生产企业：Atavistik Bio
ATV-1601 is an investigational oral selective allosteric AKT1 inhibitor developed by Atavistik Bio for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT). It has received FDA IND clearance and Fast Track Designation. The Harmony-HHT Phase 1/2 trial (NCT07601425) is a randomized, double-blind, multicenter, placebo-controlled study evaluating three oral dosing regimens over 16 weeks in patients with moderate to severe HHT.
ATV-1601 is an investigational oral selective allosteric AKT1 inhibitor developed by Atavistik Bio for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT). It has received FDA IND clearance and Fast Track Designation. The Harmony-HHT Phase 1/2 trial (NCT07601425) is a randomized, double-blind, multicenter, placebo-controlled study evaluating three oral dosing regimens over 16 weeks in patients with moderate to severe HHT.
ATV-1601 is an oral selective allosteric inhibitor of AKT1. HHT is caused by loss-of-function mutations in ENG, ALK1, or SMAD4 genes, which encode proteins that regulate endothelial cell growth and branching. Impaired function of these proteins results in hyperactivation of the AKT1 pathway, leading to malformed blood vessels (arteriovenous malformations, AVMs). By selectively inhibiting AKT1, ATV-1601 addresses all HHT-driver mutations (ENG, ALK1, and SMAD4) and has the potential to provide a disease-modifying approach. In preclinical HHT models harboring ENG, ALK1, and SMAD4 mutations, ATV-1601 significantly reduced AVM formation.
ATV-1601 is being developed for the treatment of moderate to severe Hereditary Hemorrhagic Telangiectasia (HHT), the second most prevalent inherited bleeding disorder affecting more than 80,000 people in the U.S. and approximately 1.6 million people globally. Currently, no approved therapies are available for HHT. The Harmony-HHT Phase 1/2 trial (NCT07601425) is evaluating ATV-1601 in this indication.
共 1 项相关疾病
