Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2023 | Feb. 23, 2024 | Jun. 30, 2023 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Period End Date | Dec. 31, 2023 | ||
Current Fiscal Year End Date | --12-31 | ||
Document Transition Report | false | ||
Entity File Number | 000-23186 | ||
Entity Registrant Name | BIOCRYST PHARMACEUTICALS, INC. | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 62-1413174 | ||
Entity Address, Address Line One | 4505 Emperor Blvd., Suite 200 | ||
Entity Address, City or Town | Durham | ||
Entity Address, State or Province | NC | ||
Entity Address, Postal Zip Code | 27703 | ||
City Area Code | 919 | ||
Local Phone Number | 859-1302 | ||
Title of 12(b) Security | Common Stock, $0.01 par value | ||
Trading Symbol | BCRX | ||
Security Exchange Name | NASDAQ | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Interactive Data Current | Yes | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
ICFR Auditor Attestation Flag | true | ||
Document Financial Statement Error Correction [Flag] | false | ||
Entity Shell Company | false | ||
Entity Public Float | $ 1,322,900,797 | ||
Entity Common Stock, Shares Outstanding | 206,149,929 | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Portions of the registrant’s definitive proxy statement to be filed in connection with the solicitation of proxies for its 2024 annual meeting of stockholders are incorporated by reference into Items 10, 11, 12, 13, and 14 under Part III hereof. | ||
Entity Central Index Key | 0000882796 | ||
Document Fiscal Year Focus | 2023 | ||
Document Fiscal Period Focus | FY | ||
Amendment Flag | false |
Audit Information
Audit Information | 12 Months Ended |
Dec. 31, 2023 | |
Audit Information [Abstract] | |
Auditor Name | Ernst & Young LLP |
Auditor Location | Raleigh, NC |
Auditor Firm ID | 42 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
ASSETS | ||
Cash and cash equivalents | $ 110,643 | $ 304,767 |
Restricted cash | 1,804 | 1,472 |
Investments | 278,344 | 119,543 |
Trade receivables | 56,950 | 50,599 |
Inventory, net | 28,683 | 27,533 |
Prepaid expenses and other current assets | 19,542 | 12,586 |
Total current assets | 495,966 | 516,500 |
Property and equipment, net | 7,910 | 8,617 |
Long-term investments | 0 | 18,077 |
Other assets | 13,084 | 6,806 |
Total assets | 516,960 | 550,000 |
LIABILITIES AND STOCKHOLDERS’ DEFICIT | ||
Accounts payable | 20,893 | 14,356 |
Accrued expenses | 102,882 | 87,565 |
Deferred revenue | 0 | 1,224 |
Operating lease liabilities | 1,058 | 1,516 |
Finance lease liabilities | 1,590 | 853 |
Royalty financing obligations | 23,565 | 0 |
Total current liabilities | 149,988 | 105,514 |
Operating lease liabilities | 8,390 | 4,027 |
Finance lease liabilities | 2,845 | 1,777 |
Royalty financing obligations | 508,034 | 501,655 |
Secured term loan | 303,231 | 231,624 |
Stockholders’ deficit: | ||
Preferred stock, $0.01 par value; shares authorized — 5,000; no shares outstanding at December 31, 2023 and 2022 | 0 | 0 |
Common stock, $0.01 par value; shares authorized — 450,000; shares issued and outstanding – 205,771 and 187,906 at December 31, 2023 and 2022, respectively | 2,058 | 1,879 |
Additional paid-in capital | 1,222,236 | 1,158,118 |
Accumulated other comprehensive income | 1,337 | 26 |
Accumulated deficit | (1,681,159) | (1,454,620) |
Total stockholders’ deficit | (455,528) | (294,597) |
Total liabilities and stockholders’ deficit | $ 516,960 | $ 550,000 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parentheticals) - $ / shares | Dec. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par or stated value (in usd per share) | $ 0.01 | $ 0.01 |
Preferred stock, shares authorized (shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par or stated value (in usd per share) | $ 0.01 | $ 0.01 |
Common stock, shares authorized (in shares) | 450,000,000 | 450,000,000 |
Common stock, shares, issued (in shares) | 205,771,000 | 187,906,000 |
Common stock, shares, outstanding (in shares) | 205,771,000 | 187,906,000 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Revenues [Abstract] | |||
Revenues | $ 331,412 | $ 270,827 | $ 157,170 |
Expenses: | |||
Cost of product sales | 4,481 | 6,408 | 7,229 |
Research and development | 216,566 | 253,297 | 208,808 |
Selling, general and administrative | 213,894 | 159,371 | 118,818 |
Royalty | 180 | 186 | 35 |
Total operating expenses | 435,121 | 419,262 | 334,890 |
Loss from operations | (103,709) | (148,435) | (177,720) |
Interest and other income | 15,777 | 5,127 | 62 |
Interest expense | (108,239) | (99,092) | (59,294) |
(Loss) gain on extinguishment of debt | (29,019) | 0 | 55,838 |
Foreign currency losses, net | (1,039) | (1,983) | (695) |
Loss before income taxes | (226,229) | (244,383) | (181,809) |
Income tax expense | 310 | 2,733 | 2,253 |
Net loss | (226,539) | (247,116) | (184,062) |
Foreign currency translation adjustment | 180 | 890 | 189 |
Unrealized gain (loss) on available for sale investments | 1,131 | (1,041) | (15) |
Net comprehensive loss | $ (225,228) | $ (247,267) | $ (183,888) |
Earnings per share, basic (in usd per share) | $ (1.18) | $ (1.33) | $ (1.03) |
Earnings per share, diluted (in usd per share) | $ (1.18) | $ (1.33) | $ (1.03) |
Weighted average shares outstanding, basic (in shares) | 192,198 | 185,908 | 179,117 |
Weighted average shares outstanding, diluted (in shares) | 192,198 | 185,908 | 179,117 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Cash flows from operating activities: | |||
Net loss | $ (226,539) | $ (247,116) | $ (184,062) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 1,655 | 1,437 | 777 |
Inventory obsolescence | 422 | 932 | 0 |
Stock-based compensation expense | 55,615 | 44,701 | 34,640 |
Non-cash interest expense on royalty financing obligations and secured term loan and amortization of debt issuance costs | 85,803 | 98,918 | 54,204 |
Amortization of premium/discount on investments | (10,263) | (1,777) | (2) |
Loss (gain) on extinguishment of debt | 29,019 | 0 | (55,838) |
Loss on impairment | 1,548 | 0 | 0 |
Changes in operating assets and liabilities: | |||
Receivables | (6,095) | (21,470) | (20,817) |
Inventory | (1,450) | (12,423) | (8,767) |
Prepaid expenses and other assets | (6,820) | (2,583) | (7,155) |
Accounts payable and accrued expenses | (16,806) | (22,360) | 39,412 |
Interest payable | 0 | 0 | 4,168 |
Deferred revenue | (1,230) | (109) | 1,283 |
Net cash used in operating activities | (95,141) | (161,850) | (142,157) |
Cash flows from investing activities: | |||
Acquisitions of property and equipment | (2,168) | (1,351) | (2,385) |
Purchases of investments | (514,407) | (244,283) | (10,012) |
Sales and maturities of investments | 385,077 | 117,396 | 28,201 |
Net cash (used in) provided by investing activities | (131,498) | (128,238) | 15,804 |
Cash flows from financing activities: | |||
Sale of common stock, net | 0 | 0 | 50,000 |
Net proceeds from common stock issued under stock-based compensation plans | 8,340 | 14,765 | 15,734 |
Net proceeds from common stock issued to directors in lieu of cash retainer | 342 | 190 | 60 |
Withholding taxes paid on stock-based awards | (2,172) | 0 | 0 |
Net proceeds from secured term loans | 300,000 | 73,072 | 0 |
Repayment of Athyrium secured term loans principal | (240,452) | 0 | 0 |
Prepayment and repayment fees on Athyrium secured term loans | (21,261) | 0 | 0 |
Payment of debt issuance costs on Pharmakon Tranche A term loan | (11,147) | 0 | 0 |
Principal payments on finance lease liabilities | (1,165) | 0 | 0 |
Net proceeds from royalty financing liabilities | 0 | 0 | 293,874 |
Net cash provided by financing activities | 32,485 | 88,027 | 359,668 |
Effects of exchange rates on cash, cash equivalents and restricted cash | 362 | 566 | 71 |
(Decrease) increase in cash, cash equivalents and restricted cash | (193,792) | (201,495) | 233,386 |
Cash, cash equivalents and restricted cash at beginning of year | 306,239 | 507,734 | 274,348 |
Cash, cash equivalents and restricted cash at end of year | 112,447 | 306,239 | 507,734 |
Supplemental cash flow disclosure: | |||
Cash paid for interest | 22,139 | 0 | 900 |
Cash paid for taxes | 1,434 | 3,542 | 118 |
Taxes withheld on stock-based awards included in accrued expenses | $ 4,199 | $ 1,990 | $ 984 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Deficit - USD ($) $ in Thousands | Total | Common Stock | Common Stock Restricted Stock Units (RSUs) | Additional Paid-In Capital | Additional Paid-In Capital Restricted Stock Units (RSUs) | Accumulated Other Comprehensive Income | Accumulated Deficit |
Beginning balance at Dec. 31, 2020 | $ (19,262) | $ 1,769 | $ 1,002,408 | $ 3 | $ (1,023,442) | ||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||
Net loss | (184,062) | (184,062) | |||||
Other comprehensive income | 174 | 174 | |||||
Exercise of stock options, net | 13,745 | 33 | 13,712 | ||||
Employee stock purchase plan sales, net | 1,989 | 3 | 1,986 | ||||
Issuance of common stock, net | 45,730 | 38 | 45,692 | ||||
Issuance of shares to directors in lieu of cash retainer | 60 | 60 | |||||
Stock-based compensation expense | 34,640 | 34,640 | |||||
Ending balance at Dec. 31, 2021 | (106,986) | 1,843 | 1,098,498 | 177 | (1,207,504) | ||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||
Net loss | (247,116) | (247,116) | |||||
Other comprehensive income | (151) | (151) | |||||
Exercise of stock options, net | 11,900 | 26 | 11,874 | ||||
Employee stock purchase plan sales, net | 2,862 | 3 | 2,859 | ||||
Issuance of common stock, net | 3 | 3 | |||||
Issuance of shares to directors in lieu of cash retainer | 190 | 190 | |||||
Stock-based compensation expense | 44,701 | 44,701 | |||||
Vesting of restricted stock units | $ 4 | $ (4) | |||||
Ending balance at Dec. 31, 2022 | (294,597) | 1,879 | 1,158,118 | 26 | (1,454,620) | ||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||
Net loss | (226,539) | (226,539) | |||||
Other comprehensive income | 1,311 | 1,311 | |||||
Exercise of stock options, net | 6,114 | 13 | 6,101 | ||||
Employee stock purchase plan sales, net | 2,596 | 4 | 2,592 | ||||
Issuance of shares to directors in lieu of cash retainer | 342 | 342 | |||||
Stock-based compensation expense | 55,615 | 55,615 | |||||
Vesting of restricted stock units | $ 13 | $ (13) | |||||
Shares withheld for taxes for vesting of restricted stock units, 59 shares | (370) | (1) | (369) | ||||
Exercise of warrants, 14,997 shares | 150 | (150) | |||||
Ending balance at Dec. 31, 2023 | $ (455,528) | $ 2,058 | $ 1,222,236 | $ 1,337 | $ (1,681,159) |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Deficit Parentheticals - shares | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Exercise of stock options, net (in shares) | 1,276,000 | ||
Employee stock purchase plan sales, net (in shares) | 338,000 | 260,000 | 321,000 |
Issuance of common stock, net (in shares) | 3,846,000 | ||
Warrants exercised (in shares) | 14,997,000 | 253,000 | |
Shares withheld for taxes for vesting of restricted stock units (in shares) | 59,000 | ||
Common Stock | |||
Exercise of stock options, net (in shares) | 2,573,000 | 3,290,000 | |
Employee stock purchase plan sales, net (in shares) | 338,000 | 260,000 | 321,000 |
Issuance of shares to directors in lieu of cash retainer (in shares) | 37,000 | 32,000 | 9,000 |
Common Stock | Restricted Stock Units (RSUs) | |||
Stock option awards vested (in shares) | 1,276,000 | 439,000 |
Significant Accounting Policies
Significant Accounting Policies and Concentrations of Risk | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Significant Accounting Policies and Concentrations of Risk | Significant Accounting Policies and Concentrations of Risk The Company BioCryst Pharmaceuticals, Inc. (the “Company”) is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. The Company leverages its expertise in structure-guided drug design with the goal of developing first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat rare diseases. The Company was founded in 1986 and incorporated in Delaware in 1991, and its headquarters is located in Durham, North Carolina. The Company integrates the disciplines of biology, crystallography, medicinal chemistry and computer modeling to discover and develop small molecule and protein therapeutics through the process known as structure-guided drug design. The Company’s marketed products include oral, once-daily ORLADEYO® for the prevention of hereditary angioedema (“HAE”) attacks and RAPIVAB® (peramivir injection) for the treatment of acute uncomplicated influenza in the United States. ORLADEYO received regulatory approval in the United States in December 2020. ORLADEYO has also received regulatory approvals in multiple global markets. The Company is commercializing ORLADEYO in each of these territories directly or through distributors, except in Japan where Torii Pharmaceutical Co., Ltd. (“Torii”), the Company’s collaborative partner, conducts certain commercialization activities with respect to ORLADEYO for the prevention of HAE attacks in exchange for certain royalty payments to the Company. In addition to its approval in the United States, peramivir injection has received regulatory approvals in Canada, Australia, Japan, Taiwan and Korea. Based on the Company’s expectations for revenue and operating expenses, the Company believes its financial resources available at December 31, 2023 will be sufficient to fund its operations for at least the next 12 months. The Company has sustained operating losses for the majority of its corporate history and expects that its total 2024 expenses will exceed its total 2024 revenues. The Company expects to continue to incur operating losses and negative cash flows until revenues reach a level sufficient to support ongoing operations. The Company’s liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of its products and the progression of its product candidates in the future. The Company regularly evaluates other opportunities to fund future operations, including: (1) out-licensing rights to certain of its products or product candidates, pursuant to which the Company would receive cash milestone payments; (2) raising additional capital through equity or debt financings or from other sources, including royalty or other monetization transactions; (3) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (4) reducing spending on one or more research and development programs, including by discontinuing development; (5) restructuring operations to change its overhead structure; and/or (6) securing U.S. Government funding of its programs, including obtaining procurement contracts. The Company may, in the future, issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities and units, through private placement transactions or registered public offerings. The Company’s future liquidity needs, and ability to address those needs, will largely be determined by the success of its products and product candidates; the timing, scope and magnitude of its research and development and commercial expenses; and key developments and regulatory events and its decisions in the future. Basis of Presentation The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany transactions and balances among the consolidated entities have been eliminated from the consolidated financial statements. The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). Such consolidated financial statements reflect all adjustments that are, in management’s opinion, necessary to present fairly, in all material respects, the Company’s consolidated financial position, results of operations, and cash flows. There were no adjustments other than normal recurring adjustments. Certain prior year amounts have been reclassified to conform to the current year presentation. The Company has made certain presentation changes relative to its revenue, which management considers fundamental to understanding the Company’s current business and financial performance related to its primary product, ORLADEYO, including expanded international sales of ORLADEYO, relative to the Company’s other sources of revenue. Accordingly, certain disaggregated revenue information has been provided in this Note 1 and “Note 2—Revenue” to these consolidated financial statements. These presentation changes have been applied to prior year revenue amounts for consistency and comparability. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses, and related disclosure of contingent assets and liabilities. Significant estimates in the Company’s consolidated financial statements have been made relative to the calculation of net product sales, the ORLADEYO and Factor D inhibitors royalty financing obligations, inventory reserves, certain accruals, primarily related to the Company’s research and development expenses, the valuation of stock options and the valuation allowance for deferred tax assets resulting from net operating losses. These estimates are based on historical experience and on various other assumptions that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates. Revenue Recognition The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands): Years Ended December 31, 2023 2022 2021 Product sales, net 324,696 267,710 136,350 Collaborative and other revenues 6,716 3,117 20,820 Total revenues $ 331,412 $ 270,827 $ 157,170 Pursuant to Accounting Standards Codification (“ASC”) Topic 606, the Company recognizes revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To achieve this core principle, Topic 606 includes provisions within a five step model that includes (i) identifying the contract with a customer, (ii) identifying the performance obligations in the contract, (iii) determining the transaction price, (iv) allocating the transaction price to the performance obligations, and (v) recognizing revenue when, or as, an entity satisfies a performance obligation. At contract inception, the Company identifies the goods or services promised within each contract, assesses whether each promised good or service is distinct and determines those that are performance obligations. The Company recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when the performance obligation is satisfied. Product Sales, Net The Company’s principal sources of product sales are sales of ORLADEYO, which the Company began shipping to patients in December 2020, sales of peramivir to the Company’s licensing partners, and in prior years, sales of RAPIVAB to the U.S. Department of Health and Human Services (“HHS”) under the Company’s historical procurement contract, which was completed in 2022. In the United States, the Company ships ORLADEYO directly to patients through a single specialty pharmacy, which is considered its customer. In the European Union, United Kingdom and elsewhere, the Company sells ORLADEYO to specialty distributors as well as hospitals and pharmacies, which collectively are considered its customers. The Company recognizes revenue for sales when its customers obtain control of the product, which generally occurs upon delivery. For ORLADEYO, the Company classifies payments to its specialty pharmacy customer for certain services provided by its customer as selling, general and administrative expenses to the extent such services provided are determined to be distinct from the sale of ORLADEYO. Net revenue from sales of ORLADEYO is recorded at net selling price (transaction price), which includes estimates of variable consideration for which reserves are established for (i) estimated government rebates, such as Medicaid and Medicare Part D reimbursements, and estimated managed care rebates, (ii) estimated chargebacks, (iii) estimated costs of co-payment assistance programs and (iv) product returns. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable or as a current liability. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the applicable contract. The amount of variable consideration included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company's estimates. If actual results in the future vary from estimates, the Company adjusts these estimates, which would affect net product revenue and earnings in the period such variances become known. Government and Managed Care Rebates . The Company contracts with government agencies and managed care organizations or, collectively, third-party payors, so that ORLADEYO will be eligible for purchase by, or partial or full reimbursement from, such third-party payors. The Company estimates the rebates it will provide to third-party payors and deducts these estimated amounts from total gross product revenues at the time the revenues are recognized. These reserves are recorded in the same period in which the revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability. The Company estimates the rebates that it will provide to third-party payors based upon (i) the Company's contracts with these third-party payors, (ii) the government mandated discounts applicable to government-funded programs, (iii) a range of possible outcomes that are probability-weighted for the estimated payor mix, and (iv) product distribution information obtained from the Company's specialty pharmacy. Chargebacks . Chargebacks are discounts that occur when certain contracted customers, pharmacy benefit managers, insurance companies, and government programs purchase directly from the Company’s specialty pharmacy. These customers purchase the Company’s product under contracts negotiated between them and the Company’s specialty pharmacy. The specialty pharmacy, in turn, charges back to the Company the difference between the price the specialty pharmacy paid and the negotiated price paid by the contracted customers, which may be higher or lower than the specialty pharmacy’s purchase price from the Company. The Company estimates chargebacks and adjusts gross product revenues and accounts receivable based on the estimates at the time revenues are recognized. Co-payment assistance and patient assistance programs . Patients who have commercial insurance and meet certain eligibility requirements may receive co-payment assistance. Based upon the terms of the program and co-payment assistance utilization reports received from the specialty pharmacy, the Company is able to estimate the co-payment assistance amounts, which are recorded in the same period in which the related revenue is recognized, resulting in a reduction of product revenue. The Company also offers a patient assistance program that provides free drug product, for a limited period of time, to allow a patient’s insurance coverage to be established. Based on patient assistance program utilization reports provided by the specialty pharmacy, the Company records gross revenue of the product provided and a full reduction of the revenue amount for the free drug discount. Product returns . The Company does not provide contractual return rights to its customers, except in instances where the product is damaged or defective. Non-acceptance by the patient of shipped drug product by the specialty pharmacy is reflected as a reversal of sales in the period in which the sales were originally recorded. Reserves for estimated non-acceptances by patients are recorded as a reduction of revenue in the period that the related revenue is recognized, as well as a reduction to accounts receivable. Estimates of non-acceptance are based on quantitative information provided by the specialty pharmacy. Collaborative and Other Revenues The Company has collaboration and license agreements with a number of third parties, as well as research and development agreements with certain government entities. The Company’s primary sources of revenue from these collaborative and other research and development arrangements are license, service and royalty revenues. Revenue from license fees, royalty payments, milestone payments, and research and development fees are recognized as revenue when the earnings process is complete and the Company has no further continuing performance obligations or the Company has completed the performance obligations under the terms of the agreement. Arrangements that involve the delivery of more than one performance obligation are initially evaluated as to whether the intellectual property licenses granted by the Company represent distinct performance obligations. If they are determined to be distinct, the value of the intellectual property licenses would be recognized up front while the research and development service fees would be recognized as the performance obligations are satisfied. For performance obligations based on services performed, the Company measures progress using an input method based on the effort it expends or costs it incurs toward the satisfaction of the performance obligation in relation to the total estimated effort or costs. Variable consideration is assessed at each reporting period as to whether it is not subject to significant future reversal and, therefore, should be included in the transaction price at the inception of the contract. If a contract includes a fixed or minimum amount of research and development support, this also would be included in the transaction price. Changes to collaborations, such as the extensions of the research term or increasing the number of targets or technology covered under an existing agreement, are assessed for whether they represent a modification or should be accounted for as a new contract. For contracts with multiple performance obligations, revenue is allocated to each performance obligation based on its relative standalone selling price. Standalone selling prices are based on observable prices at which the Company separately sells the products or services. If a standalone selling price is not directly observable, then the Company estimates the standalone selling price using either an adjusted market assessment approach or an expected cost plus margin approach, representing the amount that the Company believes the market is willing to pay for the product or service. Analyzing the arrangement to identify performance obligations requires the use of judgment, and each may be an obligation to deliver services, a right or license to use an asset, or another performance obligation. Milestone payments are recognized as licensing revenue upon the achievement of specified milestones if (i) the milestone is substantive in nature and the achievement of the milestone was not probable at the inception of the agreement; and (ii) the Company has a right to payment. Any milestone payments received prior to satisfying these revenue recognition criteria are recorded as deferred revenue. Reimbursements received for direct out-of-pocket expenses related to research and development costs are recorded as revenue in the Consolidated Statements of Comprehensive Loss rather than as a reduction in expenses. Under the Company’s historical contracts with the Biomedical Advanced Research and Development Authority within the HHS (“BARDA/HHS”) and the National Institute of Allergy and Infectious Diseases (“NIAID/HHS”), revenue was recognized as reimbursable direct and indirect costs are incurred. Under certain of the Company’s license agreements, the Company receives royalty payments based upon its licensees’ net sales of covered products. Royalties are recognized at the later of when (i) the subsequent sale or usage occurs, or (ii) the performance obligation to which some or all of the sales-based or usage-based royalty has been satisfied. Cash and Cash Equivalents The Company generally considers cash equivalents to be all cash held in commercial checking accounts, money market accounts, or investments in debt instruments and certificates of deposit with maturities of three months or less at the time of purchase. The carrying value of cash and cash equivalents approximates fair value due to the short-term nature of these items. Restricted Cash Total restricted cash was $1,804 and $1,472 as of December 31, 2023 and 2022, respectively, and primarily consisted of $1,493 and $1,449 as of December 31, 2023 and 2022, respectively, for a letter of credit the Company is required to maintain associated with its Birmingham lease. Investments The Company invests in high credit quality investments in accordance with its investment policy, which is designed to minimize the possibility of loss. The objective of the Company’s investment policy is to ensure the safety and preservation of invested funds, as well as maintaining liquidity sufficient to meet cash flow requirements. The Company places its excess cash with high credit quality financial institutions, commercial companies, and government agencies in order to limit the amount of its credit exposure. In accordance with its policy, the Company is able to invest in marketable debt securities that may consist of U.S. Government and government agency securities, money market and mutual fund investments, certificates of deposits, municipal and corporate