Verge reports positive clinical trial results for AI-discovered ALS drug

AI-based drug discovery biotech Verge Genomics has announced positive safety and tolerability data from the Phase 1 clinical trial of VRG50635, a potential therapeutic for amyotrophic lateral sclerosis (ALS).

The trial, conducted on healthy adult volunteers, demonstrated a favorable safety and tolerability profile, supporting the advancement of VRG50635 into a proof-of-concept study in people with ALS. It is one of the first drugs entirely developed from an AI-enabled platform to enter clinical trials and has progressed from discovery to the clinic in just four years.

The Phase 1 trial, which included 80 healthy adult volunteers, showed that VRG50635 was safe and well-tolerated, with no serious adverse events. The compound was escalated to the highest planned doses, and the pharmacokinetic analysis demonstrated dose-proportional increases in key parameters, supporting once-daily oral dosing of VRG50635. Verge plans to move forward with a proof-of-concept study in ALS patients with both sporadic and familial forms of the disease later this year.

“The lack of predictive animal models is one of the greatest challenges to developing effective ALS therapies today,” said Alice Zhang, CEO of Verge. “This is why we’re excited to advance VRG50635, a drug derived directly from human data, into trials in people with ALS. Our innovative proof-of-concept ALS study is designed to overcome historical challenges in ALS clinical trials by using state of the art technology, such as digital at-home devices and blood-based biomarkers, that can capture richer, higher-fidelity patient data and have the potential to detect efficacy with greater sensitivity.”

VRG50635 is a small molecule inhibitor of PIKfyve, a newly discovered therapeutic target for ALS. Verge discovered PIKfyve as a therapeutic target for ALS using its CONVERGE platform, which integrates large multi-omic datasets from CNS tissues of ALS patients. Verge has developed novel PIKfyve inhibitors optimized for CNS penetration that have successfully reversed disease-relevant pathology in multiple preclinical models.

Detailed results from the Phase 1 trial of VRG50635 will be presented at the upcoming European Network to Cure ALS (ENCALS) meeting in Barcelona, Spain.