Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2023 | Aug. 07, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2023 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | AMLX | |
Entity Registrant Name | Amylyx Pharmaceuticals, Inc. | |
Entity Central Index Key | 0001658551 | |
Current Fiscal Year End Date | --12-31 | |
Entity File Number | 001-41199 | |
Entity Tax Identification Number | 46-4600503 | |
Entity Address, Address Line One | 43 Thorndike St. | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02141 | |
City Area Code | 617 | |
Local Phone Number | 682-0917 | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 67,377,743 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 247,760 | $ 62,526 |
Short-term investments | 109,516 | 284,419 |
Accounts receivable, net | 33,473 | 15,306 |
Total inventories | 24,858 | 9,769 |
Prepaid expenses and other current assets | 11,787 | 10,113 |
Total current assets | 427,394 | 382,133 |
Property and equipment, net | 2,637 | 2,611 |
Restricted cash equivalents | 719 | 719 |
Operating lease right-of-use assets | 4,643 | 5,524 |
Long-term inventories | 17,729 | |
Other assets | 466 | 466 |
Total assets | 453,588 | 391,453 |
Current liabilities: | ||
Accounts payable | 24,695 | 6,257 |
Accrued expenses | 37,241 | 38,312 |
Operating lease liabilities, current portion | 2,146 | 2,040 |
Total current liabilities | 64,082 | 46,609 |
Operating lease liabilities, net of current portion | 3,141 | 4,237 |
Total liabilities | 67,223 | 50,846 |
Commitments and contingencies (Note 13) | ||
Stockholders' equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized no shares issued or outstanding | ||
Common stock, $0.0001 par value; 300,000,000 shares authorized; 67,354,417 and 66,512,011 shares issued and outstanding as of June 30, 2023 and December 31, 2022, respectively | 7 | 7 |
Additional paid-in capital | 717,003 | 694,906 |
Accumulated deficit | (330,573) | (354,220) |
Accumulated other comprehensive loss | (72) | (86) |
Total stockholders' equity | 386,365 | 340,607 |
Total liabilities, redeemable convertable preferred stock and stockholders' equity | $ 453,588 | $ 391,453 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Jun. 30, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock par value | $ 0.0001 | $ 0.0001 |
Preferred stock shares authorized | 10,000,000 | 10,000,000 |
Preferred stock shares issued | 0 | 0 |
Preferred stock shares outstanding | 0 | 0 |
Common stock, stated par value per share | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares, issued | 67,354,417 | 66,512,011 |
Common stock, shares, outstanding | 67,354,417 | 66,512,011 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Income Statement [Abstract] | ||||
Product revenue, net | $ 98,216 | $ 169,644 | ||
Revenue from Contract with Customer, Product and Service [Extensible Enumeration] | us-gaap:ProductMember | us-gaap:ProductMember | ||
Operating expenses: | ||||
Cost of sales | $ 5,580 | $ 10,863 | ||
Research and development | 29,044 | $ 24,259 | 53,236 | $ 45,723 |
Selling, general and administrative | 43,391 | 29,994 | 87,397 | 56,344 |
Total operating expenses | 78,015 | 54,253 | 151,496 | 102,067 |
Income (loss) from operations | 20,201 | (54,253) | 18,148 | (102,067) |
Other income, net: | ||||
Interest income | 3,887 | 402 | 7,605 | 533 |
Other expense, net | (81) | (42) | (343) | (61) |
Total other income, net | 3,806 | 360 | 7,262 | 472 |
Income (loss) before income taxes | 24,007 | (53,893) | 25,410 | (101,595) |
Provision for income taxes | 1,933 | 174 | 1,763 | 320 |
Net income (loss) | $ 22,074 | $ (54,067) | $ 23,647 | $ (101,915) |
Net income (loss) per share attributable to common stockholders -basic | $ 0.33 | $ (0.93) | $ 0.35 | $ (1.85) |
Net income (loss) per share attributable to common stockholders -diluted | $ 0.31 | $ (0.93) | $ 0.34 | $ (1.85) |
Weighted-average shares used in computing net income (loss) per share attributable to common stockholders-basic | 67,233,617 | 58,275,903 | 66,976,871 | 54,958,537 |
Weighted-average shares used in computing net income (loss) per share attributable to common stockholders-diluted | 70,132,040 | 58,275,903 | 70,471,821 | 54,958,537 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Statement of Comprehensive Income [Abstract] | ||||
Net income (loss) | $ 22,074 | $ (54,067) | $ 23,647 | $ (101,915) |
Other comprehensive income (loss): | ||||
Foreign currency translation gain (loss) | (18) | 58 | 60 | (10) |
Net unrealized loss on investments held | (79) | (103) | (46) | (195) |
Other comprehensive income (loss) | (97) | (45) | 14 | (205) |
Comprehensive income (loss) | $ 21,977 | $ (54,112) | $ 23,661 | $ (102,120) |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF REDEEMABLE CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Series A Redeemable Convertible Preferred Share | Series B Redeemable Convertible Preferred Shares | Series C-1 Redeemable Convertible Preferred Shares | Series C-2 Redeemable Convertible Preferred Shares | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Beginning Balance, Shares at Dec. 31, 2021 | 6,289,609 | 14,496,835 | 13,150,430 | 3,170,585 | |||||
Beginning Balance at Dec. 31, 2021 | $ 7,675 | $ 64,387 | $ 134,791 | $ 32,498 | |||||
Beginning Balance, Shares at Dec. 31, 2021 | 7,020,487 | ||||||||
Beginning Balance at Dec. 31, 2021 | $ (151,168) | $ 1 | $ 4,667 | $ 9 | $ (155,845) | ||||
Conversion of preferred stock into common stock upon initial public offering, Shares | 39,474,330 | ||||||||
Conversion of preferred stock into common stock upon initial public offering | 239,351 | $ 4 | 239,347 | ||||||
Conversion of preferred stock into common stock upon initial public offering, Shares | (6,289,609) | (14,496,835) | (13,150,430) | (3,170,585) | |||||
Conversion of preferred stock into common stock upon initial public offering | $ (7,675) | $ (64,387) | $ (134,791) | $ (32,498) | |||||
Issuance of common stock upon initial public offering, net of issuance costs, Shares | 11,369,369 | ||||||||
Issuance of common stock upon initial public offering, net of issuance costs | 196,379 | $ 1 | 196,378 | ||||||
Stock-based compensation expense | 4,392 | 4,392 | |||||||
Other comprehensive income (loss) | (160) | (160) | |||||||
Net income (loss) | (47,848) | (47,848) | |||||||
Ending Balance, Shares at Mar. 31, 2022 | 57,864,186 | ||||||||
Ending Balance at Mar. 31, 2022 | 240,946 | $ 6 | 444,784 | (151) | (203,693) | ||||
Beginning Balance, Shares at Dec. 31, 2021 | 6,289,609 | 14,496,835 | 13,150,430 | 3,170,585 | |||||
Beginning Balance at Dec. 31, 2021 | $ 7,675 | $ 64,387 | $ 134,791 | $ 32,498 | |||||
Beginning Balance, Shares at Dec. 31, 2021 | 7,020,487 | ||||||||
Beginning Balance at Dec. 31, 2021 | (151,168) | $ 1 | 4,667 | 9 | (155,845) | ||||
Net income (loss) | (101,915) | ||||||||
Ending Balance, Shares at Jun. 30, 2022 | 58,533,226 | ||||||||
Ending Balance at Jun. 30, 2022 | 192,789 | $ 6 | 450,739 | (196) | (257,760) | ||||
Beginning Balance, Shares at Mar. 31, 2022 | 57,864,186 | ||||||||
Beginning Balance at Mar. 31, 2022 | 240,946 | $ 6 | 444,784 | (151) | (203,693) | ||||
Issuance of common stock upon exercise of stock options, Shares | 669,040 | ||||||||
Issuance of common stock upon exercise of stock options | 248 | 248 | |||||||
Stock-based compensation expense | 5,707 | 5,707 | |||||||
Other comprehensive income (loss) | (45) | (45) | |||||||
Net income (loss) | (54,067) | (54,067) | |||||||
Ending Balance, Shares at Jun. 30, 2022 | 58,533,226 | ||||||||
Ending Balance at Jun. 30, 2022 | $ 192,789 | $ 6 | 450,739 | (196) | (257,760) | ||||
Beginning Balance, Shares at Dec. 31, 2022 | 66,512,011 | 66,512,011 | |||||||
Beginning Balance at Dec. 31, 2022 | $ 340,607 | $ 7 | 694,906 | (86) | (354,220) | ||||
Issuance of common stock upon exercise of stock options, Shares | 451,298 | ||||||||
Issuance of common stock upon exercise of stock options | 2,777 | 2,777 | |||||||
Issuance of common stock upon vesting of RSUs | 132,294 | ||||||||
Stock-based compensation expense | 7,580 | 7,580 | |||||||
Other comprehensive income (loss) | 111 | 111 | |||||||
Net income (loss) | 1,573 | 1,573 | |||||||
Ending Balance, Shares at Mar. 31, 2023 | 67,095,603 | ||||||||
Ending Balance at Mar. 31, 2023 | $ 352,648 | $ 7 | 705,263 | 25 | (352,647) | ||||
Beginning Balance, Shares at Dec. 31, 2022 | 66,512,011 | 66,512,011 | |||||||
Beginning Balance at Dec. 31, 2022 | $ 340,607 | $ 7 | 694,906 | (86) | (354,220) | ||||
Issuance of common stock upon exercise of stock options, Shares | 677,436 | ||||||||
Net income (loss) | $ 23,647 | ||||||||
Ending Balance, Shares at Jun. 30, 2023 | 67,354,417 | 67,354,417 | |||||||
Ending Balance at Jun. 30, 2023 | $ 386,365 | $ 7 | 717,003 | (72) | (330,573) | ||||
Beginning Balance, Shares at Mar. 31, 2023 | 67,095,603 | ||||||||
Beginning Balance at Mar. 31, 2023 | 352,648 | $ 7 | 705,263 | 25 | (352,647) | ||||
Issuance of common stock upon exercise of stock options, Shares | 226,138 | ||||||||
Issuance of common stock upon exercise of stock options | 1,687 | 1,687 | |||||||
Issuance of common stock upon vesting of RSUs | 32,676 | ||||||||
Stock-based compensation expense | 10,053 | 10,053 | |||||||
Other comprehensive income (loss) | (97) | (97) | |||||||
