Will Sangamo and Pfizer’s Hemophilia Gene Therapy Keep Building Momentum?

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Sangamo Therapeutics, Inc. (NASDAQ: SGMO) made a handy gain to start out the week after the firm, in conjunction with Pfizer Inc. (NYSE: PFE), announced updated results from a mid-stage trial evaluating a treatment for hemophilia.

Specifically, data came from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The data demonstrated that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels.

The first two patients treated at the 3e13 vg/kg dose rapidly achieved normal levels of FVIII activity, with no reported bleeding events, and the response continues to be durable for as long as 24 weeks—the extent of follow-up. The two patients more recently treated are demonstrating FVIII activity kinetics that appear consistent with the first two patients treated in this dose cohort at similar early time points.

One treatment-related serious adverse event (SAE) was reported. This patient experienced hypotension and fever six hours after completion of SB-525 infusion; this fully resolved with treatment and the patient was discharged as planned within 24 hours.

Barbara Konkle, M.D., Bloodworks Northwest, Professor of Medicine at University of Washington and a Principal Investigator of the Alta study, commented:

The initial results with SB-525 gene therapy for patients with severe hemophilia A continue to look very promising. It is encouraging that patients in the 3e13 vg/kg cohort have attained normal Factor VIII levels within 5-7 weeks of treatment with SB-525 gene therapy and have sustained Factor VIII activity with no bleeding episodes. It will be important to continue to follow these patients to understand the potential long-term durability of this gene therapy.

Shares of Sangamo were last seen up about 4% at $11.83, with a 52-week range of $6.26 to $19.25. The consensus analyst price target is $23.50.

Shares of Pfizer were recently seen down 1.6% at $43.24, with a 52-week range of $37.01 to $46.47. The consensus analyst price target is $45.92.

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