Vaderis Therapeutics AG Emerges from Stealth and Announces Initiation of Clinical Proof-of-Concept Trial in HHT
Tuesday, August 23, 2022
Vaderis Therapeutics AG, a clinical stage biotechnology company specializing in treatments for rare diseases associated with vascular malformations, has emerged from stealth mode and commenced its INSIGHT proof-of-concept clinical trial. This trial focuses on patients with Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu Syndrome. Vaderis, established in 2019, raised over CHF18 million from Medicxi in 2020, enabling the advancement of its lead asset into clinical trials and the successful completion of Phase 1a.
Emergence from Stealth
Vaderis was founded with a unique mission 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 in Belfast, UK. AKT, a serine kinase, plays a critical role in vascular growth and overgrowth. Many rare diseases, including HHT, involve upstream genetic mutations that trigger over-activation of AKT, resulting in abnormal vascular growth. These diseases often cause debilitation, disfigurement, pain, and even life-threatening complications. Currently, most of these diseases lack targeted treatments and rely on symptomatic interventions that offer limited temporary relief. VAD044, an orally administered allosteric AKT inhibitor taken once daily, has the potential to address the underlying cause of these diseases. If successful, Vaderis would be the first company worldwide to develop a medicine for the treatment of HHT and other diseases associated with vascular malformations.
Initiation of Clinical Proof-of-Concept (PoC) in HHT
Aligned with its core objective, Vaderis has announced the initiation of the first study of VAD044 in HHT patients. The INSIGHT proof-of-concept trial (INternational Study InvestiGating HHT) is designed to provide a comprehensive understanding of the safety of VAD044 in HHT patients. The study will include 80 HHT patients from the USA, Canada, and Europe, who will be randomized in a double-blind, controlled trial comparing two doses of VAD044 to a placebo. The initiation of the INSIGHT trial follows approvals from regulatory bodies such as the FDA, Health Canada, and key European agencies.
Dr. 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, expressed enthusiasm, stating, "The recruitment of the first patients into the INSIGHT trial signifies an exciting milestone in HHT clinical research. If successful, this trial may demonstrate the potential of this new drug to go beyond symptomatic treatments and address the course of this rare, progressive, and debilitating disease."
Nicholas Benedict, CEO of Vaderis, emphasized the company's commitment to collaborate with the HHT community, including caregivers and patient associations, with the aim of transforming patient care for HHT. Success in the INSIGHT trial would be a significant step towards developing the first registered treatment for HHT globally.
Giovanni Mariggi, Partner at Medicxi and Chairman of Vaderis, highlighted the company's vision to deliver groundbreaking therapies for rare diseases caused by vascular malformations. The initiation of the INSIGHT trial represents an important milestone in realizing this vision and underscores the significant progress made by Vaderis over the past two years.