Net income (loss) | $ 22,074 | 22,074 | |||||||
Ending Balance, Shares at Jun. 30, 2023 | 67,354,417 | 67,354,417 | |||||||
Ending Balance at Jun. 30, 2023 | $ 386,365 | $ 7 | $ 717,003 | $ (72) | $ (330,573) |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF REDEEMABLE CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY (Parenthetical) $ in Thousands | 3 Months Ended |
Mar. 31, 2022 USD ($) | |
Common Stock | Initial Public Offering | |
Issuance costs | $ 19,639 |
CONDENSED CONSOLIDATED STATEM_5
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2023 | Jun. 30, 2022 | |
Cash flows provided by (used in) operating activities: | ||
Net income (loss) | $ 23,647 | $ (101,915) |
Adjustments to reconcile net income (loss) to net cash used in operating activities: | ||
Stock-based compensation expense | 17,451 | 10,099 |
Depreciation expense | 485 | 140 |
Accretion of investment discounts, net | (4,886) | (107) |
Changes in operating assets and liabilities: | ||
Accounts receivable, net | (18,167) | |
Inventories | (32,636) | |
Interest receivable | (356) | (13) |
Prepaid expenses and other current assets | (1,318) | (4,053) |
Operating lease right-of-use assets | 881 | 790 |
Other assets | (456) | |
Accounts payable | 18,455 | 3,755 |
Accrued expenses | 971 | 4,784 |
Operating lease liabilities | (990) | (120) |
Net cash provided by (used in) operating activities | 3,537 | (87,096) |
Cash flows provided by (used in) investing activities: | ||
Purchases of property and equipment | (528) | (1,447) |
Purchases of investments | (9,756) | (154,313) |
Proceeds from maturities of short-term investments | 189,500 | 60,911 |
Net cash provided by (used in) investing activities | 179,216 | (94,849) |
Cash flows provided by financing activities: | ||
Proceeds from initial public offering | 200,897 | |
Initial public offering costs paid | (1,518) | |
Follow-on offering costs paid | (136) | |
Proceeds from exercise of stock options | 5,663 | 248 |
Withholding taxes paid on stock-based awards | (3,103) | |
Net cash provided by financing activities | 2,424 | 199,627 |
Effect of exchange rate changes on cash, cash equivalents and restricted cash equivalents | 57 | (203) |
Net increase in cash, cash equivalents and restricted cash equivalents | 185,234 | 17,479 |
Cash, cash equivalents and restricted cash equivalents, beginning of period | 63,245 | 50,380 |
Cash, cash equivalents and restricted cash equivalents, end of period | 248,479 | 67,859 |
Supplemental disclosure of cash flow information: | ||
Unrealized loss on short-term investments | 46 | 195 |
Taxes withheld on stock-based awards included in accrued expenses | 67 | |
Purchases of property and equipment included in accounts payable | 81 | 76 |
Right-of-use assets and liabilities upon ASC 842 adoption | 2,201 | |
Right-of-use assets obtained in exchange for lease liabilities | 4,958 | |
Movement of deferred offering costs to equity | 4,518 | |
Initial public offering costs included in accounts payable and accrued expenses | 526 | |
Conversion of preferred stock to common stock upon initial public offering | $ 239,351 | |
Income taxes paid | $ 2,562 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2022 | Mar. 31, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Pay vs Performance Disclosure | ||||||
Net Income (Loss) | $ 22,074 | $ 1,573 | $ (54,067) | $ (47,848) | $ 23,647 | $ (101,915) |
Insider Trading Arrangements
Insider Trading Arrangements | 6 Months Ended |
Jun. 30, 2023 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | During the three months ended June 30, 2023, the following officers of the Company (as defined in Rule 16a-1(f)) adopted the following trading plans for the sale of our common stock pursuant to the terms of the applicable plan; such plans are intended to satisfy the affirmative defense conditions of Rule 10b5–1(c)(1) of the Exchange Act: • Joshua Cohen , our Co-Chief Executive Officer and a member of our board of directors, adopted a new Rule 10b5-1 trading plan on May 22, 2023 , which is scheduled to expire on November 30, 2023 . The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 75,000 ; • Justin Klee , our Co-Chief Executive Officer and a member of our board of directors, adopted a new Rule 10b5-1 trading plan on May 22, 2023 , which is scheduled to expire on November 30, 2023 . The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 75,000 ; • James Frates , our Chief Financial Officer , adopted a new Rule 10b5-1 trading plan on May 22, 2023 , which is scheduled to expire on December 14, 2023 . The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 60,000 ; and • Gina M. Mazzariello , our Chief Legal Officer and General Counsel, adopted a new Rule 10b5-1 trading plan on May 16, 2023 , which is scheduled to expire on March 8, 2024 . The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 111,119 , which includes shares that may be withheld or sold to cover withholding taxes at the time of vesting. No other director or officer has adopted or terminated any non-Rule 10b5-1 trading arrangements during the quarter ended June 30, 2023. |
Non-Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
Joshua Cohen [Member] | |
Trading Arrangements, by Individual | |
Name | Joshua Cohen |
Title | Co-Chief Executive Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 22, 2023 |
Termination Date | November 30, 2023 |
Aggregate Available | 75,000 |
Justin Klee [Member] | |
Trading Arrangements, by Individual | |
Name | Justin Klee |
Title | Co-Chief Executive Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 22, 2023 |
Termination Date | November 30, 2023 |
Aggregate Available | 75,000 |
James Frates [Member] | |
Trading Arrangements, by Individual | |
Name | James Frates |
Title | Chief Financial Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 22, 2023 |
Termination Date | December 14, 2023 |
Aggregate Available | 60,000 |
Gina M. Mazzariello [Member] | |
Trading Arrangements, by Individual | |
Name | Gina M. Mazzariello |
Title | Chief Legal Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | May 16, 2023 |
Termination Date | March 8, 2024 |
Aggregate Available | 111,119 |
Nature of the Business
Nature of the Business | 6 Months Ended |
Jun. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business | 1. Nature of the Business Amylyx Pharmaceuticals, Inc., together with its wholly owned subsidiaries, known as Amylyx or the Company, is a commercial-stage biotechnology company with a mission to one day end the suffering caused by neurodegenerative diseases. The Company is pursuing amyotrophic lateral sclerosis, or ALS, as its first indication and is focused on the development and potential commercialization of AMX0035 for ALS globally. Amylyx’ first product, RELYVRIO ® (sodium phenylbutyrate and taurursodiol, also known as ursodoxicoltaurine), also known as AMX0035, is approved by the U.S. Food and Drug Administration, or the FDA, for the treatment of ALS in adults in the U.S. AMX0035 is also approved with conditions by Health Canada and marketed as ALBRIOZA for the treatment of ALS in Canada. In June 2023, the Company announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, adopted a negative opinion on the application for conditional marketing authorisation of AMX0035, under the trade name ALBRIOZA ® , for the treatment of adults with ALS in the European Union, or EU. Following the Company's request for a formal re-examination in July 2023, CHMP is re-examining its initial opinion on the current application for conditional marketing authorisation. The re-examination procedure is an approximately four-month process, which includes the appointment of a different rapporteur and co-rapporteur from the initial evaluation. At the end of the re-examination, the EMA will review its initial opinion before issuing a final recommendation, which is expected in the fall of 2023. The Company is also developing AMX0035 for other neurodegenerative diseases by leveraging its scientific expertise and relationships in the neurodegenerative space. The Company dosed the first participant in the HELIOS trial, a Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome, in April 2023 and plans to initiate the ORION trial, a global, pivotal Phase 3 trial of AMX0035 for the treatment of progressive supranuclear palsy, or PSP by the end of 2023. The Company is also advancing additional drug candidates for neurodegenerative diseases including AMX0114, an antisense oligonucleotide targeting Calpain-2, a key protein in axonal degeneration, among others. Risks and Uncertainties The Company is subject to risks and uncertainties common to companies in the biotechnology industry, including, but not limited to, the outcome of preclinical studies and clinical trials, market acceptance and the successful commercialization of its approved products ALBRIOZA, which received marketing authorization with conditions in Canada in June 2022, and RELYVRIO, which was approved by the FDA in the U.S. in September 2022, potential difficulties with or delays in timing with respect to the regulatory approval processes of the EMA and other comparable foreign authorities, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, ability to secure additional capital to fund operations, and risks associated with the economic challenges caused by the COVID-19 pandemic or other public health concerns and economic uncertainty in various global markets caused by geopolitical instability and conflict. The Company and its contractors may experience disruptions in supply of items that are essential for its research and development and commercial activities, including, for example, raw materials and bulk drug substances that the Company imports from Europe and Canada used in the manufacturing of AMX0035, and any additional or future product candidates . |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Significant Accounting Policies The Company’s significant accounting policies are disclosed in the audited consolidated financial statements for the year ended December 31, 2022 and the notes thereto, which are included in the Company’s most recent Annual Report on Form 10-K. Since the date of those consolidated financial statements, there have been no material changes to its significant accounting policies, with the exception of significant accounting policies related to the adoption of the Financial Accounting Standards Board, or FASB, Accounting Standards Codification, or ASC, Topic 326, Financial Instruments—Credit Losses , or ASC 326, effective January 1, 2023, as described below. Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the U.S., or GAAP, and include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. In the opinion of the Company's management, all normal and recurring adjustments necessary for a fair presentation have been reflected. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the ASC, and Accounting Standards Update, or ASU, of the FASB. Use of Estimates The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: gross-to-net, or GTN, adjustments; recoverability of inventories, including those produced in preparation for product launches; accrued expenses; stock option valuations; valuation allowance for deferred tax assets and research and development expense s. Recently Adopted Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326) Measurement of Credit Losses on Financial Instruments , or ASU 2016-13. The provisions of ASU 2016-13 modify the impairment model to utilize an expected loss methodology in place of the currently used incurred loss methodology and require a consideration of a broader range of reasonable and supportable information to inform credit loss estimates. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. ASU 2016-13 requires a cumulative effect adjustment to the consolidated balance sheet as of the beginning of the first reporting period in which the guidance is effective. The Company adopted ASU 2016-13 effective January 1, 2023, with no material impact on its consolidated financial statements and related disclosures. |
Product Revenue, Net
Product Revenue, Net | 6 Months Ended |
Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Product Revenue, Net | 3. PRODUCT REVENUE, NET To date, the Company’s only source of product revenue has been from the sales of RELYVRIO, known as ALBRIOZA in Canada. Significant judgment is required in estimating GTN adjustments considering historical experience, payer channel mix, current contract prices, unbilled claims, processing time lags, inventory levels in the distribution ch annel and estimated product returns. The following table reconciles gross product revenue to net product revenue (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Product revenue, gross $ 108,405 $ — $ 192,958 $ — GTN adjustments ( 10,189 ) — ( 23,314 ) — Product revenue, net $ 98,216 $ — $ 169,644 $ — The activity and ending reserve balance for GTN adjustments were as follows for the six months ended June 30, 2023 (in thousands): Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2022 $ 648 $ 1,992 $ 1,664 $ 4,304 Provision related to sales in the current year 10,053 6,516 7,147 23,716 Adjustments related to prior period sales — ( 236 ) ( 166 ) ( 402 ) Credits and payments made ( 5,831 ) ( 4,294 ) ( 5,988 ) ( 16,113 ) Ending balance at June 30, 2023 $ 4,870 $ 3,978 $ 2,657 $ 11,505 Included in the ending reserve balance for GTN adjustments are chargebacks resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to customers who directly purchase the product from the Company, discounts to customers for prompt payment and estimates for product returns. Chargebacks, discounts and returns are recorded as reductions of accounts receivable, net on the condensed consolidated balance sheets. In addition, included in the ending reserve balance for GTN adjustments are Medicaid and Medicare rebates, other rebates for obligations under voluntary patient assistance programs, and accrued fees payable to customers. Medicaid and Medicare rebates, other rebates and fees are recorded as a component of accrued expenses on the condensed consolidated balance sheets. |
Short-Term Investments
Short-Term Investments | 6 Months Ended |
Jun. 30, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Short-Term Investments | 4. SHORT-TERM INVESTMENTS The Company determines the appropriate classification of marketable securities at the time of purchase and reevaluates such designation at each balance sheet date. The Company has classified all of its marketable securities at June 30, 2023 as “available-for-sale” pursuant to ASC 320, Investments – Debt and Equity Securities . The Company records available-for-sale securities at fair value, with the unrealized gains and losses included as a separate component of other accumulated comprehensive income (loss). There were no realized gains or losses recognized during the three and six months ended June 30, 2023 and 2022. The Company adjusts the cost of available-for-sale debt securities for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion are included in interest income. The cost of securities sold is based on the specific identification method. The Company includes interest and dividends on securities classified as available-for-sale in interest. Accrued interest receivable relating to the Company's available-for-sale securities is presented within prepaid expenses and other current assets in the accompanying condensed consolidated balance sheets, and amounted to $ 0.8 million and $ 0.5 million at June 30, 2023 and December 31, 2022, respectively. The following is a summary of available-for-sale securities with unrealized losses for less than 12 months as of June 30, 2023 and December 31, 2022 (in thousands): June 30, 2023 December 31, 2022 Fair Value Unrealized Losses Fair Value Unrealized Losses Treasury notes $ 27,762 $ ( 24 ) $ 27,159 $ ( 14 ) Treasury bills — — 9,839 ( 2 ) Commercial paper 46,371 ( 26 ) — — Corporate debt securities 15,516 ( 11 ) 33,486 ( 55 ) Agency bonds 9,870 ( 20 ) — — Total available-for-sale securities in an unrealized loss position $ 99,519 $ ( 81 ) $ 70,484 $ ( 71 ) At June 30, 2023, the Company's security portfolio consisted of 15 securities related to investments in debt securities available-for-sale, of which 14 se curities were in an unrealized loss position. There were no securities in an unrealized loss position for greater than 12 months as of June 30, 2023. The contractual terms of these investments do not permit the issuer to settle the securities at a price less than the amortized cost bases of the investments. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost bases. The Company did not record an allowance for credit losses as of June 30, 2023. Prior to January 1, 2023, the Company evaluated short-term investments for other-than-temporary impairment at the balance sheet date. Declines in fair value, if any, determined to be other-than-temporary were also included in other income, net. When assessing short-term investments for other-than-temporary declines in value, the Company considered such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, and the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value and market conditions in general. The Company determined it did not hold any investments with any other-than-temporary impairment as of December 31, 2022. Short-term investments, which are classified as available-for-sale, consisted of the following (in thousands): Balance at June 30, 2023: Amortized Unrealized Unrealized Fair Treasury notes $ 27,786 $ — $ ( 24 ) $ 27,762 Commercial paper 46,397 — ( 26 ) 46,371 Corporate debt securities 25,521 3 ( 11 ) 25,513 Agency bonds 9,890 — ( 20 ) 9,870 Total short-term investments $ 109,594 $ 3 $ ( 81 ) $ 109,516 Balance at December 31, 2022: Amortized Unrealized Unrealized Fair Treasury notes $ 27,173 $ — $ ( 14 ) $ 27,159 Treasury bills 59,326 10 ( 2 ) 59,334 Commercial paper 134,375 — — 134,375 Corporate debt securities 58,795 13 ( 55 ) 58,753 Agency bonds 4,781 17 — 4,798 Total short-term investments $ 284,450 $ 40 $ ( 71 ) $ 284,419 |
Inventories
Inventories | 6 Months Ended |
