Cover Page
Cover Page - shares | 9 Months Ended | |
Sep. 30, 2023 | Oct. 27, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2023 | |
Document Transition Report | false | |
Entity File Number | 001-36014 | |
Entity Registrant Name | AGIOS PHARMACEUTICALS, INC. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 26-0662915 | |
Entity Address, Address Line One | 88 Sidney Street | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 617 | |
Local Phone Number | 649-8600 | |
Title of 12(b) Security | Common Stock, Par Value $0.001 per share | |
Trading Symbol | AGIO | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 55,891,284 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 | |
Amendment Flag | false | |
Entity Central Index Key | 0001439222 | |
Current Fiscal Year End Date | --12-31 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 64,333 | $ 139,259 |
Marketable securities | 583,155 | 643,860 |
Accounts receivable, net | 1,176 | 2,206 |
Inventory | 17,274 | 8,492 |
Prepaid expenses and other current assets | 38,414 | 38,955 |
Total current assets | 704,352 | 832,772 |
Marketable securities | 224,902 | 313,874 |
Operating lease assets | 57,162 | 65,129 |
Property and equipment, net | 16,785 | 22,987 |
Other non-current assets | 4,057 | 3,956 |
Total assets | 1,007,258 | 1,238,718 |
Current liabilities: | ||
Accounts payable | 13,271 | 18,616 |
Accrued expenses | 30,490 | 30,350 |
Operating lease liabilities | 14,664 | 13,663 |
Total current liabilities | 58,425 | 62,629 |
Operating lease liabilities, net of current portion | 60,834 | 71,996 |
Other non-current liabilities | 1,156 | 3,279 |
Total liabilities | 120,415 | 137,904 |
Stockholders’ equity: | ||
Preferred stock, $0.001 par value; 25,000,000 shares authorized; no shares issued or outstanding at September 30, 2023 and December 31, 2022 | 0 | 0 |
Common stock, $0.001 par value; 125,000,000 shares authorized; 72,100,384 shares issued and 55,883,973 shares outstanding at September 30, 2023, and 71,256,118 shares issued and 55,039,707 shares outstanding at December 31, 2022 | 72 | 71 |
Additional paid-in capital | 2,421,862 | 2,386,325 |
Accumulated other comprehensive loss | (5,896) | (12,535) |
Treasury stock, at cost (16,216,411 shares at September 30, 2023 and December 31, 2022) | (802,486) | (802,486) |
Accumulated deficit | (726,709) | (470,561) |
Total stockholders’ equity | 886,843 | 1,100,814 |
Total liabilities and stockholders’ equity | $ 1,007,258 | $ 1,238,718 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (parenthetical) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, authorized (in shares) | 25,000,000 | 25,000,000 |
Preferred stock, issued (in shares) | 0 | 0 |
Preferred stock, outstanding (in shares) | 0 | 0 |
Common stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Common stock, authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, issued (in shares) | 72,100,384 | 71,256,118 |
Common stock, outstanding (in shares) | 55,883,973 | 55,039,707 |
Treasury stock (in shares) | 16,216,411 | 16,216,411 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenues: | ||||
Total revenue | $ 7,399 | $ 3,516 | $ 19,720 | $ 9,930 |
Operating expenses: | ||||
Cost of sales | 633 | 517 | 2,295 | 1,291 |
Research and development | 81,841 | 64,966 | 218,037 | 209,612 |
Selling, general and administrative | 25,822 | 29,123 | 84,598 | 88,902 |
Total operating expenses | 108,296 | 94,606 | 304,930 | 299,805 |
Loss from operations | (100,897) | (91,090) | (285,210) | (289,875) |
Royalty income from gain on sale of oncology business | 0 | 4,443 | 0 | 9,851 |
Interest income, net | 8,375 | 3,818 | 24,720 | 6,305 |
Other income, net | 1,198 | 1,082 | 4,342 | 5,392 |
Net loss | $ (91,324) | $ (81,747) | $ (256,148) | $ (268,327) |
Net loss per share - basic (in usd per share) | $ (1.64) | $ (1.49) | $ (4.61) | $ (4.90) |
Net loss per share - diluted (in usd per share) | $ (1.64) | $ (1.49) | $ (4.61) | $ (4.90) |
Weighted-average number of common shares used in computing net loss per share - basic (in shares) | 55,803,663 | 54,844,579 | 55,559,766 | 54,734,301 |
Weighted-average number of common shares used in computing net loss per share – diluted (in shares) | 55,803,663 | 54,844,579 | 55,559,766 | 54,734,301 |
Product revenue, net | ||||
Revenues: | ||||
Total revenue | $ 7,399 | $ 3,516 | $ 19,720 | $ 7,430 |
Milestone revenue | ||||
Revenues: | ||||
Total revenue | $ 0 | $ 0 | $ 0 | $ 2,500 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (91,324) | $ (81,747) | $ (256,148) | $ (268,327) |
Other comprehensive income (loss) | ||||
Unrealized gain (loss) on available-for-sale securities | 2,974 | (4,581) | 6,639 | (13,885) |
Comprehensive loss | $ (88,350) | $ (86,328) | $ (249,509) | $ (282,212) |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive (Loss) Income | Accumulated Deficit | Treasury Stock |
Beginning balance (in shares) at Dec. 31, 2021 | 70,550,631,000 | |||||
Beginning balance at Dec. 31, 2021 | $ 1,291,975 | $ 71 | $ 2,334,348 | $ (1,198) | $ (238,760) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Dec. 31, 2021 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | (6,547) | (6,547) | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 442,646,000 | |||||
Common stock issued under stock incentive plan and ESPP | 1,289 | 1,289 | ||||
Stock-based compensation expense | 15,510 | 15,510 | ||||
Net loss | (94,774) | (94,774) | ||||
Ending balance (in shares) at Mar. 31, 2022 | 70,993,277,000 | |||||
Ending balance at Mar. 31, 2022 | 1,207,453 | $ 71 | 2,351,147 | (7,745) | (333,534) | $ (802,486) |
Treasury stock, ending balance (in shares) at Mar. 31, 2022 | (16,216,411,000) | |||||
Beginning balance (in shares) at Dec. 31, 2021 | 70,550,631,000 | |||||
Beginning balance at Dec. 31, 2021 | 1,291,975 | $ 71 | 2,334,348 | (1,198) | (238,760) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Dec. 31, 2021 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Net loss | (268,327) | |||||
Ending balance (in shares) at Sep. 30, 2022 | 71,110,442,000 | |||||
Ending balance at Sep. 30, 2022 | 1,050,170 | $ 71 | 2,374,755 | (15,083) | (507,087) | $ (802,486) |
Treasury stock, ending balance (in shares) at Sep. 30, 2022 | (16,216,411,000) | |||||
Beginning balance (in shares) at Mar. 31, 2022 | 70,993,277,000 | |||||
Beginning balance at Mar. 31, 2022 | 1,207,453 | $ 71 | 2,351,147 | (7,745) | (333,534) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Mar. 31, 2022 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | (2,757) | (2,757) | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 38,515,000 | |||||
Common stock issued under stock incentive plan and ESPP | 15 | 15 | ||||
Stock-based compensation expense | 11,165 | 11,165 | ||||
Net loss | (91,806) | (91,806) | ||||
Ending balance (in shares) at Jun. 30, 2022 | 71,031,792,000 | |||||
Ending balance at Jun. 30, 2022 | 1,124,070 | $ 71 | 2,362,327 | (10,502) | (425,340) | $ (802,486) |
Treasury stock, ending balance (in shares) at Jun. 30, 2022 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | (4,581) | (4,581) | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 78,650,000 | |||||
Common stock issued under stock incentive plan and ESPP | 1,272 | 1,272 | ||||
Stock-based compensation expense | 11,156 | 11,156 | ||||
Net loss | (81,747) | (81,747) | ||||
Ending balance (in shares) at Sep. 30, 2022 | 71,110,442,000 | |||||
Ending balance at Sep. 30, 2022 | $ 1,050,170 | $ 71 | 2,374,755 | (15,083) | (507,087) | $ (802,486) |
Treasury stock, ending balance (in shares) at Sep. 30, 2022 | (16,216,411,000) | |||||
Beginning balance (in shares) at Dec. 31, 2022 | 55,039,707 | 71,256,118,000 | ||||
Beginning balance at Dec. 31, 2022 | $ 1,100,814 | $ 71 | 2,386,325 | (12,535) | (470,561) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Dec. 31, 2022 | (16,216,411) | (16,216,411,000) | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | $ 4,124 | 4,124 | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 501,660,000 | |||||
Common stock issued under stock incentive plan and ESPP | 2,467 | $ 1 | 2,466 | |||
Stock-based compensation expense | 10,139 | 10,139 | ||||
Net loss | (81,018) | (81,018) | ||||
Ending balance (in shares) at Mar. 31, 2023 | 71,757,778,000 | |||||
Ending balance at Mar. 31, 2023 | $ 1,036,526 | $ 72 | 2,398,930 | (8,411) | (551,579) | $ (802,486) |
Treasury stock, ending balance (in shares) at Mar. 31, 2023 | (16,216,411,000) | |||||
Beginning balance (in shares) at Dec. 31, 2022 | 55,039,707 | 71,256,118,000 | ||||
Beginning balance at Dec. 31, 2022 | $ 1,100,814 | $ 71 | 2,386,325 | (12,535) | (470,561) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Dec. 31, 2022 | (16,216,411) | (16,216,411,000) | ||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 188,591 | |||||
Net loss | $ (256,148) | |||||
Ending balance (in shares) at Sep. 30, 2023 | 55,883,973 | 72,100,384,000 | ||||
