Pfizer's Gene Therapy for Duchenne Muscular Dystrophy Fails in Phase 3 Trial on June 12, Showing No Improvement

Jun 12, 2024, 09:15 PM

Pfizer's phase 3 study of its investigational gene therapy for Duchenne muscular dystrophy (DMD) failed to meet its primary endpoint, showing no significant improvement in motor function among ambulatory boys aged 4-7 compared to placebo. The trial also did not show significant differences in key secondary endpoints. This marks the second large, randomized, placebo-controlled trial of a Duchenne gene therapy to fail in achieving its primary goal. On June 12, Pfizer confirmed the failure of the trial, which targeted a muscle-wasting disorder.