Jun. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Inventories | 5. INVENTORIES Inventories consisted of the following (in thousands): June 30, 2023 December 31, 2022 Raw materials $ 28,915 $ 7,151 Work in process 8,702 1,681 Finished goods 4,970 937 Total inventories $ 42,587 $ 9,769 The Company capitalizes inventory costs associated with its products after regulatory approval when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. As of June 30, 2023, the Company had $ 12.0 million of in ventory on hand that was acquired prior to regulatory approvals. This inventory was expensed to research and development as the future economic benefit was not probable. The Company began to capitalize inventory costs upon receipt of regulatory approvals in 2022. Finished goods have a shelf life of 12 - 18 months from the date of manufacture. |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2023 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses consisted of the following (in thousands): June 30, 2023 December 31, 2022 Accrued external research and development $ 9,298 $ 8,424 Accrued benefits and incentive compensation 7,946 15,231 Accrued manufacturing 4,339 4,596 Accrued consulting and other professional fees 5,407 4,116 Accrued rebates and co-pay assistance 6,456 3,582 Accrued royalties 3,111 1,358 Other accrued expenses 684 1,005 Total accrued expenses $ 37,241 $ 38,312 |
Fair Value Measurements
Fair Value Measurements | 6 Months Ended |
Jun. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | . FAIR VALUE MEASUREMENTS The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values (in thousands): June 30, 2023 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 150,723 $ — $ — $ 150,723 Short-term investments: Treasury notes 27,762 — — 27,762 Commercial paper — 46,371 — 46,371 Corporate debt securities — 25,513 — 25,513 Agency bonds — 9,870 — 9,870 Total short-term investments 27,762 81,754 — 109,516 Restricted cash equivalents 719 — — 719 Total financial assets $ 179,204 $ 81,754 $ — $ 260,958 December 31, 2022 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 23,567 $ 9,989 $ — $ 33,556 Short-term investments: Treasury notes 27,159 — — 27,159 Treasury bills 59,334 — — 59,334 Commercial paper — 134,375 — 134,375 Corporate debt securities — 58,753 — 58,753 Agency bonds — 4,798 — 4,798 Total short-term investments 86,493 197,926 — 284,419 Restricted cash equivalents 719 — — 719 Total financial assets $ 110,779 $ 207,915 $ — $ 318,694 The Company classifies its money market funds, treasury notes and treasury bills as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices for identical assets in active markets without any valuation adjustment. The Company classifies its commercial paper, corporate debt securities, and agency bonds as Level 2 assets under the fair value hierarchy, as these assets have been valued using information obtained through a third-party pricing service at each balance sheet date, using observable market inputs that may include trade information, broker or dealer quotes, bids, offers, or a combination of these data sources. |
Leases
Leases | 6 Months Ended |
Jun. 30, 2023 | |
Leases [Abstract] | |
Leases | 8. Leases The Company adopted ASC 842 on January 1, 2022. ASC 842 allows the Company to elect a package of practical expedients, which include: (i) an entity need not reassess whether any expired or existing contracts are or contain leases; (ii) an entity need not reassess the lease classification for any expired or existing leases; and (iii) an entity need not reassess any initial direct costs for any existing leases. Another practical expedient allows the Company to use hindsight in determining the lease term when considering lessee options to extend or terminate the lease and to purchase the underlying asset. The Company elected to utilize this package of practical expedients and did not elect the hindsight methodology in its implementation of ASC 842. The Company leases its office facilities under non-cancelable operating leases that expire at various dates through October 2026. Components of lease expense required by ASC 842 are presented below for the three and six months ended June 30, 2023 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Lease cost Operating lease cost $ 544 $ 544 $ 1,088 $ 1,048 Total lease cost $ 544 $ 544 $ 1,088 $ 1,048 During the six months ended June 30, 2023, the Company made cash payments of $ 1.2 million for operating leases. Future minimum lease payments under non-cancelable leases as of June 30, 2023, were as detailed below (in thousands): June 30, 2023 2023 (remaining 6 months) $ 1,220 2024 2,478 2025 1,586 2026 476 2027 — Total undiscounted lease payments 5,760 Less: imputed interest ( 473 ) Total operating lease liabilities $ 5,287 As of June 30, 2023, the weighted average remaining lease term was 2.4 years and the weighted average incremental borrowing rate used to determine the operating lease right-of-use assets was 7.3 % . |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2023 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 9. Income taxes For the three months ended June 30, 2023 and 2022, the Company recorded an income tax provision of $ 1.9 million and $ 0.2 million , respectively. The income tax provision for each of the three months ended June 30, 2023 and 2022 was primarily driven by the estimated effective tax rate for the year, as well as discrete income tax benefit of $ 0.1 million and zero , respectively. For the six months ended June 30, 2023 and 2022, the Company recorded an income tax provision of $ 1.8 million and $ 0.3 million , respectively. The income tax provision for the six months ended June 30, 2023 and 2022 was primarily driven by the estimated effective tax rate for the year, as well as discrete income tax benefit of $ 0.4 million and zero , respectively. The income tax provision for the three and six months ended June 30, 2023 includes the release of a portion of the Company’s valuation allowance that has been reversed through the annual effective tax rate with respect to amounts expected to be realized through current year taxable income. |
Stock Option and Grant Plan
Stock Option and Grant Plan | 6 Months Ended |
Jun. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Stock Option and Grant Plan | 10. Stock Option and Grant Plan Stock Incentive Plan General Option Information A summary of option activity for the six months ended June 30, 2023, is as follows: Number of Weighted- Weighted- Aggregate Outstanding at December 31, 2022 8,480,950 $ 13.19 8.2 $ 201,765 Granted 2,399,056 $ 31.96 Exercised ( 677,436 ) $ 6.59 $ 16,202 Cancelled or forfeited ( 210,721 ) $ 17.54 Outstanding at June 30, 2023 9,991,849 $ 18.05 8.2 $ 66,550 Options exercisable as of June 30, 2023 3,172,459 $ 11.01 7.2 $ 34,430 Options unvested as of June 30, 2023 6,819,390 $ 21.32 8.7 $ 32,120 Weighted average grant-date fair value of options granted during the period $ 21.15 The aggregate intrinsic value of stock options is calculated as the difference between the exercise price of the options and the fair value of the Company’s common stock for those options that had exercise prices lower than the fair value of the Company’s common stock. The aggregate intrinsic value of options exercised during the six months ended June 30, 2023 and 2022 was $ 16.2 million and $ 6.0 million , respectively. The total fair value of stock options vested during the six months ended June 30, 2023 and 2022 was $ 19.9 million and $ 3.2 million , respectively. Restricted Stock Unit Activity A summary of restricted stock unit activity for the six months ended June 30, 2023, is as follows: Number of Shares Weighted Average Grant Date Fair Value Nonvested as of December 31, 2022 740,297 $ 20.02 Granted 512,878 $ 32.11 Vested ( 164,970 ) $ 18.42 Forfeited ( 26,141 ) $ 25.66 Nonvested as of June 30, 2023 1,062,064 $ 25.97 Summary of Stock-Based Compensation Expense Stock-based compensation expense recorded in the condensed consolidated statements of operations for the three and six months ended June 30, 2023 and 2022, is as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Research and development $ 2,535 $ 1,460 $ 4,426 $ 2,585 Selling, general and administrative 7,415 4,247 13,025 7,514 Total stock-based compensation expense $ 9,950 $ 5,707 $ 17,451 $ 10,099 The Company capitalized stock-based compensation expense of $ 0.1 million and $ 0.2 million for the three and six months ended June 30, 2023 , respectively. The Company did no t capitalize any stock-based compensation expense during the three and six months ended June 30, 2022. Stock-based compensation recognized through cost of sales was less than $ 0.1 million for the three and six months ended June 30, 2023. The following table summarizes stock-based compensation by type of award (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Stock options $ 8,115 $ 4,955 $ 14,371 $ 8,863 Restricted stock units 1,835 752 3,080 1,236 Total stock-based compensation expense $ 9,950 $ 5,707 $ 17,451 $ 10,099 The following table summarizes unrecognized stock-based compensation expense as of June 30, 2023, by type of awards (in thousands), and the weighted-average period over which that expense is expected to be recognized (in years). The total unrecognized stock-based compensation expense will be adjusted for actual forfeitures as they occur. June 30, 2023 Unrecognized Expense Weighted-average Recognition Period Stock options $ 94,199 3.01 Restricted stock units $ 24,635 3.31 |
Net Income (Loss) Per Share
Net Income (Loss) Per Share | 6 Months Ended |