notes and bonds, and commercial paper, among others. The Company’s investment policy requires it to purchase high-quality marketable securities with a maximum individual maturity of three years and requires an average portfolio maturity of no more than 12 months. Some of the securities in which the Company invests may have market risk. This means that a change in prevailing interest rates may cause the principal amount of the investment to fluctuate. To minimize this risk, the Company schedules its investments with maturities that coincide with expected cash flow needs, thus avoiding the need to redeem an investment prior to its maturity date. Accordingly, the Company does not believe it has a material exposure to interest rate risk arising from its investments. Generally, the Company’s investments are not collateralized. The Company has not realized any significant losses from its investments. The Company classifies all of its investments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive income, unless an unrealized loss is considered to be other than temporary, in which case the unrealized loss is charged to operations. The Company reviews its investments for other than temporary declines in fair value below cost basis at the end of each reporting period and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Factors considered include whether a decline in fair value below the amortized cost basis is due to credit-related factors or non-credit-related factors, the financial condition and near-term prospects of the Company, and the Company's intent and ability to hold the investment to allow for an anticipated recovery in fair value. A credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings. Any impairment that is not credit-related is recognized in other comprehensive income, net of applicable taxes unless deemed other than temporary. Realized gains and losses are reflected in interest and other income in the Consolidated Statements of Comprehensive Loss and are determined using the specific identification method with transactions recorded on a settlement date basis. Investments with original maturities at date of purchase beyond three months and which mature at or less than 12 months from the balance sheet date are classified as current. Investments with a maturity beyond 12 months from the balance sheet date are classified as long-term. Fair Value Measurements Assets and liabilities recorded at fair value on a recurring basis on the Consolidated Balance Sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Items measured at fair value on a recurring basis include investments (Note 3). The carrying amounts reflected in the Consolidated Balance Sheets for cash and cash equivalents, trade receivables, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities. Trade Receivables The majority of the Company’s trade receivables arise from product sales and primarily represent amounts due from its specialty pharmacy customer in the United States and other third-party distributors, hospitals and pharmacies in the European Union, United Kingdom and elsewhere and have standard payment terms that generally require payment within 30 to 90 days. Receivables from collaborations are recorded for amounts due to the Company related to reimbursable research and development costs from the HHS (Note 15), and royalty receivables from the Company’s partners, including Shionogi, Green Cross, and Torii (Note 15). Monthly invoices were submitted to the HHS related to reimbursable research and development costs. The Company was also entitled to monthly reimbursement of indirect costs based on rates stipulated in the underlying contract. The Company’s calculations of its indirect cost rates are subject to audit by the U.S. Government. The Company does not adjust its receivables for the effects of a significant financing component at contract inception if it expects to collect the receivables in one year or less from the time of sale. The Company provides reserves against trade receivables for estimated losses that may result from a customer's inability to pay. Receivables are evaluated to determine if any reserve or allowance should be recorded based on consideration of the current economic environment, expectations of future economic conditions, specific circumstances and the Company’s own historical collection experience. Amounts determined to be uncollectible are charged or written-off against the reserve. Inventory The Company’s inventories primarily relate to ORLADEYO. Additionally, the Company’s inventories include RAPIVAB and peramivir. The Company values its inventories at the lower of cost or estimated net realizable value. The Company determines the cost of its inventories, which includes amounts related to materials, labor, manufacturing overhead, and shipping and handling costs on a first-in, first-out (FIFO) basis. Raw materials and work-in-process include all inventory costs prior to packaging and labelling, including raw material, active product ingredient, and the drug product. Finished goods include packaged and labelled products. The Company’s inventories are subject to expiration dating. The Company regularly evaluates the carrying value of its inventories and provides valuation reserves for any estimated obsolete, short-dated or unmarketable inventories. In addition, the Company may experience spoilage of its raw materials and supplies. The Company’s determination that a valuation reserve might be required, in addition to the quantification of such reserve, requires it to utilize significant judgment. The Company expenses costs related to the production of inventories as research and development expenses in the period incurred until such time it is believed that future economic benefit is expected to be recognized, which generally is reliant upon receipt of regulatory approval. Upon regulatory approval, the Company capitalizes subsequent costs related to the production of inventories. Property and Equipment Property and equipment are recorded at cost. Depreciation is computed using the straight-line method over the estimated useful lives of the assets. Computer equipment is depreciated over a life of three years. Laboratory equipment, office equipment, and software are depreciated over a life of five years. Furniture and fixtures are depreciated over a life of seven years. Leasehold improvements are amortized over their estimated useful lives or the expected lease term, whichever is less. The Company periodically reviews its property and equipment for impairment when events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Determination of recoverability is based on an estimate of undiscounted future cash flows resulting from the use of the asset and its eventual disposition. In the event that such cash flows are not expected to be sufficient to recover the carrying amount of the assets, the assets are written down to their estimated fair values. Property and equipment to be disposed of are reported at the lower of carrying amount or fair value less cost to sell. Accrued Expenses The Company enters into contractual agreements with third-party vendors who provide research and development, manufacturing, distribution, and other services in the ordinary course of business. Some of these contracts are subject to milestone-based invoicing, and services are completed over an extended period of time. The Company records liabilities under these contractual commitments when it determines an obligation has been incurred, regardless of the timing of the invoice. This process involves reviewing open contracts and purchase orders, communicating with applicable Company personnel to identify services that have been performed on its behalf and estimating the level of service performed and the associated cost incurred for the service when the Company has not yet been invoiced or otherwise notified of actual cost. The majority of service providers invoice the Company monthly in arrears for services performed. The Company makes estimates of accrued expenses as of each balance sheet date in its financial statements based on the facts and circumstances which can include assumptions such as expected patient enrollment, site activation and estimated project duration. The Company periodically confirms the accuracy of its estimates with the service providers and makes adjustments if necessary. Examples of estimated accrued expenses include (i) fees paid to clinical research organizations (“CROs”) in connection with preclinical and toxicology studies and clinical trials; (ii) fees paid to investigative sites in connection with clinical trials; (iii) fees paid to contract manufacturers in connection with the production of the Company’s raw materials, drug substance, drug products, and product candidates; and (iv) professional fees. The Company bases its expenses related to clinical trials on its estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical trials on the Company’s behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. If the Company underestimates or overestimates the level of these costs, actual expenses could differ from such estimates. As of December 31, 2023 and December 31, 2022, the carrying value of accrued expenses approximates their fair value due to their short-term settlement. Cost of Product Sales Cost of product sales includes the cost of producing and distributing inventories that are related to product revenue during the respective period, including freight. In addition, shipping and handling costs for product shipments are recorded as incurred. Finally, cost of product sales may also include costs related to excess or obsolete inventory adjustment charges. Research and Development Expenses The Company’s research and development costs are expensed when incurred. Research and development expenses include all direct and indirect development costs related to the development of the Company’s portfolio of product candidates. Advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as expense when the related goods are delivered or the related services are performed. Research and development expenses include, among other items, personnel costs, including salaries and benefits, manufacturing costs, clinical, regulatory, and toxicology services performed by CROs, materials and supplies, and overhead allocations consisting of various administrative and facilities related costs, as well as termination fees and other commitments associated with discontinued programs. Most of the Company’s manufacturing and clinical and preclinical studies are performed by third-party CROs. Costs for studies performed by CROs are accrued by the Company over the service periods specified in the contracts, and estimates are adjusted based upon the Company’s ongoing review of the level of services actually performed. Additionally, the Company has license agreements with third parties, such as Albert Einstein College of Medicine of Yeshiva University, Industrial Research, Ltd., and the University of Alabama at Birmingham (“UAB”), which require fees related to sublicense agreements. The Company expenses sublicense payments as incurred. The Company groups its research and development expenses into two major categories: direct expenses and indirect expenses. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidate, conduct and manage clinical trials, as well as other costs related to the Company’s clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. These costs are accumulated and tracked by active program. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of the Company’s research and development efforts. These costs ap |
Revenue
Revenue | 12 Months Ended |
Dec. 31, 2023 | |
Revenue Recognition [Abstract] | |
Revenue | Revenue The Company recorded the following revenues for the years ended December 31 (in thousands): Years Ended December 31, 2023 2022 2021 ORLADEYO: U.S. $ 288,361 $ 226,358 $ 117,836 Outside of U.S. 37,629 25,275 4,719 Total ORLADEYO 325,990 251,633 122,555 Other revenues 5,422 19,194 34,615 Total revenues $ 331,412 $ 270,827 $ 157,170 ORLADEYO revenues represent total revenues from product sales, collaborative revenues and royalties. Royalty revenue from sales of ORLADEYO in Japan by the Company’s collaborative partner, Torii, were $2,178, $1,944, and $690 for the years ended December 31, 2023, 2022, and 2021 respectively. Other revenues primarily relate to the Company’s product sales and royalties for peramivir injection (RAPIVAB/RAPIACTA/PERAMIFLU), and for the years ended December 31, 2022 and 2021, galidesivir development contracts with BARDA/HHS and NIAID/HHS. Other revenues for the year ended December 31, 2021 also includes milestone revenue of $15,000 upon receipt from the Japanese National Health Insurance System (“NHI”) of a reimbursement price approval for ORLADEYO. |
Investments
Investments | 12 Months Ended |
Dec. 31, 2023 | |
Investments, All Other Investments [Abstract] | |
Investments | Investments Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. Fair value is determined based on observable and unobservable inputs. Observable inputs reflect readily obtainable data from independent sources, while unobservable inputs reflect certain market assumptions. As a basis for considering such assumptions, U.S. GAAP establishes a three-tier value hierarchy, which prioritizes the inputs used to develop the assumptions and for measuring fair value as follows: (Level 1) observable inputs such as quoted prices in active markets for identical assets; (Level 2) inputs other than the quoted prices in active markets that are observable either directly or indirectly; and (Level 3) unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. This hierarchy requires the Company to use observable market data, when available, and to minimize the use of unobservable inputs when determining fair value. The Company’s financial instruments that are measured at fair value on a recurring basis consist of fixed income investments. These valuations are based on observable direct and indirect inputs, primarily quoted prices of similar, but not identical, instruments in active markets or quoted prices for identical or similar instruments in markets that are not active. These fair values are obtained from independent pricing services. Assets measured at fair value on a recurring basis were as follows (in thousands): December 31, 2023 Quoted Price in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Total Assets: Obligations of U.S. Government and its agencies $ — $ 277,358 $ — $ 277,358 Certificates of deposit — 986 — 986 Total assets $ — $ 278,344 $ — $ 278,344 December 31, 2022 Quoted Price in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Total Assets: Obligations of U.S. Government and its agencies $ — $ 129,371 $ — $ 129,371 Corporate debt securities — 6,092 — 6,092 Certificates of deposit — 2,157 — 2,157 Total assets $ — $ 137,620 $ — $ 137,620 There were no changes in valuation techniques during the years ended December 31, 2023 and 2022. There were no liabilities measured at fair value on a recurring basis as of December 31, 2023 and 2022. As of December 31, 2023, the Company had 11 securities with a total estimated fair market value of $18,513 in an unrealized loss position. The Company believes the individual unrealized losses represent temporary declines primarily resulting from interest rate changes. The Company does not have intent to sell these investments, and it is more likely than not that the investments will be held until recovery of their amortized cost basis. As such, no allowance was recognized. The following tables summarize the fair value of the Company’s investments by type (in thousands): December 31, 2023 Amortized Cost Accrued Interest Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Obligations of U.S. Government and its agencies $ 277,151 $ 121 $ 150 $ (64) $ 277,358 Certificates of deposit 980 14 — (8) 986 Total investments $ 278,131 $ 135 $ 150 $ (72) $ 278,344 December 31, 2022 Amortized Cost Accrued Interest Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Obligations of U.S. Government and its agencies $ 129,940 $ 427 $ — $ (996) $ 129,371 Corporate debt securities 6,093 37 — (38) 6,092 Certificates of deposit 2,163 23 — (29) 2,157 Total investments $ 138,196 $ 487 $ — $ (1,063) $ 137,620 The following table summarizes the scheduled maturity for the Company’s investments at December 31, 2023 and 2022 (in thousands): December 31, 2023 2022 Maturing in one year or less $ 278,344 $ 119,543 Maturing after one year through two years — 18,077 Total investments $ 278,344 $ 137,620 |
Trade Receivables
Trade Receivables | 12 Months Ended |
Dec. 31, 2023 | |
Receivables [Abstract] | |
Trade Receivables | Trade Receivables Product Sales Receivables from product sales are recorded for amounts due to the Company related to sales of ORLADEYO and RAPIVAB. At December 31, 2023 and 2022, receivables, net of reserves, related to sales of ORLADEYO were $54,149 and $41,508, respectively. A returns reserve related to sales of ORLADEYO of $288 was recorded as of December 31, 2023. No reserve or allowance amounts were recorded as of December 31, 2022 related to sales of ORLADEYO. At December 31, 2023 and 2022, receivables related to sales of RAPIVAB were $505 and $823, respectively. No reserve or allowance amounts were recorded as of December 31, 2023 and 2022, related to sales of RAPIVAB. Collaborations Receivables from collaborations were as follows (in thousands): December 31, 2023 Billed Unbilled Total Royalty receivables from partners $ — $ 2,296 $ 2,296 Total receivables from collaborators $ — $ 2,296 $ 2,296 December 31, 2022 Billed Unbilled Total U.S. Department of Health and Human Services, net $ 7,218 $ 284 $ 7,502 Royalty receivables from partners 741 — 741 Other collaborations — 25 25 Total receivables from collaborators $ 7,959 $ 309 $ 8,268 As of December 31, 2023 and 2022, the reserve related to royalties from collaborations was not material. |
Inventory
Inventory | 12 Months Ended |
Dec. 31, 2023 | |
Inventory Disclosure [Abstract] | |
Inventory | Inventory At December 31, 2023 and 2022, the Company’s inventory primarily related to ORLADEYO. Additionally, inventory included RAPIVAB and peramivir, which is manufactured for the Company’s partners. The Company’s inventories consisted of the following (in thousands): December 31, 2023 2022 Raw materials $ 6,449 $ 8,906 Work-in-process 17,591 14,990 Finished goods 6,242 4,814 Total inventory $ 30,282 $ 28,710 Reserves (1,599) (1,177) Total inventory, net $ 28,683 $ 27,533 |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment | Property and Equipment Property and equipment consisted of the following (in thousands): December 31, 2023 2022 Furniture and fixtures $ 1,384 $ 1,308 Office equipment 719 633 Software 1,252 1,501 Laboratory equipment 5,559 4,588 Leasehold improvements 10,206 10,137 Total property and equipment $ 19,120 $ 18,167 Less accumulated depreciation and amortization (11,210) (9,550) Property and equipment, net $ 7,910 $ 8,617 Depreciation expense for the years ended December 31, 2023, 2022, and 2021 was $1,655, $1,437, and $777, respectively. The Company recorded an impairment loss of $1,548 and contract termination fees of $440 related to the discontinuation of the Birmingham research facilities expansion, which has been recognized in research and development expenses during the year ended December 31, 2023. The Company did not record any impairment losses during the years ended December 31, 2022 and 2021. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2023 | |
Accounts Payable and Accrued Liabilities [Abstract] | |
Accrued Expenses | Accrued Expenses Accrued expenses consisted of the following (in thousands): December 31, 2023 2022 Compensation and benefits $ 36,518 $ 22,125 Revenue-related reserves for discounts and allowances 26,509 14,332 Royalties payable 18,524 7,700 Development costs 13,677 30,360 Inventory 190 4,193 Other 7,464 8,855 Total accrued expenses $ 102,882 $ 87,565 |
Royalty Financing Obligations
Royalty Financing Obligations | 12 Months Ended |
Dec. 31, 2023 | |
Advance Royalties [Abstract] | |
Royalty Financing Obligations | Royalty Financing Obligations ORLADEYO and Factor D Inhibitors On December 7, 2020, the Company and RPI 2019 Intermediate Finance Trust (“RPI”) entered into a Purchase and Sale Agreement (the “2020 RPI Royalty Purchase Agreement”), pursuant to which the Company sold to RPI the right to receive certain royalty payments from the Company for a purchase price of $125,000 in cash (the “2020 RPI Royalty Sale”). Under the 2020 RPI Royalty Purchase Agreement, RPI is entitled to receive tiered, sales-based royalties on net product sales of ORLADEYO in the United States and certain key European markets (collectively, the “Key Territories”), and other markets where the Company sells ORLADEYO directly or through distributors (collectively, the “Direct Sales”) in an amount equal to: (i) 8.75% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 2.75% of annual net sales for annual net sales between $350,000 and $550,000. No royalty payments are payable on annual Direct Sales over $550,000. Under the 2020 RPI Royalty Purchase Agreement, RPI is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees outside of the Key Territories (the “Other Markets”) equal to: (i) 20% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets; (ii) 20% of proceeds received on annual net sales of up to $150,000 in the Other Markets; and (iii) 10% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets. On November 19, 2021, the Company and RPI entered into (i) a Purchase and Sale Agreement (the “2021 RPI Royalty Purchase Agreement” and together with the 2020 RPI Royalty Purchase Agreement, the “RPI Royalty Purchase Agreements”), pursuant to which the Company sold to RPI the right to receive certain royalty payments from the Company for a purchase price of $150,000 in cash, and (ii) a Purchase and Sale Agreement with OCM IP Healthcare Holdings Limited, an affiliate of OMERS Capital Markets (“OMERS”) (the “OMERS Royalty Purchase Agreement” and collectively with the RPI Royalty Purchase Agreements, the “Royalty Purchase Agreements”), pursuant to which the Company sold to OMERS the right to receive certain royalty payments from the Company for a purchase price of an additional $150,000 in cash. Under the 2021 RPI Royalty Purchase Agreement, RPI is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 0.75% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 1.75% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000. No royalty payments are payable on Direct Sales over $550,000. RPI is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees in the Other Markets in an amount equal to (i) 3.0% of proceeds received by the Company on annual net sales of up to $150,000 in the Other Markets and (ii) 2.0% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets. Under the 2021 RPI Royalty Purchase Agreement, RPI is also entitled to receive tiered, sales-based royalties on net product sales of BCX10013 in an amount equal to: (i) 3.0% of worldwide aggregate annual net sales up to $1,500,000 and (ii) 2.0% of worldwide aggregate annual net sales between $1,500,000 and $3,000,000. No royalty payments are payable on annual net sales above $3,000,000. RPI is also entitled to receive tiered profit share amounts of up to 3.0% from certain other permitted sales in certain other markets. The royalties payable under the 2021 RPI Royalty Purchase Agreement are in addition to the royalties payable to RPI under the 2020 RPI Royalty Purchase Agreement. Under the OMERS Royalty Purchase Agreement, commencing with the calendar quarter beginning October 1, 2023, OMERS is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 7.5% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 6.0% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000 (with no royalty payments payable on annual Direct Sales over $550,000). For each calendar quarter beginning on or after January 1, 2024, OMERS is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 10.0% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 3.0% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000 (with no royalty payments payable on annual Direct Sales over $550,000). Under the OMERS Royalty Purchase Agreement, OMERS is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees in the Other Markets in an amount equal to: (i) 20.0% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets, (ii) 20.0% of proceeds received by the Company on annual net sales of up to $150,000 in the Other Markets, and (iii) 10.0% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets. OMERS is also entitled to receive profit share amounts of up to 10% from certain other permitted sales in certain other markets. Under the 2020 RPI Royalty Purchase Agreement, the Company is required to make royalty payments of amounts owed to RPI each calendar quarter following the first commercial sale of ORLADEYO in any country. Under the 2021 RPI Royalty Purchase Agreement, the Company is required to make payments to RPI in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2021. Under the OMERS Royalty Purchase Agreement, the Company is required to make payments to OMERS in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2023. OMERS will no longer be entitled to receive any payments on the date in which aggregate payments actually received by OMERS equals 155.0% of the $150,000 purchase price. The transactions contemplated by each of the Royalty Purchase Agreements are referred to herein as the “Royalty Sales.” Under the Royalty Purchase Agreements, the Company has agreed to specified affirmative and negative covenants, including covenants regarding periodic reporting of information by the Company to RPI and OMERS, third-party audits of royalties paid under the Royalty Purchase Agreements, and restrictions on the ability of the Company or any of its subsidiaries to incur indebtedness other than certain royalty sales and as was permitted to be incurred under the terms of the Athyrium Credit Agreement (as defined in Note 9 herein) through its payoff and termination on April 17, 2023 or, subsequent to that date and the Pharmakon Loan Agreement (as defined in Note 9 herein), as applicable. See “Note 9—Debt” for further details on the Athyrium Credit Agreement and the Pharmakon Loan Agreement. The restrictions under the Royalty Purchase Agreements on the ability of the Company or any of its subsidiaries to incur indebtedness are eliminated after the achievement of certain specified milestones in the Royalty Purchase Agreements. The cash consideration obtained pursuant to the Royalty Purchase Agreements is recorded in “Royalty financing obligations” on the Company’s Consolidated Balance Sheets. The fair value for the royalty financing obligations at the time of the transactions was based on the Company’s estimates of future royalties expected to be paid to the counterparty over the life of the arrangement. The Company subsequently records the obligations at their carrying value using the effective interest method. In order to amortize the royalty financing obligations, the Company utilizes the prospective method to estimate the future royalties to be paid by the Company to the counterparty over the life of the arrangement. Under the prospective method, a new effective interest rate is determined based on the revised estimate of remaining cash flows. The new rate is the discount rate that equates the present value of the revised estimate of remaining cash flows with the carrying amount of the debt, and it will be used to recognize interest expense for the remaining periods. The Company periodically assesses the amount and timing of expected royalty payments using a combination of internal projections and forecasts from external sources. The estimates of future net product sales (and resulting royalty payments) are based on key assumptions including population, penetration, probability of success, and sales price, among others. