0001439222-23-000039 | 10-K | XBRL Viewer | Agios Pharmaceuticals INC

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Cover Page - USD ($)	12 Months Ended
Cover Page - USD ($)	Dec. 31, 2022	Feb. 17, 2023	Jun. 30, 2022
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2022
Current Fiscal Year End Date	--12-31
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 Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
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
ICFR Auditor Attestation Flag	true
Entity Shell Company	false
Entity Public Float			$ 1,200,277,504
Entity Common Stock, Shares Outstanding		55,285,223
Documents Incorporated by Reference	Portions of the registrant’s definitive proxy statement for its 2023 Annual Meeting of Stockholders to be filed pursuant to Regulation 14A within 120 days of the end of the registrant’s fiscal year ended December 31, 2022 are incorporated by reference into Part III of this Annual Report on Form 10-K to the extent stated herein.
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	FY
Amendment Flag	false
Entity Central Index Key	0001439222

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2022
Auditor Information [Abstract]
Auditor Firm ID	238
Auditor Name	PricewaterhouseCoopers LLP
Auditor Location	Boston, Massachusetts

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 139,259	$ 203,126
Marketable securities	643,860	816,892
Accounts receivable, net	2,206	0
Other receivable	0	4,378
Inventory	8,492	0
Prepaid expenses and other current assets	38,955	39,835
Total current assets	832,772	1,064,231
Marketable securities	313,874	266,375
Operating lease assets	65,129	75,124
Property and equipment, net	22,987	28,923
Financing lease assets	0	183
Other non-current assets	3,956	2,900
Total assets	1,238,718	1,437,736
Current liabilities:
Accounts payable	18,616	16,700
Accrued expenses	30,350	31,967
Operating lease liabilities	13,663	10,828
Financing lease liabilities	0	331
Total current liabilities	62,629	59,826
Operating lease liabilities, net of current portion	71,996	85,659
Financing lease liabilities, net of current portion	0	276
Other non-current liabilities	3,279	0
Total liabilities	137,904	145,761
Commitments and contingent liabilities (Note 16)
Stockholders’ equity:
Preferred stock, $0.001 par value; 25,000,000 shares authorized, no shares issued and outstanding at December 31, 2022 and 2021	0	0
Common stock, $0.001 par value; 125,000,000 shares authorized; 71,256,118 shares issued and 55,039,707 outstanding at December 31, 2022 and 70,550,631 shares issued and 54,334,220 outstanding at December 31, 2021	71	71
Additional paid-in capital	2,386,325	2,334,348
Accumulated other comprehensive loss	(12,535)	(1,198)
Accumulated deficit	(470,561)	(238,760)
Treasury stock, at cost (16,216,411 shares at December 31, 2022 and December 31, 2021)	(802,486)	(802,486)
Total stockholders’ equity	1,100,814	1,291,975
Total liabilities and stockholders’ equity	$ 1,238,718	$ 1,437,736

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2022	Dec. 31, 2021
Statement of Financial Position [Abstract]
Preferred stock, par value (in usd per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized (in shares)	25,000,000	25,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common stock, par value (in usd per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	125,000,000	125,000,000
Common stock, shares issued (in shares)	71,256,118	70,550,631
Common stock, shares outstanding (in shares)	55,039,707	54,334,220
Treasury stock (in shares)	16,216,411

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Total revenue	$ 14,240	$ 0	$ 0
Operating expenses
Cost of sales	1,704	0	0
Research and development	279,910	256,973	220,811
Selling, general and administrative	121,673	121,445	115,105
Total operating expenses	403,287	378,418	335,916
Loss from operations	(389,047)	(378,418)	(335,916)
Gain on sale of contingent payments	127,853	0	0
Royalty income from gain on sale of oncology business	9,851	6,639	0
Interest income, net	12,793	836	6,611
Other income, net	6,749	14,433	0
Net loss from continuing operations	(231,801)	(356,510)	(329,305)
Net income from discontinued operations, net of tax	0	1,961,225	1,935
Net (loss) income	$ (231,801)	$ 1,604,715	$ (327,370)
Net loss from continuing operations per share - basic (in usd per share)	$ (4.23)	$ (5.90)	$ (4.77)
Net loss from continuing operations per share - diluted (in usd per share)	(4.23)	(5.90)	(4.77)
Net income from discontinued operations per share - basic (in usd per share)	0	32.45	0.03
Net income from discontinued operations per share - diluted (in usd per share)	0	32.45	0.03
Net income (loss) per share – basic (in usd per share)	(4.23)	26.55	(4.74)
Net income (loss) per share – diluted (in usd per share)	$ (4.23)	$ 26.55	$ (4.74)
Weighted-average number of common shares used in computing net loss per share – basic (in shares)	54,789,435	60,447,346	68,997,879
Weighted-average number of common shares used in computing net loss per share – diluted (in shares)	54,789,435	60,447,346	68,997,879
Product revenue, net
Total revenue	$ 11,740	$ 0	$ 0
Milestone revenue
Total revenue	$ 2,500	$ 0	$ 0

Consolidated Statements of Comprehensive (Loss) Income - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Statement of Comprehensive Income [Abstract]
Net (loss) income	$ (231,801)	$ 1,604,715	$ (327,370)
Other comprehensive (loss) income
Unrealized loss on available-for-sale securities	(11,337)	(1,303)	(97)
Comprehensive (loss) income	$ (243,138)	$ 1,603,412	$ (327,467)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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+ Details

Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Treasury
Common stock, beginning balance (in shares) at Dec. 31, 2019		68,401,105
Beginning balance at Dec. 31, 2019	$ 640,528	$ 68	$ 2,156,363	$ 202	$ (1,516,105)	$ 0
Treasury stock, beginning balance (in shares) at Dec. 31, 2019						0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	(97)			(97)
Net (loss) income	(327,370)				(327,370)
Stock-based compensation expense	61,602		61,602
Issuance of common stock under stock incentive and employee stock purchase plans (in shares)		892,815
Issuance of common stock under stock incentive and employee stock purchase plans	11,317	$ 1	11,316
Disposition of oncology business	13,520		13,520
Common stock, ending balance (in shares) at Dec. 31, 2020		69,293,920
Ending balance at Dec. 31, 2020	399,500	$ 69	2,242,801	105	(1,843,475)	$ 0
Treasury stock, ending balance (in shares) at Dec. 31, 2020						0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	(1,303)			(1,303)
Net (loss) income	1,604,715				1,604,715
Stock-based compensation expense	53,508		53,508
Issuance of common stock under stock incentive and employee stock purchase plans (in shares)		1,256,711
Issuance of common stock under stock incentive and employee stock purchase plans	37,296	$ 2	37,294
Repurchase of common stock (in shares)						(16,216,411)
Repurchase of common stock	(802,486)					$ (802,486)
Disposition of oncology business	$ 745		745
Common stock, ending balance (in shares) at Dec. 31, 2021	54,334,220	70,550,631
Ending balance at Dec. 31, 2021	$ 1,291,975	$ 71	2,334,348	(1,198)	(238,760)	$ (802,486)
Treasury stock, ending balance (in shares) at Dec. 31, 2021						(16,216,411)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	(11,337)			(11,337)
Net (loss) income	(231,801)				(231,801)
Stock-based compensation expense	$ 49,296		49,296
Issuance of common stock under stock incentive and employee stock purchase plans (in shares)	15,539	705,487
Issuance of common stock under stock incentive and employee stock purchase plans	$ 2,681		2,681
Common stock, ending balance (in shares) at Dec. 31, 2022	55,039,707	71,256,118
Ending balance at Dec. 31, 2022	$ 1,100,814	$ 71	$ 2,386,325	$ (12,535)	$ (470,561)	$ (802,486)
Treasury stock, ending balance (in shares) at Dec. 31, 2022						(16,216,411)

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Operating activities
Net (loss) income	$ (231,801)	$ 1,604,715	$ (327,370)
Less: Net income from discontinued operations	0	1,961,225	1,935
Net loss from continuing operations	(231,801)	(356,510)	(329,305)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	8,564	9,240	9,790
Stock-based compensation expense	49,296	53,508	61,602
Net (accretion of discount) amortization of premium on marketable securities	(1,198)	6,949	3,022
Gain on sale of contingent payments	(127,853)	0	0
(Gain) loss on disposal of property and equipment	(48)	12	0
Non-cash operating lease expense	9,995	9,537	8,982
Changes in operating assets and liabilities:
Accounts receivable, net	(2,206)	0	0
Inventory	(8,492)	0	0
Other receivables	447	(4,378)	0
Prepaid expenses and other current and non-current assets	(176)	(26,846)	(3)
Accounts payable	3,436	1,863	3,330
Accrued expenses	(1,617)	66	6,765
Operating lease liabilities	(10,828)	(7,527)	(8,127)
Other liabilities	3,003	0	0
Net cash used in operating activities	(309,478)	(314,086)	(243,944)
Net cash used in operating activities - discontinued operations	0	(93,234)	(46,815)
Net cash used in operating activities	(309,478)	(407,320)	(290,759)
Investing activities
Purchases of marketable securities	(1,030,781)	(1,378,221)	(557,030)
Proceeds from maturities and sales of marketable securities	1,146,175	829,804	647,685
Proceeds from sale of contingent payments	131,784	0	0
Purchases of property and equipment	(4,881)	(5,741)	(14,106)
Proceeds from sale of equipment	964	0	0
Net cash provided by (used in) investing activities	243,261	(554,158)	76,549
Net cash provided by (used in) investing activities - discontinued operations	0	1,802,936	(803)
Net cash provided by investing activities	243,261	1,248,778	75,746
Financing activities
Payments on financing lease obligations	(331)	(578)	(336)
Purchase of treasury stock	0	(802,486)	0
Net proceeds from stock option exercises and employee stock purchase plan	2,681	37,296	11,317
Net cash provided by (used in) financing activities	2,350	(765,768)	10,981
Net cash provided by financing activities - discontinued operations	0	0	250,537
Net cash provided by (used in) financing activities	2,350	(765,768)	261,518
Net change in cash and cash equivalents	(63,867)	75,690	46,505
Cash and cash equivalents at beginning of the period	203,126	127,436	80,931
Cash and cash equivalents at end of the period	139,259	203,126	127,436
Supplemental disclosure of non-cash investing and financing transactions:
Additions to property and equipment in accounts payable and accrued expenses	158	1,678	465
Cash taxes paid	0	16,078	0
Financing lease liabilities arising from obtaining financing lease assets	$ 0	$ 511	$ 0

