Verge Genomics (Verge) announced that the first subject has been dosed in its Phase 1 clinical trial evaluating VRG50635, a small molecule inhibitor of PIKfyve, as a treatment for amyotrophic lateral sclerosis (ALS), also known as motor neuron.
Characterized by progressive degeneration of motor nerve cells in the brain and spinal cord, ALS leads to deterioration of muscle function, inability to move and speak, respiratory paralysis and eventually death.
VRG50635 was discovered by the company’s all-human AI-powered platform – CONVERGE – and is the only PIKfyve inhibitor in clinical development that has been specifically optimized for the treatment of central nervous system disorders like ALS .
The platform uncovered PIKfyve as a promising new therapeutic target through the evaluation of over 11.4 million data points from genetic and tissue datasets of ALS patients, uncovering the loss of endolysosomal function as a new causal mechanism in ALS.
VRG50635 restores endolysosomal function in neurons of ALS patients and has been shown to be effective in multiple preclinical studies in motor neuron degeneration models relevant to ALS.
“For years, researchers have relied heavily on animal or cell models to identify new targets, in a way that oversimplifies the enormous complexity of human biology, especially for diseases like ALS. “said Robert Scannevin, scientific director of Verge.
“The CONVERGE platform begins 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 may have a broad impact on these complex, disease-relevant processes,” he added.
This first-in-man study is a randomized, double-blind, placebo-controlled, single-dose, and multiple-escalating-dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VRG50635 in healthy volunteers – phase 1a – and multiple dose study in ALS patients – phase 1b.
Alice Zhang, CEO of Verge Genomics, said, “We are proud to be not only one of the few AI-focused biotech companies to reach the clinical stage, but also one of the first to offer a new clinical project. against a new target, which was entirely discovered and developed in-house on our platform.
“…This is just the beginning of our plan to develop a robust clinical pipeline of new drugs demonstrating that a human-to-human rather than animal-based approach has the potential to transform clinical success rates.”