AstraZeneca’s Alexion division and Verge Genomics Embark on a $840 Million Odyssey to Unearth Rare Disease Miracles with AI
In an extraordinary leap forward, AstraZeneca’s rare disease branch, Alexion, has joined forces with Verge Genomics in a multi-target, high-stakes quest to discover groundbreaking treatments for rare neurodegenerative and neuromuscular diseases. The announcement of this groundbreaking partnership promises to reshape the landscape of drug discovery.
AstraZeneca, through Alexion, is set to invest an initial $42 million, combined with equity and near-term investments, laying the foundation for a four-year collaboration that could unlock up to a staggering $840 million for Verge, not to mention potential future royalties. As a testament to their commitment, AstraZeneca is also becoming a stakeholder in Verge, solidifying their belief in the project’s potential.
At the heart of this partnership is access to Verge’s cutting-edge CONVERGE platform, a revolutionary system that harnesses the power of AI and human tissue data. Unlike traditional drug discovery methods, CONVERGE begins its journey with genomic data sourced directly from human tissue, offering a uniquely human-centered approach. This innovative platform has already shown its promise with the Phase I data showcasing VRG50635, an investigational PIKfyve inhibitor discovered using CONVERGE. With outstanding safety, tolerability, and pharmacokinetic profiles in healthy volunteers, VRG50635 holds the promise of being best-in-class.
But the excitement doesn’t stop there. This collaboration will apply the AI-driven approach to a range of rare neurodegenerative and neuromuscular conditions, the specifics of which remain a tantalizing mystery. Alexion will have the privilege of selecting “high-potential targets” for these enigmatic conditions, guiding them through clinical development and eventual commercialization.
Seng Cheng, Alexion’s head of research and product development, encapsulates the spirit of this endeavor: “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.”
With this partnership, AstraZeneca is poised to build an even more formidable neurology pipeline, adding to its promising arsenal of treatments for Alzheimer’s disease, multiple system atrophy, and Parkinson’s disease. And let’s not forget their robust rare disease pipeline, featuring late-stage contenders like acoramidis for transthyretin amyloidosis cardiomyopathy and danicopan for paroxysmal nocturnal hemoglobinuria.
This extraordinary collaboration follows AstraZeneca’s recent $1 billion acquisition of preclinical gene therapies and technologies from Pfizer, demonstrating their unwavering dedication to pushing the boundaries of medical innovation. In this new era of drug discovery, the sky’s the limit, and AstraZeneca is leading the way in the search for rare disease miracles.