Release Details
Translarna™ Phase 2b Data Published in Muscle & Nerve
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 < /=0.05 level due to a higher than expected variability in a heterogeneous patient population. In a retrospective analysis, more severely affected patients whose baseline 6MWD was less than 350 meters and were receiving Translarna had a 68.2 meter benefit relative to the placebo group (nominal p=0.0053). Patients receiving Translarna also demonstrated a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD. Additionally, based on a retrospective analysis, patients receiving treatment also trended better in the stair climb and stair descend time-function tests as well as other secondary endpoints of physical function. Safety results showed that Translarna was generally well tolerated. Serious adverse events were infrequent and none were considered to be related to Translarna. The most frequent adverse reactions at the recommended dose were nausea, vomiting, and headache. These adverse reactions generally did not require medical intervention, and no patients discontinued Translarna treatment due to any adverse reaction.
"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
"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
"We are pleased by the results observed in this study," stated
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
About Translarna™ (ataluren)
Translarna, discovered and developed by
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
About
PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are essential to proper cellular function. PTC's internally discovered
pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has developed proprietary technologies that it applies in its drug discovery activities and in collaborations with leading biopharmaceutical companies. For more information on the company, please visit our website www.ptcbio.com.
Any statements in this press release about future expectations, plans and prospects for PTC, the development of and potential market for PTC's product candidates, our clinical trials, including ACT DMD, our current and planned regulatory submissions, our earlier stage programs, the potential advantages of Translarna and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan" "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Forward-looking statements involve substantial risks and uncertainties that could cause our future results,
performance or achievements to differ significantly from those expressed or implied by these forward-looking statements. Such risks and uncertainties include, among others, those related to the timing and conduct of clinical trials, including ACT DMD, availability of data from clinical trials, expectations for regulatory approvals, our scientific approach and general development progress, the availability or commercial potential of our product candidates and other factors discussed in the "Risk Factors" in PTC's most recent Quarterly Report, which is on file with the
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