Pfizer has transferred an AAV capsid designed for a rare neurological disease target to AstraZeneca’s rare disease subsidiary, Alexion, as part of a larger, previously announced bundled deal.
This announcement, included in Voyager Therapeutics’ third-quarter earnings report, offers more details on a deal between Pfizer and Alexion that was initially disclosed in late July. At that time, AstraZeneca revealed that Alexion had acquired a portfolio of early-stage rare disease gene therapies for up to $1 billion, along with tiered royalties if any candidates reached the market. The specific assets included in the deal were not disclosed by Alexion at that time.
Voyager’s agreement with Pfizer dates back more than two years when the gene therapy biotech entered into a licensing agreement, receiving an upfront payment of $30 million for up to two capsids. Voyager also had the potential to earn $10 million for each option, with additional biobucks totaling more than $500 million. Pfizer paid one of the two option payments in October 2022, with $290 million in milestone payments specifically allocated for one asset, which is now being transferred to Alexion.
Voyager CEO Al Sandrock, M.D., Ph.D., expressed enthusiasm for the partnership, especially given Alexion’s commitment to advancing next-generation genomic medicines.
This deal with Pfizer-turned-Alexion is one of three collaborations that have provided validation for Voyager’s early pipeline. In March, Novartis opted for two gene therapies, resulting in a $25 million payment. A few months later, Sangamo Therapeutics partnered with Voyager to leverage its licensing technology in developing a candidate for prion disease, with plans to submit a new drug application in 2025.
While none of Voyager’s wholly owned programs have entered clinical trials so far, that may change soon. The company intends to seek regulatory approval for a Phase 1 study of an anti-tau Alzheimer’s disease treatment in the first half of 2024. Regarding gene therapy, Voyager plans to select a development candidate for its SOD1 amyotrophic lateral sclerosis program by the end of the year, with a new drug submission scheduled for mid-2025. This program targets the same subset of patients as Biogen and Ionis Pharmaceuticals’ Qalsody treatment, which received accelerated approval in April.