Agios Pharmaceuticals (AGIO) 10-Q 31 Oct 24 2024 Q3 Quarterly report Financial data

Cover Page

Cover Page - shares	9 Months Ended
	Sep. 30, 2024	Oct. 25, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2024
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		57,030,195
Document Fiscal Year Focus	2024
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, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 253,730	$ 88,205
Marketable securities	751,027	688,723
Accounts receivable, net	3,118	2,810
Inventory	26,429	19,076
Prepaid expenses and other current assets	39,885	35,021
Total current assets	1,074,189	833,835
Marketable securities	655,889	29,435
Operating lease assets	45,841	54,409
Property and equipment, net	11,819	15,382
Other non-current assets	4,056	4,057
Total assets	1,791,794	937,118
Current liabilities:
Accounts payable	17,143	9,780
Accrued expenses	33,307	43,167
Income tax payable	52,682	0
Operating lease liabilities	16,319	15,008
Total current liabilities	119,451	67,955
Operating lease liabilities, net of current portion	44,515	56,988
Other non-current liabilities	1,156	1,156
Total liabilities	165,122	126,099
Stockholders’ equity:
Preferred stock, $0.001 par value; 25,000,000 shares authorized; no shares issued or outstanding at September 30, 2024 and December 31, 2023	0	0
Common stock, $0.001 par value; 125,000,000 shares authorized; 73,237,954 shares issued and 57,021,543 shares outstanding at September 30, 2024, and 72,161,489 shares issued and 55,945,078 shares outstanding at December 31, 2023	73	72
Additional paid-in capital	2,478,066	2,436,523
Accumulated other comprehensive income (loss)	3,420	(441)
Treasury stock, at cost (16,216,411 shares at September 30, 2024 and December 31, 2023)	(802,486)	(802,486)
Accumulated deficit	(52,401)	(822,649)
Total stockholders’ equity	1,626,672	811,019
Total liabilities and stockholders’ equity	$ 1,791,794	$ 937,118

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (parenthetical) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
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)	73,237,954	72,161,489
Common stock, outstanding (in shares)	57,021,543	55,945,078
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, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Revenues:
Total revenue	$ 8,964	$ 7,399	$ 25,768	$ 19,720
Operating expenses:
Cost of sales	783	633	2,905	2,295
Research and development	72,455	81,841	218,476	218,037
Selling, general and administrative	38,537	25,822	105,087	84,598
Total operating expenses	111,775	108,296	326,468	304,930
Loss from operations	(102,811)	(100,897)	(300,700)	(285,210)
Gain on sale of contingent payments	889,136	0	889,136	0
Milestone payment from gain on sale of oncology business	200,000	0	200,000	0
Interest income, net	13,059	8,375	30,068	24,720
Other income, net	1,651	1,198	4,864	4,342
Net income (loss) before taxes	1,001,035	(91,324)	823,368	(256,148)
Income tax expense	53,120	0	53,120	0
Net income (loss)	$ 947,915	$ (91,324)	$ 770,248	$ (256,148)
Net income (loss) per share - basic (in usd per share)	$ 16.65	$ (1.64)	$ 13.58	$ (4.61)
Net income (loss) per share - diluted (in usd per share)	$ 16.22	$ (1.64)	$ 13.38	$ (4.61)
Weighted-average number of common shares used in computing net income (loss) per share - basic (in shares)	56,939,403	55,803,663	56,709,318	55,559,766
Weighted-average number of common shares used in computing net income (loss) per share – diluted (in shares)	58,432,796	55,803,663	57,581,382	55,559,766
Product revenue, net
Revenues:
Total revenue	$ 8,964	$ 7,399	$ 25,768	$ 19,720

Condensed Consolidated Statem_2

Condensed Consolidated Statements of Comprehensive Income (Loss) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Statement of Comprehensive Income [Abstract]
Net income (loss)	$ 947,915	$ (91,324)	$ 770,248	$ (256,148)
Other comprehensive income
Unrealized gain on available-for-sale securities	4,465	2,974	3,861	6,639
Comprehensive income (loss)	$ 952,380	$ (88,350)	$ 774,109	$ (249,509)