Ending balance at Sep. 30, 2023 | $ 886,843 | $ 72 | 2,421,862 | (5,896) | (726,709) | $ (802,486) |
Treasury stock, ending balance (in shares) at Sep. 30, 2023 | (16,216,411) | (16,216,411,000) | ||||
Beginning balance (in shares) at Mar. 31, 2023 | 71,757,778,000 | |||||
Beginning balance at Mar. 31, 2023 | $ 1,036,526 | $ 72 | 2,398,930 | (8,411) | (551,579) | $ (802,486) |
Treasury stock, beginning balance (in shares) at Mar. 31, 2023 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | (459) | (459) | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 193,408,000 | |||||
Common stock issued under stock incentive plan and ESPP | 238 | 238 | ||||
Stock-based compensation expense | 11,737 | 11,737 | ||||
Net loss | (83,806) | (83,806) | ||||
Ending balance (in shares) at Jun. 30, 2023 | 71,951,186,000 | |||||
Ending balance at Jun. 30, 2023 | 964,236 | $ 72 | 2,410,905 | (8,870) | (635,385) | $ (802,486) |
Treasury stock, ending balance (in shares) at Jun. 30, 2023 | (16,216,411,000) | |||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Unrealized gain (loss) on available-for-sale securities | 2,974 | 2,974 | ||||
Common stock issued under stock incentive plan and ESPP (in shares) | 149,198,000 | |||||
Common stock issued under stock incentive plan and ESPP | 1,881 | 1,881 | ||||
Stock-based compensation expense | 9,076 | 9,076 | ||||
Net loss | $ (91,324) | (91,324) | ||||
Ending balance (in shares) at Sep. 30, 2023 | 55,883,973 | 72,100,384,000 | ||||
Ending balance at Sep. 30, 2023 | $ 886,843 | $ 72 | $ 2,421,862 | $ (5,896) | $ (726,709) | $ (802,486) |
Treasury stock, ending balance (in shares) at Sep. 30, 2023 | (16,216,411) | (16,216,411,000) |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Operating activities | ||
Net loss | $ (256,148) | $ (268,327) |
Adjustments to reconcile net loss from operations to net cash used in operating activities: | ||
Depreciation and amortization | 5,220 | 6,750 |
Stock-based compensation expense | 30,952 | 37,831 |
Net (accretion of discount) amortization of premium on marketable securities | (4,658) | 721 |
Loss on disposal of property and equipment | 278 | 29 |
Non-cash operating lease expense | 7,967 | 7,432 |
Expense associated with license agreement | 17,500 | 0 |
Realized gain on investments | (28) | 0 |
Changes in operating assets and liabilities: | ||
Accounts receivable, net | 1,030 | (1,818) |
Inventory | (8,782) | (5,176) |
Other receivables | 0 | 604 |
Prepaid expenses and other current and non-current assets | 440 | (7,655) |
Accounts payable | (5,201) | (4,628) |
Accrued expenses and other current liabilities | 140 | (2,211) |
Operating lease liabilities | (10,161) | (7,646) |
Other non-current liabilities | (2,123) | 779 |
Net cash used in operating activities | (223,574) | (243,315) |
Investing activities | ||
Purchases of marketable securities | (327,490) | (782,218) |
Proceeds from maturities and sales of marketable securities | 488,492 | 947,296 |
Payments associated with license agreement | (17,500) | 0 |
Purchases of property and equipment | (765) | (4,768) |
Proceeds from sale of equipment | 1,325 | 0 |
Net cash provided by investing activities | 144,062 | 160,310 |
Financing activities | ||
Payments on financing lease obligations | 0 | (248) |
Net proceeds from stock option exercises and employee stock purchase plan | 4,586 | 2,576 |
Net cash provided by financing activities | 4,586 | 2,328 |
Net change in cash and cash equivalents | (74,926) | (80,677) |
Cash and cash equivalents at beginning of the period | 139,259 | 203,126 |
Cash and cash equivalents at end of the period | 64,333 | 122,449 |
Supplemental disclosure of non-cash investing and financing transactions | ||
Additions to property and equipment in accounts payable and accrued expenses | 14 | 21 |
Net cash taxes paid | $ 1,586 | $ 1,940 |
Overview and Basis of Presentat
Overview and Basis of Presentation | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Overview and Basis of Presentation | Overview and Basis of Presentation References to Agios Throughout this Quarterly Report on Form 10-Q, “we,” “us,” and “our,” and similar expressions, except where the context requires otherwise, refer to Agios Pharmaceuticals, Inc. and its consolidated subsidiaries, and “our Board of Directors” refers to the board of directors of Agios Pharmaceuticals, Inc. Overview We are a biopharmaceutical company committed to transforming patients’ lives through leadership in the field of cellular metabolism, with the goal of creating differentiated medicines for rare diseases. With a history of focused study on cellular metabolism, we have a deep and mature understanding of this biology, which is involved in the healthy functioning of nearly every system in the body. Building on this expertise, these learnings can be rapidly applied to our clinical trials with the goal of developing medicines that can have a significant impact for patients. We accelerate the impact of our portfolio by cultivating connections with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver potential therapies for rare diseases. We are located in Cambridge, Massachusetts. The lead product candidate in our portfolio, PYRUKYND® (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. In February 2022, the U.S. Food and Drug Administration, or FDA, approved PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency in the United States. In November 2022, we received marketing authorization from the European Commission for PYRUKYND® for the treatment of PK deficiency in adult patients in the European Union, or EU. In December 2022, we received marketing authorization in Great Britain for PYRUKYND® for the treatment of PK deficiency in adult patients under the European Commission Decision Reliance Procedure. In addition, we are currently evaluating PYRUKYND® in clinical trials for the treatment of thalassemia, sickle cell disease, or SCD, and in pediatric patients with PK deficiency. We are also developing AG-946, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndrome, or LR MDS, and hemolytic anemias. In addition to the aforementioned development programs, we continue to invest in our late-stage research program focused on advancing a phenylalanine hydroxylase, or PAH, stabilizer for the treatment of phenylketonuria, or PKU. Also, in July 2023 we entered into a license agreement with Alnylam Pharmaceuticals, Inc., or Alnylam, for the development and commercialization of products containing or comprised of an siRNA development candidate discovered by Alnylam and targeting the transmembrane serine protease 6, or TMPRSS6, gene, and we intend to pursue development of a licensed product for the potential treatment of patients with polycythemia vera, or PV, a rare blood disorder. See below for more information on the license agreement with Alnylam. We are subject to risks common to companies in our industry including, but not limited to, uncertainties relating to conducting preclinical and clinical research and development, the manufacture and supply of products for clinical and commercial use, obtaining and maintaining regulatory approvals and pricing and reimbursement for our products, market acceptance, managing global growth and operating expenses, availability of additional capital, competition, obtaining and enforcing patents, stock price volatility, dependence on collaborative relationships and third-party service providers, dependence on key personnel, potential litigation, potential product liability claims and potential government investigations. Sale of our Oncology Business to Servier On March 31, 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier, which represented a discontinued operation. The transaction included the sale of our oncology business, including TIBSOVO®, our clinical-stage product candidates vorasidenib, AG-270 and AG-636, and our oncology research programs for a payment of approximately $1.8 billion in cash at the closing, subject to certain adjustments, and a payment of $200.0 million in cash, if, prior to January 1, 2027, vorasidenib is granted new drug application approval from the FDA with an approved label that permits vorasidenib’s use as a single agent for the adjuvant treatment of patients with Grade 2 glioma that have an isocitrate dehydrogenase 1 or 2 mutation (and, to the extent required by such approval, the vorasidenib companion diagnostic test is granted an FDA premarket approval), as well as a royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through loss of exclusivity, and a royalty of 15% of U.S. net sales of vorasidenib from the first commercial sale of vorasidenib through loss of exclusivity. Servier also acquired our co-commercialization rights for Bristol Myers Squibb’s IDHIFA® and the right to receive a $25.0 million potential milestone payment under our prior collaboration agreement with Celgene Corporation, Servier is responsible for conducting certain clinical development activities within the