By Tristan Manalac

AstraZeneca, through its rare disease division Alexion, has entered into a multi-target agreement with Verge Genomics to discover and develop drugs against novel targets in rare neurodegenerative and neuromuscular diseases, the companies announced Friday.
Under the agreement, Alexion will make a $42 million payment in upfront fees, as well as equity and near-term investments. Counting milestones, the four-year collaboration can yield up to $840 million for Verge, plus potential royalties down the line. Alongside the drug discovery pact, AstraZeneca will also take an equity position in Verge.
In return, Alexion will gain access to the California biotech’s proprietary full-stack CONVERGE platform, which applies machine learning to human tissue data in order to identify novel disease targets that have a high probability of yielding clinical success.
Friday’s partnership will apply the AI-driven approach to several yet-undisclosed rare neurodegenerative and neuromuscular conditions. Alexion will have the option to select “high-potential targets” for each of these indications, as well as license and advance successful targets through clinical development, according to the announcement.
Alexion will also take charge of commercialization of products that result from the collaboration.
“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,” Seng Cheng, Alexion’s head of research and product development, said in a statement.
Unlike the typical drug discovery process, which usually begins with animal or cell models, CONVERGE starts off with genomic data obtained directly from human tissue. This is bolstered by a human-centered biology platform, yielding new insights into candidates.
Verge validated this approach with Phase I data in June 2023 demonstrating that its investigational PIKfyve inhibitor VRG50635 had promising safety, tolerability and pharmacokinetic profiles in healthy volunteers. The company is planning to launch a proof-of-concept study of the candidate in amyotrophic lateral sclerosis in the fourth quarter.
VRG50635 was discovered using CONVERGE and has best-in-class potential.
Friday’s agreement is AstraZeneca’s second rare disease deal recently. In late July, the company paid $1 billion to fellow powerhouse Pfizer to acquire “a dozen” of the latter’s portfolio of preclinical gene therapies and technologies, Alexion CEO Marc Dunoyer said during AstraZeneca’s second-quarter earnings call.
The deal brings several novel AAV capsids into the AstraZeneca fold, along with some Pfizer employees.
The Verge partnership will also help AstraZeneca build a more robust neurology pipeline, which already includes the early-stage Alzheimer’s disease hopeful MEDI1814 and the mid-stage MEDI1341 for multiple system atrophy and Parkinson’s disease. Its rare disease pipeline is more robust in comparison, with the late-stage molecules acoramidis for transthyretin amyloidosis cardiomyopathy and danicopanfor paroxysmal nocturnal hemoglobinuria.