Business
Sangamo Therapeutics Reports on Pfizer’s Announcement of Positive Topline Results From Phase 3 Trial of Hemophilia A Gene Therapy Candidate
Giroctocogene fitelparvovec trial meets primary and key secondary objectives of superiority compared to prophylaxis RICHMOND, Calif.--(BUSINESS WIRE)--
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"\nGiroctocogene fitelparvovec trial meets primary and key secondary objectives of superiority compared to prophylaxis\n\n\n RICHMOND, Calif.--(BUSINESS WIRE)--\nSangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported on Pfizer Inc.’s announcement of positive topline results from the Phase 3 AFFINE trial (NCT04370054) 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.\n\n\nSangamo is eligible to earn from Pfizer up to $220 million 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.\n\n\nPfizer reported that the AFFINE trial achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040).\n\n\nKey secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis. 84% of participants maintained FVIII activity >5% at 15 months post-infusion (one-sided p-value = 0.0086) with the majority of participants having FVIII activity ≥15%, and the mean treated ABR showed a statistically significant 98.3% reduction from 4.08 in the pre-infusion period to 0.07 post-infusion (from Week 12 up to at least 15 months [15-44 months]; one-sided p-value \nIn the AFFINE trial, giroctocogene fitelparvovec was generally well tolerated. Transiently elevated FVIII levels ≥150% were observed in 49.3% of dosed participants, as measured via chromogenic assay, with no impact on efficacy and safety results. Serious adverse events were reported in 15 patients (20%), including 13 events reported by 10 patients (13.3%) assessed as related to treatment. Treatment-related adverse events generally resolved in response to clinical mana...