0001439222-24-000069 | 10-Q | XBRL Viewer | Agios Pharmaceuticals INC

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Condensed Consolidated Balance Sheets (parenthetical) - $ / shares	Mar. 31, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Preferred stock, par value (in usd per share)	$ 0.001	$ 0.001
Preferred stock, authorized (in shares)	25,000,000	25,000,000
Preferred stock, issued (in shares)	0	0
Preferred stock, outstanding (in shares)	0	0
Common stock, par value (in usd per share)	$ 0.001	$ 0.001
Common stock, authorized (in shares)	125,000,000	125,000,000
Common stock, issued (in shares)	72,967,922	72,161,489
Common stock, outstanding (in shares)	56,751,511	55,945,078
Treasury stock (in shares)	16,216,411	16,216,411

Accrued Expenses

3 Months Ended

Mar. 31, 2024

Payables and Accruals [Abstract]

Accrued Expenses

Accrued expenses consisted of the following:


(In thousands)	March 31, 2024		December 31, 2023
Accrued compensation	$	5,403	$	23,232
Accrued research and development costs	15,182		15,463
Accrued professional fees	2,737		3,115
Accrued other	1,807		1,357
Total accrued expenses	$	25,129	$	43,167

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Mar. 31, 2024
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

Loss per Share (Tables)

3 Months Ended

Mar. 31, 2024

Earnings Per Share [Abstract]

Schedule of Common Stock Equivalents were Excluded from Calculation of Diluted Net Loss Per Share

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


	Three Months Ended March 31,
	2024	2023
Stock options	5,908,863	5,969,886
Restricted stock units	1,865,185	1,531,802

Employee stock purchase plan shares	11,016	14,143
Total common stock equivalents	7,785,064	7,515,831

Inventory (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 90	$ 51
Work-in-process	20,787	17,568
Finished goods	2,193	1,457
Total inventory	$ 23,070	$ 19,076

Marketable Securities - Additional Information (Details)	3 Months Ended
	Mar. 31, 2024 USD ($) security	Mar. 31, 2023 USD ($)	Dec. 31, 2023 USD ($) security
Investments, Debt and Equity Securities [Abstract]
Realized gain (loss) on marketable securities	$ 0	$ 0
Number of debt securities in unrealized loss position for less than one year \| security	160		151
Allowance for credit losses	$ 0		$ 0
Aggregate fair value of debt securities in unrealized loss position	$ 490,100,000		$ 513,500,000
Number of unrealized loss position \| security	0		0

Product Revenue - Schedule of Product Revenue, Net (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Disaggregation of Revenue [Line Items]
Product revenue, net	$ 8,189	$ 5,609
Product revenue, net
Disaggregation of Revenue [Line Items]
Product revenue, net	$ 8,189	$ 5,609

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

- 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.

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+ Details

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Pay vs Performance Disclosure
Net loss	$ (81,549)	$ (81,018)

- 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

Condensed Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Revenues:
Total revenue	$ 8,189	$ 5,609
Operating expenses:
Cost of sales	627	554
Research and development	68,620	67,301
Selling, general and administrative	31,014	28,367
Total operating expenses	100,261	96,222
Loss from operations	(92,072)	(90,613)
Interest income, net	8,889	8,091
Other income, net	1,634	1,504
Net loss	$ (81,549)	$ (81,018)
Net loss per share - basic (in usd per share)	$ (1.45)	$ (1.47)
Net loss per share - diluted (in usd per share)	$ (1.45)	$ (1.47)
Weighted-average number of common shares used in computing net loss per share - basic (in shares)	56,383,475	55,265,390
Weighted-average number of common shares used in computing net loss per share – diluted (in shares)	56,383,475	55,265,390
Product revenue, net
Revenues:
Total revenue	$ 8,189	$ 5,609

Product Revenue - Schedule of Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Dec. 31, 2023
Contractual Adjustments
Contractual adjustments, beginning balance	$ 156
Contractual adjustments, current provisions relating to sales in the current year	511
Contractual adjustments, adjustments relating to prior year	(40)
Contractual adjustments, payments/returns relating to sales in the current year	(427)
Contractual adjustments, payments/returns relating to sales in the prior year	(79)
Contractual adjustments, ending balance	121
Government Rebates
Government rebates, beginning balance	1,084
Government rebates, current provisions relating to sales in the current year	616
Government rebates, adjustments relating to prior years	0
Government rebates, payments/returns relating to sales in the current year	0
Government rebates, payments/returns relating to sales in the prior years	(306)
Government rebates, ending balance	1,394
Returns/ Replacement
Returns/ replacement, beginning balance	232
Returns/ replacement, current provisions relating to sales in the current year	93
Returns/ replacement, adjustments relating to prior years	40
Returns/ replacement, payments/returns relating to sales in the current year	0
Returns/ replacement, payments/returns relating to sales in the prior years	0
Returns/ replacement, ending balance	365
Total
Total revenue-related reserves	1,880	$ 1,472
Total, current provisions relating to sales in the current year	1,220
Total, adjustments relating to prior years	0
Total, payments/returns relating to sales in the current year	(427)
Total, payments/returns relating to sales in the prior years	$ (385)

Product Revenue

3 Months Ended

Mar. 31, 2024

Revenue from Contract with Customer [Abstract]

Product Revenue

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

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

Product revenue, net, were as follows:


	Three Months Ended March 31,
(In thousands)	2024		2023
Product revenue, net	$	8,189	$	5,609

Reserves for Variable Consideration

Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products.

Contractual Adjustments

We generally provide Customers with discounts, including prompt pay discounts, and allowances that are explicitly stated in the contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from certain Customers.

Chargebacks and discounts represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to Customers who directly purchase the product from us. Customers charge us for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are estimated using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated channel mix and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue.

Government Rebates

Government rebates include Medicare, TriCare, and Medicaid rebates, which we estimate using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program.

Returns / Replacement

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

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the three months ended March 31, 2024:


(In thousands)	Contractual Adjustments		Government Rebates		Returns/ Replacement		Total
Balance at December 31, 2023	$	156	$	1,084	$	232	$	1,472
Current provisions relating to sales in the current year	511		616		93		1,220
Adjustments relating to prior years	(40)		—		40		—
Payments/returns relating to sales in the current year	(427)		—		—		(427)
Payments/returns relating to sales in the prior years	(79)		(306)		—		(385)
Balance at March 31, 2024	$	121	$	1,394	$	365	$	1,880

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


(In thousands)	March 31, 2024		December 31, 2023
Reduction of accounts receivable	$	115	$	151
Component of accrued expenses	1,765		1,321
Total revenue-related reserves	$	1,880	$	1,472

The following table presents changes in our contract assets during the three months ended March 31, 2024:


(In thousands)	December 31, 2023		Additions		Deductions		March 31, 2024
Contract assets(1)
Accounts receivable, net	$	2,810	$	9,409	$	(8,766)	$	3,453

(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.