Jun. 30, 2023 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Share | 11. NET INCOME (LOSS) PER SHARE Net Income (Loss) per Share Attributable to Common Stockholders Basic earnings per share is computed by dividing net income (loss) by the weighted average number of common shares outstanding during the period. Diluted earnings per share is calculated based on the combined weighted average number of common shares and potentially dilutive shares, which include the assumed exercise of employee stock options and unvested restricted stock units. In computing diluted earnings per share, the Company utilizes the treasury stock method. A summary of the numerator and denominators used in the computation of earnings per share follows (in thousands, except share and per share data): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Numerator: Net income (loss) $ 22,074 $ ( 54,067 ) $ 23,647 $ ( 101,915 ) Denominator: Weighted-average shares used to compute basic net income (loss) per share 67,233,617 58,275,903 66,976,871 54,958,537 Dilutive effect of employee stock options and restricted stock units 2,898,423 — 3,494,950 — Weighted-average shares used to compute diluted net income (loss) per share 70,132,040 58,275,903 70,471,821 54,958,537 Net income (loss) per share attributable to common stockholders Basic $ 0.33 $ ( 0.93 ) $ 0.35 $ ( 1.85 ) Diluted $ 0.31 $ ( 0.93 ) $ 0.34 $ ( 1.85 ) Because the Company reported a net loss attributable to common stockholders for the three and six months ended June 30, 2022, basic and diluted net loss per share attributable to common stockholders were the same. All stock options and restricted stock units were excluded from the computation of diluted weighted-average shares outstanding because such securities would have an antidilutive impact for the three and six months ended June 30, 2022 . The following stock options and restricted stock units outstanding at each period end have been excluded from the calculation of diluted net income (loss) per share because their inclusion would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Options to purchase common stock 5,831,498 8,528,039 3,198,508 8,528,039 Restricted stock units 567,671 670,013 85,658 670,013 Total excluded common stock equivalents 6,399,169 9,198,052 3,284,166 9,198,052 |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2023 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 12. Related party transactions Supplier Agreements In the ordinary course of business, the Company may purchase materials or supplies or services from entities that are associated with a party that meets the criteria of a related party of the Company. These transactions are reviewed quarterly and to date have not been material to the Company’s condensed consolidated financial statements. |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 13. Commitments and Contingencies Lease Commitments The Company has two operating lease agreements for its office space. See Note 8 for additional information. Letter of Credit Restricted cash equivalents consist of $ 0.2 million of cash serving as collateral for a letter of credit issued for the Company’s office space, and $ 0.5 million as collateral for a corporate credit card program. As of June 30, 2023 and December 31, 2022, the Company’s restricted cash equivalents balance was $ 0.7 million on its condensed consolidated balance sheets. Legal Proceedings The Company is not currently a party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or potential range of loss is probable and reasonably estimated under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company recognizes expenses for its costs related to its legal proceedings, as incurred. Royalty Payments Between August 2016 and February 2019, the Company entered into agreements with the ALS Association, ALS Finding a Cure Foundation, Alzheimer’s Drug Discovery Foundation, Alzheimer’s Association and Cure Alzheimer’s Fund, or Grantors. Under the terms of the agreements, the Company was granted, in aggregate, $ 4.3 million. These grants were provided to the Company for the purpose of furthering the research and development of AMX0035 as a therapeutic benefit for ALS and Alzheimer’s disease. Under the terms of the arrangements, the Company would receive a tranche of funds as it completes certain milestones. Pursuant to the terms of the grant agreements, the Company has certain payment obligations that are contingent upon future events such as the achievement of commercialization or the receipt of proceeds from a revenue generating transaction resulting from the projects for which the grants are used for. Pursuant to the terms of the respective grant agreements among the Company, ALS Association and ALS Finding a Cure, the Company will be required to make royalty payments to each Grantor in the total amount equal to 150 % of the grant received. The royalty payments will be achieved through a combination of the following payment methods: (i) an annual installment payment of 3 % of net sales of any products developed under the project for which the grant was used for and (ii) 3 % of cash proceeds resulting from revenue generating transaction under the project for which the grants are used for. During the three and six months ended June 30, 2023, the Company recorded $ 0.6 million and $ 3.1 million in royalty expense, respectively, which is included in cost of sales in the condensed consolidated financial statements. As of June 30, 2023 , no further royalties remain to be accrued under the grant agreements with the ALS Association and ALS Finding a Cure Foundation. Under the terms of the respective grant agreements among the Company, Alzheimer’s Drug Discovery Foundation, the Alzheimer’s Association, and Cure Alzheimer’s Fund, the Company will make royalty payments up to the maximum amount of $ 15.0 million to each Grantor (or $ 45.0 million in aggregate). The royalty payment will be made through a combination of the following payment methods: (i) 4 % of annual net sales of any product commercialized from the project for which the grant was used for and directly related to the treatment of the Alzheimer’s disease and (ii) 15 % of all royalties and cash proceeds resulting from revenue generating transactions associated with the projects for which the grants were used for under the grant agreements. As the conditions that would trigger royalty payments under the agreements have not occurred, no amounts have been recorded in the condensed consolidated financial statements for the three and six months ended June 30, 2023 and 2022. Purchase Commitments The Company enters into agreements in the normal course of business with contract manufacturing organizations, or CMOs, for raw material purchases and manufacturing services. As of June 30, 2023, the Company had committed approximately $ 86.8 million under these agreements related to raw material purchases and manufacturing services, which are expected to be paid through 2025. |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2023 | |
Subsequent Events [Abstract] | |
Subsequent Events | 14. Subsequent Events The Company has evaluated all subsequent events after June 30, 2023 and through the date of this filing, and there were no material subsequent events requiring disclosure, except as described below. 2023 Inducement Plan In July 2023, the Company’s board of directors adopted the Amylyx Pharmaceuticals, Inc. 2023 Inducement Plan, or the Inducement Plan, to grant equity awards to induce highly-qualified prospective officers and employees who are not currently employed by the Company to accept employment and provide them with a proprietary interest in the Company, pursuant to Rule 5635(c)(4) of the Marketplace Rules of the NASDAQ Stock Market, Inc. The Company has reserved 750,000 shares of its common stock that may be issued under the Inducement Plan. Supplier Agreements In August 2023, the Company entered into an agreement with a CMO for the purchase of raw materials. Under the terms of the agreement, the Company committed to minimum annual raw material purchases. As a result, the Company's total purchase commitments related to raw material purchases and manufacturing services have increased to approximately $ 223.3 million , which are expected to be paid through 2028. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Consolidation | Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the U.S., or GAAP, and include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. In the opinion of the Company's management, all normal and recurring adjustments necessary for a fair presentation have been reflected. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the ASC, and Accounting Standards Update, or ASU, of the FASB. |
