Cover Page
Cover Page - shares | 9 Months Ended | |
Sep. 30, 2022 | Nov. 07, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2022 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q3 | |
Entity Registrant Name | CATALYST PHARMACEUTICALS, INC. | |
Entity Central Index Key | 0001369568 | |
Current Fiscal Year End Date | --12-31 | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity Filer Category | Accelerated Filer | |
Trading Symbol | CPRX | |
Entity Emerging Growth Company | false | |
Entity Small Business | false | |
Entity Address, State or Province | FL | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock | |
Security Exchange Name | NASDAQ | |
Entity Common Stock, Shares Outstanding | 104,071,350 | |
Entity Tax Identification Number | 76-0837053 | |
Entity Incorporation, State or Country Code | DE | |
Entity Address, Address Line One | 355 Alhambra Circle | |
Entity Address, Address Line Two | Suite 801 | |
Entity Address, City or Town | Coral Gables | |
Entity Address, Postal Zip Code | 33134 | |
City Area Code | 305 | |
Local Phone Number | 420-3200 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity File Number | 001-33057 |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Current Assets: | ||
Cash and cash equivalents | $ 256,065 | $ 171,445 |
Short-term investments | 0 | 19,821 |
Accounts receivable, net | 9,337 | 6,619 |
Inventory | 7,132 | 7,870 |
Prepaid expenses and other current assets | 3,776 | 4,351 |
Total current assets | 276,310 | 210,106 |
Operating lease right-of-use asset | 2,833 | 3,017 |
Property and equipment, net | 882 | 959 |
License and acquired intangibles, net | 33,051 | 0 |
Deferred tax assets, net | 20,029 | 23,697 |
Deposits | 9 | 9 |
Total assets | 333,114 | 237,788 |
Current Liabilities: | ||
Accounts payable | 2,529 | 2,768 |
Accrued expenses and other liabilities | 42,152 | 24,295 |
Total current liabilities | 44,681 | 27,063 |
Operating lease liability, net of current portion | 3,643 | 3,894 |
Other non-current liabilities | 14,749 | 0 |
Total liabilities | 63,073 | 30,957 |
Commitments and contingencies (Note 12) | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value, 5,000,000 shares authorized: none issued and outstanding at September 30, 2022 and December 31, 2021 | 0 | 0 |
Common stock, $0.001 par value, 200,000,000 shares authorized; 104,064,683 shares and 102,992,913 shares issued and outstanding at September 30, 2022 and December 31, 2021, respectively | 104 | 103 |
Additional paid-in capital | 245,514 | 233,186 |
Retained earnings (accumulated deficit) | 24,391 | (26,310) |
Accumulated other comprehensive income (loss) (Note 4) | 32 | (148) |
Total stockholders' equity | 270,041 | 206,831 |
Total liabilities and stockholders' equity | $ 333,114 | $ 237,788 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Sep. 30, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 104,064,683 | 102,992,913 |
Common stock, shares outstanding | 104,064,683 | 102,992,913 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Revenues: | ||||
Total revenues | $ 57,244 | $ 35,954 | $ 153,446 | $ 102,524 |
Operating costs and expenses: | ||||
Cost of sales | 9,665 | 5,310 | 23,198 | 14,536 |
Research and development | 8,310 | 4,487 | 15,696 | 11,944 |
Selling, general and administrative | 14,167 | 12,153 | 43,515 | 36,401 |
Total operating costs and expenses | 32,142 | 21,950 | 82,409 | 62,881 |
Operating income | 25,102 | 14,004 | 71,037 | 39,643 |
Other income (expense), net | 905 | 68 | 674 | 211 |
Net income before income taxes | 26,007 | 14,072 | 71,711 | 39,854 |
Income tax provision | 3,259 | 3,743 | 14,103 | 9,681 |
Net income | $ 22,748 | $ 10,329 | $ 57,608 | $ 30,173 |
Net income per share: | ||||
Basic | $ 0.22 | $ 0.1 | $ 0.56 | $ 0.29 |
Diluted | $ 0.2 | $ 0.1 | $ 0.52 | $ 0.28 |
Weighted average shares outstanding: | ||||
Basic | 103,318,572 | 103,196,550 | 102,967,280 | 103,470,762 |
Diluted | 111,986,025 | 107,843,196 | 110,352,214 | 107,476,175 |
Net income | $ 22,748 | $ 10,329 | $ 57,608 | $ 30,173 |
Other comprehensive income (Note 4): | ||||
Unrealized gain (loss) on available-for-sale securities, net of tax of ($52), $0, ($59) and $0, respectively | 162 | (26) | 180 | (94) |
Comprehensive income | 22,910 | 10,303 | 57,788 | 30,079 |
Product revenue, net [Member] | ||||
Revenues: | ||||
Total revenues | 57,173 | 35,890 | 153,255 | 99,731 |
License and other revenue [Member] | ||||
Revenues: | ||||
Total revenues | $ 71 | $ 64 | $ 191 | $ 2,793 |
CONSOLIDATED STATEMENTS OF OP_2
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Statement of Comprehensive Income [Abstract] | ||||
Net of tax impact | $ (52) | $ 0 | $ (59) | $ 0 |
CONSOLIDATED STATEMENTS OF CHAN
CONSOLIDATED STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Preferred Stock [Member] | Common Stock [Member] | Additional Paid-in Capital [Member] | Retained Earnings (Accumulated Deficit) [Member] | Accumulated Other Comprehensive Gain (Loss) [Member] |
Beginning Balance at Dec. 31, 2020 | $ 169,598 | $ 0 | $ 104 | $ 223,168 | $ (53,705) | $ 31 |
Beginning Balance (shares) at Dec. 31, 2020 | 103,782,000 | |||||
Issuance of stock options for services | 1,442 | 1,442 | ||||
Exercise of stock options for common stock | 188 | 188 | ||||
Exercise of stock options for common stock (shares) | 90,000 | |||||
Amortization of restricted stock for services | 129 | 129 | ||||
Repurchase of common stock | (293) | (293) | ||||
Repurchase of common stock (shares) | (67,000) | |||||
Other comprehensive gain (loss) | (76) | (76) | ||||
Net income | 7,663 | 7,663 | ||||
Ending Balance at Mar. 31, 2021 | 178,651 | 0 | $ 104 | 224,927 | (46,335) | (45) |
Ending Balance (shares) at Mar. 31, 2021 | 103,805,000 | |||||
Beginning Balance at Dec. 31, 2020 | 169,598 | 0 | $ 104 | 223,168 | (53,705) | 31 |
Beginning Balance (shares) at Dec. 31, 2020 | 103,782,000 | |||||
Net income | 30,173 | |||||
Ending Balance at Sep. 30, 2021 | 198,661 | 0 | $ 103 | 231,165 | (32,544) | (63) |
Ending Balance (shares) at Sep. 30, 2021 | 103,153,000 | |||||
Beginning Balance at Mar. 31, 2021 | 178,651 | 0 | $ 104 | 224,927 | (46,335) | (45) |
Beginning Balance (shares) at Mar. 31, 2021 | 103,805,000 | |||||
Issuance of stock options for services | 1,388 | 1,388 | ||||
Exercise of stock options for common stock | 271 | 271 | ||||
Exercise of stock options for common stock (shares) | 83,000 | |||||
Amortization of restricted stock for services | 130 | 130 | ||||
Repurchase of common stock | (3,668) | $ (1) | (3,667) | |||
Repurchase of common stock (shares) | (733,000) | |||||
Issuance of common stock upon vesting of restricted stock units, net | (17) | (17) | ||||
Issuance of common stock upon vesting of restricted stock units, net (shares) | 7,000 | |||||
Other comprehensive gain (loss) | 8 | 8 | ||||
Net income | 12,181 | 12,181 | ||||
Ending Balance at Jun. 30, 2021 | 188,944 | 0 | $ 103 | 226,699 | (37,821) | (37) |
Ending Balance (shares) at Jun. 30, 2021 | 103,162,000 | |||||
Issuance of stock options for services | 1,380 | 1,380 | ||||
Exercise of stock options for common stock | 2,955 | $ 1 | 2,954 | |||
Exercise of stock options for common stock (shares) | 941,000 | |||||
Amortization of restricted stock for services | 132 | 132 | ||||
Repurchase of common stock | (5,053) | $ (1) | (5,052) | |||
Repurchase of common stock (shares) | (950,000) | |||||
Other comprehensive gain (loss) | (26) | (26) | ||||
Net income | 10,329 | 10,329 | ||||
Ending Balance at Sep. 30, 2021 | 198,661 | 0 | $ 103 | 231,165 | (32,544) | (63) |
Ending Balance (shares) at Sep. 30, 2021 | 103,153,000 | |||||
Beginning Balance at Dec. 31, 2021 | 206,831 | 0 | $ 103 | 233,186 | (26,310) | (148) |
Beginning Balance (shares) at Dec. 31, 2021 | 102,993,000 | |||||
Issuance of stock options for services | 1,623 | 1,623 | ||||
Exercise of stock options for common stock | 1,102 | 1,102 | ||||
Exercise of stock options for common stock (shares) | 364,000 | |||||
Amortization of restricted stock for services | 280 | 280 | ||||
Repurchase of common stock | (2,551) | (2,551) | ||||
Repurchase of common stock (shares) | (400,000) | |||||
Other comprehensive gain (loss) | (305) | (305) | ||||
Net income | 13,241 | 13,241 | ||||
Ending Balance at Mar. 31, 2022 | 220,221 | 0 | $ 103 | 236,191 | (15,620) | (453) |
Ending Balance (shares) at Mar. 31, 2022 | 102,957,000 | |||||
Beginning Balance at Dec. 31, 2021 | 206,831 | 0 | $ 103 | 233,186 | (26,310) | (148) |
Beginning Balance (shares) at Dec. 31, 2021 | 102,993,000 | |||||
Net income | 57,608 | |||||
Ending Balance at Sep. 30, 2022 | 270,041 | 0 | $ 104 | 245,514 | 24,391 | 32 |
Ending Balance (shares) at Sep. 30, 2022 | 104,065,000 | |||||
Beginning Balance at Mar. 31, 2022 | 220,221 | 0 | $ 103 | 236,191 | (15,620) | (453) |
Beginning Balance (shares) at Mar. 31, 2022 | 102,957,000 | |||||
Issuance of stock options for services | 1,594 | 1,594 | ||||
Exercise of stock options for common stock | 1,282 | 1,282 | ||||
Exercise of stock options for common stock (shares) | 345,000 | |||||
Amortization of restricted stock for services | 429 | 429 | ||||
Repurchase of common stock | (4,356) | (4,356) | ||||
Repurchase of common stock (shares) | (600,000) | |||||
Issuance of common stock upon vesting of restricted stock units, net | (20) | (20) | ||||
Issuance of common stock upon vesting of restricted stock units, net (shares) | 7,000 | |||||
Other comprehensive gain (loss) | 323 | 323 | ||||
Net income | 21,619 | 21,619 | ||||
Ending Balance at Jun. 30, 2022 | 241,092 | 0 | $ 103 | 239,476 | 1,643 | (130) |
Ending Balance (shares) at Jun. 30, 2022 | 102,709,000 | |||||
Issuance of stock options for services | 1,621 | 1,621 | ||||
Exercise of stock options for common stock | 3,984 | $ 1 | 3,983 | |||
Exercise of stock options for common stock (shares) | 1,356,000 | |||||
Amortization of restricted stock for services | 434 | 434 | ||||
Other comprehensive gain (loss) | 162 | 162 | ||||
Net income | 22,748 | 22,748 | ||||
Ending Balance at Sep. 30, 2022 | $ 270,041 | $ 0 | $ 104 | $ 245,514 | $ 24,391 | $ 32 |
Ending Balance (shares) at Sep. 30, 2022 | 104,065,000 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Operating Activities: | ||
Net income | $ 57,608 | $ 30,173 |
Adjustments to reconcile net income to net cash provided by (used in) operating activities: | ||
Depreciation | 106 | 159 |
Stock-based compensation | 5,981 | 4,601 |
Amortization of intangible assets | 518 | |
Deferred taxes | 3,652 | 6,571 |
Change in accrued interest and accretion of discount on investments | 17 | (9) |
Reduction in the carrying amount of right-of-use asset | 184 | 216 |
Realized loss on sale of available-for-sale securities | 762 | |
Acquired research and development inventory expensed from asset acquisition | 4,130 | |
(Increase) decrease in: | ||
Accounts receivable, net | (2,718) | (638) |
Inventory | 738 | (2,299) |
Prepaid expenses and other current assets and deposits | 575 | 3,900 |
Increase (decrease) in: | ||
Accounts payable | (239) | (1,504) |
Accrued expenses and other liabilities | 4,885 | (916) |
Operating lease liability | (229) | 942 |
Net cash provided by (used in) operating activities | 75,970 | 41,196 |
Investing Activities: | ||
Purchases of property and equipment | (29) | (980) |
Purchases of investments | (10,000) | |
Proceeds from sale of available-for-sale securities | 19,238 | |
Payment in connection with license agreement | (10,000) | |
Net cash provided by (used in) investing activities | 9,209 | (10,980) |
Financing Activities: | ||
Payment of employee withholding tax related to stock-based compensation | (20) | (17) |
Proceeds from exercise of stock options | 6,368 | 3,414 |
Repurchase of common stock | (6,907) | (9,014) |
Net cash provided by (used in) financing activities | (559) | (5,617) |
Net increase (decrease) in cash and cash equivalents | 84,620 | 24,599 |
Cash and cash equivalents – beginning of period | 171,445 | 130,237 |
Cash and cash equivalents – end of period | 256,065 | 154,836 |
Supplemental disclosures of cash flow information: | ||
Cash paid for income taxes | 6,344 | 1,438 |
Non-cash investing and financing activities: | ||
Operating lease liabilities arising from obtaining right-of-use assets | $ 3,309 | |