Nature of Business	12 Months Ended
Nature of Business	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of Business	Nature of Business References to Agios Throughout this Annual Report on Form 10-K, “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, small molecule medicines for rare diseases. With a history of focused study on cellular metabolism, we have a deep and mature understanding of this biology, which is involved in the healthy functioning of nearly every system in the body. 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 Food and Drug Administration, or FDA, approved PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency in the United States. In November 2022, we received marketing authorization from the European Commission for PYRUKYND® for the treatment of PK deficiency in adult patients in the European Union, or EU. In December 2022, we received marketing authorization in Great Britain for PYRUKYND® for the treatment of PK deficiency in adult patients under the European Commission Decision Reliance Procedure. In addition, we are currently evaluating PYRUKYND® in clinical trials for the treatment of thalassemia, sickle cell disease, or SCD, and in pediatric patients with PK deficiency. We are also developing AG-946, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndrome, or LR MDS, and hemolytic anemias. In addition to the aforementioned clinical development programs, we continue to invest in our late-stage research program focused on advancing a phenylalanine hydroxylase, or PAH, stabilizer for the treatment of phenylketonuria, or PKU. We are subject to risks common to companies in our industry including, but not limited to, uncertainties relating to conducting 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, product liability claims and government investigations. Sale of our Oncology Business to Servier On March 31, 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier, which represented a discontinued operation. The transaction included the sale of our oncology business, including TIBSOVO®, our clinical-stage product candidates vorasidenib, AG-270 and AG-636, and our oncology research programs for a payment of approximately $1.8 billion in cash at the closing, subject to certain adjustments, and a payment of $200.0 million in cash, if, prior to January 1, 2027, vorasidenib is granted new drug application, or NDA, approval from the FDA with an approved label that permits vorasidenib’s use as a single agent for the adjuvant treatment of patients with Grade 2 glioma that have an isocitrate dehydrogenase 1 or 2 mutation (and, to the extent required by such approval, the vorasidenib companion diagnostic test is granted an FDA premarket approval), as well as a royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through loss of exclusivity, and a royalty of 15% of U.S. net sales of vorasidenib from the first commercial sale of vorasidenib through loss of exclusivity. Servier also acquired our co-commercialization rights for Bristol Myers Squibb’s IDHIFA® and the right to receive a $25.0 million potential milestone payment under our prior collaboration agreement with Celgene Corporation, or Celgene, and following the sale Servier will conduct certain clinical development activities within the IDHIFA® development program. We recorded income from royalties of approximately $9.9 million and $6.6 million on U.S. net sales of TIBSOVO® by Servier in the royalty income from gain on sale of oncology business line item within the consolidated statements of operations, for the years ended December 31, 2022 and December 31, 2021, respectively. Sale of Contingent Payments The consideration for the sale of our oncology business to Servier includes a royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through loss of exclusivity, referred to as contingent payments. We recognize the contingent payments in the royalty income from gain on sale of oncology business line item in our consolidated statement of operations in the period when realizable. On October 27, 2022, we sold our rights to future contingent payments to entities affiliated with Sagard Healthcare Partners, or Sagard, and recognized income of $127.9 million within the gain on sale of contingent payments line item in our consolidated statements of operations for the year ended December 31, 2022. We retained our rights to the potential milestone payment and royalties from Servier if vorasidenib is approved by the FDA. Reclassifications Certain amounts in prior periods have been reclassified to reflect the impact of the discontinued operations treatment of the oncology business in order to conform to the current period presentation. Liquidity On March 31, 2021, we completed the sale of our oncology business to Servier, and received approximately $1.8 billion in cash at closing. In connection with the sale, on March 25, 2021, we announced that our Board of Directors authorized the repurchase of up to $1.2 billion of our outstanding shares of common stock, or the Repurchase Program, using the proceeds from the sale of our oncology business to Servier. On March 31, 2021, in connection with the Repurchase Program, we entered into a definitive share repurchase agreement with Bristol-Myers Squibb Company, or BMS, to repurchase 7.1 million shares of our common stock held by certain subsidiaries of BMS for an aggregate purchase price of $344.5 million, or $48.38 per share. This repurchase was completed on April 5, 2021. Further, on April 2, 2021, in connection with the Repurchase Program, we entered into a Rule 10b5-1 repurchase plan pursuant to which we could repurchase up to $600.0 million of shares of our common stock. On October 5, 2021, we terminated our Rule 10b5-1 share repurchase program and on October 13, 2021 we entered into a Rule 10b-18 repurchase plan that allows us to conduct open market repurchases over time up to our remaining authorization under the Repurchase Program. We have not repurchased any shares of common stock in fiscal year 2022 and as of December 31, 2022 we have repurchased approximately 9.1 million shares of common stock for $458.0 million, or $50.35 per share, under the Rule 10b5-1 repurchase plan. As of December 31, 2022, we have not repurchased any shares under the Rule 10b-18 repurchase plan. In total, as of December 31, 2022, we repurchased 16.2 million shares of common stock for $802.5 million, or $49.49 per share, under the Repurchase Program. We have paused our share repurchases for the foreseeable future. On April 30, 2020, we entered into an at-the-market sales agreement, or the 2020 sales agreement, with Cowen & Company LLC, or Cowen, pursuant to which we may offer and sell shares of our common stock having an aggregate offering price of up to $250.0 million through Cowen pursuant to a universal shelf registration statement on Form S-3 filed with the SEC on April 30, 2020. As of December 31, 2022, $250.0 million in common stock remained available for future issuance under the 2020 sales agreement. On February 15, 2023, we delivered written notice to Cowen that we were terminating the 2020 sales agreement, effective on February 22, 2023. As of the termination of the 2020 sales agreement, we had not sold any shares of our common stock under the 2020 sales agreement. As of December 31, 2022, we had cash, cash equivalents and marketable securities of $1.1 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 Accounting Policies

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Principles of consolidation

The consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries, Agios Securities Corporation, Agios International Sarl (GmbH), Agios Germany GmbH, Agios Netherlands B.V., Agios Italy S.R.L., Agios France SARL, and Agios Limited. All intercompany transactions have been eliminated in consolidation. The consolidated financial statements have been prepared in conformity with U.S. generally accepted accounting principles, or U.S. GAAP.

Use of estimates

The preparation of our consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making

judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the COVID-19 pandemic will 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, including as a result of new information that may emerge concerning COVID-19 and any variant strains of the virus and the actions taken to contain the pandemic or treat COVID-19, as well as the economic impact on local, regional, national and international customers and markets. We have made estimates of the impact of COVID-19 within our financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates.

Cash and cash equivalents

We consider highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents are stated at fair value.

Accounts receivable, net

Our trade accounts receivable arise from product sales and represent amounts due from specialty distributors and specialty pharmacy providers in the U.S. We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile. We reserve against these receivables for estimated losses that may arise from a customer’s inability to pay. Amounts determined to be uncollectible are charged or written-off against the reserve.

Inventory

Inventory is stated at the lower of cost or estimated net realizable value on a first-in, first-out basis. Prior to the regulatory approval of our product candidates, we incur expenses for the manufacture of drug product that could potentially be available to support the commercial launch of those products. Until the date at which regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expenses. Upon approval of our wholly owned product, PYRUKYND®, by the FDA on February 17, 2022 for the treatment of hemolytic anemia in adults with PK deficiency in the United States, we began to capitalize inventories of PYRUKYND®.

Revenue recognition

Under Accounting Standards Codification 606, Revenue from Contracts with Customers, or ASC 606, revenue is recognized when the customer obtains control of promised goods or services, in an amount that reflects the consideration that we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that have been determined to be within the scope of ASC 606, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer.

This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments.

Once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations, and assess whether each promised good or service is distinct. We will then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

Product Revenue

We generate product revenue from sales of PYRUKYND® to a limited number of specialty distributors and specialty pharmacy providers, or collectively, the Customers. These Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®.

The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer.

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. We estimate the amount of product sales that may be returned by Customers and record this estimate as a reduction of revenue in the period the related product revenue is recognized. We currently estimate product return liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel.

Cost of sales

Cost of sales consists primarily of manufacturing costs for sales of PYRUKYND®. Based on our policy to expense costs associated with the manufacturing of our products prior to regulatory approval, certain of the manufacturing costs associated with product shipments of PYRUKYND® recorded during the twelve months ended December 31, 2022 were expensed prior to February 17, 2022 and, therefore, are not included in costs of sales during the twelve months ended December 31, 2022.

Marketable securities

Marketable securities at December 31, 2022 and 2021 consisted of investments in U.S. Treasuries, government securities and corporate debt securities. We determine the appropriate classification of the securities at the time they are acquired and evaluate the appropriateness of such classifications at each balance sheet date. We classify our marketable securities as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities. Marketable securities are recorded at fair value. Unrealized gains and losses are included as a component of accumulated other comprehensive (loss) income in the consolidated balance sheets and statements of stockholders’ equity and a component of total comprehensive (loss) income in the consolidated statements of comprehensive (loss) income, until realized. Realized gains and losses are included in investment income on a specific-identification basis.

At December 31, 2022 and 2021, 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 one to two years, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and therefore classified as available-for-sale.

We review marketable securities for impairment whenever the fair value of a marketable security is less than the amortized cost and evidence indicates that a marketable security’s carrying amount is not recoverable. 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 balance sheet with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive (loss) income, net of taxes. Evidence considered in this assessment includes reasons for the impairment, compliance with our investment policy, the severity of the impairment, collectability of the security, and any adverse conditions specifically related to the security, an industry, or geographic area.

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, either 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.

Our financial assets, which include cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued 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 December 31, 2022 or 2021. Fair value information for these assets, including their classification in the fair value hierarchy is included in Note 3 , Fair Value Measurements.

There have been no changes to the valuation methods during the years ended December 31, 2022 and 2021. We evaluate transfers between levels at the end of each reporting period.

The carrying amounts of other receivables, prepaid expenses and other current assets, accounts payable, and accrued expenses approximate their fair values due to their short-term maturities.

Concentrations of credit risk

Financial instruments which potentially subject us to credit risk consist primarily of cash, cash equivalents, and marketable securities. We hold these investments in highly rated financial institutions, and, by policy, limit the amounts of credit exposure to any one financial institution. These amounts at times may exceed federally insured limits. We have not experienced any credit losses in such accounts and do not believe we are exposed to any significant credit risk on these funds. We have no off-balance sheet concentrations of credit risk, such as foreign currency exchange contracts, option contracts, or other hedging arrangements.

Property and equipment

Property and equipment consist of laboratory equipment, computer equipment and software, leasehold improvements, furniture and fixtures, and office equipment. Costs of major additions and betterment are capitalized; maintenance and repairs, which do not improve or extend the life of the respective assets, are charged to expense as incurred. Upon retirement or sale, the cost of the disposed asset and the related accumulated depreciation are removed from the accounts and the resulting gain or loss is recognized.

Property and equipment is stated at cost, and depreciated using the straight-line method over the estimated useful lives of the respective assets:


						Years
Laboratory equipment						5
Computer equipment and software						3
Furniture and fixtures						5
Office equipment						5

Leasehold improvements are amortized over the lesser of the remaining lease term or the estimated useful life of the improvement.

Impairment of long-lived assets

We periodically evaluate our long-lived assets for potential impairment in accordance with ASC 360, Property, Plant and Equipment. Potential impairment is assessed when there is evidence that events or changes in circumstances indicate that the carrying amount of an asset may not be recovered. Recoverability of these assets is assessed based on the undiscounted expected future cash flows from the assets, considering a number of factors, including past operating results, budgets and

economic projections, market trends and product development cycles. If impairments are identified, assets are written down to their estimated fair value. We did not recognize any impairment charges through December 31, 2022.

Leases

We determine if an arrangement is a lease at inception. An arrangement is determined to contain a lease if the contract conveys the right to control the use of an identified property or equipment for a period of time in exchange for consideration. If we can benefit from the various underlying assets of a lease on their own or together with other resources that are readily available, or if the various underlying assets are neither highly dependent on nor highly interrelated with other underlying assets in the arrangement, they are considered to be a separate lease component. In the event multiple underlying assets are identified, the lease consideration is allocated to the various components based on each of the component’s relative fair value.

Operating lease assets represent our right to use an underlying asset for the lease term and operating lease liabilities represent our obligation to make lease payments arising from the leasing arrangement. Operating lease assets and operating lease liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As most of our leases do not provide an implicit rate, in determining the operating lease liabilities we use an estimate of our incremental borrowing rate. The incremental borrowing rate is determined using two alternative credit scoring models to estimate our credit rating, adjusted for collateralization. The calculation of the operating lease assets includes any lease payments made and excludes any lease incentives. Our lease terms may include options to extend or terminate the lease when it is reasonably certain that we will exercise that option.

For operating leases, we record operating lease assets and lease liabilities in our consolidated balance sheets. Lease expense for lease payments is recognized on a straight-line basis over the lease term. Short-term leases, or leases that have a lease term of 12 months or less at commencement date, are excluded from this treatment and are recognized on a straight-line basis over the term of the lease.

We have not entered into any material short-term leases or financing leases as of December 31, 2022.

Research and development costs

Research and development costs, including those accrued as of each balance sheet date, are expensed as incurred. These costs include salaries and personnel-related costs, consulting fees, fees paid for contract research services, fees paid to contract research organizations, or CROs, and other third parties in connection with clinical trials and preclinical development activities, fees paid to investigative sites in connection with clinical studies, the costs associated with the product manufacturing, development, and distribution of clinical supplies, the costs of laboratory equipment and facilities, and other external costs.

Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized. Additionally, there may be instances in which payments made to our vendors will exceed the level of services provided, and result in a prepayment of the research and development expense. The capitalized amounts are expensed as the related goods are delivered or the services are performed. We estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual or prepaid accordingly.

Stock-based compensation

We account for stock-based compensation awards in accordance with ASC 718, Compensation –Stock Compensation, or ASC 718. For stock-based awards granted to employees, non-employees and members of the board of directors for their services and for participation in our employee stock purchase plan, we estimate the grant date fair value of each option award using the Black-Scholes option-pricing model. The use of the Black-Scholes option-pricing model requires us to make assumptions with respect to the expected term of the option, the expected volatility of the common stock consistent with the expected life of the option, risk-free interest rates and expected dividend yields of the common stock. For awards subject to service-based vesting conditions, we recognize stock-based compensation expense equal to the grant date fair value of stock options on a straight-line basis over the requisite service period. For awards subject to both performance and service-based vesting conditions, we recognize stock-based compensation expense over the remaining service period if the performance condition is considered probable of achievement using management’s best estimates.

Income taxes

Income taxes are recorded in accordance with ASC 740, Accounting for Income Taxes, or ASC 740, which provides for deferred taxes using an asset and liability approach. We recognize deferred tax assets and liabilities for the expected future tax consequences of events that have been included in our financial statements or tax returns. We determine our deferred tax assets and liabilities based on differences between the financial reporting and tax bases of assets and liabilities, which are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. Valuation allowances are

provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized.

We also account for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, we recognize the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances.

Comprehensive income (loss)

Comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances, and currently consists of net loss and unrealized gains and losses on available-for-sale securities. Accumulated other comprehensive (loss) income consists entirely of unrealized gains and losses from available-for-sale securities as of December 31, 2022 and 2021.

Net 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 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, restricted stock units, or RSUs, performance-based stock units, or PSUs, and market-based stock units, or MSUs, for which the performance and market vesting conditions, respectively, have been deemed probable, and employee stock purchase plan shares are considered to be common stock equivalents but are excluded from the calculation of diluted net income (loss) per share as their effect would be anti-dilutive.

