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 companion to take the remedy by regulatory evaluate 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 provide issue VIII, bringing that protein nearer to regular ranges. Beneath the collaboration settlement signed in 2017, Sangamo was liable for Part 1/2 improvement of the gene remedy. Pfizer’s accountability spanned late-stage improvement, regulatory submissions, and commercialization.
This previous summer time, 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 fulfill with regulators. In keeping with Sangamo, Pfizer had mentioned it anticipated U.S. and European regulatory submissions would occur in early 2025. As lately as final month, Pfizer indicated it was discussing the information with regulators.
Hemophilia gene therapies have made it by regulatory evaluate. Pfizer did it earlier this yr, profitable FDA approval for Beqvez, a hemophilia B gene remedy that was licensed from Spark Therapeutics. However commercializing expensive hemophilia gene therapies has confirmed to be tough. For sufferers who can handle hemophilia with infusions of clotting proteins or persistent dosing of sure medicine, one-time remedy from gene remedy has been a troublesome promote. Newer hemophilia medicine are getting into the market, giving sufferers much more selections. Pfizer has certainly one of them with Hympavzi, a once-weekly injectable drug permitted by the FDA in October for each hemophilia A and B.
The commercialization challenges dealing with hemophilia gene therapies are forcing firms to make exhausting selections. Lackluster gross sales of Roctavian, a BioMarin Pharmaceutical gene remedy for hemophilia A permitted final yr, 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 reviews, it had obtained $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 yr, 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 reviews, 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, in line with 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 information could be ample 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 may possibly chart a path ahead for its applications, however the firm acknowledged that extra funding is critical 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 disenchanted 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 appropriate companion with the main focus and understanding of the genomic drugs business atmosphere to carry this drugs to sufferers,” he mentioned.
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