Rare Daily Staff

Verge Genomics said it has entered a three-year collaboration with Eli Lilly to use its genetic disease focused all-in-human, artificial-intelligence-powered drug discovery and development platform to research and develop novel therapies for the treatment of amyotrophic lateral sclerosis, a devastating progressive, and fatal motor neuron disease.

The average life expectancy of a person with amyotrophic lateral sclerosis (ALS) is approximately two to five years, and there is currently no cure for the disease. One of the biggest challenges in the treatment of ALS is the underlying complex biology and lack of predictive animal models. Recent advances in genetic sequencing and human tissue banking offer a new opportunity to develop breakthrough therapies. Artificial intelligence allows the processing and integration of multiple types of human data generated by these technologies and is uniquely poised to address diseases with complex biology, such as ALS.

“This approach complements and enhances Lilly’s neuroscience portfolio and will help facilitate development of what we hope will be transformative new therapies for people with ALS,” said Michael Hutton, vice president, Neurodegeneration Research at Lilly.

Under the terms of their agreement, Verge will receive up to $25 million in upfront, equity investment, and potential near-term payments, with an additional $694 million in potential milestones and downstream royalties.

Verge will apply its all-in-human platform to discover and validate new targets for ALS. The all-in-human platform is based on a proprietary collection of patient brain transcriptomes across a variety of neurodegenerative diseases. Through its application, the all-in-human platform provides insights into novel causal disease mechanisms in genetically segmented patient populations and enables the discovery of therapeutic targets. Based on these insights, Verge will apply its human-based discovery capabilities to validate targets. Lilly will select up to four targets identified by Verge with plans to advance through clinical development and commercialization.

“Through this partnership with Lilly, we will examine the use of human data and machine learning to potentially overcome translational hurdles in historically challenging diseases with complex biology,” commented Alice Zhang, CEO and co-founder, Verge Genomics. “This collaboration also builds on the significant momentum for Verge in 2021, as we advance our wholly-owned lead PIKFyve programs for ALS and COVID-19 and continue to expand our discovery and pipeline development efforts in disease areas with significant unmet need.”

Photo: Photo: Alice Zhang, CEO and co-founder, Verge Genomics

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