IDHIFA® development program. We recorded income from royalties of approximately $4.4 million and $9.9 million on U.S. net sales of TIBSOVO® by Servier in the royalty income from gain on sale of oncology business line item within the condensed consolidated statements of operations, for the three and nine months ended September 30, 2022, respectively. Sale of Contingent Payments The consideration for the sale of our oncology business to Servier included a royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through the loss of exclusivity, referred to as contingent payments. We recognized the contingent payments in the royalty income from gain on sale of oncology business line item in our consolidated statements of operations in the period when realizable. In October 2022, we sold our rights to future contingent payments to entities affiliated with Sagard Healthcare Partners, or Sagard, and recognized income of $127.9 million within the gain on sale of contingent payments line item in our consolidated statements of operations for the year ended December 31, 2022. We retain our rights to the potential milestone payment and royalties from Servier if vorasidenib is approved by the FDA. Alnylam License Agreement On July 28, 2023, Agios and Alnylam Pharmaceuticals, Inc., or Alnylam, entered into a license agreement under which Agios acquired the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with PV. Because the acquired assets do not meet the definition of a business in accordance with ASC 805, Business Combinations, Agios will account for the agreement as an asset acquisition. In accordance with the agreement, in the three months ended September 30, 2023, Agios made an up-front payment to Alnylam and recognized in-process research and development of $17.5 million which was recorded in research and development expense within our Consolidated Statements of Operations and classified as investing activities within our Consolidated Statements of Cash Flows. Agios will also pay Alnylam for certain expenses associated with the development of TMPRSS6 and these will be recorded in our Consolidated Statements of Operations as incurred. Additionally, Agios is responsible to pay up to $130.0 million in potential development and regulatory milestones, in addition to sales milestones as well as tiered royalties on annual net sales, if any, of licensed products, which may be subject to specified reductions and offsets. Basis of Presentation The condensed consolidated balance sheet as of September 30, 2023, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three and nine months ended September 30, 2023 and 2022, and the condensed consolidated statements of cash flows for the nine months ended September 30, 2023 and 2022 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of September 30, 2023, our results of operations and stockholders' equity for the three and nine months ended September 30, 2023 and 2022, and cash flows for the nine months ended September 30, 2023 and 2022. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three and nine-month periods are also unaudited. The results of operations for the three and nine months ended September 30, 2023 are not necessarily indicative of the results to be expected for the year ending December 31, 2023 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2022 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022 that was filed with the Securities and Exchange Commission, or SEC, on February 23, 2023. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP. Use of Estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the recent COVID-19 pandemic, or other pandemics or public health emergencies, may in the future directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain. We have made estimates of the impact of the recent COVID-19 pandemic within our financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates. Liquidity As of September 30, 2023, we had cash, cash equivalents and marketable securities of $872.4 million. Although we have incurred recurring losses and expect to continue to incur losses for the foreseeable future, we expect our cash, cash equivalents and marketable securities will be sufficient to fund current operations for at least the next twelve months from the issuance date of these financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies There have been no material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2022. Recent Accounting Pronouncements Accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption. |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements We record cash equivalents and marketable securities at fair value. Accounting Standards Codification, or ASC, 820, Fair Value Measurements and Disclosures , establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date. The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of September 30, 2023: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 5,187 $ — $ — $ 5,187 Total cash equivalents 5,187 — — 5,187 Marketable securities: U.S. Treasuries — 32,973 — 32,973 Government securities — 363,542 — 363,542 Corporate debt securities — 411,542 — 411,542 Total marketable securities — 808,057 — 808,057 Total cash equivalents and marketable securities $ 5,187 $ 808,057 $ — $ 813,244 Cash equivalents and marketable securities have been initially valued at the transaction price and are subsequently valued, at the end of each reporting period, utilizing third-party pricing services or other observable market data. The pricing services utilize industry standard valuation models, including both income and market-based approaches, and observable market inputs to determine value. After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of September 30, 2023. There have been no changes to the valuation methods during the nine months ended September 30, 2023, and we had no financial assets or liabilities that were classified as Level 3 at any point during the nine months ended September 30, 2023. |
Marketable Securities
Marketable Securities | 9 Months Ended |
Sep. 30, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | Marketable Securities Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities , and are recorded at fair value. Unrealized gains and losses are included as a component of accumulated other comprehensive loss in the condensed consolidated balance sheets and statements of stockholders’ equity and a component of total comprehensive loss in the condensed consolidated statements of comprehensive loss, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses , to determine if the impairment is credit-related or noncredit-related. Credit-related impairment is recognized as an allowance on the condensed consolidated balance sheets with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive income, net of taxes. Realized gains and losses are included in investment income on a specific-identification basis. There were no material realized gains or losses on marketable securities for the three and nine months ended September 30, 2023 or 2022. Marketable securities at September 30, 2023 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 33,214 $ — $ (241) $ 32,973 Government securities 293,501 — (2,136) 291,365 Corporate debt securities 260,658 — (1,841) 258,817 Total Current 587,373 — (4,218) 583,155 Non-current: U.S. Treasuries — — — — Government securities 72,630 — (453) 72,177 Corporate debt securities 153,950 — (1,225) 152,725 Total Non-current 226,580 — (1,678) 224,902 Total marketable securities $ 813,953 $ — $ (5,896) $ 808,057 Marketable securities at December 31, 2022 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 68,175 $ 3 $ (811) $ 67,367 Government securities 220,901 8 (5,289) 215,620 Corporate debt securities 363,263 1 (2,391) 360,873 Total Current 652,339 12 (8,491) 643,860 Non-current: U.S. Treasuries 17,418 4 (193) 17,229 Government securities 117,475 7 (1,659) 115,823 Corporate debt securities 183,037 76 (2,291) 180,822 Total Non-current 317,930 87 (4,143) 313,874 Total marketable securities $ 970,269 $ 99 $ (12,634) $ 957,734 As of September 30, 2023 and December 31, 2022, we held both current and non-current investments. Investments classified as current have maturities of less than one year. Investments classified as non-current are those that: (i) have a maturity of greater than one year, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and, therefore, are classified as available-for-sale. As of September 30, 2023 and December 31, 2022, we held 233 and 259 debt securities, respectively, that were in an unrealized loss position for less than one year. We did not record an allowance for credit losses as of September 30, 2023 and December 31, 2022 related to these securities. The aggregate fair value of debt securities in an unrealized loss position at September 30, 2023 and December 31, 2022 was $808.1 million and $868.2 million, respectively. There were no individual securities that were in a significant unrealized loss position as of September 30, 2023 and December 31, 2022. We regularly review the securities in an unrealized loss position and evaluate the current expected credit loss by considering factors such as historical experience, market data, issuer-specific factors, and current economic conditions. We do not consider these marketable securities to be impaired as of September 30, 2023 and December 31, 2022. |
