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 large should pay pricey milestone funds for a product with unsure industrial prospects.
In response to 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 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 supply issue VIII, bringing that protein nearer to regular ranges. Below the collaboration settlement signed in 2017, Sangamo was liable for Section 1/2 growth of the gene remedy. Pfizer’s accountability spanned late-stage growth, 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 by 15 months. The pharma large stated it deliberate to fulfill with regulators. In response to Sangamo, Pfizer had stated 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 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 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 medicine, one-time remedy from gene remedy has been a troublesome promote. Newer hemophilia medicine are coming into the market, giving sufferers much more selections. Pfizer has one in every of them with Hympavzi, a once-weekly injectable drug authorized by the FDA in October for each hemophilia A and B.
The commercialization challenges going through hemophilia gene therapies are forcing corporations to make exhausting selections. Lackluster gross sales of Roctavian, a BioMarin Pharmaceutical gene remedy for hemophilia A authorized 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.
Below the gene remedy alliance, Sangamo obtained $70 million up entrance. In response to the biotech’s monetary stories, it had obtained $55 million in milestone funds to this point. 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 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 which may be years away. In its monetary stories, 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 enough 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 will possibly 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 stunned and disenchanted 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 proper companion with the main focus and understanding of the genomic medication industrial surroundings to deliver this medication to sufferers,” he stated.
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