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the amortization of the royalty financing obligations and the effective interest rate. On a quarterly basis, the Company assesses the projected royalty payments relative to the projected interest accretion for the next twelve months to determine if the royalty liability balance is reduced relative to the current outstanding liability, which would signify a repayment of the liability. In such case of excess payments relative to interest accretion for the next twelve months, the excess payments are considered to be a short-term liability and classified within current liabilities on the Company’s Consolidated Balance Sheets. During the year ended December 31, 2023, the Company adjusted its forecasts related to its BCX10013 program and updated its ORLADEYO forecast based on actual results for the year ended December 31, 2023. The primary factors that impacted forecasts on the BCX10013 development program were development delays, reduced probability of success, reduced pricing assumptions and reduced market share assumptions. These adjustments impacted the amount and timing of expected royalties to be made under the Royalty Purchase Agreements. As a result, the effective interest rate related to the 2020 RPI Royalty Purchase Agreement decreased from 22.4% to 22.3%, the effective interest rate related to the 2021 RPI Royalty Purchase Agreement decreased from 13.1% to 0.0%, and the effective interest rate related to the OMERS Royalty Purchase Agreement decreased from 10.6% to 10.0%. The following table shows the activity within the Royalty financing obligations account (in thousands) as well as the effective interest rate as of December 31, 2023: 2020 RPI Royalty Agreement 2021 RPI Royalty Agreement OMERS Royalty Agreement Total Balance as of December 31, 2020 $ 124,717 $ — $ — $ 124,717 2021 Royalty sale: Royalty financing obligations, net of issuance costs — 150,833 147,309 298,142 Non-cash Interest expense on Royalty financing obligations 33,308 2,897 1,465 37,670 Royalty revenues paid and payable (10,801) (353) — (11,154) Balance as of December 31, 2021 $ 147,224 $ 153,377 $ 148,774 $ 449,375 Deferred financing costs — (34) — (34) Non-cash Interest expense on Royalty financing obligations 39,994 22,239 14,249 76,482 Royalty revenues paid and payable (22,237) (1,931) — (24,168) Balance as of December 31, 2022 $ 164,981 $ 173,651 $ 163,023 $ 501,655 Non-cash Interest expense on Royalty financing obligations 38,267 14,188 17,901 70,356 Royalty revenues paid and payable (28,768) (2,494) (9,150) (40,412) Balance as of December 31, 2023 $ 174,480 $ 185,345 $ 171,774 $ 531,599 Effective interest rate 22.3 % — % 10.0 % Deferred issuance costs pursuant to the Royalty financing obligations, which consist primarily of advisory and legal fees, totaled $8,532 as of December 31, 2023 and 2022. The Royalty financing obligations liabilities and the associated deferred issuance costs are amortized using the effective interest method over the term of the arrangement. Concurrent with entering into the 2021 RPI Royalty Purchase Agreement, the Company and RPI entered into a Common Stock Purchase Agreement (the “Common Stock Purchase Agreement”), pursuant to which the Company sold common stock to RPI for a premium of $4,269. This premium has been deferred and is being amortized through interest expense using the effective interest method over the term of the applicable arrangement. See “Note 11—Stockholders’ Equity” for further details on the common stock sale premium. RAPIACTA Overview On March 9, 2011, the Company completed a $30,000 financing transaction to monetize certain future royalty and milestone payments under the Company’s agreement (the “Shionogi Agreement”) with Shionogi & Co., Ltd. (“Shionogi”), pursuant to which Shionogi licensed from the Company the rights to market RAPIACTA in Japan and Taiwan. The Company received net proceeds of $22,691 from the transaction. As part of the transaction, the Company entered into a purchase and sale agreement dated as of March 9, 2011 with JPR Royalty Sub LLC, a wholly-owned subsidiary of the Company (“Royalty Sub”), whereby the Company transferred to Royalty Sub, among other things, its rights to receive certain royalty and milestone payments from Shionogi arising under the Shionogi Agreement. Royalty payments are paid by Shionogi in Japanese Yen, and any milestone payments will be paid in U.S. dollars. The Company’s collaboration with Shionogi was not impacted as a result of this transaction. Non-Recourse Notes Payable On March 9, 2011, Royalty Sub completed a private placement to institutional investors of $30,000 in aggregate principal amount of its PhaRMA Senior Secured 14% Notes due on December 1, 2020 (the “PhaRMA Notes”). The PhaRMA Notes were issued by Royalty Sub under an Indenture, dated as of March 9, 2011 (the “Indenture”), by and between Royalty Sub and U.S. Bank National Association, as Trustee. Principal and interest on the PhaRMA Notes are payable from, and are secured by, the rights to royalty and milestone payments under the Shionogi Agreement transferred by the Company to Royalty Sub. The PhaRMA Notes bear interest at 14% per annum, payable annually in arrears on September 1st of each year. The Company remains entitled to receive any royalties and milestone payments related to sales of peramivir by Shionogi following repayment of the PhaRMA Notes. Royalty Sub’s obligations to pay principal and interest on the PhaRMA Notes are obligations solely of Royalty Sub and are without recourse to any other person, including the Company, except to the extent of the Company’s pledge of its equity interests in Royalty Sub in support of the PhaRMA Notes. In September 2014, Royalty Sub was unable to pay the accrued interest obligation due September 3, 2013. Under the terms of the Indenture, Royalty Sub’s inability to pay the full amount of interest payable in September 2013 by the next succeeding payment date for the PhaRMA Notes, which was September 1, 2014, constituted an event of default. Due to the non-recourse nature of the PhaRMA Notes, in the event of any potential foreclosure, the primary impact to the Company would be the loss of future royalty payments, if any, from Shionogi and legal costs associated with retiring the PhaRMA Notes. The PhaRMA Notes had a final legal maturity date of December 1, 2020, at which time the outstanding principal amount of the PhaRMA Notes of $30,000, together with accrued and unpaid interest of $20,614, was due in full. Non-Recourse Notes Payable – Debt Extinguishment |
Debt
Debt | 12 Months Ended |
Dec. 31, 2023 | |
Debt Disclosure [Abstract] | |
Debt | Debt Pharmakon Loan Agreement On April 17, 2023, the Company entered into a $450,000 Loan Agreement (the “Pharmakon Loan Agreement”) with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, as lenders, and BioPharma Credit PLC, as collateral agent for the lenders. Certain of the Company’s wholly-owned subsidiaries are guarantors to the Pharmakon Loan Agreement. The Pharmakon Loan Agreement provides for an initial term loan in the principal amount of $300,000 (the “Tranche A Loan”) funded on April 17, 2023 (the “Tranche A Closing Date”). The Company used a portion of the proceeds from the Tranche A Loan to repay the $241,787 of outstanding indebtedness (principal and interest due as of April 17, 2023) under the then-existing Athyrium Credit Agreement (defined below) and to pay associated transaction costs and fees, and used the remaining net proceeds of $25,805 for other general corporate purposes. The Pharmakon Loan Agreement also provides for three additional term loan tranches, at the Company’s option, in principal amounts of $50,000 each (each a “Subsequent Tranche Loan” and, collectively with the Tranche A Loan, the “Pharmakon Term Loans” and each, a “Pharmakon Term Loan”), which may be requested on or prior to September 30, 2024. The maturity date of the Pharmakon Loan Agreement is April 17, 2028 (the “Maturity Date”), the fifth anniversary of the Tranche A Closing Date. The Pharmakon Loan Agreement provides for quarterly interest-only payments until the Maturity Date, with the unpaid principal amount of the outstanding Pharmakon Term Loans due and payable on the Maturity Date. During the first 18 months following the Tranche A Closing Date, the Company has the option to make a portion of the applicable interest payment on the Tranche A Loan in-kind (a “Pharmakon PIK Interest Payment”) by capitalizing as principal up to 50% of the amount of interest accrued on the Tranche A Loan during the applicable interest period. The Pharmakon Term Loans will bear interest at a rate equal to the three-month Secured Overnight Financing Rate (“SOFR”) rate, which shall be no less than 1.75%, plus 7.00%, per annum or, for each interest period in which a Pharmakon PIK Interest Payment is made, with respect to the Tranche A Loan, SOFR plus 7.25%, per annum. The Tranche A Loan accrued interest at an effective interest rate of 13.30% for the year ended December 31, 2023. The Company is required to make a mandatory prepayment of the Pharmakon Term Loans (i) upon the occurrence of a change of control and (ii) prior to any repayment of any convertible debt that the Company may issue in the future, subject to certain exceptions. The Company may make voluntary prepayments in whole or in part, in minimum $25,000 increments. Prepayments are subject to a prepayment premium equal to, (i) with respect to any prepayment made prior to the second anniversary of the applicable Pharmakon Term Loan borrowing date, the sum of (1) 3.00% of the principal amount of the Pharmakon Term Loan being prepaid plus (2) the aggregate amount of all interest that would have accrued on the principal amount of the Pharmakon Term Loan being prepaid from the date of prepayment through and including the second anniversary of the date of the borrowing of such Pharmakon Term Loan; (ii) with respect to any prepayment made on or after the second anniversary and prior to the third anniversary of the applicable Pharmakon Term Loan borrowing date, 3.00% of the principal amount of the Pharmakon Term Loan being prepaid; (iii) with respect to any prepayment made on or after the third anniversary and prior to the fourth anniversary of the applicable Pharmakon Term Loan, 2.00% of the principal amount of the Pharmakon Term Loan being prepaid; and (iv) with respect to any prepayment made on or after the fourth anniversary of the applicable Pharmakon Term Loan borrowing date and before the Maturity Date, 1.00% of the principal amount of the Pharmakon Term Loan being prepaid. In addition, upon the drawing of any Subsequent Tranche Loan, certain funding fees are required to be paid. The Pharmakon Loan Agreement also contains representations and warranties and affirmative and negative covenants customary for financings of this type, as well as customary events of default. Certain of the customary negative covenants limit the ability of the Company and certain of its subsidiaries to, among other things, dispose of assets, engage in mergers, acquisitions, and similar transactions, incur additional indebtedness, grant liens, make investments, pay dividends or make distributions or certain other restricted payments in respect of equity, prepay other indebtedness, enter into restrictive agreements, undertake fundamental changes or amend certain material contracts, among other customary covenants, in each case subject to certain exceptions. A failure to comply with the covenants in the Pharmakon Loan Agreement, or an occurrence of any other event of default, could permit the lenders under the Pharmakon Loan Agreement to declare the borrowings thereunder, together with accrued interest and fees, and any applicable prepayment premium, to be immediately due and payable. The Company’s obligations under the Pharmakon Loan Agreement are secured by a security interest in, subject to certain exceptions, substantially all of the Company’s assets. As of December 31, 2023, the Company had total borrowings of $300,000 under the Pharmakon Loan Agreement. Interest expense on the Tranche A Loan for the year ended December 31, 2023 totaled $27,326. As allowable under the Pharmakon Loan Agreement, the Company has designated and accounted for 50% of the quarterly interest payments for the year ended December 31, 2023 as a Pharmakon PIK Interest Payment and the amount of $13,663 has been added to the outstanding principal balance of the borrowing. The remaining 50% of the quarterly interest payments of $13,663 have been paid at the end of each quarterly period. As of December 31, 2023, borrowings, including the Pharmakon PIK Interest Payments, totaled $313,663. The fair value of the debt approximates its carrying value based on prevailing interest rates as of the balance sheet date and is considered as Level 2 in the fair value hierarchy. Incurred debt fees and issuance costs associated with the Tranche A Loan under the Pharmakon Loan Agreement totaled $11,147 and have been deferred and are being amortized as interest expense on an effective interest rate method over the remaining term of the Tranche A Loan. Deferred financing amortization of $715 was recognized for the year ended December 31, 2023. Athyrium Credit Agreement On December 7, 2020, the Company entered into a $200,000 Credit Agreement (the “Athyrium Credit Agreement”) with Athyrium Opportunities III Co-Invest 1 LP (“Athyrium”), as lender and as administrative agent for the lenders. Certain of the Company’s direct and indirect subsidiaries were guarantors to the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for an initial term loan in the principal amount of $125,000 (the “Term A Loan”), which was received by the Company on December 7, 2020 and is recorded in “Secured term loan” on the Company’s balance sheet. The Company used a portion of the proceeds from the Term A Loan to repay $43,298 of outstanding indebtedness, including accrued interest, under its prior credit facility with MidCap Financial Trust. The Athyrium Credit Agreement also provided for two additional term loans, at the Company’s option, in the respective principal amounts of $25,000 (the “Term B Loan”) and $50,000 (the “Term C Loan” and, collectively with the Term A Loan and the Term B Loan, the “Athyrium Term Loans”). Having achieved all required revenue-based milestones, the Company exercised its option to draw upon the additional funding available under the Athyrium Credit Agreement, borrowing the principal amounts of $25,000 under the Term B Loan and $50,000 under the Term C Loan. Both the Term B Loan and the Term C Loan were funded on July 29, 2022 in the aggregate principal amount of $75,000. The Company incurred deferred debt fees and issuance costs associated with the Term B and Term C Loans of $3,428. The Term B Loan and the Term C Loan were subject to all the provisions under the Athyrium Credit Agreement. On November 19, 2021, the Company entered into an amendment to the Athyrium Credit Agreement to, among other things, (i) permit the Company to enter into the 2021 RPI Royalty Purchase Agreement, the OMERS Royalty Purchase Agreement, and the other definitive documentation related thereto and to perform its obligations thereunder; and (ii) require the Company to pay to Athyrium, for the account of the lenders, a make-whole premium plus certain fees set forth in the Athyrium Credit Agreement in the event that the Company prepaid or repaid, or was required to prepay or repay, voluntarily or pursuant to mandatory prepayment obligations under the Athyrium Credit Agreement (e.g., with the proceeds of certain asset sales, certain ORLADEYO out-licensing or royalty financing transactions (excluding the Royalty Sales), extraordinary receipts, debt issuances, or upon a change of control of the Company and specified other events, subject to certain exceptions), all of the then-outstanding Athyrium Term Loans, in each case, subject to certain exceptions set forth in the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for quarterly interest-only payments until the maturity date, with the unpaid principal amount of the outstanding Athyrium Term Loans due and payable on the maturity date. For each of the first eight full fiscal quarters following December 7, 2020, the Company had the option to make the applicable interest payment-in-kind (an “Athyrium PIK Interest Payment”) by capitalizing the entire amount of interest accrued during the applicable interest period with the unpaid original principal amount outstanding on the last day of such period. The Athyrium Term Loans accrued interest at a rate equal to the three-month LIBOR rate, which was no less than 1.75% and no more than 3.50% (“LIBOR”), plus 8.25%, or for each interest period in which an Athyrium PIK Interest Payment was made, LIBOR plus 10.25%. The quarter ended December 31, 2022 was the last period eligible for the Athyrium PIK Interest Payment designation. The Athyrium Term Loans accrued interest at an effective interest rate of 13.71% during the period in which the debt was outstanding for the year ended 2023 compared to 12.87% for fiscal year 2022. Quarterly interest payments under the Athyrium Credit Agreement for the year ended December 31, 2023 totaled $8,476. Quarterly interest payments under the Athyrium Credit Agreement for the year ended December 31, 2022 totaled $23,387 and were designated and accounted for as Athyrium PIK Interest Payments and added to the outstanding principal balance of the borrowing. From the Athyrium Term Loans’ inception through December 31, 2022, the quarterly interest payments were designated and accounted for as Athyrium PIK Interest Payments and added to the outstanding principal balance of the borrowing. The quarter ended December 31, 2022 was the last period eligible for the Athyrium PIK Interest Payment designation. Deferred financing amortization of $1,069 and $916, and $531 was recognized for the years ended December 31, 2023, 2022, and 2021 respectively. On April 17, 2023, the outstanding principal of the Athyrium Term Loans, including the Athyrium PIK Interest Payments of $240,452 along with interest accrued of $1,335 for the first 17 days of the quarterly interest period ended June 30, 2023, was repaid with the funding received through the Pharmakon Loan Agreement. In accordance with the Athyrium Credit Agreement, upon the prepayment or repayment of all or any of the Athyrium Term Loans, the Company was obligated to pay an exit fee in an amount equal to 2.00% of the principal amount of the Athyrium Term Loans prepaid or repaid. In addition, each Athyrium Term Loan was subject to a 1.00% commitment fee at its respective borrowing date. As a result, the Company incurred prepayment and final payment fees of $17,261 upon repayment of the Athyrium Term Loans. Additionally, unamortized deferred financing costs of $11,758 associated with the Athyrium Term Loans were written-off at the time of repayment. Collectively, the prepayment and final payment fees and unamortized deferred financing costs totaled $29,019 and are reflected as a one-time loss on extinguishment of debt on the Consolidated Statements of Comprehensive Loss for the year ended December 31, 2023. |
Lease Obligations
Lease Obligations | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Lease, Obligations | Lease Obligations The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. Renewal options for the Company’s leases range from 1 to 3 years in length and begin from 2024 through 2030. Lease expense under operating and finance leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Operating lease expense $ 2,018 $ 1,578 $ 1,660 Finance lease expense: Amortization of right-of-use assets $ 1,212 $ 816 113 Interest on lease liabilities $ 201 $ 138 22 Total finance lease expense $ 1,413 $ 954 $ 135 Other supplemental information related to leases was as follows: As of December 31, 2023 2022 Weighted average remaining lease term: Operating leases 9.7 years 10.6 years Finance leases 3.0 years 3.0 years Weighted average discount rate: Operating leases 10.88 % 12.54 % Finance leases 7.46 % 5.00 % The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Operating lease assets: Operating lease assets, net Other Assets $ 8,682 $ 4,242 Operating lease liabilities: Current operating lease liabilities Operating lease liabilities – current liabilities $ 1,058 $ 1,516 Non-current operating lease liabilities Operating lease liabilities – long-term liabilities 8,390 4,027 Total operating lease liabilities $ 9,448 $ 5,543 The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Finance lease assets: Finance lease assets, net Other Assets $ 4,322 $ 2,564 Finance lease liabilities: Current finance lease liabilities Finance lease liabilities – current liabilities $ 1,590 $ 853 Non-current finance lease liabilities Finance lease liabilities – long-term liabilities 2,845 1,777 Total finance lease liabilities $ 4,435 $ 2,630 Operating lease assets are recorded net of accumulated amortization of $4,794 and $3,268 as of December 31, 2023 and 2022, respectively. Finance lease assets are recorded net of accumulated amortization of $2,293 and $1,081 as of December 31, 2023 and 2022, respectively. Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands): Operating Leases Finance Leases 2024 $ 2,014 $ 1,857 2025 1,701 1,732 2026 1,509 899 2027 1,463 437 2028 1,312 27 Thereafter 7,952 — Total lease payments 15,951 4,952 Less imputed interest (6,503) (517) Total $ 9,448 $ 4,435 Supplemental cash flow information related to leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows for finance leases $ 201 $ 898 $ 125 Operating cash flows for operating leases $ 1,920 $ 1,555 $ 1,490 Financing cash flows for finance leases $ 1,165 $ — $ — Operating lease assets obtained in exchange for operating lease liabilities: $ 4,695 $ 755 $ 1,536 Finance lease assets obtained in exchange for finance lease liabilities: $ 2,971 $ 1,302 $ 2,190 Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities: $ 924 $ — $ — |
Lessee, Finance Leases | Lease Obligations The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. Renewal options for the Company’s leases range from 1 to 3 years in length and begin from 2024 through 2030. Lease expense under operating and finance leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Operating lease expense $ 2,018 $ 1,578 $ 1,660 Finance lease expense: Amortization of right-of-use assets $ 1,212 $ 816 113 Interest on lease liabilities $ 201 $ 138 22 Total finance lease expense $ 1,413 $ 954 $ 135 Other supplemental information related to leases was as follows: As of December 31, 2023 2022 Weighted average remaining lease term: Operating leases 9.7 years 10.6 years Finance leases 3.0 years 3.0 years Weighted average discount rate: Operating leases 10.88 % 12.54 % Finance leases 7.46 % 5.00 % The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Operating lease assets: Operating lease assets, net Other Assets $ 8,682 $ 4,242 Operating lease liabilities: Current operating lease liabilities Operating lease liabilities – current liabilities $ 1,058 $ 1,516 Non-current operating lease liabilities Operating lease liabilities – long-term liabilities 8,390 4,027 Total operating lease liabilities $ 9,448 $ 5,543 The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Finance lease assets: Finance lease assets, net Other Assets $ 4,322 $ 2,564 Finance lease liabilities: Current finance lease liabilities Finance lease liabilities – current liabilities $ 1,590 $ 853 Non-current finance lease liabilities Finance lease liabilities – long-term liabilities 2,845 1,777 Total finance lease liabilities $ 4,435 $ 2,630 Operating lease assets are recorded net of accumulated amortization of $4,794 and $3,268 as of December 31, 2023 and 2022, respectively. Finance lease assets are recorded net of accumulated amortization of $2,293 and $1,081 as of December 31, 2023 and 2022, respectively. Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands): Operating Leases Finance Leases 2024 $ 2,014 $ 1,857 2025 1,701 1,732 2026 1,509 899 2027 1,463 437 2028 1,312 27 Thereafter 7,952 — Total lease payments 15,951 4,952 Less imputed interest (6,503) (517) Total $ 9,448 $ 4,435 Supplemental cash flow information related to leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows for finance leases $ 201 $ 898 $ 125 Operating cash flows for operating leases $ 1,920 $ 1,555 $ 1,490 Financing cash flows for finance leases $ 1,165 $ — $ — Operating lease assets obtained in exchange for operating lease liabilities: $ 4,695 $ 755 $ 1,536 Finance lease assets obtained in exchange for finance lease liabilities: $ 2,971 $ 1,302 $ 2,190 Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities: $ 924 $ — $ — |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2023 | |
Equity [Abstract] | |
Stockholders' Equity | Stockholders ’ Equity Sales of Common Stock On March 1, 2021, the Company filed an automatic shelf registration statement on Form S-3 with the SEC. This shelf registration statement became effective automatically upon filing and allows the Company to sell an indeterminate number of securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities, and units, from time to time at prices and on terms to be determined at the time of sale. On November 19, 2021, concurrent with the Company entering into the 2021 RPI Royalty Purchase Agreement, the Company and RPI entered into the Common Stock Purchase Agreement, pursuant to which the Company issued 3,846 shares of the Company’s common stock to RPI for an aggregate purchase price of $50,000, at a price of $13.00 per share, calculated based on the 20-day volume weighted average price. The $13.00 per share price represented a premium of $1.11 over the closing price of $11.89 of the Company’s common stock on November 19, 2021, the last trading day prior to the execution of the Common Stock Purchase Agreement. The premium of $4,269 paid by RPI on the purchase of the Company’s common stock has been deferred and is being amortized as a component of interest expense of the 2021 RPI royalty financing obligation. On October 23, 2023, certain entities affiliated with Baker Bros. Advisors LP (the “Baker Entities”) net exercised the remaining balance of the pre-funded warrants held by such Baker Entities that were issued on November 21, 2019. Additionally, certain of the Baker Entities net exercised all of the pre-funded warrants that were issued on June 1, 2020. The exercises resulted in the issuance of 14,997 common shares. Following the exercises, there are no outstanding warrants. Shares Reserved for Future Issuance of Common Stock The Company had reserved shares of common stock for issuance as follows (in thousands): December 31, 2023 2022 Shares reserved for exercises of outstanding stock options 41,032 36,520 Shares reserved for vesting of restricted stock units 6,507 4,747 Shares reserved for exercises of warrants — 15,023 Shares reserved for future issuance under the Stock Incentive Plan 3,376 4,206 Shares reserved for future issuance under the Inducement Equity Incentive Plan 1,651 947 Shares reserved for future issuance under the Employee Stock Purchase Plan 5,454 5,792 Total shares reserved for future issuance 58,020 67,235 |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | Note 12 — Stock-Based Compensation As of December 31, 2023, the Company had three stock-based employee compensation plans: the Amended and Restated Stock Incentive Plan (“Incentive Plan”), the Amended and Restated Inducement Equity Incentive Plan (“Inducement Plan”) and the Amended and Restated Employee Stock Purchase Plan (“ESPP”). The Incentive Plan was most recently amended and restated on April 24, 2023 and approved by the Company’s stockholders on June 13, 2023. The Inducement Plan was most recently amended and restated by the Company’s Board of Directors on October 26, 2023. The ESPP was most recently amended and restated by the Company’s Board of Directors on July 7, 2023. The Company recorded the following stock-based compensation expense (in thousands): Years Ended December 31, 2023 2022 2021 Incentive Plan $ 44,581 $ 36,716 $ 27,062 Inducement Plan 9,958 6,550 6,055 ESPP 1,076 1,435 1,523 Stock-based compensation expense $ 55,615 $ 44,701 $ 34,640 Total stock-based compensation was allocated as follows: Years Ended December 31, 2023 2022 2021 Research and development $ 29,377 $ 24,936 $ 20,179 Selling, general and administrative 26,238 19,765 14,461 Total stock-based compensation expense $ 55,615 $ 44,701 $ 34,640 Stock Incentive Plan The Company grants stock option awards, restricted stock and restricted stock units to its employees, directors, and consultants under the Incentive Plan. Under the Incentive Plan, stock option awards are granted with an exercise price equal to the market price of the Company’s common stock at the date of grant. Stock option awards and restricted stock units granted to employees generally vest 25% each year until fully vested after four years. In December 2014, the Company issued 1,250 performance-based stock options. These awards vest upon successful completion of specific development milestones. As of December 31, 2023, 85% of these grants have vested. In January 2022, the Company issued 221 performance-based restricted stock unit awards. 