Inventory
Inventory | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Inventory | Inventory Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following: (In thousands) September 30, December 31, Raw materials $ 470 $ — Work-in-process 15,743 7,550 Finished goods 1,061 942 Total inventory $ 17,274 $ 8,492 |
Leases
Leases | 9 Months Ended |
Sep. 30, 2023 | |
Leases [Abstract] | |
Leases | Leases Our building leases are comprised of office and laboratory space under non-cancelable operating leases. These lease agreements have remaining lease terms of approximately four years and contain various clauses for renewal at our option. The renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options are not reasonably certain of being exercised. The lease agreements do not contain residual value guarantees. The components of lease expense and other information related to leases were as follows: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Operating lease costs $ 3,807 $ 3,807 $ 11,420 $ 11,420 Cash paid for amounts included in the measurement of operating lease liabilities $ 4,550 $ 4,420 $ 13,614 $ 12,610 We have not entered into any material short-term leases or financing leases as of September 30, 2023. In arriving at the operating lease liabilities as of September 30, 2023 and December 31, 2022, we applied the weighted-average incremental borrowing rate of 5.7% for both periods over a weighted-average remaining lease term of 4.4 and 5.2 years, respectively. As of September 30, 2023, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter were as follows: (In thousands) Remaining 2023 $ 3,037 2024 18,660 2025 19,507 2026 20,151 2027 20,755 2028 3,479 Thereafter — Undiscounted minimum rental commitments $ 85,589 Interest (10,091) Operating lease liabilities $ 75,498 We provided our landlord a security deposit of $2.9 million as security for our leases, which is included within other non-current assets on our condensed consolidated balance sheet. In August 2021, we entered into a long-term sublease agreement for 13,000 square feet of the office space at 38 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through December 2024. In April 2022, we entered into a long-term sublease agreement for 27,000 square feet of the office space at 64 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through April 2025. In May 2023, we entered into a long-term sublease agreement for 7,407 square feet of office space on the first floor of 64 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through April 2025. We recorded operating sublease income of $1.7 million and $1.0 million for the three months ended September 30, 2023 and 2022, respectively, and $4.5 million and $2.7 million for the nine months ended September 30, 2023 and 2022, respectively, in other income, net in the condensed consolidated statements of operations. We received security deposits from our sublessees of approximately $1.2 million which is recorded within other non-current assets on our condensed consolidated balance sheet. As of September 30, 2023, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2023 $ 1,247 2024 5,078 2025 1,310 Total $ 7,635 |
Accrued Expenses
Accrued Expenses | 9 Months Ended |
Sep. 30, 2023 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses Accrued expenses consisted of the following: (In thousands) September 30, December 31, Accrued compensation $ 14,022 $ 18,105 Accrued research and development costs 12,528 8,425 Accrued professional fees 1,369 2,435 Accrued other 2,571 1,385 Total accrued expenses $ 30,490 $ 30,350 |
Product Revenue
Product Revenue | 9 Months Ended |
Sep. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Product Revenue | Product Revenue We sell PYRUKYND®, our wholly owned product, to a limited number of specialty distributors and specialty pharmacy providers, or collectively, the Customers. The Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®. The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer. Product revenue, net, were as follows: Three Months Ended September 30, Nine Months Ended September 30, (In thousands) 2023 2022 2023 2022 Product revenue, net $ 7,399 $ 3,516 $ 19,720 $ 7,430 Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products. Contractual Adjustments We generally provide Customers with discounts, including prompt pay discounts, and allowances that are explicitly stated in the contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from certain Customers. Chargebacks and discounts represent the estimated obligations 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 us. Customers charge us for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are estimated using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated channel mix and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Government Rebates Government rebates include Medicare, TriCare, and Medicaid rebates, which we estimate using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. Returns / Replacement We estimate the amount of product sales that may be returned by Customers or replaced by Agios and record this estimate as a reduction of revenue in the period the related product revenue is recognized. We currently estimate product return and replacement liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the nine months ended September 30, 2023: (In thousands) Contractual Adjustments Government Rebates Returns/ Replacement Total Balance at December 31, 2022 $ 65 $ 573 $ 133 $ 771 Current provisions relating to sales in the current year 919 1,631 2,147 4,697 Adjustments relating to prior years — (7) — (7) Payments/returns relating to sales in the current year (793) (495) (1,958) (3,246) Payments/returns relating to sales in the prior years (49) (306) (48) (403) Balance at September 30, 2023 $ 142 $ 1,396 $ 274 $ 1,812 Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows: (In thousands) September 30, 2023 December 31, 2022 Reduction of accounts receivable $ 137 $ 60 Component of accrued expenses 1,675 711 Total revenue-related reserves $ 1,812 $ 771 The following table presents changes in our contract assets during the nine months ended September 30, 2023: (In thousands) December 31, 2022 Additions Deductions September 30, 2023 Contract assets (1) Accounts receivable, net $ 2,206 $ 24,411 $ (25,441) $ 1,176 |
Share-Based Payments
Share-Based Payments | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Share-Based Payments | Share-Based Payments 2023 Stock Incentive Plan and Inducement Grants In June 2023, our stockholders approved the 2023 Stock Incentive Plan, or the 2023 Plan. The 2023 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based share units, or PSUs, and other stock-based awards to employees, advisors, consultants and non-employee directors. Following the adoption of the 2023 Plan, we ceased granting equity awards under the 2013 Stock Incentive Plan, or the 2013 Plan. Any outstanding equity awards that were previously granted under the 2013 Plan continue to be governed by their terms. Following adoption of the 2013 Plan, we ceased granting equity awards under the 2007 Stock Incentive Plan, or the 2007 Plan. There are no outstanding equity awards under the 2007 Plan. In connection with the start of employment of our Chief Executive Officer and Chief Financial Officer in 2022, and our Chief Commercial Officer in 2023, our board of directors granted each of them equity awards in the form of stock options, RSUs and PSUs, which awards were made outside our equity incentive plans as inducements material to their respective entry into employment with us in accordance with Nasdaq Listing Rule 5635(c)(4). As of September 30, 2023, the maximum number of shares reserved under the 2007 Plan, the 2013 Plan, the 2023 Plan and the inducement grants described above was 12,065,656, and we had 4,762,423 shares available for future issuance under the 2023 Plan. Stock options The following table presents stock option activity for the nine months ended September 30, 2023: Number of Weighted-Average Exercise Price Outstanding at December 31, 2022 5,772,564 $ 48.81 Granted 870,924 25.88 Exercised (188,591) 11.52 Forfeited/Expired (912,479) 55.49 Outstanding at September 30, 2023 5,542,418 $ 45.37 Exercisable at September 30, 2023 3,485,110 $ 54.14 Vested and expected to vest at September 30, 2023 5,542,418 $ 45.37 At September 30, 2023, there was approximately $31.9 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.55 