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 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 from continuing operations for all periods presented, no dilutive effect has been recognized in the calculation of income (loss) from discontinued operations per share or net income (loss) per share.

Segment and geographic information

Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker or decision-making group in making decisions on how to allocate resources and assess performance. Our chief operating decision maker is the chief executive officer. Our chief operating decision maker and we view our operations and manage our business as one operating segment.

Discontinued operations

We accounted for the sale of our oncology business in accordance with ASC 205, Discontinued Operations and Accounting Standards Update, or ASU, No. 2014-08, Reporting of Discontinued Operations and Disclosures of Disposals of Components of an Entity. We followed the held-for-sale criteria as defined in ASC 360, Property, Plant and Equipment, and ASC 205. ASC 205 requires that a component of an entity that has been disposed of or is classified as held for sale and has operations and cash flows that can be clearly distinguished from the rest of the entity be reported as assets held for sale and discontinued operations. In the period a component of an entity has been disposed of or classified as held for sale, the results of operations for the periods presented are reclassified into separate line items in the consolidated statements of operations. Assets and liabilities are also reclassified into separate line items on the related consolidated balance sheets for the periods presented. The statements of cash flows for the periods presented are also reclassified to reflect the results of discontinued operations as separate line items. ASU 2014-08 requires that only a disposal of a component of an entity, or a group of components of an entity, that represents a strategic shift that has, or will have, a major effect on the reporting entity’s operations and financial results be reported in the financial statements as discontinued operations. ASU 2014-08 also provides guidance on the financial statement presentations and disclosures of discontinued operations.

Due to the sale of the oncology business during the first quarter of 2021, in accordance with ASC 205, we have classified the results of the oncology business as discontinued operations in our consolidated statements of operations and cash flows for all periods presented, and refer to Note 15 , Discontinued Operations. All assets and liabilities associated with our oncology business were therefore classified as assets and liabilities of discontinued operations in our consolidated balance sheets for the periods presented. All amounts included in the notes to the consolidated financial statements relate to continuing operations unless otherwise noted.

Treasury stock

Treasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock.

Recent accounting pronouncements

In June 2016, the Financial Accounting Standards Board, or FASB, issued ASU 2016-13, Financial Instruments - Credit Losses (Topic 326), which introduces new guidance for the accounting for credit losses on instruments within its scope. The new guidance introduces an approach based on expected losses to estimate credit losses on certain types of financial instruments. Credit losses relating to available-for-sale debt securities will also be recorded through an allowance for credit losses rather than as a reduction in the amortized cost basis of the securities. The guidance is effective for fiscal years beginning after December 31, 2019, including interim periods within those years. The Company adopted this amendment as of January 1, 2020, which eliminated the concept of other-than-temporary impairments and required credit losses on debt securities to be recorded through an allowance for credit losses instead of as a reduction in the amortized cost basis of the securities. Application of the amendments is through a cumulative-effect adjustment to retained earnings as of the effective date. There was no material impact to the Company’s consolidated financial position, results of operation, or cash flows.

Other accounting standards that have been issued by the FASB 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.

Subsequent events

We considered events or transactions occurring after the balance sheet date, but prior to the issuance of the consolidated financial statements, for potential recognition or disclosure in our consolidated financial statements. All significant subsequent events have been properly disclosed in the consolidated financial statements.

Fair Value Measurements

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

Fair Value Measurements

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level (as described in Note 2. Summary of Significant Accounting Policies) on a recurring basis as of December 31, 2022:


(In thousands)	Level 1		Level 2		Level 3		Total
Cash equivalents	$	37,093	$	50,909	$	—	$	88,002
Total cash equivalents	37,093		50,909		—		88,002

Marketable securities:

U.S. Treasuries	—		84,596		—		84,596
Government securities	—		331,443		—		331,443
Corporate debt securities	—		541,695		—		541,695
Total marketable securities	—		957,734		—		957,734
Total cash equivalents and marketable securities	$	37,093	$	1,008,643	$	—	$	1,045,736

There were no transfers between Level 1 and Level 2 and we had no financial assets or liabilities that were classified as Level 3 at any point during the year ended December 31, 2022.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

Marketable Securities

12 Months Ended

Dec. 31, 2022

Investments, Debt and Equity Securities [Abstract]

Marketable Securities

Marketable securities at December 31, 2022 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:

U.S. Treasuries	$	68,175	$	3	$	(811)	$	67,367
Government securities	220,901		8		(5,289)		215,620
Corporate debt securities	363,263		1		(2,391)		360,873
Total Current	652,339		12		(8,491)		643,860

Non-current:
U.S. Treasuries	17,418		4		(193)		17,229
Government securities	117,475		7		(1,659)		115,823
Corporate debt securities	183,037		76		(2,291)		180,822
Total Non-current	317,930		87		(4,143)		313,874
Total marketable securities	$	970,269	$	99	$	(12,634)	$	957,734

Marketable securities at December 31, 2021 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:

U.S. Treasuries	$	269,109	$	—	$	(36)	$	269,073
Government securities	17,764		1		(10)		17,755
Corporate debt securities	530,490		3		(429)		530,064
Total Current	817,363		4		(475)		816,892

Non-current:
U.S. Treasuries	40,607		—		(23)		40,584
Government securities	148,820		—		(470)		148,350
Corporate debt securities	77,675		—		(234)		77,441
Total Non-current	267,102		—		(727)		266,375
Total marketable securities	$	1,084,465	$	4	$	(1,202)	$	1,083,267

There were no material realized gains or losses on marketable securities for the years ended December 31, 2022 and 2021.

At December 31, 2022 and 2021, we held 259 and 294 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 December 31, 2022 and December 31, 2021 related to these securities. The aggregate fair value of debt securities in an unrealized loss position at December 31, 2022 and 2021 was $868.2 million and $950.5 million, respectively. There were no individual securities that were in a significant unrealized loss position as of December 31, 2022 and 2021. 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 December 31, 2022 and 2021.

Inventory

12 Months Ended

Dec. 31, 2022

Inventory Disclosure [Abstract]

Inventory

Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following:


(In thousands)	December 31, 2022		December 31, 2021
Raw materials	$	—	$	—
Work-in-process	7,550		—
Finished goods	942		—
Total inventory	$	8,492	$	—

Leases

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

Leases

Our building leases are comprised of office and laboratory space under non-cancelable operating leases. These lease agreements have remaining lease terms of five 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.

On April 11, 2019, we entered into an agreement to lease approximately 13,000 square feet of office space located at 38 Sidney Street, Cambridge, Massachusetts, or the 38 Sidney Lease, with Thirty-Eight Sidney Street, LLC. The initial term of the 38 Sidney Lease commenced on May 1, 2019 and expires on February 29, 2028. At the end of the lease term, we have the option to extend the 38 Sidney Lease for two consecutive terms of five years at fair market rent at the time of the extension. The 38 Sidney Lease provides us with the right to lease additional space within the 38 Sidney Street building and also includes rent escalation clauses and a tenant improvement allowance of $1.0 million.

In connection with the 38 Sidney Lease, we also amended our existing building leases at 88 Sidney Street, Cambridge, Massachusetts and at 64 Sidney Street, Cambridge, Massachusetts to extend the initial terms of those leases by approximately three years through February 29, 2028. The amendments also provide us with the right to lease additional space at the 64 Sidney Street building. Our existing extension options for the 88 Sidney Street building and 64 Sidney Street building continue as set forth in the existing leases for those buildings.

The components of lease expense and other information related to leases were as follows:


(In millions)	2022		2021		2020
Operating lease costs	$	15,227	$	15,229	$	15,241
Cash paid for amounts included in the measurement of operating lease liabilities	17,035		14,411		14,424

We have not entered into any material short-term leases or financing leases as of December 31, 2022.

In arriving at the operating lease liabilities as of December 31, 2022, we applied the weighted-average incremental borrowing rate of 5.7% from inception over a weighted-average remaining lease term of 5.2 years. In arriving at the operating lease liabilities as of December 31, 2021, we applied the weighted-average incremental borrowing rate of 5.7% over a weighted-average remaining lease term of 6.2 years.

As of December 31, 2022, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter, were as follows:


(In thousands)
2023	$	16,651
2024	18,660
2025	19,507
2026	20,151
2027	20,755
Thereafter	3,479
Undiscounted minimum rental commitments	99,203
Interest	(13,544)
Total operating lease liabilities	$	85,659

We provided our landlord a standby letter of credit of $2.9 million as security for our leases. We are not required to maintain any cash collateral for the standby letter of credit.

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. We recorded operating sublease income of $4.1 million and $0.5 million for the years ended December 31, 2022 and December 31, 2021, respectively, in other income, net in the consolidated statements of operations. We received a security deposit from our sublessee of approximately $1.1 million which is recorded within other non-current assets on our consolidated balance sheet.

As of December 31, 2022, the future minimum lease payments to be received under the long-term sublease agreements were as follows:


(In thousands)
2023	4,329
2024	4,459
2025	1,101
Total	$	9,889

Accrued Expenses

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

Accrued Expenses

Accrued ExpensesAccrued expenses consisted of the following at December 31:


(In thousands)	2022		2021
Accrued compensation	$	18,105	$	19,818
Accrued research and development costs	8,425		5,980
Accrued professional fees	2,435		2,335
Accrued other	1,385		3,834
Total accrued expenses	$	30,350	$	31,967

Product Revenue

12 Months Ended

Dec. 31, 2022

Revenue from Contract with Customer [Abstract]

Product Revenue

We sell PYRUKYND®, our wholly owned product, to the Customers. The Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®.

Product revenue, net, was as follows for the years ended December 31:


(In thousands)	2022		2021		2020
Product revenue, net	$	11,740	$	—	$	—

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.

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the year ended December 31, 2022:


(In thousands)	Contractual Adjustments		Government Rebates		Returns		Total
Balance at December 31, 2021	$	—	$	—	$	—	$	—
Current provisions relating to sales in the current year	497		912		133		1,542
Adjustments relating to prior years	—		—		—		—
Payments/returns relating to sales in the current year	(432)		(339)				(771)
Payments/returns relating to sales in the prior years	—		—		—		—
Balance at December 31, 2022	$	65	$	573	$	133	$	771

There were no balances or activity related to product revenue allowance and reserve categories for the year ended December 31, 2021.

Total revenue-related reserves above, included in our consolidated balance sheets, are summarized as follows:


(In thousands)	December 31, 2022		December 31, 2021
Reduction of accounts receivable	$	60	$	—
Component of accrued expenses	711		—
Total revenue-related reserves	$	771	$	—

The following table presents changes in our contract assets during the year ended December 31, 2022:


(In thousands)	December 31, 2021		Additions		Deductions		December 31, 2022
Contract assets(1)
Accounts receivable, net	$	—	$	13,283	$	(11,077)	$	2,206

(1) Additions to contract assets relate to amounts billed to Customers for product sales, and deductions to contract assets primarily relate to collection of receivables during the reporting period.

There were no balances or activity related to contract assets for the year ended December 31, 2021.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-URI https://asc.fasb.org/topic&trid=49130388

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 10
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130551-203045

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 13
-Subparagraph (b)(2)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130556-203045

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 9
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130550-203045

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (e)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (d)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 15
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130558-203045

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

+ Details

Share-Based Payments

12 Months Ended

Dec. 31, 2022

Share-Based Payment Arrangement [Abstract]

Share-Based Payments

Stock incentive plans

In June 2013, our Board of Directors adopted and, in July 2013 our stockholders approved, the 2013 Stock Incentive Plan, or the 2013 Plan. The 2013 Plan became effective upon the closing of our initial public offering and provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards, RSUs, PSUs, and other stock-based awards to employees, non-employees and non-employee directors. Following the adoption of the 2013 Plan, we granted no further stock options or other awards under the 2007 Stock Incentive Plan, or the 2007 Plan. Any options or awards outstanding under the 2007 Plan at the time of adoption of the 2013 Plan remain outstanding and effective. As of December 31, 2022, the total number of shares reserved under the 2007 Plan and the 2013 Plan was 12,821,789, and we had 5,458,366 shares available for future issuance under the 2013 Plan.

The 2013 Plan provides for an annual increase, to be added on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2014 and continuing until the expiration of the 2013 Plan, equal to the lesser of (i) 2,000,000 shares of common stock, (ii) 4% of the outstanding shares of common stock on such date or (iii) an amount determined by our Board of Directors. On January 1, 2023, the annual increase for the 2013 Plan resulted in an additional 2,000,000 shares authorized for issuance.

Stock options

The following table summarizes the stock option activity of all stock incentive plans for the year ended December 31, 2022:


	Number of Stock Options	Weighted- Average Exercise Price		Weighted- Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2021	4,798,826	$	58.51	6.24	$	4,697
Granted	1,850,093	28.89
Exercised	(15,539)	11.50
Forfeited/Expired	(860,816)	60.91
Outstanding at December 31, 2022	5,772,564	$	48.81	6.50	$	5,362
Exercisable at December 31, 2022	3,497,660	$	58.24	4.84	$	3,141
Vested and expected to vest at December 31, 2022	5,772,564	$	48.81	6.50	$	5,362

The weighted-average grant date fair value of options granted was $15.64, $31.20 and $32.10 during the years ended December 31, 2022, 2021 and 2020, respectively. The total intrinsic value of options exercised was $0.3 million, $8.5 million and $10.4 million during the years ended December 31, 2022, 2021 and 2020, respectively.

