Verge Genomics Updates on ALS Treatment Collaboration with Lilly

Eli Lilly & Company (Lilly) has decided to move forward with the development of therapeutics targeting amyotrophic lateral sclerosis (ALS), following the identification and validation of two drug targets by Verge Genomics. This collaboration, which started in July 2021, leverages Verge’s AI-driven CONVERGE® platform. This platform has demonstrated a remarkable predictive accuracy, with 83% of its prioritized targets being validated in disease-relevant models, a rate that surpasses industry standards. This milestone has triggered immediate payments from Lilly to Verge, with the potential for further financial rewards as the programs advance.

Michael Hutton, Ph.D., Senior Vice President and Chief Scientific Officer of Neurodegeneration and Genetic Medicine at Lilly, praised the collaboration. He noted that the scientifically compelling target predictions and validation experiments conducted in human cell systems by Verge’s team and their CONVERGE® platform have resulted in multiple promising therapeutic targets. Hutton expressed optimism about advancing ALS drug candidates to meet urgent patient needs in this devastating disease.

The partnership between Verge and Lilly began in 2021 with a three-year agreement to identify and validate novel therapeutic targets for ALS, setting the stage for subsequent drug discovery and development efforts. Verge received up to $25 million upfront, including equity investment and potential near-term milestone payments, with the total deal value potentially reaching $694 million, plus possible future royalties.

Robert H. Scannevin, Ph.D., Chief Scientific Officer at Verge Genomics, emphasized the significance of this achievement, highlighting the ability of the CONVERGE® platform to uncover unique insights into the biological mechanisms of ALS and identify meaningful targets for complex diseases. Scannevin noted the synergy between computational predictions and experimental data, which has enabled Lilly to move forward with developing the selected targets.

Additionally, Verge is making headway with VRG50635, its leading drug candidate for treating sporadic and familial ALS. Currently, VRG50635 is under evaluation in a Phase 1B Proof-of-Concept study in Canada and several European countries. With enrollment complete, dose escalation is ongoing, including at the highest dose level. This study uses innovative technology to collect extensive, unbiased, objective molecular and clinical data relevant to the disease, assessing safety, tolerability, pharmacological dose-response, and efficacy, including potential disease modification. VRG50635, an orally bioavailable and brain-penetrant PIKfyve inhibitor, has shown promise in improving survival in ALS patient-derived motor neurons and has demonstrated efficacy in several preclinical studies using ALS-relevant models of motor neuron degeneration.

Verge Genomics is dedicated to developing treatments for complex diseases with significant unmet medical needs by utilizing human genomics from patient disease tissues and machine learning. The company has developed the proprietary, all-in-human CONVERGE® platform, which includes one of the largest and most comprehensive databases of multi-omic patient data in the field. Led by a team of experienced computational biologists and drug developers, Verge is advancing both clinical and preclinical therapeutic programs across various diseases.