Pfizer (PFE) Reports New Phase 1b Results of Gene Therapy in Ambulatory Boys with DMD Support Advancement into Pivotal Phase 3 Study

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Go back to Pfizer (PFE) Reports New Phase 1b Results of Gene Therapy in Ambulatory Boys with DMD Support Advancement into Pivotal Phase 3 Study

Pfizer’s New Phase 1b Results of Gene Therapy in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD) Support Advancement into Pivotal Phase 3 Study

May 15, 2020 8:00 AM EDT

NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced updated Phase 1b clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy (DMD). The preliminary data from 9 ambulatory boys with DMD, aged 6 to 12 (mean age: 8 years) indicate that the intravenous administration of PF-06939926 was well-tolerated during the infusion period, with encouraging efficacy and manageable safety events, even when considering those adverse events that were more severe in nature. The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of... More