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Pfizer's Duchenne gene therapy fails in late-stage study
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Pfizer's Duchenne gene therapy fails in late-stage study
Jun 12, 2024 3:42 PM

June 12 (Reuters) - Pfizer ( PFE ) said on Wednesday 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-meter 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 ( PFE ) 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 4 to 7 years marks

another setback for Pfizer ( PFE ), which needs revenue from new

medicines as billions of dollars from sales of COVID-19 products

have evaporated due to sharply declining demand.

Pfizer ( PFE ) 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 program.

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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