Atavistik Bio Joins Cure HHT Clinical Trial Network to Advance ATV-1601 for Hereditary Haemorrhagic Telangiectasia

Atavistik Bio Expands HHT Clinical Development Efforts

Atavistik Bio has announced that it has joined the Cure HHT Clinical Trial Network (CTN) to support the clinical development of its investigational medicine, ATV-1601, for people living with Hereditary Haemorrhagic Telangiectasia (HHT).

The announcement comes alongside another important milestone for the company. The first clinical site for the Harmony-HHT Phase 1/2 clinical trial has now been activated at Massachusetts General Hospital (MGH), officially moving the study into the next stage of development.

Together, these developments are expected to strengthen Atavistik Bio's efforts to bring a potential new treatment to patients living with this rare inherited bleeding disorder.

Partnership Will Help Speed Up Clinical Research

By becoming part of the Cure HHT Clinical Trial Network, Atavistik Bio will work closely with Cure HHT throughout the Harmony-HHT clinical study.

The collaboration gives the company access to several important resources, including:

• Cure HHT's network of trial-qualified Centers of Excellence.
• An established community of patients and healthcare professionals.
• Clinical and regulatory expertise focused specifically on HHT.

The Cure HHT Clinical Trial Network was created to help accelerate the development of new therapies by bringing together researchers, treatment centers, patient advocates, and biotechnology companies.

The goal is to make clinical studies more efficient while improving patient access to investigational treatments.

First Study Site Opens at Massachusetts General Hospital

Atavistik Bio has activated the first clinical trial site for the Harmony-HHT study at Massachusetts General Hospital, one of the leading HHT Centers of Excellence.

According to Dr. Susan Pandya, Chief Medical Officer of Atavistik Bio, opening the first study site and joining the Cure HHT Clinical Trial Network allows the company to work more closely with HHT experts while also improving engagement with the patient community.

She said the partnership creates opportunities to collaborate with investigators, physicians, and patient advocates who all share the same goal of developing better treatment options for people living with HHT.

Community Outreach Will Be Part of the Collaboration

The partnership is not limited to clinical research alone.

Atavistik Bio and Cure HHT will also work together on several patient-focused initiatives, including:

• Patient education programs.
• Community outreach activities.
• Clinical trial awareness campaigns.
• Helping eligible patients learn about and participate in the Harmony-HHT study.

Cure HHT CEO Marianne Clancy welcomed Atavistik Bio into the network and said ATV-1601 represents an exciting new investigational treatment approach for people living with HHT.

She added that the organization looks forward to helping connect patients with the ongoing clinical trial.

What Is Hereditary Haemorrhagic Telangiectasia?

Hereditary Haemorrhagic Telangiectasia, commonly called HHT, is a rare inherited disease that affects blood vessel formation.

It is considered the second most common inherited bleeding disorder.

The disease affects more than 80,000 people in the United States and approximately 1.6 million people worldwide.

Despite the number of people living with HHT, there are currently no approved therapies specifically developed to treat the disease.

HHT develops because of mutations in one of three genes:

• ENG
• ALK1
• SMAD4

These genes normally help control how blood vessels grow and develop.

When they do not function properly, the AKT1 signaling pathway becomes overactive, leading to abnormal blood vessel formation.

These abnormal blood vessels, known as arteriovenous malformations (AVMs), can rupture or interfere with normal blood flow.

As a result, patients may experience:

• Frequent bleeding episodes.
• Chronic anaemia.
• Damage to vital organs.
• Serious complications that may become life-threatening.

Because there are no approved disease-modifying treatments available today, there remains a significant unmet medical need.

How ATV-1601 Works

ATV-1601 is an investigational oral medicine designed to selectively inhibit AKT1, one of the key biological pathways involved in HHT.

Unlike treatments that only manage symptoms, ATV-1601 is being developed as a disease-modifying therapy that aims to address the underlying cause of HHT.

The medicine has the potential to benefit patients regardless of whether their disease is caused by mutations in the ENG, ALK1, or SMAD4 genes.

Preclinical research has shown encouraging results.

Studies demonstrated that ATV-1601 significantly reduced the formation of abnormal blood vessels in multiple HHT disease models involving all three genetic mutations.

Because of its potential, the US Food and Drug Administration (FDA) has granted ATV-1601 Fast Track designation for the treatment of HHT.

Harmony-HHT Phase 1/2 Study Is Now Underway

The Harmony-HHT Phase 1/2 clinical trial is designed as a proof-of-concept study to evaluate both the safety and effectiveness of ATV-1601 in patients with moderate to severe HHT.

The study has two parts.

Part 1 is a randomized, double-blind, placebo-controlled, multicenter trial that will evaluate three different oral dose levels of ATV-1601 over a 16-week treatment period.

Patients who successfully complete Part 1 may continue into Part 2, which is an open-label extension study where all participants will receive ATV-1601.

Researchers will use the study to better understand the medicine's safety profile while also evaluating its ability to improve disease outcomes.

Atavistik Bio Continues to Build Its Rare Disease Pipeline

Atavistik Bio is a clinical-stage biotechnology company focused on developing new therapies for rare blood-related diseases using its proprietary allosteric drug discovery platform.

Alongside ATV-1601, the company is also advancing a selective JAK2V617F inhibitor program for the treatment of myeloproliferative neoplasms.

The company is led by an experienced drug development team with a history of bringing small-molecule therapies to market.

Its research programs are supported by several leading life science investors, including The Column Group, Nextech Invest, Lux Capital, Regeneron Ventures, and RA Capital Management.