Liabilities arising from asset acquisition | $ 27,699 |
Organization and Description of
Organization and Description of Business | 9 Months Ended |
Sep. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Description of Business | 1. Organization and Description of Business. Catalyst Pharmaceuticals, Inc. and subsidiary (collectively, the “Company”) is a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases. With exceptional patient focus, Catalyst is committed to developing a robust pipeline of cutting-edge, best-in-class medicines for rare diseases. Catalyst’s New Drug Application for FIRDAPSE® (amifampridine) Tablets 10 mg for the treatment of adults with Lambert-Eaton myasthenic syndrome (“LEMS”) was approved in 2018 by the U.S. Food & Drug Administration (“FDA”), and FIRDAPSE® is commercially available in the United States as a treatment for adults with LEMS. Further, Canada’s national healthcare regulatory agency, Health Canada, approved the use of FIRDAPSE® for the treatment of adult patients in Canada with LEMS in 2020 and FIRDAPSE® is commercially available in Canada for the treatment of patients with LEMS through a license and supply agreement with KYE Pharmaceuticals. Finally, in the third quarter of 2022, the FDA approved the Company’s sNDA approving an expansion of the FIRDAPSE® label to include pediatric patients (ages six and older ). Since inception, the Company has devoted substantially all its efforts to business planning, research and development, recruiting management and technical staff, acquiring operating assets, raising capital, and selling its product. The Company incurred operating losses in each period from inception and started reporting operating income during the year ended December 31, 2019. The Company has been able to fund its cash needs to date through offerings of its securities and from revenues from sales of its product. See Note 15 (Stockholders’ Equity). Capital Resources While there can be no assurance, based on currently available information, the Company estimates that it has sufficient resources to support its operations for at least the next 12 months from the issuance date of this report. The Company may raise funds in the future through public or private equity offerings, debt financings, corporate collaborations, governmental research grants or other means. The Company may also seek to raise new capital to fund additional drug development efforts, even if it has sufficient funds for its planned operations. Any sale by the Company of additional equity or convertible debt securities could result in dilution to the Company’s current stockholders. There can be no assurance that any required additional funding will be available to the Company at all or available on terms acceptable to the Company. Further, to the extent that the Company raises additional funds through collaborative arrangements, it may be necessary to relinquish some rights to the Company’s drug candidates or grant sublicenses on terms that are not favorable to the Company. If the Company is not able to secure additional funding when needed, the Company may have to delay, reduce the scope of, or eliminate one or more research and development programs, which could have an adverse effect on the Company’s business. Risks and Uncertainties There are numerous aspects of the coronavirus (COVID-19) |
Basis of Presentation and Signi
Basis of Presentation and Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Significant Accounting Policies | 2. Basis of Presentation and Significant Accounting Policies. a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2021 included in the 2021 Annual Report on Form 10-K b. PRINCIPLES OF CONSOLIDATION. c. USE OF ESTIMATES. d. CASH AND CASH EQUIVALENTS. e. INVESTMENTS. The U.S. Treasuries held at September 30, 2022 are classified as available-for-sale non-current non-current The Company records available-for-sale in the consolidated statements of operations and comprehensive income, available-for-sale available-for-sale available-for-sale f. ACCOUNTS RECEIVABLE, NET. g. INVENTORY Products that have been approved by the FDA or other regulatory authorities, such as FIRDAPSE®, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of FIRDAPSE® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage. h. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-clinical i. PROPERTY AND EQUIPMENT, NET. three five five j. INTANGIBLE ASSETS, NET. The Company reviews intangible assets with finite lives for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If indicators of impairment exist, an impairment test is performed to assess the recoverability of the affected assets by determining whether the carrying amount of such assets exceeds the undiscounted expected future cash flows. If the affected assets are deemed not recoverable, the Company would estimate the fair value of the assets and record an impairment loss. There were no impairment charges recognized on finite lived intangibles for the three or nine months ended September 30, 2022. There were no finite lived intangible assets at December 31, 2021. k. FAIR VALUE OF FINANCIAL INSTRUMENTS. l. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using (in thousands) Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,336 $ 10,336 $ — $ — U.S. Treasuries $ 227,349 $ 227,349 $ — $ — Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,990 $ 10,990 $ — $ — U.S. Treasuries $ 140,995 $ 140,995 $ — $ — Short-term investments: Short-term bond funds $ 19,821 $ 19,821 $ — $ — m. OPERATING LEASES. right-of-use non-lease n. SHARE REPURCHASES. The Company accounts for share repurchases by charging the excess of the repurchase price over the repurchased common stock’s par value entirely to retained earnings (accumulated deficit). All repurchased shares are retired and become authorized but unissued shares. The Company accrues for the shares purchased under the share repurchase plan based on the trade date. The Company may terminate or modify its share repurchase program at any time. o. REVENUE RECOGNITION. Product Revenues: The Company recognizes revenue when its customer obtains title of the promised goods, in an amount that reflects the consideration to which the Company expects to be entitled in exchange for these goods. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), which is the exclusive distributor of FIRDAPSE® in the United States. The Customer subsequently resells FIRDAPSE® to a small group of exclusive specialty pharmacies (“SPs”) whose dispensing activities for patients with specific payors may result in government-mandated or privately negotiated rebate obligations for the Company with respect to the purchase of FIRDAPSE®. To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company assesses the goods or services promised within each contract and determines those that are performance obligations by assessing whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under collaborative and license agreements. Collaborative and license agreement payments may include nonrefundable fees at the inception of the agreements, contingent payments for specific achievements designated in the agreements, and/or net profit-sharing payments on sales of products resulting from the collaborative and license arrangements. For a complete discussion of accounting for collaborative and licensing arrangements, see Revenues from Collaboration and Licensing Arrangements below. Product Revenue, Net: The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery or upon dispense to patient). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three and nine months ended September 30, 2022 and 2021. During the three and nine months ended September 30, 2022 and 2021, principally all of the Company’s sales of FIRDAPSE® in the United States were to its Customer. Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted Customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplates application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of September 30, 2022 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2022 and 2021. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: Prompt Payment Discounts: Funded Co-pay Assistance Program: Product Returns: Provider Chargebacks and Discounts: Government Rebates: The Company’s liability for these rebates consists of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in the distribution channel inventories at the end of each reporting period. Bridge and Patient Assistance Programs: Revenues from Collaboration and Licensing Arrangements: The Company analyzes license and collaboration arrangements pursuant to FASB ASC Topic 808, Collaborative Arrangement Guidance and Consideration, (“Topic 808”) to assess whether such arrangements, or transactions between arrangement participants, 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 or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of Topic 606. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For elements of collaboration arrangements that are not accounted for pursuant to guidance in Topic 606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance. The Company evaluates the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determines whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. The agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments as variable consideration in accordance with Topic 606. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations. After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations based on the same methodology used at contract inception. The Company recognizes sales-based royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty or net profit-sharing has been allocated has been satisfied. Payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Refer to Note 11 (Collaborative and Licensing Arrangements), for further discussion on the Company’s collaborative and licensing arrangements. p. RESEARCH AND DEVELOPMENT. q. ADVERTISING EXPENSE. In connection with the FDA approval and commercial launch of FIRDAPSE® in 2019, the Company began to incur advertising costs. Advertising costs are expensed as incurred. The C r. STOCK-BASED COMPENSATION. one s. CONCENTRATION OF RISK. The Company sells its product in the United States through an exclusive distributor (its Customer) to SPs. Therefore, its distributor and SPs account for principally all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product, which makes it difficult to evaluate its current business, predict its future prospects, and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, FIRDAPSE®, and expects FIRDAPSE® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to continue to effectively commercialize FIRDAPSE®. The Company relies exclusively on third parties to formulate and manufacture FIRDAPSE® and its drug candidates. The commercialization of FIRDAPSE® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of FIRDAPSE®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs. t. ROYALTIES. u. INCOME TAXES. The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not v. COMPREHENSIVE INCOME. available-for-sale w. NET INCOME PER COMMON SHARE. Diluted net income per common share is computed by dividing net income by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended For the Nine Months Ended 2022 2021 2022 2021 Basic weighted average common shares outstanding 103,318,572 103,196,550 102,967,280 103,470,762 Effect of dilutive securities 8,667,453 4,646,646 7,384,934 4,005,413 Diluted weighted average common shares outstanding 111,986,025 107,843,196 110,352,214 107,476,175 Outstanding common stock equivalents totaling approximately 12 thousand and 1.7 million, respectively, were excluded from the calculation of diluted net income per common share for both the three and nine months ended September 30, 2022, as their effect would be anti-dilutive. For the three and nine months ended September 30, 2021, approximately 2.6 million and 5.3 million shares, respectively, of common stock were excluded from the calculation of diluted net income per common share as their effect would be anti-dilutive. x. RECLASSIFICATIONS. y. RECENTLY ISSUED ACCOUNTING STANDARDS. |
