Sangamo Phase 3 hemophilia A gene therapy meets primary endpoint with superiority over prophylaxis

SANGAMO THERAPEUTICS, INC

AFFINE trial achieved superiority on total ABR (1.24 vs 4.73, p=0.0040) compared to FVIII prophylaxis.
84% of participants maintained FVIII activity >5% at 15 months; mean treated ABR reduced 98.3% (0.07 vs 4.08, p<0.0001).
Sangamo eligible for up to $220M in regulatory/commercial milestones plus 14%-20% royalties on sales.
One participant (1.3%) returned to prophylaxis; treatment generally well tolerated with transient elevated FVIII in 49.3%.
Pfizer to discuss data with regulatory authorities; Sangamo's first potential commercial medicine.

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