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Loss	Accumulated Deficit	Treasury Stock
Beginning balance (in shares) at Dec. 31, 2022		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						(16,216,411,000)
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 income (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		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						(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net income (loss)	(256,148)
Ending balance (in shares) at Sep. 30, 2023		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						(16,216,411,000)
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 income (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 income (loss)	(91,324)				(91,324)
Ending balance (in shares) at Sep. 30, 2023		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						(16,216,411,000)
Beginning balance (in shares) at Dec. 31, 2023	55,945,078	72,161,489,000
Beginning balance at Dec. 31, 2023	$ 811,019	$ 72	2,436,523	(441)	(822,649)	$ (802,486)
Treasury stock, beginning balance (in shares) at Dec. 31, 2023	(16,216,411)					(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	$ (646)			(646)
Common stock issued under stock incentive plan and ESPP (in shares)		806,433,000
Common stock issued under stock incentive plan and ESPP	5,864	$ 1	5,863
Stock-based compensation expense	9,234		9,234
Net income (loss)	(81,549)				(81,549)
Ending balance (in shares) at Mar. 31, 2024		72,967,922,000
Ending balance at Mar. 31, 2024	$ 743,922	$ 73	2,451,620	(1,087)	(904,198)	$ (802,486)
Treasury stock, ending balance (in shares) at Mar. 31, 2024						(16,216,411,000)
Beginning balance (in shares) at Dec. 31, 2023	55,945,078	72,161,489,000
Beginning balance at Dec. 31, 2023	$ 811,019	$ 72	2,436,523	(441)	(822,649)	$ (802,486)
Treasury stock, beginning balance (in shares) at Dec. 31, 2023	(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)	223,740
Net income (loss)	$ 770,248
Ending balance (in shares) at Sep. 30, 2024	57,021,543	73,237,954,000
Ending balance at Sep. 30, 2024	$ 1,626,672	$ 73	2,478,066	3,420	(52,401)	$ (802,486)
Treasury stock, ending balance (in shares) at Sep. 30, 2024	(16,216,411)					(16,216,411,000)
Beginning balance (in shares) at Mar. 31, 2024		72,967,922,000
Beginning balance at Mar. 31, 2024	$ 743,922	$ 73	2,451,620	(1,087)	(904,198)	$ (802,486)
Treasury stock, beginning balance (in shares) at Mar. 31, 2024						(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	42			42
Common stock issued under stock incentive plan and ESPP (in shares)		123,568,000
Common stock issued under stock incentive plan and ESPP	1,099		1,099
Stock-based compensation expense	11,565		11,565
Net income (loss)	(96,118)				(96,118)
Ending balance (in shares) at Jun. 30, 2024		73,091,490,000
Ending balance at Jun. 30, 2024	660,510	$ 73	2,464,284	(1,045)	(1,000,316)	$ (802,486)
Treasury stock, ending balance (in shares) at Jun. 30, 2024						(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	4,465			4,465
Common stock issued under stock incentive plan and ESPP (in shares)		146,464,000
Common stock issued under stock incentive plan and ESPP	2,637		2,637
Stock-based compensation expense	11,145		11,145
Net income (loss)	$ 947,915				947,915
Ending balance (in shares) at Sep. 30, 2024	57,021,543	73,237,954,000
Ending balance at Sep. 30, 2024	$ 1,626,672	$ 73	$ 2,478,066	$ 3,420	$ (52,401)	$ (802,486)
Treasury stock, ending balance (in shares) at Sep. 30, 2024	(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, 2024	Sep. 30, 2023
Operating activities
Net income (loss)	$ 770,248	$ (256,148)
Adjustments to reconcile net income (loss) to net cash used in operating activities:
Depreciation and amortization	4,286	5,220
Stock-based compensation expense	31,944	30,952
Net accretion of discount on marketable securities	(8,870)	(4,658)
(Gain) loss on disposal of property and equipment	(39)	278
Non-cash operating lease expense	8,568	7,967
Expense associated with license agreement	0	17,500
Realized gain on investments	(168)	(28)
Gain on sale of contingent payments	(889,136)	0
Milestone payment from gain on sale of oncology business	(200,000)	0
Changes in operating assets and liabilities:
Accounts receivable, net	(308)	1,030
Inventory	(7,353)	(8,782)
Prepaid expenses and other current and non-current assets	(4,863)	440
Accounts payable	7,357	(5,201)
Accrued expenses and other current liabilities	(9,860)	140
Income taxes payable	52,682	0
Operating lease liabilities	(11,162)	(10,161)
Other non-current liabilities	0	(2,123)
Net cash used in operating activities	(256,674)	(223,574)
Investing activities
Purchases of marketable securities	(1,344,107)	(327,490)
Proceeds from maturities and sales of marketable securities	668,248	488,492
Payments associated with license agreement	0	(17,500)
Proceeds from sale of contingent payments	889,136	0
Proceeds from milestone payment from gain on sale of oncology business	200,000	0
Purchases of property and equipment	(718)	(765)
Proceeds from sale of equipment	40	1,325
Net cash provided by investing activities	412,599	144,062
Financing activities
Net proceeds from stock option exercises and employee stock purchase plan	9,600	4,586
Net cash provided by financing activities	9,600	4,586
Net change in cash and cash equivalents	165,525	(74,926)
Cash and cash equivalents at beginning of the period	88,205	139,259
Cash and cash equivalents at end of the period	253,730	64,333
Supplemental disclosure of non-cash investing and financing transactions
Additions to property and equipment in accounts payable and accrued expenses	61	14
Net cash taxes (returned) paid	$ (637)	$ 1,586

Overview and Basis of Presentat

Overview and Basis of Presentation	9 Months Ended
	Sep. 30, 2024
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, "Agios," "the company," “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 focus on classical hematology. 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 (i) AG-946 (tebapivat), a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndrome, or LR MDS, and hemolytic anemias, and (ii) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU. In September 2024, AG-946 (tebapivat) was granted Orphan Drug Designation by the FDA for the treatment of MDS. In addition to the aforementioned development programs, 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 preclinical development candidate discovered by Alnylam and targeting the transmembrane serine protease 6, or TMPRSS6, gene, and we have begun preclinical development of a product candidate for the potential disease-modifying treatment of patients with polycythemia vera, or PV, a rare blood disorder. 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. Alnylam License Agreement On July 28, 2023, we entered into a license agreement with Alnylam under which we acquired the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting the TMPRSS6 gene, 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 Accounting Standards Codification, or ASC, 805, Business Combinations , we accounted for the agreement as an asset acquisition. In accordance with the license agreement, in the three months ended September 30, 2023, we made an up-front payment to Alnylam and recognized in-process research and development of $17.5 million. We will also pay Alnylam for certain expenses associated with the development of the TMPRSS6 program, and these will be recorded in our Consolidated Statements of Operations as incurred. Additionally, we are 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. Sale of Oncology Business to Servier and Sale of Contingent Payments 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, or IDH, 1 or 2 mutation (and, to the extent required by such approval, the vorasidenib companion diagnostic test is granted an FDA premarket approval), or the Vorasidenib Milestone Payment, 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, or the Vorasidenib Royalty Rights. The Vorasidenib Milestone Payment, Vorasidenib Royalty Rights and royalty payments related to TIBSOVO® are referred to as contingent payments and recognized as income when realizable. 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, or Celgene, and following the sale Servier has agreed to conduct certain clinical development activities within the IDHIFA® development program. In October 2022, we sold our rights to future contingent payments associated with the royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through the loss of exclusivity to entities affiliated with Sagard Healthcare Partners, or Sagard, and recognized income of $127.9 million in our consolidated statements of operations for the year ended December 31, 2022. In August 2024, the FDA approved vorasidenib for adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection. In September 2024, we received the Vorasidenib Milestone Payment from Servier and recognized income of $200.0 million within the milestone payment from gain on sale of oncology business line item in our consolidated statements of operations for the three and nine months ended September 30, 2024. In May 2024, we entered into a purchase and sale agreement to sell the Vorasidenib Royalty Rights to Royalty Pharma Investments 2019 ICAV, or Royalty Pharma, for $905.0 million in cash, or the Upfront Payment. The sale was contingent upon FDA approval of vorasidenib and other customary closing conditions. Upon consummation of the sale in August 2024, Royalty Pharma acquired 100% of the Vorasidenib Royalty Rights payments made by Servier on account of up to $1.0 billion in U.S. net sales for each calendar year. In addition, any such Vorasidenib Royalty Rights payments made by Servier on account of U.S. net sales in each calendar year in excess of $1.0 billion will be split, with Royalty Pharma having the rights to a 12% earn-out on those excess payments and Agios retaining the rights to a 3% earn-out on those excess payments, or the Retained Earn-Out Rights. As a result of the sale, we recognized $889.1 million ($905.0 million net of fees of $15.9 million) within the gain on sale of contingent payments line item in our consolidated statements of operations for the three and nine months ended September 30, 2024. Royalty income related to the Retained Earn-Out Rights will be recognized in the period when realizable. Basis of Presentation The condensed consolidated balance sheet as of September 30, 2024, the condensed consolidated statements of operations, comprehensive income (loss) and stockholders' equity for the three and nine months ended September 30, 2024 and 2023, and the condensed consolidated statements of cash flows for the nine months ended September 30, 2024 and 2023 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, 2024, our results of operations and stockholders' equity for the three and nine months ended September 30, 2024 and 2023, and cash flows for the nine months ended September 30, 2024 and 2023. 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, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2023 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, 2023 that was filed with the Securities and Exchange Commission, or SEC, on February 15, 2024. 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 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. Liquidity As of September 30, 2024, we had cash, cash equivalents and marketable securities of $1.7 billion. 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 to be sufficient to fund current operations for at least the next twelve months from the issuance of the financial statements. If we are unable to raise additional funds through equity or debt financings, we may be required to delay, limit, reduce or terminate product development or future commercialization efforts, or grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.

