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.