Verge Genomics and Alexion in AI-Enabled Drug Discovery Collaboration for Rare Neurodegenerative and Neuromuscular Diseases

Rare Daily Staff

Verge Genomics has entered into a multi-target collaboration with Alexion, AstraZeneca Rare Disease to identify novel drug targets for rare neurodegenerative and neuromuscular diseases.

Under the terms of the four-year agreement, Verge will receive up to $42 million, consisting of upfront fee, equity, and near-term payments, with a potential deal value of $840 million plus 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 Cheng, senior vice president, head of Research and Product Development, Alexion. “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 highly predictive human tissue datasets with machine learning to find new targets with a higher probability of clinical success. 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. This approach, validated through the successful advancement of Verge’s lead program in amyotrophic lateral sclerosis, will be used to identify novel therapeutic targets. Alexion will select high-potential targets for each indication, with the option to license and advance successful targets through clinical development and commercialization.

“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,” said Jane Rhodes, chief business officer, Verge Genomics.

Along with this collaboration, AstraZeneca will take an equity position in Verge Genomics.

Photo: Jane Rhodes, chief business officer, Verge Genomics