Verge Genomics Announces Milestones in Collaboration with Lilly to Discover and Develop Novel Treatments for ALS

— Lilly to Develop Two Targets Identified and Validated Using Verge Genomics’ AI-Enabled, All-in-Human CONVERGE® Platform

— Demonstration of the Robust Predictive Power of CONVERGE® - 83% of Prioritized Targets were Validated in Disease-Relevant Models, a Rate Significantly Higher than Industry Standards 

SOUTH SAN FRANCISCO, November 20, 2024 – Verge Genomics, a clinical-stage biotechnology company pioneering the use of artificial intelligence (AI) and human data to transform drug discovery and development, announced today that Eli Lilly & Company (“Lilly”) (NYSE: LLY) has opted to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) as part of the companies’ collaboration, which was initiated in July 2021. The targets were identified and validated by Verge’s AI-enabled CONVERGE® platform, with 83% of prioritized targets validated in disease-relevant models, a rate significantly higher than industry standards, demonstrating the robust predictive power of CONVERGE®. Target selection triggered near-term milestone payments from Lilly to Verge. Verge is eligible for additional downstream economics as the programs progress.

“By providing us with scientifically compelling target predictions, which were supported by elegant validation experiments in human cell systems, the Verge team and CONVERGE® platform have delivered multiple promising therapeutic targets,” said Michael Hutton, Ph.D., Senior Vice President and CSO, Neurodegeneration and Genetic Medicine at Lilly. “We look forward to building upon their work with the aim of advancing ALS drug candidates to address the significant unmet patient needs in this fatal disease.”

In 2021, Verge and Lilly entered into a 3-year collaboration to discover and validate novel therapeutic targets for ALS that would become the focus for subsequent drug discovery and development. Verge received up to $25 million in upfront, equity investment and potential near-term milestones, with a total deal value of $694 million plus potential downstream royalties.

“This achievement further validates the predictive power of CONVERGE® to unlock unique insights into the biological underpinnings of ALS and discover meaningful targets for complex diseases,” said Robert H. Scannevin, Ph.D., Chief Scientific Officer at Verge Genomics. “By combining robust computational predictions with compelling experimental data, we empowered Lilly to advance selected targets into further development.”

Verge also continues to advance VRG50635, its internal lead drug candidate for the treatment of sporadic and familial ALS. VRG50635 is currently being evaluated in a Phase 1B Proof-of-Concept (PoC) study in Canada and several European countries. Enrollment is complete and dose escalation, including to the highest dose level, is ongoing. Verge’s PoC study incorporates innovative technology that collects dense amounts of unbiased, objective, disease-relevant molecular and clinical data to properly assess safety, tolerability, pharmacological dose-response, and efficacy, including for disease modification. VRG50635 is a potent, orally bioavailable and brain-penetrant PIKfyve inhibitor shown to improve survival in ALS patient-derived motor neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration.

About Verge Genomics

Verge is focused on developing therapeutics for complex diseases with high unmet need, using human genomics from patient disease tissues and machine learning. Verge has created a proprietary, all-in-human CONVERGE® platform, featuring one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is led by experienced computational biologists and drug developers who are successfully advancing clinical and preclinical therapeutic programs in various diseases. For additional information, visit www.vergegenomics.com and please follow us on LinkedIn and X.

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