Investments
Investments | 9 Months Ended |
Sep. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 3. Investments. Available-for-sale investments by security type were as follows (in thousands): Estimated Gross Gross Amortized At September 30, 2022: U.S. Treasuries—Cash equivalents $ 227,349 $ 45 $ — $ 227,304 Total $ 227,349 $ 45 $ — $ 227,304 At December 31, 2021: U.S. Treasuries—Cash equivalents $ 140,995 $ 2 $ — $ 140,993 Short-term bond funds 19,821 — (196 ) 20,017 Total $ 160,816 $ 2 $ (196 ) $ 161,010 There were realized losses from sale of available-for-sale available-for-sale The estimated fair values of available-for-sale September 30, 2022 Due in one year or less $ 227,349 |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Income (Loss) | 9 Months Ended |
Sep. 30, 2022 | |
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Accumulated Other Comprehensive Income (Loss) | 4. Accumulated Other Comprehensive Income (Loss). The following table summarizes the changes in accumulated other comprehensive income (loss), net of tax from unrealized gains (losses) on available-for-sale The amount reclassified out of accumulated other comprehensive income (loss), net of tax and into net income during the three and nine months ended September 30, 2022, was solely due to a realized loss from sale of available-for-sale Total Accumulated Balance at June 30, 2022 $ (130 ) Other comprehensive gain ( ) 33 Amount reclassified from accumulated other comprehensive income 129 Net current period other comprehensive gain (loss) 162 Balance at September 30, 2022 $ 32 Balance at December 31, 2021 $ (148 ) Other comprehensive gain ( ) (582 ) Amount reclassified from accumulated other comprehensive income 762 Net current period other comprehensive gain (loss) 180 Balance at September 30, 2022 $ 32 |
Inventory
Inventory | 9 Months Ended |
Sep. 30, 2022 | |
Inventory Disclosure [Abstract] | |
Inventory | 5. Inventory. Inventory consists of the following (in thousands): September 30, 2022 December 31, 2021 Raw materials $ — $ 1,769 Work-in-process 6,252 5,172 Finished goods 880 929 Total inventory $ 7,132 $ 7,870 |
Prepaid Expenses and Other Curr
Prepaid Expenses and Other Current Assets | 9 Months Ended |
Sep. 30, 2022 | |
Text Block [Abstract] | |
Prepaid Expenses and Other Current Assets | 6. Prepaid Expenses and Other Current Assets. Prepaid expenses and other current assets consist of the following (in thousands): September 30, 2022 December 31, 2021 Prepaid manufacturing costs $ 970 $ 307 Prepaid tax 74 564 Prepaid insurance 289 1,213 Prepaid subscriptions fees 470 909 Prepaid research fees 404 452 Prepaid commercialization expenses 573 195 Due from collaborative and licensing arrangements 324 105 Prepaid conference and travel expenses 206 279 Other 466 327 Total prepaid expenses and other current assets $ 3,776 $ 4,351 |
Operating Lease
Operating Lease | 9 Months Ended |
Sep. 30, 2022 | |
Operating Lease, Lease Income [Abstract] | |
Operating Leases | 7. Operating Lease. The Company has an operating lease agreement for its corporate office. The lease includes an option to extend the lease for up to 5 years and options to terminate the lease within 6 and 7.6 years. There are no obligations under finance leases. The Company entered into an agreement in May 2020 that amended its lease for its office facilities. Under the amended lease, the Company’s leased space increased from approximately 7,800 square feet of space to approximately 10,700 square feet of space. The amended lease commenced in March 2021 when construction of the asset was completed and space became available for use. Consequently, the Company recorded the effects of the amended lease during Q1 2021. The components of lease expense were as follows (in thousands): For the Three Months Ended For the Nine Months Ended 2022 2021 2022 2021 Operating lease cost $ 108 $ 108 $ 324 $ 272 Supplemental cash flow information related to lease was as follows (in thousands): For the Nine Months Ended 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 368 $ 29 Right-of-use Operating lease $ 67 $ 58 Supplemental balance sheet information related to lease was as follows (in thousands): September 30, 2022 December 31, 2021 Operating lease right-of-use $ 2,833 $ 3,017 Other current liabilities $ 330 $ 308 Operating lease liabilities, net of current portion 3,643 3,894 Total operating lease liabilities $ 3,973 $ 4,202 As of September 30, 2022 and December 31, 2021, the weighted average remaining lease term was 8.6 years and 9.3 years, respectively. The weighted average discount rate used to determine the operating lease liabilities was 4.51% as of September 30, 2022 and December 31, 2021. Remaining payments of lease liabilities as of September 30, 2022 were as follows (in thousands): 2022 (remaining three months) $ 123 2023 506 2024 522 2025 537 2026 553 Thereafter 2,598 Total lease payments 4,839 Less: imputed interest (866 ) Total $ 3,973 Rent expense was approximately $0.1 million and $0.3 million for both the three and nine months ended September 30, 2022 and 2021, respectively. |
Property and Equipment, Net
Property and Equipment, Net | 9 Months Ended |
Sep. 30, 2022 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 8. Property and Equipment, Net. Property and equipment, net consists of the following (in thousands): September 30, 2022 December 31, 2021 Computer equipment $ 51 $ 51 Furniture and equipment 223 203 Leasehold improvements 980 980 Less: Accumulated depreciation (372 ) (275 ) Total property and equipment, net $ 882 $ 959 |
License and Acquired Intangible
License and Acquired Intangibles, Net. | 9 Months Ended |
Sep. 30, 2022 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
License and Acquired Intangibles, Net. | 9. License and Acquired Intangibles, Net. The following table presents the Company’s intangible assets at September 30, 2022 (in thousands): Gross Carrying Value Accumulated Amortization Net Carrying Value Intangible assets: License and acquired intangibles for Ruzurgi® $ 33,569 $ 518 $ 33,051 Total $ 33,569 $ 518 $ 33,051 The Company amortizes its definite-lived intangible assets using the straight-line method, which is considered the best estimate of economic benefit, over its estimated useful life of approximately years. Amortization of these assets during each of the next five years is estimated to be approximately $ million per year. The Company recorded approximately $ million in amortization expense related to the licensed and acquired intangibles for Ruzurgi® during the three and nine months ended September 30, 2022. There were no intangible assets at December 31, 2021. If all or a portion of the intangible assets are deemed not recoverable, the Company would estimate the fair value of the assets and record an impairment loss. |
Accrued Expenses and Other Liab
Accrued Expenses and Other Liabilities | 9 Months Ended |
Sep. 30, 2022 | |
Payables and Accruals [Abstract] | |
Accrued Expenses and Other Liabilities | 10. Accrued Expenses and Other Liabilities. Accrued expenses and other liabilities consist of the following (in thousands): September 30, 2022 December 31, 2021 Accrued preclinical and clinical trial expenses $ 460 $ 659 Accrued professional fees 2,503 2,391 Accrued compensation and benefits 3,412 4,035 Accrued license fees 15,528 12,819 Accrued purchases — 2,045 Operating lease liability 330 308 Accrued variable consideration 2,988 1,716 Accrued income tax 3,744 79 Due to licensor 12,950 — Other 237 243 Current accrued expenses and other liabilities 42,152 24,295 Lease liability – non-current 3,643 3,894 Due to licensor – non-current 14,749 — Non-current 18,392 3,894 Total accrued expenses and other liabilities $ 60,544 $ 28,189 |
Collaborative and Licensing Arr
Collaborative and Licensing Arrangements | 9 Months Ended |
Sep. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborative and Licensing Arrangements | 11. Collaborative and Licensing Arrangements. Endo In December 2018, the Company entered into a collaboration and license agreement (Collaboration) with Endo, for the further development and commercialization of generic Sabril® (vigabatrin) tablets through Endo’s U.S. Generic Pharmaceuticals segment, doing business as Par Pharmaceutical (Par). Under the Collaboration, Endo assumes all development, manufacturing, clinical, regulatory, sales and marketing costs under the collaboration, while the Company is responsible for exercising commercially reasonable efforts to develop, or cause the development of, a final finished, stable dosage form of generic Sabril® tablets. Under the terms of the Collaboration, the Company has received an up-front payment, and will receive a milestone payment, and a sharing of defined net profits upon commercialization from Endo consisting of a mid-double digit percent of net sales of generic Sabril®. The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is The Company evaluated the license agreement with Endo to determine whether it is a collaborative arrangement for purposes of Topic 808. As the Company shares in the significant risks and rewards, the Company has concluded that this is a collaborative arrangement. As developing a final finished dosage form of a generic product in exchange for consideration is not an output of the Company’s ongoing activities, Endo does not represent a contract with a customer. However, Topic 808 does not provide guidance on the recognition of consideration exchanged or accounting for the obligations that may arise between the parties. The Company concluded that ASC Topic 730, Research and Development The collaborative agreement included a nonrefundable upfront license fee that was recognized upon receipt following execution of the collaborative arrangement for vigabatrin tablets. The collaborative agreement provides for a $2.0 million milestone payment on the commercial launch of the product by Endo/Par. As of September 30, 2022 and 2021, milestone payment had been earned. There were no revenues from this collaborative arrangement for the three and nine months ended September 30, 2022 or 2021. There were no expenses incurred, net, in connection with the collaborative arrangement for the three and nine months ended September 30, 2022. Total expenses incurred, net, in connection with the collaborative agreement for three and nine months ended September 30, 2021 were approximately $0 and $45,000, respectively. These expenses have been included in research and development expenses in the accompanying consolidated statements of operations and comprehensive income. KYE Pharmaceuticals Inc. In August 2020, the Company entered into a collaboration and license agreement with KYE Pharmaceuticals Inc. (KYE), for the commercialization of FIRDAPSE® in Canada. Under the agreement, Catalyst granted KYE an exclusive license to commercialize and market FIRDAPSE® in Canada. KYE assumes all selling and marketing costs under the collaboration, while the Company is responsible for supply of FIRDAPSE® based on the collaboration partner’s purchase orders. Under the terms of the agreement, the Company will receive an up-front payment, received payment upon transfer of Marketing Authorization and delivery of commercial product, received payment for supply of FIRDAPSE®, will receive milestone payments, and a sharing of defined net profits upon commercialization from KYE consisting of a mid-double-digit percent of net sales of FIRDAPSE®. The Company has also agreed to a sharing of certain development expenses. Unless terminated earlier in accordance with its terms, the collaboration continues in effect until the date that is ten years following the commercial launch of the product in Canada. This agreement is in form identified as a collaborative agreement and the Company has concluded for accounting purposes that it also represents a contract with a customer. This is because the Company grants to KYE a license and provides supply of FIRDAPSE® in exchange for consideration, which are outputs of the Company’s ongoing activities. Accordingly, the Company has concluded that this collaborative arrangement will be accounted for pursuant to Topic 606. The collaborative agreement