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Income Taxes (Tables)

12 Months Ended

Dec. 31, 2021

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,
			2021						2020
Deferred income tax assets:
Federal U.S. net operating loss carryforward			$	175,802					$	136,086
State net operating loss carryforward			49,965						37,465
Research and development credit, net			60,514						50,271
Orphan drug credit, net			24,858						23,409
Operating lease liabilities			7,008						8,048
Deferred revenue			1,245						3,132
Other			16,727						14,285
Gross deferred income tax assets			336,119						272,696
Valuation allowance			(327,595)						(263,403)
Net deferred income tax assets			8,524						9,293
Deferred income tax liabilities:
Depreciation			(1,688)						(2,123)
Operating lease ROU assets			(4,576)						(5,316)
Prepaid expenditures			(2,260)						(1,854)
Gross deferred income tax liabilities			(8,524)						(9,293)
Net deferred income tax asset/(liability)			$	—					$	—

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,
			2021						2020						2019
United States federal tax at statutory rate			$	(42,445)					$	(27,245)					$	(31,880)
State taxes (net of federal benefit)			(12,806)						(8,100)						(9,524)
Deferred income tax adjustments			473						344						2,004
Research credit, net			(10,243)						(14,691)						(5,830)
Orphan drug credit, net			(1,449)						(528)						(301)
Other permanent items			1,199						1,156						1,206
Equity-based compensation			1,079						554						1,889
Change in valuation allowance			64,192						48,510						42,436
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,
			2021						2020						2019
Beginning balance			$	6,126					$	4,950					$	4,318
Increases for current year tax positions			965						839						637
Increases/(decreases) for prior year tax positions			106						337						(5)
Ending balance			$	7,197					$	6,126					$	4,950

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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 the change in unrecognized tax benefits.

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