21 of the awards met the performance objectives in 2022 and became eligible for vesting at 50% on the first anniversary of the grant date and 25% on each of the second and third anniversaries of the grant date, until fully vested after three years. The Company recognized $34 and $158 of stock compensation expense related to these awards during the years ended December 31, 2023 and December 31, 2022, respectively. Stock option awards and restricted stock unit awards granted to non-employee directors of the Company generally vest over one year. Stock option awards granted to new non-employee directors when they first join the Company’s Board of Directors generally vest, subject to the terms of the Incentive Plan, in 36 equal monthly installments over a three-year period measured from the grant date. All stock option awards have contractual terms of 10 years. Restricted stock unit awards granted to new non-employee directors when they first join the Company’s Board of Directors generally vest, subject to the terms of the Incentive Plan, in three equal annual installments beginning on the first anniversary of the grant date. The vesting and exercise provisions of all awards granted under the Incentive Plan are subject to acceleration in the event of certain stockholder-approved transactions, or upon the occurrence of a change in control as defined in the Incentive Plan. The following table summarizes stock option activity under the Incentive Plan: Shares Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value Outstanding at December 31, 2022 31,179 $ 8.56 Granted 6,625 6.67 Exercised (935) 5.21 $ 3,601 Cancelled or Forfeited (1,364) 10.00 Outstanding at December 31, 2023 35,505 $ 8.24 6.85 $ 12,280 Exercisable at December 31, 2023 21,008 $ 7.87 5.44 $ 12,280 Vested and expected to vest at December 31, 2023 33,557 $ 8.19 6.77 $ 12,280 The total intrinsic value of stock option awards exercised under the Incentive Plan was $21,150 and $25,484 during the years ended December 31, 2022 and 2021, respectively. The aggregate intrinsic value represents the total proceeds (calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date) received by all individuals who exercised stock option awards during the period. The following table summarizes restricted stock unit activity under the Incentive Plan: Shares Weighted Average Grant Date Fair Value Unvested at December 31, 2022 4,159 $ 10.69 Granted 3,106 6.71 Vested (1,137) 11.04 Forfeited (536) 7.91 Unvested at December 31, 2023 5,592 $ 8.67 For restricted stock unit awards granted under the Incentive Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during 2023, 2022, and 2021 was $6.71, $11.20, and 11.36, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method. As of December 31, 2023, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Incentive Plan was $40,981, which is expected to be recognized over a weighted average period of 2.0 years. Inducement Equity Incentive Plan The Company has the ability to grant stock option and restricted stock unit awards to newly-hired employees as inducements material to each employee entering employment with the Company. Awards granted to newly hired employees generally vest 25% each year until fully vested after four years and are subject to the terms and conditions of the Inducement Plan. Each stock option has a term of 10 years. The vesting and exercise provisions of all awards granted under the Inducement Plan are subject to acceleration in the event of certain stockholder-approved transactions, or upon the occurrence of a change in control as defined in the Inducement Plan. The following table summarizes stock option activity under the Inducement Plan: Shares Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value Outstanding at December 31, 2022 5,341 $ 8.80 Granted 1,196 8.15 Exercised (344) 3.70 $ 1,803 Cancelled or Forfeited (666) 10.47 Outstanding at December 31, 2023 5,527 $ 8.77 7.76 $ 4,463 Exercisable at December 31, 2023 2,448 $ 7.17 6.89 $ 3,434 Vested and expected to vest at December 31, 2023 5,122 $ 8.68 7.72 $ 4,291 The total intrinsic value of stock option awards exercised under the Inducement Plan was $3,710 and $6,700 during the years ended December 31, 2022 and 2021, respectively. The aggregate intrinsic value represents the total proceeds (calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date) received by all individuals who exercised stock option awards during the period. The following table summarizes restricted stock unit activity under the Inducement Plan: Shares Weighted Average Grant Date Fair Value Unvested at December 31, 2022 588 $ 13.14 Granted 581 7.81 Vested (139) 13.13 Forfeited (115) 11.98 Unvested at December 31, 2023 915 $ 9.90 For restricted stock unit awards granted under the Inducement Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during 2023 and 2022 was $7.81 and 13.21, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method. No restricted stock unit awards were granted under the Inducement Plan during 2021. As of December 31, 2023, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Inducement Plan was $7,044, which is expected to be recognized over a weighted average period of 1.8 years. Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors under the Incentive and Inducement Plans For stock option awards granted under the Incentive Plan and the Inducement Plan, the fair value is estimated on the date of grant using a Black-Scholes option pricing model and the assumptions noted below. The fair value of the stock option awards is amortized to expense over the vesting periods using a straight-line expense attribution method. Historically, the expected life was based on the average of the assumption that all outstanding stock option awards will be exercised at full vesting and the assumption that all outstanding stock option awards will be exercised at the midpoint of the current date (if already vested) or at full vesting (if not yet vested) and the full contractual term. Effective July 1, 2023, the expected life is based on the historical settlement of options by taking into account exercises and post- vesting terminations and weighing them based on the number of options settled. This change in approach did not have a significant impact on the value of the stock option awards granted. The expected volatility represents the historical volatility on the Company’s publicly-traded common stock. The Company has assumed no expected dividend yield, as dividends have never been paid to stock or option holders and will not be paid for the foreseeable future. The weighted average risk-free interest rate is the implied yield currently available on zero-coupon government issues with a remaining term equal to the expected term. Stock Incentive Plan The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Incentive Plan during the years ended December 31, 2023, 2022, and 2021: Years Ended December 31, 2023 2022 2021 Expected Life in Years 5.7 5.5 5.5 Expected Volatility 82.5 % 84.2 % 84.2 % Expected Dividend Yield 0.0 % 0.0 % 0.0 % Risk-Free Interest Rate 3.9 % 3.6 % 1.1 % Weighted average grant date fair value per share $ 4.75 $ 7.57 $ 7.93 The total fair value of the stock option awards vested under the Incentive Plan was $33,731, $28,916, and $20,510 during the years ended December 31, 2023, 2022, and 2021, respectively. As of December 31, 2023, total unrecognized compensation cost related to unvested stock option awards granted under the Incentive Plan was $75,164, which is expected to be recognized over a weighted average period of 1.9 years. Inducement Equity Incentive Plan The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the years ended December 31, 2023, 2022, and 2021: Years Ended December 31, 2023 2022 2021 Expected Life in Years 5.6 5.5 5.5 Expected Volatility 83.5 % 84.2 % 84.2 % Expected Dividend Yield 0.0 % 0.0 % 0.0 % Risk-Free Interest Rate 4.0 % 3.2 % 0.9 % Weighted average grant date fair value per share $ 5.79 $ 9.54 $ 9.65 The total fair value of the stock option awards vested under the Inducement Plan was $7,698, $4,659, and $2,885 during the years ended December 31, 2023, 2022, and 2021, respectively. As of December 31, 2023, total unrecognized compensation cost related to unvested stock option awards granted under the Inducement Plan was $16,216, which is expected to be recognized over a weighted average period of 1.5 years. Employee Stock Purchase Plan The Company has reserved a total of 7,975 shares of common stock to be purchased under the ESPP, of which 5,454 shares remain available for purchase at December 31, 2023. Eligible employees may authorize up to 15% of their salary to purchase common stock at the lower of 85% of the beginning or 85% of the ending price during six-month purchase intervals. No more than three thousand shares may be purchased by any one employee at each purchase date, and no employee may purchase stock having a fair market value at the commencement date of $25 or more in any one calendar year. During the years ended December 31, 2023, 2022, and 2021, the Company issued 338, 260, and 321 shares of common stock under the ESPP, respectively, at a weighted average price per share of $7.68, $11.03, and $6.20, respectively. Compensation expense for shares purchased under the ESPP related to the purchase discount and the “look-back” option were determined using a Black-Scholes option pricing model. The weighted average grant date fair values of shares granted under the ESPP during the years ended December 31, 2023, 2022, and 2021, were $3.82, $6.02, and $2.80, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes The components of loss before provision for income taxes were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Domestic $ (206,674) $ (225,127) $ (159,632) Foreign (19,555) (19,256) (22,177) Loss before provision for income taxes $ (226,229) $ (244,383) $ (181,809) The components of the (benefit) expense for income taxes were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Current expense (benefit) provision: U.S. Federal and state $ (45) $ 2,430 $ 2,179 Foreign 1,037 292 233 Total current expense provision 992 2,722 2,412 Deferred expense (benefit) provision: U.S. Federal and state (120) 11 (159) Foreign (562) — — Total deferred expense provision (682) 11 (159) Total expense provision $ 310 $ 2,733 $ 2,253 The differences between the Company’s effective tax rate and the statutory tax rate in 2023, 2022, and 2021 were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021) $ (47,328) $ (51,321) $ (38,175) State and local income taxes net of federal tax benefit (3,477) (1,816) (2,288) Permanent items 3,015 (1,608) (1,343) Expiration of attribute carryforwards — — (1,057) Research and development tax credits (3,301) (9,793) (5,994) Foreign rate differential 2,255 1,862 1,940 Other 1,656 (5,485) 1,216 Change in valuation allowance 47,490 70,894 47,954 Income tax expense $ 310 $ 2,733 $ 2,253 The Company recognizes the impact of a tax position in its financial statements if it is more likely than not that the position will be sustained on audit based on the technical merits of the position. The Company has concluded that it has an uncertain tax position pertaining to its research and development and orphan drug credit carryforwards. The Company has established these credits based on information and calculations it believes are appropriate and the best estimate of the underlying credit. Any changes to the Company’s unrecognized tax benefits are offset by an adjustment to the valuation allowance and there would be no impact on the Company’s financial statements. The Company does not expect its unrecognized tax benefits to change significantly over the next 12 months. A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): 2023 2022 Balance at January 1, $ 13,844 $ 9,729 Additions to current period tax positions 825 4,115 Reductions to prior period tax provisions (307) — Balance at December 31, $ 14,362 $ 13,844 The Company’s ability to utilize the net operating loss and tax credit carryforwards in the future may be subject to substantial restrictions in the event of past or future ownership changes as defined in Section 382 of the IRC and similar state tax law. Significant components of the Company’s deferred tax assets and liabilities are as follows (in thousands): December 31, 2023 2022 Deferred tax assets: Net federal and state operating losses $ 108,753 $ 101,600 Research and development credits 88,390 86,321 Royalty income 122,391 115,554 Stock-based compensation 26,113 19,374 Capitalized R&D 75,081 62,794 Leasing obligations 3,076 1,842 Other 17,515 4,354 Total deferred tax assets 441,319 391,839 Deferred tax liabilities: Fixed assets (678) (717) Right of use asset (2,872) (1,525) Total deferred tax liabilities (3,550) (2,242) Valuation allowance (437,098) (389,608) Net deferred tax assets (liabilities) $ 671 $ (11) The majority of the Company’s deferred tax assets relate to net operating loss and research and development carryforwards that can only be realized if the Company is profitable in future periods. It is uncertain whether the Company will realize any tax benefit related to these carryforwards. Accordingly, the Company has provided a valuation allowance against substantially all the net deferred tax assets due to uncertainties as to their ultimate realization. The valuation allowance will remain at the full amount of the deferred tax assets until it is more likely than not that the related tax benefits will be realized. The Company’s valuation allowance increased by $47,490, $70,894, and $47,954 in 2023, 2022, and 2021, respectively. As of December 31, 2023, the Company had U.S. federal operating loss carryforwards of $432,328, state operating loss carryforwards of $183,478, foreign net operating losses of $65,416, and U.S. research and development and orphan drug credit carryforwards of $102,752, which will expire at various dates from 2024 through 2043. Federal losses, state losses, and research and development credit carryforwards began expiring in 2021. The foreign net operating losses have an indefinite carryforward period. Tax years 2020-2023 remain open to examination by the major taxing jurisdictions to which the Company is subject. Additionally, years prior to 2020 are also open to examination to the extent of loss and credit carryforwards from those years. The Company recognizes interest and penalties accrued related to unrecognized tax benefits as components of its income tax provision. However, there were no provisions or accruals for interest and penalties in 2023, 2022, and 2021. As of December 31, 2023, the Company has minimal accumulated undistributed earnings generated by its foreign subsidiaries which have already been subject to local and U.S. tax (as part of the global intangible low-taxed income provisions). The Company intends to indefinitely reinvest these earnings, as well as future earnings from its foreign subsidiaries to fund its international operations. In addition, the Company expects future U.S. cash generation will be sufficient to meet future U.S. cash needs. |
Employee 401(k) Plan
Employee 401(k) Plan | 12 Months Ended |
Dec. 31, 2023 | |
Retirement Benefits [Abstract] | |
Employee 401(k) Plan | Employee 401(k) Plan In January 1991, the Company adopted an employee retirement plan (“401(k) Plan”) under Section 401(k) of the IRC covering all employees. Employee contributions may be made to the 401(k) Plan up to limits established by the Internal Revenue Service. Company matching contributions may be made at the discretion of the Board of Directors. The Company made matching contributions of $5,716, $3,758, and $2,834 in 2023, 2022, and 2021, respectively. |
Collaborative and Other Relatio
Collaborative and Other Relationships | 12 Months Ended |
Dec. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborative and Other Relationships | Collaborative and Other Relationships Government Collaborations National Institute of Allergy and Infectious Diseases (“NIAID/HHS”) In September 2013, NIAID/HHS contracted with the Company for the development of galidesivir as a treatment for Marburg virus disease and subsequently, Yellow Fever and Ebola virus disease. On September 15, 2021, the Company entered into an amendment to pay for certain additional costs, including additional manufacturing development costs and overhead, and to change the total value of the contract, as amended, to $47,315 from $45,931. All options under the contract have been awarded. In August 2020, NIAID/HHS awarded the Company a new contract, with potential aggregate funding of up to $43,908 if all contract options were exercised, to manufacture and evaluate the safety, efficacy and tolerability of galidesivir. NIAID/HHS made an initial award of $6,326 to the Company under this contract. Revenue related to this contract was recognized during the years ended December 31, 2022 and 2021. Biomedical Advanced Research and Development Authority (“BARDA/HHS”) In March 2015, BARDA/HHS awarded the Company a contract for the continued development of galidesivir as a potential treatment for diseases caused by RNA pathogens, including filoviruses. This BARDA/HHS contract included a base contract of $16,265 to support galidesivir drug manufacturing, as well as $22,855 in additional development options that could be exercised by the government, bringing the potential value of the contract to $39,120. As of December 31, 2022, a total of $20,574 was awarded under exercised options within this contract. The most recent development option was completed as of December 31, 2022. The contracts with NIAID/HHS and BARDA/HHS were cost-plus-fixed-fee contracts. That is, the Company was entitled to receive reimbursement for all costs incurred in accordance with the contract provisions that were related to the development of galidesivir plus a fixed fee, or profit. BARDA/HHS and NIAID/HHS made periodic assessments of progress, and the continuation of the contracts was based on the Company’s performance, the timeliness and quality of deliverables, and other factors. The government had rights under certain contract clauses to terminate these contracts. These contracts were terminable by the government at any time for breach or without cause. As of December 31, 2022, all of the Company’s government funding for galidesivir had expired. U.S. Department of Health and Human Services (“HHS”) In September 2018, HHS awarded the Company a $34,660 contract for the procurement of up to 50,000 doses of RAPIVAB (peramivir injection) over a five-year period. The Company initially delivered 20,000 doses of RAPIVAB under this contract in 2019 for a total price of approximately $13,864. The Company further delivered 20,000 and 9,980 doses of RAPIVAB in 2022 and 2021, respectively, and recorded revenue of $13,864 and $6,918 for the years ending December 31, 2022 and 2021, respectively. As of December 31, 2022, the Company had delivered a total of 49,980 RAPIVAB doses of the 50,000 RAPIVAB doses available under the contract, effectively completing the contract with HHS. ORLADEYO Torii Pharmaceutical Co., Ltd. (“Torii”) On November 5, 2019, the Company entered into a Commercialization and License Agreement with Torii (the “Original Torii Agreement”), granting Torii the exclusive right to commercialize ORLADEYO for the prevention of hereditary angioedema (“HAE”) attacks in Japan. Under the Original Torii Agreement, the Company received an upfront, non-refundable payment of $22,000. The Company received an additional milestone payment of $15,000 in the second quarter of 2021 upon receipt from the Japanese NHI of a reimbursement price approval for ORLADEYO. In addition, the Company was entitled to receive tiered royalty payments, ranging from 20% to 40% of annual net sales of ORLADEYO in Japan during each calendar year. Torii’s royalty payment obligations were subject to customary reductions in certain circumstances, but could not be reduced by more than 50% of the amount that otherwise would have been payable to the Company in the applicable calendar quarter. The Company identified performance obligations under the Original Torii Agreement related to (i) the license to develop and commercialize ORLADEYO, (ii) regulatory approval support, and (iii) reimbursement pricing approval support. These were each determined to be distinct from the other performance obligations. The Company allocated the $22,000 upfront consideration to the identified performance obligations using estimation approaches to determine the standalone selling prices under ASC Topic 606. Specifically, in determining the value related to the license, a valuation approach utilizing risk adjusted discounted cash flow projections was used, and an expected cost plus margin approach was utilized for the other performance obligations. On November 30, 2023, the Company entered into an Amended and Restated Commercialization and License Agreement with Torii (as amended, the “Torii Agreement”). Under the Torii Agreement, the Company is entitled to receive tiered royalty payments, ranging from 20% to 80% of annual net sales of ORLADEYO in Japan during each calendar year. The Company is now responsible for all commercial promotion activities to support ORLADEYO sales in Japan, and Torii is responsible for HAE disease awareness activities in Japan. The Company will receive a 20% royalty on annual Japanese sales below a prespecified threshold and an 80% royalty on annual Japanese sales above the prespecified threshold. Torii’s updated royalty payment obligations commenced upon November 30, 2023 and expire upon the later of (i) the tenth anniversary of the date of first commercial sale of ORLADEYO in Japan, (ii) the expiration of the Company’s patents covering ORLADEYO, and (iii) the expiration of regulatory exclusivity for ORLADEYO in Japan. The Company determined that the Torii Agreement represented a contract modification to be accounted for as if it were part of the Original Torii Agreement under ASC Topic 606. As the performance obligations under the Original Torii agreement had been fully satisfied, the Company was not required to adjust revenue previously recognized. Peramivir Injection (RAPIVAB, RAPIACTA, PERAMIFLU) Shionogi & Co., Ltd. (“Shionogi”) In February 2007, the Company entered into an exclusive license agreement with Shionogi to develop and commercialize peramivir in Japan for the treatment of seasonal and potentially life-threatening human influenza. Under the terms of the agreement, Shionogi obtained rights to injectable formulations of peramivir in Japan. In October 2008, the Company and Shionogi amended the license agreement to expand the territory covered by the agreement to include Taiwan. Shionogi has commercially launched peramivir under the commercial name RAPIACTA in Japan and Taiwan. The Company developed peramivir under a license from UAB and will owe sublicense payments to UAB on any future milestone payments and/or royalties received by the Company from Shionogi. Green Cross Corporation (“Green Cross”) In June 2006, the Company entered into an agreement with Green Cross to develop and commercialize peramivir in Korea. Under the terms of the agreement, Green Cross is responsible for all development, regulatory, and commercialization costs in Korea and the Company is entitled to share in profits resulting from the sale of peramivir in Korea, including the sale of peramivir to the Korean government for stockpiling purposes. Furthermore, Green Cross will pay the Company a premium over its cost to supply peramivir for development and any future marketing of peramivir products in Korea. Other Collaborations Clearside Biomedical, Inc. (“Clearside”) On November 3, 2023, the Company announced that it entered into a license agreement (the “Clearside Agreement”) with Clearside, enabling the Company to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector® to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema. Under the Clearside Agreement, Clearside received a $5,000 upfront license fee from the Company, which has been recognized in research and development expenses during the year ended December 31, 2023. Clearside is eligible to receive up to an additional $30,000 in clinical and regulatory milestone payments, and up to a total of $47,500 in three post-approval sales-based milestone payments as annual global net sales progress to $2,000,000. The Company will pay Clearside tiered mid-single digit royalties on annual global net product sales, at three tiers, including a top tier of >$1,500,000. |
Workforce Reduction
Workforce Reduction | 12 Months Ended |
Dec. 31, 2023 | |
Restructuring and Related Activities [Abstract] | |
Workforce Reduction | Workforce Reduction In January 2024, the Company announced a reduction of workforce. The majority of the impacted employees had a termination date in January 2024, with certain employees exiting later in 2024. The Company notified the impacted employees in January 2024. The Company incurred costs related to employee severance, benefits, and related costs which were accounted for as ongoing terminations benefits under ASC Topic 712, Nonretirement Postemployment Benefits . As of December 31, 2023, it was considered probable that payment would be owed and the amount of payment was considered to be reasonably estimable, which resulted in the recognition of $3,380 of costs related to the workforce reduction during the year ended December 31, 2023, of which $3,026 has been recognized in research and development expenses and $354 has been recognized in selling, general and administrative expenses in the Consolidated Statement of Comprehensive Loss. As of December 31, 2023, none of the costs were paid and are included within accrued expenses on the Consolidated Balance Sheet. These costs are expected to be disbursed during the period of January 1, 2024 through December 31, 2024. In addition, the employees impacted by the workforce reduction received an amount equal to the bonus amount the employee would have received through continued employment with the Company, which was considered a one-time termination benefit pursuant to ASC Topic 420, Exit or Disposal Costs . As a result, $1,264 will be recognized during the three months ended March 31, 2024, the period in which the communication occurred, of which $1,201 will be recognized in research and development expenses, and $63 will be recognized in selling, general and administrative expenses in the Consolidated Statement of Comprehensive Loss. This cost is expected to be disbursed during the period of January 1, 2024 through March 31, 2024. The Company does not expect to incur any additional significant costs related to this workforce reduction. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2023 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. The Company has concluded that no subsequent events have occurred that require disclosure, except as described in “Note 16— Workforce Reduction”. |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Pay vs Performance Disclosure | |||
Net loss | $ (226,539) | $ (247,116) | $ (184,062) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended | 12 Months Ended |
Dec. 31, 2023 | Dec. 31, 2023 | |
Trading Arrangements, by Individual | ||
Rule 10b5-1 Arrangement Adopted | false | |
Non-Rule 10b5-1 Arrangement Adopted | false | |
Rule 10b5-1 Arrangement Terminated | true | |
Non-Rule 10b5-1 Arrangement Terminated | false | |
Officer Trading Arrangement [Member] | ||
Trading Arrangements, by Individual | ||
Material Terms of Trading Arrangement | During the three months ended December 31, 2023, the directors and officers of the Company adopted or terminated the “Rule 10b5-1 trading arrangements” or “non-Rule 10b5-1 trading arrangements” (as each term is defined in Item 408(a) of Regulation S-K) set forth in the table below. Name (Title) Action Termination Date Type of Trading Arrangement Duration of Trading Arrangement Aggregate Number of Securities Jon P. Stonehouse, President and Chief Executive Officer Termination November 7, 2023 Rule 10b5-1 trading arrangement (1) (1) Anthony J. Doyle, Chief Financial Officer and Treasurer Termination November 7, 2023 Rule 10b5-1 trading arrangement (1) (1) Helen M. Thackray, M.D., Chief Research & Development Officer Termination November 8, 2023 Rule 10b5-1 trading arrangement (2) (2) Alane P. Barnes, Chief Legal Officer and Secretary Termination November 7, 2023 Rule 10b5-1 trading arrangement (1) (1) Charles K. Gayer, Chief Commercial Officer Termination November 7, 2023 Rule 10b5-1 trading arrangement (2) (2) (1) This trading plan was originally adopted on March 11, 2022, to cover tax withholding obligations, commissions and any fees related to the vesting of restricted stock units, with a scheduled expiration date of December 31, 2025. The aggregate number of securities that would have been sold under this plan is indeterminable because the number of shares that would have been sold to satisfy applicable tax withholding obligations upon vesting is unknown, as the number would vary based on the extent to which vesting conditions were satisfied and the market price of the Company’s common stock at the time of settlement. (2) This trading plan was originally adopted on March 14, 2022, to cover tax withholding obligations, commissions and any fees related to the vesting of restricted stock units, with a scheduled expiration date of December 31, 2025. The aggregate number of securities that would have been sold under this plan is indeterminable because the number of shares that would have been sold to satisfy applicable tax withholding obligations upon vesting is unknown, as the number would vary based on the extent to which vesting conditions were satisfied and the market price of the Company’s common stock at the time of settlement. | |
