Vaderis Therapeutics AG, a clinical stage biotech company focused on developing treatments for rare diseases associated with vascular malformations, has emerged from stealth.
The company has also started its INSIGHT proof-of-concept clinical trial in patients suffering from hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu syndrome.
About HHT
HHT is a disorder where some blood vessels do not develop properly. A person with HHT may form blood vessels without the capillaries. This causes abnormal connections, called arteriovenous malformations (AVMs), to develop between arteries and veins. Most commonly affected are the nose, lungs, brain and liver. AVMs may enlarge over time and can bleed or rupture.
Emergence from stealth
Vaderis Therapeutics was established in 2019 and in 2020 raised more than CHF18 million ($18.8 million) from Medicxi, enabling progression of its lead asset into the clinic and successful completion of phase 1a.
Vaderis Therapeutics was founded with the aim of developing 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 in Belfast, U.K.
AKT is a serine kinase that plays a crucial role in vascular growth and in vascular overgrowth. There are 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 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, 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.
HHT trial
Vaderis’ first study of VAD044 in HHT patients, the INSIGHT proof-of-concept trial, aims to achieve a thorough understanding of the safety of VAD044 in HHT patients. A total of 80 HHT patients across the U.S., 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 FDA, Health Canada and key European agencies.
J.J. Mager, 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, said: “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 Therapeutics, said: “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.”