Engasertib is an oral drug, an AKT inhibitor, showing significant success in clinical trials for Hereditary Hemorrhagic Telangiectasia (HHT), reducing nosebleed frequency and duration by targeting abnormal blood vessel development, showing safety and effectiveness, and longer-term benefits observed in ongoing studies.
Engasertib is a selective, allosteric inhibitor of the AKT protein kinase (AKT1/2).
It targets the AKT pathway, which is involved in cell growth and blood vessel formation.
By inhibiting AKT, it helps correct the faulty blood vessel development (arteriovenous malformations, AVMs) that causes HHT symptoms.
It significantly decreases the frequency and duration of epistaxis compared to placebo.
Improves quality of life.
It is generally well-tolerated, with mild, reversible rash and hyperglycemia being the most common side effects.
It has sustained and even greater reductions in bleeding over 12 months.
HHT is a rare genetic disorder with no approved treatments, but Engasertib offers hope as the first potential therapy.