Document And Entity Information
Document And Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2022 | Mar. 03, 2023 | Jun. 30, 2022 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2022 | ||
Document Fiscal Year Focus | 2022 | ||
Document Fiscal Period Focus | FY | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Registrant Name | Mirum Pharmaceuticals, Inc. | ||
Entity Central Index Key | 0001759425 | ||
Entity Current Reporting Status | Yes | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Interactive Data Current | Yes | ||
Entity Voluntary Filers | No | ||
Entity Well-known Seasoned Issuer | Yes | ||
ICFR Auditor Attestation Flag | false | ||
Entity Public Float | $ 609 | ||
Entity Common Stock, Shares Outstanding | 37,950,968 | ||
Entity Shell Company | false | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | true | ||
Entity File Number | 001-38981 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 83-1281555 | ||
Entity Address, Address Line One | 950 Tower Lane, Suite 1050 | ||
Entity Address, City or Town | Foster City | ||
Entity Address, State or Province | CA | ||
Entity Address, Postal Zip Code | 94404 | ||
City Area Code | 650 | ||
Local Phone Number | 667-4085 | ||
Document Transition Report | false | ||
Document Annual Report | true | ||
Title of 12(b) Security | Common stock, par value $0.0001 per share | ||
Trading Symbol | MIRM | ||
Security Exchange Name | NASDAQ | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Portions of the Registrant’s definitive proxy statement for its 2023 Annual Meeting of Stockholders, which the Registrant intends to file pursuant to Regulation 14A with the Securities and Exchange Commission not later than 120 days after the Registrant’s fiscal year ended December 31, 2022, are incorporated by reference into Part III of this Annual Report on Form 10-K. | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Location | Irvine, California | ||
Auditor Firm ID | 42 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 28,003 | $ 31,340 |
Short-term investments | 123,716 | 125,201 |
Accounts receivable | 23,994 | 3,267 |
Inventory | 5,565 | 1,513 |
Prepaid expenses and other current assets | 8,947 | 5,271 |
Total current assets | 190,225 | 166,592 |
Restricted cash equivalents | 100,000 | 100,000 |
Long-term investments | 0 | 4,983 |
Property and equipment, net | 914 | 981 |
Operating lease right-of-use assets | 1,431 | 1,569 |
Intangible assets, net | 58,954 | 18,740 |
Other assets | 1,382 | 1,786 |
Total assets | 352,906 | 294,651 |
Current liabilities: | ||
Accounts payable | 8,690 | 9,166 |
Accrued expenses | 54,018 | 30,723 |
Operating lease liabilities | 931 | 711 |
Derivative liability | 1,090 | 1,996 |
Total current liabilities | 64,729 | 42,596 |
Revenue interest liability, net | 140,351 | 129,923 |
Operating lease liabilities, noncurrent | 1,257 | 1,903 |
Other liabilities | 4,532 | 17 |
Total liabilities | 210,869 | 174,439 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized as of December 31, 2022 and 2021; no shares issued and outstanding as of December 31, 2022 and 2021; and liquidation value of zero as of December 31, 2022 and 2021 | 0 | 0 |
Common stock, $0.0001 par value; 200,000,000 shares authorized; 36,956,345 shares issued and outstanding, excluding zero shares subject to repurchase as of December 31, 2022; and 30,705,060 shares issued and 30,582,596 shares outstanding, excluding 122,464 shares subject to repurchase as of December 31, 2021 | 4 | 3 |
Additional paid-in capital | 535,074 | 377,403 |
Accumulated deficit | (392,824) | (257,159) |
Accumulated other comprehensive income (loss) | (217) | (35) |
Total stockholders’ equity | 142,037 | 120,212 |
Total liabilities and stockholders’ equity | $ 352,906 | $ 294,651 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares, issued | 0 | 0 |
Preferred stock, shares, outstanding | 0 | 0 |
Preferred stock, liquidation preference | $ 0 | $ 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares, issued | 36,956,345 | 30,705,060 |
Common stock, shares, outstanding | 36,956,345 | 30,582,596 |
Common stock, subject to repurchase | 0 | 122,464 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Revenues [Abstract] | ||
Total revenue | $ 77,062 | $ 19,138 |
Operating expenses: | ||
Cost of sales | 12,374 | 1,903 |
Research and development | 106,842 | 131,428 |
Selling, general and administrative | 89,066 | 59,220 |
Total operating expenses | 208,282 | 192,551 |
Loss from operations | (131,220) | (173,413) |
Other income (expense): | ||
Interest income | 3,857 | 366 |
Interest expense | (15,979) | (17,590) |
Change in fair value of derivative liability | 906 | (732) |
Other income (expense), net | 365 | (582) |
Gain from sale of priority review voucher, net | 0 | 108,000 |
Loss before income taxes | (142,071) | (83,951) |
(Benefit from) provision for income taxes | (6,406) | 37 |
Net loss | $ (135,665) | $ (83,988) |
Earnings Per Share, Basic [Abstract] | ||
Basic | $ (4.01) | $ (2.77) |
Diluted | $ (4.02) | $ (2.77) |
Earnings Per Share, Diluted [Abstract] | ||
Basic | 33,839,072 | 30,321,722 |
Diluted | 33,982,493 | 30,321,722 |
Product [Member] | ||
Revenues [Abstract] | ||
Total revenue | $ 75,062 | $ 3,138 |
License [Member] | ||
Revenues [Abstract] | ||
Total revenue | $ 2,000 | $ 16,000 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (135,665) | $ (83,988) |
Other comprehensive gain (loss): | ||
Unrealized loss on available-for-sale investments | (194) | (117) |
Cumulative translation adjustments | 12 | (1) |
Comprehensive loss | $ (135,847) | $ (84,106) |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Equity Incentive Plan | Employee Stock Purchase Plan (ESPP) | At-the-market Offering | Public Offering | Common Stock | Common Stock Equity Incentive Plan | Common Stock Employee Stock Purchase Plan (ESPP) | Common Stock At-the-market Offering | Common Stock Public Offering | Additional Paid-In Capital | Additional Paid-In Capital Equity Incentive Plan | Additional Paid-In Capital Employee Stock Purchase Plan (ESPP) | Additional Paid-In Capital At-the-market Offering | Additional Paid-In Capital Public Offering | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Balance at Dec. 31, 2020 | $ 172,095 | $ 3 | $ 345,180 | $ (173,171) | $ 83 | ||||||||||||
Balance, Shares at Dec. 31, 2020 | 29,776,544 | ||||||||||||||||
Issuance of common stock | $ 7,038 | $ 7,038 | |||||||||||||||
Issuance of common stock, Shares | 375,654 | ||||||||||||||||
Issuance of common stock in connection with Employee Stock Purchase Plan | $ 844 | $ 1,254 | $ 844 | $ 1,254 | |||||||||||||
Issuance of common stock in connection with Employee Stock Purchase Plan, Shares | 207,595 | 89,211 | |||||||||||||||
Restricted common stock vested in the period, Shares | 133,592 | ||||||||||||||||
Stock-based compensation | 23,087 | 23,087 | |||||||||||||||
Net loss | (83,988) | (83,988) | |||||||||||||||
Other comprehensive loss | (118) | (118) | |||||||||||||||
Balance at Dec. 31, 2021 | 120,212 | $ 3 | 377,403 | (257,159) | (35) | ||||||||||||
Balance, Shares at Dec. 31, 2021 | 30,582,596 | ||||||||||||||||
Issuance of common stock | $ 21,289 | $ 86,078 | $ 1 | $ 21,289 | $ 86,077 | ||||||||||||
Issuance of common stock, Shares | 1,160,915 | 4,000,000 | |||||||||||||||
Issuance of common stock in connection with asset acquisition, shares | 609,305 | ||||||||||||||||
Issuance of common stock in connection with asset acquisition | 15,585 | 15,585 | |||||||||||||||
Issuance of common stock in connection with Employee Stock Purchase Plan | $ 5,438 | $ 1,834 | $ 5,438 | $ 1,834 | |||||||||||||
Issuance of common stock in connection with Employee Stock Purchase Plan, Shares | 357,934 | 123,131 | |||||||||||||||
Restricted common stock vested in the period, Shares | 122,464 | ||||||||||||||||
Stock-based compensation | 27,448 | 27,448 | |||||||||||||||
Net loss | (135,665) | (135,665) | |||||||||||||||
Other comprehensive loss | (182) | (182) | |||||||||||||||
Balance at Dec. 31, 2022 | $ 142,037 | $ 4 | $ 535,074 | $ (392,824) | $ (217) | ||||||||||||
Balance, Shares at Dec. 31, 2022 | 36,956,345 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity (Parenthetical) - Common Stock - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
At-the-market Offering | ||
Issuance Costs | $ 785 | |
Public Offering | ||
Issuance Costs | $ 5,922 | $ 476 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Operating activities | ||
Net loss | $ (135,665) | $ (83,988) |
Reconciliation of net loss to net cash used in operating activities: | ||
Stock-based compensation | 27,007 | 23,087 |
Depreciation and amortization | 3,238 | 595 |
Amortization of operating lease right-of-use assets | 443 | 380 |
Net accretion of discounts on investments | (1,703) | (137) |
Non-cash interest expense related to the revenue interest liability | 15,979 | 17,590 |
Change in fair value of derivative liability | (906) | 732 |
Change in fair value of contingent liabilities associated with acquisition | (899) | 0 |
Deferred income tax taxes benefit associated with an acquisition | (6,580) | 0 |
Gain on sale of priority review voucher, net | 0 | (108,000) |
Change in operating assets and liabilities: | ||
Accounts receivable | (20,727) | 0 |
Prepaid and other current assets | (3,676) | (4,009) |
Inventory | (3,448) | (495) |
Other assets | (127) | (519) |
Accounts payable, accrued expenses and other liabilities | 7,639 | 22,655 |
Operating lease liabilities | (711) | (649) |
Net cash used in operating activities | (120,136) | (132,758) |
Investing activities | ||
Purchase of investments | (132,322) | (198,029) |
Proceeds from maturities of investments | 140,300 | 155,600 |
Proceeds from paydown of investments | 0 | 2,000 |
Purchase of property and equipment | (278) | (24) |
Proceeds from sale of priority review voucher, net | 0 | 108,000 |
Additions to intangible assets | 0 | (19,000) |
Net cash provided by investing activities | 7,700 | 48,547 |
Financing activities | ||
Proceeds from issuance of common stock in public offerings, net of issuance costs | 86,078 | 6,914 |
Proceeds from issuance of common stock in at-the-market offerings, net of issuance costs | 21,289 | 0 |
Proceeds from issuance of common stock pursuant to equity plans | 7,272 | 2,098 |
Proceeds from revenue interest liability, net of issuance costs | 0 | 64,575 |
Payments on revenue interest liability | (5,552) | (121) |
Net cash provided by financing activities | 109,087 | 73,466 |
Effect of exchange rate on cash, cash equivalents and restricted cash equivalents | 12 | (1) |
Net decrease increase in cash, cash equivalents and restricted cash equivalents | (3,337) | (10,746) |
Cash, cash equivalents and restricted cash equivalents at beginning of period | 131,340 | 142,086 |
Cash, cash equivalents and restricted cash equivalents at end of period | 128,003 | 131,340 |
Supplemental disclosure of cash flow information: | ||
Operating cash flows paid for operating lease | 911 | 864 |
Noncash investing and financing activities: | ||
Issuance of common stock in exchange for acquired intangible assets | 15,585 | 0 |
Accrued milestone payments classified as intangible assets, net | 15,000 | 0 |
Deferred Tax Liability Incurred From Acquired Intangible | 6,580 | 0 |
Contingent milestone liability for common stock issuance tly issuable common stock for acquired intangible assets | 4,600 | 0 |
Indemnification Holdback liability for common stock issuance in exchange for acquired intangible assets | 831 | 0 |
Stock Based Compensation Capitalized to Inventory | 441 | 71 |
Right-of-use asset obtained in exchange for lease liability | 285 | 0 |
Inventory purchases in accrued liabilities | $ 163 | $ 1,018 |
Organization and Description of
Organization and Description of Business | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Description of Business | 1. Organization and Description of Business Mirum Pharmaceuticals, Inc. (the “Company”) was incorporated in the State of Delaware on May 2, 2018 , and is headquartered in Foster City, California. The Company is a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases. The Company’s product LIVMARLI® (maralixibat) oral solution (“Livmarli”), was approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) one year of age and older in the United States in September 2021 and for the treatment of cholestatic pruritus in patients with ALGS two months and older in Europe in December 2022. The Company’s development pipeline consists of two clinical-stage product candidates, Livmarli and volixibat. The Company commenced significant operations in November 2018. The Company views its operations and manages its business as one operating segment. The Company determined its operating segment on the same basis that it uses to evaluate its performance internally. Liquidity The Company has a limited operating history, has incurred significant operating losses since its inception, and the revenue and income potential of the Company’s business and market are unproven. As of December 31, 2022, the Company had an accumulated deficit of $ 392.8 million and cash, cash equivalents, restricted cash equivalents and investments of $ 251.7 million. The Company believes that its unrestricted cash, cash equivalents and investments of $ 151.7 million as of December 31, 2022, provide sufficient capital resources to continue its operations for at least twelve months from the issuance date of the accompanying consolidated financial statements. T he consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. The consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of this uncertainty. Management expects to continue to incur additional substantial losses in the foreseeable future as a result of the Company’s research and development activities. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. The Company translates the financial statements of its foreign subsidiaries using end-of-period exchange rates for assets and liabilities and average exchange rates during each reporting period for results of operations. All significant intercompany balances and transactions among the consolidated entities have been eliminated in consolidation. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”). Use of Estimates The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the financial statements and accompanying notes. These estimates and assumptions are based upon historical experience, knowledge of current events and various other factors 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 and the recording of expenses that are not readily apparent from other sources. Actual results could differ materially from those estimates. The Company’s consolidated financial statements as of and for the year ended December 31, 2022 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, higher interest rates, foreign exchange rate fluctuations and the COVID-19 pandemic. The duration and the scope of these conditions cannot be predicted; therefore, the extent to which these conditions will directly or indirectly impact the Company’s business, results of operations and financial condition, is uncertain. The Company is not aware of any specific event or circumstance that would require an update to its estimates, judgments and assumptions or a revision of the carrying value of the Company’s assets or liabilities as of the date of this filing. Cash, Cash Equivalents and Restricted Cash Equivalents The Company considers all highly liquid investments that are readily convertible into cash without penalty and with original maturities of three months or less at the date of purchase to be cash equivalents. The carrying amounts reported in the consolidated balance sheets for cash and cash equivalents are valued at cost, which approximate their fair value. Restricted cash equivalents consist of deposits placed in a segregated bank account as required under the terms of the Company’s Revenue Interest Purchase Agreement ("RIPA"), as amended September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the purchasers party thereto (the "Purchasers"), and the Purchasers in connection with the sale of the Priority Review Voucher ("PRV") in December 2021. The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the consolidated balance sheets that together reflect the same amounts shown in the consolidated statements of cash flows (in thousands): December 31, 2022 2021 Cash and cash equivalents $ 28,003 $ 31,340 Restricted cash equivalents 100,000 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 128,003 $ 131,340 Concentrations of Credit Risk and Off-Balance Sheet Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, accounts receivable and investments. The Company limits the amount of credit exposure by investing cash that is not required for immediate operating needs in money market funds, government obligations and/or commercial paper with short maturities. Additionally, the Company has established guidelines regarding diversification of its investments and their maturities, which are designed to maintain principal and maximize liquidity. To date, the Company has not experienced any losses associated with this credit risk and continues to believe that this exposure is not significant. The Company relies on a specialty pharmacy and a single distributor for all of the Company’s sales of Livmarli in the United States as well as a single distributor for sales outside the United States. The Company sources materials and services through several vendors. Certain materials are sourced from a single vendor. The loss of certain vendors could result in a temporary disruption of the Company’s commercialization efforts. For the years ended December 31, 2022 and 2021, the Company had one customer that accounted for approximately 23% and zero, respectively, of accounts receivable. For the years ended 2022 and 2021, the Company did not have revenue attributable to any one customer in excess of 10% of sales. Investments The Company classifies all investments in securities as available-for-sale, as the sale of such securities may be required prior to maturity. Management determines the appropriate classification of its investments in debt securities at the time of purchase. Investments with original maturities beyond three months at the date of purchase and which mature at, or less than twelve months from the balance sheet date, are classified as a current asset. Investments are recorded at fair value, with unrealized gains and losses reported as accumulated other comprehensive income (loss) until realized. The Company periodically evaluates whether declines in fair values of its available-for-sale securities below their cost basis are other-than-temporary and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. This evaluation consists of several qualitative and quantitative factors regarding the severity and duration of the unrealized loss as well as the Company’s ability and intent to hold the available-for-sale security until a forecasted recovery occurs. Additionally, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale securities before recovery of its amortized cost basis. The cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, are included in interest income. Realized gains and losses from the sale of available-for-sale securities, if any, are determined on a specific identification basis and are also included in interest income (loss). To date, the Company has no t identified any other than temporary declines in fair value of its investments. Fair Value of Financial Instruments Fair value is defined as an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement determined based on assumptions that market participants would use in pricing an asset or liability. The following fair value hierarchy distinguishes between observable and unobservable inputs, which are categorized in one of the following levels: Level 1: Observable inputs (unadjusted) such as quoted prices in active markets for identical assets or liabilities; Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly for similar assets or liabilities; and Level 3: Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. Accounts Receivable Accounts receivable are recorded net of allowances for sales discounts and any allowance for doubtful accounts. Management estimates the allowance for doubtful accounts based on existing contractual payment terms, actual payment patterns of customers and individual customer circumstances. To date, an allowance for doubtful accounts has not been material. Inventory Inventory is valued at the lower of cost or net realizable value, with cost determined on a first-in, first-out (FIFO) basis. The Company periodically reviews the composition of inventory to identify excess, obsolete, slow-moving or otherwise unsaleable items. If unsaleable items are observed and there are no alternate uses for the inventory, the Company will record a write-down to net realizable value in the period that the decline in value is recognized through a charge to cost of sales. The determination of whether inventory costs will be realizable requires estimates by management. If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required. Prior to the initial regulatory approval for Livmarli, the Company expensed costs relating to raw materials and production of inventory as research and development expense in the accompanying consolidated statements of operations, in the period incurred. The Company expects it will deplete such inventories within 12 months of the balance sheet date. Property and Equipment Property and equipment are recorded at cost less accumulated depreciation. Depreciation is computed on a straight-line basis over the estimated useful lives of the related assets, ranging from three to five years . Leasehold improvements are amortized over the shorter of their useful lives or the related lease term. As of December 31, 2022, property and equipment consisted primarily of leasehold improvements of $ 1.3 million and furniture and equipment of $ 0.6 million. As of December 31, 2021, property and equipment consisted primarily of leasehold improvements of $ 1.3 million and furniture and equipment of $ 0.2 million. Accumulated depreciation as of December 31, 2022 and 2021 was $ 1.0 million and $ 0.7 million, respectively. Depreciation expense was $ 0.3 million for the years ended December 31, 2022 and 2021, respectively. Impairment of Long-Lived Assets Long-lived assets are reviewed for indications of possible impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability is measured by comparison of the carrying amounts to the future undiscounted cash flows attributable to these assets. An impairment loss is recognized to the extent an asset group is not recoverable, and the carrying amount exceeds the projected discounted future cash flows arising from these assets. There were no impairments of long-lived assets for any of the periods presented. Intangible Assets, Net The Company accounts for asset acquisitions that do not meet the definition of a business using the cost accumulation method, whereby the cost of the acquisition, including certain transaction costs, is allocated to the asset (or assets) acquired on the basis of its (or their) relative fair value(s) on the measurement date. No goodwill is recognized in an asset acquisition. Intangible