Officer Trading Arrangement [Member] | Jon P. Stonehouse [Member] | ||
Trading Arrangements, by Individual | ||
Name | Jon P. Stonehouse | |
Title | President and Chief Executive Officer | |
Rule 10b5-1 Arrangement Terminated | true | |
Termination Date | November 7, 2023 | |
Officer Trading Arrangement [Member] | Anthony J. Doyle [Member] | ||
Trading Arrangements, by Individual | ||
Name | Anthony J. Doyle | |
Title | Chief Financial Officer and Treasurer | |
Rule 10b5-1 Arrangement Terminated | true | |
Termination Date | November 7, 2023 | |
Officer Trading Arrangement [Member] | Helen M. Thackray [Member] | ||
Trading Arrangements, by Individual | ||
Name | Helen M. Thackray, M.D. | |
Title | Chief Research & Development Officer | |
Rule 10b5-1 Arrangement Terminated | true | |
Termination Date | November 8, 2023 | |
Officer Trading Arrangement [Member] | Alane P. Barnes [Member] | ||
Trading Arrangements, by Individual | ||
Name | Alane P. Barnes | |
Title | Chief Legal Officer and Secretary | |
Rule 10b5-1 Arrangement Terminated | true | |
Termination Date | November 7, 2023 | |
Officer Trading Arrangement [Member] | Charles K. Gayer [Member] | ||
Trading Arrangements, by Individual | ||
Name | Charles K. Gayer | |
Title | Chief Commercial Officer | |
Rule 10b5-1 Arrangement Terminated | true | |
Termination Date | November 7, 2023 |
Significant Accounting Polici_2
Significant Accounting Policies and Concentrations of Risk (Policies) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
The Company | The Company BioCryst Pharmaceuticals, Inc. (the “Company”) is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. The Company leverages its expertise in structure-guided drug design with the goal of developing first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat rare diseases. The Company was founded in 1986 and incorporated in Delaware in 1991, and its headquarters is located in Durham, North Carolina. The Company integrates the disciplines of biology, crystallography, medicinal chemistry and computer modeling to discover and develop small molecule and protein therapeutics through the process known as structure-guided drug design. The Company’s marketed products include oral, once-daily ORLADEYO® for the prevention of hereditary angioedema (“HAE”) attacks and RAPIVAB® (peramivir injection) for the treatment of acute uncomplicated influenza in the United States. ORLADEYO received regulatory approval in the United States in December 2020. ORLADEYO has also received regulatory approvals in multiple global markets. The Company is commercializing ORLADEYO in each of these territories directly or through distributors, except in Japan where Torii Pharmaceutical Co., Ltd. (“Torii”), the Company’s collaborative partner, conducts certain commercialization activities with respect to ORLADEYO for the prevention of HAE attacks in exchange for certain royalty payments to the Company. In addition to its approval in the United States, peramivir injection has received regulatory approvals in Canada, Australia, Japan, Taiwan and Korea. Based on the Company’s expectations for revenue and operating expenses, the Company believes its financial resources available at December 31, 2023 will be sufficient to fund its operations for at least the next 12 months. The Company has sustained operating losses for the majority of its corporate history and expects that its total 2024 expenses will exceed its total 2024 revenues. The Company expects to continue to incur operating losses and negative cash flows until revenues reach a level sufficient to support ongoing operations. The Company’s liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of its products and the progression of its product candidates in the future. The Company regularly evaluates other opportunities to fund future operations, including: (1) out-licensing rights to certain of its products or product candidates, pursuant to which the Company would receive cash milestone payments; (2) raising additional capital through equity or debt financings or from other sources, including royalty or other monetization transactions; (3) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (4) reducing spending on one or more research and development programs, including by discontinuing development; (5) restructuring operations to change its overhead structure; and/or (6) securing U.S. Government funding of its programs, including obtaining procurement contracts. The Company may, in the future, issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities and units, through private placement transactions or registered public offerings. The Company’s future liquidity needs, and ability to address those needs, will largely be determined by the success of its products and product candidates; the timing, scope and magnitude of its research and development and commercial expenses; and key developments and regulatory events and its decisions in the future. |
Basis of Presentation | Basis of Presentation The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany transactions and balances among the consolidated entities have been eliminated from the consolidated financial statements. The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). Such consolidated financial statements reflect all adjustments that are, in management’s opinion, necessary to present fairly, in all material respects, the Company’s consolidated financial position, results of operations, and cash flows. There were no adjustments other than normal recurring adjustments. Certain prior year amounts have been reclassified to conform to the current year presentation. The Company has made certain presentation changes relative to its revenue, which management considers fundamental to understanding the Company’s current business and financial performance related to its primary product, ORLADEYO, including expanded international sales of ORLADEYO, relative to the Company’s other sources of revenue. Accordingly, certain disaggregated revenue information has been provided in this Note 1 and “Note 2—Revenue” to these consolidated financial statements. These presentation changes have been applied to prior year revenue amounts for consistency and comparability. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses, and related disclosure of contingent assets and liabilities. Significant estimates in the Company’s consolidated financial statements have been made relative to the calculation of net product sales, the ORLADEYO and Factor D inhibitors royalty financing obligations, inventory reserves, certain accruals, primarily related to the Company’s research and development expenses, the valuation of stock options and the valuation allowance for deferred tax assets resulting from net operating losses. These estimates are based on historical experience and on various other assumptions that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates. |
Revenue Recognition | Revenue Recognition The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands): Years Ended December 31, 2023 2022 2021 Product sales, net 324,696 267,710 136,350 Collaborative and other revenues 6,716 3,117 20,820 Total revenues $ 331,412 $ 270,827 $ 157,170 Pursuant to Accounting Standards Codification (“ASC”) Topic 606, the Company recognizes revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To achieve this core principle, Topic 606 includes provisions within a five step model that includes (i) identifying the contract with a customer, (ii) identifying the performance obligations in the contract, (iii) determining the transaction price, (iv) allocating the transaction price to the performance obligations, and (v) recognizing revenue when, or as, an entity satisfies a performance obligation. At contract inception, the Company identifies the goods or services promised within each contract, assesses whether each promised good or service is distinct and determines those that are performance obligations. The Company recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when the performance obligation is satisfied. Product Sales, Net The Company’s principal sources of product sales are sales of ORLADEYO, which the Company began shipping to patients in December 2020, sales of peramivir to the Company’s licensing partners, and in prior years, sales of RAPIVAB to the U.S. Department of Health and Human Services (“HHS”) under the Company’s historical procurement contract, which was completed in 2022. In the United States, the Company ships ORLADEYO directly to patients through a single specialty pharmacy, which is considered its customer. In the European Union, United Kingdom and elsewhere, the Company sells ORLADEYO to specialty distributors as well as hospitals and pharmacies, which collectively are considered its customers. The Company recognizes revenue for sales when its customers obtain control of the product, which generally occurs upon delivery. For ORLADEYO, the Company classifies payments to its specialty pharmacy customer for certain services provided by its customer as selling, general and administrative expenses to the extent such services provided are determined to be distinct from the sale of ORLADEYO. Net revenue from sales of ORLADEYO is recorded at net selling price (transaction price), which includes estimates of variable consideration for which reserves are established for (i) estimated government rebates, such as Medicaid and Medicare Part D reimbursements, and estimated managed care rebates, (ii) estimated chargebacks, (iii) estimated costs of co-payment assistance programs and (iv) product returns. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable or as a current liability. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the applicable contract. The amount of variable consideration included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company's estimates. If actual results in the future vary from estimates, the Company adjusts these estimates, which would affect net product revenue and earnings in the period such variances become known. Government and Managed Care Rebates . The Company contracts with government agencies and managed care organizations or, collectively, third-party payors, so that ORLADEYO will be eligible for purchase by, or partial or full reimbursement from, such third-party payors. The Company estimates the rebates it will provide to third-party payors and deducts these estimated amounts from total gross product revenues at the time the revenues are recognized. These reserves are recorded in the same period in which the revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability. The Company estimates the rebates that it will provide to third-party payors based upon (i) the Company's contracts with these third-party payors, (ii) the government mandated discounts applicable to government-funded programs, (iii) a range of possible outcomes that are probability-weighted for the estimated payor mix, and (iv) product distribution information obtained from the Company's specialty pharmacy. Chargebacks . Chargebacks are discounts that occur when certain contracted customers, pharmacy benefit managers, insurance companies, and government programs purchase directly from the Company’s specialty pharmacy. These customers purchase the Company’s product under contracts negotiated between them and the Company’s specialty pharmacy. The specialty pharmacy, in turn, charges back to the Company the difference between the price the specialty pharmacy paid and the negotiated price paid by the contracted customers, which may be higher or lower than the specialty pharmacy’s purchase price from the Company. The Company estimates chargebacks and adjusts gross product revenues and accounts receivable based on the estimates at the time revenues are recognized. Co-payment assistance and patient assistance programs . Patients who have commercial insurance and meet certain eligibility requirements may receive co-payment assistance. Based upon the terms of the program and co-payment assistance utilization reports received from the specialty pharmacy, the Company is able to estimate the co-payment assistance amounts, which are recorded in the same period in which the related revenue is recognized, resulting in a reduction of product revenue. The Company also offers a patient assistance program that provides free drug product, for a limited period of time, to allow a patient’s insurance coverage to be established. Based on patient assistance program utilization reports provided by the specialty pharmacy, the Company records gross revenue of the product provided and a full reduction of the revenue amount for the free drug discount. Product returns . The Company does not provide contractual return rights to its customers, except in instances where the product is damaged or defective. Non-acceptance by the patient of shipped drug product by the specialty pharmacy is reflected as a reversal of sales in the period in which the sales were originally recorded. Reserves for estimated non-acceptances by patients are recorded as a reduction of revenue in the period that the related revenue is recognized, as well as a reduction to accounts receivable. Estimates of non-acceptance are based on quantitative information provided by the specialty pharmacy. Collaborative and Other Revenues The Company has collaboration and license agreements with a number of third parties, as well as research and development agreements with certain government entities. The Company’s primary sources of revenue from these collaborative and other research and development arrangements are license, service and royalty revenues. Revenue from license fees, royalty payments, milestone payments, and research and development fees are recognized as revenue when the earnings process is complete and the Company has no further continuing performance obligations or the Company has completed the performance obligations under the terms of the agreement. Arrangements that involve the delivery of more than one performance obligation are initially evaluated as to whether the intellectual property licenses granted by the Company represent distinct performance obligations. If they are determined to be distinct, the value of the intellectual property licenses would be recognized up front while the research and development service fees would be recognized as the performance obligations are satisfied. For performance obligations based on services performed, the Company measures progress using an input method based on the effort it expends or costs it incurs toward the satisfaction of the performance obligation in relation to the total estimated effort or costs. Variable consideration is assessed at each reporting period as to whether it is not subject to significant future reversal and, therefore, should be included in the transaction price at the inception of the contract. If a contract includes a fixed or minimum amount of research and development support, this also would be included in the transaction price. Changes to collaborations, such as the extensions of the research term or increasing the number of targets or technology covered under an existing agreement, are assessed for whether they represent a modification or should be accounted for as a new contract. For contracts with multiple performance obligations, revenue is allocated to each performance obligation based on its relative standalone selling price. Standalone selling prices are based on observable prices at which the Company separately sells the products or services. If a standalone selling price is not directly observable, then the Company estimates the standalone selling price using either an adjusted market assessment approach or an expected cost plus margin approach, representing the amount that the Company believes the market is willing to pay for the product or service. Analyzing the arrangement to identify performance obligations requires the use of judgment, and each may be an obligation to deliver services, a right or license to use an asset, or another performance obligation. Milestone payments are recognized as licensing revenue upon the achievement of specified milestones if (i) the milestone is substantive in nature and the achievement of the milestone was not probable at the inception of the agreement; and (ii) the Company has a right to payment. Any milestone payments received prior to satisfying these revenue recognition criteria are recorded as deferred revenue. Reimbursements received for direct out-of-pocket expenses related to research and development costs are recorded as revenue in the Consolidated Statements of Comprehensive Loss rather than as a reduction in expenses. Under the Company’s historical contracts with the Biomedical Advanced Research and Development Authority within the HHS (“BARDA/HHS”) and the National Institute of Allergy and Infectious Diseases (“NIAID/HHS”), revenue was recognized as reimbursable direct and indirect costs are incurred. Under certain of the Company’s license agreements, the Company receives royalty payments based upon its licensees’ net sales of covered products. Royalties are recognized at the later of when (i) the subsequent sale or usage occurs, or (ii) the performance obligation to which some or all of the sales-based or usage-based royalty has been satisfied. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company generally considers cash equivalents to be all cash held in commercial checking accounts, money market accounts, or investments in debt instruments and certificates of deposit with maturities of three months or less at the time of purchase. The carrying value of cash and cash equivalents approximates fair value due to the short-term nature of these items. |
Restricted Cash | Restricted Cash Total restricted cash was $1,804 and $1,472 as of December 31, 2023 and 2022, respectively, and primarily consisted of $1,493 and $1,449 as of December 31, 2023 and 2022, respectively, for a letter of credit the Company is required to maintain associated with its Birmingham lease. |
Investments | Investments The Company invests in high credit quality investments in accordance with its investment policy, which is designed to minimize the possibility of loss. The objective of the Company’s investment policy is to ensure the safety and preservation of invested funds, as well as maintaining liquidity sufficient to meet cash flow requirements. The Company places its excess cash with high credit quality financial institutions, commercial companies, and government agencies in order to limit the amount of its credit exposure. In accordance with its policy, the Company is able to invest in marketable debt securities that may consist of U.S. Government and government agency securities, money market and mutual fund investments, certificates of deposits, municipal and corporate notes and bonds, and commercial paper, among others. The Company’s investment policy requires it to purchase high-quality marketable securities with a maximum individual maturity of three years and requires an average portfolio maturity of no more than 12 months. Some of the securities in which the Company invests may have market risk. This means that a change in prevailing interest rates may cause the principal amount of the investment to fluctuate. To minimize this risk, the Company schedules its investments with maturities that coincide with expected cash flow needs, thus avoiding the need to redeem an investment prior to its maturity date. Accordingly, the Company does not believe it has a material exposure to interest rate risk arising from its investments. Generally, the Company’s investments are not collateralized. The Company has not realized any significant losses from its investments. The Company classifies all of its investments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive income, unless an unrealized loss is considered to be other than temporary, in which case the unrealized loss is charged to operations. The Company reviews its investments for other than temporary declines in fair value below cost basis at the end of each reporting period and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Factors considered include whether a decline in fair value below the amortized cost basis is due to credit-related factors or non-credit-related factors, the financial condition and near-term prospects of the Company, and the Company's intent and ability to hold the investment to allow for an anticipated recovery in fair value. A credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings. Any impairment that is not credit-related is recognized in other comprehensive income, net of applicable taxes unless deemed other than temporary. Realized gains and losses are reflected in interest and other income in the Consolidated Statements of Comprehensive Loss and are determined using the specific identification method with transactions recorded on a settlement date basis. Investments with original maturities at date of purchase beyond three months and which mature at or less than 12 months from the balance sheet date are classified as current. Investments with a maturity beyond 12 months from the balance sheet date are classified as long-term. |
Fair Value Measurements | Fair Value Measurements Assets and liabilities recorded at fair value on a recurring basis on the Consolidated Balance Sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Items measured at fair value on a recurring basis include investments (Note 3). The carrying amounts reflected in the Consolidated Balance Sheets for cash and cash equivalents, trade receivables, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities. |
Trade Receivables | Trade Receivables The majority of the Company’s trade receivables arise from product sales and primarily represent amounts due from its specialty pharmacy customer in the United States and other third-party distributors, hospitals and pharmacies in the European Union, United Kingdom and elsewhere and have standard payment terms that generally require payment within 30 to 90 days. Receivables from collaborations are recorded for amounts due to the Company related to reimbursable research and development costs from the HHS (Note 15), and royalty receivables from the Company’s partners, including Shionogi, Green Cross, and Torii (Note 15). Monthly invoices were submitted to the HHS related to reimbursable research and development costs. The Company was also entitled to monthly reimbursement of indirect costs based on rates stipulated in the underlying contract. The Company’s calculations of its indirect cost rates are subject to audit by the U.S. Government. The Company does not adjust its receivables for the effects of a significant financing component at contract inception if it expects to collect the receivables in one year or less from the time of sale. The Company provides reserves against trade receivables for estimated losses that may result from a customer's inability to pay. Receivables are evaluated to determine if any reserve or allowance should be recorded based on consideration of the current economic environment, expectations of future economic conditions, specific circumstances and the Company’s own historical collection experience. Amounts determined to be uncollectible are charged or written-off against the reserve. |
Inventory | Inventory The Company’s inventories primarily relate to ORLADEYO. Additionally, the Company’s inventories include RAPIVAB and peramivir. The Company values its inventories at the lower of cost or estimated net realizable value. The Company determines the cost of its inventories, which includes amounts related to materials, labor, manufacturing overhead, and shipping and handling costs on a first-in, first-out (FIFO) basis. Raw materials and work-in-process include all inventory costs prior to packaging and labelling, including raw material, active product ingredient, and the drug product. Finished goods include packaged and labelled products. The Company’s inventories are subject to expiration dating. The Company regularly evaluates the carrying value of its inventories and provides valuation reserves for any estimated obsolete, short-dated or unmarketable inventories. In addition, the Company may experience spoilage of its raw materials and supplies. The Company’s determination that a valuation reserve might be required, in addition to the quantification of such reserve, requires it to utilize significant judgment. The Company expenses costs related to the production of inventories as research and development expenses in the period incurred until such time it is believed that future economic benefit is expected to be recognized, which generally is reliant upon receipt of regulatory approval. Upon regulatory approval, the Company capitalizes subsequent costs related to the production of inventories. |
Property and Equipment | Property and Equipment Property and equipment are recorded at cost. Depreciation is computed using the straight-line method over the estimated useful lives of the assets. Computer equipment is depreciated over a life of three years. Laboratory equipment, office equipment, and software are depreciated over a life of five years. Furniture and fixtures are depreciated over a life of seven years. Leasehold improvements are amortized over their estimated useful lives or the expected lease term, whichever is less. The Company periodically reviews its property and equipment for impairment when events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Determination of recoverability is based on an estimate of undiscounted future cash flows resulting from the use of the asset and its eventual disposition. In the event that such cash flows are not expected to be sufficient to recover the carrying amount of the assets, the assets are written down to their estimated fair values. Property and equipment to be disposed of are reported at the lower of carrying amount or fair value less cost to sell. |
Accrued Expenses | Accrued Expenses The Company enters into contractual agreements with third-party vendors who provide research and development, manufacturing, distribution, and other services in the ordinary course of business. Some of these contracts are subject to milestone-based invoicing, and services are completed over an extended period of time. The Company records liabilities under these contractual commitments when it determines an obligation has been incurred, regardless of the timing of the invoice. This process involves reviewing open contracts and purchase orders, communicating with applicable Company personnel to identify services that have been performed on its behalf and estimating the level of service performed and the associated cost incurred for the service when the Company has not yet been invoiced or otherwise notified of actual cost. The majority of service providers invoice the Company monthly in arrears for services performed. The Company makes estimates of accrued expenses as of each balance sheet date in its financial statements based on the facts and circumstances which can include assumptions such as expected patient enrollment, site activation and estimated project duration. The Company periodically confirms the accuracy of its estimates with the service providers and makes adjustments if necessary. Examples of estimated accrued expenses include (i) fees paid to clinical research organizations (“CROs”) in connection with preclinical and toxicology studies and clinical trials; (ii) fees paid to investigative sites in connection with clinical trials; (iii) fees paid to contract manufacturers in connection with the production of the Company’s raw materials, drug substance, drug products, and product candidates; and (iv) professional fees. The Company bases its expenses related to clinical trials on its estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical trials on the Company’s behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. If the Company underestimates or overestimates the level of these costs, actual expenses could differ from such estimates. As of December 31, 2023 and December 31, 2022, the carrying value of accrued expenses approximates their fair value due to their short-term settlement. |
