We have a diversified and balanced clinical development pipeline that covers multiple therapeutic areas, including CNS, cardiovascular and immunological disorders, as well as orphan diseases.

Compound Mechanism of Action Target Indication Status More information
         
Daridorexant Dual orexin receptor antagonist Insomnia Under review with FDA/EMA

Investor webcast (first study)

Investor webcast (second study)

Aprocitentan*  Dual endothelin receptor antagonist Resistant hypertension management Phase 3 Investor webcast
Clazosentan Endothelin receptor antagonist Vasospasm associated with aneurysmal subarachnoid hemorrhage (aSAH) Japan: NDA submitted
Global: Phase 3
Investor webcast
Lucerastat Glucosylceramide synthase inhibitor Fabry disease Phase 3 Investor webcast
Selatogrel P2Y12 receptor antagonist Suspected acute myocardial infarction Phase 3 in preparation  
Cenerimod S1P1 receptor modulator Systemic lupus erythematosus Phase 2b  
ACT-539313 Selective orexin 1 receptor antagonist Binge eating disorder Phase 2   
Sinbaglustat
GBA2/GCS inhibitor Rare lysosomal storage disorders Phase 1 complete  
ACT-1004-1239 CXCR7 antagonist Immunology  Phase 1  
ACT-1014-6470 - Immunology Phase 1  
ACT-541478 - CNS Phase 1  
ACT-777991 - Immunology Phase 1  


* In collaboration with Janssen Biotech to jointly develop aprocitentan, Janssen Biotech has sole commercialization rights worldwide

Neurocrine Biosciences has a global license to develop and commercialize our ACT-709478, a novel T-type calcium channel blocker. In November 2020, Neurocrine announced it had initiated a Phase 2 study investigating ACT-709478 for the treatment of a rare form of pediatric epilepsy.