Pfizer's Late-Stage Gene Therapy Trial for Duchenne Muscular Dystrophy Fails to Meet Primary Endpoint

Gelonghui June 13 | Pfizer, Inc. stated that in a late-stage trial, its experimental gene therapy for Duchenne muscular dystrophy (DMD) failed to improve patients' motor function compared to placebo. DMD is a hereditary muscular atrophy condition where most patients lack the protein dystrophin, which is essential for maintaining muscle integrity. It is estimated that one in every 3,500 male infants worldwide is affected by this disease. Regarding secondary objectives of the study—such as reducing the time it takes for patients to rise from the floor or improving the speed of running or walking 10 meters—the therapy also showed no significant difference compared to placebo. The primary goal of the trial was to improve motor speed and function in ambulatory DMD patients. The trial results mark another setback for Pfizer, Inc., which needs revenue from new drugs as billions of dollars in sales from COVID-19 products have evaporated due to sharply declining demand. Pfizer will continue to closely monitor all participants in the late-stage study and is evaluating appropriate next steps for the program.