Cost of Product Sales | Cost of Product Sales Cost of product sales includes the cost of producing and distributing inventories that are related to product revenue during the respective period, including freight. In addition, shipping and handling costs for product shipments are recorded as incurred. Finally, cost of product sales may also include costs related to excess or obsolete inventory adjustment charges. |
Research and Development Expenses | Research and Development Expenses The Company’s research and development costs are expensed when incurred. Research and development expenses include all direct and indirect development costs related to the development of the Company’s portfolio of product candidates. Advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as expense when the related goods are delivered or the related services are performed. Research and development expenses include, among other items, personnel costs, including salaries and benefits, manufacturing costs, clinical, regulatory, and toxicology services performed by CROs, materials and supplies, and overhead allocations consisting of various administrative and facilities related costs, as well as termination fees and other commitments associated with discontinued programs. Most of the Company’s manufacturing and clinical and preclinical studies are performed by third-party CROs. Costs for studies performed by CROs are accrued by the Company over the service periods specified in the contracts, and estimates are adjusted based upon the Company’s ongoing review of the level of services actually performed. Additionally, the Company has license agreements with third parties, such as Albert Einstein College of Medicine of Yeshiva University, Industrial Research, Ltd., and the University of Alabama at Birmingham (“UAB”), which require fees related to sublicense agreements. The Company expenses sublicense payments as incurred. The Company groups its research and development expenses into two major categories: direct expenses and indirect expenses. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidate, conduct and manage clinical trials, as well as other costs related to the Company’s clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. These costs are accumulated and tracked by active program. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of the Company’s research and development efforts. These costs apply to work on non-active product candidates and the Company’s discovery research efforts. |
Selling, General and Administrative Expenses | Selling, General and Administrative Expenses Selling, general and administrative expenses are primarily comprised of compensation and benefits associated with sales and marketing, finance, human resources, legal, information technology and other administrative personnel. Additionally, selling, general and administrative expenses are comprised of market research, marketing, advertising and legal expenses, including patent costs, licenses and other general and administrative costs. Advertising costs related to ORLADEYO of $14,404, $14,891 and $5,705 were expensed as incurred for the years ended December 31, 2023, 2022 and 2021 respectively. All patent related costs are expensed to selling, general and administrative expenses when incurred as recoverability of such expenditures is uncertain. |
Leases | Leases The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. The Company accounts for lease obligations in accordance with ASU 2016-02: Leases (Topic 842) , which requires a lessee to recognize a right-of-use asset and a lease liability on its balance sheet for most leases. Certain of the Company’s operating leases provide for renewal options, which can vary by lease. The right-of-use asset and lease liabilities on the Company’s Consolidated Balance Sheets represent payments over the lease term, which includes renewal options for certain real estate leases that the Company is likely to exercise. As part of the Company’s assessment of the lease term, the Company elected the hindsight practical expedient, which allows companies to use current knowledge and expectations when determining the likelihood to extend lease options. Certain operating leases include rent escalation provisions, which the Company recognizes as expense on a straight-line basis. Lease expense for leases with an initial term of twelve months or less was not material. The discount rate used in the calculation of the Company’s right-of-use asset and lease liability was determined based on the stated rate within each contract when available, or the Company’s collateralized borrowing rate from lending institutions. The Company has not made any residual value guarantees related to its leases; therefore, the Company has no corresponding liability recorded on its Consolidated Balance Sheets. |
Share-Based Compensation | Stock-Based Compensation All share-based payments, including grants of stock option awards and restricted stock unit awards, are recognized in the Company’s Consolidated Statements of Comprehensive Loss based on their fair values. Stock-based compensation cost is estimated at the grant date based on the fair value of the award and is recognized as expense on a straight-line basis over the requisite service period of the award. Determining the appropriate fair value model and the related assumptions for the model requires judgment, including estimating the life of an award, the stock price volatility, and the expected term. The Company utilizes the Black-Scholes option-pricing model to value its stock option awards and recognize compensation expense on a straight-line basis over the vesting periods. The Company reduces stock-based compensation expense for estimated forfeitures. The estimation of share-based payment awards that will ultimately vest requires judgment, and to the extent actual results or updated estimates differ from the Company’s current estimates, such amounts will be recorded as a cumulative adjustment in the period estimates are revised. In addition, the Company has outstanding performance-based stock options and restricted stock units for which no compensation expense is recognized until “performance” is deemed to have occurred. Significant management judgment is also required in determining estimates of future stock price volatility to be used in the valuation of the options. Actual results, and future changes in estimates, may differ substantially from the Company’s current estimates. |
Interest Expense and Deferred Financing Costs | Interest Expense and Deferred Financing Costs Interest expense primarily relates to the PhaRMA Notes (Note 8), royalty financing obligations (Note 8), the secured term loan borrowings under the Athyrium Credit Agreement (Note 9), and during 2023, the term loan borrowings under the Pharmakon Loan Agreement (Note 9). |
Interest Expense and Royalty Financing Obligations | Interest Expense and Royalty Financing Obligations The royalty financing obligations are eligible to be repaid based on royalties from net sales of ORLADEYO and BCX10013. Interest expense is accrued using the effective interest rate method over the estimated period each of the related liabilities will be paid. This requires the Company to estimate the total amount of future royalty payments to be generated from product sales over the life of the agreement. The Company imputes interest on the carrying value of each of the royalty financing obligations and records interest expense using an imputed effective interest rate. The Company reassesses the expected royalty payments each reporting period and accounts for any changes through an adjustment to the effective interest rate on a prospective basis. The assumptions used in determining the expected repayment term of the debt and amortization period of the issuance costs require that the Company make estimates that could impact the carrying value of each of the liabilities, as well as the periods over which associated issuance costs will be amortized. A significant increase or decrease in forecasted net sales could materially impact each of the liability balances, interest expense and the time periods for repayment. |
Income Tax | Income Taxes The liability method is used in the Company’s accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on differences between financial reporting and tax bases of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. The Company accounts for uncertain tax positions in accordance with U.S. GAAP. Significant management judgment is required in determining the Company’s provision for income taxes, deferred tax assets and liabilities and any valuation allowance recorded against net deferred tax assets. The Company has recorded a valuation allowance against substantially all potential tax assets, due to uncertainties in its ability to utilize deferred tax assets, primarily consisting of certain net operating losses carried forward, before they expire. The valuation allowance is based on estimates of taxable income in each of the jurisdictions in which the Company operates and the period over which its deferred tax assets will be recoverable. Beginning in fiscal year 2021, the Company began accruing for U.S. state taxes and foreign income taxes as a result of increased nexus in both U.S. state and foreign jurisdictions where historically the Company had no presence. |
Foreign Currency | Foreign Currency The functional currencies of the Company’s foreign subsidiaries primarily are the local currencies of the country in which the subsidiary operates. The Company’s asset and liability accounts are translated at the current exchange rate as of the balance sheet date. Revenue and expense accounts are translated at the average exchange rate over the period. Adjustments resulting from the translation of the financial statements of the Company’s foreign subsidiaries into U.S. dollars are accumulated as a separate component of stockholders’ equity within accumulated other comprehensive income. Gains or losses resulting from transactions denominated in foreign currencies are included in foreign currency losses, net, within the Consolidated Statement of Comprehensive Loss. |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is based upon the weighted average number of common shares outstanding during the period. Diluted net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding during the period, after giving consideration to the dilutive effect of potentially dilutive common shares. The Company has generated a net loss in all periods presented, so the diluted net loss per share is equivalent to basic net loss per share for all periods presented herein because common equivalent shares from unexercised stock options, warrants and common shares expected to be issued under the Company’s equity compensation plans would be anti-dilutive. The Company excluded the following potential common shares, presented based on amounts outstanding as of December 31, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect: December 31, 2023 2022 Outstanding stock options 41,032 36,520 Unvested restricted stock unit awards 6,507 4,747 Warrants to purchase common stock — 15,023 Total 47,539 56,290 |
Accumulated Other Comprehensive Income | Accumulated Other Comprehensive Income Accumulated other comprehensive income is comprised of cumulative foreign currency translation adjustments and unrealized gains and losses on available-for-sale investments and is disclosed as a separate component of stockholders’ equity. Realized gain and loss amounts on available-for-sale investments are reclassified from accumulated other comprehensive loss and recorded as interest and other income on the Consolidated Statements of Comprehensive Loss. There were no realized gains or losses reclassified out of accumulated other comprehensive income for the years ended December 31, 2023 and 2022. For the year ended December 31, 2021, realized gains of $1 were reclassified out of accumulated other comprehensive income. |
Significant Customers and Other Risks | Significant Customers and Other Risks Significant Customers The Company’s primary sources of revenue and cash flow are the sales of ORLADEYO in the United States and other global markets and, during the years ended December 31, 2022 and 2021, sales of RAPIVAB (peramivir injection) under the Company’s historical procurement contract with the Assistant Secretary for Preparedness and Response within HHS. Additionally, the Company previously received reimbursement of galidesivir (formerly BCX4430) development expenses earned under cost-plus-fixed-fee contracts with BARDA/HHS and NIAID/HHS during the years ended December 31, 2022 and 2021. ORLADEYO is distributed through an arrangement with a single specialty pharmacy in the United States, which represents the substantial majority of the ORLADEYO net product sales. The specialty pharmacy subsequently sells ORLADEYO to its customers (pharmacy benefit managers, insurance companies, government programs and group purchasing organizations) and dispenses product to patients. The specialty pharmacy’s inability or unwillingness to continue these distribution activities could adversely impact the Company’s business, results of operations and financial condition. The Company is distributing ORLADEYO in other global markets directly or through distributors, except in Japan where Torii, the Company’s collaborative partner, has the exclusive right to commercialize ORLADEYO. The Company relied on BARDA/HHS and NIAID/HHS to reimburse predominantly all of the development costs for its galidesivir program and stockpiling sales of RAPIVAB to HHS. Accordingly, reimbursement of these expenses represented a significant portion of the Company’s collaborative and other research and development revenues. All government funding for galidesivir expired in 2022. Additionally, HHS was the primary customer for RAPIVAB, and it exercised the remaining options for the purchase of RAPIVAB under the procurement contract with the Company during 2022. Further, the Company’s drug development activities are performed by a limited group of third-party vendors. If any of these vendors were unable to perform its services, this could significantly impact the Company’s ability to complete its drug development activities. Risks from Third-Party Manufacturing and Distribution Concentration The Company relies on a single source manufacturer for active pharmaceutical ingredient and finished drug product manufacturing of product candidates in development and on a single specialty pharmacy for distribution of approved drug product in the United States. Delays or disruption in the manufacture or distribution of any product could adversely impact the future procurement stockpiling of the Company’s commercial product, commercial revenue and product candidates. Credit Risk Cash equivalents and investments are financial instruments that potentially subject the Company to concentration of risk to the extent recorded on the Consolidated Balance Sheets. The Company deposits excess cash with major financial institutions in the United States. Balances may exceed the amount of insurance provided on such deposits. The Company believes it has established guidelines for investment of its excess cash relative to diversification and maturities that maintain safety and liquidity. To minimize the exposure due to adverse shifts in interest rates, the Company maintains a portfolio of investments with an average maturity of approximately 12 months or less. The Company’s receivables from sales of ORLADEYO are primarily due from one customer, resulting in a concentration of credit risk. Sales of ORLADEYO from the Company to the specialty pharmacy only occur once an order of product has been received by the specialty pharmacy from one of its customers, which include pharmacy benefit managers, insurance companies, government programs and group purchasing organizations. The majority of the Company’s receivables from collaborations are due from the U.S. Government, for which there is no assumed credit risk. |
Recently Adopted Accounting Pronouncements and New Accounting Pronouncements Not Yet Adopted | Recently Adopted Accounting Pronouncements In December 2020, the FASB issued ASU No. 2019-12 (ASC Topic 740), Simplifying the Accounting for Income Taxes . ASU 2019-12 simplifies accounting for income taxes by removing certain exceptions to the general principles and clarifying existing guidance. This standard is effective for fiscal years, and interim periods within those years, beginning after December 15, 2020. The Company adopted ASU No. 2019-12 as of January 1, 2021. The adoption of this standard did not have a material impact to the Company’s financial position, results of operations or cash flows. New Accounting Pronouncements Not Yet Adopted The Company has reviewed new accounting pronouncements that were issued as of December 31, 2023 and does not believe that these pronouncements are either applicable to the Company, or that they will have a material impact on its financial position or results of operations. |
Significant Accounting Polici_3
Significant Accounting Policies and Concentrations of Risk (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Accounting Policies [Abstract] | |
Summary of Revenues | The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands): Years Ended December 31, 2023 2022 2021 Product sales, net 324,696 267,710 136,350 Collaborative and other revenues 6,716 3,117 20,820 Total revenues $ 331,412 $ 270,827 $ 157,170 The Company recorded the following revenues for the years ended December 31 (in thousands): Years Ended December 31, 2023 2022 2021 ORLADEYO: U.S. $ 288,361 $ 226,358 $ 117,836 Outside of U.S. 37,629 25,275 4,719 Total ORLADEYO 325,990 251,633 122,555 Other revenues 5,422 19,194 34,615 Total revenues $ 331,412 $ 270,827 $ 157,170 |
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share | The Company excluded the following potential common shares, presented based on amounts outstanding as of December 31, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect: December 31, 2023 2022 Outstanding stock options 41,032 36,520 Unvested restricted stock unit awards 6,507 4,747 Warrants to purchase common stock — 15,023 Total 47,539 56,290 |
Revenue (Tables)
Revenue (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Revenue Recognition [Abstract] | |
Summary of Revenues | The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands): Years Ended December 31, 2023 2022 2021 Product sales, net 324,696 267,710 136,350 Collaborative and other revenues 6,716 3,117 20,820 Total revenues $ 331,412 $ 270,827 $ 157,170 The Company recorded the following revenues for the years ended December 31 (in thousands): Years Ended December 31, 2023 2022 2021 ORLADEYO: U.S. $ 288,361 $ 226,358 $ 117,836 Outside of U.S. 37,629 25,275 4,719 Total ORLADEYO 325,990 251,633 122,555 Other revenues 5,422 19,194 34,615 Total revenues $ 331,412 $ 270,827 $ 157,170 |
Investments (Tables)
Investments (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Investments, All Other Investments [Abstract] | |
Schedule of Fair Value, Assets and Liabilities Measured on Recurring Basis | Assets measured at fair value on a recurring basis were as follows (in thousands): December 31, 2023 Quoted Price in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Total Assets: Obligations of U.S. Government and its agencies $ — $ 277,358 $ — $ 277,358 Certificates of deposit — 986 — 986 Total assets $ — $ 278,344 $ — $ 278,344 December 31, 2022 Quoted Price in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) Total Assets: Obligations of U.S. Government and its agencies $ — $ 129,371 $ — $ 129,371 Corporate debt securities — 6,092 — 6,092 Certificates of deposit — 2,157 — 2,157 Total assets $ — $ 137,620 $ — $ 137,620 |
Fair Value of the Company's Investments by Type | December 31, 2023 Amortized Cost Accrued Interest Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Obligations of U.S. Government and its agencies $ 277,151 $ 121 $ 150 $ (64) $ 277,358 Certificates of deposit 980 14 — (8) 986 Total investments $ 278,131 $ 135 $ 150 $ (72) $ 278,344 December 31, 2022 Amortized Cost Accrued Interest Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value Obligations of U.S. Government and its agencies $ 129,940 $ 427 $ — $ (996) $ 129,371 Corporate debt securities 6,093 37 — (38) 6,092 Certificates of deposit 2,163 23 — (29) 2,157 Total investments $ 138,196 $ 487 $ — $ (1,063) $ 137,620 |
Schedule of the Maturity of Investments | The following table summarizes the scheduled maturity for the Company’s investments at December 31, 2023 and 2022 (in thousands): December 31, 2023 2022 Maturing in one year or less $ 278,344 $ 119,543 Maturing after one year through two years — 18,077 Total investments $ 278,344 $ 137,620 |
Trade Receivables (Tables)
Trade Receivables (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Receivables [Abstract] | |
Summary of Receivables | Receivables from collaborations were as follows (in thousands): December 31, 2023 Billed Unbilled Total Royalty receivables from partners $ — $ 2,296 $ 2,296 Total receivables from collaborators $ — $ 2,296 $ 2,296 December 31, 2022 Billed Unbilled Total U.S. Department of Health and Human Services, net $ 7,218 $ 284 $ 7,502 Royalty receivables from partners 741 — 741 Other collaborations — 25 25 Total receivables from collaborators $ 7,959 $ 309 $ 8,268 |
Inventory (Tables)
Inventory (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories | The Company’s inventories consisted of the following (in thousands): December 31, 2023 2022 Raw materials $ 6,449 $ 8,906 Work-in-process 17,591 14,990 Finished goods 6,242 4,814 Total inventory $ 30,282 $ 28,710 Reserves (1,599) (1,177) Total inventory, net $ 28,683 $ 27,533 |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property and Equipment | Property and equipment consisted of the following (in thousands): December 31, 2023 2022 Furniture and fixtures $ 1,384 $ 1,308 Office equipment 719 633 Software 1,252 1,501 Laboratory equipment 5,559 4,588 Leasehold improvements 10,206 10,137 Total property and equipment $ 19,120 $ 18,167 Less accumulated depreciation and amortization (11,210) (9,550) Property and equipment, net $ 7,910 $ 8,617 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Accounts Payable and Accrued Liabilities [Abstract] | |
Schedule of Accrued Liabilities | Accrued expenses consisted of the following (in thousands): December 31, 2023 2022 Compensation and benefits $ 36,518 $ 22,125 Revenue-related reserves for discounts and allowances 26,509 14,332 Royalties payable 18,524 7,700 Development costs 13,677 30,360 Inventory 190 4,193 Other 7,464 8,855 Total accrued expenses $ 102,882 $ 87,565 |
Royalty Financing Obligations (
Royalty Financing Obligations (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Advance Royalties [Abstract] | |
Schedule of Royalty Financing Obligations | The following table shows the activity within the Royalty financing obligations account (in thousands) as well as the effective interest rate as of December 31, 2023: 2020 RPI Royalty Agreement 2021 RPI Royalty Agreement OMERS Royalty Agreement Total Balance as of December 31, 2020 $ 124,717 $ — $ — $ 124,717 2021 Royalty sale: Royalty financing obligations, net of issuance costs — 150,833 147,309 298,142 Non-cash Interest expense on Royalty financing obligations 33,308 2,897 1,465 37,670 Royalty revenues paid and payable (10,801) (353) — (11,154) Balance as of December 31, 2021 $ 147,224 $ 153,377 $ 148,774 $ 449,375 Deferred financing costs — (34) — (34) Non-cash Interest expense on Royalty financing obligations 39,994 22,239 14,249 76,482 Royalty revenues paid and payable (22,237) (1,931) — (24,168) Balance as of December 31, 2022 $ 164,981 $ 173,651 $ 163,023 $ 501,655 Non-cash Interest expense on Royalty financing obligations 38,267 14,188 17,901 70,356 Royalty revenues paid and payable (28,768) (2,494) (9,150) (40,412) Balance as of December 31, 2023 $ 174,480 $ 185,345 $ 171,774 $ 531,599 Effective interest rate 22.3 % — % 10.0 % |
Lease Obligations (Tables)
Lease Obligations (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Leases [Abstract] | |
Lease, Cost | Lease expense under operating and finance leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Operating lease expense $ 2,018 $ 1,578 $ 1,660 Finance lease expense: Amortization of right-of-use assets $ 1,212 $ 816 113 Interest on lease liabilities $ 201 $ 138 22 Total finance lease expense $ 1,413 $ 954 $ 135 Supplemental cash flow information related to leases was as follows (in thousands): Years Ended December 31, 2023 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows for finance leases $ 201 $ 898 $ 125 Operating cash flows for operating leases $ 1,920 $ 1,555 $ 1,490 Financing cash flows for finance leases $ 1,165 $ — $ — Operating lease assets obtained in exchange for operating lease liabilities: $ 4,695 $ 755 $ 1,536 Finance lease assets obtained in exchange for finance lease liabilities: $ 2,971 $ 1,302 $ 2,190 Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities: $ 924 $ — $ — |
Other Supplemental Information Related to Leases | Other supplemental information related to leases was as follows: As of December 31, 2023 2022 Weighted average remaining lease term: Operating leases 9.7 years 10.6 years Finance leases 3.0 years 3.0 years Weighted average discount rate: Operating leases 10.88 % 12.54 % Finance leases 7.46 % 5.00 % |
Lessee, Operating Lease, Assets and Liabilities | The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Operating lease assets: Operating lease assets, net Other Assets $ 8,682 $ 4,242 Operating lease liabilities: Current operating lease liabilities Operating lease liabilities – current liabilities $ 1,058 $ 1,516 Non-current operating lease liabilities Operating lease liabilities – long-term liabilities 8,390 4,027 Total operating lease liabilities $ 9,448 $ 5,543 The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands): As of December 31, Balance Sheet Location 2023 2022 Finance lease assets: Finance lease assets, net Other Assets $ 4,322 $ 2,564 Finance lease liabilities: Current finance lease liabilities Finance lease liabilities – current liabilities $ 1,590 $ 853 Non-current finance lease liabilities Finance lease liabilities – long-term liabilities 2,845 1,777 Total finance lease liabilities $ 4,435 $ 2,630 |
Maturities of Operating Lease Liabilities | Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands): Operating Leases Finance Leases 2024 $ 2,014 $ 1,857 2025 1,701 1,732 2026 1,509 899 2027 1,463 437 2028 1,312 27 Thereafter 7,952 — Total lease payments 15,951 4,952 Less imputed interest (6,503) (517) Total $ 9,448 $ 4,435 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Equity [Abstract] | |
Schedule of Stock by Class | The Company had reserved shares of common stock for issuance as follows (in thousands): December 31, 2023 2022 Shares reserved for exercises of outstanding stock options 41,032 36,520 Shares reserved for vesting of restricted stock units 6,507 4,747 Shares reserved for exercises of warrants — 15,023 Shares reserved for future issuance under the Stock Incentive Plan 3,376 4,206 Shares reserved for future issuance under the Inducement Equity Incentive Plan 1,651 947 Shares reserved for future issuance under the Employee Stock Purchase Plan 5,454 5,792 Total shares reserved for future issuance 58,020 67,235 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Disclosure of Share-Based Compensation Arrangements by Share-Based Payment Award | The Company recorded the following stock-based compensation expense (in thousands): Years Ended December 31, 2023 2022 2021 Incentive Plan $ 44,581 $ 36,716 $ 27,062 Inducement Plan 9,958 6,550 6,055 ESPP 1,076 1,435 1,523 Stock-based compensation expense $ 55,615 $ 44,701 $ 34,640 The following table summarizes stock option activity under the Incentive Plan: Shares Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value Outstanding at December 31, 2022 31,179 $ 8.56 Granted 6,625 6.67 Exercised (935) 5.21 $ 3,601 Cancelled or Forfeited (1,364) 10.00 Outstanding at December 31, 2023 35,505 $ 8.24 6.85 $ 12,280 Exercisable at December 31, 2023 21,008 $ 7.87 5.44 $ 12,280 Vested and expected to vest at December 31, 2023 33,557 $ 8.19 6.77 $ 12,280 The following table summarizes stock option activity under the Inducement Plan: Shares Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value Outstanding at December 31, 2022 5,341 $ 8.80 Granted 1,196 8.15 Exercised (344) 3.70 $ 1,803 Cancelled or Forfeited (666) 10.47 Outstanding at December 31, 2023 5,527 $ 8.77 7.76 $ 4,463 Exercisable at December 31, 2023 2,448 $ 7.17 6.89 $ 3,434 Vested and expected to vest at December 31, 2023 5,122 $ 8.68 7.72 $ 4,291 |
