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

Approved as QUVIVIQ™ in the US

MAA under review in other countries

Investor webcast

Aprocitentan*  Dual endothelin receptor antagonist Resistant hypertension management Phase 3 recruitment complete Investor webcast
Clazosentan Endothelin receptor antagonist Cerebral vasospasm assoc.
with aneurysmal subarachnoid hemorrhage

Approved as PIVLAZ™ in Japan

Global Phase 3 study ongoing

Investor webcast
Lucerastat Glucosylceramide synthase inhibitor Fabry disease

Phase 3 – primary endpoint not met

Open Label Extension study (OLE) ongoing

Selatogrel P2Y12 receptor antagonist Suspected acute myocardial infarction Phase 3 Investor webcast
Cenerimod S1P1 receptor modulator Systemic lupus erythematosus Phase 3 in preparation  
ACT-539313 Selective orexin 1 receptor antagonist Binge eating disorder Phase 2   
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-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 ACT-709478 (NBI-827104), Idorsia's novel T-type calcium channel blocker. ACT-709478 is currently investigated in two Phase 2 studies for the treatment of a rare form of pediatric epilepsy and essential tremor.