AstraZeneca, through its rare diseases unit Alexion, has entered into a four-year multi-target collaboration with Verge Genomics to identify novel drug targets for rare neurodegenerative and neuromuscular diseases.

Under the terms of the agreement, Verge will receive up to $42m in upfront, equity, and near-term payments, in addition to $840m in potential milestone payments and royalties.

In exchange, Alexion will obtain access to the US-based biotechnology company’s CONVERGE platform to identify novel disease targets with high potential for clinical success.

In addition to the collaboration, Alexion will acquire an equity stake in Verge, based on its strategic investors, including Eli Lilly and Merck Global Health Innovation Fund.

Alexion senior vice president, research and product development head Seng H Cheng said: “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.

“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 diseases.”

CONVERGE is a full-stack platform that combines highly predictive human tissue datasets with machine learning and features comprehensive databases of multi-omic patient data.

It is powered by a library of genomic datasets derived directly from human tissue and an advanced human-centred biology platform to advance new data insights into clinical candidates.

Verge validated the drug discovery platform’s approach through a lead program in amyotrophic lateral sclerosis (ALS), used to identify novel therapeutic targets.

Alexion will select the targets for each indication, with an option to license and advance the successful targets through clinical development and commercialisation.

Verge Genomics chief business officer Jane Rhodes said: “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 commercialising rare disease treatments.

“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.”