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Pfizer Begins Clinical Trial With Mini-dystrophin Gene Therapy Candidate

Pfizer Inc. (PFE) announced it has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy. Screening and enrollment of patients is expected to continue at up to four clinical research sites in the United States. Early data from the trial are expected in the first half of 2019, once all patients have been evaluated for one full year post-treatment.

The multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 will enroll approximately 12 ambulatory boys aged 5 to 12 years with Duchenne muscular dystrophy. In addition to evaluating safety and tolerability, the study will evaluate measurements of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.

The clinical trial is Pfizer's first recombinant AAV-based gene therapy program to enter the clinic stemming from Pfizer's 2016 acquisition of Bamboo Therapeutics. PF-06939926 was granted Orphan Drug and Pediatric Rare Disease Designations by the FDA and Orphan Medical Product Designation by the European Medicines Agency in May 2017.

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