Illustration of a human head and neck showing a cut-through to the brain on the left hand side on a background picture of fluorescent neurons and with a DNA helix to the right hand side to represent genetic brain and neurological disorders such as the movement disorder ataxia.

Artificial intelligence (AI) pioneer Verge Genomics today announced a four-year, multi-target, collaboration with Alexion (AstraZeneca Rare Disease) to identify novel drug targets for rare neurodegenerative and neuromuscular diseases. Verge will receive up to $42 million up front, consisting of a fee, equity, and near-term payments, with $840 million of potential downstream royalties.

“By leveraging Verge’s AI-enabled platform in combination with data from patient tissue samples, we see potential in helping researchers more efficiently identify and validate therapeutic targets for rare diseases,” said Seng H. Cheng, senior vice president, head of research and product development, Alexion.

He added that, “This collaboration will contribute to Alexion’s science-led innovation and may help accelerate our efforts to transform the discovery and development of new medicines for patients with rare disease.”

The collaboration, which is Verge’s second with a leading global pharmaceutical company, will leverage CONVERGE, a full-stack platform that combines human tissue datasets with machine learning to find new targets with a higher probability of clinical success.

In July 2021 Verge announced a three-year collaboration with Eli Lilly and Company to research and develop novel therapies for the treatment of amyotrophic lateral sclerosis (ALS), a devastating motor neuron disease.

Instead of starting with cell or animal models, Verge’s drug discovery platform is based on a proprietary library of genomic datasets derived directly from human tissue that is coupled with an advanced human-centered biology platform to rapidly advance new data insights into clinical candidates. The company says it has validated this approach through the successful advancement of Verge’s lead program in amyotrophic lateral sclerosis (ALS). Verge has almost a dozen programs in the works. All are preclinical except for its lead ALS candidate,

Alexion will select high-potential targets for each indication, with the option to license and advance successful targets through clinical development and commercialization.

“Our collaboration with Alexion is a tremendous opportunity to leverage the combined power of CONVERGE’s technology-enabled approach to drug discovery with Alexion’s expertise in developing and commercializing rare disease treatments,” said Jane Rhodes, chief business officer, Verge Genomics. “We believe that our work with Alexion will build on our achievements in CNS drug discovery and is indicative of the sustained interest from pharma in the potential of AI to transform drug discovery and development.”

Alongside this collaboration agreement, AstraZeneca will take an equity position in Verge Genomics. Other Verge investors include Eli Lilly and Merck Global Health Innovation Fund.

Verge develops therapeutics using human genomics from patient disease tissues and machine learning. The company has created a proprietary all-in-human CONVERGE platform, which it says features one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is currently focused on ALS and Parkinson’s disease.

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