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Income Taxes (Tables)

12 Months Ended

Dec. 31, 2020

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,
			2020						2019
Deferred income tax assets:
Federal U.S. net operating loss carryforward			$	136,086					$	116,436
State net operating loss carryforward			37,465						31,110
Research and development credit, net			50,271						35,580
Orphan drug credit, net			23,409						22,881
Operating lease liabilities			8,048						8,413
Deferred revenue			3,132						367
Other			14,285						9,123
Gross deferred income tax assets			272,696						223,910
Valuation allowance			(263,403)						(214,893)
Net deferred income tax assets			9,293						9,017
Deferred income tax liabilities:
Depreciation			(2,123)						(438)
Operating lease ROU assets			(5,316)						(5,547)
Prepaid expenditures			(1,854)						(3,032)
Gross deferred income tax liabilities			(9,293)						(9,017)
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,
			2020						2019						2018
United States federal tax at statutory rate			$	(27,245)					$	(31,880)					$	(36,005)
State taxes (net of federal benefit)			(8,100)						(9,524)						(11,133)
Deferred income tax adjustments			344						2,004						(4,435)
Research credit, net			(14,691)						(5,830)						(8,466)
Orphan drug credit, net			(528)						(301)						(872)
Other permanent items			1,156						1,206						148
Equity-based compensation			554						1,889						758
Change in valuation allowance			48,510						42,436						60,005
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,
			2020						2019						2018
Beginning balance			$	4,950					$	4,318					$	3,395
Increases for current year tax positions			839						637						642
Increases/(decreases) for prior year tax positions			337						(5)						281
Ending balance			$	6,126					$	4,950					$	4,318

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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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