- 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
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-9

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 10
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-10

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 15
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-15

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-13

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org//606/tableOfContent

+ Details

Leases - Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases (Details) $ in Thousands	Mar. 31, 2024 USD ($)
Leases [Abstract]
Remaining 2024	$ 12,488
2025	19,507
2026	20,151
2027	20,755
2028	3,479
Undiscounted minimum rental commitments	76,380
Interest	(8,033)
Operating lease liabilities	$ 68,347

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Operating activities
Net loss	$ (81,549)	$ (81,018)
Adjustments to reconcile net loss from operations to net cash used in operating activities:
Depreciation and amortization	1,348	1,790
Stock-based compensation expense	9,234	10,139
Net accretion of discount on marketable securities	(2,527)	(2,776)
Gain on disposal of property and equipment	(39)	(150)
Non-cash operating lease expense	2,803	2,608
Realized gain on investments	(152)	0
Changes in operating assets and liabilities:
Accounts receivable, net	(643)	428
Inventory	(3,994)	(2,882)
Prepaid expenses and other current and non-current assets	(4,091)	(348)
Accounts payable	1,430	(9,796)
Accrued expenses and other current liabilities	(18,038)	(7,891)
Operating lease liabilities	(3,649)	(3,321)
Other non-current liabilities	0	(1,966)
Net cash used in operating activities	(99,867)	(95,183)
Investing activities
Purchases of marketable securities	(244,059)	(128,351)
Proceeds from maturities and sales of marketable securities	368,722	186,196
Purchases of property and equipment	(141)	(399)
Proceeds from sale of equipment	40	150
Net cash provided by investing activities	124,562	57,596
Financing activities
Net proceeds from stock option exercises and employee stock purchase plan	5,864	2,467
Net cash provided by financing activities	5,864	2,467
Net change in cash and cash equivalents	30,559	(35,120)
Cash and cash equivalents at beginning of the period	88,205	139,259
Cash and cash equivalents at end of the period	118,764	104,139
Supplemental disclosure of non-cash investing and financing transactions
Net cash taxes (returned) paid	$ (525)	$ 0

Share-Based Payments - Schedule of PSU, Market-based Stock Unit or MSU Activity (Details)	3 Months Ended
	Mar. 31, 2024 $ / shares shares
Performance-based stock units
Number of Stock Units
Unvested shares, beginning balance (in shares) \| shares	362,133
Granted (in shares) \| shares	183,000
Vested (in shares) \| shares	(145,023)
Unvested shares, ending balance (in shares) \| shares	400,110
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) \| $ / shares	$ 30.66
Granted (in usd per share) \| $ / shares	32.27
Vested (in usd per share) \| $ / shares	36.04
Unvested shares, ending balance (in usd per share) \| $ / shares	$ 29.45
Market-Based Stock Units
Number of Stock Units
Unvested shares, beginning balance (in shares) \| shares	42,695
Forfeited/Expired (in shares) \| shares	(42,695)
Unvested shares, ending balance (in shares) \| shares	0
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) \| $ / shares	$ 41.50
Forfeited/Expired (in usd per share) \| $ / shares	41.50
Unvested shares, ending balance (in usd per share) \| $ / shares	$ 0

Marketable Securities (Tables)

3 Months Ended

Mar. 31, 2024

Investments, Debt and Equity Securities [Abstract]

Schedule of Marketable Securities

Marketable securities at March 31, 2024 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:
Certificates of deposit	$	11,384	$	—	$	(3)	$	11,381
U.S. Treasuries	79,485		6		(17)		79,474
Government securities	156,434		20		(454)		156,000
Corporate debt securities	232,852		12		(492)		232,372
Total Current	480,155		38		(966)		479,227

Non-current:

U.S. Treasuries	32,989		6		(14)		32,981
Government securities	18,684		—		(60)		18,624
Corporate debt securities	64,787		33		(124)		64,696
Total Non-current	116,460		39		(198)		116,301
Total marketable securities	$	596,615	$	77	$	(1,164)	$	595,528

Marketable securities at December 31, 2023 consisted of the following:


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

U.S. Treasuries	$	30,876	$	—	$	(56)	$	30,820
Government securities	247,460		194		(695)		246,959
Corporate debt securities	411,045		874		(975)		410,944
Total Current	689,381		1,068		(1,726)		688,723

Non-current:

U.S. Treasuries	4,802		30		—		4,832
Government securities	9,986		75		—		10,061
Corporate debt securities	14,430		112		—		14,542
Total Non-current	29,218		217		—		29,435
Total marketable securities	$	718,599	$	1,285	$	(1,726)	$	718,158

Fair Value Measurements - Summary of Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	$ 595,528	$ 718,158
Fair Value, Measurements, Recurring
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	77,421
Total marketable securities	595,528
Total cash equivalents and marketable securities	672,949
Fair Value, Measurements, Recurring \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	77,421
Fair Value, Measurements, Recurring \| Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	11,381
Fair Value, Measurements, Recurring \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	112,455
Fair Value, Measurements, Recurring \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	174,624
Fair Value, Measurements, Recurring \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	297,068
Fair Value, Measurements, Recurring \| Level 1
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	21,530
Total marketable securities	0
Total cash equivalents and marketable securities	21,530
Fair Value, Measurements, Recurring \| Level 1 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	21,530
Fair Value, Measurements, Recurring \| Level 1 \| Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 1 \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 1 \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 1 \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 2
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	55,891
Total marketable securities	595,528
Total cash equivalents and marketable securities	651,419
Fair Value, Measurements, Recurring \| Level 2 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	55,891
Fair Value, Measurements, Recurring \| Level 2 \| Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	11,381
Fair Value, Measurements, Recurring \| Level 2 \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	112,455
Fair Value, Measurements, Recurring \| Level 2 \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	174,624
Fair Value, Measurements, Recurring \| Level 2 \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	297,068
Fair Value, Measurements, Recurring \| Level 3
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	0
Total marketable securities	0
Total cash equivalents and marketable securities	0
Fair Value, Measurements, Recurring \| Level 3 \| Cash equivalents
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total cash equivalents	0
Fair Value, Measurements, Recurring \| Level 3 \| Certificates of deposit
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 3 \| U.S. Treasuries
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 3 \| Government securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	0
Fair Value, Measurements, Recurring \| Level 3 \| Corporate debt securities
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items]
Total marketable securities	$ 0

