The businesses said on Friday that AstraZeneca and Verge Genomics have signed a multi-target agreement to find and develop therapies against novel targets in rare neurodegenerative and neuromuscular illnesses. The deal will be carried out through Alexion, a division of AstraZeneca that specializes in rare diseases.
By the contract, Alexion will pay upfront fees of $42 million as well as stock and short-term investments. When milestones are taken into account, the four-year partnership may bring Verge up to $840 million in revenue in addition to future royalties. In addition to the drug discovery agreement, AstraZeneca will purchase stock in Verge.
In exchange, Alexion will get access to the California biotech company’s fully integrated, patented CONVERGE platform, which uses data from human tissue samples to find novel disease targets with a high likelihood of achieving clinical success.
The agreement announced on Friday would use the AI-driven methodology to treat several uncommon neurodegenerative and neuromuscular diseases. According to the release, Alexion will have the choice of choosing “high-potential targets” for every one of these indications, as well as licensing and advancing successful candidates through clinical development.
The commercialization of the joint venture’s products will also fall within Alexion’s purview.
Seng Cheng, head of research and product development at Alexion, stated in a statement: “We see promise in helping researchers more effectively find and evaluate therapeutic targets for rare diseases by employing Verge’s AI-enabled platform in combination with data from patient tissue samples.
Contrary to conventional drug development methods, which frequently start with animal or cell models, CONVERGE begins with genetic information directly derived from human tissue. A human-centered biology platform that produces fresh perceptions of candidates supports this.
In June 2023, Phase I data from Verge’s investigational PIKfyve inhibitor VRG50635 showed that it had good safety, tolerability, and pharmacokinetic profiles in healthy volunteers, thereby validating this strategy. In the fourth quarter, the company intends to start a proof-of-concept trial for the candidate in amyotrophic lateral sclerosis.
CONVERGE was used to find VRG50635 and it offers best-in-class potential.
The transaction on Friday is AstraZeneca’s second recent agreement related to a rare condition. According to Alexion CEO Marc Dunoyer, during AstraZeneca’s second-quarter results call, the business paid fellow giant Pfizer $1 billion in late July to purchase “a dozen” of the latter’s portfolio of preclinical gene medicines and technology.
Along with some Pfizer employees, the transaction brings several innovative AAV capsids into the AstraZeneca fold.
The Verge cooperation will also assist AstraZeneca in expanding its neurology portfolio, which currently includes the promising early-stage MEDI1814 for Alzheimer’s disease and the mid-stage MEDI1341 for Parkinson’s disease and multiple system atrophy. With the late-stage compounds ceramides for transthyretin amyloidosis cardiomyopathy and danicopanfor paroxysmal nocturnal hemoglobinuria, its pipeline for uncommon diseases is more robust in comparison.
