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Verge Genomics initiates first-in-human dosing in phase 1 trial of VRG50635, a novel therapeutic for Amyotrophic Lateral Sclerosis

By November 4, 2022April 9th, 2024No Comments

• New target discovered and delivered into the clinic in just four years using CONVERGE™, Verge’s all-in-human, AI-powered platform

• VRG50635 is one of the first drugs entirely discovered and developed from a tech-enabled platform to enter clinical trials

San Francisco, CA, October 31, 2022 – Verge Genomics, a clinical-stage, tech-enabled biotechnology company pioneering the use of artificial intelligence (AI) and human data to transform drug discovery, announced today that the first subject has been dosed in a Phase 1 clinical trial of VRG50635. VRG50635 is a small molecule inhibitor of PIKfyve, a novel therapeutic target for amyotrophic lateral sclerosis (ALS) discovered by CONVERGE™, Verge’s all-in-human, AI-powered platform.

“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 H. 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.”

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 optimized for treatment of central nervous system disorders like ALS, and has potential for best-in-class characteristics.

This first-in-human study is a randomized, 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 1a) and multiple-dose study in patients with ALS (Phase 1b). More information is available at

“We are proud to be not just one of the few AI-driven biotech companies to have made it to the clinical stage, but also one of the first to bring forward a novel clinical compound against a novel target, that was entirely discovered and developed internally on our platform,” said Alice Zhang, CEO of Verge Genomics. “Advancing VRG50635 from research to clinic in just four years offers clear proof of our team’s commitment to disciplined execution and is a leading indicator of the potential efficiency gains from tech-enabled drug discovery. This is just the start of our plan to develop a robust clinical pipeline of new medicines demonstrating that a human-to-human rather than an animal-to-human approach has the potential to transform clinical success rates.”

About PIKfyve

Verge discovered the relevance of PIKfyve in ALS using their computational platform, CONVERGE™, which incorporates large multi-’omic data sets from patients with disease. PIKfyve is a kinase that is believed to regulate endolysosomal function within a variety of cells, including neurons. The endolysosomal pathway is a critical cellular process involved in protein homeostasis. Verge and other top research groups have shown that in ALS, this pathway is dysregulated, leading to neuronal death and disease progression. After the discovery of PIKfyve as a therapeutic target for ALS, Verge developed PIKfyve inhibitors that have been effective in reversing disease-relevant pathology in multiple preclinical models of ALS.

About Amyotrophic Lateral Sclerosis (ALS)

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects neuronal cells in the brain and spinal cord. ALS often strikes people between the ages of 40 and 70, and it is estimated that 100,000 people worldwide may have the disease at any given time. ALS is fatal and there is currently no cure, and available therapies have only modest benefits. The majority of patients succumb to the disease within 2 to 5 years after diagnosis.

About Verge Genomics

Verge uses AI and human data to develop better drugs faster for the most challenging diseases of our generation. Verge has built CONVERGE™, an end-to-end drug discovery and development platform, that integrates multiple technological innovations from discovery to translation to streamline drug development. Its pioneering use of the industry’s most advanced all-in-human, AI-powered drug discovery platform identifies new medicines more efficiently and with an improved probability of success.

Verge is one of the first AI-enabled drug discovery companies to independently develop a clinical candidate from a novel target discovered from its platform. Verge has further demonstrated the power of CONVERGE™ by delivering a broad pipeline spanning diverse therapeutic areas, with its first program, an investigational PIKfyve inhibitor for the treatment of ALS, now in the clinic. The company is led by experienced drug developers and computational biologists with a shared belief that technology has created a new opportunity to deliver life-changing medicines more efficiently.

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Adam Silverstein
Scient PR

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