assets are measured at their fair values as of the acquisition date or, in the case of commercial milestone payments, the date they become due. The evaluation of intangible assets includes assessing the amortization period for which the asset is expected to contribute to the future cash flows of the Company. Intangible assets with finite useful lives are amortized over their estimated useful lives, primarily on a straight-line basis when the Company is unable to reliably estimate the pattern of cash flow. The Company tests its finite lived intangible assets for impairment annually or if events or changes in circumstances indicate that it is more likely than not that the asset is impaired. If it is determined that the asset is impaired, the carrying value is written down to its estimated fair value, with the related impairment charge recognized in the consolidated statements of operations in the period in which the impairment occurs. The Company has not recorded any impairments to its intangible assets for any of the periods presented. The following table provides detail of the carrying amount of the Company’s intangible assets (in thousands): December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Value Intangible asset - commercial milestone payments $ 34,000 $ ( 1,333 ) $ 32,667 Intangible assets - Satiogen acquisition 28,107 ( 1,820 ) 26,287 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 December 31, 2021 Gross Carrying Value Accumulated Amortization Net Carrying Amount Intangible asset - commercial milestone payments $ 19,000 $ ( 260 ) $ 18,740 Total intangible assets $ 19,000 $ ( 260 ) $ 18,740 As of December 31, 2022, the remaining weighted-average amortization period of the Company’s intangible assets was 11.9 years. The remaining weighted-average amortization periods of the commercial milestone payments and the Satiogen Pharmaceutical, Inc. ("Satiogen") acquisition intangible assets were 17.1 years and 8.4 years, respectively. Amortization expense was $ 2.9 million and $ 0.3 million for the years ended December 31, 2022 and 2021, respectively, and was included in cost of sales on the accompanying consolidated statements of operations. The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of December 31, 2022 (in thousands): Years Ended December 31, Amount 2023 $ 5,035 2024 5,035 2025 5,035 2026 5,035 2027 5,035 Thereafter 33,779 $ 58,954 Leases The Company determines if a contractual arrangement is or contains a lease at inception. Operating lease right-of-use (“ROU”) assets represent the Company’s right to use an underlying asset during the lease term, and operating lease liabilities represent the Company’s obligation to make lease payments arising from the lease. Operating leases are included in ROU assets, current operating lease liabilities, and long-term operating lease liabilities on the accompanying consolidated balance sheets. Operating lease ROU assets and lease liabilities are initially recognized based on the present value of the future minimum lease payments over the lease term at commencement date calculated using the Company’s incremental borrowing rate applicable to the leased asset, unless the implicit rate is readily determinable. Operating lease ROU assets also include any lease payments made at or before lease commencement and exclude any lease incentives received. The Company determines the lease term as the noncancelable period of the lease and may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Leases with a term of 12 months or less are not recognized on the consolidated balance sheets. The Company’s leases do not contain any residual value guarantees. Lease expense for minimum lease payments is recognized as rent expense on a straight-line basis over the lease term. Variable lease payments include lease operating expenses. Accrued Research and Development Expenses The Company accrues and expenses clinical trial activities performed by third parties based upon estimates of the proportion of work completed over the life of the individual study and patient enrollment rates in accordance with agreements established with clinical research organizations and clinical trial sites. The Company determines the estimates by reviewing contracts, vendor agreements and purchase orders and through discussions with internal clinical personnel and external service providers as to the progress or stage of completion of trials or services and the agreed-upon fee to be paid for such services. The Company makes estimates of accrued expenses as of each balance sheet date based on facts and circumstances known to the Company at that time. 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. The Company has not experienced any material differences between accrued costs and actual costs incurred for the periods presented. Nonrefundable advance payments for goods and services are deferred and recognized as expense in the period that the related goods are consumed or services are performed. Revenue Interest Liability, Net The revenue interest liability, net, associated with the RIPA that the Company entered into in December 2020, as amended in September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC (“Oberland”), as agent for purchasers party thereto (the “Purchasers”), and the Purchasers, is presented net of issuance costs and a debt discount on the consolidated balance sheets. The Company imputes interest expense associated with this liability using the effective interest rate method. The effective interest rate is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement. The interest rate on the liability may vary during the term of the agreement depending on a number of factors, including the level of actual and forecasted product sales, net. The Company evaluates the interest rate quarterly based on actual product sales, net and forecast product sales, net, utilizing the prospective method. A significant increase or decrease in product sales, net will materially impact the revenue interest liability, interest expense and the time period for repayment. Derivative Liability The RIPA contains certain features that meet the definition of being an embedded derivative requiring bifurcation as a separate compound financial instrument apart from the RIPA. The derivative liability is initially measured at fair value on issuance and is subject to remeasurement at each reporting period with changes in fair value recognized as other income (expense) in the accompanying consolidated statements of operations as the change in fair value of derivative liability. Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration the Company expects to receive in exchange for those goods or services. Product Sales, Net The Company recognizes product sales, net when the customer obtains control of our product, which occurs at a point in time, typically upon delivery of the Company’s product to the customer. Revenues from product sales are recorded at the net sales price, or the transaction price, which may include fixed or variable consideration for discounts, government rebates, co-pay assistance, returns and other allowances that are offered within contracts with a customer relating to the sale of Livmarli. Estimates of variable consideration are calculated based on the actual product sales each reporting period and the nature of the variable consideration related to those sales. Overall, these estimates reflect the Company’s best estimate of the amount of consideration to which the Company expects to be entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in product sales, net only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Estimates are reviewed and updated quarterly as additional information becomes known. Actual amounts of consideration ultimately received may differ materially from estimates. If actual results in the future vary from estimates, the Company will adjust these estimates, which would affect product sales, net and earnings in the period such variances are adjusted. Significant categories of sales discounts and allowances are as follows: Government Rebates : The Company records rebates payable under Medicaid and other government programs as a reduction of revenue at the time product revenues are generated. The Company’s rebate calculations may require estimates, including estimates of customer mix, to determine which sales will be subject to rebates and the amount of such rebates. The Company updates its estimates and assumptions on a quarterly basis and records any necessary adjustments to revenue in the period identified. The liability for unpaid rebates is included in accrued expenses in the accompanying consolidated balance sheets. To date, actual government rebates have not differed materially from the Company’s estimates. Other Incentives: Other incentives include a branded co-pay assistance program for eligible patients with commercial insurance in the United States. The branded co-pay assistance program assists commercially insured patients who have coverage for Livmarli and is intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance. The calculation of the accrual for co-pay assistance is based upon an identification of claims and the cost per claims associated with product that has been recognized as revenue. The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales. To date, actual other incentives have not differed materially from the Company’s estimates. Product Returns: The Company records revenue for product sales, net of estimated product returns. Customers have limited return rights related only to the product’s damage or defect identified upon delivery of the product. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. To date, actual returns have not differed materially from the Company’s estimates. License and Collaboration Arrangements The Company enters into collaborative arrangements with partners and analyzes the collaboration arrangements to assess whether they are within the scope of Collaborative Arrangements (Topic 808) ("Topic 808") and determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. The accounting for some of the activities under collaboration arrangements may be subject to Revenue from Contracts with Customers (Topic 606) (“Topic 606”) for distinct units of account that are reflective of a vendor-customer relationship. For other elements of collaboration arrangements, such as reimbursements of certain development costs, the Company generally records reimbursements received as a reduction of research and development expenses. In determining the appropriate amount of revenue to be recognized under Topic 606 as the Company fulfills its obligations under each of its agreements, the Company performs the following steps: (i) identification of the contracts with customers; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) determination and measurement of the transaction price, including any constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations in the contract; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The terms of the Company’s license and collaborative research and development agreements include upfront license fees, research, development and other funding or reimbursements, milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and sales-based events, as well as royalties on sales of commercialized products. Arrangements that include upfront payments may require deferral of revenue recognition to a future period until the Company satisfies performance obligations under these arrangements. A performance obligation is a promise in a contract to transfer a distinct good or service and is the unit of accounting in Topic 606. A contract’s transaction price is allocated among each distinct performance obligation based on relative standalone selling price and recognized as revenue when, or as, the applicable performance obligation is satisfied. If a license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues attributed to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. At the inception of each arrangement that includes development milestone payments, the Company evaluates whether the milestones are considered probable of being achieved and estimates the amount to be included in the transaction price as variable consideration using the most likely amount method or expected value method, depending on the nature of the contingency and the variable payments. If it is probable that a significant reversal of cumulative revenue recognized for the contract would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not generally considered probable of being achieved until those approvals are received. Given the high degree of uncertainty around the occurrence of these events, the Company generally determines the milestone and other contingent amounts to be fully constrained until the uncertainty associated with these payments is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of any development milestones, and if necessary, adjusts its estimate of the transaction price. Any such adjustments would be recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. For arrangements that include sales-based royalties, including milestone payments based on the level of sales, the Company recognizes revenue at the later of (i) when or as the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Accounting for these arrangements requires the Company to develop assumptions that require judgment to determine the stand-alone selling price of each performance obligation identified in the contract. The Company has never sold the performance obligations in its collaborative arrangements separately; therefore, an observable stand-alone selling price does not exist. Accordingly, the Company estimates a stand-alone selling price through maximizing the use of observable inputs such as market data, project cost estimates, and targeted margins. Cost of Product Sales Prior to receiving approval from the U.S. Food and Drug Administration ("FDA") in September 2021 to sell Livmarli in the United States, the Company expensed all costs incurred related to the manufacture of Livmarli as research and development expense because of the inherent risks associated with the development of a drug candidate, the uncertainty about the regulatory approval process and the lack of history for the Company of regulatory approval of drug candidates. Subsequent to receiving FDA approval, when commercialization was considered probable and the future economic benefit was expected to be realized, the Company began capitalizing inventory costs incurred. Cost of product sales consist of manufacturing costs, transportation and freight, amortization of capitalized intangible assets, royalties and indirect overhead costs associated with the manufacturing and distribution of Livmarli. Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges. Research and Development Expenses Research and development expenses consists primarily of fees paid to contract research organizations and other vendors for clinical, non-clinical and manufacturing services, salaries and employee benefits, including stock-based compensation, consultant expenses, costs related to acquiring manufacturing materials, costs related to compliance with regulatory requirements and license payments related to acquiring intellectual property rights for the Company’s product candidates. Research and development expenses are expensed as incurred. Selling, General and Administrative Expenses Selling, general and administrative (“SG&A”) expenses relate to sales and marketing, finance, human resources, legal and other administrative activities. SG&A expenses consist primarily of personnel costs, facilities and overhead costs, outside marketing, advertising and legal expenses, and other general and administrative costs. The Company expenses the costs of advertising, including promotional expenses, as incurred. Advertising expenses were $ 4.0 million and $ 9.2 million for the years ended December 31, 2022 and 2021, respectively. Stock-Based Compensation The Company recognizes stock-based compensation for all stock-based awards based on the grant date fair value of the award granted to employees and nonemployees, including members of its board of directors. For stock-based awards with service conditions, the fair value of the awards is recognized on a straight-line basis over the requisite service period in which the awards are expected to vest. For stock-based awards with performance vesting conditions, stock-based compensation is recognized when it is considered probable that the performance conditions will be satisfied. At each reporting period, the Company reassesses the probability of the achievement of the performance vesting conditions. Any change in stock-based compensation resulting from an adjustment in the vesting is treated as a cumulative catch-up in the period of adjustment. For stock-based awards with market conditions, stock-based compensation is recognized over the appropriate requisite service period. The Company accounts for forfeitures as they occur. The Company evaluates the grant date fair value of awards granted for impacts of any potential material non-public information at the time of grant. Income Taxes Income taxes are recorded using the liability method, under which deferred tax assets and liabilities are determined based on differences between the financial reporting and tax basis of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. Valuation allowances are recorded against deferred tax assets, including net operating losses and tax credits, when it is determined it is more-likely-than-not that some or all of the tax benefits will not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of Financial Accounting Standards Board (“FASB”) Accounting Standards Codification No. 740, Income Taxes (“ASC 740”). When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit would more likely than not be realized assuming examination by the taxing authority. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. Interest and penalties related to unrecognized tax benefits, if any, are recorded as a component of income tax expense. Net Loss Per Share Basic net loss per share is computed by dividing net loss attributable to common stockholders by the weighted-average shares of common stock outstanding for the period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss attributable to common stockholders by the weighted-average shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury-stock and if-converted methods. The following table sets for the computation of basic and diluted earnings per share (in thousands, except share and per share data): Year Ended December 31, 2022 2021 Numera |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements Financial assets and liabilities subject to fair value measurements on a recurring basis and the level of inputs used in such measurements by major security type are presented in the following table (in thousands): December 31, 2022 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,975 — — 4,975 Commercial paper — 74,386 — 74,386 U.S. government bonds — 44,354 — 44,354 Total financial assets $ 129,202 $ 118,740 $ — $ 247,942 Financial liabilities: Contingent milestone liability $ 3,900 $ — $ — $ 3,900 Derivative liability — — 1,090 1,090 Indemnification holdback — — 617 617 Total financial liabilities $ 3,900 $ — $ 1,707 $ 5,607 December 31, 2021 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 128,420 $ — $ — $ 128,420 Commercial paper — 115,221 — 115,221 U.S. government bonds — 14,963 — 14,963 Total financial assets $ 128,420 $ 130,184 $ — $ 258,604 Financial liabilities: Derivative liability $ — $ — $ 1,996 $ 1,996 Total financial liabilities $ — $ — $ 1,996 $ 1,996 The carrying amounts of certain financial instruments such as cash and cash equivalents, restricted cash equivalents, accounts receivable, prepaid expenses, other current assets, accounts payable and accrued expenses as of December 31, 2022 and 2021 approximate their related fair values due to the short-term maturities of these instruments. Money market funds and U.S. treasury bills are highly liquid investments and are actively traded. The pricing information on these investment instruments is readily available and can be independently validated as of the measurement date. This approach results in the classification of these securities as Level 1 of the fair value hierarchy. U.S. government agency bonds, U.S. government bonds and commercial paper, are measured at fair value using Level 2 inputs. The Company reviews trading activity and pricing for these investments as of each measurement date. The carrying amount of the revenue interest liability as of December 31, 2022 and 2021 approximates its fair value and is based on the Company’s contractual repayment obligation to the Purchasers, based on the current estimates of future revenues, over the life of the RIPA. The derivative liability and Indemnification Holdback liability (each as defined below) are each considered a Level 3 input based on the three-level hierarchy. At time of issuance, the Contingent Milestone liability at fair value was recognized using Level 3 inputs as no observable inputs were available at the time in the market. At December 31, 2022, the Contingent Milestone liability has been reclassified as a Level 1 input as the significant inputs were known and observable. Derivative Liability The debt pursuant to the RIPA (refer to Note 6 "Revenue Interest Purchase Agreement" for further information) contains an embedded derivative requiring bifurcation as a single compound derivative instrument. The Company estimated the fair value of the derivative liability using a “with-and-without” method. The “with-and-without” methodology involves valuing the whole instrument on an as-is basis and then valuing the instrument without the individual embedded derivative. The difference between the entire instrument with the embedded derivative compared to the instrument without the embedded derivative was the fair value of the derivative liability at December 31, 2022 and 2021. The estimated probability and timing of underlying events triggering the exercisability of the put option contained within the RIPA, forecasted cash flows and the discount rate are significant unobservable inputs used to determine the estimated fair value of the entire instrument with the embedded derivative. As of December 31, 2022 and 2021, the discount rate used for valuation of the derivative liability was 15.7 %, respectively. The following table provides a summary of the change in the estimated fair value of the Company’s derivative liability, classified as Level 3 in the fair value hierarchy (in thousands): Balance at January 1, 2021 $ 1,264 Change in fair value of derivative liability 732 Balance at December 31, 2021 1,996 Change in fair value of derivative liability ( 906 ) Balance at December 31, 2022 $ 1,090 Indemnification Holdback and Contingent Milestone Liabilities In May 2022, in connection with the acquisition of Satiogen (refer to Note 7 “Asset Acquisitions” for further information), the Company recorded at fair value liabilities related to the Company’s common stock issuable upon satisfaction of certain purchase price adjustments and indemnification obligations that may arise during the 12 month period following the asset acquisition date (“Indemnification Holdback”) and contingent consideration upon achievement of a certain milestone (“Contingent Milestone”). The fair value of the Indemnification Holdback and the Contingent Milestone was classified within Level 3 of the fair value hierarchy and was estimated based upon the value of the Company’s common stock price. The fair value of the Indemnification Holdback was additionally determined based on management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date, while the fair value of the Contingent Milestone was additionally determined based upon management’s estimate of the probability of the milestone being met. The fair value of the Indemnification Holdback and Contingent Milestone was initially measured on May 20, 2022, the date on which the Company completed the acquisition of Satiogen. The Company assesses the fair value of the Indemnification Holdback and Contingent Milestone each reporting period until resolution of the related contingency and changes in fair value are recorded in other income (expense), net in the accompanying consolidated statements of operations. As of December 31, 2022, the contingency for the Contingent Milestone was resolved and the fair value of the liability was determined based solely upon the closing price of the Company’s common stock. The following table provides a summary of the changes in the estimated fair value of the Indemnification Holdback and Contingent Milestone liability (in thousands): Indemnification Holdback Liability Contingent Milestone Liability Balance at January 1, 2022 $ — $ — Initial recognition 831 4,600 Change in fair value ( 214 ) ( 700 ) Transfer out of Level 3 fair value hierarchy — ( 3,900 ) Balance at December 31, 2022 $ 617 $ — The Indemnification Holdback and Contingent Milestone liabilities are included in other liabilities in the accompanying consolidated balance sheets as of December 31, 2022. The change in fair value was recorded in Other income (expense), net in the consolidated statements of operations. |
