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Translarna Phase 2b Data Published In Muscle & Nerve


PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of data and related analysis which provide strong support for concluding that Translarna™ (ataluren) was active and showed clinically meaningful improvements in Translarna-treated patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The data was published in the October issue of Muscle & Nerve. Translarna received marketing authorization in the European Union in August 2014 for the treatment of ambulatory nmDMD patients five years and older.

The data demonstrated that from baseline to Week 48 nmDMD patients treated with Translarna (40 mg/kg/day given in three doses) had a 31.3 meter benefit in six minute walk distance (6MWD) relative to patients in the placebo group (post hoc p=0.056). Though having achieved the targeted treatment effect, the results did not achieve significance at the p

"Prolonging ambulatory function produces a myriad of benefits that are essential and important to boys living with DMD and affords boys a longer period of self-sufficiency," stated Professor Kate Bushby, M.D., Institute of Genetic Medicine, Newcastle University. "During a one-year clinical trial, substantial increases in muscle strength and function from a dystrophin-restoration therapy like Translarna are not generally anticipated but rather demonstrating the ability to stabilize or slow the decline in muscle function is the real goal. Translarna offers real promise for patients as a treatment that addresses the underlying cause of this rare genetic disorder."

"The results presented in this publication show that patients treated with Translarna demonstrated clinically meaningful trends across all patient groups as measured by the six minute walk distance," stated Robert Spiegel, M.D., FACP, Chief Medical Officer of PTC Therapeutics, Inc. "The results also showed that Translarna showed an even more pronounced effect for those patients who are rapidly declining in walking ability. Importantly, the results observed in the 6 minute walk test were also supported by positive trends in certain other important secondary endpoints that were measured during the course of the trial."

"We are pleased by the results observed in this study," stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "These results, as well as the work we did in defining and understanding the natural history of DMD utilizing the 6 minute walk test, give us a high degree of confidence that our ACT DMD confirmatory trial is both well designed and powered for success. We look forward to the Phase 3 trial results in 2015 and remain committed to bringing Translarna to all patients who can benefit from it."

About the Phase 2b Clinical Trial
The randomized, double-blind, placebo-controlled Phase 2b trial was designed to evaluate the safety and efficacy of 48 weeks of Translarna therapy in patients with nmDMD. The study enrolled 173 participants at 37 sites in North America, Europe, Australia, and Israel. Participants, males ≥5 years, were randomized to receive received placebo, Translarna 40 mg/kg/day or Translarna 80 mg/kg/day given at morning, midday and evening. The primary outcome measure was the total distance walked during a 6-minute walk test, a standardized test of ambulation. Other outcome measures in the study evaluated activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and Translarna blood levels were also monitored.

About Translarna™ (ataluren)
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. PTC has received conditional marketing authorization in the European Economic Area for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

About Duchenne Muscular Dystrophy (DMD)
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD, the more severe form of the disorder, lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. It is estimated that a nonsense mutation is the cause of DMD in approximately 13% of patients, or approximately 2,000 patients in the United States and 2,500 patients in the European Union. More information about DMD is available through the Muscular Dystrophy Association (, Parent Project Muscular Dystrophy (, Action Duchenne (, United Parent Projects Muscular Dystrophy (, Muscular Dystrophy Campaign ( and AFM (l'Association francaise contre les myopathies), (

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