years. Restricted stock units The following table presents RSU activity for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2022 1,117,921 $ 38.30 Granted 899,072 25.97 Vested (450,586) 41.42 Forfeited (210,420) 33.46 Unvested shares at September 30, 2023 1,355,987 $ 29.84 As of September 30, 2023, there was approximately $27.5 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 1.87 years. Performance-based stock units The following table presents PSU activity for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2022 430,243 $ 35.87 Granted 125,897 25.23 Vested (92,257) 30.18 Forfeited (101,750) 46.39 Unvested shares at September 30, 2023 362,133 $ 30.66 Stock-based compensation expense associated with these PSUs is recognized if the underlying performance condition is considered probable of achievement using our management’s best estimates. As of September 30, 2023, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $11.1 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement. Market-based stock units The following table presents market-based stock unit, or MSU, activity for the nine months ended September 30, 2023: Number of Weighted-Average Unvested shares at December 31, 2022 42,695 $ 41.50 Granted — — Unvested shares at September 30, 2023 42,695 $ 41.50 The fair value of MSUs are estimated using a Monte Carlo simulation model. Assumptions and estimates utilized in the model include the risk-free interest rate, dividend yield, expected stock volatility and the estimated period to achievement of the market condition. As of September 30, 2023, there was no remaining unrecognized compensation expense related to MSUs. 2013 Employee Stock Purchase Plan In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 Employee Stock Purchase Plan, or the 2013 ESPP. We issued and sold 112,832 and 104,867 shares of common stock during the nine months ended September 30, 2023 and 2022, respectively, under the 2013 ESPP. The 2013 ESPP provides participating employees with the opportunity to purchase up to an aggregate of 2,363,636 shares of our common stock. As of September 30, 2023, we had 1,686,039 shares of common stock available for future issuance under the 2013 ESPP. Stock-based compensation expense Stock-based compensation expense by award type included within the condensed consolidated statements of operations is as follows: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Stock options $ 4,171 $ 5,864 $ 13,065 $ 17,720 Restricted stock units 4,735 5,115 14,436 16,512 Performance-based stock units — — 2,784 2,919 Employee stock purchase plan 170 177 667 680 Total stock-based compensation expense $ 9,076 $ 11,156 $ 30,952 $ 37,831 Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Research and development expense $ 3,635 $ 4,628 $ 12,530 $ 16,207 Selling, general and administrative expense 5,441 6,528 18,422 21,624 Total stock-based compensation expense $ 9,076 $ 11,156 $ 30,952 $ 37,831 |
Loss per Share
Loss per Share | 9 Months Ended |
Sep. 30, 2023 | |
Earnings Per Share [Abstract] | |
Loss per Share | Loss per Share Basic net loss per share is calculated by dividing net loss by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of September 30, 2023 are not considered to be common stock equivalents. We utilize the control number concept in the computation of diluted earnings per share to determine whether potential common stock equivalents are dilutive. The control number used is net loss from continuing operations. The control number concept requires that the same number of potentially dilutive securities applied in computing diluted earnings per share from continuing operations be applied to all other categories of income or loss, regardless of their anti-dilutive effect on such categories. Since we had a net loss for all periods presented, no dilutive effect has been recognized in the calculation of loss per share. Basic and diluted net loss per share was the same for all periods presented. The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three and Nine Months Ended September 30, 2023 2022 Stock options 5,542,418 5,909,087 Restricted stock units 1,355,987 1,264,377 Employee stock purchase plan shares 11,383 10,503 Total common stock equivalents 6,909,788 7,183,967 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2023 | Jun. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Pay vs Performance Disclosure | ||||||||
Net loss | $ (91,324) | $ (83,806) | $ (81,018) | $ (81,747) | $ (91,806) | $ (94,774) | $ (256,148) | $ (268,327) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended | 9 Months Ended |
Sep. 30, 2023 | Sep. 30, 2023 | |
Trading Arrangements, by Individual | ||
Material Terms of Trading Arrangement | (c) Director and Officer Trading Arrangements A significant portion of the compensation of our directors and officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, is in the form of equity awards and, from time to time, directors and officers engage in open-market transactions with respect to the securities acquired pursuant to such equity awards or other of our securities, including to satisfy tax withholding obligations when equity awards vest or are exercised, and for diversification or other personal reasons. Transactions in our securities by directors and officers are required to be made in accordance with our insider trading policy, which requires that the transactions be in accordance with applicable U.S. federal securities laws that prohibit trading while in possession of material nonpublic information. Rule 10b5-1 under the Exchange Act provides an affirmative defense that enables directors and officers to prearrange transactions in our securities in a manner that avoids concerns about initiating transactions while in possession of material nonpublic information. During the quarterly period covered by this report, Sarah Gheuens, M.D., Ph.D., our chief medical officer and head of research & development, entered into a Rule 10b5-1 trading arrangement that is intended to qualify as an “eligible sell-to-cover transaction” (as described in Rule 10b5-1(c)(1)(ii)(D)(3) under the Exchange Act). This sell-to-cover arrangement applies to restricted stock units, or RSUs, whether vesting is based on the passage of time and/or the achievement of performance goals, granted to Dr. Gheuens on July 1, 2023 and any RSUs that may be granted to her from time to time thereafter (other than those which by their terms require us to withhold shares for tax withholding obligations in connection with vesting and settlement). This arrangement provides for the automatic sale of shares of our common stock that would otherwise be issuable on each settlement date of a covered RSU in an amount necessary to satisfy the applicable withholding obligation, with the proceeds of the sale delivered to us in satisfaction of the applicable withholding obligation. The number of shares that will be sold under this arrangement is not currently determinable as the number will vary based on the extent to which vesting conditions are satisfied, the market price of our common stock at the time of settlement and the potential future grant of additional RSUs subject to this arrangement. None of our directors or officers terminated a Rule 10b5-1 trading arrangement or adopted or terminated a non-Rule 10b5-1 trading arrangement (as defined in Item 408(c) of Regulation S-K) during the quarterly period covered by this report. | |
Non-Rule 10b5-1 Arrangement Adopted | false | |
Rule 10b5-1 Arrangement Terminated | false | |
Non-Rule 10b5-1 Arrangement Terminated | false | |
Sarah Gheuens [Member] | ||
Trading Arrangements, by Individual | ||
Name | Sarah Gheuens | |
Title | chief medical officer and head of research & development | |
Rule 10b5-1 Arrangement Adopted | true |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The condensed consolidated balance sheet as of September 30, 2023, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three and nine months ended September 30, 2023 and 2022, and the condensed consolidated statements of cash flows for the nine months ended September 30, 2023 and 2022 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of September 30, 2023, our results of operations and stockholders' equity for the three and nine months ended September 30, 2023 and 2022, and cash flows for the nine months ended September 30, 2023 and 2022. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three and nine-month periods are also unaudited. The results of operations for the three and nine months ended September 30, 2023 are not necessarily indicative of the results to be expected for the year ending December 31, 2023 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2022 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022 that was filed with the Securities and Exchange Commission, or SEC, on February 23, 2023. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP. |