Financial Instruments
Financial Instruments | 12 Months Ended |
Dec. 31, 2022 | |
Financial Instruments, Owned, at Fair Value, by Type, Alternative [Abstract] | |
Financial Instruments | 4. Financial Instruments The fair value and amortized cost of cash equivalents and available-for-sale investments by major security type are presented in the following table (in thousands): December 31, 2022 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market fund $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,980 — ( 5 ) 4,975 Commercial paper 74,386 — — 74,386 U.S. government bonds 44,579 — ( 225 ) 44,354 Total cash equivalents and investments $ 248,172 $ — $ ( 230 ) $ 247,942 Classified as: Cash equivalents $ 24,226 Cash equivalents - restricted 100,000 Short-term investments 123,716 Total cash equivalents, restricted cash equivalents and investments $ 247,942 December 31, 2021 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market fund $ 128,420 $ — $ — $ 128,420 Commercial paper 115,221 — — 115,221 U.S. government bonds 14,999 — ( 36 ) 14,963 Total cash equivalents and investments $ 258,640 $ — $ ( 36 ) $ 258,604 Classified as: Cash equivalents $ 28,420 Cash equivalents - restricted 100,000 Short-term investments 125,201 Long-term investments 4,983 Total cash equivalents, restricted cash equivalents and investments $ 258,604 As of December 31, 2022, the remaining contractual maturities of available-for-sale debt securities were less than 12 months. During the years ended December 31, 2022 and 2021, there have been no significant realized gains or losses on available-for-sale investments, no investments have been in a continuous unrealized loss position for more than 12 months, and the Company did no t recognize any other-than-temporary impairment losses on these securities. |
Balance Sheet Components
Balance Sheet Components | 12 Months Ended |
Dec. 31, 2022 | |
Balance Sheet Components [Abstract] | |
Balance Sheet Components | 5. Balance Sheet Components Inventory Inventory consists of the following (in thousands): December 31, 2022 2021 Raw material $ — $ 856 Work in progress 5,351 570 Finished goods 214 87 Total inventory $ 5,565 $ 1,513 Accrued Expenses Accrued expenses consist of the following (in thousands): December 31, 2022 2021 Accrued milestone payments $ 15,000 $ - Accrued compensation and related benefits 14,660 9,988 Accrued clinical trials 8,319 6,732 Accrued professional service fees 5,372 2,458 Accrued rebates payable 4,284 265 Accrued contract manufacturing and non-clinical costs 3,927 4,635 Accrued royalties payable 2,456 345 Accrued collaboration funding — 6,300 Total accrued expenses $ 54,018 $ 30,723 |
Revenue Interest Purchase Agree
Revenue Interest Purchase Agreement | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Revenue Interest Purchase Agreement | 6. Revenue Interest Purchase Agreement I n December 2020, the Company entered into the RIPA, as amended in September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the Purchasers, and the Purchasers to obtain financing for the commercialization and further development of Livmarli and other working capital needs. Pursuant to the RIPA, the Company has received $ 115.0 million consisting of an upfront payment of $ 50.0 million in December 2020 and $ 65.0 million in April 2021 associated with the acceptance for filing by the FDA of a New Drug Application for Livmarli for the treatment of cholestatic pruritus in patients with ALGS, less certain transaction expenses . The Company was entitled to receive an additional $ 35.0 million upon FDA approval of Livmarli, which it elected to forgo. The Company was also entitled to receive up to approximately $ 50.0 million at the option of the Purchasers to finance in-licenses or other acquisitions on or prior to December 31, 2022, which the Company did not request. As consideration for such payments, the Purchasers have the right to receive certain revenue interests (the "Revenue Interests") from the Company based on annual product sales, net of Livmarli, which will be tiered payments (the “Revenue Interest Payments”) based on whether such annual product sales, net are (i) less than or equal to $ 350.0 million (“Tier 1”), (ii) exceeding $ 350.0 million and less than or equal to $ 1.1 billion (“Tier 2”), or (iii) exceeding $ 1.1 billion (“Tier 3”). The Revenue Interest Payments will initially be 9.75 % (at Tier 1) and 2.0 % (at Tier 2 and Tier 3) of such annual net sales. If the Purchasers have received Revenue Interest Payments in an amount equal to or greater than 110.0% of the total payments actually made by the Purchasers to the Company, exclusive of transaction expenses (the “Cumulative Purchaser Payments”), on or prior to December 31, 2026, the Revenue Interests shall be reduced to 2.0 % at Tier 1 and 0.0 % at Tier 3 for all subsequent calendar years beginning on January 1, 2027. If the Purchasers have not received Revenue Interest Payments in an amount equal to or greater than 110.0 % of the Cumulative Purchaser Payments on or prior to December 31, 2026, the Revenue Interests shall be increased for all subsequent calendar years beginning on January 1, 2027 to a single defined rate (with no separate tiers) that would have provided the Purchasers with an amount equal to 110.0% of the Cumulative Purchaser Payments on or prior to December 31, 2026 had such rate applied to Tier 1 of initial Revenue Interest Payments. The Purchasers’ rights to receive the Revenue Interest Payments shall terminate on the date on which the Purchasers have received Revenue Interest Payments of 195.0 % of the Cumulative Purchaser Payments, unless the RIPA is terminated earlier. Under the RIPA, the Company has an option (the “Call Option”) to terminate the RIPA and repurchase future Revenue Interests at any time upon advance written notice. Additionally, the Purchasers have an option (the “Put Option”) to terminate the RIPA and to require the Company to repurchase future Revenue Interests upon enumerated events such as a bankruptcy event, an uncured material breach, a material adverse effect or a change of control, or upon the 12 th anniversary of the first payment made by Purchasers. If the Put Option is exercised prior to the first anniversary of the closing date by the Purchasers (except pursuant to a change of control), the required repurchase price will be 120.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests). In all other cases, if the Put Option or the Call Option are exercised, the required repurchase price will be 175.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests), if such option is exercised prior to the third anniversary of the closing date, and 195.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests) if such option is exercised thereafter. In addition, the RIPA contains various representations and warranties, information rights, non-financial and financial covenants, indemnification obligations and other provisions that are customary for a transaction of this nature. The Purchasers’ obligations to fund the scheduled installments are subject to certain customary conditions as set forth in the RIPA. Concurrently with the RIPA, the Company entered into a Common Stock Purchase Agreement (“CSPA”) with certain affiliates of Oberland, pursuant to which the Company sold an aggregate of 509,164 shares of its common stock for an aggregate purchase price of $ 10.0 million. The $ 50.0 million upfront payment received pursuant to the RIPA and $ 10.0 million received pursuant to the CSPA was allocated between the resulting financial instruments on a relative fair value basis, with $ 49.2 million allocated to the debt under the RIPA and $ 10.8 million allocated to the common stock issued under the CSPA. The Put Option under the RIPA that is exercisable by Purchasers upon certain contingent events was determined to be an embedded derivative requiring bifurcation and separately accounted for as a single compound derivative instrument. The Company recorded the initial fair value of the derivative liability of $ 1.3 million as a debt discount, which is amortized to interest expense over the expected term of the debt using the effective interest method. As of December 31, 2022 and 2021, $ 140.4 million and $ 129.9 million, respectively, was recorded as a revenue interest liability on the accompanying consolidated balance sheets. The Company imputes interest expense associated with this liability using the effective interest rate method. The effective interest rate is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement. The interest rate on this liability may vary during the term of the agreement depending on a number of factors, including the level of forecasted product sales, net. The Company evaluates the interest rate quarterly based on its current product sales, net forecasts utilizing the prospective method. A significant increase or decrease in product sales, net will materially impact the revenue interest liability, interest expense and the time period for repayment. The Company recorded interest expense related to this arrangement of $ 16.0 million and $ 17.6 million for the years ended December 31, 2022 and 2021, respectively. The Company incurred $ 0.9 million of issuance costs in connection with the RIPA, which are being amortized to interest expense over the estimated term of the debt. Revenue Interest Payments made as a result of the Company’s product sales, net reduce the revenue interest liability. During the year ended December 31, 2022, the Company made payments of $ 5.6 million in connection with the RIPA. The following table summarizes the revenue interest liability activity during the years ended December 31, 2022 and 2021 (in thousands): Revenue interest liability at January 1, 2021 $ 47,651 Proceeds from purchaser payments 65,000 Interest expense recognized 17,590 Capitalized issuance costs ( 197 ) Revenue interest payments ( 121 ) Revenue interest liability at December 31, 2021 129,923 Interest expense recognized 15,979 Revenue interest payments ( 5,551 ) Revenue interest liability at December 31, 2022 $ 140,351 |
Asset Acquisitions
Asset Acquisitions | 12 Months Ended |
Dec. 31, 2022 | |
Asset Acquisitions [Abstract] | |
Asset Acquisitions | 7. Asset Acquisitions Assignment and License Agreement with Shire International GmbH (Takeda) In November 2018, the Company entered into an Assignment and License Agreement (the “Shire Agreement”) with Shire International GmbH (“Shire”), which was subsequently acquired by Takeda Pharmaceutical Company Limited ("Takeda"). Under the terms of the Shire Agreement, Shire granted the Company an exclusive, royalty bearing worldwide license to develop and commercialize its two product candidates, Livmarli and volixibat. As part of the Shire Agreement, the Company was assigned license agreements held by Shire with Satiogen, Pfizer Inc. (“Pfizer”) and Sanofi-Aventis Deutschland GmbH (“Sanofi”). The Company has the right to sublicense under the Shire Agreement and additionally has the right to sublicense under the Satiogen, Pfizer and Sanofi licenses subject to the terms of those license agreements. The Company is obligated to pay Shire up to an aggregate of $ 109.5 million upon the achievement of certain clinical development and regulatory milestones for Livmarli in certain indications and an additional $ 25.0 million upon regulatory approval of Livmarli for each and every other indication. In addition, the Company is required to pay up to an aggregate of $ 30.0 million upon the achievement of certain clinical development and regulatory milestones for volixibat solely for the first indication sought. Upon commercialization, the Company is obligated to pay Shire product sales milestones on total licensed products up to an aggregate of $ 30.0 million. The Company is also obligated to pay tiered royalties with rates ranging from low double-digits to mid-teens based upon annual worldwide net sales for all licensed products; however, these royalties are reduced in part by royalties due under the Satiogen and Sanofi licenses, as discussed below, related to Livmarli and volixibat, as applicable. The Company’s royalty obligations will continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country, expiration of any regulatory exclusivity for the licensed product in a country and ten years after the first commercial sale of a licensed product in such country. As of December 31, 2022, the Company accrued $ 15.0 million for a regulatory milestone associated with approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older. No additional milestones have been accrued as there were no potential milestones yet considered probable. There were $ 15.0 million of Livmarli development or regulatory milestones achieved during the year ended December 31, 2022, compared to $ 32.0 million for the year ended December 31, 2021. There were no volixibat development and regulatory milestones achieved during the year ended December 21, 2022, compared to $ 2.0 million during the year ended December 31, 2021. Satiogen License Through the Shire Agreement, the Company was assigned a license agreement with Satiogen pursuant to which the Company obtained an exclusive, worldwide license to certain patents and know-how, with the right to sublicense to a third party subject to certain financial considerations. Pursuant to the terms of the license agreement, the Company is obligated to pay to Satiogen up to an aggregate of $ 10.5 million upon the achievement of certain milestones, of which $ 0.5 million was for initiation of certain development activities, $ 5.0 million for the completion of regulatory approvals and $ 5.0 million for commercialization activities. Additionally, the Company will be required to pay a low single-digit royalty on net sales. The Company’s royalty obligations continue on a licensed product-by-licensed product and country-by-country basis until the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country. Royalty obligations under the Satiogen license are creditable against the royalty obligations to Shire under the Shire Agreement. The Company has not paid milestone payments pursuant to this agreement for the periods presented. In May 2022, the Company completed the merger and acquisition of Satiogen for total consideration of approximately $ 24.2 million. At acquisition, Satiogen’s assets consisted of cash and intangible assets related to developed technology. The purchase consideration consisted of 609,305 shares of the Company’s common stock issued upon the closing of the acquisition and cash consideration of $ 2.6 million, excluding $ 0.2 million of stock option exercise prices deemed to have been paid immediately prior to the acquisition, in respect of an equivalent amount of cash on the books of Satiogen, with up to an additional 32,494 shares of common stock that would have been issued upon the closing of the acquisition except the parties agreed to such shares being held back by the Company for 12 months from the acquisition date to satisfy certain purchase price adjustments and indemnification obligations that may arise during this period. Specifically, purchase price adjustments and indemnification obligations that arise will reduce the number of shares issuable by the Company at settlement in accordance with the terms of the definitive acquisition agreement. The purchase consideration also included issuance of up to an additional 199,993 shares of the Company’s common stock, contingent upon the achievement of a certain milestone by June 30, 2025, subject to adjustment to satisfy certain purchase price adjustments and indemnification obligations that may arise. In December 2022, with the approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older, the milestone was achieved. The Company issued 199,993 shares of common stock in January 2023. Through the transaction, the Company obtained all Satiogen licensing payments and Satiogen-owned intellectual property relating to Livmarli and volixibat. The transaction resulted in a reduction of total licensing royalty obligations for Livmarli and volixibat. The Company accounted for the transaction as an asset acquisition as the set of acquired assets did not constitute a business and substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets, namely, the Satiogen intangible assets comprised of intellectual property. The Company evaluated that the intellectual property assets acquired were deemed to be commercially viable and the cost of the acquisition was recorded as an intangible asset in the accompanying consolidated balance sheets as of December 31, 2022. There was no gain or loss recognized from settlement of the preexisting contractual relationship with Satiogen as the pre-existing contract was determined to be at fair value on the date of acquisition. For the year ended December 31, 2022, there were no significant expenses incurred that were approved for settlement against the Indemnification Holdback. As the number of shares potentially issuable upon the resolution of the Indemnification Holdback and the Contingent Milestone is variable, they were recorded as liabilities at their respective fair values on the date of acquisition using the Company’s common stock price. The fair value of the Indemnification Holdback was additionally determined based on management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date, while the fair value of the Contingent Milestone was additionally determined based upon management’s estimate of the probability of the milestone being met until the contingency was resolved in December 2022. The fair value of the Indemnification Holdback liability and the Contingent Milestone liability are remeasured at each reporting period until settled, with resulting changes in the fair value recorded in other income (expense) in the accompanying consolidated statements of operations. The following represents the consideration paid and allocation of purchase price for the acquisition of Satiogen (in thousands, except per share data): Issued common stock $ 15,585 Cash consideration 2,600 Indemnification Holdback 831 Contingent consideration settled in common stock 4,600 Transaction costs 545 Total purchase consideration 24,161 Assets acquired: Intangible assets - developed technology 21,561 Cash consideration 2,600 Total assets acquired $ 24,161 Pfizer License Through the Shire Agreement, the Company was assigned a license agreement with Pfizer pursuant to which the Company obtained an exclusive, worldwide license to certain Pfizer know-how with a right to sublicense. Upon commercialization of any product utilizing the licensed product, the Company will be required to pay to Pfizer a low single-digit royalty on net sales of product sold by the Company, its affiliates or sublicensees. The Company’s royalty obligations continue on a licensed product-by-licensed product basis until the eighth anniversary of the first commercial sale of such licensed product anywhere in the world. Sanofi License Through the Shire Agreement, the Company was assigned a license agreement with Sanofi pursuant to which the Company obtained an exclusive, worldwide license to certain patents and know-how with the right to sublicense to a third party subject to certain financial considerations. The Company is obligated to pay up to an aggregate of $ 36.0 million upon the achievement of certain regulatory, commercialization and product sales milestones. Additionally, upon commercialization, the Company is required to pay tiered royalties in the mid to high single-digit range based upon net sales of licensed products sold by the Company and sublicensees in a calendar year, subject to adjustments in certain circumstances. The Company’s royalty obligations continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country and ten years after the first commercial sale of a licensed product in such country. Royalty obligations under the Sanofi license are creditable against the royalty obligations to Shire under the Shire Agreement . The Company has not paid milestone payments pursuant to this agreement for the periods presented. As of December 31, 2022, no milestones had been accrued as there were no potential milestones considered probable. |
Collaboration and License Agree
