Verge Genomics, a clinical-stage biotechnology company transforming drug discovery and development using artificial intelligence (AI) and human data, announced the initiation of its Phase 1b proof-of-concept study of VRG50635 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 potential best-in-class, 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, and 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 the team to personalize the assessment of change for each study participant as they move from no treatment to increasingly higher doses of VRG50635, and for longer treatment periods.
“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,” said Diego Cadavid, M.D., FAAN, FANA, Chief Medical Officer at Verge Genomics.
“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,” said Alice Zhang, CEO and co-founder of Verge Genomics.