Use of Estimates | Use of Estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the recent COVID-19 pandemic, or other pandemics or public health emergencies, may in the future directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are |
Recent Accounting Pronouncements | Recent Accounting Pronouncements Accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption. |
Fair Value Measurements | We record cash equivalents and marketable securities at fair value. Accounting Standards Codification, or ASC, 820, Fair Value Measurements and Disclosures , establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date. |
Marketable Securities | Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities , and are recorded at fair value. Unrealized gains and losses are included as a component of accumulated other comprehensive loss in the condensed consolidated balance sheets and statements of stockholders’ equity and a component of total comprehensive loss in the condensed consolidated statements of comprehensive loss, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses |
Loss per Share | Basic net loss per share is calculated by dividing net loss by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of September 30, 2023 are not considered to be common stock equivalents. |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Summary of Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis | The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of September 30, 2023: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 5,187 $ — $ — $ 5,187 Total cash equivalents 5,187 — — 5,187 Marketable securities: U.S. Treasuries — 32,973 — 32,973 Government securities — 363,542 — 363,542 Corporate debt securities — 411,542 — 411,542 Total marketable securities — 808,057 — 808,057 Total cash equivalents and marketable securities $ 5,187 $ 808,057 $ — $ 813,244 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Marketable Securities | Marketable securities at September 30, 2023 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 33,214 $ — $ (241) $ 32,973 Government securities 293,501 — (2,136) 291,365 Corporate debt securities 260,658 — (1,841) 258,817 Total Current 587,373 — (4,218) 583,155 Non-current: U.S. Treasuries — — — — Government securities 72,630 — (453) 72,177 Corporate debt securities 153,950 — (1,225) 152,725 Total Non-current 226,580 — (1,678) 224,902 Total marketable securities $ 813,953 $ — $ (5,896) $ 808,057 Marketable securities at December 31, 2022 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 68,175 $ 3 $ (811) $ 67,367 Government securities 220,901 8 (5,289) 215,620 Corporate debt securities 363,263 1 (2,391) 360,873 Total Current 652,339 12 (8,491) 643,860 Non-current: U.S. Treasuries 17,418 4 (193) 17,229 Government securities 117,475 7 (1,659) 115,823 Corporate debt securities 183,037 76 (2,291) 180,822 Total Non-current 317,930 87 (4,143) 313,874 Total marketable securities $ 970,269 $ 99 $ (12,634) $ 957,734 |
Inventory (Tables)
Inventory (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following: (In thousands) September 30, December 31, Raw materials $ 470 $ — Work-in-process 15,743 7,550 Finished goods 1,061 942 Total inventory $ 17,274 $ 8,492 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Leases [Abstract] | |
Schedule of Lease Expense and Other Information Related to Leases | The components of lease expense and other information related to leases were as follows: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Operating lease costs $ 3,807 $ 3,807 $ 11,420 $ 11,420 Cash paid for amounts included in the measurement of operating lease liabilities $ 4,550 $ 4,420 $ 13,614 $ 12,610 |
Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases | As of September 30, 2023, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter were as follows: (In thousands) Remaining 2023 $ 3,037 2024 18,660 2025 19,507 2026 20,151 2027 20,755 2028 3,479 Thereafter — Undiscounted minimum rental commitments $ 85,589 Interest (10,091) Operating lease liabilities $ 75,498 |
Schedule of Future Minimum Lease Payments to be Received under the Long-term Sublease Agreements | As of September 30, 2023, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2023 $ 1,247 2024 5,078 2025 1,310 Total $ 7,635 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: (In thousands) September 30, December 31, Accrued compensation $ 14,022 $ 18,105 Accrued research and development costs 12,528 8,425 Accrued professional fees 1,369 2,435 Accrued other 2,571 1,385 Total accrued expenses $ 30,490 $ 30,350 |
Product Revenue (Tables)
Product Revenue (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Schedule of Product Revenue, Net | Product revenue, net, were as follows: Three Months Ended September 30, Nine Months Ended September 30, (In thousands) 2023 2022 2023 2022 Product revenue, net $ 7,399 $ 3,516 $ 19,720 $ 7,430 |
Schedule of Product Revenue Allowance and Reserve | The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the nine months ended September 30, 2023: (In thousands) Contractual Adjustments Government Rebates Returns/ Replacement Total Balance at December 31, 2022 $ 65 $ 573 $ 133 $ 771 Current provisions relating to sales in the current year 919 1,631 2,147 4,697 Adjustments relating to prior years — (7) — (7) Payments/returns relating to sales in the current year (793) (495) (1,958) (3,246) Payments/returns relating to sales in the prior years (49) (306) (48) (403) Balance at September 30, 2023 $ 142 $ 1,396 $ 274 $ 1,812 |
Schedule of Revenue-related Reserves | Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows: (In thousands) September 30, 2023 December 31, 2022 Reduction of accounts receivable $ 137 $ 60 Component of accrued expenses 1,675 711 Total revenue-related reserves $ 1,812 $ 771 |
Schedule of Changes in Contract Assets | The following table presents changes in our contract assets during the nine months ended September 30, 2023: (In thousands) December 31, 2022 Additions Deductions September 30, 2023 Contract assets (1) Accounts receivable, net $ 2,206 $ 24,411 $ (25,441) $ 1,176 |
Share-Based Payments (Tables)
Share-Based Payments (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Schedule of Company's Stock Option Activity | The following table presents stock option activity for the nine months ended September 30, 2023: Number of Weighted-Average Exercise Price Outstanding at December 31, 2022 5,772,564 $ 48.81 Granted 870,924 25.88 Exercised (188,591) 11.52 Forfeited/Expired (912,479) 55.49 Outstanding at September 30, 2023 5,542,418 $ 45.37 Exercisable at September 30, 2023 3,485,110 $ 54.14 Vested and expected to vest at September 30, 2023 5,542,418 $ 45.37 |
Schedule of RSU Activity | The following table presents RSU activity for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2022 1,117,921 $ 38.30 Granted 899,072 25.97 Vested (450,586) 41.42 Forfeited (210,420) 33.46 Unvested shares at September 30, 2023 1,355,987 $ 29.84 |
Schedule of PSU Activity | The following table presents PSU activity for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2022 430,243 $ 35.87 Granted 125,897 25.23 Vested (92,257) 30.18 Forfeited (101,750) 46.39 Unvested shares at September 30, 2023 362,133 $ 30.66 |
Schedule of Market-based Stock Unit or MSU Activity | The following table presents market-based stock unit, or MSU, activity for the nine months ended September 30, 2023: Number of Weighted-Average Unvested shares at December 31, 2022 42,695 $ 41.50 Granted — — Unvested shares at September 30, 2023 42,695 $ 41.50 |
Schedule of Stock-Based Compensation Expense by Award Type Included within the Condensed Consolidated Statements of Operations | Stock-based compensation expense by award type included within the condensed consolidated statements of operations is as follows: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Stock options $ 4,171 $ 5,864 $ 13,065 $ 17,720 Restricted stock units 4,735 5,115 14,436 16,512 Performance-based stock units — — 2,784 2,919 Employee stock purchase plan 170 177 667 680 Total stock-based compensation expense $ 9,076 $ 11,156 $ 30,952 $ 37,831 |
Schedule of Expenses Related to Stock Options and Stock-based Awards | Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations: Three Months Ended Nine Months Ended (In thousands) 2023 2022 2023 2022 Research and development expense $ 3,635 $ 4,628 $ 12,530 $ 16,207 Selling, general and administrative expense 5,441 6,528 18,422 21,624 Total stock-based compensation expense $ 9,076 $ 11,156 $ 30,952 $ 37,831 |
Loss per Share (Tables)
Loss per Share (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Earnings Per Share [Abstract] | |
Schedule of Common Stock Equivalents were Excluded from Calculation of Diluted Net Loss Per Share | The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three and Nine Months Ended September 30, 2023 2022 Stock options 5,542,418 5,909,087 Restricted stock units 1,355,987 1,264,377 Employee stock purchase plan shares 11,383 10,503 Total common stock equivalents 6,909,788 7,183,967 |