included a nonrefundable upfront license fee that was recognized upon transfer of the license based on a determination that the right is provided as the intellectual property exists at the point in time in which the license is granted. Under the arrangement, the Company will receive profit-sharing reports within nine days after quarter end from KYE. Revenue from sales of FIRDAPSE® by KYE will be recognized in the quarter in which the sales occurred. Revenues from the arrangement with KYE for the three and nine months ended September 30, 2022 and 2021 were not material. Revenue from sales of FIRDAPSE® to KYE is included in product revenue, net and revenues from profit sharing and milestones is included in license and other revenue in the accompanying consolidated statements of operations and comprehensive income. Total expenses incurred, net in connection with the agreement with KYE for the three and nine months ended September 30, 2022 and 2021 were DyDo Pharma, Inc. On June 28, 2021, the Company entered into a license agreement with DyDo Pharma, Inc. (DyDo), for the development and commercialization of FIRDAPSE® in Japan. Under the agreement, DyDo has joint rights to develop FIRDAPSE®, and exclusive rights to commercialize the product, in Japan. DyDo is responsible for funding all clinical, regulatory, marketing and commercialization activities in Japan, while the Company is responsible for clinical and commercial supply based on purchase orders, as well as providing support to DyDo in its efforts to obtain regulatory approval for the product from the Japanese regulatory authorities. Under the terms of the agreement, the Company has earned an up-front payment and may earn further development and sales milestones for FIRDAPSE®, as well as revenue on product supplied to DyDo. The Company has concluded that this license agreement will be accounted for pursuant to Topic 606. The agreement included a nonrefundable upfront license fee that was recognized upon the effective date of the agreement as the intellectual property exists at the point in time in which the right to the license is granted. The Company determined the granting of the right to the license is distinct from the supply of FIRDAPSE® and represents a separate performance obligation in the agreement. The agreement includes milestones that are considered a sales-based royalty in which the license is deemed to be the predominant item to which these milestones relate. Revenue will be recognized when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty has been allocated has been satisfied. Additionally, the agreement includes regulatory milestone payments which represent variable consideration, and due to uncertainty are fully constrained and only recognized when the uncertainty is subsequently resolved. For clinical and commercial supply of the product, the Company will recognize revenue when the Customer obtains control of the Company’s product, which will occur at a point in time which is generally at time of shipment. There were $0 and $0.5 million in revenue, respectively, from the arrangement with DyDo for the three and nine months ended September 30, 2022, which is included in product revenue, net in the accompanying consolidated statements of operations and comprehensive income. Revenues from the arrangement with DyDo for the three and nine months ended September 30, 2021 were approximately $0 and $2.7 million, respectively, relating to the nonrefundable upfront license fee, which is included in licensing and other revenue in the accompanying consolidated statements of operations and comprehensive income. As of September 30, 2022, no milestone payments have been earned. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 12. Commitments and Contingencies. In May 2019, the FDA approved a New Drug Application (NDA) filed by Jacobus Pharmaceutical Company, Inc., or Jacobus, for Ruzurgi®, another formulation of amifampridine, for the treatment of pediatric LEMS patients (ages 6 to under 17). As a result of the approval of Ruzurgi® and based on the Company’s belief that the approval violated its statutory rights to exclusivity under the Orphan Drug Act, in June 2019 the Company filed suit against the FDA in the U.S. District Court for the Southern District of Florida challenging this approval, and Jacobus intervened in the Company’s case. While the District Court granted summary judgement in favor of the FDA and Jacobus in 2020, on September 30, 2021, a three-judge panel of the U.S. Court of Appeals for the 11th Circuit issued a unanimous decision overturning the District Court’s decision. On January 31, 2022, after Jacobus’ motions for stay were denied by the 11th Circuit and the U.S. Supreme Court, the U.S. District Court for the Southern District of Florida, based on a mandate issued by the 11th Circuit, entered summary judgement in the Company’s favor. On February 1, 2022, the FDA informed Jacobus that, consistent with the 11th Circuit’s decision, the final approval of the Ruzurgi® NDA had been switched to a tentative approval until the 7-year orphan-drug exclusivity, or ODE, for FIRDAPSE® has expired, and Ruzurgi® was no longer available on the U.S. market. Further, in October 2020, the Company filed lawsuits against Jacobus and the specialty pharmacy marketing Ruzurgi® for infringement of the ‘893 patent. The lawsuits alleged that the Ruzurgi® product infringes the ‘893 patent when administered in accordance with its product labeling. Further, in August 2021, the lawsuits were amended to include alleged infringement of the ‘128 patent. The lawsuit sought damages and injunctive relief to prevent further marketing of Ruzurgi® in violation of the Company’s patent rights. On July 11, 2022, the Company settled certain of its disputes with Jacobus. In connection with the settlement, the Company licensed the rights to develop and commercialize Ruzurgi® in the United States and Mexico (the “Territory”). Simultaneously, the Company purchased, among other intellectual property rights, Jacobus’ U.S. patents related to Ruzurgi®, its new drug applications in the United States for Ruzurgi®, and certain Ruzurgi® inventory previously manufactured by Jacobus. At the same time, the Company received a license from Jacobus for use of its know-how related to the manufacture of Ruzurgi®. Further, the Company settled the patent case, which has been dismissed without prejudice. Finally, Jacobus agreed that until the later of (i) the expiration of the royalty term or (ii) December 31, 2034, Jacobus and its affiliates, will not, directly or indirectly, research, develop, manufacture, commercialize, distribute, use or otherwise exploit any product competitive to FIRDAPSE® or Ruzurgi® in the Territory, and Laura Jacobus, the sole shareholder of Jacobus, and two of Jacobus’ other officers, also signed individual non-competition agreements containing the same terms. The Company’s New Drug Submission filing for FIRDAPSE® for the symptomatic treatment of LEMS was approved when Health Canada issued a Notice of Compliance, or NOC, on July 31, 2020. In August 2020, the Company entered into a license agreement with KYE Pharmaceuticals, or KYE, pursuant to which the Company licensed to KYE the Canadian rights for FIRDAPSE® for the treatment of LEMS. On August 10, 2020, Health Canada issued a NOC to Medunik (Jacobus’ licensee in Canada for Ruzurgi®) for the treatment of LEMS. Shortly thereafter, the Company initiated a legal proceeding in Canada seeking judicial review of Health Canada’s decision to issue the NOC for Ruzurgi® as incorrect and unreasonable under Canadian law. Data protection, per Health Canada regulations, is supposed to prevent Health Canada from issuing an NOC to a drug that directly or indirectly references an innovative drug’s data, for eight years from the date of the innovative drug’s approval. The Ruzurgi® Product Monograph clearly references pivotal nonclinical carcinogenicity and reproductive toxicity data for amifampridine phosphate developed by the Company. As such, the Company believes that its data was relied upon to establish the nonclinical safety profile of Ruzurgi® needed to meet the standards of the Canadian Food and Drugs Act. On June 3, 2021, the Company announced a positive decision in this proceeding that quashed the NOC previously issued for Ruzurgi® and remanded the matter to the Minister of Health to redetermine its decision to grant marketing authorization to Ruzurgi® in spite of FIRDAPSE®’s data protection rights. However, on June 28, 2021, the Company announced that Health Canada had re-issued an NOC for Ruzurgi®, once again allowing the product to be marketed in Canada for patients with LEMS. As a result, in July 2021 the Company, along with its partner in Canada, KYE, filed a second suit against Health Canada to overturn this decision. On March 11, 2022, the Company announced that it had received a favorable decision from the Canadian court setting aside, for the second time, the decision of Health Canada approving Ruzurgi® for the treatment of LEMS patients. In its ruling, the court determined that the Minister of Health’s approach to evaluating whether FIRDAPSE®’s data deserved protection based on FIRDAPSE®’s status as an innovative drug, which protects by regulation the use of such data as part of a submission seeking an NOC for eight years from approval of the innovative drug, was legally flawed and not supported by the evidence. As a result, the matter has, once again, been remanded to the Minister of Health to redetermine its decision on Ruzurgi® in light of the court’s ruling, and, in that regard, the Minister of Health recently sought appellate review of the Court’s decision (which appeal is ongoing). As a result, Ruzurgi® is no longer currently available for sale in Canada pending both a potential redetermination by the Minister and also the appeal of the second favorable decision. The settlement between Catalyst and Jacobus described above did not resolve the outstanding issues between the parties in Canada, and this dispute continues. There can be no assurance as to the outcome of this proceeding. Additionally, from time to time the Company may become involved in legal proceedings arising in the ordinary course of business. Except as set forth above, the Company believes that there is no other litigation pending at this time that could have, individually or in the aggregate, a material adverse effect on its results of operations, financial condition, or cash flows. |
Agreements
Agreements | 9 Months Ended |
Sep. 30, 2022 | |
Text Block [Abstract] | |