Summary of Significant Accounting Policies (Policies)	3 Months Ended
Summary of Significant Accounting Policies (Policies)	Mar. 31, 2024
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The condensed consolidated balance sheet as of March 31, 2024, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three months ended March 31, 2024 and 2023, and the condensed consolidated statements of cash flows for the three months ended March 31, 2024 and 2023 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of March 31, 2024, our results of operations and stockholders' equity for the three months ended March 31, 2024 and 2023, and cash flows for the three months ended March 31, 2024 and 2023. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three-month periods are also unaudited. The results of operations for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2023 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2023 that was filed with the Securities and Exchange Commission, or SEC, on February 15, 2024. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP.
Use of Estimates	Use of Estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the recent COVID-19 pandemic, or other pandemics or public health emergencies, may in the future directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain.
Recent Accounting Pronouncements	Recent Accounting Pronouncements Accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption.
Fair Value Measurements	We record cash equivalents and marketable securities at fair value. Accounting Standards Codification, or ASC, 820, Fair Value Measurements and Disclosures, establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date.
Marketable Securities	Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities, and are recorded at fair value. Unrealized gains and losses are included as a component of accumulated other comprehensive loss in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive loss in the condensed consolidated statements of comprehensive loss, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses, to determine if the impairment is credit-related or noncredit-related. Credit-related impairment is recognized as an allowance on the condensed consolidated balance sheets with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive income, net of taxes. Realized gains and losses are included in investment income on a specific-identification basis.
Loss per Share	Basic net loss per share is calculated by dividing net loss by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of March 31, 2024 are not considered to be common stock equivalents.

Summary of Significant Accounting Policies	3 Months Ended
Summary of Significant Accounting Policies	Mar. 31, 2024
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	Summary of Significant Accounting Policies There have been no material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2023. Recent Accounting Pronouncements Accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption.

Share-Based Payments

3 Months Ended

Mar. 31, 2024

Share-Based Payment Arrangement [Abstract]

Share-Based Payments

2023 Stock Incentive Plan and Inducement Grants

In June 2023, our stockholders approved the 2023 Stock Incentive Plan, or the 2023 Plan. The 2023 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based share units, or PSUs, and other stock-based awards to employees, advisors, consultants and non-employee directors.

Following the adoption of the 2023 Plan, we ceased granting equity awards under the 2013 Stock Incentive Plan, or the 2013 Plan. Any outstanding equity awards that were previously granted under the 2013 Plan continue to be governed by their terms. Following adoption of the 2013 Plan, we ceased granting equity awards under the 2007 Stock Incentive Plan, or the 2007 Plan. There are no outstanding equity awards under the 2007 Plan.

In connection with the start of employment of our Chief Executive Officer and Chief Financial Officer in 2022, and our Chief Commercial Officer in 2023, our board of directors granted each of them equity awards in the form of stock options, RSUs and

PSUs, which awards were made outside our equity incentive plans as inducements material to their respective entry into employment with us in accordance with Nasdaq Listing Rule 5635(c)(4).

As of March 31, 2024, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 11,250,432, and we had 3,076,274 shares available for future issuance under the 2023 Plan.

Stock options

The following table presents stock option activity for the three months ended March 31, 2024:


	Number of Stock Options	Weighted-Average Exercise Price
Outstanding at December 31, 2023	5,263,681	$	44.94
Granted	808,415	32.20
Exercised	(148,626)	31.04
Cancelled/Forfeited	(9,357)	41.86
Expired	(5,250)	26.15
Outstanding at March 31, 2024	5,908,863	$	43.56
Exercisable at March 31, 2024	3,475,385	$	52.67
Vested and expected to vest at March 31, 2024	5,908,863	$	43.56

At March 31, 2024, there was approximately $38.0 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.86 years.

Restricted stock units

The following table presents RSU activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	1,346,701	$	29.67
Granted	989,378	30.91
Vested	(460,470)	33.54
Forfeited	(10,424)	29.75
Unvested shares at March 31, 2024	1,865,185	$	29.37

As of March 31, 2024, there was approximately $47.9 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 2.33 years.

Performance-based stock units

The following table presents PSU activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	362,133	$	30.66
Granted	183,000	32.27
Vested	(145,023)	36.04

Unvested shares at March 31, 2024	400,110	$	29.45

Stock-based compensation expense associated with these PSUs is recognized if the underlying performance condition is considered probable of achievement using our management’s best estimates.

As of March 31, 2024, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $11.8 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement.

Market-based stock units

The following table presents market-based stock unit, or MSU, activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	42,695	$	41.50


Expired	(42,695)	41.50
Unvested shares at March 31, 2024	—	$	—

The fair value of MSUs are estimated using a Monte Carlo simulation model. Assumptions and estimates utilized in the model include the risk-free interest rate, dividend yield, expected stock volatility and the estimated period to achievement of the market condition. As of March 31, 2024, there was no remaining unrecognized compensation expense related to MSUs.

2013 Employee Stock Purchase Plan

In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 Employee Stock Purchase Plan, or the 2013 ESPP. We issued and sold 52,514 and 54,867 shares of common stock during the three months ended March 31, 2024 and 2023, respectively, under the 2013 ESPP. The 2013 ESPP provides participating employees with the opportunity to purchase up to an aggregate of 2,363,636 shares of our common stock. As of March 31, 2024, we had 1,633,525 shares of common stock available for future issuance under the 2013 ESPP.

Stock-based compensation expense

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


	Three Months Ended March 31,
(In thousands)	2024		2023
Stock options	$	4,071	$	4,957
Restricted stock units	4,966		4,947

Employee stock purchase plan	197		235

Total stock-based compensation expense	$	9,234	$	10,139

Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations:


	Three Months Ended March 31,
(In thousands)	2024		2023
Research and development expense	$	3,775	$	4,355
Selling, general and administrative expense	5,459		5,784
Total stock-based compensation expense	$	9,234	$	10,139

Inventory (Tables)

3 Months Ended

Mar. 31, 2024

Inventory Disclosure [Abstract]

Schedule of Inventory

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


(In thousands)	March 31, 2024		December 31, 2023
Raw materials	$	90	$	51
Work-in-process	20,787		17,568
Finished goods	2,193		1,457
Total inventory	$	23,070	$	19,076

Inventory

3 Months Ended

Mar. 31, 2024

Inventory Disclosure [Abstract]

Inventory

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


(In thousands)	March 31, 2024		December 31, 2023
Raw materials	$	90	$	51
Work-in-process	20,787		17,568
Finished goods	2,193		1,457
Total inventory	$	23,070	$	19,076

