From stealth mode into clinical trials

Established in 2019, Vaderis Therapeutics AG aimed 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 that plays a crucial role in vascular growth and overgrowth. 

There are many rare diseases such as HHT in which upstream genetic mutations trigger over-activation of the serine kinase AKT, resulting in vascular overgrowth.  Such diseases frequently manifest in debilitating, disfiguring, painful, and sometimes life-threatening ways.  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. The company's lead candidate VAD044 is a once-daily, orally administered, allosteric AKT inhibitor which 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 announces the initiation of the first study of VAD044 in HHT patients. Known as the INSIGHT proof-of-concept trial (INternational Study InvestiGating HHT), the study is unique in its robust design aimed at achieving a thorough understanding of the safety of VAD044 in HHT patients. A total of 80 HHT patients across the USA, Canada and Europe will be randomised in a double-blind, controlled trial comparing two doses of VAD044 to placebo.  Initiation of the INSIGHT trial follows Health Authority approvals including FDA, Health Canada and key European agencies.

J.J. Mager, MD, PhD, pulmonologist at St. Antonius Hospital Utrecht, Chairman of the Global Research and Medical Advisory Board of Cure HHT and lead investigator of the INSIGHT trial commented, "The first patients recruited into the INSIGHT trial mark an exciting milestone in HHT clinical research.  If successful, this trial may demonstrate the potential of this new drug to be effective beyond the symptomatic treatments we currently offer HHT patients, by addressing the course of this rare, progressive, and debilitating disease."

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

(Press release/RAN)