- PTC plans to complete rolling NDA submission to the FDA by end of 2015 -
- Totality of clinical data demonstrate Translarna's ability to slow disease progression for patients with nonsense mutation Duchenne muscular dystrophy -
- 15 meter benefit observed in 6MWD in overall study population (p=0.213, n=228) -
- 47 meter benefit observed in 6MWD in pre-specified subgroup of 300 - 400 meters at baseline (p=0.007, n=99) -
- Pre-specified meta-analysis of combined ACT DMD and Phase 2b studies demonstrated benefit in Translarna-treated patients across primary (p=0.015, n=291) and key secondary efficacy endpoints -
- Strong safety profile of Translarna confirmed, consistent with previous studies -
--Conference call scheduled today at 5:00 PM ET--
"These results show Translarna's ability to change the course of DMD disease progression. The totality of the data from our two robust placebo-controlled studies across over 400 patients demonstrate a clinically relevant impact on patients' lives," said
After the completion of ACT DMD, both placebo and treated patients were given the opportunity to continue on Translarna in an open-label extension study. Ninety-seven percent of the patients who completed ACT DMD enrolled in the extension study.
"These important results demonstrate positive treatment effects across multiple endpoints and validate our emerging understanding of the optimal patient group in which to demonstrate benefit in the 6-minute walk test. It is particularly impressive that these results were observed in a one-year study," said
ACT DMD, the largest placebo-controlled study ever conducted in patients with DMD, is a multi-center, randomized, double-blind, Phase 3 clinical trial involving 228 patients in 53 sites across 18 countries. Patients between the ages of 7 and 16 with nmDMD were randomized to receive either Translarna 40mg/kg per day (n=114) or placebo (n=114) over 48 weeks. The primary endpoint was change from baseline in the 6MWT. Analyses of data from pre-specified subgroups, including the pre-specified subgroup of patients with baseline 6-minute walk distance (6MWD) of 300 - 400 meters, were also completed. Key secondary outcome measures were timed-function tests, including time to run or walk 10 meters and the time to ascend or descend four stairs. Tertiary endpoints included the North Star Ambulatory Assessment test, a functional scale designed for ambulant boys affected by DMD, and the Pediatric Outcomes Data Collection Instrument (PODCI), a validated tool for measuring quality of life in pediatric patients with orthopedic conditions. Supportive analyses of ambulation were conducted, including the proportion of patients with at least 10% worsening in 6MWD. A pre-specified meta-analysis of combined data from the ACT DMD and Phase 2b (ambulatory decline phase) studies was also performed.
"Duchenne muscular dystrophy is a devastating, progressive muscle-wasting disease, which cuts short the lives of boys and young men," said
The ACT DMD study confirmed the favorable safety profile of Translarna, which was generally well-tolerated, consistent with results from previous studies. More than 500 nmDMD patients have now received Translarna, the largest population to be treated with a disease-modifying agent in DMD.
Translarna received marketing authorization from the
About Duchenne Muscular Dystrophy
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. More information about DMD is available through the
Translarna, discovered and developed by
About PTC Therapeutics
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA, or mRNA, molecule is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology, and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com
Conference Call Today
PTC will host a conference call and provide slides on our website to discuss the results of ACT DMD.
The call can be accessed by dialing (877) 303-9216 (domestic) or +1 973 935-8152 (international) five minutes prior to the start of the call and providing the passcode 61746193. The accompanying slide presentation will be posted at
A webcast replay of the call will be available approximately two hours after completion of the call. The webcast and slide presentation and will be archived on the company's website for two weeks.
For More Information:
+1 (908) 912-9327
+1 (908) 912-9551
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release are forward-looking statements, including statements regarding the future expectations, plans and prospects for PTC; the timing of PTC's planned regulatory filings, including with the
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of final analyses of the data from ACT DMD, which may vary from PTC's initial analysis, lead to different (including more or less favorable) interpretations of the results than the analyses conducted to date, and identify further important information that is not available at the time of this press release; whether the
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this release except as required by law.