Summary of Significant Accounti

Summary of Significant Accounting Policies	9 Months Ended
	Sep. 30, 2024
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, 2023. 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, 2024
Fair Value Disclosures [Abstract]
Fair Value Measurements	Fair Value Measurements We record cash equivalents and marketable securities at fair value. 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, 2024: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 20,154 $ 149,936 $ — $ 170,090 Total cash equivalents 20,154 149,936 — 170,090 Marketable securities: Certificates of deposit $ — $ 13,743 $ — $ 13,743 U.S. Treasuries — 266,598 — 266,598 Government securities — 297,557 — 297,557 Corporate debt securities — 829,018 — 829,018 Total marketable securities — 1,406,916 — 1,406,916 Total cash equivalents and marketable securities $ 20,154 $ 1,556,852 $ — $ 1,577,006 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, 2024. There have been no changes to the valuation methods during the nine months ended September 30, 2024, and we had no financial assets or liabilities that were classified as Level 3 at any point during the nine months ended September 30, 2024.

Marketable Securities

Marketable Securities	9 Months Ended
	Sep. 30, 2024
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 income (loss) in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive income (loss) in the condensed consolidated statements of comprehensive income (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, 2024 or 2023. Marketable securities at September 30, 2024 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: Certificates of deposit $ 13,724 $ 19 $ — $ 13,743 U.S. Treasuries 179,560 299 (10) 179,849 Government securities 160,442 235 (34) 160,643 Corporate debt securities 396,143 718 (69) 396,792 Total Current 749,869 1,271 (113) 751,027 Non-current: U.S. Treasuries 86,299 477 (27) 86,749 Government securities 136,747 250 (83) 136,914 Corporate debt securities 430,581 1,733 (88) 432,226 Total Non-current 653,627 2,460 (198) 655,889 Total marketable securities $ 1,403,496 $ 3,731 $ (311) $ 1,406,916 Marketable securities at December 31, 2023 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 30,876 $ — $ (56) $ 30,820 Government securities 247,460 194 (695) 246,959 Corporate debt securities 411,045 874 (975) 410,944 Total Current 689,381 1,068 (1,726) 688,723 Non-current: U.S. Treasuries 4,802 30 — 4,832 Government securities 9,986 75 — 10,061 Corporate debt securities 14,430 112 — 14,542 Total Non-current 29,218 217 — 29,435 Total marketable securities $ 718,599 $ 1,285 $ (1,726) $ 718,158 As of September 30, 2024 and December 31, 2023, 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, 2024 and December 31, 2023, we held 91 and 151 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, 2024 and December 31, 2023 related to these securities. The aggregate fair value of debt securities in an unrealized loss position at September 30, 2024 and December 31, 2023 was $336.6 million and $513.5 million, respectively. There were no individual securities that were in a significant unrealized loss position as of September 30, 2024 and December 31, 2023. 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, 2024 and December 31, 2023.

Inventory

Inventory	9 Months Ended
	Sep. 30, 2024
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 $ 90 $ 51 Work-in-process 24,079 17,568 Finished goods 2,260 1,457 Total inventory $ 26,429 $ 19,076

Leases

Leases	9 Months Ended
	Sep. 30, 2024
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 three 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) 2024 2023 2024 2023 Operating lease costs $ 3,807 $ 3,807 $ 11,420 $ 11,420 Cash paid for amounts included in the measurement of operating lease liabilities 4,684 4,550 14,015 $ 13,614 We have not entered into any material short-term leases or financing leases as of September 30, 2024. In arriving at the operating lease liabilities as of September 30, 2024 and December 31, 2023, we applied the weighted-average incremental borrowing rate of 5.7% for both periods over a weighted-average remaining lease term of 3.4 and 4.2 years, respectively. As of September 30, 2024, undiscounted minimum rental commitments under non-cancelable leases were as follows: (In thousands) Remaining 2024 $ 3,126 2025 19,507 2026 20,151 2027 20,755 2028 3,479 Undiscounted minimum rental commitments $ 67,018 Interest (6,184) Operating lease liabilities $ 60,834 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 $1.6 million and $1.7 million for the three months ended September 30, 2024 and 2023, respectively, and $4.8 million and $4.5 million for the nine months ended September 30, 2024 and 2023, respectively, in other income, net in the condensed consolidated statements of operations. We hold 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, 2024, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2024 $ 1,284 2025 1,310 Total $ 2,594

Accrued Expenses

Accrued Expenses	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
Accrued Expenses	Accrued Expenses Accrued expenses consisted of the following: (In thousands) September 30, December 31, Accrued compensation $ 17,686 $ 23,232 Accrued research and development costs 9,731 15,463 Accrued professional fees 3,273 3,115 Accrued other 2,617 1,357 Total accrued expenses $ 33,307 $ 43,167