Stock-Based Compensation Allocation | Total stock-based compensation was allocated as follows: Years Ended December 31, 2023 2022 2021 Research and development $ 29,377 $ 24,936 $ 20,179 Selling, general and administrative 26,238 19,765 14,461 Total stock-based compensation expense $ 55,615 $ 44,701 $ 34,640 |
Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans | Stock Incentive Plan The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Incentive Plan during the years ended December 31, 2023, 2022, and 2021: Years Ended December 31, 2023 2022 2021 Expected Life in Years 5.7 5.5 5.5 Expected Volatility 82.5 % 84.2 % 84.2 % Expected Dividend Yield 0.0 % 0.0 % 0.0 % Risk-Free Interest Rate 3.9 % 3.6 % 1.1 % Weighted average grant date fair value per share $ 4.75 $ 7.57 $ 7.93 Inducement Equity Incentive Plan The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the years ended December 31, 2023, 2022, and 2021: Years Ended December 31, 2023 2022 2021 Expected Life in Years 5.6 5.5 5.5 Expected Volatility 83.5 % 84.2 % 84.2 % Expected Dividend Yield 0.0 % 0.0 % 0.0 % Risk-Free Interest Rate 4.0 % 3.2 % 0.9 % Weighted average grant date fair value per share $ 5.79 $ 9.54 $ 9.65 |
Share-Based Payment Arrangement, Restricted Stock and Restricted Stock Unit, Activity | The following table summarizes restricted stock unit activity under the Incentive Plan: Shares Weighted Average Grant Date Fair Value Unvested at December 31, 2022 4,159 $ 10.69 Granted 3,106 6.71 Vested (1,137) 11.04 Forfeited (536) 7.91 Unvested at December 31, 2023 5,592 $ 8.67 The following table summarizes restricted stock unit activity under the Inducement Plan: Shares Weighted Average Grant Date Fair Value Unvested at December 31, 2022 588 $ 13.14 Granted 581 7.81 Vested (139) 13.13 Forfeited (115) 11.98 Unvested at December 31, 2023 915 $ 9.90 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2023 | |
Income Tax Disclosure [Abstract] | |
Schedule Of Loss Before Provision For Income Taxes | The components of loss before provision for income taxes were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Domestic $ (206,674) $ (225,127) $ (159,632) Foreign (19,555) (19,256) (22,177) Loss before provision for income taxes $ (226,229) $ (244,383) $ (181,809) |
Schedule of Components of Income Tax (Benefit) Expense | The components of the (benefit) expense for income taxes were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Current expense (benefit) provision: U.S. Federal and state $ (45) $ 2,430 $ 2,179 Foreign 1,037 292 233 Total current expense provision 992 2,722 2,412 Deferred expense (benefit) provision: U.S. Federal and state (120) 11 (159) Foreign (562) — — Total deferred expense provision (682) 11 (159) Total expense provision $ 310 $ 2,733 $ 2,253 |
Summary Of The Differences Between The Company's Effective Tax Rate And Statutory Tax Rate | The differences between the Company’s effective tax rate and the statutory tax rate in 2023, 2022, and 2021 were as follows (in thousands): Years Ended December 31, 2023 2022 2021 Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021) $ (47,328) $ (51,321) $ (38,175) State and local income taxes net of federal tax benefit (3,477) (1,816) (2,288) Permanent items 3,015 (1,608) (1,343) Expiration of attribute carryforwards — — (1,057) Research and development tax credits (3,301) (9,793) (5,994) Foreign rate differential 2,255 1,862 1,940 Other 1,656 (5,485) 1,216 Change in valuation allowance 47,490 70,894 47,954 Income tax expense $ 310 $ 2,733 $ 2,253 |
Schedule Of Unrecognized Tax Benefits | A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): 2023 2022 Balance at January 1, $ 13,844 $ 9,729 Additions to current period tax positions 825 4,115 Reductions to prior period tax provisions (307) — Balance at December 31, $ 14,362 $ 13,844 |
Schedule Of Deferred Tax Assets And Liabilities | Significant components of the Company’s deferred tax assets and liabilities are as follows (in thousands): December 31, 2023 2022 Deferred tax assets: Net federal and state operating losses $ 108,753 $ 101,600 Research and development credits 88,390 86,321 Royalty income 122,391 115,554 Stock-based compensation 26,113 19,374 Capitalized R&D 75,081 62,794 Leasing obligations 3,076 1,842 Other 17,515 4,354 Total deferred tax assets 441,319 391,839 Deferred tax liabilities: Fixed assets (678) (717) Right of use asset (2,872) (1,525) Total deferred tax liabilities (3,550) (2,242) Valuation allowance (437,098) (389,608) Net deferred tax assets (liabilities) $ 671 $ (11) |
Significant Accounting Polici_4
Significant Accounting Policies and Concentrations of Risk Textual (Details) - USD ($) | 12 Months Ended | |||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Apr. 17, 2023 | |
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Restricted cash | $ 1,804,000 | $ 1,472,000 | ||
Long-term investment maturity, minimum (in months) | 12 months | |||
Advertising expense | $ 14,404,000 | $ 14,891,000 | $ 5,705,000 | |
Antidilutive securities excluded from computation of earnings per share, amount (in shares) | 47,539,000 | 56,290,000 | ||
Reclassification from accumulated other comprehensive income, current period, before tax | $ 0 | $ 0 | $ 1,000 | |
Computer Equipment | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Property, plant and equipment, useful life (in years) | 3 years | |||
Laboratory Equipment, Office Equipment and Software | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Property, plant and equipment, useful life (in years) | 5 years | |||
Furniture and fixtures | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Property, plant and equipment, useful life (in years) | 7 years | |||
Royalty Receivable | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Restricted cash | $ 1,804,000 | 1,472,000 | ||
Collateral for Credit | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Restricted cash | $ 1,493,000 | $ 1,449,000 | ||
Maximum | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Maturity period of high quality marketable securities (in years) | 3 years | |||
Average maturity period of high quality marketable securities (in months) | 12 months | |||
Maturity period of short term investment (in months) | 12 months | |||
Pharmakon Advisors, LP | Term Loan Tranche A | Line of Credit | ||||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||||
Interest expense, debt, total | $ 27,326,000 | |||
Debt instrument, face amount | $ 300,000,000 |
Significant Accounting Polici_5
Significant Accounting Policies and Concentrations of Risk - Summary of Revenues (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Significant Accounting Policies and Concentrations of Risk [Line Items] | |||
Revenues | $ 331,412 | $ 270,827 | $ 157,170 |
Revenues | |||
Significant Accounting Policies and Concentrations of Risk [Line Items] | |||
Revenues | 324,696 | 267,710 | 136,350 |
Collaborative and other revenues | |||
Significant Accounting Policies and Concentrations of Risk [Line Items] | |||
Revenues | $ 6,716 | $ 3,117 | $ 20,820 |
Significant Accounting Polici_6
Significant Accounting Policies and Concentrations of Risk - Net Loss Per Share (Details) - shares | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount (in shares) | 47,539,000 | 56,290,000 |
Share-based Payment Arrangement, Option | ||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount (in shares) | 41,032,000 | 36,520,000 |
Restricted Stock Units (RSUs) | ||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount (in shares) | 6,507,000 | 4,747,000 |
Warrant | ||
Significant Accounting Policies and Concentrations of Risk [Line Items] | ||
Antidilutive securities excluded from computation of earnings per share, amount (in shares) | 0 | 15,023,000 |
Revenue - Summary of Revenues (
Revenue - Summary of Revenues (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Disaggregation of Revenue [Line Items] | |||
Revenues | $ 331,412 | $ 270,827 | $ 157,170 |
Revenues | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | 324,696 | 267,710 | 136,350 |
Revenues | Geographic Distribution, Domestic | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | 288,361 | 226,358 | 117,836 |
Revenues | Geographic Distribution, Foreign | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | 37,629 | 25,275 | 4,719 |
ORLADEYO | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | 325,990 | 251,633 | 122,555 |
Other revenues | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | $ 5,422 | $ 19,194 | $ 34,615 |
Revenue - Narrative (Details)
Revenue - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Disaggregation of Revenue [Line Items] | |||
Revenues | $ 331,412 | $ 270,827 | $ 157,170 |
Royalty | Torii Pharmaceutical Co | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | $ 2,178 | $ 1,944 | 690 |
Milestone | |||
Disaggregation of Revenue [Line Items] | |||
Revenues | $ 15,000 |
Investments - Fair Value, Recur
Investments - Fair Value, Recurring Basis (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Marketable Securities [Line Items] | ||
Estimated Fair Value | $ 278,344 | $ 137,620 |
Obligations of U.S. Government and its agencies | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 277,358 | 129,371 |
Corporate debt securities | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 6,092 | |
Certificates of deposit | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 986 | 2,157 |
Fair Value, Inputs, Level 1 | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | 0 |
Fair Value, Inputs, Level 1 | Obligations of U.S. Government and its agencies | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | 0 |
Fair Value, Inputs, Level 1 | Corporate debt securities | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | |
Fair Value, Inputs, Level 1 | Certificates of deposit | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | 0 |
Fair Value, Inputs, Level 2 | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 278,344 | 137,620 |
Fair Value, Inputs, Level 2 | Obligations of U.S. Government and its agencies | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 277,358 | 129,371 |
Fair Value, Inputs, Level 2 | Corporate debt securities | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 6,092 | |
Fair Value, Inputs, Level 2 | Certificates of deposit | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 986 | 2,157 |
Fair Value, Inputs, Level 3 | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | 0 |
Fair Value, Inputs, Level 3 | Obligations of U.S. Government and its agencies | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | 0 |
Fair Value, Inputs, Level 3 | Corporate debt securities | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | 0 | |
Fair Value, Inputs, Level 3 | Certificates of deposit | ||
Marketable Securities [Line Items] | ||
Estimated Fair Value | $ 0 | $ 0 |
Investments - Narrative (Detail
Investments - Narrative (Details) | Dec. 31, 2023 USD ($) securities |
Investments, All Other Investments [Abstract] | |
Securities | securities | 11 |
Estimated fair market value | $ | $ 18,513,000 |
Investments- Fair Value of the
Investments- Fair Value of the Company's Investments by Type (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Marketable Securities [Line Items] | ||
Amortized Cost | $ 278,131 | $ 138,196 |
Accrued Interest | 135 | 487 |
Gross Unrealized Gains | 150 | 0 |
Gross Unrealized Losses | (72) | (1,063) |
Estimated Fair Value | $ 278,344 | $ 137,620 |
Debt Securities, Available-for-Sale, Accrued Interest, after Allowance for Credit Loss, Statement of Financial Position [Extensible Enumeration] | Investments, Long-term investments | Investments, Long-term investments |
Obligations of U.S. Government and its agencies | ||
Marketable Securities [Line Items] | ||
Amortized Cost | $ 277,151 | $ 129,940 |
Accrued Interest | 121 | 427 |
Gross Unrealized Gains | 150 | 0 |
Gross Unrealized Losses | (64) | (996) |
Estimated Fair Value | 277,358 | 129,371 |
Certificates of deposit | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 980 | 2,163 |
Accrued Interest | 14 | 23 |
Gross Unrealized Gains | 0 | 0 |
Gross Unrealized Losses | (8) | (29) |
Estimated Fair Value | $ 986 | 2,157 |
Corporate debt securities | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 6,093 | |
Accrued Interest | 37 | |
Gross Unrealized Gains | 0 | |
Gross Unrealized Losses | (38) | |
Estimated Fair Value | $ 6,092 |
Investment - Schedule of Maturi
Investment - Schedule of Maturity of Investments (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Investments, All Other Investments [Abstract] | ||
Maturing in one year or less | $ 278,344 | $ 119,543 |
Maturing after one year through two years | 0 | 18,077 |
Estimated Fair Value | $ 278,344 | $ 137,620 |
Trade Receivables Textual (Deta
Trade Receivables Textual (Details) - USD ($) | Dec. 31, 2023 | Dec. 31, 2022 |
Trade Accounts Receivable | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Accounts receivable, after allowance for credit loss, total | $ 288,000 | $ 288,000 |
ORLADEYO | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Allowance | 0 | |
ORLADEYO | Trade Accounts Receivable | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Accounts receivable, after allowance for credit loss, total | 54,149,000 | 41,508,000 |
RAPIVAB | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Allowance | 0 | 0 |
RAPIVAB | Trade Accounts Receivable | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Accounts receivable, after allowance for credit loss, total | $ 505,000 | $ 823,000 |
Trade Receivables - Summary of
Trade Receivables - Summary of Receivables (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | $ 2,296 | $ 8,268 |
Billed | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 0 | 7,959 |
Unbilled | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 2,296 | 309 |
U.S. Department of Health and Human Services, net | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 7,502 | |
U.S. Department of Health and Human Services, net | Billed | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 7,218 | |
U.S. Department of Health and Human Services, net | Unbilled | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 284 | |
Royalty receivables from partners | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 2,296 | 741 |
Royalty receivables from partners | Billed | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 0 | 741 |
Royalty receivables from partners | Unbilled | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | $ 2,296 | 0 |
Other collaborations | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 25 | |
Other collaborations | Billed | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | 0 | |
Other collaborations | Unbilled | ||
Accounts, Notes, Loans and Financing Receivable [Line Items] | ||
Receivables | $ 25 |
Inventory - Summary of Inventor
Inventory - Summary of Inventories (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 6,449 | $ 8,906 |
Work-in-process | 17,591 | 14,990 |
Finished goods | 6,242 | 4,814 |
Total inventory | 30,282 | 28,710 |
Reserves | (1,599) | (1,177) |
Total inventory, net | $ 28,683 | $ 27,533 |
Property and Equipment - Proper
Property and Equipment - Property and Equipment (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 19,120 | $ 18,167 |
Less accumulated depreciation and amortization | (11,210) | (9,550) |
Property and equipment, net | 7,910 | 8,617 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 1,384 | 1,308 |
Office equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 719 | 633 |
Software | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 1,252 | 1,501 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | 5,559 | 4,588 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment | $ 10,206 | $ 10,137 |
Property and Equipment Textual
Property and Equipment Textual (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation and amortization | $ 1,655 | $ 1,437 | $ 777 |
Loss on impairment | 1,548 | $ 0 | $ 0 |
Contract termination | $ 440 |
Accrued Expenses - Accrued Expe
Accrued Expenses - Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Accounts Payable and Accrued Liabilities [Abstract] | ||
Compensation and benefits | $ 36,518 | $ 22,125 |
Revenue-related reserves for discounts and allowances | 26,509 | 14,332 |
Royalties payable | 18,524 | 7,700 |
Development costs | 13,677 | 30,360 |
Inventory | 190 | 4,193 |
Other | 7,464 | 8,855 |
Total accrued expenses | $ 102,882 | $ 87,565 |
Royalty Financing Obligations_2
Royalty Financing Obligations (Details) - USD ($) | 12 Months Ended | ||||||
Nov. 19, 2021 | Dec. 07, 2020 | Mar. 09, 2011 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 01, 2020 | |
Royalties Monetizations [Line Items] | |||||||
(Loss) gain on extinguishment of debt | $ (29,019,000) | $ 0 | $ 55,838,000 | ||||
RPI 2019 Intermediate Finance Trust | |||||||
Royalties Monetizations [Line Items] | |||||||
Shares issued, common stock, premium | $ 4,269,000 | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 20% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Under $350,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 8.75% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Between $350,000 and $550,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 2.75% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Over $550,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 0% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Under $150,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 20% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Between $150,000 and $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 10% | ||||||
RPI 2019 Intermediate Finance Trust | ORLADEYO | Annual Net Sales Over $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 0% | ||||||
RPI 2019 Intermediate Finance Trust | BCX9930 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, maximum tiered profit share percentage on permitted sales in other markets | 3% | ||||||
RPI 2019 Intermediate Finance Trust | BCX9930 | Annual Net Sales Over $3 Billions | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, annual net sales payment threshold | $ 3,000,000,000 | ||||||
RPI 2019 Intermediate Finance Trust | Future Royalties Payable | |||||||
Royalties Monetizations [Line Items] | |||||||
Proceeds from issuance of debt | $ 125,000,000 | ||||||
Debt issuance costs, net, total | $ 8,532,000 | $ 8,532,000 | |||||
RPI 2021 and 2020 Intermediate Finance Trust | Future Royalties Payable | |||||||
Royalties Monetizations [Line Items] | |||||||
Proceeds from issuance of debt | $ 150,000,000 | ||||||
The 2021 RPI Royalty Purchase Agreement | Annual Net Sales Under $350,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 0.75% | ||||||
The 2021 RPI Royalty Purchase Agreement | Annual Net Sales Between $350,000 and $550,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 1.75% | ||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 0% | ||||||
The 2021 RPI Royalty Purchase Agreement | Annual Net Sales Under $150,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 3% | ||||||
The 2021 RPI Royalty Purchase Agreement | Annual Net Sales Between $150,000 and $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 2% | ||||||
The 2021 RPI Royalty Purchase Agreement | Annual Net Sales Over $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 0% | ||||||
The 2021 RPI Royalty Purchase Agreement | BCX9930 | Annual Net Sales Under $1.5 Million | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 3% | ||||||
The 2021 RPI Royalty Purchase Agreement | BCX9930 | Annual Net Sales Between $1.5 And $3 Million | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in key territories | 2% | ||||||
The 2021 RPI Royalty Purchase Agreement | BCX9930 | Annual Net Sales Over $3 Billions | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 0% | ||||||
OMERS Capital Markets | ORLADEYO | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 20% | ||||||
Royalty purchase agreement, royalties, percentage of annual net sales in other markets | 10% | ||||||
Royalty purchase agreement, royalties, percentage of purchase price | 155% | ||||||
Royalty purchase agreement, royalties, purchase price, amount | $ 150,000,000 | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Under $350,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales | 7.50% | ||||||
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales | 10% | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Between $350,000 and $550,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales | 6% | ||||||
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales | 3% | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Over $550,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of annual net sales | 0% | ||||||
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales | 0% | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Under $150,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 20% | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Between $150,000 and $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 10% | ||||||
OMERS Capital Markets | ORLADEYO | Annual Net Sales Over $230,000 | |||||||
Royalties Monetizations [Line Items] | |||||||
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets | 0% | ||||||
OMERS Royalty Agreement | |||||||
Royalties Monetizations [Line Items] | |||||||
Effective interest rate | 10% | 10.60% | |||||
2020 RPI Royalty Agreement | |||||||
Royalties Monetizations [Line Items] | |||||||
Effective interest rate | 22.30% | 22.40% | |||||
2021 RPI Royalty Agreement | |||||||
Royalties Monetizations [Line Items] | |||||||
Effective interest rate | 0% | 13.10% | |||||
JPR Royalty Sub LLC | PhaRMA Notes | |||||||
Royalties Monetizations [Line Items] | |||||||
Private placement of senior secured notes | $ 30,000,000 | ||||||
Debt instrument, interest rate, stated percentage | 14% | ||||||
Secured debt, total | $ 30,000,000 | ||||||
Interest payable | $ 20,614,000 | ||||||
(Loss) gain on extinguishment of debt | 25,838,000 | ||||||
JPR Royalty Sub LLC | PhaRMA Notes | Fair Value, Inputs, Level 2 | |||||||
Royalties Monetizations [Line Items] | |||||||
Notes payable, fair value disclosure | $ 30,000,000 | ||||||
JPR Royalty Sub LLC | Royalty Monetization | |||||||
Royalties Monetizations [Line Items] | |||||||
Debt instrument, face amount | $ 30,000,000 | ||||||
Prepayment and repayment fees on Athyrium secured term loans | $ 22,691,000 |
Royalty Financing Obligations -
Royalty Financing Obligations - Schedule of Royalty Financing Obligations (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Royalties Monetizations [Line Items] | |||
Balance | $ 501,655 | $ 449,375 | $ 124,717 |
Long-term debt, total | 298,142 | ||
Non-cash Interest expense on Royalty financing obligations | 70,356 | 76,482 | 37,670 |
Royalty revenues paid and payable | (40,412) | (24,168) | (11,154) |
Deferred financing costs | (34) | ||
Balance | 531,599 | 501,655 | 449,375 |
2020 RPI Royalty Agreement | |||
Royalties Monetizations [Line Items] | |||
Balance | 164,981 | 147,224 | 124,717 |
Non-cash Interest expense on Royalty financing obligations | 38,267 | 39,994 | 33,308 |
Royalty revenues paid and payable | (28,768) | (22,237) | (10,801) |
Balance | $ 174,480 | $ 164,981 | 147,224 |
Effective interest rate | 22.30% | 22.40% | |
2021 RPI Royalty Agreement | |||
Royalties Monetizations [Line Items] | |||
Balance | $ 173,651 | $ 153,377 | |
Long-term debt, total | 150,833 | ||
Non-cash Interest expense on Royalty financing obligations | 14,188 | 22,239 | 2,897 |
Royalty revenues paid and payable | (2,494) | (1,931) | (353) |
Deferred financing costs | (34) | ||
Balance | $ 185,345 | $ 173,651 | 153,377 |
Effective interest rate | 0% | 13.10% | |
OMERS Royalty Agreement | |||
Royalties Monetizations [Line Items] | |||
Balance | $ 163,023 | $ 148,774 | |
Long-term debt, total | 147,309 | ||
Non-cash Interest expense on Royalty financing obligations | 17,901 | 14,249 | 1,465 |
Royalty revenues paid and payable | (9,150) | ||
Balance | $ 171,774 | $ 163,023 | $ 148,774 |
Effective interest rate | 10% | 10.60% |
Debt Textual (Details)
Debt Textual (Details) | 12 Months Ended | |||||
Apr. 17, 2023 USD ($) | Dec. 07, 2020 USD ($) | Dec. 31, 2023 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Jul. 29, 2022 USD ($) | |
Debt Instrument [Line Items] | ||||||
Net proceeds from secured term loans | $ 300,000,000 | $ 73,072,000 | $ 0 | |||
Long-term debt, total | 298,142,000 | |||||
Gain (loss) on extinguishment of debt | 29,019,000 | 0 | (55,838,000) | |||
Pharmakon Advisors, LP | ||||||
Debt Instrument [Line Items] | ||||||
Capitalized interest rate | 0.50 | |||||
Pharmakon Advisors, LP | Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate | ||||||
Debt Instrument [Line Items] | ||||||
Variable rate floor | 0.0175 | |||||
Debt instrument, basis spread on variable rate | 7% | |||||
Pharmakon Advisors, LP | Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate | PIK Interest Payment is Made | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, basis spread on variable rate | 7.25% | |||||
Pharmakon Advisors, LP | Line of Credit | ||||||
Debt Instrument [Line Items] | ||||||
Periodic payment | $ 25,000,000 | |||||
Debt instrument, prepayment fee, percentage of principal voluntary payments | 3% | |||||
Pharmakon Advisors, LP | Line of Credit | Between the Second and Third Anniversaries | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, prepayment fee, percentage of principal voluntary payments | 3% | |||||
Pharmakon Advisors, LP | Line of Credit | After Third Anniversary | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, prepayment fee, percentage of principal voluntary payments | 2% | |||||
Pharmakon Advisors, LP | Line of Credit | After Fourth Anniversary | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, prepayment fee, percentage of principal voluntary payments | 1% | |||||
Athyrium | Senior Credit Facility | ||||||
Debt Instrument [Line Items] | ||||||
Repayments of lines of credit | $ 241,787,000 | |||||
MidCap Financial Services, LLC | Senior Credit Facility | ||||||
Debt Instrument [Line Items] | ||||||
Repayments of lines of credit | $ 43,298,000 | |||||
Loan Agreement | Pharmakon Advisors, LP | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | 450,000,000 | |||||
Net proceeds from secured term loans | 25,805,000 | |||||
Term Loan Tranche A | Pharmakon Advisors, LP | Line of Credit | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | $ 300,000,000 | |||||
Net proceeds from secured term loans | $ 300,000,000 | |||||
Effective interest rate | 13.30% | |||||
Interest expense, debt, total | $ 27,326,000 | |||||
Periodic payment, interest | 13,663,000 | |||||
Fair value disclosure | 313,663,000 | |||||
Debt instrument, unamortized discount (premium) and debt issuance costs, net, total | 11,147,000 | |||||
Amortization of debt issuance costs | 715,000 | |||||
Debt instrument, term (month) | 18 months | |||||
Term Loan Tranche A | Pharmakon Advisors, LP | Line of Credit | PIK Interest Payment is Made | ||||||
Debt Instrument [Line Items] | ||||||
Percent of interest payment | 0.50 | |||||
Term Loan Tranche B | Pharmakon Advisors, LP | Line of Credit | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | $ 50,000,000 | |||||
Term Loan Tranche C | Pharmakon Advisors, LP | Line of Credit | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | 50,000,000 | |||||
Term Loan Tranche D | Pharmakon Advisors, LP | Line of Credit | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | 50,000,000 | |||||
Credit Agreement | Athyrium | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | $ 200,000,000 | |||||
Debt instrument, LIBOR floor | 1.75% | |||||
Debt instrument, LIBOR cap | 3.50% | |||||
Debt instrument, exit fee, percentage of principal | 2% | |||||
Debt instrument, commitment fee percentage | 1% | |||||
Paid-in-kind interest | 8,476,000 | 23,387,000 | ||||
Long-term debt, total | 240,452,000 | |||||
Interest expense, long-term debt | $ 1,335,000 | |||||
Debt instrument, term (month) | 17 days | |||||
Debt instrument, fee amount | $ 17,261,000 | |||||
Debt issuance costs, net, total | 11,758,000 | |||||
Gain (loss) on extinguishment of debt | $ 29,019,000 | |||||
Credit Agreement | Athyrium | London Interbank Offered Rate (LIBOR) | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, basis spread on variable rate | 8.25% | |||||
Credit Agreement | Athyrium | London Interbank Offered Rate (LIBOR) | PIK Interest Payment is Made | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, basis spread on variable rate | 10.25% | |||||
Credit Agreement | Athyrium | Term A Loan | ||||||
Debt Instrument [Line Items] | ||||||
Net proceeds from secured term loans | $ 125,000,000 | |||||
Amortization of debt issuance costs | $ 1,069,000 | $ 916,000 | $ 531,000 | |||
Credit Agreement | Athyrium | Term B Loan | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | $ 25,000,000 | |||||
Credit Agreement | Athyrium | Term C Loan | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | 50,000,000 | |||||
