Sangamo reports on Pfizer’s Phase 3 results of Hemophilia A candidate

Sangamo Therapeutics (SGMO) reported on Pfizer’s (PFE) announcement of positive topline results from the Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec, an investigational gene therapy that Sangamo is co-developing with and licensing to Pfizer for the treatment of adults with moderately severe to severe hemophilia A. Sangamo is eligible to earn from Pfizer up to $220M in milestone payments upon the achievement of certain regulatory and commercial milestones for giroctocogene fitelparvovec and product sales royalties of 14% – 20% if giroctocogene fitelparvovec is approved and commercialized, subject to certain reductions. Pfizer reported that the AFFINE trial achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII replacement prophylaxis treatment. Giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period. Key secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis. The goal of this investigational treatment for people living with hemophilia A is that a single infusion of giroctocogene fitelparvovec may allow them to produce FVIII themselves for an extended period of time, provide bleed protection and reduce the need for routine prophylaxis with intravenous (IV) infusions or injections.