Share-Based Payments - Additional Information (Details) - USD ($)	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted-average period to recognize compensation expense (in years)	2 years 3 months 29 days
Unrecognized compensation expense excluding options	$ 47,900,000
Performance-based stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense excluding options	0
Unrecognized compensation cost not expected to be recognized	11,800,000
Market-Based Stock Units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense excluding options	$ 0
2007 Plan and 2013 Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock reserved for issuance (in shares)	11,250,432
2013 Stock Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)	3,076,274
Unrecognized compensation expense related to options	$ 38,000,000
Weighted-average period to recognize compensation expense (in years)	2 years 10 months 9 days
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares available for future issuance (in shares)	1,633,525
Shares issued under 2013 ESPP (in shares)	52,514	54,867
Opportunity to purchase of common stock (in shares)	2,363,636

Leases - Schedule of Future Minimum Lease Payments to be Received under the Long-term Sublease Agreements (Details) $ in Thousands	Mar. 31, 2024 USD ($)
Leases [Abstract]
Remaining 2024	$ 3,831
2025	1,310
Total	$ 5,141

Share-Based Payments - Schedule of RSU Activity (Details) - Restricted stock units	3 Months Ended
	Mar. 31, 2024 $ / shares shares
Number of Stock Units
Unvested shares, beginning balance (in shares) \| shares	1,346,701
Granted (in shares) \| shares	989,378
Vested (in shares) \| shares	(460,470)
Forfeited/Expired (in shares) \| shares	(10,424)
Unvested shares, ending balance (in shares) \| shares	1,865,185
Weighted-Average Grant Date Fair  Value
Unvested shares, beginning balance (in usd per share) \| $ / shares	$ 29.67
Granted (in usd per share) \| $ / shares	30.91
Vested (in usd per share) \| $ / shares	33.54
Forfeited/Expired (in usd per share) \| $ / shares	29.75
Unvested shares, ending balance (in usd per share) \| $ / shares	$ 29.37

Overview and Basis of Presentation (Details) - USD ($) $ in Millions		12 Months Ended
	Mar. 31, 2021	Dec. 31, 2023	Dec. 31, 2022	Mar. 31, 2024
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Cash, cash equivalents, and marketable securities				$ 714.3
Alnylam Pharmaceuticals, Inc.
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Payments associated with license agreement		$ 17.5
Potential development milestone payments, maximum		$ 130.0
2010 Agreement
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone payment for achievement of specified ex-U.S. commercial milestone event	$ 25.0
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Cash proceeds	1,800.0
Contingent milestone payment	$ 200.0
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business \| TIBSOVO \| UNITED STATES
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Contingent royalty payment	5.00%
Gain on sale of contingent payments			$ 127.9
Discontinued Operations, Disposed of by Sale \| Agios Oncology Business \| Vorasidenib \| UNITED STATES
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Contingent royalty payment	15.00%

Leases (Tables)

3 Months Ended

Mar. 31, 2024

Leases [Abstract]

Schedule of Lease Expense and Other Information Related to Leases

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


	Three Months Ended March 31,
(In thousands)	2024		2023
Operating lease costs	$	3,807	$	3,807
Cash paid for amounts included in the measurement of operating lease liabilities	4,653		4,520

Schedule of Undiscounted Minimum Rental Commitments under Non-cancelable Leases

As of March 31, 2024, undiscounted minimum rental commitments under non-cancelable leases were as follows:



(In thousands)
Remaining 2024	$	12,488
2025	19,507
2026	20,151
2027	20,755
2028	3,479


Undiscounted minimum rental commitments	$	76,380
Interest	(8,033)
Operating lease liabilities	$	68,347

Schedule of Future Minimum Lease Payments to be Received under the Long-term Sublease Agreements

As of March 31, 2024, the future minimum lease payments to be received under the long-term sublease agreements were as follows:


(In thousands)
Remaining 2024	$	3,831
2025	1,310


Total	$	5,141

Fair Value Measurements (Tables)

3 Months Ended

Mar. 31, 2024

Fair Value Disclosures [Abstract]

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

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of March 31, 2024:


(In thousands)	Level 1		Level 2		Level 3		Total
Cash equivalents	$	21,530	$	55,891	$	—	$	77,421
Total cash equivalents	21,530		55,891		—		77,421

Marketable securities:
Certificates of deposit	$	—	$	11,381	$	—	$	11,381
U.S. Treasuries	—		112,455		—		112,455
Government securities	—		174,624		—		174,624
Corporate debt securities	—		297,068		—		297,068
Total marketable securities	—		595,528		—		595,528
Total cash equivalents and marketable securities	$	21,530	$	651,419	$	—	$	672,949

Leases

3 Months Ended

Mar. 31, 2024

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 approximately four years and contain various clauses for renewal at our option. The renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options are not reasonably certain of being exercised. The lease agreements do not contain residual value guarantees.

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


	Three Months Ended March 31,
(In thousands)	2024		2023
Operating lease costs	$	3,807	$	3,807
Cash paid for amounts included in the measurement of operating lease liabilities	4,653		4,520

We have not entered into any material short-term leases or financing leases as of March 31, 2024.

In arriving at the operating lease liabilities as of March 31, 2024 and December 31, 2023, we applied the weighted-average incremental borrowing rate of 5.7% for both periods over a weighted-average remaining lease term of 3.9 and 4.2 years, respectively.

As of March 31, 2024, undiscounted minimum rental commitments under non-cancelable leases were as follows:



(In thousands)
Remaining 2024	$	12,488
2025	19,507
2026	20,151
2027	20,755
2028	3,479


Undiscounted minimum rental commitments	$	76,380
Interest	(8,033)
Operating lease liabilities	$	68,347

We provided our landlord a security deposit of $2.9 million as security for our leases, which is included within other non-current assets on our condensed consolidated balance sheet.

In August 2021, we entered into a long-term sublease agreement for 13,000 square feet of the office space at 38 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through December 2024. In April 2022, we entered into a long-term sublease agreement for 27,000 square feet of the office space at 64 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through April 2025. In May 2023, we entered into a long-term sublease agreement for 7,407 square feet of office space on the first floor of 64 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through April 2025. We recorded operating sublease income of $1.6 million and $1.4 million for the three months ended March 31, 2024 and 2023, respectively, in other income, net in the condensed consolidated statements of operations. We received security

deposits from our sublessees of approximately $1.2 million which is recorded within other non-current assets on our condensed consolidated balance sheet.