Agreements | 13. Agreements. a. LICENSE AGREEMENT FOR FIRDAPSE® FIRDAPSE® FIRDAPSE® 100 FIRDAPSE® On May 29, 2019, the Company and BioMarin entered into an amendment to the Company’s license agreement for FIRDAPSE®. Under the amendment, the Company has expanded its commercial territory for FIRDAPSE®, which originally was comprised of North America, to include Japan. Additionally, the Company has an option to further expand its territory under the license agreement to include most of Asia, as well as Central and South America, upon the achievement of certain milestones in Japan. Under the amendment, the Company will pay royalties to our licensor on net sales in Japan of a similar percentage to the royalties that the Company is currently paying under its original license agreement for North America. In January 2020, the Company was advised that BioMarin has transferred certain rights under the license agreement to SERB S.A. b. LICENSE AGREEMENT FOR RUZURGI® Ruzurgi® Pursuant to the terms of the license agreement, the Company paid Jacobus a $10 million up-front Firdapse® million unless a competing product or generic version of Firdapse® is being marketed or sold in the United States. As these minimum payments are not probable and estimable, they will be charged to cost of sales as revenue from product sales is recognized. Royalties over the minimum, if any, will be paid based on the agreement terms on a quarterly basis. Assets acquired as part of the license agreement include among other intellectual property rights, Jacobus’ U.S. patents related to Ruzurgi®, its new drug applications in the United States for Ruzurgi®, its Trademark for Ruzurgi®, the Orphan Drug Designation for Ruzurgi® and a license from Jacobus for use of its know-how related to the manufacture of Ruzurgi®. Additionally, the Company also purchased from Jacobus approximately $4.1 million of Ruzurgi® inventory previously manufactured by Jacobus, which have been recorded as an expense in research and development expenses in the accompanying consolidated statement of operations and comprehensive income for the three and nine months ended September 30, 2022. Under business combination guidance, the screen test states that if substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset or group of similar identifiable assets, the set is not considered a business and is accounted for as an asset acquisition. The Company has determined that the screen test was not met. However, the Company determined that the acquisition did not meet the definition of a business under ASC 805, Business Combination. The Company believes that the licensing agreement and other assets acquired from Jacobus are similar and considered them all to be intangible assets with the exception of the inventory acquired. As the screen test was not met, further determination was required to determine that the Company had not acquired inputs and processes that have the ability to create outputs, which would meet the requirements of a business, and therefore, determined that this was an asset acquisition. The Company accounted for the Jacobus license agreement as an asset acquisition under ASC 805, which requires the acquiring entity in an asset acquisition to recognize assets acquired and liabilities assumed based on the cost to the acquiring entity on a relative fair value basis, which includes consideration given. Goodwill is not recognized in an asset acquisition and any excess consideration transferred over the fair value of the net assets acquired is allocated to the identifiable assets based on relative fair values. The total purchase price was allocated to the acquired assets based on their relative fair values, as follows (in thousands): License and acquired intangibles $ 33,569 Acquired research and development expensed from asset acquisition 4,130 Total purchase price $ 37,699 The straight-line method is used to amortize the license and acquired intangibles, as disclosed in Note 9 (License and Acquired Intangibles, Net). c. AGREEMENTS FOR DRUG MANUFACTURING, DEVELOPMENT, PRECLINICAL AND CLINICAL STUDIES. |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 2022 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income Taxes. The Company’s effective income tax rate was 19.7% and 24.3% for the nine months ended September 30, 2022 and 2021, respectively. Differences in the effective tax and the statutory federal income tax rate of 21% are driven by state income taxes and anticipated annual permanent differences and offset by the orphan drug credit claimed. The Company had no uncertain tax positions as of September 30, 2022 and December 31, 2021. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2022 | |
Equity [Abstract] | |
Stockholders' Equity | 15. Stockholders’ Equity. Preferred Stock The Company has 5,000,000 shares of authorized preferred stock, $0.001 par value per share, at September 30, 2022 and December 31, 2021. No shares of preferred stock were outstanding at September 30, 2022 and December 31, 2021. Common Stock The Company has 200,000,000 shares of authorized common stock, par value $0.001 per share. At September 30, 2022 and December 31, 2021, 104,064,683 and 102,992,913 shares, respectively, of common stock were issued and outstanding. Each holder of common stock is entitled to one vote of each share of common stock held of record on all matters on which stockholders generally are entitled to vote. Share Repurchases In March 2021, the Company’s Board of Directors approved a share repurchase program that authorizes the repurchase of up to $40 million of the Company’s common stock, pursuant to a repurchase plan under Rule 10b-18 , respectively , 2020 Shelf Registration Statement On July 23, 2020, the Company filed a shelf registration statement with the SEC to sell up to $200 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the “2020 Shelf Registration Statement”). The 2020 Shelf Registration Statement (file no. 333-240052) |
Stock Compensation
Stock Compensation | 9 Months Ended |
Sep. 30, 2022 | |
Share-based Payment Arrangement [Abstract] | |
Stock Compensation | 16. Stock Compensation. For the three and nine months ended September 30, 2022 and 2021, the Company recorded stock-based compensation expense as follows (in thousands): Three months ended Nine months ended 2022 2021 2022 2021 Research and development $ 450 $ 428 $ 1,307 $ 1,193 Selling, general and administrative 1,605 1,084 4,674 3,408 Total stock-based compensation $ 2,055 $ 1,512 $ 5,981 $ 4,601 Stock Options As of September 30, 2022, there were outstanding stock options to purchase 12,516,584 shares of common stock, of which stock options to purchase 8,124,977 shares of common stock were exercisable. During the three and nine months ended September 30, 2022, the Company granted seven-year term options to purchase an aggregate of 12,000 and 455,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.6 million and $4.8 million, respectively, during the three and nine months ended September 30, 2022. During the three and nine months ended September 30, 2021, the Company granted seven-year term options to purchase an aggregate of 400,000 and 1,030,000 shares, respectively, of the Company’s common stock to employees. The Company recorded stock-based compensation related to stock options totaling $1.4 million and $4.2 million, respectively, during the three and nine months ended September 30, 2021. During the three and nine months ended September 30, 2022, options to purchase 1,355,335 shares and 2,064,700 shares, respectively, of the Company’s common stock were exercised, with proceeds of $4.0 million and $6.4 million respectively, to the Company. During the three and nine months ended September 30, 2021, options to purchase 941,164 shares and 1,114,494 shares, respectively, of the Company’s common stock were exercised, with proceeds of $3.0 million and $3.4 million respectively, to the Company. As of September 30, 2022, there was approximately $8.3 million of unrecognized compensation expense related to non-vested Restricted Stock Units There were no grants of restricted stock units to employees or directors during the three months ended September 30, 2022. The Company granted 474,500 restricted stock units during the nine months ended September 30, 2022. There were no grants of restricted stock units to employees or directors during the three or nine months ended September 30, 2021. During the three and nine months ended September 30, 2022, the Company recorded non-cash stock-based compensation expense related to restricted stock units totaling non-cash As of September 30, 2022, there was approximately $3.0 million of unrecognized compensation expense related to non-vested |
Basis of Presentation and Sig_2
Basis of Presentation and Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
INTERIM FINANCIAL STATEMENTS | a. INTERIM FINANCIAL STATEMENTS. 10-Q In the opinion of management, the accompanying unaudited interim consolidated financial statements of the Company contain all adjustments (consisting of only normal recurring adjustments) necessary to present fairly the financial position of the Company as of the dates and for the periods presented. Accordingly, these consolidated statements should be read in conjunction with the financial statements and notes thereto for the year ended December 31, 2021 included in the 2021 Annual Report on Form 10-K |
PRINCIPLES OF CONSOLIDATION. | b. PRINCIPLES OF CONSOLIDATION. |
USE OF ESTIMATES | c. USE OF ESTIMATES. |
CASH AND CASH EQUIVALENTS | d. CASH AND CASH EQUIVALENTS. |
INVESTMENTS | e. INVESTMENTS. The U.S. Treasuries held at September 30, 2022 are classified as available-for-sale non-current non-current The Company records available-for-sale in the consolidated statements of operations and comprehensive income, available-for-sale available-for-sale available-for-sale |
ACCOUNTS RECEIVABLE, NET | f. ACCOUNTS RECEIVABLE, NET. |
INVENTORY | g. INVENTORY Products that have been approved by the FDA or other regulatory authorities, such as FIRDAPSE®, are also used in clinical programs to assess the safety and efficacy of the products for usage in treating diseases that have not been approved by the FDA or other regulatory authorities. The form of FIRDAPSE® utilized for both commercial and clinical programs is identical and, as a result, the inventory has an “alternative future use” as defined in authoritative guidance. Raw materials associated with clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes and, therefore, does not have an “alternative future use”. The Company evaluates for potential excess inventory by analyzing current and future product demand relative to the remaining product shelf life. The Company builds demand forecasts by considering factors such as, but not limited to, overall market potential, market share, market acceptance, and patient usage. |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | h. PREPAID EXPENSES AND OTHER CURRENT ASSETS. pre-clinical |
PROPERTY AND EQUIPMENT, NET | i. PROPERTY AND EQUIPMENT, NET. three five five |
INTANGIBLE ASSETS, NET | j. INTANGIBLE ASSETS, NET. The Company reviews intangible assets with finite lives for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If indicators of impairment exist, an impairment test is performed to assess the recoverability of the affected assets by determining whether the carrying amount of such assets exceeds the undiscounted expected future cash flows. If the affected assets are deemed not recoverable, the Company would estimate the fair value of the assets and record an impairment loss. There were no impairment charges recognized on finite lived intangibles for the three or nine months ended September 30, 2022. There were no finite lived intangible assets at December 31, 2021. |
FAIR VALUE OF FINANCIAL INSTRUMENTS | k. FAIR VALUE OF FINANCIAL INSTRUMENTS. |
FAIR VALUE MEASUREMENTS | l. FAIR VALUE MEASUREMENTS. Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which are typically based on an entity’s own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company’s assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability. Fair Value Measurements at Reporting Date Using (in thousands) Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,336 $ 10,336 $ — $ — U.S. Treasuries $ 227,349 $ 227,349 $ — $ — Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,990 $ 10,990 $ — $ — U.S. Treasuries $ 140,995 $ 140,995 $ — $ — Short-term investments: Short-term bond funds $ 19,821 $ 19,821 $ — $ — |
OPERATING LEASES | m. OPERATING LEASES. right-of-use non-lease |
SHARE REPURCHASES | n. SHARE REPURCHASES. The Company accounts for share repurchases by charging the excess of the repurchase price over the repurchased common stock’s par value entirely to retained earnings (accumulated deficit). All repurchased shares are retired and become authorized but unissued shares. The Company accrues for the shares purchased under the share repurchase plan based on the trade date. The Company may terminate or modify its share repurchase program at any time. |
