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Income Taxes (Tables)

12 Months Ended

Dec. 31, 2023

Income Tax Disclosure [Abstract]

Components of the Company's Deferred Income Tax Assets (Liabilities)

The significant components of the Company's deferred income tax assets (liabilities) were as follows (in thousands):

			December 31,
			2023						2022
Deferred income tax assets:
Federal U.S. net operating loss carryforward			$	140,706					$	163,071
State net operating loss carryforward			33,350						44,784
Research and development credit, net			68,251						65,084
Orphan drug credit, net			33,330						35,703
Operating lease liabilities			9,078						9,585
Deferred revenue			17,442						—
Section 174 deferred tax asset			48,421						43,192
Equity based compensation			16,217						15,228
Other			6,617						4,663
Gross deferred income tax assets			373,412						381,310
Valuation allowance			(365,010)						(372,267)
Net deferred income tax assets			8,402						9,043
Deferred income tax liabilities:
Operating lease ROU assets			(6,372)						(7,522)
Prepaid expenditures			(2,030)						(1,521)
Gross deferred income tax liabilities			(8,402)						(9,043)
Net deferred income tax asset/(liability)			$	—					$	—

Summary of Valuation Allowance

The activity in the valuation allowance on deferred tax assets was as follows (in thousands):

			Balance at Beginning of Year						Additions						Deductions						Balance at End of Year
Year Ended December 31, 2023			$	372,267					$	—					$	(7,257)					$	365,010
Year Ended December 31, 2022			$	327,592					$	44,675					$	—					$	372,267
Year Ended December 31, 2021			$	263,400					$	64,192					$	—					$	327,592

Reconciliation of Reported Estimated Income Tax Benefit

The reconciliation of the reported estimated income tax benefit to the amount that would result by applying the U.S. federal statutory tax rate to the net income is as follows (in thousands):

			Year Ended December 31,
			2023						2022						2021
United States federal tax at statutory rate			$	(1,902)					$	(25,149)					$	(42,445)
State taxes (net of federal benefit)			(163)						(7,385)						(12,806)
Deferred income tax adjustments			5,024						308						473
Deferred state blended rate adjustments			1,841						—						—
Research credit, net			(3,168)						(4,569)						(10,243)
Orphan drug credit, net			2,374						(10,846)						(1,449)
Other permanent items			1,301						1,362						1,199
Equity-based compensation			1,950						1,604						1,079
Change in valuation allowance			(7,257)						44,675						64,192
Income tax expense/(benefit)			$	—					$	—					$	—

Reconciliation of the Beginning and Ending Amount of Gross Unrecognized Tax Benefits

A reconciliation of the beginning and ending amount of gross unrecognized tax benefits is as follows (in thousands):

			Year Ended December 31,
			2023						2022						2021
Beginning balance			$	7,376					$	7,197					$	6,126
Increases for current year tax positions			449						548						965
Increases/(decreases) for prior year tax positions			(4)						(369)						106
Ending balance			$	7,821					$	7,376					$	7,197

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Tabular disclosure of the components of net deferred tax asset or liability recognized in an entity's statement of financial position, including the following: the total of all deferred tax liabilities, the total of all deferred tax assets, the total valuation allowance recognized for deferred tax assets.

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Tabular disclosure of the reconciliation using percentage or dollar amounts of the reported amount of income tax expense attributable to continuing operations for the year to the amount of income tax expense that would result from applying domestic federal statutory tax rates to pretax income from continuing operations.

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Tabular disclosure of valuation allowances to reduce deferred tax assets to net realizable value, including identification of the deferred tax asset more likely than not will not be fully realized and the corresponding amount of the valuation allowance.

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