As of March 31, 2024, the future minimum lease payments to be received under the long-term sublease agreements were as follows:


(In thousands)
Remaining 2024	$	3,831
2025	1,310


Total	$	5,141

Product Revenue - Schedule of Revenue-related Reserves (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Revenue from Contract with Customer [Abstract]
Reduction of accounts receivable	$ 115	$ 151
Component of accrued expenses	1,765	1,321
Total revenue-related reserves	$ 1,880	$ 1,472

Marketable Securities - Schedule of Marketable Securities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 596,615	$ 718,599
Unrealized Gains	77	1,285
Unrealized Losses	(1,164)	(1,726)
Fair Value	595,528	718,158
Current:
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	480,155	689,381
Unrealized Gains	38	1,068
Unrealized Losses	(966)	(1,726)
Fair Value	479,227	688,723
Current: \| Certificates of deposit
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	11,384
Unrealized Gains	0
Unrealized Losses	(3)
Fair Value	11,381
Current: \| U.S. Treasuries
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	79,485	30,876
Unrealized Gains	6	0
Unrealized Losses	(17)	(56)
Fair Value	79,474	30,820
Current: \| Government securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	156,434	247,460
Unrealized Gains	20	194
Unrealized Losses	(454)	(695)
Fair Value	156,000	246,959
Current: \| Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	232,852	411,045
Unrealized Gains	12	874
Unrealized Losses	(492)	(975)
Fair Value	232,372	410,944
Non-current:
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	116,460	29,218
Unrealized Gains	39	217
Unrealized Losses	(198)	0
Fair Value	116,301	29,435
Non-current: \| U.S. Treasuries
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	32,989	4,802
Unrealized Gains	6	30
Unrealized Losses	(14)	0
Fair Value	32,981	4,832
Non-current: \| Government securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	18,684	9,986
Unrealized Gains	0	75
Unrealized Losses	(60)	0
Fair Value	18,624	10,061
Non-current: \| Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	64,787	14,430
Unrealized Gains	33	112
Unrealized Losses	(124)	0
Fair Value	$ 64,696	$ 14,542

Overview and Basis of Presentation	3 Months Ended
Overview and Basis of Presentation	Mar. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Overview and Basis of Presentation	Overview and Basis of Presentation References to Agios Throughout this Quarterly Report on Form 10-Q, "Agios," "the company," “we,” “us,” and “our,” and similar expressions, except where the context requires otherwise, refer to Agios Pharmaceuticals, Inc. and its consolidated subsidiaries, and “our Board of Directors” refers to the board of directors of Agios Pharmaceuticals, Inc. Overview We are a biopharmaceutical company committed to transforming patients’ lives through leadership in the field of cellular metabolism, with the goal of creating differentiated medicines for rare diseases, with a focus on classical hematology. With a history of focused study on cellular metabolism, we have a deep and mature understanding of this biology, which is involved in the healthy functioning of nearly every system in the body. Building on this expertise, these learnings can be rapidly applied to our clinical trials with the goal of developing medicines that can have a significant impact for patients. We accelerate the impact of our portfolio by cultivating connections with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver potential therapies for rare diseases. We are located in Cambridge, Massachusetts. The lead product candidate in our portfolio, PYRUKYND® (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. In February 2022, the U.S. Food and Drug Administration, or FDA, approved PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency in the United States. In November 2022, we received marketing authorization from the European Commission for PYRUKYND® for the treatment of PK deficiency in adult patients in the European Union, or EU. In December 2022, we received marketing authorization in Great Britain for PYRUKYND® for the treatment of PK deficiency in adult patients under the European Commission Decision Reliance Procedure. In addition, we are currently evaluating PYRUKYND® in clinical trials for the treatment of thalassemia, sickle cell disease, or SCD, and in pediatric patients with PK deficiency. We are also developing (i) AG-946, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndrome, or LR MDS, and hemolytic anemias, and (ii) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU. In addition to the aforementioned development programs, in July 2023 we entered into a license agreement with Alnylam Pharmaceuticals, Inc., or Alnylam, for the development and commercialization of products containing or comprised of an siRNA preclinical development candidate discovered by Alnylam and targeting the transmembrane serine protease 6, or TMPRSS6, gene, and we intend to pursue development of a licensed product for the potential disease-modifying treatment of patients with polycythemia vera, or PV, a rare blood disorder. We are subject to risks common to companies in our industry including, but not limited to, uncertainties relating to conducting preclinical and clinical research and development, the manufacture and supply of products for clinical and commercial use, obtaining and maintaining regulatory approvals and pricing and reimbursement for our products, market acceptance, managing global growth and operating expenses, availability of additional capital, competition, obtaining and enforcing patents, stock price volatility, dependence on collaborative relationships and third-party service providers, dependence on key personnel, potential litigation, potential product liability claims and potential government investigations. Alnylam License Agreement On July 28, 2023, we entered into a license agreement with Alnylam under which we acquired the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting the TMPRSS6 gene, as a potential disease-modifying treatment for patients with PV. Because the acquired assets do not meet the definition of a business in accordance with ASC 805, Business Combinations, we accounted for the agreement as an asset acquisition. In accordance with the license agreement, in the twelve months ended December 31, 2023, we made an up-front payment to Alnylam and recognized in-process research and development of $17.5 million. We will also pay Alnylam for certain expenses associated with the development of the TMPRSS6 gene and these will be recorded in our Consolidated Statements of Operations as incurred. Additionally, we are responsible to pay up to $130.0 million in potential development and regulatory milestones, in addition to sales milestones as well as tiered royalties on annual net sales, if any, of licensed products, which may be subject to specified reductions and offsets. Sale of Oncology Business to Servier and Sale of Contingent Payments On March 31, 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier, which represented a discontinued operation. The transaction included the sale of our oncology business, including TIBSOVO®, our clinical-stage product candidates vorasidenib, AG-270 and AG-636, and our oncology research programs for a payment of approximately $1.8 billion in cash at the closing, subject to certain adjustments, and a payment of $200.0 million in cash, if, prior to January 1, 2027, vorasidenib is granted new drug application, 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, with these royalties referred to as contingent payments and recognized as income when realizable. Servier also acquired our co-commercialization rights for Bristol Myers Squibb’s IDHIFA® and the right to receive a $25.0 million potential milestone payment under our prior collaboration agreement with Celgene Corporation, or Celgene, and following the sale Servier will conduct certain clinical development activities within the IDHIFA® development program. In October 2022, we sold our rights to future contingent payments associated with the royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through the loss of exclusivity to entities affiliated with Sagard Healthcare Partners, or Sagard, and recognized income of $127.9 million within the gain on sale of contingent payments line item in our Consolidated Statements of Operations for the year ended December 31, 2022. We retain our rights to the potential milestone payment and royalties from Servier if a new drug application for vorasidenib is approved by the FDA. In February 2024, Servier announced that it received filing acceptance and priority review from the FDA for its NDA for vorasidenib for the treatment of certain IDH-mutant diffuse glioma and accelerated assessment from the European Medicines Agency, or EMA, for its Marketing Authorization Application, or MAA, for vorasidenib. The FDA assigned the NDA a Prescription Drug User Fee Act action date of August 20, 2024, and Servier anticipates that the European Commission will approve its MAA in the second half of 2024. Basis of Presentation The condensed consolidated balance sheet as of March 31, 2024, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three months ended March 31, 2024 and 2023, and the condensed consolidated statements of cash flows for the three months ended March 31, 2024 and 2023 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of March 31, 2024, our results of operations and stockholders' equity for the three months ended March 31, 2024 and 2023, and cash flows for the three months ended March 31, 2024 and 2023. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three-month periods are also unaudited. The results of operations for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2023 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2023 that was filed with the Securities and Exchange Commission, or SEC, on February 15, 2024. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP. Use of Estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the recent COVID-19 pandemic, or other pandemics or public health emergencies, may in the future directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain. Liquidity As of March 31, 2024, we had cash, cash equivalents and marketable securities of $714.3 million. Although we have incurred recurring losses and expect to continue to incur losses for the foreseeable future, we expect our cash, cash equivalents and marketable securities 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.

