Eli Lilly, Verge Partner to Identify and Test Potential ALS Therapies

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by Forest Ray PhD |

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Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS).

Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of bringing them into commercial use.

“Lilly’s focus and leadership in neurology matches well with Verge’s ability to identify high-potential targets for devastating neurological diseases,” Alice Zhang, PhD, Verge’s CEO and co-founder, said in a press release. “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.”

Verge reports that it has established a large collection of gene expression data related to ALS and other neurodegenerative disorders, including Parkinson’s disease and frontotemporal dementia. The company uses a proprietary computational platform based on machine learning, an artificial intelligence technique, to mine these data for potential treatment targets.

Essentially, the company’s algorithm estimates the ways in which genetic variants can lead to disease. These variants are then tested to validate their predicted effects.

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“Verge Genomics is advancing an innovative approach to identifying high-potential drug targets that are validated through artificial intelligence algorithms and a large library of human data,” said Michael Hutton, PhD, vice president of neurodegeneration research at Lilly.

“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,” Hutton added.

Under the agreement’s terms, Lilly will pay Verge up to $25 million in upfront, equity investment, and potential near-term payments. Additional milestone payments could amount to a further $694 million. The company is also eligible for royalties on commercial products stemming from this work.

“This collaboration also builds on the significant momentum for Verge in 2021,” said Zhang, “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.”

Some studies have shown that lowering levels of the PIKFyve enzyme increases the number of functional lysosomes, compartments in which cellular waste is broken down inside the cell. Problems with lysosome functioning are known in ALS and several other neurodegenerative disorders.

Current ALS treatments aim to slow the disease’s progression and/or ease its symptoms, but none are able to stop disability accumulation.