Pfizer’s analysis partnership with Sangamo Therapeutics produced a hemophilia A gene remedy that reached FDA discussions a couple of 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 big should pay pricey milestone funds for a product with unsure business prospects.
In keeping with Sangamo, Pfizer mentioned the termination displays its determination 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 associate to take the remedy by 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 chargeable for Part 1/2 improvement of the gene remedy. Pfizer’s duty spanned late-stage improvement, regulatory submissions, and commercialization.
This previous summer season, Pfizer reported preliminary Part 3 outcomes exhibiting the gene remedy led to statistically important reductions in annualized bleeding charges by 15 months. The pharma big mentioned it deliberate to satisfy with regulators. In keeping with 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 info with regulators.
Hemophilia gene therapies have made it by regulatory assessment. Pfizer did it earlier this 12 months, successful 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 persistent dosing of sure medication, one-time remedy from gene remedy has been a troublesome promote. Newer hemophilia medication are coming into the market, giving sufferers much more decisions. Pfizer has one in every of them with Hympavzi, a once-weekly injectable drug permitted by the FDA in October for each hemophilia A and B.
The commercialization challenges going through hemophilia gene therapies are forcing firms to make arduous decisions. Lackluster gross sales of Roctavian, a BioMarin Pharmaceutical gene remedy for hemophilia A permitted 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 obtained $70 million up entrance. In keeping with the biotech’s monetary studies, it had obtained $55 million in milestone funds up to now. As much as $220 million in further 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 opportunity of submitting for chapter safety. The corporate’s money place as of Sept. 30 was $39.2 million, in response to 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 assist 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 could be adequate to assist a regulatory submission underneath 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 will possibly chart a path ahead for its applications, however the firm acknowledged that further 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 determination 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 best associate with the main target and understanding of the genomic medication business setting to carry this medication to sufferers,” he mentioned.
Illustration: Kuzma, Getty Photos
