Boston, MA 09/10/2014 (wallstreetpr) – PTC Therapeutics, Inc. (NASDAQ:PTCT) announced that the enrollment of ACT DMD completes as per the plans and in defined time limit. The enrollment is for the Phase 3 confirmatory trial in Duchenne Muscular Dystrophy. It is the trial of Translarna for the patients who are suffering from Duchenne muscular dystrophy. PTC Therapeutics, Inc. (NASDAQ:PTCT) is expected to release the data in the second half of 2015. The top-line data will act as a platform to get the additional approvals globally.
The trial
ACT DMD can be precisely defined as a clinical trial to find the impact of the on ambulation in patients suffering from nmDMD. It is 48-week trial for which the enrollment has completed across 54 sites globally. The patients who have already completed the initial 48 weeks of treatment can become a part of open-label extension study. The first endpoint in the trial is the change in walking distance that is measured by conducting a six-minute walk test.
Significant achievement
Dr. Haluk Topaloglu, working in Dept. of Neurology in Hacettepe Children’s Hospital considers the full enrollment in a clinical trial as a significant achievement towards developing effective disease-modifying treatments. The new treatments will work for the betterment of patients and will set advance standards of care in the field of DMD. He also praised the commitment of patients, their families, study investigators, PC therapeutics and advocacy groups in supporting the trial.
Management View
Stuart Peltz, Ph.D., the CEO of PTC Therapeutics, Inc. (NASDAQ:PTCT), said that the completion of enrollment is an important achievement in the ACT DMD study. The new trial design is optimized by the use of previous results of Translarna’s clinical activity and the natural history data from the past studies. The existing study comes in the list of largest DMD trials ever conducted. The management expects to get a successful outcome of the study.