At December 31, 2022, the total unrecognized compensation expense related to unvested stock option awards was $38.1 million, which we expect to recognize over a weighted-average period of approximately 2.71 years.

Restricted stock units

Upon vesting, each RSU entitles the holder to receive a specified number of shares of our common stock. The following table presents RSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	1,002,924	$	51.51
Granted	869,766	31.16
Vested	(531,304)	50.16
Forfeited	(223,465)	41.84
Unvested shares at December 31, 2022	1,117,921	$	38.30

As of December 31, 2022, there was approximately $25.6 million of total unrecognized compensation expense related to RSUs, which we expect to be recognized over a weighted-average period of 1.76 years.

Performance-based stock units

At the achievement of the performance-based and service-based vesting criteria, each PSU entitles the holder to receive a specified number of shares of our common stock. The following table presents PSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	234,059	$	54.28
Granted	337,243	30.33
Vested	(53,777)	54.28
Forfeited	(47,190)	49.06
Expired	(40,092)	56.52
Unvested shares at December 31, 2022	430,243	$	35.87

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 December 31, 2022, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement that we expect to recognize. There is $15.4 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

We have issued certain equity awards that contain market based vesting conditions, in which shares of stock are earned at vesting based on stock price performance. 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.

The following table presents MSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	42,695	$	41.50
Granted	—	—
Unvested shares at December 31, 2022	42,695	$	41.50

As of December 31, 2022, 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. On January 1, 2023, the annual increase for the 2013 ESPP resulted in an additional 509,091 shares authorized for issuance. We issued 104,867 shares and 94,888 shares during the years ended December 31, 2022 and 2021, respectively, under the 2013 ESPP. The 2013 ESPP provides participating employees with the opportunity to purchase up to an aggregate of 1,854,545 shares of our common stock. As of December 31, 2022, we had 1,289,780 shares available for future issuance under the 2013 ESPP.

Stock-based compensation expense

During the years ended December 31, 2022, 2021 and 2020, we recorded stock-based compensation expense for employee and non-employee stock options, RSUs, PSUs, ESPP shares and other stock-based awards. Stock-based compensation expense by award type included within the consolidated statements of operations is as follows:


(In thousands)	2022		2021		2020
Stock options	$	23,731	$	30,985	$	37,705
Restricted stock units	21,670		21,510		19,893
Performance-based stock units	2,919		—		1,760
Employee Stock Purchase Plan	976		1,013		1,463
Other stock awards	—		—		781
Total stock-based compensation expense	$	49,296	$	53,508	$	61,602

Expenses related to equity-based awards were allocated as follows in the consolidated statements of operations:


(In thousands)	2022		2021		2020
Research and development expense	$	20,988	$	24,527	$	27,119
Selling, general and administrative expense	28,308		28,981		34,483
Total stock-based compensation expense	$	49,296	$	53,508	$	61,602

No related tax benefits were recognized for the years ended December 31, 2022, 2021 and 2020.

The fair value of each stock option granted to employees and non-employees is estimated on the date of grant using the Black-Scholes option-pricing model. The following table summarizes the weighted average assumptions used in calculating the grant date fair value of the awards:


	2022		2021		2020
Risk-free interest rate	2.55	%	0.72	%	1.24	%
Expected dividend yield	—		—		—
Expected term (in years)	6.03		6.05		6.05
Expected volatility	55.30	%	61.72	%	73.80	%

Expected term

We use the “simplified method” as prescribed by the Securities and Exchange Commission Staff Accounting Bulletin No. 107, Share Based Payments, to estimate the expected term of stock option grants. Under this approach, the weighted-average expected life is presumed to be the average of the contractual term of ten years and the weighted-average vesting term of the stock options, taking into consideration multiple vesting tranches. We utilize this method due to lack of historical data and the plain-vanilla nature of our share-based awards.

Volatility

The expected volatility has been determined using Agios' historical volatilities for a period equal to the expected term of the option grant.

Risk-free rate

The risk-free rate is based on the yield curve of U.S. Treasury securities with periods commensurate with the expected term of the options being valued.

Dividends

We have never paid, and do not anticipate paying, any cash dividends in the foreseeable future, and, therefore, use an expected dividend yield of zero in the option-pricing model.

Forfeitures

We account for forfeitures as they occur and, therefore, do not estimate forfeitures.

Net Income (Loss) per Share

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

Net 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 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, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of December 31, 2022 are not considered to be common stock equivalents.

The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:


	Years ended December 31,
	2022	2021	2020
Stock options	5,772,564	4,798,826	6,143,046
Restricted stock units	1,117,921	1,002,924	1,284,378

Employee Stock Purchase Plan shares	42,026	39,864	46,439
Total	6,932,511	5,841,614	7,473,863

Income Taxes

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

Income Taxes

The domestic and foreign components of loss from continuing operations before income taxes are as follows:


(In thousands)	2022		2021		2020
Domestic	$	(231,767)	$	(356,665)	$	(330,669)
Foreign	(34)		155		1,364
Total	$	(231,801)	$	(356,510)	$	(329,305)

We did not have any material provision for income taxes for the years ended December 31, 2022, 2021 and 2020.

A reconciliation of the expected income tax benefit (expense) computed using the federal statutory income tax rate to our effective income tax rate is as follows for the years ended December 31, 2022, 2021 and 2020:


	2022		2021		2020
Income tax benefit computed at federal statutory tax rate	21.0	%	21.0	%	21.0	%
State taxes, net of federal benefit	2.9	%	2.6	%	2.5	%
Change in valuation allowance	(25.7)	%	(24.5)	%	(28.2)	%
General business credits and other credits	5.2	%	5.3	%	7.0	%
Permanent differences and other adjustments	(2.3)	%	(3.9)	%	(1.6)	%
Stock based compensation	(1.1)	%	(0.5)	%	(0.7)	%


Total	—	%	—	%	—	%

Deferred income taxes reflect the net tax effects of temporary differences between the carrying amount of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. Significant components of our deferred tax assets and liabilities for the years ended December 31, 2022 and 2021 are as follows:


(In thousands)	2022		2021
Deferred tax assets:
Net operating loss carryforwards	$	32,907	$	39,186

Tax credit carryforwards	163,780		152,128
Purchased intangible assets	11,583		12,150
Stock-based compensation	26,236		27,217
Operating lease liability	21,042		22,963
Non-deductible accruals and reserves, including inventory	3,992		4,033
Section 174 R&D expense	56,565		—

Total deferred tax assets	316,105		257,677
Depreciation and amortization	(3,767)		(3,168)
Operating lease right of use asset	(16,345)		(18,031)
Less: valuation allowance	(295,993)		(236,478)
Net deferred taxes	$	—	$	—

The Tax Cuts and Jobs Act (TCJA) requires taxpayers to capitalize and amortize research and experimental expenditures under section 174 for tax years beginning after December 31, 2021. This rule became effective for the Company during the year ending December 31, 2022 and resulted in the capitalization of research and development costs of $261.4 million. We will amortize these costs for tax purposes over 5 years if the research and development was performed in the U.S. and over 15 years if the research and development was performed outside the U.S.

As of December 31, 2022, we had net operating loss carryforwards, or NOLs, available to reduce state and foreign income taxes of approximately $1.2 billion and $65.2 million, respectively. At December 31, 2022, we also had available research and development tax credits for federal and state income tax purposes of approximately $16.9 million and $25.7 million, respectively. If not utilized, the credits begin to expire in 2039 and 2027 for federal and state income tax purposes, respectively. We engaged in clinical testing activities and incurred expenses that qualify for the federal orphan drug tax credit. At December 31, 2022, we had available orphan drug tax credits for federal purposes only of approximately $126.5 million. If not utilized, the orphan drug credits begin to expire in 2035.

As provided by Section 382 of the Internal Revenue Code of 1986, or Section 382, and similar state provisions, utilization of NOLs and tax credit carryforwards may be subject to substantial annual limitations due to ownership change limitations that have previously occurred or that could occur in the future. Ownership changes may limit the amount of NOLs and tax credit carryforwards that can be utilized annually to offset future taxable income and tax, respectively. In general, an ownership change, as defined by Section 382, results from transactions that increase the ownership of five percent stockholders in the stock of a corporation by more than 50 percent in the aggregate over a three year period. We completed a review of our changes in ownership through December 31, 2022 and determined that transactions have resulted in no ownership changes during the year ended December 31, 2022, as defined by Section 382. The impact of the historical ownership changes has been reflected in our deferred tax assets in the table above.

As required by ASC 740, we have evaluated the positive and negative evidence bearing upon the realizability of our deferred tax assets. Based on the weight of available evidence, both positive and negative, we recorded a valuation allowance of $296.0 million and $236.5 million as of December 31, 2022 and December 31, 2021, respectively, because we have determined that it is more likely than not that these assets will not be fully realized. The valuation allowance increased by $59.5 million for the year ended December 31, 2022 primarily due to the Section 174 R&D expense capitalization and decreased by $358.3 million for the year ended December 31, 2021 primarily due to the utilization of net operating losses and tax credits.

The following table presents our change in valuation allowance for the years ended December 31, 2022 and, 2021:


(in thousands)	2022		2021
Valuation allowance at the beginning of the year	$	236,478	$	594,752
Increase (decrease) for the current period	59,515		(358,274)
Valuation allowance at the end of the year	$	295,993	$	236,478

As of December 31, 2022, the unremitted earnings of our foreign subsidiaries are not material. We have not provided for U.S. income taxes or foreign withholding taxes on these earnings as it is our current intention to permanently reinvest these earnings outside the U.S. The tax liability on these earnings is also not material. Events that could trigger a tax liability include, but are not limited to, distributions, reorganizations or restructurings and/or tax law changes.

We apply the accounting guidance in ASC 740 related to accounting for uncertainty in income taxes. Our reserves related to taxes are based on a determination of whether, and how much of, a tax benefit taken by us in our tax filings or positions is more likely than not to be realized following resolution of any potential contingencies present related to the tax benefit.

The following table presents our unrecognized tax benefits activity for the years ended December 31, 2022 and 2021:


(In thousands)	2022		2021
Unrecognized tax benefits at the beginning of the year	$	24,220	$	21,131
Gross increases - current period tax positions	1,970		3,089
Unrecognized tax benefits at the end of the year	$	26,190	$	24,220

We will recognize interest and penalties related to uncertain tax positions above the line as an expense to continuing operations. As of December 31, 2022 and 2021, we had no accrued interest or penalties related to uncertain tax positions and no such amounts have been recognized. If all of the Company’s unrecognized tax benefits as of December 31, 2022 were to become recognizable in the future, we would record $26.2 million of unrecognized tax benefits. The uncertain tax position does not impact our effective income tax rate due to the full valuation allowance.

We are subject to taxation in the United States, Switzerland, Netherlands, Germany, Italy and France. The statute of limitations for assessment by the IRS and state tax authorities is open for tax years ending December 31, 2022, 2021, 2020, and 2019, although carryforward attributes that were generated for tax years prior to 2019 may still be adjusted upon examination by the IRS or state tax authorities if they either have been, or will be, used in a future period. The statute of limitations for assessment in Switzerland remains open for tax years ending December 31, 2022, 2021, 2020, and 2019. The Company’s subsidiaries in the Netherlands and Germany were incorporated in 2019 and therefore the statute of limitations for assessment that remain open in these jurisdictions are for the tax years ending December 31, 2022, 2021, 2020 and 2019. The Company’s subsidiaries in Italy and France were incorporated in 2020 and therefore the statute of limitations for assessment that remain open in these jurisdictions are for the tax years ending December 31, 2022, 2021 and 2020. There are currently no federal, state or foreign audits in progress.

As of December 31, 2022 and 2021, we had an income tax receivable of $0.3 million and $2.9 million, respectively, recorded within prepaid expenses and other assets.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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+ Details

Property and Equipment, net

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

Property and Equipment, net

Property and equipment, net consisted of the following at December 31:


(In thousands)	2022		2021
Laboratory equipment	$	23,182	$	22,165
Computer equipment and software	6,179		6,913
Leasehold improvements	37,277		32,726
Furniture and fixtures	3,514		3,035
Office equipment	2,248		1,690
Construction in progress	657		7,368
Total property and equipment	73,057		73,897
Less: accumulated depreciation	(50,070)		(44,974)
Total property and equipment, net	$	22,987	$	28,923

Depreciation expense for the years ended December 31, 2022, 2021 and 2020 was $8.4 million, $8.8 million and $9.4 million, respectively.

