Lilly to develop ALS therapies against targets ID’d with Verge tech
Converge draws on human data, machine learning toward treatment targets
Eli Lilly will develop therapies against two drug targets for amyotrophic lateral sclerosis (ALS) that were identified and validated by Verge Genomics as part of a three-year collaboration between the companies.
The targets, which weren’t disclosed, were discovered with Converge, Verge’s platform that draws on human data and machine learning technology to discover and validate new treatment targets. The platform validated as many as 83% of its treatment targets in disease models, a success rate much higher than the usual industry rates, according to the company.
This rate of success suggests the platform can reliably predict which treatments are most likely to work in clinical settings.
“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,” Robert H. Scannevin, PhD, Verge’s chief scientific officer, said in a company press release.
Verge and Lilly partnered in 2021 to identify new targets for treating ALS over three years. Verge received up to $25 million up front and could realize up to $694 million plus royalties if the partnership leads to effective treatments.
“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,” said Michael Hutton, PhD, senior vice president and chief scientific officer of neurodegeneration and genetic medicine at Lilly.
More work by Verge
In ALS, damage to the nerve cells that control voluntary movement, called motor neurons, leads to muscle weakness and problems with walking, balance, breathing, swallowing, and speaking as the disease progresses and symptoms worsen.
Verge feeds data on human genetics and protein-protein interactions into machine learning models that can recognize patterns and predict possible treatment targets. It then combines disease models and data from patients to identify candidate compounds with a chance of success in clinical trials.
“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,” Hutton said.
Verge continues testing VRG50635, an experimental oral compound, in a fully enrolled proof-of-concept Phase 1b clinical trial (NCT06215755) the company launched this year. Its goal is to see how safe and well tolerated VRG50635 is in 50 adults with sporadic or familial ALS.
VRG50635, which can enter the brain, blocks a protein called PIKfyve, which should increase the amount of lysosomes, cellular organelles that can break down faulty or unwanted molecules, including the abnormal protein clumps that build up in ALS nerve cells. The therapy has been shown to prolong the survival of lab-grown motor neurons from patients. It was also deemed safe in healthy adults who participated in a Phase 1 clinical trial (ISRCTN14792372).
About the Author