Product Revenue

Product Revenue	9 Months Ended
	Sep. 30, 2024
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. These Customers subsequently resell PYRUKYND® to pharmacies or dispense PYRUKYND® 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) 2024 2023 2024 2023 Product revenue, net $ 8,964 $ 7,399 $ 25,768 $ 19,720 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, 2024: (In thousands) Contractual Adjustments Government Rebates Returns/ Replacement Total Balance at December 31, 2023 $ 156 $ 1,084 $ 232 $ 1,472 Current provisions relating to sales in the current year 1,021 1,915 289 3,225 Adjustments relating to prior years (39) (49) 40 (48) Payments/returns relating to sales in the current year (860) (731) — (1,591) Payments/returns relating to sales in the prior years (85) (373) (71) (529) Balance at September 30, 2024 $ 193 $ 1,846 $ 490 $ 2,529 Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows: (In thousands) September 30, 2024 December 31, 2023 Reduction of accounts receivable $ 91 $ 151 Component of accrued expenses 2,438 1,321 Total revenue-related reserves $ 2,529 $ 1,472 The following table presents changes in our contract assets during the nine months ended September 30, 2024: (In thousands) December 31, 2023 Additions Deductions September 30, 2024 Contract assets (1) Accounts receivable, net $ 2,810 $ 28,921 $ (28,613) $ 3,118

Share-Based Payments

Share-Based Payments	9 Months Ended
	Sep. 30, 2024
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, 2024, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 11,030,891, and we had 2,819,436 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, 2024: Number of Weighted-Average Exercise Price Outstanding at December 31, 2023 5,263,681 $ 44.94 Granted 1,023,433 34.04 Exercised (223,740) 31.13 Cancelled/Forfeited (46,427) 42.18 Expired (24,750) 35.37 Outstanding at September 30, 2024 5,992,197 $ 43.65 Exercisable at September 30, 2024 3,840,761 $ 50.47 Vested and expected to vest at September 30, 2024 5,992,197 $ 43.65 At September 30, 2024, there was approximately $33.5 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.49 years. Restricted stock units The following table presents RSU activity for the nine months ended September 30, 2024: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2023 1,346,701 $ 29.67 Granted 1,117,510 31.95 Vested (579,370) 32.72 Forfeited (40,166) 30.41 Unvested shares at September 30, 2024 1,844,675 $ 30.08 As of September 30, 2024, there was approximately $39.6 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 2.01 years. Performance-based stock units The following table presents PSU activity for the nine months ended September 30, 2024: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2023 362,133 $ 30.66 Granted 183,000 32.27 Vested (170,550) 35.04 Unvested shares at September 30, 2024 374,583 $ 29.45 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, 2024, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $11.0 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, 2024: Number of Weighted-Average Unvested shares at December 31, 2023 42,695 $ 41.50 Expired (42,695) 41.50 Unvested shares at September 30, 2024 — $ — 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, 2024, 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 102,805 and 112,832 shares of common stock during the nine months ended September 30, 2024 and 2023, 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, 2024, we had 1,583,234 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) 2024 2023 2024 2023 Stock options $ 4,579 $ 4,171 $ 13,011 $ 13,065 Restricted stock units 6,314 4,735 17,491 14,436 Performance-based stock units — — 750 2,784 Employee stock purchase plan 252 170 692 667 Total stock-based compensation expense $ 11,145 $ 9,076 $ 31,944 $ 30,952 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) 2024 2023 2024 2023 Research and development expense $ 4,391 $ 3,635 $ 12,626 $ 12,530 Selling, general and administrative expense 6,754 5,441 19,318 18,422 Total stock-based compensation expense $ 11,145 $ 9,076 $ 31,944 $ 30,952

Income (Loss) per Share

Income (Loss) per Share	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
Income (Loss) per Share	Income (Loss) per Share Basic net income (loss) per share is calculated by dividing net income (loss) by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (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 income (loss) per share calculation, stock options, RSUs and PSUs 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 with performance and market vesting conditions, respectively, that were not deemed probable as of September 30, 2024 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 income (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 the three and nine months ended September 30, 2023, no dilutive effect was recognized in the calculation of loss per share and basic and diluted net loss per share was the same for those periods. The following is a reconciliation of basic weighted-average number of common shares used in computing net income (loss) per share to diluted weighted-average number of common shares used in computing net income (loss) per share for the periods indicated: Three Months Ended Nine Months Ended 2024 2023 2024 2023 Basic shares 56,939,403 55,803,663 56,709,318 55,559,766 Effect of dilutive securities Stock options 557,300 — 231,082 — Restricted stock units 927,149 — 626,208 — Performance-based stock units — — 11,793 — Employee stock purchase plan shares 8,944 — 2,981 — Diluted shares 58,432,796 55,803,663 57,581,382 55,559,766 The following common stock equivalents were excluded from the calculation of diluted net income (loss) per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three Months Ended Nine Months Ended 2024 2023 2024 2023 Stock options 3,463,344 5,542,418 5,176,610 5,542,418 Restricted stock units — 1,355,987 90,480 1,355,987 Employee stock purchase plan shares 3,139 11,383 4,393 11,383 Total common stock equivalents 3,466,483 6,909,788 5,271,483 6,909,788

Income Taxes

Income Taxes	9 Months Ended
	Sep. 30, 2024
Income Tax Disclosure [Abstract]
Income Taxes	Income Taxes We are subject to taxation in numerous U.S. states and territories. As a result, our effective tax rate is derived from a combination of applicable tax rates in the various places that we operate. In preparing our financial statements, we estimate the amount of tax that will become payable in each of such places. Nevertheless, our effective tax rate may be different from previous periods or our current expectations due to numerous factors, including as a result of changes in the mix of our profitability from state to state, the results of examinations and audits of our tax filings, our inability to secure or sustain acceptable agreements with tax authorities, changes in accounting for income taxes and changes in tax laws. Any of these factors may result in tax obligations in excess of amounts accrued in our financial statements. We recorded income tax expense of $53.1 million for the three and nine months ended September 30, 2024 compared to no income tax expense for the three and nine months ended September 30, 2023. The income tax expense in the three and nine months ended September 30, 2024 was predominantly due to the current tax impact of the sale of the Vorasidenib Royalty Rights and the Vorasidenib Milestone Payment received in the three months ended September 30, 2024 as discussed above in Note 1 , Overview and Basis of Presentation . While we released a portion of our valuation allowance when we utilized certain net operating loss carryforwards, or NOLs, and research and development tax credits as a result of the income associated with the sale of Vorasidenib Royalty Rights and the Vorasidenib Milestone Payment in the three months ended September 30, 2024, we continue to maintain a full valuation allowance against all of our net deferred tax assets.