Credit Agreement | Athyrium | Term B And C Loan | ||||||
Debt Instrument [Line Items] | ||||||
Debt instrument, face amount | 75,000,000 | |||||
Deferred debt fees and issuance costs | $ 3,428,000 | |||||
Credit Agreement | Athyrium | Term Loans | ||||||
Debt Instrument [Line Items] | ||||||
Effective interest rate | 13.71% | 12.87% |
Lease Obligations (Details Text
Lease Obligations (Details Textual) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Lessee, Lease, Description [Line Items] | |||
Accumulated amortization | $ 4,794 | $ 3,268 | |
Accumulated amortization | 2,293 | 1,081 | |
Operating cash flows for operating leases | 1,920 | 1,555 | $ 1,490 |
Principal payments on finance lease liabilities | $ 1,165 | $ 0 | $ 0 |
Minimum | |||
Lessee, Lease, Description [Line Items] | |||
Lessee, operating lease, term of contract (in years) | 1 year | ||
Maximum | |||
Lessee, Lease, Description [Line Items] | |||
Lessee, operating lease, term of contract (in years) | 3 years |
Lease Obligations - Aggregate L
Lease Obligations - Aggregate Lease Expense Under Operating Leases (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Leases [Abstract] | |||
Operating lease expense | $ 2,018 | $ 1,578 | $ 1,660 |
Amortization of right-of-use assets | 1,212 | 816 | 113 |
Interest on lease liabilities | 201 | 138 | 22 |
Total finance lease expense | $ 1,413 | $ 954 | $ 135 |
Lease Obligations - Other Suppl
Lease Obligations - Other Supplemental Information Related to Leases (Details) | Dec. 31, 2023 | Dec. 31, 2022 |
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract] | ||
Weighted average remaining lease term (in years) | 9 years 8 months 12 days | 10 years 7 months 6 days |
Weighted average discount rate: | 10.88% | 12.54% |
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract] | ||
Weighted average remaining lease term (in years) | 3 years | 3 years |
Weighted average discount rate: | 7.46% | 5% |
Lease Obligations -Balance Shee
Lease Obligations -Balance Sheets of the Company's Operating Leases (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Assets: | ||
Operating lease assets, net | $ 8,682 | $ 4,242 |
Operating Lease, Liability [Abstract] | ||
Operating lease liabilities | 1,058 | 1,516 |
Operating lease liabilities | 8,390 | 4,027 |
Total operating lease liabilities | $ 9,448 | $ 5,543 |
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration] | Other assets | Other assets |
Lease Obligations - Balance She
Lease Obligations - Balance Sheets of the Company's Finance Leases (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Finance Lease Assets [Abstract] | ||
Finance Lease, Right-of-Use Asset, after Accumulated Amortization | $ 4,322 | $ 2,564 |
Finance Lease, Liability [Abstract] | ||
Finance lease liabilities | 1,590 | 853 |
Finance lease liabilities | 2,845 | 1,777 |
Finance lease liabilities | $ 4,435 | $ 2,630 |
Finance Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration] | Other assets | Other assets |
Lease Obligations - Maturities
Lease Obligations - Maturities of Operating Lease Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract] | ||
2024 | $ 2,014 | |
2025 | 1,701 | |
2026 | 1,509 | |
2027 | 1,463 | |
2028 | 1,312 | |
Thereafter | 7,952 | |
Total lease payments | 15,951 | |
Less imputed interest | (6,503) | |
Total | 9,448 | $ 5,543 |
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract] | ||
2024 | 1,857 | |
2025 | 1,732 | |
2026 | 899 | |
2027 | 437 | |
2028 | 27 | |
Thereafter | 0 | |
Total lease payments | 4,952 | |
Less imputed interest | (517) | |
Total | $ 4,435 | $ 2,630 |
Lease Obligations - Supplementa
Lease Obligations - Supplemental Cash Flows (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Leases [Abstract] | |||
Operating cash flows for finance leases | $ 201 | $ 898 | $ 125 |
Operating cash flows for operating leases | 1,920 | 1,555 | 1,490 |
Financing cash flows for finance leases | 1,165 | 0 | 0 |
Operating lease assets obtained in exchange for operating lease liabilities: | 4,695 | 755 | 1,536 |
Finance lease assets obtained in exchange for finance lease liabilities: | 2,971 | 1,302 | 2,190 |
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities: | $ 924 | $ 0 | $ 0 |
Stockholders' Equity Textual (D
Stockholders' Equity Textual (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||||
Oct. 23, 2023 | Nov. 19, 2021 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Subsidiary, Sale of Stock [Line Items] | |||||
Issuance of common stock, net (in shares) | 3,846,000 | ||||
Sale of common stock, net | $ 0 | $ 0 | $ 50,000 | ||
Common Stock | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Stock issued during period (in shares) | 14,997,000 | ||||
Warrant | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Outstanding warrants (in shares) | 0 | ||||
Common Stock Purchase Agreement with RPI | |||||
Subsidiary, Sale of Stock [Line Items] | |||||
Issuance of common stock, net (in shares) | 3,846,000 | ||||
Shares issued, price per share (in dollars per share) | $ 13 | ||||
Sale of common stock, net | $ 50,000 | ||||
Shares issued, price per share, premium (in dollars per share) | $ 1.11 | ||||
Shares issued, price per share, closing price (in dollars per share) | $ 11.89 | ||||
Shares issued, common stock, premium | $ 4,269 |
Stockholders' Equity - Shares R
Stockholders' Equity - Shares Reserved For Future Issuance (Details) - shares shares in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 58,020 | 67,235 |
Incentive Plan | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 3,376 | 4,206 |
Inducement Plan | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 1,651 | 947 |
ESPP | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 5,454 | 5,792 |
Share-based Payment Arrangement, Option | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 41,032 | 36,520 |
Restricted Stock Units (RSUs) | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 6,507 | 4,747 |
Warrant | ||
Subsidiary, Sale of Stock [Line Items] | ||
Shares reserved for future issuance (in shares) | 0 | 15,023 |
Stock-Based Compensation Textua
Stock-Based Compensation Textual (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Jan. 31, 2022 shares | Dec. 31, 2014 shares | Dec. 31, 2023 USD ($) plan $ / shares shares | Dec. 31, 2022 USD ($) $ / shares shares | Dec. 31, 2021 USD ($) $ / shares shares | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Number of plans | plan | 3 | ||||
Total stock-based compensation expense | $ 55,615 | $ 44,701 | $ 34,640 | ||
Exercise of stock options, net (in shares) | shares | 1,276,000 | ||||
Employee stock purchase plan sales, net (in shares) | shares | 338,000 | 260,000 | 321,000 | ||
Share-based Payment Arrangement, Option | Non-employee Directors | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting period (year) | 1 year | ||||
Incentive Plan | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Total stock-based compensation expense | $ 44,581 | $ 36,716 | $ 27,062 | ||
Options, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 4.75 | $ 7.57 | $ 7.93 | ||
Total unrecognized compensation cost | $ 75,164 | ||||
Expected Dividend Yield | 0% | 0% | 0% | ||
Options, exercises in period, intrinsic value | $ 21,150 | $ 25,484 | |||
Options, vested in period, fair value | $ 33,731 | $ 28,916 | $ 20,510 | ||
Weighted average period for recognition | 1 year 10 months 24 days | ||||
Incentive Plan | Share-based Payment Arrangement, Option | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Options, grants in period, gross (in shares) | shares | 6,625,000 | ||||
Options, exercisable, weighted average remaining contractual term (year) | 5 years 5 months 8 days | ||||
Options, exercises in period, intrinsic value | $ 3,601 | ||||
Exercise of stock options, net (in shares) | shares | 935,000 | ||||
Options, aggregate intrinsic value | $ 12,280 | ||||
Number of stock option awards vested and expected to vest (in shares) | shares | 33,557,000 | ||||
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) | $ / shares | $ 8.19 | ||||
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) | 6 years 9 months 7 days | ||||
Incentive Plan | Share-based Payment Arrangement, Option | Maximum | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Options, exercisable, weighted average remaining contractual term (year) | 10 years | ||||
Incentive Plan | Share-based Payment Arrangement, Option | Vest 25% Each Year Until Fully Vested | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting rights, percentage | 25% | ||||
Award vesting period (year) | 4 years | ||||
Incentive Plan | Share-based Payment Arrangement, Option | Thirty-six Equal Monthly Installments | Non-employee Directors | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting period (year) | 3 years | ||||
Incentive Plan | Performance Shares | Vest Upon Successful Completion of Specific Development Milestones | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting rights, percentage | 85% | ||||
Options, grants in period, gross (in shares) | shares | 1,250,000 | ||||
Incentive Plan | Restricted Stock Units (RSUs) | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 6.71 | $ 11.20 | $ 11.36 | ||
Total unrecognized compensation cost | $ 40,981 | ||||
Weighted average period for recognition | 2 years | ||||
Incentive Plan | Performance-based Restricted Stock Units | Vest 25% Each Year Until Fully Vested | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Total stock-based compensation expense | $ 34 | $ 158 | |||
Award vesting rights, percentage | 25% | ||||
Incentive Plan | Performance-based Restricted Stock Units | Vest Upon Successful Completion of Specific Development Milestones | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Issued performance-based RSU award (in shares) | shares | 221 | 21,000 | |||
Incentive Plan | Performance-based Restricted Stock Units | Share-Based Payment Arrangement, Tranche One | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting rights, percentage | 50% | ||||
Incentive Plan | Performance-based Restricted Stock Units | Thirty-six Equal Monthly Installments | Non-employee Directors | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting period (year) | 3 years | ||||
Inducement Plan | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Total stock-based compensation expense | $ 9,958 | $ 6,550 | $ 6,055 | ||
Award vesting period (year) | 4 years | ||||
Options, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 5.79 | $ 9.54 | $ 9.65 | ||
Total unrecognized compensation cost | $ 16,216 | ||||
Expiration period (year) | 10 years | ||||
Expected Dividend Yield | 0% | 0% | 0% | ||
Options, exercises in period, intrinsic value | $ 3,710 | $ 6,700 | |||
Options, vested in period, fair value | $ 7,698 | $ 4,659 | $ 2,885 | ||
Weighted average period for recognition | 1 year 6 months | ||||
Inducement Plan | Vest 25% Each Year Until Fully Vested | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Award vesting rights, percentage | 25% | ||||
Inducement Plan | Share-based Payment Arrangement, Option | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Options, grants in period, gross (in shares) | shares | 1,196,000 | ||||
Options, exercisable, weighted average remaining contractual term (year) | 6 years 10 months 20 days | ||||
Expected Dividend Yield | 0% | ||||
Options, exercises in period, intrinsic value | $ 1,803 | ||||
Exercise of stock options, net (in shares) | shares | 344,000 | ||||
Options, aggregate intrinsic value | $ 3,434 | ||||
Number of stock option awards vested and expected to vest (in shares) | shares | 5,122,000 | ||||
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) | $ / shares | $ 8.68 | ||||
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) | 7 years 8 months 19 days | ||||
Inducement Plan | Restricted Stock Units (RSUs) | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Options, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 7.81 | $ 13.21 | $ 0 | ||
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 7.81 | ||||
Total unrecognized compensation cost | $ 7,044 | ||||
Weighted average period for recognition | 1 year 9 months 18 days | ||||
ESPP | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Total stock-based compensation expense | $ 1,076 | $ 1,435 | $ 1,523 | ||
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) | $ / shares | $ 3.82 | $ 6.02 | $ 2.80 | ||
Number of shares authorized (in shares) | shares | 7,975,000 | ||||
Number of shares available for grant (in shares) | shares | 5,454,000 | ||||
Percentage of salary to purchase common stock, maximum | 15% | ||||
Percentage of common stock shares, beginning | 85% | ||||
Percentage of common stock shares, ending | 85% | ||||
Maximum number of shares per employee (in shares) | shares | 3,000 | ||||
Maximum number of shares per employee, amount | $ 25 | ||||
Weighted average purchase price of shares purchased (in dollars per share) | $ / shares | $ 7.68 | $ 11.03 | $ 6.20 |
Stock-Based Compensation - Shar
Stock-Based Compensation - Share-Based Compensation (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 55,615 | $ 44,701 | $ 34,640 |
Incentive Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | 44,581 | 36,716 | 27,062 |
Inducement Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | 9,958 | 6,550 | 6,055 |
ESPP | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 1,076 | $ 1,435 | $ 1,523 |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock-based Compensation Allocation (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 55,615 | $ 44,701 | $ 34,640 |
Research and development | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | 29,377 | 24,936 | 20,179 |
Selling, general and administrative | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Total stock-based compensation expense | $ 26,238 | $ 19,765 | $ 14,461 |
Stock-Based Compensation - Weig
Stock-Based Compensation - Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans (Details) - $ / shares | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Incentive Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Expected Life in Years | 5 years 8 months 12 days | 5 years 6 months | 5 years 6 months |
Expected Volatility | 82.50% | 84.20% | 84.20% |
Expected Dividend Yield | 0% | 0% | 0% |
Risk-Free Interest Rate | 3.90% | 3.60% | 1.10% |
Options, grants in period, weighted average grant date fair value (in dollars per share) | $ 4.75 | $ 7.57 | $ 7.93 |
Inducement Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||
Expected Life in Years | 5 years 7 months 6 days | 5 years 6 months | 5 years 6 months |
Expected Volatility | 83.50% | 84.20% | 84.20% |
Expected Dividend Yield | 0% | 0% | 0% |
Risk-Free Interest Rate | 4% | 3.20% | 0.90% |
Options, grants in period, weighted average grant date fair value (in dollars per share) | $ 5.79 | $ 9.54 | $ 9.65 |
Stock-Based Compensation - Opti
Stock-Based Compensation - Option Activity (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward] | |||
Options exercised (in shares) | (1,276) | ||
Incentive Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract] | |||
Options, exercises in period, intrinsic value | $ 21,150 | $ 25,484 | |
Inducement Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract] | |||
Options, exercises in period, intrinsic value | $ 3,710 | $ 6,700 | |
Share-based Payment Arrangement, Option | Incentive Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward] | |||
Options outstanding, beginning balance (in shares) | 31,179 | ||
Options, grants in period, gross (in shares) | 6,625 | ||
Options exercised (in shares) | (935) | ||
Options cancelled or forfeited (in shares) | (1,364) | ||
Options outstanding, ending balance (in shares) | 35,505 | 31,179 | |
Options exercisable (in shares) | 21,008 | ||
Number of stock option awards vested and expected to vest (in shares) | 33,557 | ||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | |||
Weighted average exercise price, beginning balance (in dollars per share) | $ 8.56 | ||
Weighted average exercise price, stock option awards granted (in dollars per share) | 6.67 | ||
Weighted average exercise price, stock option awards exercised (in dollars per share) | 5.21 | ||
Weighted average exercise price, stock option awards cancelled (in dollars per share) | 10 | ||
Weighted average exercise price, ending balance (in dollars per share) | 8.24 | $ 8.56 | |
Weighted average exercise price, stock option awards, exercisable (in dollars per share) | 7.87 | ||
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) | $ 8.19 | ||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract] | |||
Weighted average remaining contractual life | 6 years 10 months 6 days | ||
Options, exercises in period, intrinsic value | $ 3,601 | ||
Options outstanding, aggregate intrinsic value | $ 12,280 | ||
Options, exercisable, weighted average remaining contractual term (year) | 5 years 5 months 8 days | ||
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) | 6 years 9 months 7 days | ||
Options, aggregate intrinsic value | $ 12,280 | ||
Options, exercisable, aggregate intrinsic value | $ 12,280 | ||
Share-based Payment Arrangement, Option | Inducement Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward] | |||
Options outstanding, beginning balance (in shares) | 5,341 | ||
Options, grants in period, gross (in shares) | 1,196 | ||
Options exercised (in shares) | (344) | ||
Options cancelled or forfeited (in shares) | (666) | ||
Options outstanding, ending balance (in shares) | 5,527 | 5,341 | |
Options exercisable (in shares) | 2,448 | ||
Number of stock option awards vested and expected to vest (in shares) | 5,122 | ||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | |||
Weighted average exercise price, beginning balance (in dollars per share) | $ 8.80 | ||
Weighted average exercise price, stock option awards granted (in dollars per share) | 8.15 | ||
Weighted average exercise price, stock option awards exercised (in dollars per share) | 3.70 | ||
Weighted average exercise price, stock option awards cancelled (in dollars per share) | 10.47 | ||
Weighted average exercise price, ending balance (in dollars per share) | 8.77 | $ 8.80 | |
Weighted average exercise price, stock option awards, exercisable (in dollars per share) | 7.17 | ||
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) | $ 8.68 | ||
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract] | |||
Weighted average remaining contractual life | 7 years 9 months 3 days | ||
Options, exercises in period, intrinsic value | $ 1,803 | ||
Options outstanding, aggregate intrinsic value | $ 4,463 | ||
Options, exercisable, weighted average remaining contractual term (year) | 6 years 10 months 20 days | ||
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) | 7 years 8 months 19 days | ||
Options, aggregate intrinsic value | $ 3,434 | ||
Options, exercisable, aggregate intrinsic value | $ 4,291 |
Stock-Based Compensation - Rest
Stock-Based Compensation - Restricted Stock Unit Activity (Details) - Restricted Stock Units (RSUs) - $ / shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Incentive Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward] | |||
Unvested RSU beginning balance (in shares) | 4,159 | ||
Issued RSU (in shares) | 3,106 | ||
Vested RSU (in shares) | (1,137) | ||
Forfeited RSU (in shares) | (536) | ||
Unvested RSU ending balance (in shares) | 5,592 | 4,159 | |
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract] | |||
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share) | $ 10.69 | ||
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) | 6.71 | $ 11.20 | $ 11.36 |
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share) | 11.04 | ||
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share) | 7.91 | ||
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share) | $ 8.67 | $ 10.69 | |
Inducement Plan | |||
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward] | |||
Unvested RSU beginning balance (in shares) | 588 | ||
Issued RSU (in shares) | 581 | ||
Vested RSU (in shares) | (139) | ||
Forfeited RSU (in shares) | (115) | ||
Unvested RSU ending balance (in shares) | 915 | 588 | |
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract] | |||
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share) | $ 13.14 | ||
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) | 7.81 | ||
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share) | 13.13 | ||
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share) | 11.98 | ||
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share) | $ 9.90 | $ 13.14 |
Income Taxes - Loss Before Prov
Income Taxes - Loss Before Provision for Income Taxes (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ (206,674) | $ (225,127) | $ (159,632) |
Foreign | (19,555) | (19,256) | (22,177) |
Loss before income taxes | $ (226,229) | $ (244,383) | $ (181,809) |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Income Tax (Benefit) Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Current expense (benefit) provision: | |||
U.S. Federal and state | $ (45) | $ 2,430 | $ 2,179 |
Foreign | 1,037 | 292 | 233 |
Total current expense provision | 992 | 2,722 | 2,412 |
Deferred expense (benefit) provision: | |||
U.S. Federal and state | (120) | 11 | (159) |
Foreign | (562) | 0 | 0 |
Total deferred expense provision | (682) | 11 | (159) |
Income tax expense | $ 310 | $ 2,733 | $ 2,253 |
Income Taxes - Differences Betw
Income Taxes - Differences Between the Company's Effective Tax Rate and the Statutory Tax Rate (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |||
Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021) | $ (47,328) | $ (51,321) | $ (38,175) |
State and local income taxes net of federal tax benefit | (3,477) | (1,816) | (2,288) |
Permanent items | 3,015 | (1,608) | (1,343) |
Expiration of attribute carryforwards | 0 | 0 | (1,057) |
Research and development tax credits | (3,301) | (9,793) | (5,994) |
Foreign rate differential | 2,255 | 1,862 | 1,940 |
Other | 1,656 | (5,485) | 1,216 |
Change in valuation allowance | 47,490 | 70,894 | 47,954 |
Income tax expense | $ 310 | $ 2,733 | $ 2,253 |
Income Taxes - Differences Be_2
Income Taxes - Differences Between the Company's Effective Tax Rate and the Statutory Tax Rate Narrative (Details) | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |||
Income tax benefit, federal statutory rate | 21% | 21% | 21% |
Income Taxes - Unrecognized Tax
Income Taxes - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2023 | Dec. 31, 2022 | |
Income Tax Disclosure [Roll Forward] | ||
Beginning balance | $ 13,844 | $ 9,729 |
Additions to current period tax positions | 825 | 4,115 |
Reductions to prior period tax provisions | (307) | 0 |
Ending balance | $ 14,362 | $ 13,844 |
Income Taxes - Deferred Tax Ass
Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2023 | Dec. 31, 2022 |
Deferred tax assets: | ||
Net federal and state operating losses | $ 108,753 | $ 101,600 |
Research and development credits | 88,390 | 86,321 |
Royalty income | 122,391 | 115,554 |
Stock-based compensation | 26,113 | 19,374 |
Capitalized R&D | 75,081 | 62,794 |
Leasing obligations | 3,076 | 1,842 |
Other | 17,515 | 4,354 |
Total deferred tax assets | 441,319 | 391,839 |
Deferred tax liabilities: | ||
Fixed assets | (678) | (717) |
Right of use asset | (2,872) | (1,525) |
Total deferred tax liabilities | (3,550) | (2,242) |
Valuation allowance | (437,098) | (389,608) |
Net deferred tax assets (liabilities) | $ 671 | |
Net deferred tax assets (liabilities) | $ (11) |
Income Taxes Textual (Details)
Income Taxes Textual (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Line Items] | |||
Change in valuation allowance | $ 47,490 | $ 70,894 | $ 47,954 |
Provisions and accruals for interest and penalties | $ 0 | $ 0 | $ 0 |
Open tax year | 2018 2019 2020 2021 | ||
Research Tax Credit Carryforward | |||
Income Tax Disclosure [Line Items] | |||
Tax credit carryforward, amount | $ 102,752 | ||
Domestic Tax Authority | |||
Income Tax Disclosure [Line Items] | |||
Operating loss carryforwards, total | 432,328 | ||
State and Local Jurisdiction | |||
Income Tax Disclosure [Line Items] | |||
Operating loss carryforwards, total | 183,478 | ||
Foreign Tax Authority | |||
Income Tax Disclosure [Line Items] | |||
Operating loss carryforwards, total | $ 65,416 |
Employee 401(k) Plan Textual (D
Employee 401(k) Plan Textual (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |||
Matching contributions | $ 5,716 | $ 3,758 | $ 2,834 |
Collaborative and Other Relat_2
Collaborative and Other Relationships Textual (Details) | 1 Months Ended | 12 Months Ended | |||||||||
Nov. 03, 2023 USD ($) payment | Sep. 15, 2021 USD ($) | Nov. 05, 2019 USD ($) | Sep. 30, 2013 USD ($) | Aug. 31, 2020 USD ($) | Sep. 30, 2018 USD ($) dose | Dec. 31, 2022 USD ($) dose | Dec. 31, 2021 USD ($) dose | Dec. 31, 2019 USD ($) dose | Nov. 30, 2023 | Mar. 31, 2015 USD ($) | |
U.S. Department of Health and Human Services, net | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Collaborative agreement contract value | $ 34,660,000 | ||||||||||
Contract term (year) | 5 years | ||||||||||
U.S. Department of Health and Human Services, net | RAPIVAB | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Maximum number of products, doses | dose | 50,000 | ||||||||||
Number of product delivered, doses | dose | 49,980 | 20,000 | |||||||||
Proceeds from collaborators | $ 13,864,000 | $ 6,918,000 | $ 13,864,000 | ||||||||
Number of additional products, doses | dose | 20,000 | 9,980 | |||||||||
Torii Pharmaceutical Co | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Contract with customer, liability | $ 22,000,000 | ||||||||||
Potential milestone payments receivable if regulatory approval before december 31, 2021 | $ 15,000,000 | ||||||||||
Maximum customary reduction on royalty rate | 50% | ||||||||||
Royalty payments receivable, expiration term from first commercial (year) | 10 years | ||||||||||
Torii Pharmaceutical Co | Minimum | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Royalty rate if maintains sakigake designation | 20% | 20% | |||||||||
Torii Pharmaceutical Co | Maximum | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Royalty rate if maintains sakigake designation | 40% | 80% | |||||||||
Clearside Biomedical, Inc. | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
License agreement, upfront payment | $ 5,000,000 | ||||||||||
Number of milestone payments | payment | 3 | ||||||||||
Royalty payment, number of tiers | payment | 3 | ||||||||||
Clearside Biomedical, Inc. | Annual Net Sales Of $2 Million | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Annual global net sales | $ 2,000,000,000 | ||||||||||
Clearside Biomedical, Inc. | Annual Net Sales Greater Than $1.5 Million | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Annual global net sales | 1,500,000,000 | ||||||||||
Clearside Biomedical, Inc. | Maximum | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Potential milestone payments payable | 30,000,000 | ||||||||||
Potential milestone payments payable if regulatory approval before milestone | $ 47,500,000 | ||||||||||
Base Contract | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Government contract receivable | $ 16,265,000 | ||||||||||
Additional Development Options | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Government contract receivable | 22,855,000 | ||||||||||
ASPRBARDA Contract | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Government contract receivable | $ 39,120,000 | ||||||||||
Proceeds from awards for research and development contracts | $ 20,574,000 | ||||||||||
National Institute of Allergy and Infectious Diseases | |||||||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||||||||
Receivable from awards for research and development contracts | $ 47,315,000 | $ 45,931,000 | |||||||||
Potential aggregate maximum amount of funding | $ 43,908,000 | ||||||||||
Collaborative agreement contract value | $ 6,326,000 |
Workforce Reduction (Details)
Workforce Reduction (Details) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Employee Severance | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | $ 3,380,000 | |
Restructuring reserve | 0 | |
Employee Severance | Research and Developement | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | 3,026,000 | |
Employee Severance | Selling, general and administrative | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | $ 354,000 | |
One-time Termination Benefits | Subsequent Event | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | $ 1,264,000 | |
One-time Termination Benefits | Research and Developement | Subsequent Event | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | 1,201,000 | |
One-time Termination Benefits | Selling, general and administrative | Subsequent Event | ||
Restructuring Cost and Reserve [Line Items] | ||
Restructuring costs | $ 63,000 |