engasertib

Engasertib (VAD044) is an oral, small molecule, selective allosteric inhibitor of AKT (protein kinase B) being developed by Vaderis Therapeutics. It is specifically designed to treat Hereditary Hemorrhagic Telangiectasia (HHT), a rare genetic vascular disorder characterized by malformed blood vessels that are prone to bleeding. By targeting the AKT pathway, which is overactive in HHT-related vascular lesions, engasertib aims to reduce the frequency and duration of epistaxis (nosebleeds) and improve anemia. Clinical data from Phase 2 studies demonstrated significant reductions in bleeding duration and frequency, leading to FDA Fast Track designation. The drug is currently being prepared for Phase 3 clinical evaluation.