Overview and Basis of Present_2
Overview and Basis of Presentation (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | |||
Mar. 31, 2021 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Gain on sale of contingent payments | $ 127,900 | |||||
Payments associated with license agreement | $ 17,500 | $ 0 | ||||
Cash, cash equivalents, and marketable securities | $ 872,400 | $ 872,400 | ||||
Alnylam Pharmaceuticals, Inc. | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Payments associated with license agreement | 17,500 | |||||
Potential development milestone payments, maximum | $ 130,000 | |||||
2010 Agreement | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Milestone payment for achievement of specified ex-U.S. commercial milestone event | $ 25,000 | |||||
TIBSOVO | UNITED STATES | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Earn-out revenue for royalties | $ 4,400 | $ 9,900 | ||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Cash proceeds | 1,800,000 | |||||
Contingent milestone payment | $ 200,000 | |||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | TIBSOVO | UNITED STATES | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Contingent royalty payment | 5% | |||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | Vorasidenib | UNITED STATES | ||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||
Contingent royalty payment | 15% |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | $ 808,057 | $ 957,734 |
Fair Value, Measurements, Recurring | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 5,187 | |
Total marketable securities | 808,057 | |
Total cash equivalents and marketable securities | 813,244 | |
Fair Value, Measurements, Recurring | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 5,187 | |
Fair Value, Measurements, Recurring | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 32,973 | |
Fair Value, Measurements, Recurring | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 363,542 | |
Fair Value, Measurements, Recurring | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 411,542 | |
Fair Value, Measurements, Recurring | Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 5,187 | |
Total marketable securities | 0 | |
Total cash equivalents and marketable securities | 5,187 | |
Fair Value, Measurements, Recurring | Level 1 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 5,187 | |
Fair Value, Measurements, Recurring | Level 1 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 1 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 1 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Total marketable securities | 808,057 | |
Total cash equivalents and marketable securities | 808,057 | |
Fair Value, Measurements, Recurring | Level 2 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Fair Value, Measurements, Recurring | Level 2 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 32,973 | |
Fair Value, Measurements, Recurring | Level 2 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 363,542 | |
Fair Value, Measurements, Recurring | Level 2 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 411,542 | |
Fair Value, Measurements, Recurring | Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Total marketable securities | 0 | |
Total cash equivalents and marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Fair Value, Measurements, Recurring | Level 3 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) | Sep. 30, 2023 USD ($) |
Fair Value, Measurements, Recurring | Level 3 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |
Fair value of assets (liabilities) | $ 0 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Details) | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2023 USD ($) security | Sep. 30, 2022 USD ($) | Sep. 30, 2023 USD ($) security | Sep. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) security | |
Investments, Debt and Equity Securities [Abstract] | |||||
Realized gain (loss) on marketable securities | $ 0 | $ 0 | $ 0 | $ 0 | |
Number of debt securities in unrealized loss position for less than one year | security | 233 | 233 | 259 | ||
Allowance for credit losses | $ 0 | $ 0 | $ 0 | ||
Aggregate fair value of debt securities in unrealized loss position | $ 808,100,000 | $ 808,100,000 | $ 868,200,000 | ||
Number of debt securities in significant unrealized loss position | security | 0 | 0 | 0 |
Marketable Securities - Schedul
Marketable Securities - Schedule of Marketable Securities (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 813,953 | $ 970,269 |
Unrealized Gains | 0 | 99 |
Unrealized Losses | (5,896) | (12,634) |
Fair Value | 808,057 | 957,734 |
Current: | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 587,373 | 652,339 |
Unrealized Gains | 0 | 12 |
Unrealized Losses | (4,218) | (8,491) |
Fair Value | 583,155 | 643,860 |
Current: | U.S. Treasuries | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 33,214 | 68,175 |
Unrealized Gains | 0 | 3 |
Unrealized Losses | (241) | (811) |
Fair Value | 32,973 | 67,367 |
Current: | Government securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 293,501 | 220,901 |
Unrealized Gains | 0 | 8 |
Unrealized Losses | (2,136) | (5,289) |
Fair Value | 291,365 | 215,620 |
Current: | Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 260,658 | 363,263 |
Unrealized Gains | 0 | 1 |
Unrealized Losses | (1,841) | (2,391) |
Fair Value | 258,817 | 360,873 |
Non-current: | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 226,580 | 317,930 |
Unrealized Gains | 0 | 87 |
Unrealized Losses | (1,678) | (4,143) |
Fair Value | 224,902 | 313,874 |
Non-current: | U.S. Treasuries | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 0 | 17,418 |
Unrealized Gains | 0 | 4 |
Unrealized Losses | 0 | (193) |
Fair Value | 0 | 17,229 |
Non-current: | Government securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 72,630 | 117,475 |
Unrealized Gains | 0 | 7 |
Unrealized Losses | (453) | (1,659) |
Fair Value | 72,177 | 115,823 |
Non-current: | Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 153,950 | 183,037 |
Unrealized Gains | 0 | 76 |
Unrealized Losses | (1,225) | (2,291) |
Fair Value | $ 152,725 | $ 180,822 |
Inventory (Details)
Inventory (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 470 | $ 0 |
Work-in-process | 15,743 | 7,550 |
Finished goods | 1,061 | 942 |
Total inventory | $ 17,274 | $ 8,492 |
Leases - Additional Information
Leases - Additional Information (Details) $ in Millions | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | May 31, 2023 ft² | Dec. 31, 2022 | Apr. 30, 2022 ft² | Aug. 31, 2021 ft² | |
Lessor, Lease, Description [Line Items] | ||||||||
Remaining lease terms | 4 years | 4 years | ||||||
Weighted-average incremental borrowing rate | 5.70% | 5.70% | 5.70% | |||||
Weighted-average remaining lease term | 4 years 4 months 24 days | 4 years 4 months 24 days | 5 years 2 months 12 days | |||||
Sublease income | $ 1.7 | $ 1 | $ 4.5 | $ 2.7 | ||||
Thirty-Eight Sydney Street Lease | ||||||||
Lessor, Lease, Description [Line Items] | ||||||||
Area of premises subleased (in square feet) | ft² | 13,000 | |||||||
Sixty-Four Sydney Street Lease | ||||||||
Lessor, Lease, Description [Line Items] | ||||||||
Area of premises subleased (in square feet) | ft² | 7,407 | 27,000 | ||||||
Other Noncurrent Assets | ||||||||
Lessor, Lease, Description [Line Items] | ||||||||
Standby letter of credit | 2.9 | 2.9 | ||||||
Security deposit liability | $ 1.2 | $ 1.2 |
Leases - Schedule of Lease Expe
Leases - Schedule of Lease Expense and Other Information Related to Leases (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Leases [Abstract] | ||||
Operating lease costs | $ 3,807 | $ 3,807 | $ 11,420 | $ 11,420 |
Cash paid for amounts included in the measurement of operating lease liabilities | $ 4,550 | $ 4,420 | $ 13,614 | $ 12,610 |
Leases - Schedule of Undiscount
Leases - Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases (Details) $ in Thousands | Sep. 30, 2023 USD ($) |
Leases [Abstract] | |
Remaining 2023 | $ 3,037 |
2024 | 18,660 |
2025 | 19,507 |
2026 | 20,151 |
2027 | 20,755 |
2028 | 3,479 |
Thereafter | 0 |
Undiscounted minimum rental commitments | 85,589 |
Interest | (10,091) |
Operating lease liabilities | $ 75,498 |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Lease Payments to be Received under the Long-term Sublease Agreements (Details) $ in Thousands | Sep. 30, 2023 USD ($) |
Leases [Abstract] | |
Remaining 2023 | $ 1,247 |
2024 | 5,078 |
2025 | 1,310 |
Total | $ 7,635 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Accrued compensation | $ 14,022 | $ 18,105 |
Accrued research and development costs | 12,528 | 8,425 |
Accrued professional fees | 1,369 | 2,435 |
Accrued other | 2,571 | 1,385 |
Total accrued expenses | $ 30,490 | $ 30,350 |
Product Revenue - Schedule of P
