Atavistik Bio Announces U.S. FDA Clearance of Investigational New Drug Application and Fast Track Designation for ATV-1601 for the Treatment of Hereditary Hemorrhagic Telangiectasia (HHT)
- FDA cleared the Harmony-HHT Phase 1/2 study design, enabling Atavistik Bio to leverage existing clinical data with ATV-1601 and advance directly into a randomized study with individuals who have moderate to severe HHT
- Fast Track Designation supports the development of ATV-1601 for HHT, the second most prevalent inherited bleeding disorder, affecting more than 80,000 people in the U.S. and 1.6 million people globally
- ATV-1601 is an oral, selective allosteric AKT1 inhibitor with disease-modifying potential for all driver mutations of HHT, which currently has no approved therapies for people with this severe and debilitating chronic disease
CAMBRIDGE, Mass., June 09, 2026 (GLOBE NEWSWIRE) -- Atavistik Bio, a biotechnology company discovering the next generation of selective allosteric therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for the Harmony-HHT study, a Phase 1/2 trial evaluating ATV-1601 in individuals with moderate to severe Hereditary Hemorrhagic Telangiectasia (HHT). In addition to the IND clearance, Atavistik Bio also announced that ATV-1601 has been granted Fast Track Designation by the FDA.
The FDA’s clearance of the Harmony-HHT Phase 1/2 study design allows Atavistik Bio to leverage existing clinical data with ATV-1601 and proceed directly into a randomized study. This approach creates the opportunity to accelerate development timelines while rapidly generating clinical proof-of-concept data.
“We recognize the significant burden HHT places on individuals and families, as recurrent bleeding, chronic anemia, and progressive complications can affect daily life and well-being,” said Susan Pandya, M.D., Chief Medical Officer at Atavistik Bio. “Treatment options are largely limited to supportive care and invasive interventions. ATV-1601 has the potential to provide a disease-modifying approach for people living with HHT. FDA clearance of our study design allows us to move directly into a randomized trial and, together with Fast Track Designation and existing ATV-1601 clinical experience, positions us to efficiently advance development for the HHT community.”
HHT is the second most prevalent inherited bleeding disorder, affecting more than 80,000 people in the U.S. and 1.6 million people globally, with no approved therapies currently available. HHT is caused by loss-of-function mutations in ENG, ALK1 or SMAD4 genes which encode proteins that regulate the growth and branching of endothelial cells into blood vessels. Impaired function of these proteins results in hyperactivation of the AKT1 pathway resulting in malformed blood vessels called arteriovenous malformations (AVMs), which can rupture or cause abnormal blood flow, leading to chronic bleeding, anemia, organ damage, and for some, life-threatening complications.
ATV-1601 is an investigational oral allosteric inhibitor that selectively targets AKT1, a key driver of the vasculopathy underlying HHT. By selectively inhibiting AKT1, ATV-1601 has the potential to provide a disease-modifying approach for HHT, with the potential to address all HHT-driver mutations (ENG, ALK1, and SMAD4) with a well-tolerated profile. In preclinical HHT models with ENG, ALK1, and SMAD4 mutations, ATV-1601 significantly reduced AVM formation, supporting its potential as a novel therapy applicable for all people living with HHT.
The Harmony-HHT study is a Phase 1/2 trial (NCT07601425) proof-of-concept study designed to evaluate the safety and efficacy of ATV-1601 for individuals with moderate to severe HHT. Part 1 is a randomized, double-blind, multicenter, placebo-controlled study evaluating three oral dosing regimens of ATV-1601 over a 16-week treatment period. Eligible participants who complete Part 1 may enroll in an open-label extension (Part 2) to receive ATV-1601.
About Atavistik Bio
Atavistik Bio is a clinical stage biotechnology company developing novel allosteric therapeutics, with an internal pipeline focused on rare hematology. Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of Hereditary Hemorrhagic Telangiectasia (HHT) and advancing a JAK2V617F mutant-selective inhibitor program for myeloproliferative neoplasms.
Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management. To learn more, visit us at atavistikbio.com and follow us on LinkedIn.
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