Common Stock	12 Months Ended
Common Stock	Dec. 31, 2022
Equity [Abstract]
Common Stock	Common StockWe are authorized to issue 125,000,000 shares of our common stock. Holders of common stock are entitled to one vote per share. Additionally, holders of common stock are entitled to receive dividends, if and when declared by our board of directors, and to share ratably in our assets legally available for distribution to our shareholders in the event of liquidation.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
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+ Details

Share Repurchase Program	12 Months Ended
Share Repurchase Program	Dec. 31, 2022
Equity [Abstract]
Share Repurchase Program	Share Repurchase Program On March 25, 2021, we announced that our board of directors authorized the Repurchase Program for the repurchase of up to $1.2 billion of our outstanding shares of common stock. On March 31, 2021, in connection with the Repurchase Program, we entered into a definitive share repurchase agreement with BMS to repurchase 7.1 million shares of our common stock held by certain subsidiaries of BMS for an aggregate purchase price of $344.5 million, or $48.38 per share. This repurchase was completed on April 5, 2021. Further, on April 2, 2021, in connection with the Repurchase Program, we entered into a Rule 10b5-1 repurchase plan to which we may repurchase up to $600.0 million of shares of our common stock. As of December 31, 2022, we have repurchased approximately 9.1 million shares of common stock for $458.0 million, or $50.35 per share, under the Rule 10b5-1 repurchase plan. In total, as of December 31, 2022, we have repurchased 16.2 million shares of common stock for $802.5 million, or $49.49 per share, under the Repurchase Program. On October 5, 2021, we terminated our Rule 10b5-1 share repurchase plan and on October 13, 2021, we entered into a Rule 10b-18 repurchase plan that allows us to conduct open market repurchases over time up to our remaining authorization under the Repurchase Program. We have paused our share repurchases for the foreseeable future. Repurchased shares are held as treasury stock until they are retired or re-issued. Treasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock. Repurchases of our common stock are accounted for as of the settlement date. There were no retirements or re-issuances of treasury stock during the year ended December 31, 2022.

Discontinued Operations

12 Months Ended

Dec. 31, 2022

Discontinued Operations and Disposal Groups [Abstract]

Discontinued Operations

Discontinued OperationsOn March 31, 2021, we completed the sale of our oncology business to Servier. We determined the sale of the oncology business represented a strategic shift that had a major effect on our business and therefore met the criteria for classification as discontinued operations at March 31, 2021. Accordingly, the oncology business is reported as discontinued operations in accordance with ASC 205-20, Discontinued Operations. The related assets and liabilities of the oncology business were classified as assets and liabilities of discontinued operations in the consolidated balance sheets and the results of operations from the oncology business as discontinued operations in the consolidated statements of operations. Applicable amounts in prior years have been recast to conform to this discontinued operations presentation. We recognized a gain on the sale of the oncology business upon closing.

The following table presents the net liabilities transferred for the sale of the oncology business at March 31, 2021:


(in thousands)	March 31, 2021
Assets
Current assets:
Accounts receivable, net	$	25,386
Collaboration receivable – related party	2,253
Collaboration receivable – other	2,438
Inventory	16,190
Prepaid expenses and other current assets	7,125
Total current assets of discontinued operations	53,392
Other non-current assets	2,234
Total assets of discontinued operations	$	55,626

Liabilities
Current liabilities:
Accounts payable	$	4,245
Accrued expenses	30,288
Total current liabilities of discontinued operations	34,533
Liability related to the sale of future revenue, net of debt issuance costs	264,281
Total liabilities of discontinued operations	298,814
Net liabilities distributed to Servier	$	(243,188)

The following table presents the gain on the sale for the year ended December 31, 2021:


(in thousands)	December 31, 2021
Cash proceeds	$	1,802,936
Less: transaction and insurance costs	(53,573)
Plus: net liabilities distributed, including working capital adjustment	239,770
Gain on sale, pre-tax	1,989,133
Income tax expense	(12,799)
Gain on sale, net of tax	$	1,976,334

As of December 31, 2022 and December 31, 2021, there were no assets or liabilities classified as discontinued operations.

The following table presents the financial results of the discontinued operations:


(in thousands)	2021		2020
Revenues:
Product revenue, net	$	36,909	$	121,089
Collaboration revenue – related party	1,350		68,274
Collaboration revenue – other	491		3,571
Royalty revenue – related party	2,659		10,262
Total revenue	41,409		203,196
Cost and expenses:
Cost of sales	706		2,805
Research and development	41,564		146,659
Selling, general and administrative	8,551		33,965
Total cost and expenses	50,821		183,429
(Loss) income from discontinued operations	(9,412)		19,767
Non-cash interest expense for the sale of future revenue	(5,697)		(17,832)
Gain on the sale of the oncology business	1,989,133		—
Income from discontinued operations, pre-tax	1,974,024		1,935
Income tax expense	(12,799)		—
Net income from discontinued operations	$	1,961,225	$	1,935

In accordance with ASC 205-20, only expenses specifically identifiable and related to a business to be disposed may be presented in discontinued operations. As such, the research and development, marketing, selling and general and administrative expenses in discontinued operations include corporate costs incurred directly to solely support our oncology business.

We also entered into a Transition Services Agreement with Servier, through which we provided transitional services related to discovery, clinical development, technical operations, commercial and general and administrative related activities through March 31, 2022.

The milestone payment for approval of vorasidenib and royalty payments related to vorasidenib and TIBSOVO® represent contingent consideration. Contingent consideration has been accounted for as a gain contingency in accordance with ASC 450, Contingencies, and will be recognized in earnings in the period when realizable. As described in Note 1, Nature of Business, on October 27, 2022, we sold our rights to future contingent payments to entities affiliated with Sagard and recognized income of $127.9 million within the gain on sale of contingent payments line item in our consolidated statements of operations for the year ended December 31, 2022.

Commitments and Contingent Liabilities	12 Months Ended
Commitments and Contingent Liabilities	Dec. 31, 2022
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingent Liabilities	Commitments and Contingent Liabilities Manufacturing Commitments We are party to various agreements with contract manufacturing organizations that we are not contractually able to terminate for convenience and avoid any and all future obligations to the vendors. Under such agreements, we are obligated to make certain minimum payments, with the exact amounts in the event of termination to be based on the timing of the termination and the exact terms of the agreement. Legal Contingencies From time to time, we may be involved in disputes and legal proceedings in the ordinary course of business. These proceedings may include allegations of infringement of intellectual property, employment or other matters. We do not have any ongoing legal proceedings that, based on our estimates, could have a material effect on our consolidated financial statements.

Defined Contribution Benefit Plan	12 Months Ended
Defined Contribution Benefit Plan	Dec. 31, 2022
Retirement Benefits [Abstract]
Defined Contribution Benefit Plan	Defined Contribution Benefit PlanWe sponsor a 401(k) retirement plan, in which substantially all of our full-time employees are eligible to participate. Participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations. We will make matching contributions equal to 100% of the employee’s contributions, subject to a maximum of 4% of eligible compensation.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/exampleRef
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+ Details

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Principles of consolidation

Use of estimates

Cash and cash equivalents

We consider highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. Cash equivalents are stated at fair value.

Accounts receivable, net

Inventory

Revenue recognition

Product Revenue

allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products.

Cost of sales

Marketable securities

Fair value measurements

Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities.

There have been no changes to the valuation methods during the years ended December 31, 2022 and 2021. We evaluate transfers between levels at the end of each reporting period.

The carrying amounts of other receivables, prepaid expenses and other current assets, accounts payable, and accrued expenses approximate their fair values due to their short-term maturities.

Concentrations of credit risk

Property and equipment

Property and equipment is stated at cost, and depreciated using the straight-line method over the estimated useful lives of the respective assets:


						Years
Laboratory equipment						5
Computer equipment and software						3
Furniture and fixtures						5
Office equipment						5

Leasehold improvements are amortized over the lesser of the remaining lease term or the estimated useful life of the improvement.

Impairment of long-lived assets

Leases

We have not entered into any material short-term leases or financing leases as of December 31, 2022.

Research and development costs

Stock-based compensation

Income taxes

provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized.

Comprehensive income (loss)

Net income (loss) per share

Segment and geographic information

Discontinued operations

Treasury stock

Treasury stockTreasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock.

Recent accounting pronouncements

Subsequent events

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Estimated Useful Lives of Property and Equipment

Property and equipment is stated at cost, and depreciated using the straight-line method over the estimated useful lives of the respective assets:


						Years
Laboratory equipment						5
Computer equipment and software						3
Furniture and fixtures						5
Office equipment						5

Fair Value Measurements (Tables)

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis


(In thousands)	Level 1		Level 2		Level 3		Total
Cash equivalents	$	37,093	$	50,909	$	—	$	88,002
Total cash equivalents	37,093		50,909		—		88,002

Marketable securities:

U.S. Treasuries	—		84,596		—		84,596
Government securities	—		331,443		—		331,443
Corporate debt securities	—		541,695		—		541,695
Total marketable securities	—		957,734		—		957,734
Total cash equivalents and marketable securities	$	37,093	$	1,008,643	$	—	$	1,045,736

Marketable Securities (Tables)

12 Months Ended

Dec. 31, 2022

Investments, Debt and Equity Securities [Abstract]

Summary of Marketable Securities

Marketable securities at December 31, 2022 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:

U.S. Treasuries	$	68,175	$	3	$	(811)	$	67,367
Government securities	220,901		8		(5,289)		215,620
Corporate debt securities	363,263		1		(2,391)		360,873
Total Current	652,339		12		(8,491)		643,860

Non-current:
U.S. Treasuries	17,418		4		(193)		17,229
Government securities	117,475		7		(1,659)		115,823
Corporate debt securities	183,037		76		(2,291)		180,822
Total Non-current	317,930		87		(4,143)		313,874
Total marketable securities	$	970,269	$	99	$	(12,634)	$	957,734

Marketable securities at December 31, 2021 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:

U.S. Treasuries	$	269,109	$	—	$	(36)	$	269,073
Government securities	17,764		1		(10)		17,755
Corporate debt securities	530,490		3		(429)		530,064
Total Current	817,363		4		(475)		816,892

Non-current:
U.S. Treasuries	40,607		—		(23)		40,584
Government securities	148,820		—		(470)		148,350
Corporate debt securities	77,675		—		(234)		77,441
Total Non-current	267,102		—		(727)		266,375
Total marketable securities	$	1,084,465	$	4	$	(1,202)	$	1,083,267

Inventory (Tables)

12 Months Ended

Dec. 31, 2022

Inventory Disclosure [Abstract]

Schedule of Inventory

Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following:


(In thousands)	December 31, 2022		December 31, 2021
Raw materials	$	—	$	—
Work-in-process	7,550		—
Finished goods	942		—
Total inventory	$	8,492	$	—

Leases (Tables)

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

Schedule of Lease Cost and Other Information

The components of lease expense and other information related to leases were as follows:


(In millions)	2022		2021		2020
Operating lease costs	$	15,227	$	15,229	$	15,241
Cash paid for amounts included in the measurement of operating lease liabilities	17,035		14,411		14,424

Schedule of Undiscounted Minimum Rental Commitments

As of December 31, 2022, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter, were as follows:


(In thousands)
2023	$	16,651
2024	18,660
2025	19,507
2026	20,151
2027	20,755
Thereafter	3,479
Undiscounted minimum rental commitments	99,203
Interest	(13,544)
Total operating lease liabilities	$	85,659

Schedule of Future Minimum Lease Payments to be Received

As of December 31, 2022, the future minimum lease payments to be received under the long-term sublease agreements were as follows:


(In thousands)
2023	4,329
2024	4,459
2025	1,101
Total	$	9,889

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

Summary of Accrued Expenses

Accrued expenses consisted of the following at December 31:


(In thousands)	2022		2021
Accrued compensation	$	18,105	$	19,818
Accrued research and development costs	8,425		5,980
Accrued professional fees	2,435		2,335
Accrued other	1,385		3,834
Total accrued expenses	$	30,350	$	31,967

Product Revenue (Tables)

12 Months Ended

Dec. 31, 2022

Revenue from Contract with Customer [Abstract]

Schedule of Product Revenue

Product revenue, net, was as follows for the years ended December 31:


(In thousands)	2022		2021		2020
Product revenue, net	$	11,740	$	—	$	—

Schedule of Product Revenue Allowance and Reserves

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the year ended December 31, 2022:


(In thousands)	Contractual Adjustments		Government Rebates		Returns		Total
Balance at December 31, 2021	$	—	$	—	$	—	$	—
Current provisions relating to sales in the current year	497		912		133		1,542
Adjustments relating to prior years	—		—		—		—
Payments/returns relating to sales in the current year	(432)		(339)				(771)
Payments/returns relating to sales in the prior years	—		—		—		—
Balance at December 31, 2022	$	65	$	573	$	133	$	771

Schedule of Revenue Related Reserves

Total revenue-related reserves above, included in our consolidated balance sheets, are summarized as follows:


(In thousands)	December 31, 2022		December 31, 2021
Reduction of accounts receivable	$	60	$	—
Component of accrued expenses	711		—
Total revenue-related reserves	$	771	$	—

Schedule of Changes in Contract Assets

The following table presents changes in our contract assets during the year ended December 31, 2022:


(In thousands)	December 31, 2021		Additions		Deductions		December 31, 2022
Contract assets(1)
Accounts receivable, net	$	—	$	13,283	$	(11,077)	$	2,206

(1) Additions to contract assets relate to amounts billed to Customers for product sales, and deductions to contract assets primarily relate to collection of receivables during the reporting period.

