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.
Based on Sangamo, Pfizer stated 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 stated it nonetheless goals to advance this system and can discover all choices, together with in search of a brand new collaboration associate to take the remedy via 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 therapy is meant to allow sufferers to provide issue VIII, bringing that protein nearer to regular ranges. Below the collaboration settlement signed in 2017, Sangamo was accountable for Section 1/2 improvement of the gene remedy. Pfizer’s duty spanned late-stage improvement, regulatory submissions, and commercialization.
This previous summer time, Pfizer reported preliminary Section 3 outcomes displaying the gene remedy led to statistically vital reductions in annualized bleeding charges via 15 months. The pharma big stated it deliberate to satisfy with regulators. Based on Sangamo, Pfizer had stated it anticipated U.S. and European regulatory submissions would occur in early 2025. As not too long ago as final month, Pfizer indicated it was discussing the info with regulators.
Hemophilia gene therapies have made it via regulatory evaluate. 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 expensive hemophilia gene therapies has confirmed to be troublesome. For sufferers who can handle hemophilia with infusions of clotting proteins or continual dosing of sure medication, one-time therapy from gene remedy has been a troublesome promote. Newer hemophilia medication are coming into the market, giving sufferers much more selections. Pfizer has considered 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 dealing with hemophilia gene therapies are forcing corporations to make onerous selections. 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.
Below the gene remedy alliance, Sangamo acquired $70 million up entrance. Based on the biotech’s monetary experiences, it had acquired $55 million in milestone funds thus far. 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 experiences, Sangamo stated it has explored the opportunity of submitting for chapter safety. The corporate’s money place as of Sept. 30 was $39.2 million, in accordance with its report for the third quarter of 2024. Sangamo stated 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 Section 1/2 information could be ample to help 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 stated it believes it could actually chart a path ahead for its packages, however the firm acknowledged that further funding is critical for advancing every of them, together with the hemophilia A gene remedy. In a ready assertion, Sangamo CEO Sandy Macrae stated the corporate was shocked and dissatisfied 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 therapy, together with in search of the appropriate associate with the main focus and understanding of the genomic medication business atmosphere to carry this medication to sufferers,” he stated.
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