PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation

- Analysis of data to date shows Translarna Preserved Ambulation for up to 5 Years Compared to Natural History -

SOUTH PLAINFIELD, New Jersey, Oct. 6, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna (ataluren), underscoring the long-term clinical benefit of Translarna when used in routine clinical practice in delaying irreversible muscle loss in children with Duchenne caused by a nonsense mutation, when compared with published natural history. The data show that children and adolescents receiving Translarna in the real-world setting are continuing to walk years longer than untreated children, and are staying more physically able. A time-to-event analysis for loss of ambulation has shown that patients on Translarna had a median age of loss of ambulation of 16.5 years of age – up to 5 years later than seen with natural disease progression in untreated children. The data were presented as a late breaker at the 23rd International Annual Congress of the World Muscle Society in Argentina.

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"The results from the Registry data are exceptionally important because they further substantiate the results observed in our analyses of data from previous clinical trials studying Translarna in Duchenne," said Stuart W. Peltz, Ph.D., CEO, PTC Therapeutics.  "Delaying the loss of ambulation is critical to these patients, as it predicts the time to loss of pulmonary function and mortality."

Patients who received Translarna in routine clinical practice also experienced a slower decline in their physical function compared with the placebo arm of Phase 3 Study 020, as measured by a series of timed function tests. Safety outcomes for patients in the STRIDE Registry were consistent with the known safety profile of Translarna.

"These initial data are very encouraging because they provide the first, real-world evidence for the impact of Translarna when used routinely, longer term," said Dr. Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University.  "We are seeing children who have been exhibiting first clinical symptoms of Duchenne at approximately 3 years of age and yet are still able to walk years after they would normally be wheelchair-bound, which is what matters most to patients and their families."