Use of Estimates | Use of Estimates The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: gross-to-net, or GTN, adjustments; recoverability of inventories, including those produced in preparation for product launches; accrued expenses; stock option valuations; valuation allowance for deferred tax assets and research and development expense |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326) Measurement of Credit Losses on Financial Instruments , or ASU 2016-13. The provisions of ASU 2016-13 modify the impairment model to utilize an expected loss methodology in place of the currently used incurred loss methodology and require a consideration of a broader range of reasonable and supportable information to inform credit loss estimates. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. ASU 2016-13 requires a cumulative effect adjustment to the consolidated balance sheet as of the beginning of the first reporting period in which the guidance is effective. The Company adopted ASU 2016-13 effective January 1, 2023, with no material impact on its consolidated financial statements and related disclosures. |
Product Revenue, Net (Tables)
Product Revenue, Net (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Summary of Gross Product Revenue to Net Product Revenue | The following table reconciles gross product revenue to net product revenue (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Product revenue, gross $ 108,405 $ — $ 192,958 $ — GTN adjustments ( 10,189 ) — ( 23,314 ) — Product revenue, net $ 98,216 $ — $ 169,644 $ — |
Schedule of Activity and Ending Reserve Balance for GTN Adjustments | The activity and ending reserve balance for GTN adjustments were as follows for the six months ended June 30, 2023 (in thousands): Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2022 $ 648 $ 1,992 $ 1,664 $ 4,304 Provision related to sales in the current year 10,053 6,516 7,147 23,716 Adjustments related to prior period sales — ( 236 ) ( 166 ) ( 402 ) Credits and payments made ( 5,831 ) ( 4,294 ) ( 5,988 ) ( 16,113 ) Ending balance at June 30, 2023 $ 4,870 $ 3,978 $ 2,657 $ 11,505 |
Short-Term Investments (Tables)
Short-Term Investments (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-sale Securities with Unrealized Losses for Less Than 12 Months | The following is a summary of available-for-sale securities with unrealized losses for less than 12 months as of June 30, 2023 and December 31, 2022 (in thousands): June 30, 2023 December 31, 2022 Fair Value Unrealized Losses Fair Value Unrealized Losses Treasury notes $ 27,762 $ ( 24 ) $ 27,159 $ ( 14 ) Treasury bills — — 9,839 ( 2 ) Commercial paper 46,371 ( 26 ) — — Corporate debt securities 15,516 ( 11 ) 33,486 ( 55 ) Agency bonds 9,870 ( 20 ) — — Total available-for-sale securities in an unrealized loss position $ 99,519 $ ( 81 ) $ 70,484 $ ( 71 ) |
Schedule of Short-term Investments Classified as Available-for-sale | Short-term investments, which are classified as available-for-sale, consisted of the following (in thousands): Balance at June 30, 2023: Amortized Unrealized Unrealized Fair Treasury notes $ 27,786 $ — $ ( 24 ) $ 27,762 Commercial paper 46,397 — ( 26 ) 46,371 Corporate debt securities 25,521 3 ( 11 ) 25,513 Agency bonds 9,890 — ( 20 ) 9,870 Total short-term investments $ 109,594 $ 3 $ ( 81 ) $ 109,516 Balance at December 31, 2022: Amortized Unrealized Unrealized Fair Treasury notes $ 27,173 $ — $ ( 14 ) $ 27,159 Treasury bills 59,326 10 ( 2 ) 59,334 Commercial paper 134,375 — — 134,375 Corporate debt securities 58,795 13 ( 55 ) 58,753 Agency bonds 4,781 17 — 4,798 Total short-term investments $ 284,450 $ 40 $ ( 71 ) $ 284,419 |
Inventories (Tables)
Inventories (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Summary of Inventories | Inventories consisted of the following (in thousands): June 30, 2023 December 31, 2022 Raw materials $ 28,915 $ 7,151 Work in process 8,702 1,681 Finished goods 4,970 937 Total inventories $ 42,587 $ 9,769 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following (in thousands): June 30, 2023 December 31, 2022 Accrued external research and development $ 9,298 $ 8,424 Accrued benefits and incentive compensation 7,946 15,231 Accrued manufacturing 4,339 4,596 Accrued consulting and other professional fees 5,407 4,116 Accrued rebates and co-pay assistance 6,456 3,582 Accrued royalties 3,111 1,358 Other accrued expenses 684 1,005 Total accrued expenses $ 37,241 $ 38,312 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values (in thousands): June 30, 2023 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 150,723 $ — $ — $ 150,723 Short-term investments: Treasury notes 27,762 — — 27,762 Commercial paper — 46,371 — 46,371 Corporate debt securities — 25,513 — 25,513 Agency bonds — 9,870 — 9,870 Total short-term investments 27,762 81,754 — 109,516 Restricted cash equivalents 719 — — 719 Total financial assets $ 179,204 $ 81,754 $ — $ 260,958 December 31, 2022 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 23,567 $ 9,989 $ — $ 33,556 Short-term investments: Treasury notes 27,159 — — 27,159 Treasury bills 59,334 — — 59,334 Commercial paper — 134,375 — 134,375 Corporate debt securities — 58,753 — 58,753 Agency bonds — 4,798 — 4,798 Total short-term investments 86,493 197,926 — 284,419 Restricted cash equivalents 719 — — 719 Total financial assets $ 110,779 $ 207,915 $ — $ 318,694 |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Leases [Abstract] | |
Components of Lease Expense | Components of lease expense required by ASC 842 are presented below for the three and six months ended June 30, 2023 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Lease cost Operating lease cost $ 544 $ 544 $ 1,088 $ 1,048 Total lease cost $ 544 $ 544 $ 1,088 $ 1,048 |
Summary of Future Minimum Lease Payments | Future minimum lease payments under non-cancelable leases as of June 30, 2023, were as detailed below (in thousands): June 30, 2023 2023 (remaining 6 months) $ 1,220 2024 2,478 2025 1,586 2026 476 2027 — Total undiscounted lease payments 5,760 Less: imputed interest ( 473 ) Total operating lease liabilities $ 5,287 |
Stock Option and Grant Plan (Ta
Stock Option and Grant Plan (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Stock Option Activity | A summary of option activity for the six months ended June 30, 2023, is as follows: Number of Weighted- Weighted- Aggregate Outstanding at December 31, 2022 8,480,950 $ 13.19 8.2 $ 201,765 Granted 2,399,056 $ 31.96 Exercised ( 677,436 ) $ 6.59 $ 16,202 Cancelled or forfeited ( 210,721 ) $ 17.54 Outstanding at June 30, 2023 9,991,849 $ 18.05 8.2 $ 66,550 Options exercisable as of June 30, 2023 3,172,459 $ 11.01 7.2 $ 34,430 Options unvested as of June 30, 2023 6,819,390 $ 21.32 8.7 $ 32,120 Weighted average grant-date fair value of options granted during the period $ 21.15 |
Summary of Restricted Stock Unit Activity | A summary of restricted stock unit activity for the six months ended June 30, 2023, is as follows: Number of Shares Weighted Average Grant Date Fair Value Nonvested as of December 31, 2022 740,297 $ 20.02 Granted 512,878 $ 32.11 Vested ( 164,970 ) $ 18.42 Forfeited ( 26,141 ) $ 25.66 Nonvested as of June 30, 2023 1,062,064 $ 25.97 |
Summary of Stock-Based Compensation Expense | Stock-based compensation expense recorded in the condensed consolidated statements of operations for the three and six months ended June 30, 2023 and 2022, is as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Research and development $ 2,535 $ 1,460 $ 4,426 $ 2,585 Selling, general and administrative 7,415 4,247 13,025 7,514 Total stock-based compensation expense $ 9,950 $ 5,707 $ 17,451 $ 10,099 |
Summary of Stock-Based Compensation by Type of Award | The following table summarizes stock-based compensation by type of award (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Stock options $ 8,115 $ 4,955 $ 14,371 $ 8,863 Restricted stock units 1,835 752 3,080 1,236 Total stock-based compensation expense $ 9,950 $ 5,707 $ 17,451 $ 10,099 |
Summary of Unrecognized Stock-Based Compensation Expense and Weighted-Average Recognition Period | The following table summarizes unrecognized stock-based compensation expense as of June 30, 2023, by type of awards (in thousands), and the weighted-average period over which that expense is expected to be recognized (in years). The total unrecognized stock-based compensation expense will be adjusted for actual forfeitures as they occur. June 30, 2023 Unrecognized Expense Weighted-average Recognition Period Stock options $ 94,199 3.01 Restricted stock units $ 24,635 3.31 |
Net Income (Loss) Per Share (Ta
Net Income (Loss) Per Share (Tables) | 6 Months Ended |
Jun. 30, 2023 | |
Earnings Per Share [Abstract] | |
Summary of the Numerator and Denominators Used in Computation of Earnings Per Share | A summary of the numerator and denominators used in the computation of earnings per share follows (in thousands, except share and per share data): Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Numerator: Net income (loss) $ 22,074 $ ( 54,067 ) $ 23,647 $ ( 101,915 ) Denominator: Weighted-average shares used to compute basic net income (loss) per share 67,233,617 58,275,903 66,976,871 54,958,537 Dilutive effect of employee stock options and restricted stock units 2,898,423 — 3,494,950 — Weighted-average shares used to compute diluted net income (loss) per share 70,132,040 58,275,903 70,471,821 54,958,537 Net income (loss) per share attributable to common stockholders Basic $ 0.33 $ ( 0.93 ) $ 0.35 $ ( 1.85 ) Diluted $ 0.31 $ ( 0.93 ) $ 0.34 $ ( 1.85 ) |