Share-Based Payments (Tables)

12 Months Ended

Dec. 31, 2022

Share-Based Payment Arrangement [Abstract]

Summary of Company's Stock Option Activity of all Stock Incentive Plans

The following table summarizes the stock option activity of all stock incentive plans for the year ended December 31, 2022:


	Number of Stock Options	Weighted- Average Exercise Price		Weighted- Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2021	4,798,826	$	58.51	6.24	$	4,697
Granted	1,850,093	28.89
Exercised	(15,539)	11.50
Forfeited/Expired	(860,816)	60.91
Outstanding at December 31, 2022	5,772,564	$	48.81	6.50	$	5,362
Exercisable at December 31, 2022	3,497,660	$	58.24	4.84	$	3,141
Vested and expected to vest at December 31, 2022	5,772,564	$	48.81	6.50	$	5,362

Schedule of Restricted Stock Unit Activity

The following table presents RSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	1,002,924	$	51.51
Granted	869,766	31.16
Vested	(531,304)	50.16
Forfeited	(223,465)	41.84
Unvested shares at December 31, 2022	1,117,921	$	38.30

Schedule of Performance-Based Unit Activity

The following table presents PSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	234,059	$	54.28
Granted	337,243	30.33
Vested	(53,777)	54.28
Forfeited	(47,190)	49.06
Expired	(40,092)	56.52
Unvested shares at December 31, 2022	430,243	$	35.87

Schedule of Market-Based Unit Activity

The following table presents MSU activity for the year ended December 31, 2022:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2021	42,695	$	41.50
Granted	—	—
Unvested shares at December 31, 2022	42,695	$	41.50

Schedule of Stock-Based Compensation Expense by Award Type Included Within the Condensed Consolidated Statements of Operations

Stock-based compensation expense by award type included within the consolidated statements of operations is as follows:


(In thousands)	2022		2021		2020
Stock options	$	23,731	$	30,985	$	37,705
Restricted stock units	21,670		21,510		19,893
Performance-based stock units	2,919		—		1,760
Employee Stock Purchase Plan	976		1,013		1,463
Other stock awards	—		—		781
Total stock-based compensation expense	$	49,296	$	53,508	$	61,602

Stock-Based Compensation Expense for Employee and Non-Employee Stock Options, Restricted Stock Units, Performance-Based Stock Options, Performance-Based Stock Units and Employee Stock Purchase Plan Shares

Expenses related to equity-based awards were allocated as follows in the consolidated statements of operations:


(In thousands)	2022		2021		2020
Research and development expense	$	20,988	$	24,527	$	27,119
Selling, general and administrative expense	28,308		28,981		34,483
Total stock-based compensation expense	$	49,296	$	53,508	$	61,602

Schedule of Grant Date Fair Value Option Award Weighted Average Assumptions Used

The following table summarizes the weighted average assumptions used in calculating the grant date fair value of the awards:


	2022		2021		2020
Risk-free interest rate	2.55	%	0.72	%	1.24	%
Expected dividend yield	—		—		—
Expected term (in years)	6.03		6.05		6.05
Expected volatility	55.30	%	61.72	%	73.80	%

Net Income (Loss) per Share (Tables)

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

Common Stock Excluded from Calculation of Diluted Earnings Per Share


	Years ended December 31,
	2022	2021	2020
Stock options	5,772,564	4,798,826	6,143,046
Restricted stock units	1,117,921	1,002,924	1,284,378

Employee Stock Purchase Plan shares	42,026	39,864	46,439
Total	6,932,511	5,841,614	7,473,863

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

Schedule of Domestic and Foreign Components of Loss Before Income Taxes

The domestic and foreign components of loss from continuing operations before income taxes are as follows:


(In thousands)	2022		2021		2020
Domestic	$	(231,767)	$	(356,665)	$	(330,669)
Foreign	(34)		155		1,364
Total	$	(231,801)	$	(356,510)	$	(329,305)

Reconciliation of Expected Income Tax Benefit (Expense) Computed Using Federal Statutory Income Tax Rate


	2022		2021		2020
Income tax benefit computed at federal statutory tax rate	21.0	%	21.0	%	21.0	%
State taxes, net of federal benefit	2.9	%	2.6	%	2.5	%
Change in valuation allowance	(25.7)	%	(24.5)	%	(28.2)	%
General business credits and other credits	5.2	%	5.3	%	7.0	%
Permanent differences and other adjustments	(2.3)	%	(3.9)	%	(1.6)	%
Stock based compensation	(1.1)	%	(0.5)	%	(0.7)	%


Total	—	%	—	%	—	%

Company's Deferred Tax Assets and Liabilities

Significant components of our deferred tax assets and liabilities for the years ended December 31, 2022 and 2021 are as follows:


(In thousands)	2022		2021
Deferred tax assets:
Net operating loss carryforwards	$	32,907	$	39,186

Tax credit carryforwards	163,780		152,128
Purchased intangible assets	11,583		12,150
Stock-based compensation	26,236		27,217
Operating lease liability	21,042		22,963
Non-deductible accruals and reserves, including inventory	3,992		4,033
Section 174 R&D expense	56,565		—

Total deferred tax assets	316,105		257,677
Depreciation and amortization	(3,767)		(3,168)
Operating lease right of use asset	(16,345)		(18,031)
Less: valuation allowance	(295,993)		(236,478)
Net deferred taxes	$	—	$	—

Changes in Valuation Allowance

The following table presents our change in valuation allowance for the years ended December 31, 2022 and, 2021:


(in thousands)	2022		2021
Valuation allowance at the beginning of the year	$	236,478	$	594,752
Increase (decrease) for the current period	59,515		(358,274)
Valuation allowance at the end of the year	$	295,993	$	236,478

Schedule of Unrecognized Tax Benefits Rollforward

The following table presents our unrecognized tax benefits activity for the years ended December 31, 2022 and 2021:


(In thousands)	2022		2021
Unrecognized tax benefits at the beginning of the year	$	24,220	$	21,131
Gross increases - current period tax positions	1,970		3,089
Unrecognized tax benefits at the end of the year	$	26,190	$	24,220

Property and Equipment, net (Tables)

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

Summary of Property and Equipment

Property and equipment, net consisted of the following at December 31:


(In thousands)	2022		2021
Laboratory equipment	$	23,182	$	22,165
Computer equipment and software	6,179		6,913
Leasehold improvements	37,277		32,726
Furniture and fixtures	3,514		3,035
Office equipment	2,248		1,690
Construction in progress	657		7,368
Total property and equipment	73,057		73,897
Less: accumulated depreciation	(50,070)		(44,974)
Total property and equipment, net	$	22,987	$	28,923

Discontinued Operations and Disposal Groups (Tables)

12 Months Ended

Dec. 31, 2022

Discontinued Operations and Disposal Groups [Abstract]

Disposal Groups, Including Discontinued Operations

The following table presents the net liabilities transferred for the sale of the oncology business at March 31, 2021:


(in thousands)	March 31, 2021
Assets
Current assets:
Accounts receivable, net	$	25,386
Collaboration receivable – related party	2,253
Collaboration receivable – other	2,438
Inventory	16,190
Prepaid expenses and other current assets	7,125
Total current assets of discontinued operations	53,392
Other non-current assets	2,234
Total assets of discontinued operations	$	55,626

Liabilities
Current liabilities:
Accounts payable	$	4,245
Accrued expenses	30,288
Total current liabilities of discontinued operations	34,533
Liability related to the sale of future revenue, net of debt issuance costs	264,281
Total liabilities of discontinued operations	298,814
Net liabilities distributed to Servier	$	(243,188)

The following table presents the gain on the sale for the year ended December 31, 2021:


(in thousands)	December 31, 2021
Cash proceeds	$	1,802,936
Less: transaction and insurance costs	(53,573)
Plus: net liabilities distributed, including working capital adjustment	239,770
Gain on sale, pre-tax	1,989,133
Income tax expense	(12,799)
Gain on sale, net of tax	$	1,976,334

The following table presents the financial results of the discontinued operations:


(in thousands)	2021		2020
Revenues:
Product revenue, net	$	36,909	$	121,089
Collaboration revenue – related party	1,350		68,274
Collaboration revenue – other	491		3,571
Royalty revenue – related party	2,659		10,262
Total revenue	41,409		203,196
Cost and expenses:
Cost of sales	706		2,805
Research and development	41,564		146,659
Selling, general and administrative	8,551		33,965
Total cost and expenses	50,821		183,429
(Loss) income from discontinued operations	(9,412)		19,767
Non-cash interest expense for the sale of future revenue	(5,697)		(17,832)
Gain on the sale of the oncology business	1,989,133		—
Income from discontinued operations, pre-tax	1,974,024		1,935
Income tax expense	(12,799)		—
Net income from discontinued operations	$	1,961,225	$	1,935

- Definition

Tabular disclosure of information related to a disposal group. Includes, but is not limited to, a discontinued operation, disposal classified as held-for-sale or disposed of by means other than sale or disposal of an individually significant component.

+ References

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Reference 3: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 4: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 8: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 9: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 13: http://fasb.org/us-gaap/role/ref/legacyRef
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+ Details

Nature of Business - Additional Information (Details) $ / shares in Units, $ in Thousands			12 Months Ended			21 Months Ended
	Apr. 05, 2021 USD ($) $ / shares shares	Mar. 31, 2021 USD ($)	Dec. 31, 2022 USD ($) $ / shares shares	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)	Dec. 31, 2022 USD ($) $ / shares shares	Apr. 02, 2021 USD ($)	Mar. 25, 2021 USD ($)	Apr. 30, 2020 USD ($)
Subsidiary, Sale of Stock [Line Items]
Royalty income from gain on sale of oncology business			$ 9,851	$ 6,639	$ 0
Gain on sale of contingent payments			127,853	0	$ 0
Stock repurchase program, authorized amount								$ 1,200,000
Repurchase of common stock				802,486
Cash, cash equivalents and marketable securities			$ 1,100,000			$ 1,100,000
Repurchase Program
Subsidiary, Sale of Stock [Line Items]
Stock repurchase program, authorized amount								$ 1,200,000
Shares repurchased (in shares) \| shares						16,200,000
Repurchase of common stock						$ 802,500
Shares repurchased (in usd per share) \| $ / shares						$ 49.49
BMS Repurchase
Subsidiary, Sale of Stock [Line Items]
Shares repurchased (in shares) \| shares	7,100,000
Repurchase of common stock	$ 344,500
Shares repurchased (in usd per share) \| $ / shares	$ 48.38
Rule 10b5-1 Repurchase
Subsidiary, Sale of Stock [Line Items]
Stock repurchase program, authorized amount							$ 600,000
Shares repurchased (in shares) \| shares			9,100,000
Repurchase of common stock			$ 458,000
Shares repurchased (in usd per share) \| $ / shares			$ 50.35
October 13 2021 Repurchase Program , Rule 10b-18 Repurchase
Subsidiary, Sale of Stock [Line Items]
Shares repurchased (in shares) \| shares			0
Repurchase Program
Subsidiary, Sale of Stock [Line Items]
Shares repurchased (in shares) \| shares			16,200,000
Repurchase of common stock			$ 802,500
Shares repurchased (in usd per share) \| $ / shares			$ 49.49
U.S. \| TIBSOVO
Subsidiary, Sale of Stock [Line Items]
Earn-out revenue for royalties				6,600
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business
Subsidiary, Sale of Stock [Line Items]
Cash proceeds		$ 1,800,000		$ 1,802,936
Contingent milestone payment		$ 200,000
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business \| U.S. \| TIBSOVO
Subsidiary, Sale of Stock [Line Items]
Contingent royalty payment		0.05
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business \| U.S. \| vorasidenib
Subsidiary, Sale of Stock [Line Items]
Contingent royalty payment		0.15
At-the-market Offering
Subsidiary, Sale of Stock [Line Items]
Maximum value of shares issued									$ 250,000
Value of common stock reserved for future issuance			$ 250,000			$ 250,000
2010 Agreement
Subsidiary, Sale of Stock [Line Items]
Milestone payment for achievement of specified ex-U.S. commercial milestone event		$ 25,000

Summary of Significant Accounting Policies - Additional Information (Details)	12 Months Ended
	Dec. 31, 2022 segment
Schedule of Significant Accounting Policies [Line Items]
Minimum period to liquidate	12 months
Number of operating segments	1
Minimum
Schedule of Significant Accounting Policies [Line Items]
Investment maturity period, non-current	1 year
Maximum
Schedule of Significant Accounting Policies [Line Items]
Investment maturity period, current	1 year
Investment maturity period, non-current	2 years