Subsequent Events

Subsequent Events	9 Months Ended
	Sep. 30, 2024
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events [Open until filing]

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net income (loss)	$ 947,915	$ (96,118)	$ (81,549)	$ (91,324)	$ (83,806)	$ (81,018)	$ 770,248	$ (256,148)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended	9 Months Ended
	Sep. 30, 2024 shares	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement		The following table describes, for the quarterly period covered by this report, each trading arrangement for the sale or purchase of Company securities adopted or terminated by our directors and officers that is either (1) a contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) (a “Rule 10b5-1 trading arrangement”) or (2) a “non-Rule 10b5-1 trading arrangement” (as defined in Item 408(c) of Regulation S-K): Name (Title) Action Taken (Date of Action) Type of Trading Arrangement Nature of Trading Arrangement Duration of Trading Arrangement Aggregate Number of Securities Rahul Ballal (Director) Adoption (August 3, 2024) Rule 10b5-1 trading arrangement Sale Through and including October 31, 2025 Up to 20,000 shares James Burns (Chief Legal Officer) Adoption (August 5, 2024) Rule 10b5-1 trading arrangement Sale Through and including December 31, 2024 Up to 21,752 shares David Scadden (Director) Adoption (September 6, 2024) Rule 10b5-1 trading arrangement Sale Through and including December 10, 2025 Up to 2,400 shares
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Rahul Ballal [Member]
Trading Arrangements, by Individual
Name	Rahul Ballal
Title	Director
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	August 3, 2024
Expiration Date	October 31, 2025
Arrangement Duration	454 days
Aggregate Available	20,000	20,000
James Burns [Member]
Trading Arrangements, by Individual
Name	James Burns
Title	Chief Legal Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	August 5, 2024
Expiration Date	December 31, 2024
Arrangement Duration	148 days
Aggregate Available	21,752	21,752
David Scadden [Member]
Trading Arrangements, by Individual
Name	David Scadden
Title	Director
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	September 6, 2024
Expiration Date	December 10, 2025
Arrangement Duration	460 days
Aggregate Available	2,400	2,400

Summary of Significant Accoun_2

Summary of Significant Accounting Policies (Policies)	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The condensed consolidated balance sheet as of September 30, 2024, the condensed consolidated statements of operations, comprehensive income (loss) and stockholders' equity for the three and nine months ended September 30, 2024 and 2023, and the condensed consolidated statements of cash flows for the nine months ended September 30, 2024 and 2023 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, 2024, our results of operations and stockholders' equity for the three and nine months ended September 30, 2024 and 2023, and cash flows for the nine months ended September 30, 2024 and 2023. 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, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2023 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, 2023 that was filed with the Securities and Exchange Commission, or SEC, on February 15, 2024. 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 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.
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. 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 income (loss) in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive income (loss) in the condensed consolidated statements of comprehensive income (loss), until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses
Loss per Share	Basic net income (loss) per share is calculated by dividing net income (loss) by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (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 income (loss) per share calculation, stock options, RSUs and PSUs 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 with performance and market vesting conditions, respectively, that were not deemed probable as of September 30, 2024 are not considered to be common stock equivalents.

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	9 Months Ended
	Sep. 30, 2024
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, 2024: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 20,154 $ 149,936 $ — $ 170,090 Total cash equivalents 20,154 149,936 — 170,090 Marketable securities: Certificates of deposit $ — $ 13,743 $ — $ 13,743 U.S. Treasuries — 266,598 — 266,598 Government securities — 297,557 — 297,557 Corporate debt securities — 829,018 — 829,018 Total marketable securities — 1,406,916 — 1,406,916 Total cash equivalents and marketable securities $ 20,154 $ 1,556,852 $ — $ 1,577,006

Marketable Securities (Tables)

Marketable Securities (Tables)	9 Months Ended
	Sep. 30, 2024
Investments, Debt and Equity Securities [Abstract]
Schedule of Marketable Securities	Marketable securities at September 30, 2024 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: Certificates of deposit $ 13,724 $ 19 $ — $ 13,743 U.S. Treasuries 179,560 299 (10) 179,849 Government securities 160,442 235 (34) 160,643 Corporate debt securities 396,143 718 (69) 396,792 Total Current 749,869 1,271 (113) 751,027 Non-current: U.S. Treasuries 86,299 477 (27) 86,749 Government securities 136,747 250 (83) 136,914 Corporate debt securities 430,581 1,733 (88) 432,226 Total Non-current 653,627 2,460 (198) 655,889 Total marketable securities $ 1,403,496 $ 3,731 $ (311) $ 1,406,916 Marketable securities at December 31, 2023 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 30,876 $ — $ (56) $ 30,820 Government securities 247,460 194 (695) 246,959 Corporate debt securities 411,045 874 (975) 410,944 Total Current 689,381 1,068 (1,726) 688,723 Non-current: U.S. Treasuries 4,802 30 — 4,832 Government securities 9,986 75 — 10,061 Corporate debt securities 14,430 112 — 14,542 Total Non-current 29,218 217 — 29,435 Total marketable securities $ 718,599 $ 1,285 $ (1,726) $ 718,158

Inventory (Tables)

Inventory (Tables)	9 Months Ended
	Sep. 30, 2024
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 $ 90 $ 51 Work-in-process 24,079 17,568 Finished goods 2,260 1,457 Total inventory $ 26,429 $ 19,076

Leases (Tables)

Leases (Tables)	9 Months Ended
	Sep. 30, 2024
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) 2024 2023 2024 2023 Operating lease costs $ 3,807 $ 3,807 $ 11,420 $ 11,420 Cash paid for amounts included in the measurement of operating lease liabilities 4,684 4,550 14,015 $ 13,614
Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases	As of September 30, 2024, undiscounted minimum rental commitments under non-cancelable leases were as follows: (In thousands) Remaining 2024 $ 3,126 2025 19,507 2026 20,151 2027 20,755 2028 3,479 Undiscounted minimum rental commitments $ 67,018 Interest (6,184) Operating lease liabilities $ 60,834
Schedule of Future Minimum Lease Payments to be Received under the Long-term Sublease Agreements	As of September 30, 2024, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2024 $ 1,284 2025 1,310 Total $ 2,594