Schedule of Antidilutive Shares Excluded from the Calculation of Diluted Net Income (Loss) Per Share | The following stock options and restricted stock units outstanding at each period end have been excluded from the calculation of diluted net income (loss) per share because their inclusion would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Options to purchase common stock 5,831,498 8,528,039 3,198,508 8,528,039 Restricted stock units 567,671 670,013 85,658 670,013 Total excluded common stock equivalents 6,399,169 9,198,052 3,284,166 9,198,052 |
Product Revenue, Net - Summary
Product Revenue, Net - Summary of Gross Product Revenue to Net product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2023 | Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | ||
Product revenue, gross | $ 108,405 | $ 192,958 |
GTN adjustments | (10,189) | (23,314) |
Product revenue, net | $ 98,216 | $ 169,644 |
Product Revenue, Net - Schedule
Product Revenue, Net - Schedule of Activity and Ending Reserve Balance for GTN Adjustments (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
Disaggregation of Revenue [Line Items] | |
GTN Adjustments, Ending balance at December 31, 2022 | $ 4,304 |
GTN Adjustments, Provision related to sales in the current year | 23,716 |
GTN Adjustments, Adjustments related to prior period sales | (402) |
GTN Adjustments, Credits and payments made | (16,113) |
GTN Adjustments, Ending balance at June 30, 2023 | 11,505 |
Chargebacks and Cash Discounts | |
Disaggregation of Revenue [Line Items] | |
GTN Adjustments, Ending balance at December 31, 2022 | 648 |
GTN Adjustments, Provision related to sales in the current year | 10,053 |
GTN Adjustments, Credits and payments made | (5,831) |
GTN Adjustments, Ending balance at June 30, 2023 | 4,870 |
Medicaid and Medicare Rebates | |
Disaggregation of Revenue [Line Items] | |
GTN Adjustments, Ending balance at December 31, 2022 | 1,992 |
GTN Adjustments, Provision related to sales in the current year | 6,516 |
GTN Adjustments, Adjustments related to prior period sales | (236) |
GTN Adjustments, Credits and payments made | (4,294) |
GTN Adjustments, Ending balance at June 30, 2023 | 3,978 |
Other Rebates, Returns, Discounts and Adjustments | |
Disaggregation of Revenue [Line Items] | |
GTN Adjustments, Ending balance at December 31, 2022 | 1,664 |
GTN Adjustments, Provision related to sales in the current year | 7,147 |
GTN Adjustments, Adjustments related to prior period sales | (166) |
GTN Adjustments, Credits and payments made | (5,988) |
GTN Adjustments, Ending balance at June 30, 2023 | $ 2,657 |
Short-Term Investments - Additi
Short-Term Investments - Additional Information (Details) | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2023 USD ($) Security | Jun. 30, 2022 USD ($) | Jun. 30, 2023 USD ($) Security | Jun. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) | |
Debt Securities, Available-for-Sale [Line Items] | |||||
Realized gains | $ | $ 0 | $ 0 | $ 0 | $ 0 | |
Realized loss | $ | $ 0 | $ 0 | $ 0 | $ 0 | |
Debt securities available-for-sale number of positions | Security | 15 | 15 | |||
Debt securities available-for-sale number of positions in an unrealized loss position | Security | 14 | 14 | |||
Debt securities available-for-sale in an unrealized loss position for greater than 12 months | Security | 0 | 0 | |||
Available-for-Sale Securities | |||||
Debt Securities, Available-for-Sale [Line Items] | |||||
Accrued interest receivable | $ | $ 800,000 | $ 800,000 | $ 500,000 |
Short-Term Investments - Summar
Short-Term Investments - Summary of Available-for-sale Securities with Unrealized Losses for Less Than 12 Months (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | $ 99,519 | $ 70,484 |
Unrealized Losses | (81) | (71) |
Commercial Paper | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | 46,371 | |
Unrealized Losses | (26) | |
Treasury notes | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | 27,762 | 27,159 |
Unrealized Losses | (24) | (14) |
Treasury Bills | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | 9,839 | |
Unrealized Losses | (2) | |
Corporate Debt Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | 15,516 | 33,486 |
Unrealized Losses | (11) | $ (55) |
Agency Bonds | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Fair Value | 9,870 | |
Unrealized Losses | $ (20) |
Short-Term Investments - Schedu
Short-Term Investments - Schedule of Short-term Investments Classified as Available-for-sale (Details) - Short-term Investments - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | $ 109,594 | $ 284,450 |
Unrealized Gain | 3 | 40 |
Unrealized Loss | (81) | (71) |
Fair Value | 109,516 | 284,419 |
Commercial Paper | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 46,397 | 134,375 |
Unrealized Loss | (26) | |
Fair Value | 46,371 | 134,375 |
Treasury notes | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 27,786 | 27,173 |
Unrealized Loss | (24) | (14) |
Fair Value | 27,762 | 27,159 |
Treasury Bills | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 59,326 | |
Unrealized Gain | 10 | |
Unrealized Loss | (2) | |
Fair Value | 59,334 | |
Corporate Debt Securities | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 25,521 | 58,795 |
Unrealized Gain | 3 | 13 |
Unrealized Loss | (11) | (55) |
Fair Value | 25,513 | 58,753 |
Agency Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 9,890 | 4,781 |
Unrealized Gain | 17 | |
Unrealized Loss | (20) | |
Fair Value | $ 9,870 | $ 4,798 |
Inventories - Summary of Invent
Inventories - Summary of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 28,915 | $ 7,151 |
Work in process | 8,702 | 1,681 |
Finished goods | 4,970 | 937 |
Total inventories | $ 42,587 | $ 9,769 |
Inventories - Additional Inform
Inventories - Additional Information (Details) $ in Millions | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
Public Utilities, Inventory [Line Items] | |
Inventory acquired expensed as research and development expense | $ 12 |
Finished Goods | Maximum | |
Public Utilities, Inventory [Line Items] | |
Shelf life | 18 months |
Finished Goods | Minimum | |
Public Utilities, Inventory [Line Items] | |
Shelf life | 12 months |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Accrued external research and development | $ 9,298 | $ 8,424 |
Accrued benefits and incentive compensation | 7,946 | 15,231 |
Accrued Manufacturing | 4,339 | 4,596 |
Accrued consulting and other professional fees | 5,407 | 4,116 |
Accrued rebates and co-pay assistance | 6,456 | 3,582 |
Accrued royalties | 3,111 | 1,358 |
Other accrued expenses | 684 | 1,005 |
Total accrued expenses | $ 37,241 | $ 38,312 |
Fair Value Measurements - Sched
Fair Value Measurements - Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value Measurements, Recurring - USD ($) $ in Thousands | Jun. 30, 2023 | Dec. 31, 2022 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | $ 260,958 | $ 318,694 |
Cash equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 150,723 | 33,556 |
Restricted Cash Equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 719 | 719 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 179,204 | 110,779 |
Level 1 | Cash equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 150,723 | 23,567 |
Level 1 | Restricted Cash Equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 719 | 719 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 81,754 | 207,915 |
Level 2 | Cash equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 9,989 | |
Short-term Investments | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 109,516 | 284,419 |
Short-term Investments | Treasury notes | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 27,762 | 27,159 |
Short-term Investments | Treasury Bills | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 59,334 | |
Short-term Investments | Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 46,371 | 134,375 |
Short-term Investments | Corporate Debt Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 25,513 | 58,753 |
Short-term Investments | Agency Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 9,870 | 4,798 |
Short-term Investments | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 27,762 | 86,493 |
Short-term Investments | Level 1 | Treasury notes | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 27,762 | 27,159 |
Short-term Investments | Level 1 | Treasury Bills | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 59,334 | |
Short-term Investments | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 81,754 | 197,926 |
Short-term Investments | Level 2 | Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 46,371 | 134,375 |
Short-term Investments | Level 2 | Corporate Debt Securities | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 25,513 | 58,753 |
Short-term Investments | Level 2 | Agency Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | $ 9,870 | $ 4,798 |
Leases- Additional Information
Leases- Additional Information (Details) $ in Millions | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
Leases [Abstract] | |
Operating lease, cash payments | $ 1.2 |
Weighted-average remaining lease term (years) | 2 years 4 months 24 days |
Weighted average incremental borrowing rate | 7.30% |
Leases - Components of Lease Ex
Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Leases [Abstract] | ||||
Operating lease cost | $ 544 | $ 544 | $ 1,088 | $ 1,048 |
Total lease cost | $ 544 | $ 544 | $ 1,088 | $ 1,048 |
Leases - Summary of Future Mini
Leases - Summary of Future Minimum Lease Payments (Detail) $ in Thousands | Jun. 30, 2023 USD ($) |