REVENUE RECOGNITION | o. REVENUE RECOGNITION. Product Revenues: The Company recognizes revenue when its customer obtains title of the promised goods, in an amount that reflects the consideration to which the Company expects to be entitled in exchange for these goods. Subsequent to receiving FDA approval, the Company entered into an arrangement with one distributor (the “Customer”), which is the exclusive distributor of FIRDAPSE® in the United States. The Customer subsequently resells FIRDAPSE® to a small group of exclusive specialty pharmacies (“SPs”) whose dispensing activities for patients with specific payors may result in government-mandated or privately negotiated rebate obligations for the Company with respect to the purchase of FIRDAPSE®. To determine revenue recognition for arrangements that are within the scope of Accounting Standards Codification (“ASC”) Topic 606 – Revenue from Contracts with Customers (“Topic 606”), the Company performs the following five steps: (i) identify the contract(s) with a customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the performance obligations in the contract, and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company assesses the goods or services promised within each contract and determines those that are performance obligations by assessing whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. For a complete discussion of accounting for product revenue, see Product Revenue, Net below. The Company also may generate revenues from payments received under collaborative and license agreements. Collaborative and license agreement payments may include nonrefundable fees at the inception of the agreements, contingent payments for specific achievements designated in the agreements, and/or net profit-sharing payments on sales of products resulting from the collaborative and license arrangements. For a complete discussion of accounting for collaborative and licensing arrangements, see Revenues from Collaboration and Licensing Arrangements below. Product Revenue, Net: The Company recognizes revenue on product sales when the Customer obtains control of the Company’s product, which occurs at a point in time (upon delivery or upon dispense to patient). Product revenue is recorded net of applicable reserves for variable consideration, including discounts and allowances. The Company’s payment terms range between 15 and 30 days. Shipping and handling costs for product shipments occur prior to the customer obtaining control of the goods and are recorded in cost of sales. If taxes should be collected from the Customer relating to product sales and remitted to governmental authorities, they will be excluded from revenue. The Company expenses incremental costs of obtaining a contract when incurred if the expected amortization period of the asset that the Company would have recognized is one year or less. However, no such costs were incurred during the three and nine months ended September 30, 2022 and 2021. During the three and nine months ended September 30, 2022 and 2021, principally all of the Company’s sales of FIRDAPSE® in the United States were to its Customer. Reserves for Variable Consideration: These estimates take into consideration a range of possible outcomes which are probability-weighted in accordance with the expected value method in Topic 606 for relevant factors such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted Customer buying and payment patterns. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the respective underlying contracts. The amount of variable consideration which is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contract will not occur in a future period. The Company’s analyses also contemplates application of the constraint in accordance with the guidance, under which it determined a material reversal of revenue would not occur in a future period for the estimates detailed below as of September 30, 2022 and, therefore, the transaction price was not reduced further during the three and nine months ended September 30, 2022 and 2021. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results in the future vary from the Company’s estimates, the Company will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. Trade Discounts and Allowances: Prompt Payment Discounts: Funded Co-pay Assistance Program: Product Returns: Provider Chargebacks and Discounts: Government Rebates: The Company’s liability for these rebates consists of invoices received for claims from prior quarters that have not been paid or for which an invoice has not yet been received, estimates of claims for the current quarter, and estimated future claims that will be made for product that has been recognized as revenue, but which remains in the distribution channel inventories at the end of each reporting period. Bridge and Patient Assistance Programs: Revenues from Collaboration and Licensing Arrangements: The Company analyzes license and collaboration arrangements pursuant to FASB ASC Topic 808, Collaborative Arrangement Guidance and Consideration, (“Topic 808”) to assess whether such arrangements, or transactions between arrangement participants, 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 or are more akin to a vendor-customer relationship. In making this evaluation, the Company considers whether the activities of the collaboration are considered to be distinct and deemed to be within the scope of the collaborative arrangement guidance or if they are more reflective of a vendor-customer relationship and, therefore, within the scope of Topic 606. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For elements of collaboration arrangements that are not accounted for pursuant to guidance in Topic 606, an appropriate recognition method is determined and applied consistently, generally by analogy to the revenue from contracts with customers guidance. The Company evaluates the performance obligations promised in the contract that are based on goods and services that will be transferred to the customer and determines whether those obligations are both (i) capable of being distinct and (ii) distinct in the context of the contract. Goods or services that meet these criteria are considered distinct performance obligations. The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. The agreements provide for milestone payments upon achievement of development and regulatory events. The Company accounts for milestone payments as variable consideration in accordance with Topic 606. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential transaction price and the likelihood that the transaction price will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and, if so, these options are considered performance obligations. After contract inception, the transaction price is reassessed at every period end and updated for changes such as resolution of uncertain events. Any change in the overall transaction price is allocated to the performance obligations based on the same methodology used at contract inception. The Company recognizes sales-based royalties or net profit-sharing when the later of (a) the subsequent sale occurs, or (b) the performance obligation to which the sales-based royalty or net profit-sharing has been allocated has been satisfied. Payments to and from the collaborator are presented in the statement of operations based on the nature of the Company’s business operations, the nature of the arrangement, including the contractual terms, and the nature of the payments. Refer to Note 11 (Collaborative and Licensing Arrangements), for further discussion on the Company’s collaborative and licensing arrangements. |
RESEARCH AND DEVELOPMENT | p. RESEARCH AND DEVELOPMENT. |
ADVERTISING EXPENSE | q. ADVERTISING EXPENSE. In connection with the FDA approval and commercial launch of FIRDAPSE® in 2019, the Company began to incur advertising costs. Advertising costs are expensed as incurred. The C |
STOCK-BASED COMPENSATION | r. STOCK-BASED COMPENSATION. one |
CONCENTRATION OF CREDIT RISK | s. CONCENTRATION OF RISK. The Company sells its product in the United States through an exclusive distributor (its Customer) to SPs. Therefore, its distributor and SPs account for principally all of its trade receivables and net product revenues. The creditworthiness of its Customer is continuously monitored, and the Company has internal policies regarding customer credit limits. The Company estimates an allowance for expected credit loss primarily based on the credit worthiness of its Customer, historical payment patterns, aging of receivable balances and general economic conditions. The Company currently has a single product, which makes it difficult to evaluate its current business, predict its future prospects, and forecast financial performance and growth. The Company has invested a significant portion of its efforts and financial resources in the development and commercialization of the lead product, FIRDAPSE®, and expects FIRDAPSE® to constitute virtually all of product revenue for the foreseeable future. The Company’s success depends on its ability to continue to effectively commercialize FIRDAPSE®. The Company relies exclusively on third parties to formulate and manufacture FIRDAPSE® and its drug candidates. The commercialization of FIRDAPSE® and any other drug candidates, if approved, could be stopped, delayed or made less profitable if those third parties fail to provide sufficient quantities of product or fail to do so at acceptable quality levels or prices. The Company does not intend to establish its own manufacturing facilities. The Company is using the same third-party contractors to manufacture, supply, store and distribute drug supplies for clinical trials and for the commercialization of FIRDAPSE®. If the Company is unable to continue its relationships with one or more of these third-party contractors, it could experience delays in the development or commercialization efforts as it locates and qualifies new manufacturers. The Company intends to rely on one or more third-party contractors to manufacture the commercial supply of its drugs. |
ROYALTIES | t. ROYALTIES. |
INCOME TAXES | u. INCOME TAXES. The Company recognizes the financial statement benefit of a tax position only after determining that the relevant tax authority would more likely than not sustain the position following an audit. For tax positions meeting the more-likely-than-not |
COMPREHENSIVE INCOME | v. COMPREHENSIVE INCOME. available-for-sale |
NET INCOME PER COMMON SHARE | w. NET INCOME PER COMMON SHARE. Diluted net income per common share is computed by dividing net income by the weighted average number of common shares outstanding, increased by the assumed conversion of other potentially dilutive securities during the period. The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended For the Nine Months Ended 2022 2021 2022 2021 Basic weighted average common shares outstanding 103,318,572 103,196,550 102,967,280 103,470,762 Effect of dilutive securities 8,667,453 4,646,646 7,384,934 4,005,413 Diluted weighted average common shares outstanding 111,986,025 107,843,196 110,352,214 107,476,175 Outstanding common stock equivalents totaling approximately 12 thousand and 1.7 million, respectively, were excluded from the calculation of diluted net income per common share for both the three and nine months ended September 30, 2022, as their effect would be anti-dilutive. For the three and nine months ended September 30, 2021, approximately 2.6 million and 5.3 million shares, respectively, of common stock were excluded from the calculation of diluted net income per common share as their effect would be anti-dilutive. |
RECLASSIFICATIONS | x. RECLASSIFICATIONS. |
RECENTLY ISSUED ACCOUNTING STANDARDS | y. RECENTLY ISSUED ACCOUNTING STANDARDS. |
Basis of Presentation and Sig_3
Basis of Presentation and Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Fair Value Measurement Specific to Assets or Liability | Fair Value Measurements at Reporting Date Using (in thousands) Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,336 $ 10,336 $ — $ — U.S. Treasuries $ 227,349 $ 227,349 $ — $ — Balances as of Quoted Prices in Significant Other Significant Cash and cash equivalents: Money market funds $ 10,990 $ 10,990 $ — $ — U.S. Treasuries $ 140,995 $ 140,995 $ — $ — Short-term investments: Short-term bond funds $ 19,821 $ 19,821 $ — $ — |
Basic and Dilutive Weighted Average Common Shares | The following table reconciles basic and diluted weighted average common shares: For the Three Months Ended For the Nine Months Ended 2022 2021 2022 2021 Basic weighted average common shares outstanding 103,318,572 103,196,550 102,967,280 103,470,762 Effect of dilutive securities 8,667,453 4,646,646 7,384,934 4,005,413 Diluted weighted average common shares outstanding 111,986,025 107,843,196 110,352,214 107,476,175 |
Investments (Tables)