Accrued Expenses (Tables)

Accrued Expenses (Tables)	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
Schedule of Accrued Expenses	Accrued expenses consisted of the following: (In thousands) September 30, December 31, Accrued compensation $ 17,686 $ 23,232 Accrued research and development costs 9,731 15,463 Accrued professional fees 3,273 3,115 Accrued other 2,617 1,357 Total accrued expenses $ 33,307 $ 43,167

Product Revenue (Tables)

Product Revenue (Tables)	9 Months Ended
	Sep. 30, 2024
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) 2024 2023 2024 2023 Product revenue, net $ 8,964 $ 7,399 $ 25,768 $ 19,720
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, 2024: (In thousands) Contractual Adjustments Government Rebates Returns/ Replacement Total Balance at December 31, 2023 $ 156 $ 1,084 $ 232 $ 1,472 Current provisions relating to sales in the current year 1,021 1,915 289 3,225 Adjustments relating to prior years (39) (49) 40 (48) Payments/returns relating to sales in the current year (860) (731) — (1,591) Payments/returns relating to sales in the prior years (85) (373) (71) (529) Balance at September 30, 2024 $ 193 $ 1,846 $ 490 $ 2,529
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, 2024 December 31, 2023 Reduction of accounts receivable $ 91 $ 151 Component of accrued expenses 2,438 1,321 Total revenue-related reserves $ 2,529 $ 1,472
Schedule of Changes in Contract Assets	The following table presents changes in our contract assets during the nine months ended September 30, 2024: (In thousands) December 31, 2023 Additions Deductions September 30, 2024 Contract assets (1) Accounts receivable, net $ 2,810 $ 28,921 $ (28,613) $ 3,118

Share-Based Payments (Tables)

Share-Based Payments (Tables)	9 Months Ended
	Sep. 30, 2024
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, 2024: Number of Weighted-Average Exercise Price Outstanding at December 31, 2023 5,263,681 $ 44.94 Granted 1,023,433 34.04 Exercised (223,740) 31.13 Cancelled/Forfeited (46,427) 42.18 Expired (24,750) 35.37 Outstanding at September 30, 2024 5,992,197 $ 43.65 Exercisable at September 30, 2024 3,840,761 $ 50.47 Vested and expected to vest at September 30, 2024 5,992,197 $ 43.65
Schedule of RSU Activity	The following table presents RSU activity for the nine months ended September 30, 2024: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2023 1,346,701 $ 29.67 Granted 1,117,510 31.95 Vested (579,370) 32.72 Forfeited (40,166) 30.41 Unvested shares at September 30, 2024 1,844,675 $ 30.08
Schedule of PSU Activity	The following table presents PSU activity for the nine months ended September 30, 2024: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2023 362,133 $ 30.66 Granted 183,000 32.27 Vested (170,550) 35.04 Unvested shares at September 30, 2024 374,583 $ 29.45
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, 2024: Number of Weighted-Average Unvested shares at December 31, 2023 42,695 $ 41.50 Expired (42,695) 41.50 Unvested shares at September 30, 2024 — $ —
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) 2024 2023 2024 2023 Stock options $ 4,579 $ 4,171 $ 13,011 $ 13,065 Restricted stock units 6,314 4,735 17,491 14,436 Performance-based stock units — — 750 2,784 Employee stock purchase plan 252 170 692 667 Total stock-based compensation expense $ 11,145 $ 9,076 $ 31,944 $ 30,952
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) 2024 2023 2024 2023 Research and development expense $ 4,391 $ 3,635 $ 12,626 $ 12,530 Selling, general and administrative expense 6,754 5,441 19,318 18,422 Total stock-based compensation expense $ 11,145 $ 9,076 $ 31,944 $ 30,952

Income (Loss) per Share (Tables

Income (Loss) per Share (Tables)	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
Schedule of Earnings Per Share, Basic and Diluted	The following is a reconciliation of basic weighted-average number of common shares used in computing net income (loss) per share to diluted weighted-average number of common shares used in computing net income (loss) per share for the periods indicated: Three Months Ended Nine Months Ended 2024 2023 2024 2023 Basic shares 56,939,403 55,803,663 56,709,318 55,559,766 Effect of dilutive securities Stock options 557,300 — 231,082 — Restricted stock units 927,149 — 626,208 — Performance-based stock units — — 11,793 — Employee stock purchase plan shares 8,944 — 2,981 — Diluted shares 58,432,796 55,803,663 57,581,382 55,559,766
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 income (loss) per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three Months Ended Nine Months Ended 2024 2023 2024 2023 Stock options 3,463,344 5,542,418 5,176,610 5,542,418 Restricted stock units — 1,355,987 90,480 1,355,987 Employee stock purchase plan shares 3,139 11,383 4,393 11,383 Total common stock equivalents 3,466,483 6,909,788 5,271,483 6,909,788

