US-based biopharmaceutical firm PTC Therapeutics has completed patient enrolment in ACT DMD, the Phase III confirmatory trial of Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD).
The company intends to report top-line data from the 48-week Phase III trial in the second half of 2015.
The trial has reached full enrolment across 54 sites globally and is designed to confirm the effect of the investigational new drug Translarna on ambulation in patients with nmDMD.
The primary endpoint of the trial is the change in walking distance as measured by the six-minute walk test.
Hacettepe Children’s Hospital Department of Neurology Dr Haluk Topaloglu said: "The enrolment of this trial represents a significant achievement in our efforts to develop disease-modifying treatments that advance the standard of care in DMD and improve the quality of life for patients.
"The enormous commitment on the part of patients and their families, advocacy groups, study investigators and PTC Therapeutics should be commended."
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The company said that all patients in the Phase III trial are eligible to participate in an open-label extension study, which has already started enrolling patients who have completed the initial 48 weeks of treatment.
PTC Therapeutics chief executive officer Stuart Peltz said: "The completion of enrolment marks an important milestone in the ACT DMD study.
"We utilised the previous results demonstrating Translarna’s clinical activity, as well as the natural history data from ours and other studies to optimise the ACT DMD trial design.
"This study is one of the largest DMD trials ever performed and we believe it is well powered for a successful outcome. We are pleased to reach this important landmark as it brings us one step closer to broadening Translarna’s availability to all patients who may benefit."
Discovered and developed by PTC, Translarna is a protein restoration therapy designed to allow the formation of functioning protein in patients with genetic disorders caused by a nonsense mutation.