Collaboration and License Agreements | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaboration and License Agreements | 8. Collaboration and License Agreements License and Collaboration Agreement with CANbridge In April 2021, the Company entered into an exclusive license and collaboration agreement with CANbridge Pharmaceuticals, Inc. (“CANbridge”). Under the terms of the agreement, CANbridge has obtained the exclusive right to develop and commercialize Livmarli within the Greater China regions (China, Hong Kong, Macau and Taiwan). In connection with the agreement, the Company received an upfront payment of $ 11.0 million, which, upon satisfaction of the performance obligation and receipt by CANbridge of the right to use and benefit from the license, was recorded as license revenue in the accompanying consolidated statements of operations. Additionally, the Company is eligible to receive up to $ 5.0 million in research and development funding, and up to $ 109.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales. The Company concluded at inception of the agreement that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in cumulative revenue. The Company will recognize any consideration related to sales-based payments when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized at the later of (i) when or as the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). The Company re-evaluates the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur. For the year ended December 31, 2022 and December 31, 2021, the Company recorded research and development funding of $ 0.9 million and $ 1.9 million, respectively, payable by CANbridge to the Company which is reflected as a reduction of research and development expense in the accompanying consolidated statements of operations. As of December 31, 2022 and 2021, such research and development funding of $ 0.2 million and $ 0.8 million, respectively, was recorded as a receivable which was included in accounts receivable on the accompanying consolidated balance sheets. In January 2022, CANbridge achieved a regulatory milestone, triggering a milestone payment to the Company of $ 2.0 million, which was recorded as license revenue on the accompanying consolidated statements of operations for the year ended December 31, 2022. License and Collaboration Agreement with GC Biopharma In July 2021, the Company entered into an exclusive license and collaboration agreement with GC Biopharma. Under the terms of the agreement, GC Biopharma has obtained the exclusive right to develop and commercialize Livmarli within South Korea for ALGS, PFIC, and BA. In connection with the agreement, the Company received a $ 5.0 million upfront payment, which, upon satisfaction of the performance obligation and receipt by GC Biopharma of the right to use and benefit from the license, was recorded as license revenue in the accompanying consolidated financial statements of operations for the year ended December 31, 2021. There was no license revenue recorded for the year ended December 31, 2022. Additionally, the Company is entitled to certain research and development funding and up to $ 23.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales. At inception of the agreement, the Company concluded that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in cumulative revenue for this contract when the uncertainty is resolved in the future. The Company will recognize any consideration related to sales-based payments (including milestones and royalties) when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized on the later to occur of satisfaction of the performance obligation or the occurrence of the related sales. The Company will re-evaluate the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur. For the years ended December 31, 2022 and December 31, 2021, no adjustments were made to the transaction price. For the years ended December 31, 2022 and December 31, 2021, the Company recorded research and development funding of zero and $ 0.3 million, respectively, payable by GC Biopharma to the Company which is reflected as a reduction of research and development expense in the accompanying consolidated statements of operations. As of December 31, 2022 and December 31, 2021, such research and development funding of zero and $ 0.3 million, respectively, was recorded as a receivable which was included in prepaids and other current assets on the accompanying consolidated balance sheets. In February 2023, GC Biopharma achieved a regulatory milestone under this agreement triggering a milestone payment to the Company of $ 2.5 million. Licensing Agreement with Takeda In September 2021, the Company entered into an exclusive licensing agreement with Takeda for the development and commercialization of Livmarli in Japan for ALGS, PFIC, and BA. Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of Livmarli in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications. The Company is responsible for commercial supply to Takeda. In exchange, the Company is eligible to receive a percentage of Takeda’s annualized net sales, which range from high double digits declining to mid double digits over the first four years from commercial launch and thereafter remains at mid double digits. The Company fully constrained all revenues upon transfer of control of the license to Takeda, which occurred when Takeda could use and benefit from the license, and will recognize any consideration related to sales-based payments when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized on the later to occur of satisfaction of the performance obligation or the occurrence of the related sales. Option, License and Collaboration Agreement with Vivet In April 2021 , the Company entered into an Option, License and Collaboration Agreement (“Vivet Collaboration Agreement”) with Vivet Therapeutics SAS (“Vivet”). Pursuant to the Vivet Collaboration Agreement, Vivet granted the Company the exclusive option, at the Company’s discretion, to develop and subsequently commercialize Vivet’s two proprietary AAV gene therapy programs for PFIC, subtypes 3 and 2. Under the terms of the Vivet Collaboration Agreement, the Company paid an upfront fee of $ 4.2 million and agreed to provide funding to support certain research and development costs associated with the two gene therapy programs through July 2023. In December 2021, the Company elected not to exercise its option, terminating the agreement. The Company made a final payment of $ 6.3 million in January 2022 under the terms of the agreement, which was reflected in accrued expenses at December 31, 2021 on the accompanying consolidated balance sheets. For the year ended December 31, 2021, pursuant to the terms of the Vivet Collaboration Agreement, the Company recorded research and development expense of $ 18.9 million on the accompanying consolidated statements of operations. |
Leases
Leases | 12 Months Ended |
Dec. 31, 2022 | |
Leases [Abstract] | |
Leases | 9. Leases In January 2019, the Company entered into an operating lease agreement for office space which consisted of approximately 5,600 square feet (the “Initial Lease”). The lease term is approximately four year s with an option to extend the term for one five-year term, which at the time was not reasonably assured of exercise and therefore, not included in the lease term. The lease contained a tenant improvement allowance of $ 0.4 million, which has been recorded as leasehold improvements in the accompanying consolidated balance sheets with a corresponding reduction of the ROU asset at inception of the lease. Rent payments commenced in August 2019. In November 2019, the Company amended the operating lease agreement (the “Amended Agreement”) to extend the term of the Initial Lease through March 2025. This extension was accounted for as a lease modification and the Company recorded an increase to the ROU asset and lease liability of $ 0.6 million at the time of the amendment. Additionally, pursuant to the Amended Agreement, the Company expanded the office space by 5,555 square feet for a five-year term expiring in March 2025 (the “Expanded Space”). The Company accounted for the Expanded Space as a separate contract as there were material additional rights of use that were not included in the Initial Lease. The Amended Agreement contained a tenant improvement allowance of $ 0.8 million in connection with the expanded space, which has been recorded as leasehold improvements within property and equipment, net on the accompanying consolidated balance sheets with a corresponding reduction of the ROU asset at inception of the lease for the expanded space. In June 2022, the Company entered into a lease agreement for approximately 3,500 square feet of office space in Switzerland. The lease commenced in November 2022 and has a term of approximately two and a half years with no option to extend the term. The Company recorded a ROU asset and lease liability of $ 0.3 million at inception of the lease. The ROU and corresponding lease liabilities were estimated using a weighted-average incremental borrowing rate of 8.0 %. As of December 31, 2022, the Company recorded an aggregate ROU asset of $ 1.4 million and an aggregate lease liability of $ 2.2 million in the accompanying consolidated balance sheets. The weighted-average remaining lease term is 2.1 years. As of December 31, 2022, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Years Ended December 31, Undiscounted 2023 $ 1,064 2024 1,072 2025 242 Total undiscounted lease payments 2,378 Less: imputed interest ( 190 ) Total lease liability $ 2,188 Rent expense was $ 0.8 million and $ 0.7 million for the years ended December 31, 2022 and 2021, respectively. Variable lease payments for the year ended December 31, 2022 were $ 0.2 million. Variable lease payments for the year ended December 31, 2021 were immaterial. |
Stockholders_ Equity (Deficit)
Stockholders’ Equity (Deficit) | 12 Months Ended |
Dec. 31, 2022 | |
Equity [Abstract] | |
Stockholders’ Equity (Deficit) | 10. Stockholders’ Equity (Deficit) Common Stock In August 2020, the SEC declared effective a registration statement on Form S-3 (“Shelf Registration”) covering the sale of up to $ 300.0 million of the Company’s securities. Also, in August 2020, the Company entered into a sales agreement (“Sales Agreement”) with SVB Leerink LLC (“SVB Leerink”) pursuant to which the Company may elect to issue and sell, from time to time, shares of common stock having an aggregate offering price of up to $ 75.0 million under the Shelf Registration through SVB Leerink acting as the sales agent and/or principal. During the year ended December 31, 2021, the Company did not issue or sell any shares of common stock pursuant to the Sales Agreement. During the year ended December 31, 2022, the Company issued and sold 1,160,915 shares of common stock pursuant to the Sales Agreement at a weighted-average price of $ 19.01 per share, resulting in aggregate gross proceeds to the Company of $ 22.1 million. The net proceeds for the year ended December 31, 2022 to the Company after deducting sales commissions to SVB Securities and other issuance expenses were approximately $ 21.3 million. Through December 31, 2022, the Company has issued and sold an aggregate of 1,466,884 shares of common stock pursuant to the Sales Agreement, resulting in aggregate gross proceeds to the Company of $ 28.7 million. The remaining capacity under the Sales Agreement is approximately $ 46.3 million as of December 31, 2022. In January and February 2023, the Company sold 658,206 shares of common stock pursuant to the Sales Agreement at a weighted-average price of $ 22.79 per share, resulting in net proceeds of $ 14.5 million. In December 2020, the Company completed an underwritten public offering of its common stock pursuant to the Shelf Registration. The Company issued and sold 3,750,000 shares of common stock at a public offering price of $ 20.00 per share, resulting in net proceeds of $ 70.0 million after deducting underwriting discounts, commissions and offering expenses. In addition, the Company granted the underwriters an option, exercisable for 30 days, to purchase up to 562,500 additional shares of its common stock at the public offering price, less the underwriting discounts and commissions. In January 2021, the underwriters partially exercised their option and purchased 375,654 shares of the Company’s common stock at a price of $ 20.00 per share, resulting in net proceeds of $ 7.1 million after deducting underwriting discounts. In August 2022, the Company completed an underwritten public offering of its common stock pursuant to the Shelf Registration. The Company issued and sold 3,478,261 shares of common stock at a public offering price of $ 23.00 per share. In addition, the Company granted the underwriters an option, exercisable for 30 days, to purchase up to 521,739 additional shares of its common stock at the public offering price, less the underwriting discounts, commissions and offering expenses, which the underwriters exercised in full. The underwritten public offering, including the underwriters’ exercise of their option, resulted in net proceeds to the Company of $ 86.1 million after deducting underwriting discounts, commissions and offering expenses. As of December 31, 2022 and 2021, zero and 122,464 shares of common stock, respectively, were subject to repurchase by the Company. The unvested stock liability related to these shares is immaterial to all periods presented. Common Stock Reserved for Issuance Common stock reserved for issuance is as follows: December 31, 2022 2021 Stock options and restricted stock units issued and outstanding 8,955,557 6,940,566 Reserved for future stock awards or option grants 1,596,947 2,434,619 Reserved for employee stock purchase plan 1,157,570 911,138 Common stock held back in connection with asset acquisition 31,638 — Common stock issuable as contingent consideration in connection with asset acquisition 199,993 — 11,941,705 10,286,323 |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | 11. Stock-Based Compensation Equity Incentive Plans In November 2018, the Company adopted the 2018 Equity Incentive Plan (the “2018 Plan”) which permits the granting of stock awards and incentive and nonstatutory stock options to employees, directors and consultants of the Company. In July 2019, the Company’s board of directors and stockholders approved and adopted the 2019 Equity Incentive Plan (the “2019 Plan”). The 2019 Plan became effective on July 17, 2019. Under the 2019 Plan, the Company may grant stock options, stock appreciation rights, restricted stock, restricted stock units and other stock or cash-based awards to individuals who are then employees, officers, directors or consultants of the Company. Shares subject to outstanding awards under the 2018 Plan as of the effective date of the 2019 Plan that are subsequently canceled, forfeited or repurchased by the Company will be added to the shares reserved under the 2019 Plan. In addition, the number of shares of common stock available for issuance under the 2019 Plan will be automatically increased on the first day of each calendar year during the ten-year term of the 2019 Plan, beginning with January 1, 2020 and ending with January 1, 2029 , by an amount equal to 5 % of the outstanding number of shares of the Company’s common stock on December 31st of the preceding calendar year or such lesser amount as determined by the Company’s board of directors. As of December 31, 2022, 1,121,179 shares of common stock were available for issuance under the 2019 Plan. In March 2020, the compensation committee of the Company’s board of directors approved and adopted the 2020 Inducement Plan (the “2020 Inducement Plan”). Under the 2020 Inducement Plan, the Company may grant nonstatutory stock options, stock appreciation rights, restricted stock and restricted stock units to new employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4). At adoption, the 2020 Inducement Plan authorized 750,000 shares of the Company’s common stock for future issuance. In 2021 and 2020, the Company’s board of directors authorized an additional 1,000,000 and 750,000 shares of the Company’s common stock for future issuance, respectively. As of December 31, 2022, 475,768 shares of common stock were available for issuance under the 2020 Inducement Plan. Stock Options The following table summarizes stock option activity during the year ended December 31, 2022 (in thousands, except share and per share data): Number of Weighted- Weighted- Aggregate Outstanding as of December 31, 2021 6,870,617 $ 12.56 8.0 $ 32,637 Granted 2,174,350 $ 18.02 Exercised ( 357,934 ) $ 15.19 Canceled and forfeited ( 346,950 ) $ 18.39 Outstanding as of December 31, 2022 8,340,083 $ 13.63 7.5 $ 51,645 Vested and exercisable as of December 31, 2022 4,578,478 $ 10.39 6.7 $ 42,123 Intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that had exercise prices that were lower than the per share fair value of the common stock on the date of exercise. The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2022 and 2021 was $ 12.73 and $ 13.39 per share, respectively. The total intrinsic value of options exercised during the years ended December 31, 2022 and 2021 was $ 3.0 million and $ 1.3 million, respectively. As of December 31, 2022, the total unrecognized stock-based compensation related to unvested stock option awards granted was $ 41.1 million, which the Company expects to recognize over a weighted-average period of approximately 2.4 years. The fair value of each employee and non-employee stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model. Due to the Company’s limited operating history and a lack of company specific historical and implied volatility data, the expected stock price volatility was based upon the weighting of the Company’s historical volatility and the historical volatility of a peer group of publicly traded companies. The historical volatility data was computed using the daily closing prices for the Company’s and its peer companies’ shares during the equivalent period of the calculated expected term of the stock-based awards. Due to the lack of historical exercise history, the expected term of the Company’s stock options for employees has been determined utilizing the “simplified” method for awards. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is zero based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. The following assumptions were used to estimate the fair value of stock option awards granted during the following periods: Year ended December 31, 2022 2021 Exercise price $ 15.50 -$ 27.46 $ 14.05 -$ 21.22 Expected term (in years) 5.5 - 6.1 5.5 - 6.1 Expected volatility 80.86 %- 83.20 % 82.76 %- 94.06 % Risk-free interest rate 1.46 %- 4.10 % 0.62 %- 1.34 % Expected dividend yield — — Restricted Stock Units The following table summarizes the activity under the Company’s restricted stock units for the year ended December 31, 2022: Number of Weighted-Average Grant Date Fair Value per Share Unvested and Outstanding as of December 31, 2021 $ 69,949 $ 18.57 Granted 586,452 18.24 Vested — — Cancelled/Forfeited ( 40,927 ) 16.92 Unvested and Outstanding as of December 31, 2022 $ 615,474 $ 18.36 The fair value of restricted stock unit ("RSU") awards made to employees and nonemployees is equal to the closing market price of the Company’s common stock on the grant date. As of December 31, 2022, the total unrecognized stock-based compensation related to restricted stock unit awards granted was $ 7.8 million, which the Company expects to recognize over a weighted-average period of approximately 2.06 years. 2019 Employee Stock Purchase Plan In July 2019, the Company’s board of directors and stockholders approved and adopted the 2019 Employee Stock Purchase Plan (“ESPP”). The ESPP became effective on July 17, 2019. A total of 500,000 shares of common stock were approved to be initially reserved for issuance under the ESPP. In addition, the number of shares of common stock available for issuance under the ESPP will be automatically increased on the first day of each calendar year during the first ten years of the term of the ESPP, beginning with January 1, 2020 and ending with January 1, 2029 , by an amount equal to the lesser of (i) 1 % of the outstanding number of shares of common stock on December 31st of the preceding calendar year, (ii) 1,500,000 shares of common stock or (iii) such lesser amount as determined by the Company’s board of directors. During the year ended December 31, 2022, 123,131 shares were issued under the ESPP. As of December 31, 2022, the Company had 1,157,570 shares available for future issuance under the ESPP. The stock-based compensation related to the ESPP for the years ended December 31, 2022 and 2021 was $ 0.7 million, respectively. Restricted Common Stock In November 2018, in connection with the issuance of Series A Preferred Stock, the Company’s founders agreed to modify their outstanding shares of common stock to include vesting provisions that require continued service to the Company in order to vest in those shares. As such, the 562,500 modified shares of common stock became compensatory upon such modification. The modified shares have a four-year vesting period and a measurement date fair value of $ 2.94 per share. For the years ended December 31, 2022 and 2021, 122,464 shares and 133,593 shares vested, respectively. The total fair value of shares vested was $ 0.4 million during each of the years ended December 31, 2022 and 2021, which was recorded as stock-based compensation in the consolidated statements of operations. As of December 31, 2022, all restricted shares were vested. Total stock-based compensation is reflected in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2022 2021 Selling, general and administrative $ 16,957 $ 13,128 Research and development 10,050 9,888 Total $ 27,007 $ 23,016 Stock-based compensation of $ 0.4 million and $ 0.1 million was capitalized into inventory for the years ended December 31, 2022 and 2021, respectively. Capitalized stock-based compensation is recognized in cost of sales when the related product is sold. |
Gain from Sale of Priority Revi
Gain from Sale of Priority Review Voucher | 12 Months Ended |
Dec. 31, 2022 | |
Gain (Loss) on Sale of Investments [Abstract] | |