Summary of Significant Accounting Policies - Estimated Useful Lives of Property and Equipment (Details)	12 Months Ended
	Dec. 31, 2022
Laboratory equipment
Property, Plant and Equipment [Line Items]
Estimated useful lives of property and equipment (in years)	5 years
Computer equipment and software
Property, Plant and Equipment [Line Items]
Estimated useful lives of property and equipment (in years)	3 years
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Estimated useful lives of property and equipment (in years)	5 years
Office equipment
Property, Plant and Equipment [Line Items]
Estimated useful lives of property and equipment (in years)	5 years

Fair Value Measurements - Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	$ 957,734	$ 1,083,267
Fair Value, Measurements, Recurring
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	88,002
Total marketable securities	957,734
Total cash equivalents and marketable securities	1,045,736
Fair Value, Measurements, Recurring \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	84,596
Fair Value, Measurements, Recurring \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	331,443
Fair Value, Measurements, Recurring \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	541,695
Fair Value, Measurements, Recurring \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	88,002
Fair Value, Measurements, Recurring \| Level 1
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	37,093
Total marketable securities	0
Total cash equivalents and marketable securities	37,093
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 1 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	37,093
Fair Value, Measurements, Recurring \| Level 2
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	50,909
Total marketable securities	957,734
Total cash equivalents and marketable securities	1,008,643
Fair Value, Measurements, Recurring \| Level 2 \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	84,596
Fair Value, Measurements, Recurring \| Level 2 \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	331,443
Fair Value, Measurements, Recurring \| Level 2 \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	541,695
Fair Value, Measurements, Recurring \| Level 2 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	50,909
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 \| 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, Recurring \| Level 3 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	$ 0

Fair Value Measurements - Additional Information (Details)	Dec. 31, 2022 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 - Summary of Marketable Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	$ 970,269	$ 1,084,465
Unrealized Gains	99	4
Unrealized Losses	(12,634)	(1,202)
Fair Value	957,734	1,083,267
Current:
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	652,339	817,363
Unrealized Gains	12	4
Unrealized Losses	(8,491)	(475)
Fair Value	643,860	816,892
Current: \| U.S. Treasuries
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	68,175	269,109
Unrealized Gains	3	0
Unrealized Losses	(811)	(36)
Fair Value	67,367	269,073
Current: \| Government securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	220,901	17,764
Unrealized Gains	8	1
Unrealized Losses	(5,289)	(10)
Fair Value	215,620	17,755
Current: \| Corporate debt securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	363,263	530,490
Unrealized Gains	1	3
Unrealized Losses	(2,391)	(429)
Fair Value	360,873	530,064
Non-current:
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	317,930	267,102
Unrealized Gains	87	0
Unrealized Losses	(4,143)	(727)
Fair Value	313,874	266,375
Non-current: \| U.S. Treasuries
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	17,418	40,607
Unrealized Gains	4	0
Unrealized Losses	(193)	(23)
Fair Value	17,229	40,584
Non-current: \| Government securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	117,475	148,820
Unrealized Gains	7	0
Unrealized Losses	(1,659)	(470)
Fair Value	115,823	148,350
Non-current: \| Corporate debt securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	183,037	77,675
Unrealized Gains	76	0
Unrealized Losses	(2,291)	(234)
Fair Value	$ 180,822	$ 77,441

Marketable Securities - Additional Information (Details)	12 Months Ended
	Dec. 31, 2022 USD ($) security	Dec. 31, 2021 USD ($) security
Investments, Debt and Equity Securities [Abstract]
Realized gain (loss) on marketable securities \| $	$ 0	$ 0
Number of debt securities in an unrealized loss position for less than one year \| security	259	294
Aggregate fair value of debt securities in an unrealized loss position \| $	$ 868,200,000	$ 950,500,000
Number of unrealized loss position \| security	0	0

Inventory - Schedule of Inventory (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Inventory Disclosure [Abstract]
Raw materials	$ 0	$ 0
Work-in-process	7,550	0
Finished goods	942	0
Total inventory	$ 8,492	$ 0

Leases - Additional Information (Details) ft² in Thousands, $ in Millions		12 Months Ended
	Apr. 11, 2019 USD ($) ft² leaseTerm	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Apr. 30, 2022 ft²	Aug. 31, 2021 ft²
Lessee, Lease, Description [Line Items]
Remaining lease terms		5 years
Weighted-average incremental borrowing rate		5.70%	5.70%
Weighted-average remaining lease term		5 years 2 months 12 days	6 years 2 months 12 days
Standby letter of credit		$ 2.9
38 Sidney Street Lease
Lessee, Lease, Description [Line Items]
Number of square feet of office space (in square feet) \| ft²	13
Number of optional terms available at end of current lease term (in lease terms) \| leaseTerm	2
Term for extension of operating lease (in years)	5 years
Tenant improvement allowance	$ 1.0
Area of premises subleased (in square feet) \| ft²				27	13
Sublease income		4.1	$ 0.5
38 Sidney Street Lease \| Other Noncurrent Assets
Lessee, Lease, Description [Line Items]
Security deposit liability		$ 1.1
64 Sidney Street Lease
Lessee, Lease, Description [Line Items]
Term for extension of operating lease (in years)	3 years
88 Sidney Street Lease
Lessee, Lease, Description [Line Items]
Term for extension of operating lease (in years)	3 years

Leases - Schedule of Lease Cost and Other Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Leases [Abstract]
Operating lease costs	$ 15,227	$ 15,229	$ 15,241
Cash paid for amounts included in the measurement of operating lease liabilities	$ 17,035	$ 14,411	$ 14,424

Leases - Schedule of Undiscounted Minimum Rental Commitments (Details) $ in Thousands	Dec. 31, 2022 USD ($)
Leases [Abstract]
2023	$ 16,651
2024	18,660
2025	19,507
2026	20,151
2027	20,755
Thereafter	3,479
Undiscounted minimum rental commitments	99,203
Interest	(13,544)
Total operating lease liabilities	$ 85,659

Leases - Schedule of Future Minimum Lease Payments to be Received (Details) $ in Thousands	Dec. 31, 2022 USD ($)
Leases [Abstract]
2023	$ 4,329
2024	4,459
2025	1,101
Total	$ 9,889

Accrued Expenses - Summary of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Payables and Accruals [Abstract]
Accrued compensation	$ 18,105	$ 19,818
Accrued research and development costs	8,425	5,980
Accrued professional fees	2,435	2,335
Accrued other	1,385	3,834
Total accrued expenses	$ 30,350	$ 31,967

Product Revenue - Schedule of Product Revenues (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Disaggregation of Revenue [Line Items]
Total revenue	$ 14,240	$ 0	$ 0
Product revenue, net
Disaggregation of Revenue [Line Items]
Total revenue	$ 11,740	$ 0	$ 0

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 30
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8906-108599

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 40
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9031-108599

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 41
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9038-108599

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 4
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130543-203045

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 22
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8736-108599

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 22
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8736-108599

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 42
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9054-108599

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 5
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130545-203045

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 924
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
-URI https://asc.fasb.org/extlink&oid=6472922&loc=d3e499488-122856

+ Details

Product Revenue - Schedule of Product Revenue Allowance and Reserves (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Contractual Adjustments
Contractual adjustments, beginning balance	$ 0
Contractual adjustments, current provisions relating to sales in the current year	497
Contractual adjustments, adjustments relating to prior years	0
Contractual adjustments, payments/returns relating to sales in the current year	(432)
Contractual adjustments, payments/returns relating to sales in the prior year	0
Contractual adjustments, ending balance	65
Government Rebates [Roll Forward]
Government rebates, beginning balance	0
Government rebates, current provisions relating to sales in the current year	912
Government rebates, adjustments relating to prior years	0
Government rebates, payments/returns relating to sales in the current year	(339)
Government rebates, payments/returns relating to sales in the prior years	0
Government rebates, ending balance	573
Returns
Returns, beginning balance	0
Returns, current provisions relating to sales in the current year	133
Returns, adjustments relating to prior years	0
Returns, payments/returns relating to sales in the current year
Returns, payments/returns relating to sales in the prior years	0
Returns, ending balance	133
Total
Total revenue-related reserves	771	$ 0
Total allowances and reserves, current provisions relating to sales in the current year	1,542
Total allowances and reserves, adjustments relating to prior years	0
Total allowances and reserves, payments/returns relating to sales in the current year	(771)
Total allowances and reserves, payments/returns relating to sales in the prior years	$ 0

Product Revenue - Schedule of Revenue-Related Reserves (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Revenue from Contract with Customer [Abstract]
Reduction of accounts receivable	$ 60	$ 0
Component of accrued expenses	711	0
Total revenue-related reserves	$ 771	$ 0

Product Revenue - Schedule of Changes in Contract Assets (Details)	12 Months Ended
	Dec. 31, 2022 USD ($)
Contract Assets and Liabilities [Roll Forward]
Contract assets, beginning balance	$ 0
Additions	13,283,000
Deductions	(11,077,000)
Contract assets, ending balance	$ 2,206,000

Product Revenue - Additional Information (Details) - USD ($)	Dec. 31, 2022	Dec. 31, 2021
Revenue from Contract with Customer [Abstract]
Contract assets	$ 2,206,000	$ 0

Share-Based Payments - Additional Information (Details) - USD ($)			12 Months Ended
	Jan. 03, 2023	Jan. 01, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Tax benefits related to stock based compensation			$ 0	$ 0	$ 0
Expected contractual term			10 years
Expected dividend yield			0.00%	0.00%	0.00%
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted-average period to recognize compensation expense			1 year 9 months 3 days
Unrecognized compensation expense excluding options			$ 25,600,000
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			15,400,000
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]
Share based awards reserved for issuance (in shares)			12,821,789
2013 Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)			5,458,366
Annual increase in common stock (in shares)			2,000,000
Percentage of outstanding shares of common stock			4.00%
Weighted-average grant date fair value of options (in usd per share)			$ 15.64	$ 31.2	$ 32.1
Intrinsic value of options exercised			$ 300,000	$ 8,500,000	$ 10,400,000
Unrecognized compensation expense related to options			$ 38,100,000
Weighted-average period to recognize compensation expense			2 years 8 months 15 days
2013 Stock Incentive Plan \| Subsequent Event
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Additional shares authorized for issuance (in shares)		2,000,000
2013 Employee Stock Purchase Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)			1,289,780
Shares issued under 2013 ESPP (in shares)			104,867	94,888
Opportunity to purchase common stock (in shares)			1,854,545
2013 Employee Stock Purchase Plan \| Subsequent Event
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Additional shares authorized for issuance (in shares)	509,091

Share-Based Payments - Summary of Company's Stock Option Activity of all Stock Incentive Plans (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding [Roll Forward]
Number of stock options, outstanding, beginning balance (in shares)	4,798,826
Number of stock options, granted (in shares)	1,850,093
Number of stock options, exercised (in shares)	(15,539)
Number of stock options, forfeited/expired (in shares)	(860,816)
Number of stock options, outstanding, ending balance (in shares)	5,772,564	4,798,826
Number of stock options, exercisable (in shares)	3,497,660
Number of stock options, vested and expected to vest (in shares)	5,772,564
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract]
Weighted-average exercise price, outstanding, beginning balance (in usd per share)	$ 58.51
Weighted-average exercise price, granted (in usd per share)	28.89
Weighted-average exercise price, exercised (in usd per share)	11.50
Weighted-average exercise price, forfeited/expired (in usd per share)	60.91
Weighted-average exercise price, outstanding, ending balance (in usd per share)	48.81	$ 58.51
Weighted-average exercise price, exercisable (in usd per share)	58.24
Weighted-average exercise price, vested and expected to vest (in usd per share)	$ 48.81
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Weighted-average remaining contractual term, outstanding (in years)	6 years 6 months	6 years 2 months 26 days
Weighted-average remaining contractual term, exercisable (in years)	4 years 10 months 2 days
Weighted-average remaining contractual term, vested and expected to vest (in years)	6 years 6 months
Aggregate intrinsic value, outstanding	$ 5,362	$ 4,697
Aggregate intrinsic value, exercisable	3,141
Aggregate intrinsic value, vested and expected to vest	$ 5,362

