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Vaderis Emerges from Stealth and Initiates Clinical Proof-of-Concept Trial in HHT

August 22, 2022

Vaderis Therapeutics, a clinical stage company focused on developing treatments for rare diseases associated with vascular malformations, emerged from stealth and initiated a proof-of-concept clinical trial in patients suffering from hereditary hemorrhagic telangiectasia, also known as Osler-Weber-Rendu Syndrome.

Photo: Nicholas Benedict, CEO of Vaderis

Vaderis was established in 2019 and in 2020 raised more than $19 million (CHF18 million) from Medicxi, enabling progression of its lead asset into the clinic and successful completion of a phase 1a trial.

Vaderis was founded to develop therapeutics for rare and orphan diseases associated with vascular malformations.  In April 2020 Vaderis acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK.  AKT is a serine kinase which plays a crucial role in vascular growth and in vascular overgrowth. 

In hereditary hemorrhagic telangiectasia (HHT), upstream genetic mutations trigger over-activation of the serine kinase AKT, resulting in vascular overgrowth.  Such diseases frequently manifest in ways that are debilitating, disfiguring, painful, and sometimes life-threatening.  The vast majority of these diseases are currently left untreated except for symptomatic interventions which tend to offer patients limited temporary relief of individual symptoms.

VAD044 is a once daily, orally administered, allosteric AKT inhibitor that has the potential to treat the underlying cause of these diseases.  If successful, Vaderis would be the first company in the world to develop a medicine for the treatment of HHT and other diseases associated with vascular malformations.

In pursuit of the company’s core objective, Vaderis said it initiated the first study of VAD044 in HHT patients. Known as the INSIGHT proof-of-concept trial (INternational Study InvestiGating HHT), the study is aimed at achieving a thorough understanding of the safety of VAD044 in HHT patients. A total of 80 HHT patients across the United States, Canada, and Europe will be randomized in a double-blind, controlled trial comparing two doses of VAD044 to placebo.  Initiation of the INSIGHT trial follows Health Authority approvals including the U.S. Food and Drug Administration, Health Canada, and key European agencies.

“Working together with the HHT community, both caregivers and patient associations, Vaderis hopes to be the catalyst which transforms patient care in HHT,” said Nicholas Benedict, CEO of Vaderis. “Success in the INSIGHT trial would be a major step towards developing the first ever registered treatment for HHT anywhere in the world.”

Author: Rare Daily Staff

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