Gain from Sale of Priority Review Voucher | 12. Gain from Sale of Priority Review Voucher In November 2021, the Company entered into a definitive agreement to sell the PRV that it received from the FDA in connection with the approval of Livmarli for the treatment of cholestatic pruritus in patients with ALGS one year of age and older, for cash proceeds of $ 110.0 million. In December 2021, the Company completed its sale of the PRV and received net proceeds of $ 108.0 million, after deducting commission costs, which was recorded as a gain within other income (loss) in the accompanying consolidated statements of operations. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2022 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 13. Income Taxes The Company’s losses before provision for income taxes are as follows (in thousands): Year Ended December 31, 2022 2021 U.S. loss before taxes $ ( 142,661 ) $ ( 84,217 ) Foreign income before taxes 590 266 Loss before income taxes $ ( 142,071 ) $ ( 83,951 ) The income tax (expense) benefit applicable to income before income taxes consists of the following: Year Ended December 31, 2022 2021 Current income taxes: State $ 18 $ — Foreign 157 37 Total current 175 37 Deferred taxes: Federal ( 5,503 ) — State ( 1,078 ) — Total deferred ( 6,581 ) — Income tax (benefit) expense $ ( 6,406 ) $ 37 The Company acquired Satiogen in a tax-free reorganization, whereby the Company did not receive a step-up in tax basis of the acquired intangible assets, resulting in a deferred tax liability of $ 6.6 million. The deferred tax liability provided an additional source of taxable income to support the realization of the pre-existing deferred tax assets. As a result, a portion of the Company’s valuation allowance was released and a $ 6.6 million tax benefit was recorded for the year ended December 31, 2022. A reconciliation of the federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2022 2021 Federal statutory income tax rate 21.00 % 21.00 % State tax 7.40 1.32 Permanent differences ( 0.65 ) ( 1.70 ) Other ( 0.93 ) 0.02 Executive compensation ( 2.99 ) — Global intangible low-taxed income ( 1.43 ) — Tax credits 4.96 7.72 Change in valuation allowance ( 22.85 ) ( 28.41 ) Total tax benefit 4.51 % ( 0.05 ) % The significant components of the Company’s deferred taxes are as follows (in thousands): December 31, 2022 2021 Deferred tax assets: Net operating losses $ 34,522 $ 31,819 Tax credit carryforwards 26,747 18,501 Capitalized research and development 23,569 — Stock-based compensation 6,726 5,815 Intangibles 4,733 10,277 Accrued expenses 3,527 4,027 Interest limitation attributes 3,127 — Lease liability 460 543 Total deferred tax assets 103,411 70,982 Deferred tax liabilities: Operating lease right-of-use assets ( 272 ) ( 319 ) Fixed assets ( 100 ) ( 138 ) Total deferred tax liabilities ( 372 ) ( 457 ) Valuation allowance ( 103,039 ) ( 70,525 ) Net deferred tax assets $ — $ — The valuation allowance increased by $ 32.5 million and $ 23.9 million for the years ended December 31, 2022 and 2021, respectively. The tax benefit of deductible temporary differences or carryforwards is recorded as a deferred tax asset to the extent that management assesses the realization is “more likely than not.” Future realization of the tax benefit ultimately depends on the existence of sufficient taxable income within the period available under the tax law. At December 31, 2022 and 2021, the Company has set up valuation allowances against all federal and state net deferred tax assets, because based on all available evidence, these deferred tax assets are not more than likely to be realizable. The Company had federal, California and other state net operating loss carryforwards of approximately $ 153.1 million, $ 19.1 million and $ 17.7 million at December 31, 2022, and $ 150.0 million, $ 2.2 million and $ 4.5 million at December 31, 2021, respectively. Federal losses do not expire, and California net operating and other state income net operating losses will begin to expire in 2038 and 2032 , respectively, if not utilized. The Company also has federal general business credit and California research and development credit carryforwards totaling $ 32.5 million and $ 4.2 million at December 31, 2022, and $ 23.1 million and $ 2.1 million at December 31, 2021, respectively. The federal research and development credit carryforwards will begin to expire in 2039, unless previously utilized. The California research credits do not expire. In general, if the Company experiences a greater than 50 percentage point aggregate change in ownership of certain significant stockholders over a three-year period (a “Section 382 ownership change”), utilization of its pre-change NOL carryforwards and the research and development credit carryforwards is subject to an annual limitation under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, and similar state laws. The annual limitation generally is determined by multiplying the value of the Company’s stock at the time of such ownership change, subject to certain adjustments, by the applicable long-term tax-exempt rate. Such limitations may result in expiration of a portion of the NOL carryforwards and research and development credit carryforwards before utilization and may be material. As of December 31, 2022, the Company determined that it has not experienced an ownership change and determined that NOLs and tax credits are not subject to a limitation pursuant to Section 382. The Company recognizes the financial statements effects of a tax position when it is more likely than not, based on technical merits, that the position will be sustained upon examination. A reconciliation of the Company’s unrecognized tax benefits is as follows (in thousands): Year Ended December 31, 2022 2021 Balance at beginning of year $ 6,399 $ 3,933 (Decrease) related to prior year tax positions — — Increases related to current year tax positions 2,855 2,466 Balance at end of year $ 9,254 $ 6,399 The Company has considered the amounts and probabilities of the outcomes that can be realized upon ultimate settlement with the tax authorities and determined unrecognized tax benefits primarily related to credits should be established as noted in the summary roll-forward above. The Company’s effective income tax rate would not be impacted if the unrecognized tax benefits were recognized in 2022 and 2021, as the Company is in a full valuation allowance position. The Company files federal, state and foreign income tax returns in jurisdictions with varying statutes of limitations. All of the Company’s tax returns in all jurisdictions remain open to examination since inception. The Company’s policy is to recognize interest expense and penalties related to income tax matters as tax expense. As of December 31, 2022 and 2021, there were no significant accruals for interest related to unrecognized tax benefits or tax penalties. The Company has no t provided U.S. income or foreign withholding taxes on the undistributed earnings of its foreign subsidiaries as of December 31, 2022 and 2021, because it intends to permanently reinvest such earnings outside of the U.S. If these foreign earnings were to be repatriated in the future, the related U.S. tax liability will be immaterial, due to the participation exemption put in place in the Tax Act. |
Contingencies
Contingencies | 12 Months Ended |
Dec. 31, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | |
Contingencies | 14. Contingencies The Company is subject to potential liabilities under government regulations and various claims and legal actions that are pending or may be asserted from time-to-time. These matters arise in the ordinary course and conduct of the Company’s business and may include, for example, commercial, intellectual property, and employment matters. The Company intends to defend itself vigorously in such matters and when warranted, take legal action against others. Furthermore, the Company regularly assesses contingencies to determine the degree of probability and range of possible loss for potential accrual in its financial statements. An estimated loss contingency is accrued in the Company’s financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. The Company does not accrue amounts for liabilities that it does not believe are probable. Litigation is inherently unpredictable, and unfavorable resolutions could occur. As a result, assessing contingencies is highly subjective and requires judgment about future events. During the periods presented, the Company has not recorded any accrual for loss contingencies associated with such government regulations, claims or legal actions, determined that an unfavorable outcome is probable or reasonably possible, or determined that the amount or range of any possible loss is reasonably estimable. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. The Company translates the financial statements of its foreign subsidiaries using end-of-period exchange rates for assets and liabilities and average exchange rates during each reporting period for results of operations. All significant intercompany balances and transactions among the consolidated entities have been eliminated in consolidation. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”). |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the financial statements and accompanying notes. These estimates and assumptions are based upon historical experience, knowledge of current events and various other factors 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 and the recording of expenses that are not readily apparent from other sources. Actual results could differ materially from those estimates. The Company’s consolidated financial statements as of and for the year ended December 31, 2022 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, higher interest rates, foreign exchange rate fluctuations and the COVID-19 pandemic. The duration and the scope of these conditions cannot be predicted; therefore, the extent to which these conditions will directly or indirectly impact the Company’s business, results of operations and financial condition, is uncertain. The Company is not aware of any specific event or circumstance that would require an update to its estimates, judgments and assumptions or a revision of the carrying value of the Company’s assets or liabilities as of the date of this filing. |
Cash Cash Equivalents and Restricted Cash Equivalents | Cash, Cash Equivalents and Restricted Cash Equivalents The Company considers all highly liquid investments that are readily convertible into cash without penalty and with original maturities of three months or less at the date of purchase to be cash equivalents. The carrying amounts reported in the consolidated balance sheets for cash and cash equivalents are valued at cost, which approximate their fair value. Restricted cash equivalents consist of deposits placed in a segregated bank account as required under the terms of the Company’s Revenue Interest Purchase Agreement ("RIPA"), as amended September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the purchasers party thereto (the "Purchasers"), and the Purchasers in connection with the sale of the Priority Review Voucher ("PRV") in December 2021. The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the consolidated balance sheets that together reflect the same amounts shown in the consolidated statements of cash flows (in thousands): December 31, 2022 2021 Cash and cash equivalents $ 28,003 $ 31,340 Restricted cash equivalents 100,000 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 128,003 $ 131,340 |
Concentrations of Credit Risk and Off-Balance Sheet Risk | Concentrations of Credit Risk and Off-Balance Sheet Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, accounts receivable and investments. The Company limits the amount of credit exposure by investing cash that is not required for immediate operating needs in money market funds, government obligations and/or commercial paper with short maturities. Additionally, the Company has established guidelines regarding diversification of its investments and their maturities, which are designed to maintain principal and maximize liquidity. To date, the Company has not experienced any losses associated with this credit risk and continues to believe that this exposure is not significant. The Company relies on a specialty pharmacy and a single distributor for all of the Company’s sales of Livmarli in the United States as well as a single distributor for sales outside the United States. The Company sources materials and services through several vendors. Certain materials are sourced from a single vendor. The loss of certain vendors could result in a temporary disruption of the Company’s commercialization efforts. For the years ended December 31, 2022 and 2021, the Company had one customer that accounted for approximately 23% and zero, respectively, of accounts receivable. For the years ended 2022 and 2021, the Company did not have revenue attributable to any one customer in excess of 10% of sales. |
Investments | Investments The Company classifies all investments in securities as available-for-sale, as the sale of such securities may be required prior to maturity. Management determines the appropriate classification of its investments in debt securities at the time of purchase. Investments with original maturities beyond three months at the date of purchase and which mature at, or less than twelve months from the balance sheet date, are classified as a current asset. Investments are recorded at fair value, with unrealized gains and losses reported as accumulated other comprehensive income (loss) until realized. The Company periodically evaluates whether declines in fair values of its available-for-sale securities below their cost basis are other-than-temporary and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. This evaluation consists of several qualitative and quantitative factors regarding the severity and duration of the unrealized loss as well as the Company’s ability and intent to hold the available-for-sale security until a forecasted recovery occurs. Additionally, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale securities before recovery of its amortized cost basis. The cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, are included in interest income. Realized gains and losses from the sale of available-for-sale securities, if any, are determined on a specific identification basis and are also included in interest income (loss). To date, the Company has no t identified any other than temporary declines in fair value of its investments. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments Fair value is defined as an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement determined based on assumptions that market participants would use in pricing an asset or liability. The following fair value hierarchy distinguishes between observable and unobservable inputs, which are categorized in one of the following levels: Level 1: Observable inputs (unadjusted) such as quoted prices in active markets for identical assets or liabilities; Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly for similar assets or liabilities; and Level 3: Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. |
Accounts Receivable | Accounts Receivable Accounts receivable are recorded net of allowances for sales discounts and any allowance for doubtful accounts. Management estimates the allowance for doubtful accounts based on existing contractual payment terms, actual payment patterns of customers and individual customer circumstances. To date, an allowance for doubtful accounts has not been material. |
Inventory | Inventory Inventory is valued at the lower of cost or net realizable value, with cost determined on a first-in, first-out (FIFO) basis. The Company periodically reviews the composition of inventory to identify excess, obsolete, slow-moving or otherwise unsaleable items. If unsaleable items are observed and there are no alternate uses for the inventory, the Company will record a write-down to net realizable value in the period that the decline in value is recognized through a charge to cost of sales. The determination of whether inventory costs will be realizable requires estimates by management. If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required. Prior to the initial regulatory approval for Livmarli, the Company expensed costs relating to raw materials and production of inventory as research and development expense in the accompanying consolidated statements of operations, in the period incurred. The Company expects it will deplete such inventories within 12 months of the balance sheet date. |
Property and Equipment | Property and Equipment Property and equipment are recorded at cost less accumulated depreciation. Depreciation is computed on a straight-line basis over the estimated useful lives of the related assets, ranging from three to five years . Leasehold improvements are amortized over the shorter of their useful lives or the related lease term. As of December 31, 2022, property and equipment consisted primarily of leasehold improvements of $ 1.3 million and furniture and equipment of $ 0.6 million. As of December 31, 2021, property and equipment consisted primarily of leasehold improvements of $ 1.3 million and furniture and equipment of $ 0.2 million. Accumulated depreciation as of December 31, 2022 and 2021 was $ 1.0 million and $ 0.7 million, respectively. Depreciation expense was $ 0.3 million for the years ended December 31, 2022 and 2021, respectively. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets Long-lived assets are reviewed for indications of possible impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability is measured by comparison of the carrying amounts to the future undiscounted cash flows attributable to these assets. An impairment loss is recognized to the extent an asset group is not recoverable, and the carrying amount exceeds the projected discounted future cash flows arising from these assets. There were no impairments of long-lived assets for any of the periods presented. |
Intangibles Assets, Net | Intangible Assets, Net The Company accounts for asset acquisitions that do not meet the definition of a business using the cost accumulation method, whereby the cost of the acquisition, including certain transaction costs, is allocated to the asset (or assets) acquired on the basis of its (or their) relative fair value(s) on the measurement date. No goodwill is recognized in an asset acquisition. Intangible assets are measured at their fair values as of the acquisition date or, in the case of commercial milestone payments, the date they become due. The evaluation of intangible assets includes assessing the amortization period for which the asset is expected to contribute to the future cash flows of the Company. Intangible assets with finite useful lives are amortized over their estimated useful lives, primarily on a straight-line basis when the Company is unable to reliably estimate the pattern of cash flow. The Company tests its finite lived intangible assets for impairment annually or if events or changes in circumstances indicate that it is more likely than not that the asset is impaired. If it is determined that the asset is impaired, the carrying value is written down to its estimated fair value, with the related impairment charge recognized in the consolidated statements of operations in the period in which the impairment occurs. The Company has not recorded any impairments to its intangible assets for any of the periods presented. The following table provides detail of the carrying amount of the Company’s intangible assets (in thousands): December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Value Intangible asset - commercial milestone payments $ 34,000 $ ( 1,333 ) $ 32,667 Intangible assets - Satiogen acquisition 28,107 ( 1,820 ) 26,287 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 December 31, 2021 Gross Carrying Value Accumulated Amortization Net Carrying Amount Intangible asset - commercial milestone payments $ 19,000 $ ( 260 ) $ 18,740 Total intangible assets $ 19,000 $ ( 260 ) $ 18,740 As of December 31, 2022, the remaining weighted-average amortization period of the Company’s intangible assets was 11.9 years. The remaining weighted-average amortization periods of the commercial milestone payments and the Satiogen Pharmaceutical, Inc. ("Satiogen") acquisition intangible assets were 17.1 years and 8.4 years, respectively. Amortization expense was $ 2.9 million and $ 0.3 million for the years ended December 31, 2022 and 2021, respectively, and was included in cost of sales on the accompanying consolidated statements of operations. The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of December 31, 2022 (in thousands): Years Ended December 31, Amount 2023 $ 5,035 2024 5,035 2025 5,035 2026 5,035 2027 5,035 Thereafter 33,779 $ 58,954 |
Leases | Leases The Company determines if a contractual arrangement is or contains a lease at inception. Operating lease right-of-use (“ROU”) assets represent the Company’s right to use an underlying asset during the lease term, and operating lease liabilities represent the Company’s obligation to make lease payments arising from the lease. Operating leases are included in ROU assets, current operating lease liabilities, and long-term operating lease liabilities on the accompanying consolidated balance sheets. Operating lease ROU assets and lease liabilities are initially recognized based on the present value of the future minimum lease payments over the lease term at commencement date calculated using the Company’s incremental borrowing rate applicable to the leased asset, unless the implicit rate is readily determinable. Operating lease ROU assets also include any lease payments made at or before lease commencement and exclude any lease incentives received. The Company determines the lease term as the noncancelable period of the lease and may include options to extend or terminate the lease when it is reasonably certain that the Company will exercise that option. Leases with a term of 12 months or less are not recognized on the consolidated balance sheets. The Company’s leases do not contain any residual value guarantees. Lease expense for minimum lease payments is recognized as rent expense on a straight-line basis over the lease term. Variable lease payments include lease operating expenses. |
Accrued Research and Development Expenses | Accrued Research and Development Expenses The Company accrues and expenses clinical trial activities performed by third parties based upon estimates of the proportion of work completed over the life of the individual study and patient enrollment rates in accordance with agreements established with clinical research organizations and clinical trial sites. The Company determines the estimates by reviewing contracts, vendor agreements and purchase orders and through discussions with internal clinical personnel and external service providers as to the progress or stage of completion of trials or services and the agreed-upon fee to be paid for such services. The Company makes estimates of accrued expenses as of each balance sheet date based on facts and circumstances known to the Company at that time. 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. The Company has not experienced any material differences between accrued costs and actual costs incurred for the periods presented. Nonrefundable advance payments for goods and services are deferred and recognized as expense in the period that the related goods are consumed or services are performed. |
Revenue Interest Liability, Net | Revenue Interest Liability, Net The revenue interest liability, net, associated with the RIPA that the Company entered into in December 2020, as amended in September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC (“Oberland”), as agent for purchasers party thereto (the “Purchasers”), and the Purchasers, is presented net of issuance costs and a debt discount on the consolidated balance sheets. The Company imputes interest expense associated with this liability using the effective interest rate method. The effective interest rate is calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement. The interest rate on the liability may vary during the term of the agreement depending on a number of factors, including the level of actual and forecasted product sales, net. The Company evaluates the interest rate quarterly based on actual product sales, net and forecast product sales, net, utilizing the prospective method. A significant increase or decrease in product sales, net will materially impact the revenue interest liability, interest expense and the time period for repayment. |
Derivative Liability | Derivative Liability The RIPA contains certain features that meet the definition of being an embedded derivative requiring bifurcation as a separate compound financial instrument apart from the RIPA. The derivative liability is initially measured at fair value on issuance and is subject to remeasurement at each reporting period with changes in fair value recognized as other income (expense) in the accompanying consolidated statements of operations as the change in fair value of derivative liability. |