Overview and Basis of Present_2

Overview and Basis of Presentation (Details) - USD ($) $ in Thousands			1 Months Ended		3 Months Ended		9 Months Ended		12 Months Ended
	Sep. 30, 2022	Mar. 31, 2021	Sep. 30, 2024	May 31, 2024	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023	Dec. 31, 2022
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Gain on sale of contingent payments					$ 889,136	$ 0	$ 889,136	$ 0
Milestone payment from gain on sale of oncology business					200,000	0	200,000	$ 0
Cash, cash equivalents, and marketable securities			$ 1,700,000		$ 1,700,000		$ 1,700,000
Alnylam Pharmaceuticals, Inc.
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Proceeds from sale of contingent payments						$ 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
Contingent Payments and Royalty Rights | Vorasidenib
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				$ 1,000,000
Gain on sale of contingent payments				$ 889,100
Royalty percent for sales over $1 billion (as a percent)				12%
Sale of royalty interests, percentage sold				100%
Proceeds from sale of royalty interests, net				$ 905,000
Sale of royalty interests, issuance costs				$ 15,900
Percentage of royalty rights retained				3%
Discontinued Operations, Disposed of by Sale | Vorasidenib
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone payment from gain on sale of oncology business			$ 200,000
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%	5%
Gain on sale of contingent payments										$ 127,900
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, 2024	Dec. 31, 2023
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	$ 1,406,916	$ 718,158
Fair Value, Measurements, Recurring
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	170,090
Total marketable securities	1,406,916
Total cash equivalents and marketable securities	1,577,006
Fair Value, Measurements, Recurring | Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	170,090
Fair Value, Measurements, Recurring | Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	13,743
Fair Value, Measurements, Recurring | U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	266,598
Fair Value, Measurements, Recurring | Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	297,557
Fair Value, Measurements, Recurring | Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	829,018
Fair Value, Measurements, Recurring | Level 1
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	20,154
Total marketable securities	0
Total cash equivalents and marketable securities	20,154
Fair Value, Measurements, Recurring | Level 1 | Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	20,154
Fair Value, Measurements, Recurring | Level 1 | Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
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	149,936
Total marketable securities	1,406,916
Total cash equivalents and marketable securities	1,556,852
Fair Value, Measurements, Recurring | Level 2 | Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	149,936
Fair Value, Measurements, Recurring | Level 2 | Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	13,743
Fair Value, Measurements, Recurring | Level 2 | U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	266,598
Fair Value, Measurements, Recurring | Level 2 | Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	297,557
Fair Value, Measurements, Recurring | Level 2 | Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	829,018
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 | Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	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, 2024 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
	Sep. 30, 2024 USD ($) security	Sep. 30, 2023 USD ($)	Dec. 31, 2023 USD ($) security
Investments, Debt and Equity Securities [Abstract]
Realized gain (loss) on marketable securities	$ 0	$ 0
Number of debt securities in unrealized loss position for less than one year | security	91		151
Allowance for credit losses	$ 0		$ 0
Aggregate fair value of debt securities in unrealized loss position	$ 336,600,000		$ 513,500,000
Number of unrealized loss position | security	0		0

Marketable Securities - Schedul

Marketable Securities - Schedule of Marketable Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 1,403,496	$ 718,599
Unrealized Gains	3,731	1,285
Unrealized Losses	(311)	(1,726)
Fair Value	1,406,916	718,158
Current:
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	749,869	689,381
Unrealized Gains	1,271	1,068
Unrealized Losses	(113)	(1,726)
Fair Value	751,027	688,723
Current: | Certificates of deposit
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	13,724
Unrealized Gains	19
Unrealized Losses	0
Fair Value	13,743
Current: | U.S. Treasuries
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	179,560	30,876
Unrealized Gains	299	0
Unrealized Losses	(10)	(56)
Fair Value	179,849	30,820
Current: | Government securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	160,442	247,460
Unrealized Gains	235	194
Unrealized Losses	(34)	(695)
Fair Value	160,643	246,959
Current: | Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	396,143	411,045
Unrealized Gains	718	874
Unrealized Losses	(69)	(975)
Fair Value	396,792	410,944
Non-current:
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	653,627	29,218
Unrealized Gains	2,460	217
Unrealized Losses	(198)	0
Fair Value	655,889	29,435
Non-current: | U.S. Treasuries
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	86,299	4,802
Unrealized Gains	477	30
Unrealized Losses	(27)	0
Fair Value	86,749	4,832
Non-current: | Government securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	136,747	9,986
Unrealized Gains	250	75
Unrealized Losses	(83)	0
Fair Value	136,914	10,061
Non-current: | Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	430,581	14,430
Unrealized Gains	1,733	112
Unrealized Losses	(88)	0
Fair Value	$ 432,226	$ 14,542

Inventory (Details)

Inventory (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 90	$ 51
Work-in-process	24,079	17,568
Finished goods	2,260	1,457
Total inventory	$ 26,429	$ 19,076

Leases - Additional Information

Leases - Additional Information (Details) $ in Millions	3 Months Ended		9 Months Ended
	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Dec. 31, 2023	May 31, 2023 ft²	Apr. 30, 2022 ft²	Aug. 31, 2021 ft²
Lessor, Lease, Description [Line Items]
Remaining lease terms	3 years		3 years
Weighted-average incremental borrowing rate	5.70%		5.70%		5.70%
Weighted-average remaining lease term	3 years 4 months 24 days		3 years 4 months 24 days		4 years 2 months 12 days
Sublease income	$ 1.6	$ 1.7	$ 4.8	$ 4.5
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, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
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	$ 4,684	$ 4,550	$ 14,015	$ 13,614

Leases - Schedule of Undiscount

Leases - Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases (Details) $ in Thousands	Sep. 30, 2024 USD ($)
Leases [Abstract]
Remaining 2024	$ 3,126
2025	19,507
2026	20,151
2027	20,755
2028	3,479
Undiscounted minimum rental commitments	67,018
Interest	(6,184)
Operating lease liabilities	$ 60,834

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, 2024 USD ($)
Leases [Abstract]
Remaining 2024	$ 1,284
2025	1,310
Total	$ 2,594

Accrued Expenses (Details)

Accrued Expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Accrued compensation	$ 17,686	$ 23,232
Accrued research and development costs	9,731	15,463
Accrued professional fees	3,273	3,115
Accrued other	2,617	1,357
Total accrued expenses	$ 33,307	$ 43,167

Product Revenue - Schedule of P

Product Revenue - Schedule of Product Revenue, Net (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Disaggregation of Revenue [Line Items]
Product revenue, net	$ 8,964	$ 7,399	$ 25,768	$ 19,720
Product revenue, net
Disaggregation of Revenue [Line Items]
Product revenue, net	$ 8,964	$ 7,399	$ 25,768	$ 19,720

Product Revenue - Schedule of_2

Product Revenue - Schedule of Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Dec. 31, 2023
Contractual Adjustments
Contractual adjustments, beginning balance	$ 156
Contractual adjustments, current provisions relating to sales in the current year	1,021
Contractual adjustments, adjustments relating to prior year	(39)
Contractual adjustments, payments/returns relating to sales in the current year	(860)
Contractual adjustments, payments/returns relating to sales in the prior year	(85)
Contractual adjustments, ending balance	193
Government Rebates
Government rebates, beginning balance	1,084
Government rebates, current provisions relating to sales in the current year	1,915
Government rebates, adjustments relating to prior years	(49)
Government rebates, payments/returns relating to sales in the current year	(731)
Government rebates, payments/returns relating to sales in the prior years	(373)
Government rebates, ending balance	1,846
Returns/ Replacement
Returns/ replacement, beginning balance	232
Returns/ replacement, current provisions relating to sales in the current year	289
Returns/ replacement, adjustments relating to prior years	40
Returns/ replacement, payments/returns relating to sales in the current year	0
Returns/ replacement, payments/returns relating to sales in the prior years	(71)
Returns/ replacement, ending balance	490
Total
Total revenue-related reserves	2,529	$ 1,472
Total, current provisions relating to sales in the current year	3,225
Total, adjustments relating to prior years	(48)
Total, payments/returns relating to sales in the current year	(1,591)
Total, payments/returns relating to sales in the prior years	$ (529)

