Sangamo Soars On Pfizer's Hemophilia A Gene Therapy Trial Results

(RTTNews) - Shares of Sangamo Therapeutics Inc. (SGMO), a genomic medicine company, were up over 70% in early trading today, following positive topline results from AFFINE trial.

AFFINE is a single-arm, multicentre phase III trial evaluating the efficacy and safety of a single infusion of Giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male participants with moderately severe to severe hemophilia A.

Hemophilia A is a rare bleeding disorder caused by mutations in the F8 gene, leading to insufficient clotting factor VIII (FVIII) in the bloodstream. FVIII is crucial for normal blood clotting, and the severity of hemophilia is determined by its deficiency. Lower levels of FVIII increase the likelihood of bleeding, potentially resulting in significant health complications.

Giroctocogene fitelparvovec is intended to deliver a working copy of the F8 gene to the liver so that liver cells can start producing functional FVIII clotting factor.

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. Key secondary endpoints were also met in the study.

Giroctocogene fitelparvovec is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo and Pfizer.

The full phase III dataset from the AFFINE trial is being analyzed by Pfizer and additional data will be presented at upcoming medical meetings.

Sangamo is entitled 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 the gene therapy is approved and commercialized, subject to certain reductions.

SGMO has traded in a range of $0.29 to $1.48 in the last 1 year. As of this writing, the stock is at $0.60, up 50%.