Revenue Recognition | Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration the Company expects to receive in exchange for those goods or services. Product Sales, Net The Company recognizes product sales, net when the customer obtains control of our product, which occurs at a point in time, typically upon delivery of the Company’s product to the customer. Revenues from product sales are recorded at the net sales price, or the transaction price, which may include fixed or variable consideration for discounts, government rebates, co-pay assistance, returns and other allowances that are offered within contracts with a customer relating to the sale of Livmarli. Estimates of variable consideration are calculated based on the actual product sales each reporting period and the nature of the variable consideration related to those sales. Overall, these estimates reflect the Company’s best estimate of the amount of consideration to which the Company expects to be entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in product sales, net only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Estimates are reviewed and updated quarterly as additional information becomes known. Actual amounts of consideration ultimately received may differ materially from estimates. If actual results in the future vary from estimates, the Company will adjust these estimates, which would affect product sales, net and earnings in the period such variances are adjusted. Significant categories of sales discounts and allowances are as follows: Government Rebates : The Company records rebates payable under Medicaid and other government programs as a reduction of revenue at the time product revenues are generated. The Company’s rebate calculations may require estimates, including estimates of customer mix, to determine which sales will be subject to rebates and the amount of such rebates. The Company updates its estimates and assumptions on a quarterly basis and records any necessary adjustments to revenue in the period identified. The liability for unpaid rebates is included in accrued expenses in the accompanying consolidated balance sheets. To date, actual government rebates have not differed materially from the Company’s estimates. Other Incentives: Other incentives include a branded co-pay assistance program for eligible patients with commercial insurance in the United States. The branded co-pay assistance program assists commercially insured patients who have coverage for Livmarli and is intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance. The calculation of the accrual for co-pay assistance is based upon an identification of claims and the cost per claims associated with product that has been recognized as revenue. The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales. To date, actual other incentives have not differed materially from the Company’s estimates. Product Returns: The Company records revenue for product sales, net of estimated product returns. Customers have limited return rights related only to the product’s damage or defect identified upon delivery of the product. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. To date, actual returns have not differed materially from the Company’s estimates. License and Collaboration Arrangements The Company enters into collaborative arrangements with partners and analyzes the collaboration arrangements to assess whether they are within the scope of Collaborative Arrangements (Topic 808) ("Topic 808") and determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. The accounting for some of the activities under collaboration arrangements may be subject to Revenue from Contracts with Customers (Topic 606) (“Topic 606”) for distinct units of account that are reflective of a vendor-customer relationship. For other elements of collaboration arrangements, such as reimbursements of certain development costs, the Company generally records reimbursements received as a reduction of research and development expenses. In determining the appropriate amount of revenue to be recognized under Topic 606 as the Company fulfills its obligations under each of its agreements, the Company performs the following steps: (i) identification of the contracts with customers; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) determination and measurement of the transaction price, including any constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations in the contract; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The terms of the Company’s license and collaborative research and development agreements include upfront license fees, research, development and other funding or reimbursements, milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and sales-based events, as well as royalties on sales of commercialized products. Arrangements that include upfront payments may require deferral of revenue recognition to a future period until the Company satisfies performance obligations under these arrangements. A performance obligation is a promise in a contract to transfer a distinct good or service and is the unit of accounting in Topic 606. A contract’s transaction price is allocated among each distinct performance obligation based on relative standalone selling price and recognized as revenue when, or as, the applicable performance obligation is satisfied. If a license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenues attributed to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. At the inception of each arrangement that includes development milestone payments, the Company evaluates whether the milestones are considered probable of being achieved and estimates the amount to be included in the transaction price as variable consideration using the most likely amount method or expected value method, depending on the nature of the contingency and the variable payments. If it is probable that a significant reversal of cumulative revenue recognized for the contract would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not generally considered probable of being achieved until those approvals are received. Given the high degree of uncertainty around the occurrence of these events, the Company generally determines the milestone and other contingent amounts to be fully constrained until the uncertainty associated with these payments is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of any development milestones, and if necessary, adjusts its estimate of the transaction price. Any such adjustments would be recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment. For arrangements that include sales-based royalties, including milestone payments based on the level of sales, the Company recognizes revenue at the later of (i) when or as the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Accounting for these arrangements requires the Company to develop assumptions that require judgment to determine the stand-alone selling price of each performance obligation identified in the contract. The Company has never sold the performance obligations in its collaborative arrangements separately; therefore, an observable stand-alone selling price does not exist. Accordingly, the Company estimates a stand-alone selling price through maximizing the use of observable inputs such as market data, project cost estimates, and targeted margins. Cost of Product Sales Prior to receiving approval from the U.S. Food and Drug Administration ("FDA") in September 2021 to sell Livmarli in the United States, the Company expensed all costs incurred related to the manufacture of Livmarli as research and development expense because of the inherent risks associated with the development of a drug candidate, the uncertainty about the regulatory approval process and the lack of history for the Company of regulatory approval of drug candidates. Subsequent to receiving FDA approval, when commercialization was considered probable and the future economic benefit was expected to be realized, the Company began capitalizing inventory costs incurred. Cost of product sales consist of manufacturing costs, transportation and freight, amortization of capitalized intangible assets, royalties and indirect overhead costs associated with the manufacturing and distribution of Livmarli. Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges. |
Research and Development Expenses | Research and Development Expenses Research and development expenses consists primarily of fees paid to contract research organizations and other vendors for clinical, non-clinical and manufacturing services, salaries and employee benefits, including stock-based compensation, consultant expenses, costs related to acquiring manufacturing materials, costs related to compliance with regulatory requirements and license payments related to acquiring intellectual property rights for the Company’s product candidates. Research and development expenses are expensed as incurred. |
Selling, General and Administrative Expenses | Selling, General and Administrative Expenses Selling, general and administrative (“SG&A”) expenses relate to sales and marketing, finance, human resources, legal and other administrative activities. SG&A expenses consist primarily of personnel costs, facilities and overhead costs, outside marketing, advertising and legal expenses, and other general and administrative costs. The Company expenses the costs of advertising, including promotional expenses, as incurred. Advertising expenses were $ 4.0 million and $ 9.2 million for the years ended December 31, 2022 and 2021, respectively. |
Stock-Based Compensation | Stock-Based Compensation The Company recognizes stock-based compensation for all stock-based awards based on the grant date fair value of the award granted to employees and nonemployees, including members of its board of directors. For stock-based awards with service conditions, the fair value of the awards is recognized on a straight-line basis over the requisite service period in which the awards are expected to vest. For stock-based awards with performance vesting conditions, stock-based compensation is recognized when it is considered probable that the performance conditions will be satisfied. At each reporting period, the Company reassesses the probability of the achievement of the performance vesting conditions. Any change in stock-based compensation resulting from an adjustment in the vesting is treated as a cumulative catch-up in the period of adjustment. For stock-based awards with market conditions, stock-based compensation is recognized over the appropriate requisite service period. The Company accounts for forfeitures as they occur. The Company evaluates the grant date fair value of awards granted for impacts of any potential material non-public information at the time of grant. |
Income Taxes | Income Taxes Income taxes are recorded using the liability method, under which deferred tax assets and liabilities are determined based on differences between the financial reporting and tax basis of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. Valuation allowances are recorded against deferred tax assets, including net operating losses and tax credits, when it is determined it is more-likely-than-not that some or all of the tax benefits will not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of Financial Accounting Standards Board (“FASB”) Accounting Standards Codification No. 740, Income Taxes (“ASC 740”). When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit would more likely than not be realized assuming examination by the taxing authority. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. Interest and penalties related to unrecognized tax benefits, if any, are recorded as a component of income tax expense. |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is computed by dividing net loss attributable to common stockholders by the weighted-average shares of common stock outstanding for the period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss attributable to common stockholders by the weighted-average shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury-stock and if-converted methods. The following table sets for the computation of basic and diluted earnings per share (in thousands, except share and per share data): Year Ended December 31, 2022 2021 Numerator: Net loss, basic $ ( 135,665 ) $ ( 83,988 ) Add: Change in fair value of Holdback Indemnification liability ( 214 ) — Add: Change in fair value of Contingent Milestone liability ( 700 ) — Net loss, diluted $ ( 136,579 ) $ ( 83,988 ) Denominator: Weighted-average shares of common stock outstanding, basic 33,839,072 30,321,722 Effect of dilutive securities: Weighted-average Holdback-Indemnification shares issuable 19,590 — Weighted-average contingent shares 123,831 — Weighted-average shares of common stock outstanding, diluted 33,982,493 30,321,722 Net loss per share, basic $ ( 4.01 ) $ ( 2.77 ) Net loss per share, diluted $ ( 4.02 ) $ ( 2.77 ) In addition to the equity instruments included above, the following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: As of December 31, 2022 2021 Options to purchase common stock and restricted stock units 8,955,557 6,940,566 Common stock subject to repurchase — 122,464 Employee stock purchase plan contingently issuable 26,305 23,116 Total 8,981,862 7,086,146 |
Recent Accounting Pronouncements Not Yet Adopted | Recent Accounting Pronouncements Not Yet Adopted In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). ASU 2016-13 requires an entity to utilize a new impairment model that requires measurement and recognition of expected credit losses for most financial assets and certain other instruments, including but not limited to available-for-sale debt securities. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. The new guidance requires the use of forward-looking expected credit loss models based on historical experience, current conditions, and reasonable and supportable forecasts that affect the collectability of the reported amount, which may result in earlier recognition of credit losses under the new guidance. The new guidance also modifies the impairment models for available-for-sale debt securities and for purchased financial assets with credit deterioration since their origination. Subsequent to the issuance of ASU 2016-13, the FASB issued ASU 2018-19, Codification Improvements to Topic 326, Financial Instruments - Credit Losses . This ASU does not change the core principle of the guidance in ASU 2016-13, instead these amendments are intended to clarify and improve operability of certain topics included within the credit losses guidance. The FASB also subsequently issued ASU No. 2019-04, Codification Improvements to Topic 326, Financial Instruments—Credit Losses, Derivatives and Hedging (Topic 815), and Financial Instruments (Topic 842), which did not change the core principle of the guidance in ASU 2016-13 but clarified that expected recoveries of amounts previously written off and expected to be written off should be included in the valuation account and should not exceed amounts previously written off and expected to be written off. In March 2020, the FASB issued ASU No. 2020-3, Codification Improvements to Financial Instruments which makes narrow-scope improvements to various financial instruments topics, including the new credit losses standard and clarifies the following areas (i) the contractual term of a net investment in a lease should be the contractual term used to measure expected credit losses; (ii) when an entity regains control of financial assets sold, an allowance for credit losses should be recorded. The guidance is effective for fiscal years, and interim periods within those years, beginning after December 15, 2019 for public business entities, excluding smaller reporting companies. For smaller reporting companies, the guidance will be effective during the first quarter of 2023. The Company does not expect the new standard to have a significant impact on its consolidated financial statements and related disclosures. In October 2021, the FASB, issued Accounting Standards Update No. 2021-08, Business Combinations (Topic 805), Accounting for Contract Assets and Contract Liabilities from Contracts with Customers , which requires an entity (acquirer) to recognize and measure contract assets and liabilities acquired in a business combination in accordance with Topic 606, Revenue from Contracts with Customers. This update is effective for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years, with early adoption permitted. The amendments should be applied prospectively to business combinations occurring on or after the effective date of the amendments. The Company does not expect the new standard to have a significant impact on its consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Accounting Policies [Abstract] | |
Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash Reported Within the Consolidated Balance Sheets | The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the consolidated balance sheets that together reflect the same amounts shown in the consolidated statements of cash flows (in thousands): December 31, 2022 2021 Cash and cash equivalents $ 28,003 $ 31,340 Restricted cash equivalents 100,000 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 128,003 $ 131,340 |
Schedule Of Finite Lived Intangible Assets | The following table provides detail of the carrying amount of the Company’s intangible assets (in thousands): December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Value Intangible asset - commercial milestone payments $ 34,000 $ ( 1,333 ) $ 32,667 Intangible assets - Satiogen acquisition 28,107 ( 1,820 ) 26,287 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 December 31, 2021 Gross Carrying Value Accumulated Amortization Net Carrying Amount Intangible asset - commercial milestone payments $ 19,000 $ ( 260 ) $ 18,740 Total intangible assets $ 19,000 $ ( 260 ) $ 18,740 |
Schedule of Estimated Future Amortization Expense Associated with Intangible Assets | The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of December 31, 2022 (in thousands): Years Ended December 31, Amount 2023 $ 5,035 2024 5,035 2025 5,035 2026 5,035 2027 5,035 Thereafter 33,779 $ 58,954 |
Schedule of Computation of Basic and Diluted Earnings per Share | The following table sets for the computation of basic and diluted earnings per share (in thousands, except share and per share data): Year Ended December 31, 2022 2021 Numerator: Net loss, basic $ ( 135,665 ) $ ( 83,988 ) Add: Change in fair value of Holdback Indemnification liability ( 214 ) — Add: Change in fair value of Contingent Milestone liability ( 700 ) — Net loss, diluted $ ( 136,579 ) $ ( 83,988 ) Denominator: Weighted-average shares of common stock outstanding, basic 33,839,072 30,321,722 Effect of dilutive securities: Weighted-average Holdback-Indemnification shares issuable 19,590 — Weighted-average contingent shares 123,831 — Weighted-average shares of common stock outstanding, diluted 33,982,493 30,321,722 Net loss per share, basic $ ( 4.01 ) $ ( 2.77 ) Net loss per share, diluted $ ( 4.02 ) $ ( 2.77 ) |
Summary of Outstanding Potentially Dilutive Shares Excluded from Calculation of Diluted Net Loss Per Share | In addition to the equity instruments included above, the following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: As of December 31, 2022 2021 Options to purchase common stock and restricted stock units 8,955,557 6,940,566 Common stock subject to repurchase — 122,464 Employee stock purchase plan contingently issuable 26,305 23,116 Total 8,981,862 7,086,146 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets and Liabilities to Fair Value Measurements On Recurring Basis and Level of Input Measurements | Financial assets and liabilities subject to fair value measurements on a recurring basis and the level of inputs used in such measurements by major security type are presented in the following table (in thousands): December 31, 2022 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,975 — — 4,975 Commercial paper — 74,386 — 74,386 U.S. government bonds — 44,354 — 44,354 Total financial assets $ 129,202 $ 118,740 $ — $ 247,942 Financial liabilities: Contingent milestone liability $ 3,900 $ — $ — $ 3,900 Derivative liability — — 1,090 1,090 Indemnification holdback — — 617 617 Total financial liabilities $ 3,900 $ — $ 1,707 $ 5,607 December 31, 2021 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 128,420 $ — $ — $ 128,420 Commercial paper — 115,221 — 115,221 U.S. government bonds — 14,963 — 14,963 Total financial assets $ 128,420 $ 130,184 $ — $ 258,604 Financial liabilities: Derivative liability $ — $ — $ 1,996 $ 1,996 Total financial liabilities $ — $ — $ 1,996 $ 1,996 |
Summary of Change in Estimated Fair Value of Company's Derivative Liability Classified as Level 3 | The following table provides a summary of the change in the estimated fair value of the Company’s derivative liability, classified as Level 3 in the fair value hierarchy (in thousands): Balance at January 1, 2021 $ 1,264 Change in fair value of derivative liability 732 Balance at December 31, 2021 1,996 Change in fair value of derivative liability ( 906 ) Balance at December 31, 2022 $ 1,090 |
Summary of Changes in Fair Value classified as Level 3 | The following table provides a summary of the changes in the estimated fair value of the Indemnification Holdback and Contingent Milestone liability (in thousands): Indemnification Holdback Liability Contingent Milestone Liability Balance at January 1, 2022 $ — $ — Initial recognition 831 4,600 Change in fair value ( 214 ) ( 700 ) Transfer out of Level 3 fair value hierarchy — ( 3,900 ) Balance at December 31, 2022 $ 617 $ — |
Financial Instruments (Tables)
Financial Instruments (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
Summary of Fair Value and Amortized Cost of Cash Equivalents and Available-for-sale Investments by Major Security Type | The fair value and amortized cost of cash equivalents and available-for-sale investments by major security type are presented in the following table (in thousands): December 31, 2022 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market fund $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,980 — ( 5 ) 4,975 Commercial paper 74,386 — — 74,386 U.S. government bonds 44,579 — ( 225 ) 44,354 Total cash equivalents and investments $ 248,172 $ — $ ( 230 ) $ 247,942 Classified as: Cash equivalents $ 24,226 Cash equivalents - restricted 100,000 Short-term investments 123,716 Total cash equivalents, restricted cash equivalents and investments $ 247,942 December 31, 2021 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market fund $ 128,420 $ — $ — $ 128,420 Commercial paper 115,221 — — 115,221 U.S. government bonds 14,999 — ( 36 ) 14,963 Total cash equivalents and investments $ 258,640 $ — $ ( 36 ) $ 258,604 Classified as: Cash equivalents $ 28,420 Cash equivalents - restricted 100,000 Short-term investments 125,201 Long-term investments 4,983 Total cash equivalents, restricted cash equivalents and investments $ 258,604 As of December 31, 2022, the remaining contractual maturities of available-for-sale debt securities were less than 12 months. |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Balance Sheet Components [Abstract] | |