Marketable Securities

3 Months Ended

Mar. 31, 2024

Investments, Debt and Equity Securities [Abstract]

Marketable Securities

Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities, and are recorded at fair value. Unrealized gains and losses are included as a component of accumulated other comprehensive

loss in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive loss in the condensed consolidated statements of comprehensive loss, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses, to determine if the impairment is credit-related or noncredit-related. Credit-related impairment is recognized as an allowance on the condensed consolidated balance sheets with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive income, net of taxes. Realized gains and losses are included in investment income on a specific-identification basis. There were no material realized gains or losses on marketable securities for the three months ended March 31, 2024 or 2023.

Marketable securities at March 31, 2024 consisted of the following:


(In thousands)	Amortized Cost		Unrealized Gains		Unrealized Losses		Fair Value
Current:
Certificates of deposit	$	11,384	$	—	$	(3)	$	11,381
U.S. Treasuries	79,485		6		(17)		79,474
Government securities	156,434		20		(454)		156,000
Corporate debt securities	232,852		12		(492)		232,372
Total Current	480,155		38		(966)		479,227

Non-current:

U.S. Treasuries	32,989		6		(14)		32,981
Government securities	18,684		—		(60)		18,624
Corporate debt securities	64,787		33		(124)		64,696
Total Non-current	116,460		39		(198)		116,301
Total marketable securities	$	596,615	$	77	$	(1,164)	$	595,528

Marketable securities at December 31, 2023 consisted of the following:


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

U.S. Treasuries	$	30,876	$	—	$	(56)	$	30,820
Government securities	247,460		194		(695)		246,959
Corporate debt securities	411,045		874		(975)		410,944
Total Current	689,381		1,068		(1,726)		688,723

Non-current:

U.S. Treasuries	4,802		30		—		4,832
Government securities	9,986		75		—		10,061
Corporate debt securities	14,430		112		—		14,542
Total Non-current	29,218		217		—		29,435
Total marketable securities	$	718,599	$	1,285	$	(1,726)	$	718,158

As of March 31, 2024 and December 31, 2023, we held both current and non-current investments. Investments classified as current have maturities of less than one year. Investments classified as non-current are those that: (i) have a maturity of greater than one year, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and, therefore, are classified as available-for-sale.

As of March 31, 2024 and December 31, 2023, we held 160 and 151 debt securities, respectively, that were in an unrealized loss position for less than one year. We did not record an allowance for credit losses as of March 31, 2024 and December 31, 2023 related to these securities. The aggregate fair value of debt securities in an unrealized loss position at March 31, 2024 and December 31, 2023 was $490.1 million and $513.5 million, respectively. There were no individual securities that were in a significant unrealized loss position as of March 31, 2024 and December 31, 2023. We regularly review the securities in an unrealized loss position and evaluate the current expected credit loss by considering factors such as historical experience,

market data, issuer-specific factors, and current economic conditions. We do not consider these marketable securities to be impaired as of March 31, 2024 and December 31, 2023.

Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Loss	Accumulated Deficit	Treasury Stock
Beginning balance (in shares) at Dec. 31, 2022		71,256,118,000
Beginning balance at Dec. 31, 2022	$ 1,100,814	$ 71	$ 2,386,325	$ (12,535)	$ (470,561)	$ (802,486)
Treasury stock, beginning balance (in shares) at Dec. 31, 2022						(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	4,124			4,124
Common stock issued under stock incentive plan and ESPP (in shares)		501,660,000
Common stock issued under stock incentive plan and ESPP	2,467	$ 1	2,466
Stock-based compensation expense	10,139		10,139
Net loss	(81,018)				(81,018)
Ending balance (in shares) at Mar. 31, 2023		71,757,778,000
Ending balance at Mar. 31, 2023	$ 1,036,526	$ 72	2,398,930	(8,411)	(551,579)	$ (802,486)
Treasury stock, ending balance (in shares) at Mar. 31, 2023						(16,216,411,000)
Beginning balance (in shares) at Dec. 31, 2023	55,945,078	72,161,489,000
Beginning balance at Dec. 31, 2023	$ 811,019	$ 72	2,436,523	(441)	(822,649)	$ (802,486)
Treasury stock, beginning balance (in shares) at Dec. 31, 2023	(16,216,411)					(16,216,411,000)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Unrealized gain (loss) on available-for-sale securities	$ (646)			(646)
Common stock issued under stock incentive plan and ESPP (in shares)	148,626	806,433,000
Common stock issued under stock incentive plan and ESPP	$ 5,864	$ 1	5,863
Stock-based compensation expense	9,234		9,234
Net loss	$ (81,549)				(81,549)
Ending balance (in shares) at Mar. 31, 2024	56,751,511	72,967,922,000
Ending balance at Mar. 31, 2024	$ 743,922	$ 73	$ 2,451,620	$ (1,087)	$ (904,198)	$ (802,486)
Treasury stock, ending balance (in shares) at Mar. 31, 2024	(16,216,411)					(16,216,411,000)

- Definition

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

+ References

+ Details

- Definition

Amount of equity (deficit) attributable to parent. Excludes temporary equity and equity attributable to noncontrolling interest.

