What’s Next for Sangamo After This Hemophilia Study

By Chris Lange Updated Jan 30, 10:35PM EST · Published Apr 2, 11:40AM EDT

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What’s Next for Sangamo After This Hemophilia Study — © PhonlamaiPhoto / Getty Images

Sangamo Therapeutics Inc. (NASDAQ: SGMO) shares jumped on Tuesday after the company announced interim data from its midstage hemophilia trial in collaboration with Pfizer Inc. (NYSE: PFE | PFE Price Prediction). The Phase 1/2 Alta study is evaluating investigational SB-525 gene therapy for severe hemophilia A.

Ultimately, the data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII levels across the four dosage cohorts. Eight patients total were dosed.

Patients in the Alta study were not treated with prophylactic steroids. No treatment-related serious adverse events were observed in the first three cohorts.

In addition to the partnership for the development and commercialization of gene therapies for hemophilia A, Sangamo and Pfizer are also collaborating on the development of gene therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration using Sangamo’s proprietary zinc finger protein transcription-factor technology.

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Edward Conner, M.D., chief medical officer of Sangamo, commented:

The interim data from the first eight patients with hemophilia A treated with SB-525 gene therapy in the Alta study are encouraging and demonstrate a dose-dependent relationship, evidence of sustained factor levels, and low variability, both within each patient and within each cohort. These interim results suggest that SB-525 may be well-tolerated and may prove to have the predictability and sustained treatment effect that can bring clinical benefit in patients with hemophilia A. We need to continue observing how the data mature and how additional patients in the expansion cohort respond to SB-525. We look forward to working with Pfizer to potentially advance SB-525 into a registrational study.

Shares of Sangamo were last seen up about 29% at $12.28, in a 52-week range of $6.26 to $20.55. The stock has a consensus price target of $18.71.

Pfizer traded at $42.97 a share. The 52-week range is $34.37 to $46.47, and the consensus price target is $44.14.
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About the Author Chris Lange →

Chris Lange is a writer for 24/7 Wall St., based in Houston. He has covered financial markets over the past decade with an emphasis on healthcare, tech, and IPOs. During this time, he has published thousands of articles with insightful analysis across these complex fields. Currently, Lange's focus is on military and geopolitical topics.

Lange's work has been quoted or mentioned in Forbes, The New York Times, Business Insider, USA Today, MSN, Yahoo, The Verge, Vice, The Intelligencer, Quartz, Nasdaq, The Motley Fool, Fox Business, International Business Times, The Street, Seeking Alpha, Barron’s, Benzinga, and many other major publications.

A graduate of Southwestern University in Georgetown, Texas, Lange majored in business with a particular focus on investments. He has previous experience in the banking industry and startups.