Pfizer's Duchenne gene therapy fails in late-stage study

Pfizer said on Wednesday (June 12) its experimental gene therapy for Duchenne muscular dystrophy (DMD) failed to improve the motion function of patients in a late-stage trial compared with a placebo.

The therapy also did not show a significant difference compared to placebo in secondary goals of the study such the time it takes for patients to rise from the floor or improve speed in a 10-metre run or walk.

DMD is a genetic muscle wasting disorder in which most patients lack the protein dystrophin, which keeps muscles intact. The disorder affects an estimated 1-in-3,500 male births worldwide.

Pfizer's one-time gene therapy aims to deliver a shortened version of the missing human dystrophin gene to DMD patient's cells.

The results of the trial in boys ages four to seven years marks another setback for Pfizer, which needs revenue from new medicines as billions of dollars from sales of Covid-19 products have evaporated due to sharply declining demand.

Pfizer said last month that a patient in an earlier stage trial of 2- to 3-year-old boys had died from a cardiac arrest after receiving the therapy. It said then it was reviewing data to understand the potential cause of death along with the independent external data monitoring committee.

The company said it will continue to closely monitor all participants enrolled in the late-stage study and is evaluating appropriate next steps for the programme.

The main goal of the trial looked for improvements in the speed and functionality of motor skills in DMD patients who can walk.

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