+ References

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-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(30))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480566/210-10-S99-1

Reference 14: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 310
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 4.E)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480418/310-10-S99-2

+ Details

Product Revenue (Tables)

3 Months Ended

Mar. 31, 2024

Revenue from Contract with Customer [Abstract]

Schedule of Product Revenue, Net

Product revenue, net, were as follows:


	Three Months Ended March 31,
(In thousands)	2024		2023
Product revenue, net	$	8,189	$	5,609

Schedule of Product Revenue Allowance and Reserve

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the three months ended March 31, 2024:


(In thousands)	Contractual Adjustments		Government Rebates		Returns/ Replacement		Total
Balance at December 31, 2023	$	156	$	1,084	$	232	$	1,472
Current provisions relating to sales in the current year	511		616		93		1,220
Adjustments relating to prior years	(40)		—		40		—
Payments/returns relating to sales in the current year	(427)		—		—		(427)
Payments/returns relating to sales in the prior years	(79)		(306)		—		(385)
Balance at March 31, 2024	$	121	$	1,394	$	365	$	1,880

Schedule of Revenue-related Reserves

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


(In thousands)	March 31, 2024		December 31, 2023
Reduction of accounts receivable	$	115	$	151
Component of accrued expenses	1,765		1,321
Total revenue-related reserves	$	1,880	$	1,472

Schedule of Changes in Contract Assets

The following table presents changes in our contract assets during the three months ended March 31, 2024:


(In thousands)	December 31, 2023		Additions		Deductions		March 31, 2024
Contract assets(1)
Accounts receivable, net	$	2,810	$	9,409	$	(8,766)	$	3,453

(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)

3 Months Ended

Mar. 31, 2024

Share-Based Payment Arrangement [Abstract]

Schedule of Company's Stock Option Activity

The following table presents stock option activity for the three months ended March 31, 2024:


	Number of Stock Options	Weighted-Average Exercise Price
Outstanding at December 31, 2023	5,263,681	$	44.94
Granted	808,415	32.20
Exercised	(148,626)	31.04
Cancelled/Forfeited	(9,357)	41.86
Expired	(5,250)	26.15
Outstanding at March 31, 2024	5,908,863	$	43.56
Exercisable at March 31, 2024	3,475,385	$	52.67
Vested and expected to vest at March 31, 2024	5,908,863	$	43.56

Schedule of RSU Activity

The following table presents RSU activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	1,346,701	$	29.67
Granted	989,378	30.91
Vested	(460,470)	33.54
Forfeited	(10,424)	29.75
Unvested shares at March 31, 2024	1,865,185	$	29.37

Schedule of PSU Activity

The following table presents PSU activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	362,133	$	30.66
Granted	183,000	32.27
Vested	(145,023)	36.04

Unvested shares at March 31, 2024	400,110	$	29.45

Schedule of Market-based Stock Unit or MSU Activity

The following table presents market-based stock unit, or MSU, activity for the three months ended March 31, 2024:


	Number of Stock Units	Weighted-Average Grant Date Fair Value
Unvested shares at December 31, 2023	42,695	$	41.50


Expired	(42,695)	41.50
Unvested shares at March 31, 2024	—	$	—

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

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


	Three Months Ended March 31,
(In thousands)	2024		2023
Stock options	$	4,071	$	4,957
Restricted stock units	4,966		4,947

Employee stock purchase plan	197		235

Total stock-based compensation expense	$	9,234	$	10,139

Schedule of Expenses Related to Stock Options and Stock-based Awards

Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations:


	Three Months Ended March 31,
(In thousands)	2024		2023
Research and development expense	$	3,775	$	4,355
Selling, general and administrative expense	5,459		5,784
Total stock-based compensation expense	$	9,234	$	10,139

Leases - Schedule of Lease Expense and Other Information Related to Leases (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Leases [Abstract]
Operating lease costs	$ 3,807	$ 3,807
Cash paid for amounts included in the measurement of operating lease liabilities	$ 4,653	$ 4,520

Accrued Expenses (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Accrued compensation	$ 5,403	$ 23,232
Accrued research and development costs	15,182	15,463
Accrued professional fees	2,737	3,115
Accrued other	1,807	1,357
Total accrued expenses	$ 25,129	$ 43,167

Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Statement of Comprehensive Income [Abstract]
Net loss	$ (81,549)	$ (81,018)
Other comprehensive (loss) income
Unrealized (loss) gain on available-for-sale securities	(646)	4,124
Comprehensive loss	$ (82,195)	$ (76,894)

- Definition

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

+ References

+ Details

Fair Value Measurements - Additional Information (Details)	Mar. 31, 2024 USD ($)
Fair Value, Measurements, Recurring \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Fair value of assets (liabilities)	$ 0

Loss per Share

3 Months Ended

Mar. 31, 2024

Earnings Per Share [Abstract]

Loss per Share

Basic net loss per share is calculated by dividing net loss by the weighted-average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of March 31, 2024 are not considered to be common stock equivalents.

We utilize the control number concept in the computation of diluted earnings per share to determine whether potential common stock equivalents are dilutive. The control number used is net loss from continuing operations. The control number concept requires that the same number of potentially dilutive securities applied in computing diluted earnings per share from continuing operations be applied to all other categories of income or loss, regardless of their anti-dilutive effect on such categories. Since

we had a net loss for all periods presented, no dilutive effect has been recognized in the calculation of loss per share. Basic and diluted net loss per share was the same for all periods presented.


	Three Months Ended March 31,
	2024	2023
Stock options	5,908,863	5,969,886
Restricted stock units	1,865,185	1,531,802

Employee stock purchase plan shares	11,016	14,143
Total common stock equivalents	7,785,064	7,515,831

Product Revenue - Schedule of Changes in Contract Assets (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($)
Contract assets
Contract assets, beginning balance	$ 2,810
Additions	9,409
Deductions	(8,766)
Contract assets, ending balance	$ 3,453

Fair Value Measurements

3 Months Ended

Mar. 31, 2024

Fair Value Disclosures [Abstract]

Fair Value Measurements

We record cash equivalents and marketable securities at fair value. Accounting Standards Codification, or ASC, 820, Fair Value Measurements and Disclosures, establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels:

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

Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability.

Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date.

