Drugs / VAD044
last change Dec 2025 re-read 3 minutes ago

VAD044

Small molecule targets AKT1 via inhibition

Developed for
hereditary hemorrhagic telangiectasia
Investigated by
Vaderis Therapeutics AG

Trials 1

20232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT05406362 Jul 2022 → Jan 2027 expected hereditary hemorrhagic telangiectasia Vaderis Therapeutics AG Active not recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 3

DateIssuerRelease
2025-12-02 Vaderis Therapeutics AG Results Vaderis Therapeutics Announces The New England Journal of Medicine Publishes Engasertib Proof-of-Concept and Long-Term Extension Results vaderis.com ↗
Vaderis Therapeutics today announced that data from its randomized, double-blind, placebo-controlled, multi-center proof-of-concept study and long-term extension of engasertib (VAD044) in Hereditary Hemorrhagic Telangiectasia (HHT) have been published in The New England Journal of Medicine (NEJM).
2024-11-17 Vaderis Therapeutics AG Regulatory Vaderis Receives FDA Fast Track Designation for VAD044 for the Treatment of Hereditary Hemorrhagic Telangiectasia vaderis.com ↗
Vaderis Therapeutics AG (Vaderis), a clinical stage biotechnology company focusing on treatments for rare diseases associated with vascular malformations, today announces that the US Food and Drug Administration (FDA) has designated the allosteric AKT-inhibitor VAD044 a Fast Track product for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT).
2024-08-27 Vaderis Therapeutics AG Results Vaderis Announces Positive Clinical Proof-of-Concept Trial in HHT vaderis.com ↗
Vaderis Therapeutics AG (Vaderis), a clinical stage biotechnology company focused on developing treatments for rare diseases associated with vascular malformations, today announces positive results from its randomized, double-blind, dose-finding placebo-controlled Proof-of-Concept (POC) clinical trial in patients suffering from HHT.

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 3 sources stand behind the page.

FieldValueCited text
Known as VAD044 ClinicalTrials.gov intervention name — accepted as the source's own label NCT05406362 ↗
Action Inhibit “a new, allosteric, selective AKT inhibitor” PMID 41297007 ↗ Nov 2025
Modality Small molecule — CHEMBL5314970 ↗
Route Oral “we evaluated the safety and efficacy of oral engasertib” PMID 41297007 ↗ Nov 2025
Target AKT “a new, allosteric, selective AKT inhibitor” PMID 41297007 ↗ Nov 2025