Annual report pursuant to Section 13 and 15(d)

INCOME TAXES (Details)

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INCOME TAXES (Details) - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2022
Dec. 31, 2021
Dec. 31, 2020
Deferred tax assets      
Net operating loss carryforwards $ 230,373 $ 229,364  
Orphan drug and research and development credits 68,646 66,616  
Capitalized research and development credits 15,680 150  
Deferred revenue 11,234 16,297  
Deferred compensation 9,620 8,819  
Other, net 2,494 1,523  
Lease liabilities 504 2,564  
Deferred tax liabilities      
Operating lease right-of-use asset (461) (2,335)  
Others (439) (607)  
Total net deferred tax assets 337,651 322,391  
Less: valuation allowance (337,651) (322,391)  
Deferred tax assets, net of allowance  
Reconciliation of the statutory federal income tax rate to the effective tax rate      
Federal statutory tax rate (as a percent) (21.00%) (21.00%) (21.00%)
State, net of federal benefit (as a percent) 0.00% 2.80% 0.10%
Valuation allowance (as a percent) 20.20% 27.50% 24.40%
Stock compensation (as a percent) 2.50% 5.60% 4.70%
Orphan drug and research and development credits (as a percent) (2.60%) (14.00%) (12.70%)
Other, net (as a percent) 1.00% 2.70% 4.60%
Effective tax rate (as a percent) 0.10% 3.60% 0.10%