Pfizer’s analysis partnership with Sangamo Therapeutics produced a hemophilia A gene remedy that reached FDA discussions a few regulatory submission. That’s so far as the alliance will go. Pfizer is terminating the seven-year-old pact, a transfer that comes earlier than the pharmaceutical large should pay pricey milestone funds for a product with unsure business prospects.
In response to Sangamo, Pfizer mentioned the termination displays its resolution to not go ahead with regulatory submissions for the hemophilia A gene remedy, giroctocogene fitelparvovec. The termination was introduced after Monday’s market shut. When the termination takes impact in April, Sangamo will regain all rights to the gene remedy. The Richmond, California-based biotech mentioned it nonetheless goals to advance this system and can discover all choices, together with searching for a brand new collaboration companion to take the remedy by way of regulatory assessment and commercialization.
Giroctocogene fitelparvovec is a functioning model of the gene that codes for issue VIII, the clotting protein that’s poor in hemophilia A sufferers. The one-time remedy is meant to allow sufferers to supply issue VIII, bringing that protein nearer to regular ranges. Beneath the collaboration settlement signed in 2017, Sangamo was answerable for Part 1/2 growth of the gene remedy. Pfizer’s accountability spanned late-stage growth, regulatory submissions, and commercialization.
This previous summer season, Pfizer reported preliminary Part 3 outcomes exhibiting the gene remedy led to statistically vital reductions in annualized bleeding charges by way of 15 months. The pharma large mentioned it deliberate to fulfill with regulators. In response to Sangamo, Pfizer had mentioned it anticipated U.S. and European regulatory submissions would occur in early 2025. As just lately as final month, Pfizer indicated it was discussing the information with regulators.
Hemophilia gene therapies have made it by way of regulatory assessment. Pfizer did it earlier this 12 months, profitable FDA approval for Beqvez, a hemophilia B gene remedy that was licensed from Spark Therapeutics. However commercializing dear hemophilia gene therapies has confirmed to be troublesome. For sufferers who can handle hemophilia with infusions of clotting proteins or power dosing of sure medication, one-time remedy from gene remedy has been a troublesome promote. Newer hemophilia medication are getting into the market, giving sufferers much more decisions. Pfizer has certainly one of them with Hympavzi, a once-weekly injectable drug accepted by the FDA in October for each hemophilia A and B.
The commercialization challenges going through hemophilia gene therapies are forcing corporations to make onerous decisions. Lackluster gross sales of Roctavian, a BioMarin Pharmaceutical gene remedy for hemophilia A accepted final 12 months, have led that firm to discover choices together with divestiture of the product. Now Pfizer has determined to not proceed with Sangamo’s hemophilia A gene remedy.
Beneath the gene remedy alliance, Sangamo acquired $70 million up entrance. In response to the biotech’s monetary studies, it had acquired $55 million in milestone funds to this point. As much as $220 million in extra milestone funds remained excellent. Sangamo was relying on the Pfizer funds for its survival.
Collaborations with Novartis and Biogen ended final 12 months, main Sangamo to implement a company restructuring and layoffs. Sangamo has since inked offers with Genentech and Astellas Pharma, however these agreements include small upfront funds and milestones that could be years away. In its monetary studies, Sangamo mentioned it has explored the potential of submitting for chapter safety. The corporate’s money place as of Sept. 30 was $39.2 million, based on its report for the third quarter of 2024. Sangamo mentioned it anticipated to have sufficient money to final solely into the primary quarter of 2025.
Sangamo wants money to help its pipeline of neurology genomic medicines, together with a gene remedy for Fabry illness. In October, the FDA confirmed to the corporate that Part 1/2 knowledge can be enough to help a regulatory submission beneath the accelerated approval pathway. The corporate deliberate a submission for the second half of 2025.
Within the announcement of the Pfizer termination, Sangamo mentioned it believes it may chart a path ahead for its packages, however the firm acknowledged that extra funding is important for advancing every of them, together with the hemophilia A gene remedy. In a ready assertion, Sangamo CEO Sandy Macrae mentioned the corporate was shocked and disillusioned by Pfizer’s resolution to finish the collaboration so near the anticipated regulatory submissions.
“We’re dedicated to exploring the optimum path ahead for this essential remedy, together with searching for the correct companion with the main target and understanding of the genomic medication business surroundings to deliver this medication to sufferers,” he mentioned.
Illustration: Kuzma, Getty Photos
