NEW YORK – Sangamo Therapeutics said on Monday that it has regained rights to its hemophilia A gene therapy giroctocogene fitelparvovec after its partner Pfizer decided to end a global collaboration and license agreement for the drug.
The companies originally entered into the agreement for giroctocogene fitelparvovec in 2017, giving Pfizer global rights to research, develop, manufacture, and commercialize the drug. Earlier this year, Pfizer said it planned to discuss with regulatory authorities the positive top-line results from a Phase III clinical trial of giroctocogene fitelparvovec in hemophilia A. The firm has since decided not to proceed with the biologics license application (BLA) and marketing authorization application (MAA) submissions for the drug.
Now, Sangamo said it is exploring options to continue advancing the program, including seeking a potential new collaboration partner. The agreement with Pfizer will terminate on April 21, 2025.
In results presented this year from the Phase III AFFINE study, patients with hemophilia A who received giroctocogene fitelparvovec experienced a significant reduction in average total annualized bleeding rate (ARB) compared to the lead-in period.
Giroctocogene fitelparvovec contains a bioengineered adeno-associated virus serotype 6 capsid that delivers a modified B-domain deleted FVIII gene, the gene that's mutated in hemophilia A. These mutations in the FVIII gene lead to a deficiency in the eponymous protein that the gene encodes, which is needed for blood clotting.
"Giroctocogene fitelparvovec has demonstrated the potential to be a life-changing gene therapy treatment for hemophilia A patients, and following positive results from the Phase III AFFINE trial, we believe it is well positioned for regulatory submissions and potential commercialization," Sangamo CEO Sandy Macrae said in a statement. “While we were surprised and extremely disappointed by Pfizer's decision to end our collaboration so close to the anticipated BLA and MAA submissions, especially given the compelling pivotal clinical trial data, we appreciate their collaboration in leading a robust and successful clinical development program and for advancing the asset to this important stage."