San Francisco-based Verge Genomics has begun a phase 1 human trial for its ALS drug candidate, VRG50635, that was discovered by Artificial Intelligence, AI. This novel method of discovery may speed up drugs to ALS patients. Verge is a AI-driven biotech company that found a novel drug to bind a novel target, they hope to “leverage the vast amounts of medical data via AI to create a short cut to find new disease treating molecules”.
Verge started by building a database of sample tissues from the brains and spines of people with neurodegenerative diseases, including ALS, Parkinson’s, and Alzheimer’s. Starting with human data and human models “provides unique insights into the biological underpinnings of ALS,” Verge Genomics CSO Robert H. Scannevin said in the company’s statement.
The AI platform identified a loss of function in the cells’ endolysosomal system from patient tissues. Published work had identified an enzyme called PIKfyve as a potential drug target to ward off loss of function and AI suggested a small molecule named VRG50635, which inhibits PIKfyve, as a possible drug candidate. The process took four years. There is pre-clinical data that had to be obtained and an expectation that the drug would be able to get to the neurons in the central nervous system needed to be made.
The Verge trial is a placebo-controlled, randomized, double-blind phase 1 meant to test the basics of the drug — safety, pharmacokinetics, and impacting the intended target. They don’t know yet if the computer identified drug works and it will take a while to find out. If it does the method could shorten the discovery pipeline for ALS treatments.