Schedule of Inventory | December 31, 2022 2021 Raw material $ — $ 856 Work in progress 5,351 570 Finished goods 214 87 Total inventory $ 5,565 $ 1,513 |
Schedule of Accrued Expenses | Accrued expenses consist of the following (in thousands): December 31, 2022 2021 Accrued milestone payments $ 15,000 $ - Accrued compensation and related benefits 14,660 9,988 Accrued clinical trials 8,319 6,732 Accrued professional service fees 5,372 2,458 Accrued rebates payable 4,284 265 Accrued contract manufacturing and non-clinical costs 3,927 4,635 Accrued royalties payable 2,456 345 Accrued collaboration funding — 6,300 Total accrued expenses $ 54,018 $ 30,723 |
Revenue Interest Purchase Agr_2
Revenue Interest Purchase Agreement (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Revenue Interest Liability | The following table summarizes the revenue interest liability activity during the years ended December 31, 2022 and 2021 (in thousands): Revenue interest liability at January 1, 2021 $ 47,651 Proceeds from purchaser payments 65,000 Interest expense recognized 17,590 Capitalized issuance costs ( 197 ) Revenue interest payments ( 121 ) Revenue interest liability at December 31, 2021 129,923 Interest expense recognized 15,979 Revenue interest payments ( 5,551 ) Revenue interest liability at December 31, 2022 $ 140,351 |
Asset Acquisitions (Tables)
Asset Acquisitions (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Asset Acquisitions [Abstract] | |
Schedule of Consideration Paid and Allocation of Costs | The following represents the consideration paid and allocation of purchase price for the acquisition of Satiogen (in thousands, except per share data): Issued common stock $ 15,585 Cash consideration 2,600 Indemnification Holdback 831 Contingent consideration settled in common stock 4,600 Transaction costs 545 Total purchase consideration 24,161 Assets acquired: Intangible assets - developed technology 21,561 Cash consideration 2,600 Total assets acquired $ 24,161 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Leases [Abstract] | |
Schedule of Undiscounted Future Minimum Payments under Operating Leases | As of December 31, 2022, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Years Ended December 31, Undiscounted 2023 $ 1,064 2024 1,072 2025 242 Total undiscounted lease payments 2,378 Less: imputed interest ( 190 ) Total lease liability $ 2,188 |
Stockholders_ Equity (Deficit)
Stockholders’ Equity (Deficit) (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Equity [Abstract] | |
Schedule of Common Stock Reserved for Issuance | Common stock reserved for issuance is as follows: December 31, 2022 2021 Stock options and restricted stock units issued and outstanding 8,955,557 6,940,566 Reserved for future stock awards or option grants 1,596,947 2,434,619 Reserved for employee stock purchase plan 1,157,570 911,138 Common stock held back in connection with asset acquisition 31,638 — Common stock issuable as contingent consideration in connection with asset acquisition 199,993 — 11,941,705 10,286,323 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Stock Option Activity | The following table summarizes stock option activity during the year ended December 31, 2022 (in thousands, except share and per share data): Number of Weighted- Weighted- Aggregate Outstanding as of December 31, 2021 6,870,617 $ 12.56 8.0 $ 32,637 Granted 2,174,350 $ 18.02 Exercised ( 357,934 ) $ 15.19 Canceled and forfeited ( 346,950 ) $ 18.39 Outstanding as of December 31, 2022 8,340,083 $ 13.63 7.5 $ 51,645 Vested and exercisable as of December 31, 2022 4,578,478 $ 10.39 6.7 $ 42,123 |
Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted | The following assumptions were used to estimate the fair value of stock option awards granted during the following periods: Year ended December 31, 2022 2021 Exercise price $ 15.50 -$ 27.46 $ 14.05 -$ 21.22 Expected term (in years) 5.5 - 6.1 5.5 - 6.1 Expected volatility 80.86 %- 83.20 % 82.76 %- 94.06 % Risk-free interest rate 1.46 %- 4.10 % 0.62 %- 1.34 % Expected dividend yield — — |
Summary of RSU Activity | The following table summarizes the activity under the Company’s restricted stock units for the year ended December 31, 2022: Number of Weighted-Average Grant Date Fair Value per Share Unvested and Outstanding as of December 31, 2021 $ 69,949 $ 18.57 Granted 586,452 18.24 Vested — — Cancelled/Forfeited ( 40,927 ) 16.92 Unvested and Outstanding as of December 31, 2022 $ 615,474 $ 18.36 |
Summary of Stock-based Compensation Reflected in Consolidated Statements of Operations | Total stock-based compensation is reflected in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2022 2021 Selling, general and administrative $ 16,957 $ 13,128 Research and development 10,050 9,888 Total $ 27,007 $ 23,016 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2022 | |
Income Tax Disclosure [Abstract] | |
Schedule of Losses before Provision for Income Taxes | The Company’s losses before provision for income taxes are as follows (in thousands): Year Ended December 31, 2022 2021 U.S. loss before taxes $ ( 142,661 ) $ ( 84,217 ) Foreign income before taxes 590 266 Loss before income taxes $ ( 142,071 ) $ ( 83,951 ) |
Schedule of Components of Income Tax (Expense) Benefit | The income tax (expense) benefit applicable to income before income taxes consists of the following: Year Ended December 31, 2022 2021 Current income taxes: State $ 18 $ — Foreign 157 37 Total current 175 37 Deferred taxes: Federal ( 5,503 ) — State ( 1,078 ) — Total deferred ( 6,581 ) — Income tax (benefit) expense $ ( 6,406 ) $ 37 |
Summary of Reconciliation of Federal Statutory Income Tax Rate | A reconciliation of the federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2022 2021 Federal statutory income tax rate 21.00 % 21.00 % State tax 7.40 1.32 Permanent differences ( 0.65 ) ( 1.70 ) Other ( 0.93 ) 0.02 Executive compensation ( 2.99 ) — Global intangible low-taxed income ( 1.43 ) — Tax credits 4.96 7.72 Change in valuation allowance ( 22.85 ) ( 28.41 ) Total tax benefit 4.51 % ( 0.05 ) % |
Significant Components of Deferred Taxes | The significant components of the Company’s deferred taxes are as follows (in thousands): December 31, 2022 2021 Deferred tax assets: Net operating losses $ 34,522 $ 31,819 Tax credit carryforwards 26,747 18,501 Capitalized research and development 23,569 — Stock-based compensation 6,726 5,815 Intangibles 4,733 10,277 Accrued expenses 3,527 4,027 Interest limitation attributes 3,127 — Lease liability 460 543 Total deferred tax assets 103,411 70,982 Deferred tax liabilities: Operating lease right-of-use assets ( 272 ) ( 319 ) Fixed assets ( 100 ) ( 138 ) Total deferred tax liabilities ( 372 ) ( 457 ) Valuation allowance ( 103,039 ) ( 70,525 ) Net deferred tax assets $ — $ — |
Reconciliation of Unrecognized Tax Benefits | A reconciliation of the Company’s unrecognized tax benefits is as follows (in thousands): Year Ended December 31, 2022 2021 Balance at beginning of year $ 6,399 $ 3,933 (Decrease) related to prior year tax positions — — Increases related to current year tax positions 2,855 2,466 Balance at end of year $ 9,254 $ 6,399 |
Organization and Description _2
Organization and Description of Business - Additional Information (Detail) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 USD ($) Segment | Dec. 31, 2021 USD ($) | |
Date of incorporation | May 02, 2018 | |
Number of operating segment | Segment | 1 | |
Accumulated deficit | $ 392,824 | $ 257,159 |
Cash, cash equivalents and investments | 251,700 | |
Unrestricted cash, cash equivalents and investments | $ 151,700 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) | 12 Months Ended | |||
Dec. 31, 2022 USD ($) Customer | Dec. 31, 2021 USD ($) Customer | Jun. 30, 2022 | Jan. 22, 2019 | |
Summary Of Significant Accounting Policies [Line Items] | ||||
Investments with original maturities at date of purchase to be cash equivalents | 3 months | |||
Other than temporary declines in fair value of investments | $ 0 | $ 0 | ||
Property and equipment, net | 914,000 | 981,000 | ||
Accumulated depreciation | 1,000,000 | 700,000 | ||
Depreciation Expense | 300,000 | 300,000 | ||
Impairment of long-lived assets | $ 0 | |||
weighted-average amortization period | 11 years 10 months 24 days | |||
Amortization expense | $ 2,900,000 | $ 300,000 | ||
No of customers | Customer | 1 | 1 | ||
Term of lease | 12 years | 2 years 6 months | 4 years | |
Inventory | $ 5,565,000 | $ 1,513,000 | ||
Advertising Expense | $ 4,000,000 | 9,200,000 | ||
Intangible Asset Milestone Payments [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
weighted-average amortization period | 17 years 1 month 6 days | |||
Intangible Assets Satiogen Acquisition [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
weighted-average amortization period | 8 years 4 months 24 days | |||
Leasehold Improvements | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, net | $ 1,300,000 | 1,300,000 | ||
Furniture and Fixtures | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, net | $ 600,000 | $ 200,000 | ||
Minimum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, useful life | 3 years | |||
Maximum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, useful life | 5 years |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash Reported Within the Consolidated Balance Sheets (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Cash and cash equivalents | $ 28,003 | $ 31,340 | |
Restricted cash equivalents | 100,000 | 100,000 | |
Cash, cash equivalents, and restricted cash equivalents | $ 128,003 | $ 131,340 | $ 142,086 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Schedule Of Finite Lived Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Summary Of Significant Accounting Policies [Line Items] | ||
Gross Carrying Value | $ 62,107 | $ 19,000 |
Accumulated Amortization | (3,153) | (260) |
Net Carrying Value | 58,954 | 18,740 |
Intangible Asset Milestone Payments Member | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Gross Carrying Value | 34,000 | 19,000 |
Accumulated Amortization | (1,333) | (260) |
Net Carrying Value | 32,667 | $ 18,740 |
Intangible Assets Satiogen Acquisition Member | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Gross Carrying Value | 28,107 | |
Accumulated Amortization | (1,820) | |
Net Carrying Value | $ 26,287 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Estimated Future Amortization Expense Associated with Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
2023 | $ 5,035 | |
2024 | 5,035 | |
2025 | 5,035 | |
2026 | 5,035 | |
2027 | 5,035 | |
Thereafter | 33,779 | |
Net Carrying Value | $ 58,954 | $ 18,740 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Schedule of Computation of Basic and Diluted Earnings per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Numerator: | ||
Net loss, basic | $ (135,665) | $ (83,988) |
Add: Change in fair value of Holdback Indemnification liability | (214) | 0 |
Add: Change in fair value of Contingent Milestone liability | (700) | 0 |
Net loss, diluted | $ (136,579) | $ (83,988) |
Denominator: | ||
Weighted-average shares of common stock outstanding, basic | 33,839,072 | 30,321,722 |
Effect of dilutive securities: | ||
Weighted Average Holdback Indemnification Shares Issuable | 19,590 | 0 |
Weighted average contingent shares | 123,831 | 0 |
Weighted-average shares of common stock outstanding, diluted | 33,982,493 | 30,321,722 |
Earnings Per Share, Basic | $ (4.01) | $ (2.77) |
Earnings Per Share, Diluted | $ (4.02) | $ (2.77) |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies - Summary of Outstanding Potentially Dilutive Shares Excluded from Calculation of Diluted Net Loss Per Share (Details) - shares | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 8,981,862 | 7,086,146 |
Employee Stock Option | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 8,955,557 | 6,940,566 |
Common Stock Subject to Repurchase | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 0 | 122,464 |
Employee Stock | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 26,305 | 23,116 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets and Liabilities to Fair Value Measurement On Recurring Basis and Level of Input Measurement (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Financial liabilities: | ||
Derivative Liability, Statement of Financial Position [Extensible Enumeration] | Derivative Liability, Current | |
Indemnification Holdback Liability | ||
Financial liabilities: | ||
Total financial liabilities | $ 617 | |
Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 247,942 | $ 258,604 |
Financial liabilities: | ||
Derivative liability | 1,090 | 1,996 |
Total financial liabilities | 5,607 | 1,996 |
Fair Value, Recurring Basis | Contingent Milestone Liability | ||
Financial liabilities: | ||
Total financial liabilities | 3,900 | |
Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 129,202 | 128,420 |
Financial liabilities: | ||
Derivative liability | 0 | 0 |
Total financial liabilities | 3,900 | 0 |
Fair Value, Recurring Basis | Level 1 | Indemnification Holdback Liability | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Fair Value, Recurring Basis | Level 1 | Contingent Milestone Liability | ||
Financial liabilities: | ||
Total financial liabilities | 3,900 | |
Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 118,740 | 130,184 |
Financial liabilities: | ||
Derivative liability | 0 | 0 |
Total financial liabilities | 0 | 0 |
Fair Value, Recurring Basis | Level 2 | Indemnification Holdback Liability | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Fair Value, Recurring Basis | Level 2 | Contingent Milestone Liability | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Financial liabilities: | ||
Derivative liability | 1,090 | 1,996 |
Total financial liabilities | 1,707 | 1,996 |
Fair Value, Recurring Basis | Level 3 | Indemnification Holdback Liability | ||
Financial liabilities: | ||
Total financial liabilities | 617 | |
Fair Value, Recurring Basis | Level 3 | Contingent Milestone Liability | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Fair Value, Recurring Basis | Money Market Fund | ||
Financial assets: | ||
Fair value measurements | 124,227 | 128,420 |
Fair Value, Recurring Basis | Money Market Fund | Level 1 | ||
Financial assets: | ||
Fair value measurements | 124,227 | 128,420 |
Fair Value, Recurring Basis | Money Market Fund | Level 2 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Fair Value, Recurring Basis | Money Market Fund | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Fair Value, Recurring Basis | U.S. treasury bills | ||
Financial assets: | ||
Fair value measurements | 4,975 | |
Fair Value, Recurring Basis | U.S. treasury bills | Level 1 | ||
Financial assets: | ||
Fair value measurements | 4,975 | |
Fair Value, Recurring Basis | U.S. treasury bills | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Fair Value, Recurring Basis | Commercial Paper | ||
Financial assets: | ||
Fair value measurements | 74,386 | 115,221 |
Fair Value, Recurring Basis | Commercial Paper | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Fair Value, Recurring Basis | Commercial Paper | Level 2 | ||
Financial assets: | ||
Fair value measurements | 74,386 | 115,221 |
Fair Value, Recurring Basis | Commercial Paper | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Fair Value, Recurring Basis | U.S. Government Bonds | ||
Financial assets: | ||
Fair value measurements | 44,354 | 14,963 |
Fair Value, Recurring Basis | U.S. Government Bonds | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Fair Value, Recurring Basis | U.S. Government Bonds | Level 2 | ||
Financial assets: | ||
Fair value measurements | 44,354 | 14,963 |
Fair Value, Recurring Basis | U.S. Government Bonds | Level 3 | ||
Financial assets: | ||
Fair value measurements | $ 0 | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) | Dec. 31, 2022 | Dec. 31, 2021 |
Discount Rate | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Discount rate used for valuation to derivative liability | 0.157 | 0.157 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Change in Estimated Fair Value of Company's Derivative Liability Classified as Level 3 (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Change in fair value of derivative liability | $ (906) | $ 732 |
Level 3 | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Beginning balance | 1,996 | 1,264 |
Ending balance | $ 1,090 | $ 1,996 |
Fair Value Measurements - Sum_3
Fair Value Measurements - Summary of Changes in Fair Value classified as Level 3 (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2022 USD ($) | |
Indemnification Holdback Liability | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning Balance | $ 0 |
Initial recognition | 831 |
Change in fair value | (214) |
Transfer out of Level 3 fair value hierarchy | 0 |
Ending Balance | 617 |
Contingent Milestone Liability | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning Balance | 0 |
Initial recognition | 4,600 |
Change in fair value | (700) |
Transfer out of Level 3 fair value hierarchy | (3,900) |
Ending Balance | $ 0 |
Financial Instruments - Summary
Financial Instruments - Summary of Fair Value and Amortized Cost of Cash Equivalents and Available-for-sale Investments by Major Security Type (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents and investments, Amortized cost | $ 248,172 | $ 258,640 |
Cash equivalents and investments, Unrealized gain | 0 | 0 |
Cash equivalents and investments, Unrealized loss | (230) | (36) |
Cash equivalents and investments, Estimated Fair Value | 247,942 | 258,604 |
Cash equivalents, Estimated Fair Value | 24,226 | 28,420 |
Cash equivalents - Restricted, Estimated Fair Value | 100,000 | 100,000 |
Short-term investments | 123,716 | 125,201 |
Long-term investments | 4,983 | |
Total cash equivalents and investments | 247,942 | 258,604 |
Money Market Fund | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents, Amortized Cost | 124,227 | 128,420 |
Cash equivalents, Estimated Fair Value | 124,227 | 128,420 |
U.S. treasury bills | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 4,980 | |
Investments, Unrealized Gain | 0 | |
Investments, Unrealized Loss | (5) | |
Investments, Estimated Fair Value | 4,975 | |
Commercial Paper | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 74,386 | 115,221 |
Investments, Unrealized Gain | 0 | 0 |
Investments, Unrealized Loss | 0 | 0 |
Investments, Estimated Fair Value | 74,386 | 115,221 |
U.S. Government Bonds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 44,579 | 14,999 |
Investments, Unrealized Gain | 0 | 0 |
Investments, Unrealized Loss | (225) | (36) |
Investments, Estimated Fair Value | $ 44,354 | $ 14,963 |
Financial Instruments - Additio
Financial Instruments - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Financial Instruments, Owned, at Fair Value, by Type, Alternative [Abstract] | ||
Realized gains or losses on available-for-sale investments | $ 0 | $ 0 |
Investments in continuous unrealized loss position for more than 12 months | 0 | 0 |
Other-than-temporary impairment losses | $ 0 | $ 0 |
Balance Sheet Components - Sche
Balance Sheet Components - Schedule of Inventory (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Raw material | $ 0 | $ 856 |
Work in progress | 5,351 | 570 |
Finished goods | 214 | 87 |
Total Inventory | $ 5,565 | $ 1,513 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Payables and Accruals [Abstract] | ||
Accrued milestone payments | $ 15,000 | $ 0 |
Accrued compensation and related benefits | 14,660 | 9,988 |
Accrued clinical trials | 8,319 | 6,732 |
Accrued professional service fees | 5,372 | 2,458 |
Accrued rebates payable | 4,284 | 265 |
Accrued contract manufacturing and non-clinical costs | 3,927 | 4,635 |
Accrued royalties payable | 2,456 | 345 |
Accrued collaboration funding | 0 | 6,300 |
Total accrued expenses | $ 54,018 | $ 30,723 |
Revenue Interest Purchase Agr_3
Revenue Interest Purchase Agreement - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Apr. 30, 2021 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Revenue interest liability | $ 140,351 | $ 129,923 | ||
Sales | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Revenues | $ 5,600 | |||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Percentage of revenue interest payments on annual net sales at Tier 1 | 9.75% | |||
Percentage of revenue interest payments on annual net sales at Tier 2 and Tier 3 | 2% | |||
Revenue interest rate percentage shall be reduced, if revenue interest payment percentage greater than or equal to 110% at Tier | 2% | |||
Revenue interest rate percentage shall be reduced, if revenue interest payment percentage greater than or equal to 110% at Tier 3 | 0% | |||
Required revenue interest payment percentage of cumulative purchaser payments for termination | 195% | |||
Required repurchase price percentage of cumulative purchaser prior to first anniversary of closing date | 120% | |||
Required repurchase price percentage of cumulative purchaser payments after first anniversary and prior to third anniversary of closing date | 175% | |||
Required repurchase price percentage of cumulative purchaser after third anniversary of closing date | 195% | |||
Purchase agreement amount allocated to debt | $ 49,200 | |||
Initial fair value of derivative liability | 1,300 | |||
Revenue interest liability | 140,400 | 129,900 | ||
Debt issuance costs | 900 | |||
Interest expense | $ 16,000 | $ 17,600 | ||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | On or Prior to December 31, 2026 | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Minimum required revenue interest payment percentage to cumulative purchaser payments to reduce interest rate | 110% | |||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Maximum | On or Prior to December 31, 2022 | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Potential milestone payment to be received at the option of purchasers | $ 50,000 | |||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Commercialization and Development of Product and Other Working Capital Needs | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Upfront payment received | 50,000 | |||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Livmarli | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Milestone and upfront payments received | 115,000 | |||
Upfront payment received | $ 50,000 | |||
Milestone payment received | $ 65,000 | |||
Potential milestone payment received upon regulatory approval | 35,000 | |||
Tier I | Mulholland SA LLC, | Livmarli | Maximum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Annual net sales | 350,000 | |||
Tier 2 | Mulholland SA LLC, | Livmarli | Minimum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Annual net sales | 350,000 | |||
Tier 2 | Mulholland SA LLC, | Livmarli | Maximum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Annual net sales | 1,100,000 | |||
Tier 3 | Mulholland SA LLC, | Livmarli | Minimum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Annual net sales | $ 1,100,000 | |||
CSPA | Mulholland SA LLC, | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Number of shares sold | 509,164 | |||
Aggregate purchase price | $ 10,000 | |||
