Pfizer’s DMD candidate misses Phase III goals

After two previous setbacks, US pharma giant Pfizer (NYSE: PFE) yesterday released disappointing new data for its Duchenne muscular dystrophy (DMD) gene therapy candidate.

Pfizer said that CIFFREO, a Phase III global, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with DMD did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age treated with the gene therapy compared to placebo.

The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment (NSAA) at one year after treatment. Key secondary endpoints, including 10-meter run/walk velocity and time to rise from floor velocity, also did not show a significant difference between participants treated with fordadistrogene movaparvovec and placebo.