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of March 31, 2024:


(In thousands)	Level 1		Level 2		Level 3		Total
Cash equivalents	$	21,530	$	55,891	$	—	$	77,421
Total cash equivalents	21,530		55,891		—		77,421

Marketable securities:
Certificates of deposit	$	—	$	11,381	$	—	$	11,381
U.S. Treasuries	—		112,455		—		112,455
Government securities	—		174,624		—		174,624
Corporate debt securities	—		297,068		—		297,068
Total marketable securities	—		595,528		—		595,528
Total cash equivalents and marketable securities	$	21,530	$	651,419	$	—	$	672,949

Cash equivalents and marketable securities have been initially valued at the transaction price and are subsequently valued, at the end of each reporting period, utilizing third-party pricing services or other observable market data. The pricing services utilize industry standard valuation models, including both income and market-based approaches, and observable market inputs to determine value. After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of March 31, 2024.

There have been no changes to the valuation methods during the three months ended March 31, 2024, and we had no financial assets or liabilities that were classified as Level 3 at any point during the three months ended March 31, 2024.

- 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

Accrued Expenses (Tables)

3 Months Ended

Mar. 31, 2024

Payables and Accruals [Abstract]

Schedule of Accrued Expenses

Accrued expenses consisted of the following:


(In thousands)	March 31, 2024		December 31, 2023
Accrued compensation	$	5,403	$	23,232
Accrued research and development costs	15,182		15,463
Accrued professional fees	2,737		3,115
Accrued other	1,807		1,357
Total accrued expenses	$	25,129	$	43,167

Cover Page - shares	3 Months Ended
Cover Page - shares	Mar. 31, 2024	Apr. 26, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2024
Document Transition Report	false
Entity File Number	001-36014
Entity Registrant Name	AGIOS PHARMACEUTICALS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	26-0662915
Entity Address, Address Line One	88 Sidney Street
Entity Address, City or Town	Cambridge
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02139
City Area Code	617
Local Phone Number	649-8600
Title of 12(b) Security	Common Stock, Par Value $0.001 per share
Trading Symbol	AGIO
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		56,772,966
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q1
Amendment Flag	false
Entity Central Index Key	0001439222
Current Fiscal Year End Date	--12-31

Share-Based Payments - Schedule of Expenses Related to Stock Options and Stock-based Awards (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 9,234	$ 10,139
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	3,775	4,355
Selling, general and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 5,459	$ 5,784

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
-Paragraph 1D
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-URI https://asc.fasb.org//1943274/2147480483/718-10-35-1D

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Loss per Share (Details) - shares	3 Months Ended
Loss per Share (Details) - shares	Mar. 31, 2024	Mar. 31, 2023
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	7,785,064	7,515,831
Stock options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	5,908,863	5,969,886
Restricted stock units
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	1,865,185	1,531,802
Employee stock purchase plan shares
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total common stock equivalents (in shares)	11,016	14,143

Share-Based Payments - Schedule of Stock-Based Compensation Expense by Award Type Included within the Condensed Consolidated Statements of Operations (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 9,234	$ 10,139
Stock options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	4,071	4,957
Restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	4,966	4,947
Employee stock purchase plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 197	$ 235

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Share-Based Payments - Schedule of Company's Stock Option Activity (Details)	3 Months Ended
	Mar. 31, 2024 $ / shares shares
Number of Stock Options
Outstanding, beginning balance (in shares) \| shares	5,263,681
Granted (in shares) \| shares	808,415
Exercised (in shares) \| shares	(148,626)
Cancelled/Forfeited (in shares) \| shares	(9,357)
Expired (in shares) \| shares	(5,250)
Outstanding, ending balance (in shares) \| shares	5,908,863
Exercisable (in shares) \| shares	3,475,385
Vested and expected to vest (in shares) \| shares	5,908,863
Weighted-Average Exercise Price
Outstanding, beginning balance (in usd per share) \| $ / shares	$ 44.94
Granted (in usd per share) \| $ / shares	32.20
Exercised (in usd per share) \| $ / shares	31.04
Cancelled/Forfeited (in usd per share) \| $ / shares	41.86
Expired (in usd per share) \| $ / shares	26.15
Outstanding, ending balance (in usd per share) \| $ / shares	43.56
Exercisable (in usd per share) \| $ / shares	52.67
Vested and expected to vest (in usd per share) \| $ / shares	$ 43.56

Leases - Additional Information (Details) $ in Millions	3 Months Ended
Leases - Additional Information (Details) $ in Millions	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Dec. 31, 2023	May 31, 2023 ft²	Apr. 30, 2022 ft²	Aug. 31, 2021 ft²
Lessor, Lease, Description [Line Items]
Remaining lease terms	4 years
Weighted-average incremental borrowing rate	5.70%		5.70%
Weighted-average remaining lease term	3 years 10 months 24 days		4 years 2 months 12 days
Sublease income	$ 1.6	$ 1.4
Thirty-Eight Sydney Street Lease
Lessor, Lease, Description [Line Items]
Area of premises subleased (in square feet) \| ft²						13,000
Sixty-Four Sydney Street Lease
Lessor, Lease, Description [Line Items]
Area of premises subleased (in square feet) \| ft²				7,407	27,000
Other Noncurrent Assets
Lessor, Lease, Description [Line Items]
Standby letter of credit	2.9
Security deposit liability	$ 1.2

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 118,764	$ 88,205
Marketable securities	479,227	688,723
Accounts receivable, net	3,453	2,810
Inventory	23,070	19,076
Prepaid expenses and other current assets	39,113	35,021
Total current assets	663,627	833,835
Marketable securities	116,301	29,435
Operating lease assets	51,606	54,409
Property and equipment, net	14,119	15,382
Other non-current assets	4,056	4,057
Total assets	849,709	937,118
Current liabilities:
Accounts payable	11,155	9,780
Accrued expenses	25,129	43,167
Operating lease liabilities	15,407	15,008
Total current liabilities	51,691	67,955
Operating lease liabilities, net of current portion	52,940	56,988
Other non-current liabilities	1,156	1,156
Total liabilities	105,787	126,099
Stockholders’ equity:
Preferred stock, $0.001 par value; 25,000,000 shares authorized; no shares issued or outstanding at March 31, 2024 and December 31, 2023	0	0
Common stock, $0.001 par value; 125,000,000 shares authorized; 72,967,922 shares issued and 56,751,511 shares outstanding at March 31, 2024, and 72,161,489 shares issued and 55,945,078 shares outstanding at December 31, 2023	73	72
Additional paid-in capital	2,451,620	2,436,523
Accumulated other comprehensive loss	(1,087)	(441)
Treasury stock, at cost (16,216,411 shares at March 31, 2024 and December 31, 2023)	(802,486)	(802,486)
Accumulated deficit	(904,198)	(822,649)
Total stockholders’ equity	743,922	811,019
Total liabilities and stockholders’ equity	$ 849,709	$ 937,118