Verge Genomics, at the forefront of using artificial intelligence (AI) and human-centric data to revolutionize drug discovery and development, has shared news that Eli Lilly & Company (Lilly) has chosen to move forward with developing treatments targeting two validated drug targets for amyotrophic lateral sclerosis (ALS) as part of a partnership dating back to July 2021. These targets were identified and validated through Verge’s AI-driven CONVERGE platform, which boasts an impressive validation rate of 83% for prioritized targets in disease-relevant models, surpassing conventional industry standards and attesting to CONVERGE’s powerful predictive capabilities.
The selection of these targets has triggered significant milestone payments from Lilly to Verge, with the latter holding the potential for further economic benefits as the projects advance.
“Thanks to the rigorous work of the Verge team and the effective validation methods within human cell systems available through the CONVERGE platform, we have numerous promising therapeutic targets that are scientifically sound,” said Michael Hutton, Ph.D., Senior VP and CSO for Neurodegeneration and Genetic Medicine at Lilly. “We’re eager to develop on their foundational work with the objective of forwarding ALS drug candidates to address the pressing unmet needs in this critical illness.”
The partnership, which began in 2021, set a course for a 3-year collaboration between Verge and Lilly to identify and validate new therapeutic targets for ALS, which would then form the core of ensuing drug discovery and development endeavors. Verge benefited from up to $25 million in initial funding, equity investment, and potential immediate milestones, with an overall deal valuation of $694 million, plus potential future royalties.
“This milestone further corroborates the potent predictive strength of CONVERGE, unlocking new understandings of ALS biology and providing meaningful targets for complex diseases,” commented Robert H. Scannevin, Ph.D., Chief Scientific Officer at Verge Genomics. “By merging strong computational forecasts with impactful experimental data, we empowered Lilly to progress chosen targets into subsequent development stages.”
Simultaneously, Verge continues to advance their leading candidate, VRG50635, targeting both sporadic and familial ALS. Currently, VRG50635 is undergoing a Phase 1B Proof-of-Concept (PoC) study in Canada and several European nations. With enrollment now complete, the study is ongoing with dose escalation reaching peak levels. Verge’s PoC study incorporates cutting-edge technology to gather extensive, unbiased molecular and clinical data relevant to the disease in order to properly evaluate safety, tolerability, dose-response, and efficacy, including potential for disease alteration. VRG50635 is an effective, orally-administered and brain-penetrant PIKfyve inhibitor, demonstrating enhanced survival in ALS patient-derived motor neurons and has shown promise in multiple preclinical studies relevant to ALS models of motor neuron degeneration.
Verge continues to prioritize the creation of therapies for intricate diseases with significant unmet needs, relying on human genomic data from patient tissues in combination with machine learning.