Investments (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-Sale Investments by Security type | Available-for-sale investments by security type were as follows (in thousands): Estimated Gross Gross Amortized At September 30, 2022: U.S. Treasuries—Cash equivalents $ 227,349 $ 45 $ — $ 227,304 Total $ 227,349 $ 45 $ — $ 227,304 At December 31, 2021: U.S. Treasuries—Cash equivalents $ 140,995 $ 2 $ — $ 140,993 Short-term bond funds 19,821 — (196 ) 20,017 Total $ 160,816 $ 2 $ (196 ) $ 161,010 |
Estimated Fair Values of Available for Sale Securities | The estimated fair values of available-for-sale September 30, 2022 Due in one year or less $ 227,349 |
Accumulated Other Comprehensi_2
Accumulated Other Comprehensive Income (Loss) (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract] | |
Summary of Reclassifications out of Accumulated Other Comprehensive Income (loss) | There were no reclassifications out of accumulated other comprehensive income (loss) during the three and nine months ended September 30, 2021. Total Accumulated Balance at June 30, 2022 $ (130 ) Other comprehensive gain ( ) 33 Amount reclassified from accumulated other comprehensive income 129 Net current period other comprehensive gain (loss) 162 Balance at September 30, 2022 $ 32 Balance at December 31, 2021 $ (148 ) Other comprehensive gain ( ) (582 ) Amount reclassified from accumulated other comprehensive income 762 Net current period other comprehensive gain (loss) 180 Balance at September 30, 2022 $ 32 |
Inventory (Tables)
Inventory (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Inventory Disclosure [Abstract] | |
Summary of current inventory | Inventory consists of the following (in thousands): September 30, 2022 December 31, 2021 Raw materials $ — $ 1,769 Work-in-process 6,252 5,172 Finished goods 880 929 Total inventory $ 7,132 $ 7,870 |
Prepaid Expenses and Other Cu_2
Prepaid Expenses and Other Current Assets (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Text Block [Abstract] | |
Prepaid expenses and other current assets | Prepaid expenses and other current assets consist of the following (in thousands): September 30, 2022 December 31, 2021 Prepaid manufacturing costs $ 970 $ 307 Prepaid tax 74 564 Prepaid insurance 289 1,213 Prepaid subscriptions fees 470 909 Prepaid research fees 404 452 Prepaid commercialization expenses 573 195 Due from collaborative and licensing arrangements 324 105 Prepaid conference and travel expenses 206 279 Other 466 327 Total prepaid expenses and other current assets $ 3,776 $ 4,351 |
Operating Lease (Tables)
Operating Lease (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Disclosure of Operating Leases [Abstract] | |
Lease, Cost | The components of lease expense were as follows (in thousands): For the Three Months Ended For the Nine Months Ended 2022 2021 2022 2021 Operating lease cost $ 108 $ 108 $ 324 $ 272 |
Schedule of Supplemental Cash Flow Information Related To Lease | Supplemental cash flow information related to lease was as follows (in thousands): For the Nine Months Ended 2022 2021 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows $ 368 $ 29 Right-of-use Operating lease $ 67 $ 58 |
Schedule of Supplemental Balance Sheet related To Lease | Supplemental balance sheet information related to lease was as follows (in thousands): September 30, 2022 December 31, 2021 Operating lease right-of-use $ 2,833 $ 3,017 Other current liabilities $ 330 $ 308 Operating lease liabilities, net of current portion 3,643 3,894 Total operating lease liabilities $ 3,973 $ 4,202 |
Lessee, Operating Lease, Liability, Maturity | Remaining payments of lease liabilities as of September 30, 2022 were as follows (in thousands): 2022 (remaining three months) $ 123 2023 506 2024 522 2025 537 2026 553 Thereafter 2,598 Total lease payments 4,839 Less: imputed interest (866 ) Total $ 3,973 Rent expense was approximately $0.1 million and $0.3 million for both the three and nine months ended September 30, 2022 and 2021, respectively. |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | Property and equipment, net consists of the following (in thousands): September 30, 2022 December 31, 2021 Computer equipment $ 51 $ 51 Furniture and equipment 223 203 Leasehold improvements 980 980 Less: Accumulated depreciation (372 ) (275 ) Total property and equipment, net $ 882 $ 959 |
License and Acquired Intangib_2
License and Acquired Intangibles, Net. (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Finite-Lived Intangible Assets, Net [Abstract] | |
Schedule of Finite-Lived Intangible Assets | The following table presents the Company’s intangible assets at September 30, 2022 (in thousands): Gross Carrying Value Accumulated Amortization Net Carrying Value Intangible assets: License and acquired intangibles for Ruzurgi® $ 33,569 $ 518 $ 33,051 Total $ 33,569 $ 518 $ 33,051 |
Accrued Expenses and Other Li_2
Accrued Expenses and Other Liabilities (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Liabilities | Accrued expenses and other liabilities consist of the following (in thousands): September 30, 2022 December 31, 2021 Accrued preclinical and clinical trial expenses $ 460 $ 659 Accrued professional fees 2,503 2,391 Accrued compensation and benefits 3,412 4,035 Accrued license fees 15,528 12,819 Accrued purchases — 2,045 Operating lease liability 330 308 Accrued variable consideration 2,988 1,716 Accrued income tax 3,744 79 Due to licensor 12,950 — Other 237 243 Current accrued expenses and other liabilities 42,152 24,295 Lease liability – non-current 3,643 3,894 Due to licensor – non-current 14,749 — Non-current 18,392 3,894 Total accrued expenses and other liabilities $ 60,544 $ 28,189 |
Agreements (Tables)
Agreements (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Text Block [Abstract] | |
Schedule Of Total Purchase Price Was Allocated To The Acquired Assets Based On Their Relative Fair Values | The total purchase price was allocated to the acquired assets based on their relative fair values, as follows (in thousands): License and acquired intangibles $ 33,569 Acquired research and development expensed from asset acquisition 4,130 Total purchase price $ 37,699 |
Stock Compensation (Tables)
Stock Compensation (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Share-based Payment Arrangement [Abstract] | |
Stock-Based Compensation Expense | For the three and nine months ended September 30, 2022 and 2021, the Company recorded stock-based compensation expense as follows (in thousands): Three months ended Nine months ended 2022 2021 2022 2021 Research and development $ 450 $ 428 $ 1,307 $ 1,193 Selling, general and administrative 1,605 1,084 4,674 3,408 Total stock-based compensation $ 2,055 $ 1,512 $ 5,981 $ 4,601 |
Basis of Presentation and Sig_4
Basis of Presentation and Significant Accounting Policies - Additional Information (Detail) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Dec. 31, 2021 | |
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Maximum maturity period of cash and cash equivalent | three months | ||||
Non current investments | $ 0 | $ 0 | $ 0 | ||
Minimum amortization period of compensation cost on straight line basis | 1 year | ||||
Maximum Amortization Period Of Compensation Cost On Straight Line Basis | 3 years | ||||
Potential equivalent common stock excluded | 12 | 2,600 | 1,700 | 5,300 | |
Impairment of Intangible Assets, Finite-Lived | $ 0 | $ 0 | |||
Selling, General and Administrative Expenses [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Advertising Expense | $ 1,000 | $ 600 | $ 2,500 | $ 1,600 | |
Minimum [Member] | Furniture and Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 5 years | ||||
Minimum [Member] | Computer Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 3 years | ||||
Minimum [Member] | Leasehold Improvements [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 5 years | ||||
Maximum [Member] | Furniture and Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 7 years | ||||
Maximum [Member] | Computer Equipment [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 5 years | ||||
Maximum [Member] | Leasehold Improvements [Member] | |||||
Summary Of Basis Of Presentation And Significant Accounting Policies [Line Items] | |||||
Useful life of assets | 10 years |
Basis of Presentation and Sig_5
Basis of Presentation and Significant Accounting Policies - Fair Value Measurement Specific to Assets or Liability (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments | $ 0 | $ 19,821 |
Short-term bond funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments | 19,821 | |
U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 227,349 | 140,995 |
Money market fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 10,336 | 10,990 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Short-term bond funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments | 19,821 | |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 227,349 | 140,995 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) [Member] | Money market fund [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 10,336 | 10,990 |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Treasuries [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 0 |
Basis of Presentation and Sig_6
Basis of Presentation and Significant Accounting Policies - Schedule Of Reconcile Basic And Dilutive Weighted Average Common Shares (Details) - shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Weighted Average Number of Shares Outstanding Reconciliation [Abstract] | ||||
Basic weighted average common shares outstanding | 103,318,572 | 103,196,550 | 102,967,280 | 103,470,762 |
Effect of dilutive securities | 8,667,453 | 4,646,646 | 7,384,934 | 4,005,413 |
Dilutive weighted average common shares outstanding | 111,986,025 | 107,843,196 | 110,352,214 | 107,476,175 |
Investments - Additional Inform
Investments - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended |
Sep. 30, 2022 | Sep. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | ||
Realized losses from sale of available-for-sale securities | $ 129 | $ 762 |
Investment - Summary of Availab
Investment - Summary of Available-for-Sale Investments by Security type (Detail) - USD ($) $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2022 | Dec. 31, 2021 | |
Net Investment Income [Line Items] | ||
Estimated Fair Value | $ 227,349 | $ 160,816 |
Gross Unrealized Gains | 45 | 2 |
Gross Unrealized Losses | 0 | (196) |
Amortized cost | 227,304 | 161,010 |
U.S. Treasuries—Cash equivalents [Member] | ||
Net Investment Income [Line Items] | ||
Estimated Fair Value | 227,349 | 140,995 |
Gross Unrealized Gains | 45 | 2 |
Gross Unrealized Losses | 0 | |
Amortized cost | $ 227,304 | 140,993 |
Short Term Bond Funds [Member] | ||
Net Investment Income [Line Items] | ||
Estimated Fair Value | 19,821 | |
Gross Unrealized Losses | (196) | |
Amortized cost | $ 20,017 |
Investment - Estimated Fair Val
Investment - Estimated Fair Values of Available for Sale Securities (Detail) $ in Thousands | Sep. 30, 2022 USD ($) |
Investments, Debt and Equity Securities [Abstract] | |
Due in one year or less | $ 227,349 |
Accumulated Other Comprehensi_3
Accumulated Other Comprehensive Income (Loss) - Summary of Reclassifications out of Accumulated Other Comprehensive Income (loss) (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Accumulated Other Comprehensive Income (Loss) [Line Items] | ||||
Beginning balance | $ (130) | $ (148) | ||
Other comprehensive gain (loss) before reclassifications | 33 | (582) | ||
Amount reclassified from accumulated other comprehensive income | 129 | 762 | ||
Net current period other comprehensive gain (loss) | 162 | $ (26) | 180 | $ (94) |
Ending balance | $ 32 | $ 32 |
Inventory - Summary of current
Inventory - Summary of current inventory (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 0 | $ 1,769 |
Work-in-process | 6,252 | 5,172 |
Finished goods | 880 | 929 |
Total inventory, net | $ 7,132 | $ 7,870 |
Prepaid Expenses and Other Cu_3
Prepaid Expenses and Other Current Assets - Prepaid Expenses and Other Current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid manufacturing costs | $ 970 | $ 307 |
Prepaid tax | 74 | 564 |
Prepaid insurance | 289 | 1,213 |