Share-Based Payments - Summary of Unvested RSUs, Performance-Based Stock and Market-Based Stock Unit Activity (Details)	12 Months Ended
	Dec. 31, 2022 $ / shares shares
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested shares beginning of period (in shares) \| shares	1,002,924
Granted (in shares) \| shares	869,766
Vested (in shares) \| shares	(531,304)
Forfeited (in shares) \| shares	(223,465)
Unvested shares end of period (in shares) \| shares	1,117,921
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Weighted-average grant date fair value, unvested shares beginning of period (in usd per share) \| $ / shares	$ 51.51
Weighted-average grant date fair value, granted (in usd per share) \| $ / shares	31.16
Weighted-average grant date fair value, vested (in usd per share) \| $ / shares	50.16
Weighted-average grant date fair value, forfeited (in usd per share) \| $ / shares	41.84
Weighted-average grant date fair value, unvested shares end of period (in usd per share) \| $ / shares	$ 38.30
Performance-based stock units
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested shares beginning of period (in shares) \| shares	234,059
Granted (in shares) \| shares	337,243
Vested (in shares) \| shares	(53,777)
Forfeited (in shares) \| shares	(47,190)
Expired (in shares) \| shares	(40,092)
Unvested shares end of period (in shares) \| shares	430,243
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Weighted-average grant date fair value, unvested shares beginning of period (in usd per share) \| $ / shares	$ 54.28
Weighted-average grant date fair value, granted (in usd per share) \| $ / shares	30.33
Weighted-average grant date fair value, vested (in usd per share) \| $ / shares	54.28
Weighted-average grant date fair value, forfeited (in usd per share) \| $ / shares	49.06
Weighted-average grant date fair value, expired (in usd per share) \| $ / shares	56.52
Weighted-average grant date fair value, unvested shares end of period (in usd per share) \| $ / shares	$ 35.87
Market-based stock units
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested shares beginning of period (in shares) \| shares	42,695
Granted (in shares) \| shares	0
Unvested shares end of period (in shares) \| shares	42,695
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Weighted-average grant date fair value, unvested shares beginning of period (in usd per share) \| $ / shares	$ 41.50
Weighted-average grant date fair value, granted (in usd per share) \| $ / shares	0
Weighted-average grant date fair value, unvested shares end of period (in usd per share) \| $ / shares	$ 41.50

Share-Based Payments - Schedule of Stock-Based Compensation by Award Type Included Within the Consolidated Statements of Operations (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 49,296	$ 53,508	$ 61,602
Stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	23,731	30,985	37,705
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	21,670	21,510	19,893
Performance-based stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	2,919	0	1,760
Employee Stock Purchase Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	976	1,013	1,463
Other stock awards
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 0	$ 0	$ 781

Share-Based Payments - Stock-Based Compensation Expense for Equity-Based Awards (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Allocated share based compensation expense	$ 49,296	$ 53,508	$ 61,602
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Allocated share based compensation expense	20,988	24,527	27,119
Selling, general and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Allocated share based compensation expense	$ 28,308	$ 28,981	$ 34,483

Share-Based Payments - Schedule of Grant Date Fair Value Option Award Weighted Average Assumptions Used (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-Based Payment Arrangement [Abstract]
Risk-free interest rate	2.55%	0.72%	1.24%
Expected dividend yield	0.00%	0.00%	0.00%
Expected term (in years)	6 years 10 days	6 years 18 days	6 years 18 days
Expected volatility	55.30%	61.72%	73.80%

Net Income (Loss) per Share - Common Stock Excluded from Calculation of Diluted Net Loss Per Share (Detail) - shares	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	6,932,511	5,841,614	7,473,863
Stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	5,772,564	4,798,826	6,143,046
Restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	1,117,921	1,002,924	1,284,378
Employee Stock Purchase Plan shares
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Antidilutive securities excluded from computation of earnings per share (in shares)	42,026	39,864	46,439

Income Taxes - Schedule of Domestic and Foreign Components of Loss Before Income Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Income Tax Disclosure [Abstract]
Domestic	$ (231,767)	$ (356,665)	$ (330,669)
Foreign	(34)	155	1,364
Net loss from continuing operations	$ (231,801)	$ (356,510)	$ (329,305)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 30
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8906-108599

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (f)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 4: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 6: http://fasb.org/us-gaap/role/ref/legacyRef
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-Name Accounting Standards Codification
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/extlink&oid=120399700&loc=SL114874048-224260

+ Details

Income Taxes - Reconciliation of Expected Income Tax Benefit (Expense) Computed Using Federal Statutory Income Tax Rate (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Income Tax Disclosure [Abstract]
Income tax benefit computed at federal statutory tax rate	21.00%	21.00%	21.00%
State taxes, net of federal benefit	2.90%	2.60%	2.50%
Change in valuation allowance	(25.70%)	(24.50%)	(28.20%)
General business credits and other credits	5.20%	5.30%	7.00%
Permanent differences and other adjustments	(2.30%)	(3.90%)	(1.60%)
Stock based compensation	(1.10%)	(0.50%)	(0.70%)
Total	0.00%	0.00%	0.00%

Income Taxes - Components of Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Deferred tax assets:
Net operating loss carryforwards	$ 32,907	$ 39,186
Tax credit carryforwards	163,780	152,128
Purchased intangible assets	11,583	12,150
Stock-based compensation	26,236	27,217
Operating lease liability	21,042	22,963
Non-deductible accruals and reserves, including inventory	3,992	4,033
Section 174 R&D expense	56,565	0
Total deferred tax assets	316,105	257,677
Depreciation and amortization	(3,767)	(3,168)
Operating lease right of use asset	(16,345)	(18,031)
Less: valuation allowance	(295,993)	(236,478)	$ (594,752)
Net deferred taxes	$ 0	$ 0

Income Taxes - Additional Information (Details)	12 Months Ended
Income Taxes - Additional Information (Details)	Dec. 31, 2022 USD ($) audit	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Income Taxes Disclosure [Line Items]
Capitalized research and development costs	$ 261,400,000
Percentage of stock owned resulting in an ownership change	50.00%
Aggregate period	3 years
Valuation allowance	$ 295,993,000	$ 236,478,000	$ 594,752,000
Increase (decrease) for the current period	59,515,000	(358,274,000)
Accrued interest and penalties related to uncertain tax positions	0	0
Unrecognized tax benefits	$ 26,190,000	24,220,000	$ 21,131,000
Number of federal, state or foreign audits in process \| audit	0
U.S.
Income Taxes Disclosure [Line Items]
Capitalized research and development costs, amortization period	5 years
Non-US
Income Taxes Disclosure [Line Items]
Capitalized research and development costs, amortization period	15 years
Prepaid Expenses and Other Current Assets
Income Taxes Disclosure [Line Items]
Income tax receivable	$ 300,000	$ 2,900,000
Federal Orphan Drug Tax Credit
Income Taxes Disclosure [Line Items]
Research and development tax credit	126,500,000
State and Local Jurisdiction
Income Taxes Disclosure [Line Items]
Net operating loss carryforwards	1,200,000,000
State and Local Jurisdiction \| Research Tax Credit Carryforward
Income Taxes Disclosure [Line Items]
Research and development tax credit	25,700,000
Foreign Income Tax
Income Taxes Disclosure [Line Items]
Net operating loss carryforwards	65,200,000
Federal \| Research Tax Credit Carryforward
Income Taxes Disclosure [Line Items]
Research and development tax credit	$ 16,900,000

Income Taxes - Changes in Valuation Allowance (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Deferred Tax Assets, Valuation Allowance [Roll Forward]
Valuation allowance at the beginning of the year	$ 236,478	$ 594,752
Increase (decrease) for the current period	59,515	(358,274)
Valuation allowance at the end of the year	$ 295,993	$ 236,478

Income Taxes - Unrecognized Tax Benefits Rollforward (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward]
Unrecognized tax benefits at the beginning of the year	$ 24,220	$ 21,131
Gross increases - current period tax positions	1,970	3,089
Unrecognized tax benefits at the end of the year	$ 26,190	$ 24,220

Property and Equipment, net - Summary of Property and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 73,057	$ 73,897
Less: accumulated depreciation	(50,070)	(44,974)
Property and equipment, net	22,987	28,923
Laboratory equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	23,182	22,165
Computer equipment and software
Property, Plant and Equipment [Line Items]
Total property and equipment	6,179	6,913
Leasehold improvements
Property, Plant and Equipment [Line Items]
Total property and equipment	37,277	32,726
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Total property and equipment	3,514	3,035
Office equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	2,248	1,690
Construction in progress
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 657	$ 7,368

Property and Equipment, net - Additional Information (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 8.4	$ 8.8	$ 9.4

Common Stock - Additional Information (Details)	Dec. 31, 2022 vote shares	Dec. 31, 2021 vote shares
Equity [Abstract]
Common stock, shares authorized (in shares) \| shares	125,000,000	125,000,000
Number of votes per share \| vote	1	1

Share Repurchase Program (Details) - USD ($) $ / shares in Units, $ in Thousands, shares in Millions		12 Months Ended		21 Months Ended
	Apr. 05, 2021	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2022	Apr. 02, 2021	Mar. 25, 2021
Equity, Class of Treasury Stock [Line Items]
Stock repurchase program, authorized amount						$ 1,200,000
Repurchase of common stock			$ 802,486
Repurchase Program
Equity, Class of Treasury Stock [Line Items]
Stock repurchase program, authorized amount						$ 1,200,000
Shares repurchased (in shares)				16.2
Repurchase of common stock				$ 802,500
Shares repurchased (in usd per share)				$ 49.49
BMS Repurchase
Equity, Class of Treasury Stock [Line Items]
Shares repurchased (in shares)	7.1
Repurchase of common stock	$ 344,500
Shares repurchased (in usd per share)	$ 48.38
Rule 10b5-1 Repurchase
Equity, Class of Treasury Stock [Line Items]
Stock repurchase program, authorized amount					$ 600,000
Shares repurchased (in shares)		9.1
Repurchase of common stock		$ 458,000
Shares repurchased (in usd per share)		$ 50.35

Discontinued Operations - Assets and Liabilities of Discontinued Operations (Details) - Agios Oncology Business - Discontinued Operations, Disposed of by Sale - USD ($)		12 Months Ended
	Mar. 31, 2021	Dec. 31, 2021	Dec. 31, 2022
Current assets:
Accounts receivable, net	$ 25,386,000
Collaboration receivable – related party	2,253,000
Collaboration receivable – other	2,438,000
Inventory	16,190,000
Prepaid expenses and other current assets	7,125,000
Total current assets of discontinued operations	53,392,000
Other non-current assets	2,234,000
Total assets of discontinued operations	55,626,000	$ 0	$ 0
Current liabilities:
Accounts payable	4,245,000
Accrued expenses	30,288,000
Total current liabilities of discontinued operations	34,533,000
Liability related to the sale of future revenue, net of debt issuance costs	264,281,000
Total liabilities of discontinued operations	298,814,000	0	$ 0
Net liabilities distributed to Servier	$ (243,188,000)	$ (239,770,000)

Discontinued Operations - Summary of Gain on Sale of Discontinued Operations (Details) - USD ($) $ in Thousands		12 Months Ended
	Mar. 31, 2021	Dec. 31, 2021	Dec. 31, 2020
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Discontinued Operation, Gain (Loss) on Disposal, Statement of Income or Comprehensive Income [Extensible Enumeration]		Less: Net income from discontinued operations
Agios Oncology Business \| Discontinued Operations, Disposed of by Sale
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Cash proceeds	$ 1,800,000	$ 1,802,936
Less: transaction and insurance costs		(53,573)
Plus: net liabilities distributed, including working capital adjustment	$ 243,188	239,770
Gain on sale, pre-tax		1,989,133	$ 0
Income tax expense		(12,799)	$ 0
Gain on sale, net of tax		$ 1,976,334

Discontinued Operations - Additional Information (Details) - USD ($)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020	Mar. 31, 2021
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Gain on sale of contingent payments	$ 127,853,000	$ 0	$ 0
Agios Oncology Business \| Discontinued Operations, Disposed of by Sale
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Total assets of discontinued operations	0	0		$ 55,626,000
Total liabilities of discontinued operations	$ 0	$ 0		$ 298,814,000

Discontinued Operations - Financial Results of Discontinued Operations (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Cost and expenses:
Net income from discontinued operations	$ 0	$ 1,961,225	$ 1,935
Agios Oncology Business \| Discontinued Operations, Disposed of by Sale
Revenues:
Total revenue		41,409	203,196
Cost and expenses:
Cost of sales		706	2,805
Research and development		41,564	146,659
Selling, general and administrative		8,551	33,965
Total cost and expenses		50,821	183,429
(Loss) income from discontinued operations		(9,412)	19,767
Non-cash interest expense for the sale of future revenue		(5,697)	(17,832)
Gain on the sale of the oncology business		1,989,133	0
Income from discontinued operations, pre-tax		1,974,024	1,935
Income tax expense		(12,799)	0
Net income from discontinued operations		1,961,225	1,935
Agios Oncology Business \| Product revenue, net \| Discontinued Operations, Disposed of by Sale
Revenues:
Total revenue		36,909	121,089
Agios Oncology Business \| Collaboration revenue – related party \| Discontinued Operations, Disposed of by Sale
Revenues:
Total revenue		1,350	68,274
Agios Oncology Business \| Collaboration revenue – other \| Discontinued Operations, Disposed of by Sale
Revenues:
Total revenue		491	3,571
Agios Oncology Business \| Milestone revenue \| Discontinued Operations, Disposed of by Sale
Revenues:
Total revenue		$ 2,659	$ 10,262

Commitments and Contingent Liabilities - Additional Information (Details)	Dec. 31, 2022 lawsuit
Commitments and Contingencies Disclosure [Abstract]
Number of ongoing legal proceedings	0

Defined Contribution Benefit Plan - Additional Information (Details)	12 Months Ended
	Dec. 31, 2022
Retirement Benefits [Abstract]
Employer's matching contribution percentage	100.00%
Employer's contribution percentage of eligible compensation	4.00%