We have a diversified and balanced portfolio covering multiple therapeutic areas, including CNS, cardiovascular, and immunological disorders, as well as orphan diseases.

         

Compound

Mechanism of action

Target indication

Status

More Information

PIVLAZ®
(clazosentan)

Endothelin receptor antagonist

Cerebral vasospasm associated with
aneurysmal subarachnoid hemorrhage

Commercially available in Japan

 

QUVIVIQ™   (daridorexant)

Dual orexin receptor antagonist

Insomnia

Commercially available in the US and the first countries in Europe;
approved in the UK and Switzerland;
under review in Canada;
Phase 3 in Japan successful – filing expected in H2 2023;
Phase 2 in pediatric insomnia recruiting

Investor webcast

Aprocitentan*

Dual endothelin receptor antagonist

Difficult-to-control (resistant) hypertension

NDA submitted in the US, MAA submitted in the EU, other filings in preparation

Investor webcast

Lucerastat

Glucosylceramide synthase inhibitor

Fabry disease

Phase 3 primary endpoint not met, Open Label Extension study (OLE) ongoing

 

Selatogrel

P2Y12 inhibitor

Suspected acute myocardial infarction

Phase 3 recruiting

Investor webcast

Cenerimod

S1P1 receptor modulator

Systemic lupus erythematosus

Phase 3 recruiting

Investor webcast

ACT-1004-1239

ACKR3 / CXCR7 antagonist

Multiple sclerosis and other demyelinating diseases

Phase 2 in preparation

 

Sinbaglustat

GBA2/GCS inhibitor

Rare lysosomal storage disorders

Phase 1 complete

 

ACT-1014-6470

C5aR1 antagonist

Immune-mediated disorders

Phase 1

 

ACT-777991

CXCR3 antagonist

Recent-onset Type 1 diabetes

Phase 1

 

IDOR-1117-2520

Undisclosed

Immune-mediated disorders

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 was investigated in a Phase 2 study for the treatment of a rare form of pediatric epilepsy. The study did not meet the primary endpoint. ACT-709478 was generally well tolerated. Neurocrine continues to analyze the data generated in the study.