Product Revenue - Schedule of Product Revenue, Net (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Disaggregation of Revenue [Line Items] | ||||
Product revenue, net | $ 7,399 | $ 3,516 | $ 19,720 | $ 9,930 |
Product revenue, net | ||||
Disaggregation of Revenue [Line Items] | ||||
Product revenue, net | $ 7,399 | $ 3,516 | $ 19,720 | $ 7,430 |
Product Revenue - Schedule of_2
Product Revenue - Schedule of Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Dec. 31, 2022 | |
Contractual Adjustments | ||
Contractual adjustments, beginning balance | $ 65 | |
Contractual adjustments, current provisions relating to sales in the current year | 919 | |
Contractual adjustments, adjustments relating to prior year | 0 | |
Contractual adjustments, payments/returns relating to sales in the current year | (793) | |
Contractual adjustments, payments/returns relating to sales in the prior year | (49) | |
Contractual adjustments, ending balance | 142 | |
Government Rebates | ||
Government rebates, beginning balance | 573 | |
Government rebates, current provisions relating to sales in the current year | 1,631 | |
Government rebates, adjustments relating to prior years | (7) | |
Government rebates, payments/returns relating to sales in the current year | (495) | |
Government rebates, payments/returns relating to sales in the prior years | (306) | |
Government rebates, ending balance | 1,396 | |
Returns/ Replacement | ||
Returns/ replacement, beginning balance | 133 | |
Returns/ replacement, current provisions relating to sales in the current year | 2,147 | |
Returns/ replacement, adjustments relating to prior years | 0 | |
Returns/ replacement, payments/returns relating to sales in the current year | (1,958) | |
Returns/ replacement, payments/returns relating to sales in the prior years | (48) | |
Returns/ replacement, ending balance | 274 | |
Total | ||
Total revenue-related reserves | 1,812 | $ 771 |
Total, current provisions relating to sales in the current year | 4,697 | |
Total, adjustments relating to prior years | (7) | |
Total, payments/returns relating to sales in the current year | (3,246) | |
Total, payments/returns relating to sales in the prior years | $ (403) |
Product Revenue - Schedule of R
Product Revenue - Schedule of Revenue-related Reserves (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Revenue from Contract with Customer [Abstract] | ||
Reduction of accounts receivable | $ 137 | $ 60 |
Component of accrued expenses | 1,675 | 711 |
Total revenue-related reserves | $ 1,812 | $ 771 |
Product Revenue - Schedule of C
Product Revenue - Schedule of Changes in Contract Assets (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Contract assets | |
Contract assets, beginning balance | $ 2,206 |
Additions | 24,411 |
Deductions | (25,441) |
Contract assets, ending balance | $ 1,176 |
Share-Based Payments - Addition
Share-Based Payments - Additional Information (Details) - USD ($) | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Restricted stock units | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Weighted-average period to recognize compensation expense (in years) | 1 year 10 months 13 days | |
Unrecognized compensation expense excluding options | $ 27,500,000 | |
Performance-based stock units | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Unrecognized compensation expense excluding options | 0 | |
Unrecognized compensation cost not expected to be recognized | 11,100,000 | |
Market-Based Stock Units | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Unrecognized compensation expense excluding options | $ 0 | |
2007 Plan and 2013 Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock reserved for issuance (in shares) | 12,065,656 | |
2013 Stock Incentive Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Shares available for future issuance (in shares) | 4,762,423 | |
Unrecognized compensation expense related to options | $ 31,900,000 | |
Weighted-average period to recognize compensation expense (in years) | 2 years 6 months 18 days | |
Employee stock purchase plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Shares available for future issuance (in shares) | 1,686,039 | |
Shares issued under 2013 ESPP (in shares) | 112,832 | 104,867 |
Opportunity to purchase of common stock (in shares) | 2,363,636 |
Share-Based Payments - Schedule
Share-Based Payments - Schedule of Company's Stock Option Activity (Details) - $ / shares | 9 Months Ended |
Sep. 30, 2023 | |
Number of Stock Options | |
Outstanding, beginning balance (in shares) | 5,772,564 |
Granted (in shares) | 870,924 |
Exercised (in shares) | (188,591) |
Forfeited/expired (in shares) | (912,479) |
Outstanding, ending balance (in shares) | 5,542,418 |
Exercisable (in shares) | 3,485,110 |
Vested and expected to vest (in shares) | 5,542,418 |
Weighted-Average Exercise Price | |
Outstanding, beginning balance (in usd per share) | $ 48.81 |
Granted (in usd per share) | 25.88 |
Exercised (in usd per share) | 11.52 |
Forfeited/expired (in usd per share) | 55.49 |
Outstanding, ending balance (in usd per share) | 45.37 |
Exercisable (in usd per share) | 54.14 |
Vested and expected to vest (in usd per share) | $ 45.37 |
Share-Based Payments - Schedu_2
Share-Based Payments - Schedule of RSU Activity (Details) - Restricted stock units | 9 Months Ended |
Sep. 30, 2023 $ / shares shares | |
Number of Stock Units | |
Unvested shares, beginning balance (in shares) | shares | 1,117,921 |
Granted (in shares) | shares | 899,072 |
Vested (in shares) | shares | (450,586) |
Forfeited (in shares) | shares | (210,420) |
Unvested shares, ending balance (in shares) | shares | 1,355,987 |
Weighted-Average Grant Date Fair Value | |
Unvested shares, beginning balance (in usd per share) | $ / shares | $ 38.30 |
Granted (in usd per share) | $ / shares | 25.97 |
Vested (in usd per share) | $ / shares | 41.42 |
Forfeited (in usd per share) | $ / shares | 33.46 |
Unvested shares, ending balance (in usd per share) | $ / shares | $ 29.84 |
Share-Based Payments - Schedu_3
Share-Based Payments - Schedule of PSU, Market-based Stock Unit or MSU Activity (Details) | 9 Months Ended |
Sep. 30, 2023 $ / shares shares | |
Performance-based stock units | |
Number of Stock Units | |
Unvested shares, beginning balance (in shares) | shares | 430,243 |
Granted (in shares) | shares | 125,897 |
Vested (in shares) | shares | (92,257) |
Forfeited (in shares) | shares | (101,750) |
Unvested shares, ending balance (in shares) | shares | 362,133 |
Weighted-Average Grant Date Fair Value | |
Unvested shares, beginning balance (in usd per share) | $ / shares | $ 35.87 |
Granted (in usd per share) | $ / shares | 25.23 |
Vested (in usd per share) | $ / shares | 30.18 |
Forfeited (in usd per share) | $ / shares | 46.39 |
Unvested shares, ending balance (in usd per share) | $ / shares | $ 30.66 |
Market-Based Stock Units | |
Number of Stock Units | |
Unvested shares, beginning balance (in shares) | shares | 42,695 |
Granted (in shares) | shares | 0 |
Unvested shares, ending balance (in shares) | shares | 42,695 |
Weighted-Average Grant Date Fair Value | |
Unvested shares, beginning balance (in usd per share) | $ / shares | $ 41.50 |
Granted (in usd per share) | $ / shares | 0 |
Unvested shares, ending balance (in usd per share) | $ / shares | $ 41.50 |
Share-Based Payments - Schedu_4
Share-Based Payments - Schedule of Stock-Based Compensation Expense by Award Type Included within the Condensed Consolidated Statements of Operations (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 9,076 | $ 11,156 | $ 30,952 | $ 37,831 |
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 4,171 | 5,864 | 13,065 | 17,720 |
Restricted stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 4,735 | 5,115 | 14,436 | 16,512 |
Performance-based stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 0 | 0 | 2,784 | 2,919 |
Employee stock purchase plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 170 | $ 177 | $ 667 | $ 680 |
Share-Based Payments - Schedu_5
Share-Based Payments - Schedule of Expenses Related to Stock Options and Stock-based Awards (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 9,076 | $ 11,156 | $ 30,952 | $ 37,831 |
Research and development expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 3,635 | 4,628 | 12,530 | 16,207 |
Selling, general and administrative expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 5,441 | $ 6,528 | $ 18,422 | $ 21,624 |
Loss per Share (Details)
Loss per Share (Details) - shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 6,909,788 | 7,183,967 | 6,909,788 | 7,183,967 |
Stock options | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 5,542,418 | 5,909,087 | 5,542,418 | 5,909,087 |
Restricted stock units | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 1,355,987 | 1,264,377 | 1,355,987 | 1,264,377 |
Employee stock purchase plan shares | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 11,383 | 10,503 | 11,383 | 10,503 |