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 |
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PIVLAZ® |
Endothelin receptor antagonist |
Cerebral vasospasm associated with |
Commercially available in Japan |
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QUVIVIQ™ (daridorexant) |
Dual orexin receptor antagonist |
Insomnia |
Commercially available in the US, Germany, and Italy; |
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Aprocitentan* |
Dual endothelin receptor antagonist |
Difficult-to-control (resistant) hypertension |
NDA under review in the US, MAA under review in the EU, other filings in preparation |
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Lucerastat |
Glucosylceramide synthase inhibitor |
Fabry disease |
Phase 3 primary endpoint not met, Open Label Extension study (OLE) ongoing |
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Selatogrel |
P2Y12 inhibitor |
Suspected acute myocardial infarction |
Phase 3 recruiting |
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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 |
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Sinbaglustat |
GBA2/GCS inhibitor |
Rare lysosomal storage disorders |
Phase 1 complete |
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ACT-1014-6470 |
C5aR1 antagonist |
Immune-mediated disorders |
Phase 1 |
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ACT-777991 |
CXCR3 antagonist |
Recent-onset Type 1 diabetes |
Phase 1 |
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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.