Pfizer has handed off an AAV capsid aimed at a rare neurological disease target to AstraZeneca’s rare disease arm Alexion as part of a larger, previously announced bundle deal.
Monday’s announcement from Voyager Therapeutics, tucked into the company’s third-quarter earnings report, provides a few more specifics into a deal between Pfizer and Alexion first disclosed in late July. AstraZeneca announced at the time that Alexion had bought a portfolio of early rare disease gene therapies for up to $1 billion plus tiered royalties should any candidates make it to market. Alexion did not specify at the time which assets were included.
Voyager’s deal with Pfizer dates back more than two years, when the gene therapy biotech accepted $30 million in upfront cash in a licensing deal for up to two capsids. Voyager also stood to gain $10 million per option with more than $500 million in biobucks also up for grabs. Pfizer paid out one of those two option payments in October 2022 and outlined $290 million in milestone payments available specifically for the one asset. That is the gene therapy now headed over to Alexion.
“We are thrilled to have them as a partner on this program, particularly given their public commitment to advancing next-generation genomic medicines,” Voyager CEO Al Sandrock, M.D., Ph.D., said in a statement to Fierce Biotech.
The deal with Pfizer-turned-Alexion was one of four partnerships that have added validation to Voyager’s early pipeline. Two other gene therapies were opted into by Novartis in March, triggering a $25 million payment. A few months later, Sangamo Therapeutics signed on to use Voyager’s licensing tech to develop a candidate for prion disease, with plans to submit a new drug application in 2025. Voyager also has an extensive collaboration with Neurocrine that includes two preclinical assets in the late research stage aimed at Parkinson's disease and Friedrich's ataxia, respectively.
So far, none of Voyager’s wholly owned programs have entered the clinic, though that may soon change. The company expects to ask U.S. regulators to greenlight a phase 1 study for an anti-tau Alzheimer’s disease treatment in the first half of 2024. As for gene therapy, Voyager expects to select a development candidate for its SOD1 amyotrophic lateral sclerosis program by the end of the year with a new drug submission planned for the middle of 2025. That's the same subset of patients that Biogen and Ionis Pharmaceuticals targeted with their treatment Qalsody, which nabbed accelerated approval in April.
Editor's note: This story was updated to include details on Voyager's ongoing collaboration with Neurocrine.