Pfizer's Duchenne Muscular Dystrophy Treatment Misses Endpoints

By Ben Glickman

Pfizer's gene therapy being developed to treat Duchenne muscular dystrophy missed its primary endpoint in a late-stage trial.

The pharmaceutical company said Wednesday the mini-dystrophin gene therapy didn't meet its primary endpoint of improvement in motor function among boys four to seven years old.

Pfizer said that secondary endpoints, such as 10-meter walk or run velocity and time to rise from the floor velocity, also didn't show a significant difference between those treated with the gene therapy and the placebo in the Phase 3 trial.

Pfizer said that it would continue to closely monitor all participants in the study and was evaluating next steps for the program.

Duchenne is a genetic disease which leads to progressive muscle degeneration and weakness. It primarily affects boys and typically appears in early childhood.

Write to Ben Glickman at ben.glickman@wsj.com

(END) Dow Jones Newswires

June 12, 2024 16:56 ET (20:56 GMT)

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