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Sangamo Therapeutics Inc (NASDAQ:SGMO), which developed a treatment in collaboration with Pfizer Inc (NYSE:PFE), received a boost to its stock price after struggling with liquidity issues.
At last check Wednesday, Sangamo was up more than 36.2%, trading at 55 cents per share.
Both companies released results from the Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for adult patients with moderately severe to severe hemophilia A.
The AFFINE study achieved its primary objective.
Following a single 3e13 vg/kg dose, the gene therapy demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73).
Key secondary endpoints were met and demonstrated superiority compared to prophylaxis.
About 84% of participants maintained FVIII activity greater than 5% at 15 months post-infusion, with the majority of participants having FVIII activity greater than or equal to 15%.
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]).
Among all dosed participants, one participant (1.3%) returned to prophylaxis post-infusion.
In the AFFINE study, giroctocogene fitelparvovec was generally well tolerated.
Serious adverse events were reported in 15 patients (20%), including 13 events reported by 10 (13.3%) assessed as related to treatment. Treatment-related adverse events were generally resolved in response to clinical management.
Clinical study participants will be evaluated in AFFINE over five years and up to 15 years as part of a long-term follow-up study.
In April, the FDA approved Pfizer’s Beqvez (fidanacogene elaparvovec) for moderate to severe hemophilia B.
Beqvez is awaiting a decision from the European Medicines Agency following a positive opinion from the EMA’s Committee for Medicinal Products for Human Use in May 2024.
The FDA put the study of giroctocogene fitelparvovec on hold in November 2021. Pfizer hit pause after some patients experienced blood clotting protein factor VIII activity greater than 150%.
Price Action: SGMO stock is up 64.5% at 66 cents, and PFE stock is down 0.10% at $29.46 at last check Wednesday.
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