Pfizer's ($PFE) Gene Therapy for Duchenne Muscular Dystrophy Fails Phase 3 Trial on Wednesday

Jun 12, 2024, 08:37 PM

Pfizer ($PFE) has announced on Wednesday that its Phase 3 study of an investigational gene therapy for ambulatory boys with Duchenne Muscular Dystrophy did not meet its primary endpoint. The study, which focused on boys aged 4-7, showed no significant improvement in motor function compared to a placebo. Additionally, key secondary endpoints also did not show significant differences. This marks the second failure of a gene therapy for Duchenne Muscular Dystrophy in recent trials, raising concerns about the efficacy of such treatments.