Atavistik Bio joins Cure HHT network as Harmony-HHT trial opens
Atavistik Bio has joined the Cure HHT Clinical Trial Network and opened the first clinical site for its Harmony-HHT Phase 1/2 study at Massachusetts General Hospital (MGH), the Cambridge, Massachusetts biotech announced on 30 July 2026. The move signals a meaningful step forward for ATV-1601, the company's investigational oral allosteric AKT1-selective inhibitor, which has received FDA Fast Track designation for hereditary haemorrhagic telangiectasia (HHT).
HHT is the second most common inherited bleeding disorder, affecting an estimated 80,000 people in the United States and 1.6 million globally. Despite that prevalence, no therapies have been approved for the condition. The disease is driven by loss-of-function mutations in the ENG, ALK1 or SMAD4 genes, which encode proteins regulating endothelial cell growth. Impaired function leads to hyperactivation of the AKT1 pathway and the formation of arteriovenous malformations, which can rupture, cause chronic bleeding, anaemia, organ damage and, in severe cases, life-threatening complications.
The collaboration
By joining the Cure HHT Clinical Trial Network, Atavistik Bio gains access to the organisation's trial-qualified Centres of Excellence, its established patient and clinician community, and disease-specific clinical and regulatory expertise. The two organisations will collaborate on patient education, community outreach and trial awareness activities aimed at connecting eligible individuals with the Harmony-HHT study.
Susan Pandya, Chief Medical Officer of Atavistik Bio, said the partnership "connects us with a global network of HHT experts, clinical investigators, and patient advocates who share our commitment to accelerating the development of novel treatment options."
The Harmony-HHT trial (NCT07601425) is structured as a proof-of-concept study. Part 1 is a randomised, double-blind, placebo-controlled, multicentre evaluation of three oral dosing regimens of ATV-1601 over 16 weeks. Participants who complete Part 1 may roll into an open-label extension, Part 2, to continue receiving the drug. ATV-1601's mechanism targets AKT1 selectively, and preclinical HHT models bearing each of the three major driver mutations showed significantly reduced arteriovenous malformation formation, which the company says supports a potential mutation-agnostic, disease-modifying profile.
Market context and competitive landscape
The rare vasculopathy space has attracted growing attention, partly because the FDA's Fast Track and Rare Disease frameworks can shorten development timelines and open priority review pathways. HHT in particular remains largely underserved: current clinical management relies heavily on symptomatic approaches such as iron supplementation, endoscopic intervention and, in some centres, off-label use of anti-angiogenic agents including bevacizumab, a monoclonal antibody not specifically approved for HHT. The absence of any approved oral disease-modifying therapy leaves a clear commercial and medical opportunity.
Atavistik Bio is backed by a notable syndicate that includes The Column Group, Lux Capital, RA Capital Management, Nextech Invest and Regeneron Ventures, suggesting the company has access to capital to sustain the multi-part trial. The company is also advancing a JAK2 V617F mutant-selective inhibitor programme for myeloproliferative neoplasms, indicating it is building a broader rare-haematology platform rather than a single-asset story.
Patient advocacy networks such as Cure HHT have increasingly taken on formal roles in trial execution, particularly in rare-disease settings where recruitment is structurally difficult due to small patient populations dispersed across specialist centres. The Cure HHT Clinical Trial Network is positioned as a first-of-its-kind platform for the indication, and its involvement may help Atavistik Bio expand site activation beyond MGH more quickly than a conventional site-by-site recruitment approach would allow. Investors will watch for additional site openings, interim safety data from Part 1 and, ultimately, evidence on AVM burden as the key efficacy read.