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Income Taxes (Tables)
12 Months Ended
Dec. 31, 2023
Income Tax Disclosure [Abstract]  
Schedule of Reconciliation of U.S. Federal Statutory Income Tax Rate

A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows:

 

 

 

Year Ended
December 31,
2023

 

 

Year Ended
December 31,
2022

 

Federal income tax at statutory rate

 

 

21.00

%

 

 

21.00

%

Permanent differences

 

 

(0.71

)

 

 

(0.46

)

Federal and state research and development credits

 

 

1.47

 

 

 

1.44

 

Federal orphan drug credits

 

 

9.41

 

 

 

7.23

 

State income tax, net of federal benefit

 

 

5.92

 

 

 

5.78

 

Officers' compensation

 

 

(1.06

)

 

 

(0.23

)

Impact of ownership change

 

 

 

 

 

(2.92

)

Other

 

 

(0.10

)

 

 

(0.13

)

Change in valuation allowance

 

 

(35.93

)

 

 

(31.71

)

Effective income tax rate

 

 

%

 

 

%

Schedule of Deferred Tax Assets and Liabilities

The Company’s deferred tax assets and liabilities consist of the following (in thousands):

 

 

 

December 31,
2023

 

 

December 31,
2022

 

Deferred tax assets:

 

 

 

 

 

 

Net operating loss carryforwards

 

$

85,622

 

 

$

75,005

 

Capitalized research and development costs

 

 

30,019

 

 

 

16,911

 

Orphan drug credit carryforwards

 

 

23,790

 

 

 

14,628

 

Research and development credit carryforwards

 

 

11,569

 

 

 

10,140

 

Intangible assets

 

 

6,291

 

 

 

6,918

 

Accrued expenses and other

 

 

6,740

 

 

 

5,232

 

Operating lease liability

 

 

2,911

 

 

 

3,613

 

Deferred revenue

 

 

 

 

 

251

 

Gross deferred tax assets

 

 

166,942

 

 

 

132,698

 

Valuation allowance

 

 

(164,483

)

 

 

(129,512

)

Net deferred tax assets

 

 

2,459

 

 

 

3,186

 

Deferred tax liability

 

 

(2,459

)

 

 

(3,186

)

Net deferred tax assets

 

$

 

 

$