AI-driven biotech initiates Phase I trial for ALS drug 

AI brain

Verge Genomics, a clinical-stage, tech-enabled biotechnology company using artificial intelligence (AI) and human data to transform drug discovery, has announced today that the first subject has been dosed in a Phase I clinical trial of VRG50635 for amyotrophic lateral sclerosis (ALS). 

VRG50635 is a small molecule inhibitor of PIKfyve, a novel therapeutic target for ALS discovered by CONVERGE, Verge’s all-in-human, AI-powered platform. 

Through the evaluation of more than 11.4 million data points from ALS patient tissue and genetics datasets, CONVERGE discovered loss of endolysosomal function as a new causative mechanism in ALS, and uncovered PIKfyve as a promising new therapeutic target.  

VRG50635 is a potent PIKfyve inhibitor that restores endolysosomal function in ALS patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration. VRG50635 is the only PIKfyve inhibitor in clinical development that has been specifically optimised for treatment of central nervous system disorders like ALS, and has potential for best-in-class characteristics. 

This first-in-human study is a randomised, double-blind, placebo-controlled, single- and multiple-ascending-dose design to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VRG50635 in healthy volunteer subjects (Phase Ia) and multiple-dose study in patients with ALS (Phase Ib). 

“For years, researchers have heavily relied on animal or cell models to identify new targets, in a way that oversimplifies the enormous complexity of human biology, particularly for diseases like ALS,” said Robert Scannevin, PhD, Chief Scientific Officer at Verge Genomics. “The CONVERGE platform starts with, and integrates human data and human model systems throughout discovery and development. This provides unique insights into the biological underpinnings of ALS, and also predicts drug targets, like PIKfyve, that can broadly impact these complex, disease-relevant processes. We look forward to sharing the progress of our VRG50635 clinical studies and continued research in due course.” 

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