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Income Taxes (Tables)

12 Months Ended

Dec. 31, 2016

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,
	2016				2015
Deferred income tax assets:
Federal U.S. net operating loss carryforward	$	75,377			$	57,949
State net operating loss carryforward	6,583				3,907
Research and development credit, net	12,829				10,278
Orphan drug credit, net	19,855				19,284
Deferred rent	2,497				2,947
Deferred revenue	5,098				6,632
Depreciation	2,926				1,597
Other	5,085				2,532
Gross deferred income tax assets	130,250				105,126
Valuation allowance	(128,844		)		(104,399		)
Net deferred income tax assets	1,406				727
Deferred income tax liabilities:
Prepaid expenditures	(1,406		)		(727		)
Gross deferred income tax liabilities	(1,406		)		(727		)
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,
	2016				2015				2014
United States federal tax at statutory rate	$	(20,489	)		$	(7,049	)		$	(13,410	)
State taxes (net of federal benefit)	(3,116		)		(897		)		(1,608		)
Deferred income tax adjustments	173				661				—
Deferred state blended rate adjustments	(32		)		(493		)		3,034
Research credit, net	(2,551		)		(3,296		)		(2,228		)
Transaction cost deduction	—				—				(379		)
Transaction cost deduction - prior year adjustment	—				—				(564		)
Orphan drug credit, net	(571		)		(106		)		(139		)
Other permanent items	145				(25		)		(382		)
Equity-based compensation	1,997				1,102				756
Change in valuation allowance	24,444				10,103				14,920
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,
	2016				2015				2014
Beginning balance	$	2,425			$	2,047			$	1,708
Increases/(decreases) for current year tax positions	308				357				242
Increases/(decreases) for prior year tax positions	(268		)		21				97
Ending balance	$	2,465			$	2,425			$	2,047

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- References

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- Definition

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.

+ References

Reference 1: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 2
-URI http://asc.fasb.org/extlink&oid=6907707&loc=d3e32537-109319

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- Definition

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.

+ References

Reference 1: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 12
-URI http://asc.fasb.org/extlink&oid=6907707&loc=d3e32687-109319

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Tabular disclosure of the change in unrecognized tax benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 55
-Paragraph 217
-URI http://asc.fasb.org/extlink&oid=51663037&loc=d3e36027-109320

Reference 2: http://www.xbrl.org/2003/role/presentationRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 15A
-URI http://asc.fasb.org/extlink&oid=6907707&loc=SL6600010-109319

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