Prepaid subscriptions fees | 470 | 909 |
Prepaid research fees | 404 | 452 |
Prepaid commercialization expenses | 573 | 195 |
Due from collaborative arrangements | 324 | 105 |
Prepaid conference and travel expenses | 206 | 279 |
Other | 466 | 327 |
Total prepaid expenses and other current assets | $ 3,776 | $ 4,351 |
Operating Lease - Operating Lea
Operating Lease - Operating Lease (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Disclosure of Operating Leases [Abstract] | ||||
Operating lease cost | $ 108 | $ 108 | $ 324 | $ 272 |
Operating Lease - Schedule of S
Operating Lease - Schedule of Supplemental Cash Flow Information Related To Lease (Detail) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Cash paid for amounts included in the measurement of lease liabilities: | ||
Operating cash flows | $ 368 | $ 29 |
Right-of-use assets obtained in exchange for lease obligations: | ||
Operating leases | $ 67 | $ 58 |
Operating Lease - Schedule of_2
Operating Lease - Schedule of Supplemental Balance Sheet related To Lease (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Disclosure of Operating Leases [Line Items] | ||
Operating lease right-of-use assets | $ 2,833 | $ 3,017 |
Other current liabilities | 330 | 308 |
Operating lease liabilities, net of current portion | 3,643 | 3,894 |
Total operating lease liabilities | $ 3,973 | $ 4,202 |
Operating Lease - Lessee, Opera
Operating Lease - Lessee, Operating Lease, Liability, Maturity (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Disclosure of Operating Leases [Line Items] | ||
2022 (remaining three months) | $ 123 | |
2023 | 506 | |
2024 | 522 | |
2025 | 537 | |
2026 | 553 | |
Thereafter | 2,598 | |
Total lease payments | 4,839 | |
Less imputed interest | (866) | |
Total | $ 3,973 | $ 4,202 |
Operating Lease - Additional I
Operating Lease - Additional Information (Detail) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) ft² | Sep. 30, 2021 USD ($) | Dec. 31, 2021 | |
Disclosure of Operating Leases [Line Items] | |||||
Finance Lease Obligations | $ | $ 0 | $ 0 | |||
Lessee, Operating Lease, Option to Extend | 5 years | ||||
Rent expense | $ | $ 108 | $ 108 | $ 324 | $ 272 | |
Before agreement of company leased spaces | ft² | 7,800 | ||||
After agreement of company leased spaces | ft² | 10,700 | ||||
Weighted average remaining lease term | 8 years 7 months 6 days | 8 years 7 months 6 days | 9 years 3 months 18 days | ||
Weighted average discount rate | 4.51% | 4.51% | 4.51% | ||
Maximum [Member] | |||||
Disclosure of Operating Leases [Line Items] | |||||
Lessee, Operating Lease, Option to Terminate | 7.6 years | ||||
Minimum [Member] | |||||
Disclosure of Operating Leases [Line Items] | |||||
Lessee, Operating Lease, Option to Terminate | 6 |
Property and Equipment, Net - P
Property and Equipment, Net - Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Property, Plant and Equipment [Line Items] | ||
Less: Accumulated depreciation | $ (372) | $ (275) |
Total property and equipment, net | 882 | 959 |
Computer equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 51 | 51 |
Furniture and equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 223 | 203 |
Leasehold improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 980 | $ 980 |
License and Acquired Intangib_3
License and Acquired Intangibles, Net - Schedule of Finite Lived Intangible Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Finite-Lived Intangible Assets [Line Items] | ||
Gross Carrying Value | $ 33,569 | |
Accumulated Amortization | 518 | |
Net Carrying Value | 33,051 | $ 0 |
License and Acquired Intangibles for Ruzurgi [Member] | ||
Finite-Lived Intangible Assets [Line Items] | ||
Gross Carrying Value | 33,569 | |
Accumulated Amortization | 518 | |
Net Carrying Value | $ 33,051 |
License and Acquired Intangib_4
License and Acquired Intangibles, Net - Additional Information (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2022 USD ($) | |
Finite-Lived Intangible Assets, Net [Abstract] | |
Finite-lived intangible asset, useful life | 14 years 6 months |
Finite lived intangible asset expected amortization per year | $ 2,300 |
Amortization of intangible assets | $ 518 |
Accrued Expenses and Other Li_3
Accrued Expenses and Other Liabilities - Schedule of Accrued Expenses and Other Liabilities (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Payables and Accruals [Abstract] | ||
Accrued preclinical and clinical trial expenses | $ 460 | $ 659 |
Accrued professional fees | 2,503 | 2,391 |
Accrued compensation and benefits | 3,412 | 4,035 |
Accrued license fees | 15,528 | 12,819 |
Accrued purchases | 0 | 2,045 |
Operating lease liability | 330 | 308 |
Accrued variable consideration | 2,988 | 1,716 |
Accrued income tax | 3,744 | 79 |
Due to licensor | 12,950 | 0 |
Other | 237 | 243 |
Current accrued expenses and other liabilities | 42,152 | 24,295 |
Lease liability – non-current | 3,643 | 3,894 |
Due to licensor – non-current | 14,749 | 0 |
Non-current accrued expenses and other liabilities | 18,392 | 3,894 |
Total accrued expenses and other liabilities | $ 60,544 | $ 28,189 |
Collaborative and Licensing A_2
Collaborative and Licensing Arrangements - Additional Information (Detail) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Collaborative Arrangements [Line Items] | ||||
Period of collaboration agreement | 10 years | |||
Total research and development expense | $ 8,310,000 | $ 4,487,000 | $ 15,696,000 | $ 11,944,000 |
Milestone payments income | 0 | 0 | ||
KYE Pharmaceuticals [Member] | Selling, General and Administrative Expenses [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Expenses incurred in connection with collaboration agreement | 0 | 0 | 0 | 0 |
Dydo Pharma Inc Member [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Milestone payments income | 0 | |||
Dydo Pharma Inc Member [Member] | Non Refundable Upfront License Fees [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Revenues | 0 | 2,700,000 | ||
Collaborative Arrangement [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Total research and development expense | 0 | 0 | ||
Milestone payments | 2,000,000 | |||
Revenues | 0 | $ 0 | 0 | $ 0 |
Collaborative Arrangement [Member] | product revenue,net | ||||
Collaborative Arrangements [Line Items] | ||||
Revenues | 0 | 500,000 | ||
Collaborative Arrangement [Member] | Research and Development Expense [Member] | ||||
Collaborative Arrangements [Line Items] | ||||
Total research and development expense | $ 0 | $ 45,000 |
Agreements - Additional Informa
Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||||||
Jul. 11, 2026 | Jul. 11, 2025 | Jul. 11, 2024 | Jul. 11, 2023 | Jul. 11, 2022 | Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
License Agreement [Line Items] | |||||||||
Percentage of royalty on net sales | 3.50% | ||||||||
Research and development expenses | $ 8,310 | $ 4,487 | $ 15,696 | $ 11,944 | |||||
License Agreement with BioMarin [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Date on which strategic collaboration is entered into | Oct. 26, 2012 | ||||||||
Royalty agreement period | 7 years | ||||||||
Net sales royalty threshold | $ 100,000 | ||||||||
License Agreement with BioMarin [Member] | Minimum [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Percentage of royalty on net sales | 7% | ||||||||
License Agreement with BioMarin [Member] | Maximum [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Percentage of royalty on net sales | 10% | ||||||||
License Agreement For Ruzurgi [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Date on which strategic collaboration is entered into | Jul. 11, 2022 | ||||||||
Minimum royalty | $ 5 | $ 3 | $ 3 | $ 3 | |||||
Research and development expenses | $ 4,100 | $ 4,100 | |||||||
Up front payment | $ 10,000 | ||||||||
Asset acquisition purchase price consideration | $ 10,000 | $ 10,000 | |||||||
License Agreement For Ruzurgi [Member] | Minimum [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Percentage of minimum royalty payable to net sales | 1.25% | ||||||||
License Agreement For Ruzurgi [Member] | Maximum [Member] | |||||||||
License Agreement [Line Items] | |||||||||
Percentage of maximum royalty payable to net sales | 2.50% |
Agreements - Schedule Of Total
Agreements - Schedule Of Total Purchase Price Was Allocated To The Acquired Assets Based On Their Relative Fair Values (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2022 USD ($) | |
Schedule Of Total Purchase Price Was Allocated To The Acquired Assets Based On Their Relative Fair Values [Line Items] | |
License and acquired intangibles | $ 33,569 |
Acquired research and development inventory expensed from asset acquisition | 4,130 |
Total purchase price | $ 37,699 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Dec. 31, 2021 | |
Income Tax Disclosure [Line Items] | |||
Effective Income Tax Rate Reconciliation, Percent | 19.70% | 24.30% | |
Unrecognized Tax Benefits that Would Impact Effective Tax Rate | $ 0 | $ 0 | |
Effective Income Tax Rate Reconciliation, at Federal Statutory Income Tax Rate, Percent | 21% |
Stockholders' Equity (Preferred
Stockholders' Equity (Preferred Stock and Common Stock) - Additional Information (Detail) | Sep. 30, 2022 Vote $ / shares shares | Dec. 31, 2021 $ / shares shares |
Stockholders Equity [Line Items] | ||
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, par value | $ / shares | $ 0.001 | $ 0.001 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, par value | $ / shares | $ 0.001 | $ 0.001 |
Common stock, shares issued | 104,064,683 | 102,992,913 |
Common stock, shares outstanding | 104,064,683 | 102,992,913 |
Number of votes entitled for each share of common stock | Vote | 1 |
Stockholders' Equity ( Share Re
Stockholders' Equity ( Share Repurchases ) - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Mar. 31, 2021 | |
Stockholders Equity [Line Items] | ||||
Aggregate purchase price | $ 5,100 | $ 6,907 | $ 9,014 | |
Average price per share | $ 5.32 | $ 6.91 | $ 5.15 | |
Common Stock [Member] | ||||
Stockholders Equity [Line Items] | ||||
Aggregate share repurchased | 950,000 | 1,000,000 | 1,750,000 | |
Share Purchase Program [Member] | Common Stock [Member] | ||||
Stockholders Equity [Line Items] | ||||
Share repurchase program, authorised | $ 40,000 |
Stockholders' Equity (2020 Shel
Stockholders' Equity (2020 Shelf Registration Statement) - Additional Information (Detail) $ in Millions | Jul. 23, 2020 USD ($) |
2020 Shelf Registration Statement [Member] | |
Stockholders' Equity [Line Items] | |
Maximum dollar amount of common stock to be issued under shelf registration statement | $ 200 |
Stock Compensation - Stock-Base
Stock Compensation - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 2,055 | $ 1,512 | $ 5,981 | $ 4,601 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | 450 | 428 | 1,307 | 1,193 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation | $ 1,605 | $ 1,084 | $ 4,674 | $ 3,408 |
Stock Compensation - Additional
Stock Compensation - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Proceeds from exercise of stock options | $ 6,368 | $ 3,414 | ||
Number of options, Outstanding | 12,516,584 | 12,516,584 | ||
Restricted Stock Units (RSUs) [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 3,000 | $ 3,000 | ||
Expected remaining weighted average vesting period | 2 years 3 months 18 days | |||
Non-cash stock-based compensation expense | $ 400 | $ 100 | $ 1,100 | $ 400 |
Common stock granted | 474,500 | |||
Options to Purchase Common Stock [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of stock options exercised | 1,355,335 | 941,164 | 2,064,700 | 1,114,494 |
Proceeds from exercise of stock options | $ 4,000 | $ 3,000 | $ 6,400 | $ 3,400 |
Stock option granted, contractual term | 7 years | 7 years | 7 years | 7 years |
Common stock unit granted | 12,000 | 400,000 | 455,000 | 1,030,000 |
Unrecognized compensation expense related to non-vested stock compensation awards granted under the Plan | $ 8,300 | $ 8,300 | ||
Expected remaining weighted average vesting period | 2 years | |||
Non-cash stock-based compensation expense | $ 1,600 | $ 1,400 | $ 4,800 | $ 4,200 |
Stock options to purchase shares of common stock | 8,124,977 | 8,124,977 |