Verge Genomics, a biotechnology company using artificial intelligence (AI) to develop new treatment options, has announced the launch of a new study for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS).
The study will assess the safety and tolerability of escalating doses of VRG50635, a small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered in diseased human tissues using CONVERGE®, the company’s all-in-human, AI-powered platform.
VRG50635 is one of the first drugs to enter the clinic that was entirely discovered and developed from an AI-enabled platform.
In traditional ALS clinical trials, a patient’s progress is measured in a clinical setting using poorly responsive physician rating scales, which typically provide sparse data to establish proof of concept early in clinical development. In contrast, Verge’s study harnesses the power of digital devices to collect thousands of objective, quantitative, relevant measurements directly from the patient, including mobility, breathing, and sleeping all from the comfort of their home.
This level of patient data greatly increases the ability to detect disease progression and treatment-related changes, allowing to personalize the assessment of change for each study participant as he/she moves from no treatment to increasingly higher doses of VRG50635, and for longer treatment periods.
Diego Cadavid, M.D., FAAN, FANA, chief medical officer at Verge Genomics, commented: “Incorporating cutting-edge technology into our ALS proof-of-concept study is a necessity born from the urgent unmet need to efficiently study promising therapeutics for people battling this devastating disease. Our approach and the technology we are incorporating into the VRG50635 proof-of-concept study will make it possible to collect dense amounts of unbiased, objective disease-relevant data to properly assess the safety, tolerability, and potential efficacy, including dose-response, early in clinical development.
Alice Zhang, CEO and co-founder of Verge Genomics, said: “Initiating our proof-of-concept study marks another important milestone for Verge and further demonstrates that by leveraging our AI-enabled platform in combination with patient data using a novel study design we are able to more efficiently discover and develop potential drugs for deadly diseases like ALS.
“We are tremendously excited by the potentially transformative power of our CONVERGE® platform in redefining the landscape and expediting the journey from lab bench to patients and improving lives worldwide.”
Dr. Angela Genge, director, ALS Centre of Excellence for Research and Patient Care, Montreal Neurological Institute-Hospital, added: “I applaud Verge for creating a bold proof-of-concept design that incorporates cutting-edge technology and scientific innovation in their efforts to create groundbreaking therapies for ALS.”