Leases [Abstract] | |
2023 (remaining 6 months) | $ 1,220 |
2024 | 2,478 |
2025 | 1,586 |
2026 | 476 |
Total undiscounted lease payments | 5,760 |
Less: imputed interest | (473) |
Total operating lease liabilities | $ 5,287 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Income Tax Disclosure [Abstract] | ||||
(Benefit) provision for income taxes | $ 1,933 | $ 174 | $ 1,763 | $ 320 |
Discrete income tax (benefit) provision | $ 100 | $ 0 | $ 400 | $ 0 |
Stock Option and Grant Plan - S
Stock Option and Grant Plan - Summary of Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 6 Months Ended | 12 Months Ended | |
Jun. 30, 2023 | Jun. 30, 2022 | Dec. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |||
Number of Options, Outstanding | 8,480,950 | ||
Number of Options, Granted | 2,399,056 | ||
Number of Options, Exercised | (677,436) | ||
Number of Options, Cancelled or forfeited | (210,721) | ||
Number of Options, Outstanding | 9,991,849 | 8,480,950 | |
Number of Options, Options exercisable | 3,172,459 | ||
Number of Options, Options unvested | 6,819,390 | ||
Weighted average grant-date fair value of options granted during the period | $ 21.15 | ||
Weighted-Average Exercise Price, Outstanding | 13.19 | ||
Weighted-Average Exercise Price, Granted | 31.96 | ||
Weighted-Average Exercise Price, Exercised | 6.59 | ||
Weighted-Average Exercise Price, Cancelled or forfeited | 17.54 | ||
Weighted-Average Exercise Price, Outstanding | 18.05 | $ 13.19 | |
Weighted-Average Exercise Price, Options exercisable | 11.01 | ||
Weighted-Average Exercise Price, Options unvested | $ 21.32 | ||
Weighted- Average Remaining Contractual Term (in years), Outstanding | 8 years 2 months 12 days | 8 years 2 months 12 days | |
Weighted- Average Remaining Contractual Term (in years), Options exercisable | 7 years 2 months 12 days | ||
Weighted- Average Remaining Contractual Term (in years), Options unvested | 8 years 8 months 12 days | ||
Aggregate Intrinsic Value, Outstanding | $ 201,765 | ||
Aggregate intrinsic value, exercised | 16,202 | $ 6,000 | |
Aggregate Intrinsic Value, Outstanding | 66,550 | $ 201,765 | |
Aggregate Intrinsic Value, Options exercisable | 34,430 | ||
Aggregate Intrinsic Value, Options unvested | $ 32,120 |
Stock Option and Grant Plan - A
Stock Option and Grant Plan - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Aggregate intrinsic value, exercised | $ 16,202,000 | $ 6,000,000 | ||
Total fair value of stock options vested | 19,900,000 | 3,200,000 | ||
Capitalized stock-based compensation expense | $ 100,000 | $ 0 | 200,000 | 0 |
Stock-based compensation recognized | 9,950,000 | $ 5,707,000 | 17,451,000 | $ 10,099,000 |
Maximum | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation recognized | $ 100,000 | $ 100,000 |
Stock Option and Grant Plan -_2
Stock Option and Grant Plan - Summary of Restricted Stock Unit Activity (Details) - Restricted Stock Unit | 6 Months Ended |
Jun. 30, 2023 $ / shares shares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Number of shares, Nonvested | shares | 740,297 |
Number of shares, Granted | shares | 512,878 |
Number of shares, Vested | shares | (164,970) |
Number of shares, Forfeited | shares | (26,141) |
Number of shares, Nonvested | shares | 1,062,064 |
Weighted Average Grant Date Fair Value, Nonvested | $ / shares | $ 20.02 |
Weighted Average Grant Date Fair Value, Granted | $ / shares | 32.11 |
Weighted Average Grant Date Fair Value, Vested | $ / shares | 18.42 |
Weighted Average Grant Date Fair Value, Forfeited | $ / shares | 25.66 |
Weighted Average Grant Date Fair Value, Nonvested | $ / shares | $ 25.97 |
Stock Option and Grant Plan -_3
Stock Option and Grant Plan - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 9,950 | $ 5,707 | $ 17,451 | $ 10,099 |
Research and Development | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 2,535 | 1,460 | 4,426 | 2,585 |
Selling, General and Administrative | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 7,415 | $ 4,247 | $ 13,025 | $ 7,514 |
Stock Option and Grant Plan -_4
Stock Option and Grant Plan - Summary of Stock-Based Compensation by Type of Award (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 9,950 | $ 5,707 | $ 17,451 | $ 10,099 |
Stock Options | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 8,115 | 4,955 | 14,371 | 8,863 |
Restricted Stock Units | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 1,835 | $ 752 | $ 3,080 | $ 1,236 |
Stock Option and Grant Plan -_5
Stock Option and Grant Plan - Summary of Unrecognized Stock-Based Compensation Expense and Weighted-Average Recognition Period (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2023 USD ($) | |
Stock Options | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Unrecognized Expense | $ 94,199 |
Weighted-average Recognition Period | 3 years 3 days |
Restricted Stock Units | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Unrecognized Expense | $ 24,635 |
Weighted-average Recognition Period | 3 years 3 months 21 days |
Net Income (Loss) Per Share - S
Net Income (Loss) Per Share - Summary of the Numerator and Denominators Used in Computation of Earnings Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2022 | Mar. 31, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Numerator: | ||||||
Net income (loss) | $ 22,074 | $ 1,573 | $ (54,067) | $ (47,848) | $ 23,647 | $ (101,915) |
Denominator: | ||||||
Weighted-average shares used to compute basic net income (loss) per share | 67,233,617 | 58,275,903 | 66,976,871 | 54,958,537 | ||
Dilutive effect of employee stock options and restricted stock units | 2,898,423 | 3,494,950 | ||||
Weighted-average shares used to compute diluted net income (loss) per share | 70,132,040 | 58,275,903 | 70,471,821 | 54,958,537 | ||
Net income (loss) per share attributable to common stockholders -basic | $ 0.33 | $ (0.93) | $ 0.35 | $ (1.85) | ||
Net income (loss) per share attributable to common stockholders -diluted | $ 0.31 | $ (0.93) | $ 0.34 | $ (1.85) |
Net Income (Loss) Per Share -_2
Net Income (Loss) Per Share - Schedule of Antidilutive Shares Excluded from the Calculation of Diluted Net Income (Loss) Per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 | Jun. 30, 2022 | Jun. 30, 2023 | Jun. 30, 2022 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 6,399,169 | 9,198,052 | 3,284,166 | 9,198,052 |
Options to Purchase Common Stock | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 5,831,498 | 8,528,039 | 3,198,508 | 8,528,039 |
Restricted Stock Unit | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 567,671 | 670,013 | 85,658 | 670,013 |
Commitment and Contingencies -
Commitment and Contingencies - Additional Information (Details) | 3 Months Ended | 6 Months Ended | 31 Months Ended | |
Jun. 30, 2023 USD ($) | Jun. 30, 2023 USD ($) LeaseAgreement | Feb. 28, 2019 USD ($) | Dec. 31, 2022 USD ($) | |
Commitments And Contingencies [Line Items] | ||||
Number of operating lease agreements | LeaseAgreement | 2 | |||
Restricted cash | $ 700,000 | $ 700,000 | $ 700,000 | |
Raw Material Purchases and Manufacturing Services | ||||
Commitments And Contingencies [Line Items] | ||||
Purchase commitments | 86,800,000 | 86,800,000 | ||
ALS Association and ALS Finding a Cure | ||||
Commitments And Contingencies [Line Items] | ||||
Accrued royalities grant agreements | 0 | 0 | ||
ALS Association and ALS Finding a Cure | Cost of Sales | ||||
Commitments And Contingencies [Line Items] | ||||
Royalty expense | 600,000 | 3,100,000 | ||
Royalty Payments Agreements with Grantors | ||||
Commitments And Contingencies [Line Items] | ||||
Proceeds from grants | $ 4,300,000 | |||
Royalty Payments Agreements with Grantors | ALS Association and ALS Finding a Cure | ||||
Commitments And Contingencies [Line Items] | ||||
Percentage of grant received | 150% | |||
Percentage of net sales of products developed under projects | 3% | |||
Percentage of cash proceeds from revenue generating transaction | 3% | |||
Royalty Payments Agreements with Grantors | Alzheimer's Drug Discovery Foundation, the Alzheimer's Association, and Cure Alzheimer's Fund | ||||
Commitments And Contingencies [Line Items] | ||||
Maximum Royalty amount payable to each grantor | $ 15,000,000 | |||
Aggregate royalty amount payable to grantor | $ 45,000,000 | |||
Percentage of annual net sales of products commercialized from project | 4% | |||
Percentage of all royalties and cash proceeds from revenue generating transaction | 15% | |||
Collateral for Letter of Credit Issued | ||||
Commitments And Contingencies [Line Items] | ||||
Restricted cash | 200,000 | 200,000 | ||
Collateral for Corporate Credit Card Program | ||||
Commitments And Contingencies [Line Items] | ||||
Restricted cash | $ 500,000 | $ 500,000 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Details) - USD ($) $ in Millions | Aug. 08, 2023 | Jul. 31, 2023 | Jun. 30, 2023 |
Raw Material Purchases and Manufacturing Services | |||
Subsequent Event [Line Items] | |||
Purchase commitments | $ 86.8 | ||
Subsequent Event | 2023 Inducement Plan | |||
Subsequent Event [Line Items] | |||
Shares reserved | 750,000 | ||
Subsequent Event | Raw Material Purchases and Manufacturing Services | |||
Subsequent Event [Line Items] | |||
Purchase commitments | $ 223.3 |