Product Revenue - Schedule of R

Product Revenue - Schedule of Revenue-related Reserves (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Revenue from Contract with Customer [Abstract]
Reduction of accounts receivable	$ 91	$ 151
Component of accrued expenses	2,438	1,321
Total revenue-related reserves	$ 2,529	$ 1,472

Product Revenue - Schedule of C

Product Revenue - Schedule of Changes in Contract Assets (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2024 USD ($)
Contract assets
Contract assets, beginning balance	$ 2,810
Additions	28,921
Deductions	(28,613)
Contract assets, ending balance	$ 3,118

Share-Based Payments - Addition

Share-Based Payments - Additional Information (Details) - USD ($)	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted-average period to recognize compensation expense (in years)	2 years 3 days
Unrecognized compensation expense excluding options	$ 39,600,000
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	11,000,000
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)	11,030,891
2013 Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)	2,819,436
Unrecognized compensation expense related to options	$ 33,500,000
Weighted-average period to recognize compensation expense (in years)	2 years 5 months 26 days
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)	1,583,234
Shares issued under 2013 ESPP (in shares)	102,805	112,832
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, 2024
Number of Stock Options
Outstanding, beginning balance (in shares)	5,263,681
Granted (in shares)	1,023,433
Exercised (in shares)	(223,740)
Cancelled/Forfeited (in shares)	(46,427)
Expired (in shares)	(24,750)
Outstanding, ending balance (in shares)	5,992,197
Exercisable (in shares)	3,840,761
Vested and expected to vest (in shares)	5,992,197
Weighted-Average Exercise Price
Outstanding, beginning balance (in usd per share)	$ 44.94
Granted (in usd per share)	34.04
Exercised (in usd per share)	31.13
Cancelled/Forfeited (in usd per share)	42.18
Expired (in usd per share)	35.37
Outstanding, ending balance (in usd per share)	43.65
Exercisable (in usd per share)	50.47
Vested and expected to vest (in usd per share)	$ 43.65

Share-Based Payments - Schedu_2

Share-Based Payments - Schedule of RSU Activity (Details) - Restricted stock units	9 Months Ended
	Sep. 30, 2024 $ / shares shares
Number of Stock Units
Unvested shares, beginning balance (in shares) | shares	1,346,701
Granted (in shares) | shares	1,117,510
Vested (in shares) | shares	(579,370)
Forfeited/Expired (in shares) | shares	(40,166)
Unvested shares, ending balance (in shares) | shares	1,844,675
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) | $ / shares	$ 29.67
Granted (in usd per share) | $ / shares	31.95
Vested (in usd per share) | $ / shares	32.72
Forfeited/Expired (in usd per share) | $ / shares	30.41
Unvested shares, ending balance (in usd per share) | $ / shares	$ 30.08

Share-Based Payments - Schedu_3

Share-Based Payments - Schedule of PSU, Market-based Stock Unit or MSU Activity (Details)	9 Months Ended
	Sep. 30, 2024 $ / shares shares
Performance-based stock units
Number of Stock Units
Unvested shares, beginning balance (in shares) | shares	362,133
Granted (in shares) | shares	183,000
Vested (in shares) | shares	(170,550)
Unvested shares, ending balance (in shares) | shares	374,583
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) | $ / shares	$ 30.66
Granted (in usd per share) | $ / shares	32.27
Vested (in usd per share) | $ / shares	35.04
Unvested shares, ending balance (in usd per share) | $ / shares	$ 29.45
Market-Based Stock Units
Number of Stock Units
Unvested shares, beginning balance (in shares) | shares	42,695
Forfeited/Expired (in shares) | shares	(42,695)
Unvested shares, ending balance (in shares) | shares	0
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) | $ / shares	$ 41.50
Forfeited/Expired (in usd per share) | $ / shares	41.50
Unvested shares, ending balance (in usd per share) | $ / shares	$ 0

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, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 11,145	$ 9,076	$ 31,944	$ 30,952
Stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	4,579	4,171	13,011	13,065
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	6,314	4,735	17,491	14,436
Performance-based stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	0	0	750	2,784
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 252	$ 170	$ 692	$ 667

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, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 11,145	$ 9,076	$ 31,944	$ 30,952
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	4,391	3,635	12,626	12,530
Selling, general and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 6,754	$ 5,441	$ 19,318	$ 18,422

Income (Loss) per Share - Sched

Income (Loss) per Share - Schedule of Dilutive Effect (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items]
Weighted-average number of common shares used in computing net income (loss) per share - basic (in shares)	56,939,403	55,803,663	56,709,318	55,559,766
Effect of dilutive securities
Diluted shares	58,432,796	55,803,663	57,581,382	55,559,766
Stock options
Effect of dilutive securities
Common stock equivalents (in shares)	557,300	0	231,082	0
Restricted stock units
Effect of dilutive securities
Common stock equivalents (in shares)	927,149	0	626,208	0
Performance-based stock units
Effect of dilutive securities
Common stock equivalents (in shares)	0	0	11,793	0
Employee stock purchase plan shares
Effect of dilutive securities
Common stock equivalents (in shares)	8,944	0	2,981	0

Income (Loss) per Share - Sch_2

Income (Loss) per Share - Schedule of Anti-Dilutive Effect (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	3,466,483	6,909,788	5,271,483	6,909,788
Stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	3,463,344	5,542,418	5,176,610	5,542,418
Restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	0	1,355,987	90,480	1,355,987
Employee stock purchase plan shares
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	3,139	11,383	4,393	11,383

Income Taxes (Details)

Income Taxes (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Income Tax Disclosure [Abstract]
Income tax expense	$ 53,120	$ 0	$ 53,120	$ 0