Net proceeds from transaction | 10,000 | |||
Purchase agreement amount allocated to common stock issued | $ 10,800 |
Revenue Interest Purchase Agr_4
Revenue Interest Purchase Agreement - Summary of Revenue Interest Liability (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Revenue interest liability | $ 129,923 | $ 47,651 |
Proceeds from purchaser payments | 65,000 | |
Interest expense recognized | 15,979 | 17,590 |
Capitalized issuance costs | (197) | |
Revenue interest payments | (5,551) | (121) |
Revenue interest liability | $ 140,351 | $ 129,923 |
Asset Acquisitions - Additional
Asset Acquisitions - Additional Information (Details) | 1 Months Ended | 12 Months Ended | |||
Jan. 31, 2023 shares | May 31, 2022 USD ($) shares | Nov. 30, 2018 USD ($) ProductCandidate | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Satiogen | |||||
Asset Acquisitions [Line Items] | |||||
Total purchase consideration | $ 24,161,000 | ||||
Business Acquisition, Equity Interest Issued or Issuable, Number of Shares | shares | 609,305 | ||||
Business Acquisition, Equity Interest Issued or Issuable, Number of Additional Shares | shares | 32,494 | ||||
Stock option exercise price | $ 200,000 | ||||
Business Acquisition Future Issuance Of Additional Shares | shares | 199,993 | ||||
Cash Consideration | $ 2,600,000 | ||||
Subsequent Event | Satiogen | |||||
Asset Acquisitions [Line Items] | |||||
Business Acquisition Future Issuance Of Additional Shares | shares | 199,993 | ||||
Shire | |||||
Asset Acquisitions [Line Items] | |||||
Accrued milestones | $ 2,000,000 | ||||
Shire | Volixibat | |||||
Asset Acquisitions [Line Items] | |||||
Milestone payment | $ 0 | ||||
Shire | Livmarli | |||||
Asset Acquisitions [Line Items] | |||||
Development or Regulatory Milestones Incurred | 15,000,000 | $ 32,000,000 | |||
Shire Agreement | Livmarli | |||||
Asset Acquisitions [Line Items] | |||||
Milestones accrued | 15,000,000 | ||||
Shire Agreement | Shire | |||||
Asset Acquisitions [Line Items] | |||||
Number of product candidates | ProductCandidate | 2 | ||||
Product sales milestone payments, payable | $ 30,000,000 | ||||
Shire Agreement | Shire | Volixibat | |||||
Asset Acquisitions [Line Items] | |||||
Milestone payments, payable upon commercialization | 30,000,000 | ||||
Shire Agreement | Shire | Livmarli | |||||
Asset Acquisitions [Line Items] | |||||
Milestone payments, payable | 109,500,000 | ||||
Milestone payments, payable upon approval | 25,000,000 | ||||
Assigned License Agreement | Satiogen Pharmaceuticals, Inc. | |||||
Asset Acquisitions [Line Items] | |||||
Milestone payments, payable | 10,500,000 | ||||
Milestone payments, payable upon approval | 5,000,000 | ||||
Milestone payments, payable upon initiation | 500,000 | ||||
Milestone payments, payable upon commercialization | 5,000,000 | ||||
Assigned License Agreement | Sanofi-Aventis Deutschland GmbH | |||||
Asset Acquisitions [Line Items] | |||||
Milestone payments, payable | $ 36,000,000 | ||||
Royalty obligations payment period | 10 years | ||||
Milestones accrued | $ 0 |
Asset Acquisitions - Schedule o
Asset Acquisitions - Schedule of Consideration Paid and Allocation of Costs (Details) - USD ($) $ in Thousands | 1 Months Ended | ||
May 31, 2022 | Dec. 31, 2022 | Dec. 31, 2021 | |
Business Acquisition [Line Items] | |||
Indemnification Holdback | $ 4,532 | $ 17 | |
Satiogen | |||
Business Acquisition [Line Items] | |||
Issued common stock | $ 15,585 | ||
Cash consideration | 2,600 | ||
Indemnification Holdback | 831 | ||
Contingent consideration settled in common stock | 4,600 | ||
Transaction costs | 545 | ||
Total purchase consideration | 24,161 | ||
Assets acquired: | |||
Intangible assets - developed technology | 21,561 | ||
Cash Consideration | 2,600 | ||
Total assets acquired | $ 24,161 |
Collaboration and License Agr_2
Collaboration and License Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Feb. 28, 2023 | Jan. 31, 2022 | Jul. 31, 2021 | Apr. 30, 2021 | Dec. 31, 2022 | Dec. 31, 2021 | |
Vivet Collaboration Agreement | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
License and collaboration agreement date | Apr. 30, 2021 | |||||
Upfront fee | $ 4,200 | |||||
Final payment on terminating agreement | $ 6,300 | |||||
Research and development expense | $ 18,900 | |||||
Exclusive Licensing Agreement | GC Pharma | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Achievement of future regulatory and commercial milestones payment | $ 2,500 | |||||
Exclusive Licensing Agreement | Livmarli | CANbridge | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Upfront payment received | 11,000 | |||||
Research and development funding received | $ 900 | 1,900 | ||||
Achievement of future regulatory and commercial milestones payment | $ 2,000 | |||||
Exclusive Licensing Agreement | Livmarli | GC Pharma | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Upfront payment received | $ 5,000 | |||||
Research and development funding received | 0 | 300 | ||||
Transaction price, adjustments | 0 | 0 | ||||
Exclusive Licensing Agreement | Livmarli | Maximum | CANbridge | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Research and development funding received | 5,000 | |||||
Potential regulatory and commercial milestone payment to be received | $ 109,000 | |||||
Exclusive Licensing Agreement | Livmarli | Maximum | GC Pharma | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Achievement of future regulatory and commercial milestones payment | $ 23,000 | |||||
Exclusive Licensing Agreement | Prepaid Expenses and Other Current Assets [Member] | Livmarli | CANbridge | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Other research and development receivable | 200 | 800 | ||||
Exclusive Licensing Agreement | Prepaid Expenses and Other Current Assets [Member] | Livmarli | GC Pharma | ||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||
Other research and development receivable | $ 0 | $ 300 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Jun. 30, 2022 USD ($) ft² | Nov. 30, 2019 USD ($) ft² | Dec. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Jan. 31, 2019 USD ($) | Jan. 22, 2019 ft² | |
Lessee Lease Description [Line Items] | ||||||
Area of office space | ft² | 3,500 | 5,600 | ||||
Term of lease | 2 years 6 months | 12 years | 4 years | |||
Operating lease, option to extend, description | The lease commenced in November 2022 and has a term of approximately two and a half years with no option to extend the term. | The lease term is approximately four years with an option to extend the term for one five-year term, which at the time was not reasonably assured of exercise and therefore, not included in the lease term. | ||||
Existence of option to extend | true | |||||
Term of extension of lease | 5 years | |||||
Tenant improvement allowance | $ 400 | |||||
Operating lease right-of-use assets | $ 300 | $ 1,431 | $ 1,569 | |||
Lease liability | $ 300 | $ 2,188 | ||||
Weighted-average incremental borrowing rate | 8% | |||||
Weighted-average remaining lease term | 2 years 1 month 6 days | |||||
Rent expense | $ 800 | $ 700 | ||||
Variable lease payments | $ 200 | |||||
Amended Operating Lease Agreement | ||||||
Lessee Lease Description [Line Items] | ||||||
Term of lease | 5 years | |||||
Additional area of office space | ft² | 5,555 | |||||
Lease expiration, month and year | 2025-03 | |||||
Amended Operating Lease Agreement | Property, Plant and Equipment | ||||||
Lessee Lease Description [Line Items] | ||||||
Tenant improvement allowance | $ 800 | |||||
Amended Operating Lease Agreement | Restatement Adjustment | ||||||
Lessee Lease Description [Line Items] | ||||||
Operating lease right-of-use assets | 600 | |||||
Lease liability | $ 600 |
Leases - Schedule of Undiscount
Leases - Schedule of Undiscounted Future Minimum Payments under Operating Leases (Detail) - USD ($) $ in Thousands | Dec. 31, 2022 | Jun. 30, 2022 |
Leases [Abstract] | ||
2023 | $ 1,064 | |
2024 | 1,072 | |
2025 | 242 | |
Total undiscounted lease payments | 2,378 | |
Less: imputed interest | (190) | |
Total lease liability | $ 2,188 | $ 300 |
Stockholders' Equity (Deficit)
Stockholders' Equity (Deficit) - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||||||
Dec. 31, 2022 | Feb. 28, 2023 | Jan. 31, 2023 | Aug. 31, 2022 | Jan. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2022 | Dec. 31, 2021 | Aug. 31, 2020 | |
Class Of Stock [Line Items] | |||||||||
Proceeds from issuance of shares | $ 21,289 | $ 0 | |||||||
Maximum amount of sale covered in shelf registration statement | $ 300,000 | ||||||||
Common stock aggregate offering price, remaining | $ 46,300 | $ 46,300 | |||||||
SVB Leerink LLC | |||||||||
Class Of Stock [Line Items] | |||||||||
Maximum amount of offering issuance and sale covered in sales agreement | $ 75,000 | ||||||||
Common Stock | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares subject to repurchase | 0 | 0 | 122,464 | ||||||
Common Stock | Sales Agreement | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares issued | 1,466,884 | 3,478,261 | 3,750,000 | 1,160,915 | |||||
Shares issued, price per share | $ 19.01 | $ 23 | $ 20 | $ 19.01 | |||||
Proceeds from issuance of shares | $ 70,000 | $ 21,300 | |||||||
Gross proceeds from issuance of common stock | $ 28,700 | $ 22,100 | |||||||
Common Stock | Sales Agreement | Subsequent Event | |||||||||
Class Of Stock [Line Items] | |||||||||
Gross proceeds from issuance of common stock | $ 14,500 | $ 14,500 | |||||||
At-the-market Offering | Common Stock | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares issued | 1,160,915 | ||||||||
At-the-market Offering | Common Stock | Sales Agreement | Subsequent Event | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares issued | 658,206 | 658,206 | |||||||
At-the-market Offering | Common Stock | Sales Agreement | Weighted Average | Subsequent Event | |||||||||
Class Of Stock [Line Items] | |||||||||
Shares issued, price per share | $ 22.79 | $ 22.79 | |||||||
Underwritten Public Offerings | Common Stock | Sales Agreement | Maximum | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares issued | 521,739 | 562,500 | |||||||
Public Offering | |||||||||
Class Of Stock [Line Items] | |||||||||
Proceeds from issuance of shares | $ 7,100 | ||||||||
Gross proceeds from issuance of common stock | $ 86,100 | ||||||||
Public Offering | Common Stock | |||||||||
Class Of Stock [Line Items] | |||||||||
Number of shares issued | 4,000,000 | 375,654 | |||||||
Shares issued, price per share | $ 20 | ||||||||
Number of shares purchased | 375,654 |
Stockholders' Equity (Deficit_2
Stockholders' Equity (Deficit) - Schedule of Common Stock Reserved for Issuance (Details) - shares | Dec. 31, 2022 | Dec. 31, 2021 |
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 11,941,705 | 10,286,323 |
Stock options and restricted stock units issued and outstanding | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 8,955,557 | 6,940,566 |
Reserved for Future Stock Awards or Option Grants | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 1,596,947 | 2,434,619 |
Reserved for Employee Stock Purchase Plan | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 1,157,570 | 911,138 |
Common stock held back in connection with asset acquisition | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 31,638 | 0 |
Common stock issuable as contingent consideration in connection with asset acquisition | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 199,993 | 0 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Dec. 31, 2020 | Jul. 31, 2019 | Nov. 30, 2018 | Dec. 31, 2022 | Dec. 31, 2021 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Number of common stock approved and reserved for issuance | 11,941,705 | 10,286,323 | ||||
Stock-based compensation expense | $ 27,007 | $ 23,016 | ||||
Share-based compensation expenses capitalized amount | 400 | 100 | ||||
Stock Options | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock-based compensation, Intrinsic value of options exercised | 3,000 | $ 1,300 | ||||
Total unrecognized stock-based compensation related to unvested stock option awards granted | $ 41,100 | |||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 2 years 4 months 24 days | |||||
Expected dividend yield | 0% | 0% | ||||
Weighted-average grant-date fair value | $ 12.73 | $ 13.39 | ||||
Issuance of common stock in connection with common stock option exercises, Shares | 357,934 | |||||
RSUs | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Total unrecognized stock-based compensation related to unvested stock option awards granted | $ 7,800 | |||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 2 years 21 days | |||||
Restricted Common Stock Member | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Vested | 122,464 | 133,593 | ||||
Total unrecognized compensation expense related to unvested restricted stock | $ 400 | $ 400 | ||||
Restricted Common Stock Member | Founder | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Granted | $ 2.94 | |||||
Restricted Common Stock Member | Common Stock | Founder | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock issued for services | 562,500 | |||||
Vesting period | 4 years | |||||
2019 Equity Incentive Plan | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Equity incentive plans, description | Shares subject to outstanding awards under the 2018 Plan as of the effective date of the 2019 Plan that are subsequently canceled, forfeited or repurchased by the Company will be added to the shares reserved under the 2019 Plan. In addition, the number of shares of common stock available for issuance under the 2019 Plan will be automatically increased on the first day of each calendar year during the ten-year term of the 2019 Plan, beginning with January 1, 2020 and ending with January 1, 2029, by an amount equal to 5% of the outstanding number of shares of the Company’s common stock on December 31st of the preceding calendar year or such lesser amount as determined by the Company’s board of directors. As of December 31, 2022, 1,121,179 shares of common stock were available for issuance under the 2019 Plan. | |||||
Shares of common stock expiration term | 10 years | |||||
Shares of common stock beginning date | Jan. 01, 2020 | |||||
Shares of common stock ending date | Jan. 01, 2029 | |||||
Percentage of annual increase in common stock available for issuance | 5% | |||||
Number of common stock for future issuance | 1,121,179 | |||||
2020 Inducement Plan | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Number of common stock for future issuance | 475,768 | 750,000 | ||||
Number of additional common stock for future issuance | 750,000 | 1,000,000 | ||||
2019 Employee Stock Purchase Plan | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Equity incentive plans, description | A total of 500,000 shares of common stock were approved to be initially reserved for issuance under the ESPP. In addition, the number of shares of common stock available for issuance under the ESPP will be automatically increased on the first day of each calendar year during the first ten years of the term of the ESPP, beginning with January 1, 2020 and ending with January 1, 2029, by an amount equal to the lesser of (i) 1% of the outstanding number of shares of common stock on December 31st of the preceding calendar year, (ii) 1,500,000 shares of common stock or (iii) such lesser amount as determined by the Company’s board of directors. During the year ended December 31, 2022, 123,131 shares were issued under the ESPP. As of December 31, 2022, the Company had 1,157,570 shares available for future issuance under the ESPP. The stock-based compensation related to the ESPP for the years ended December 31, 2022 and 2021 was $0.7 million, respectively. | |||||
Shares of common stock expiration term | 10 years | |||||
Shares of common stock beginning date | Jan. 01, 2020 | |||||
Shares of common stock ending date | Jan. 01, 2029 | |||||
Percentage of annual increase in common stock available for issuance | 1% | |||||
Number of common stock approved and reserved for issuance | 1,157,570 | |||||
Stock issued for services | 500,000 | 123,131 | ||||
Annual increase in common stock available for issuance, shares | 1,500,000 | |||||
2019 Employee Stock Purchase Plan | Executive Performance Stock Units | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock-based compensation expense | $ 700 | $ 700 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option Activity (Details) - Stock Options - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Options, Outstanding | ||
Number of shares, Outstanding, Beginning balance | 6,870,617 | |
Number of shares, Granted | 2,174,350 | |
Number of shares, Exercised | (357,934) | |
Number of shares, Canceled and forfeited | (346,950) | |
Number of shares, Outstanding, Ending balance | 8,340,083 | 6,870,617 |
Number of shares, Vested and exercisable | 4,578,478 | |
Weighted-average exercise price, Outstanding | ||
Weighted-average exercise price, Outstanding, Beginning balance | $ 12.56 | |
Weighted-average exercise price, Granted | 18.02 | |
Weighted-average exercise price, Exercised | 15.19 | |
Weighted-average exercise price, Canceled and forfeited | 18.39 | |
Weighted-average exercise price, Outstanding, Ending balance | 13.63 | $ 12.56 |
Weighted-average exercise price, Vested and exercisable | $ 10.39 | |
Share-based Payment Award, Options, Additional Disclosures | ||
Weighted-average remaining contractual life, Outstanding | 7 years 6 months | 8 years |
Weighted-average remaining contractual life, Vested and exercisable | 6 years 8 months 12 days | |
Aggregate intrinsic value, Outstanding | $ 51,645 | $ 32,637 |
Aggregate intrinsic value, Vested and exercisable | $ 42,123 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted (Details) - Stock Options - $ / shares | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Exercise price, minimum | $ 15.50 | $ 14.05 |
Exercise price, maximum | $ 27.46 | $ 21.22 |
Expected volatility, minimum | 80.86% | 82.76% |
Expected volatility, maximum | 83.20% | 94.06% |
Risk-free interest rate, minimum | 1.46% | 0.62% |
Risk-free interest rate, maximum | 4.10% | 1.34% |
Expected dividend yield | 0% | 0% |
Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 5 years 6 months | 5 years 6 months |
Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 6 years 1 month 6 days | 6 years 1 month 6 days |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of RSU Activity (Details) - RSUs - $ / shares | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||
Unvested and Outstanding as of December 31, 2021 | 615,474 | 69,949 |
Granted | 586,452 | |
Vested | 0 | |
Number of shares, Canceled and forfeited | (40,927) | |
Unvested and Outstanding as of December 31, 2022 | 615,474 | 69,949 |
Weighted-average exercise price, Granted | $ 18.24 | |
Weighted-average exercise price, Vested | 0 | |
Weighted-average exercise price, Canceled and forfeited | 16.92 | |
Weighted-average exercise price, Vested and exercisable | $ 18.36 | $ 18.57 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Stock-based Compensation Reflected in Consolidated Statements of Operations (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 27,007 | $ 23,016 |
Selling, General and Administrative | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | 16,957 | 13,128 |
Research and Development | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 10,050 | $ 9,888 |
Gain from Sale of Priority Re_2
Gain from Sale of Priority Review Voucher - Additional information (Details) - Priority review voucher of Livmarli [Member] - USD ($) $ in Millions | 1 Months Ended | |
Dec. 31, 2021 | Nov. 30, 2021 | |
Proceeds from sale of other investments | $ 110 | |
Net proceeds after commision | $ 108 |
Income Taxes - Schedule of Loss
Income Taxes - Schedule of Losses before Provision for Income Taxes (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | ||
U.S. loss before taxes | $ (142,661) | $ (84,217) |
Foreign income before taxes | 590 | 266 |
Loss before income taxes | $ (142,071) | $ (83,951) |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax [Line Items] | ||
Valuation allowance, deferred tax asset, increase | $ 32,500 | $ 23,900 |
Provision for income taxes | (6,406) | 37 |
Deferred tax assets, tax credit carryforwards, research and development | 23,569 | 0 |
Income or foreign withholding taxes on Undistributed earnings of foreign subsidiaries | 0 | 0 |
Satiogen | ||
Income Tax [Line Items] | ||
Business acquisition, deferred tax liability | 6,600 | |
Provision for income taxes | (6,600) | |
California Franchise Tax Board | ||
Income Tax [Line Items] | ||
Operating loss carryforwards | $ 17,700 | 4,500 |
Operating loss carryforwards expiration, beginning year | 2038 | |
Domestic Tax Authority | ||
Income Tax [Line Items] | ||
Operating loss carryforwards | $ 153,100 | 150,000 |
Deferred tax assets, tax credit carryforwards, research and development | 32,500 | 23,100 |
State and Local Jurisdiction | ||
Income Tax [Line Items] | ||
Operating loss carryforwards | $ 19,100 | 2,200 |
Operating loss carryforwards expiration, beginning year | 2032 | |
State and Local Jurisdiction | California Franchise Tax Board | ||
Income Tax [Line Items] | ||
Deferred tax assets, tax credit carryforwards, research and development | $ 4,200 | $ 2,100 |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Income Tax (Expense) Benefit (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Current income taxes: | ||
State | $ 18 | $ 0 |
Foreign | 157 | 37 |
Total current | 175 | 37 |
Deferred taxes: | ||
Federal | (5,503) | 0 |
State | (1,078) | 0 |
Total deferred | (6,581) | 0 |
Income tax (benefit) expense | $ (6,406) | $ 37 |
Income Taxes - Summary of Recon
Income Taxes - Summary of Reconciliation of Federal Statutory Income Tax Rate (Details) | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | ||
Federal statutory income tax rate | 21% | 21% |
State tax | 7.40% | 1.32% |
Permanent differences | (0.65%) | (1.70%) |
Other | (0.93%) | 0.02% |
Executive compensation | (2.99%) | 0% |
Global intangible low-taxed income | (1.43%) | 0% |
Tax credits | 4.96% | 7.72% |
Change in valuation allowance | (22.85%) | (28.41%) |
Total tax benefit | 4.51% | (0.05%) |
Income Taxes - Significant Comp
Income Taxes - Significant Components of Deferred Taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2022 | Dec. 31, 2021 |
Deferred tax assets: | ||
Net operating losses | $ 34,522 | $ 31,819 |
Tax credit carryforwards | 26,747 | 18,501 |
Capitalized research and development | 23,569 | 0 |
Stock-based compensation | 6,726 | 5,815 |
Intangibles | 4,733 | 10,277 |
Accrued expenses | 3,527 | 4,027 |
Interest limitation attributes | 3,127 | 0 |
Lease liability | 460 | 543 |
Total deferred tax assets | 103,411 | 70,982 |
Deferred tax liabilities: | ||
Operating lease right-of-use assets | (272) | (319) |
Fixed assets | (100) | (138) |
Total deferred tax liabilities | (372) | (457) |
Valuation allowance | (103,039) | (70,525) |
Net deferred tax assets | $ 0 | $ 0 |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2022 | Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | ||
Balance at beginning of year | $ 6,399 | $ 3,933 |
(Decrease) related to prior year tax positions | 0 | 0 |
Increases related to current year tax positions | 2,855 | 2,466 |
Balance at end of year | $ 9,254 | $ 6,399 |