Date: Jan 11, 2016 Source: PR Newswire (
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SOUTH PLAINFIELD, N.J., Jan. 11, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate update, which will be detailed at the company's presentation at the 34th Annual J.P. Morgan Healthcare Conference. Stuart W. Peltz, Ph.D., Chief Executive Officer will present the company's corporate strategy and anticipated 2016 milestones at 9:30 a.m. PST on Wednesday, Jan. 13, 2016. The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC Therapeutics' website at www.ptcbio.com.
Corporate Update
Commercial Highlights:
Completed a landmark first year ex-U.S. launch of Translarna™ (ataluren) with 206 nonsense mutation Duchenne muscular dystrophy (nmDMD) patients on commercial therapy as of January 8th, including patients from both commercial sales and reimbursed early access programs.
Translarna is now available on a commercial basis in 18 countries and PTC has a global commercial footprint of 46 countries either directly or through commercial partners.
Preparations are underway for a potential 2016 launch of Translarna for nmDMD in the U.S.
Regulatory Highlights:
Completed rolling NDA submission for Translarna to FDA and submitted Phase 3 ACT DMD results to EMA fulfilling the principal condition of the approval in the EU. Regulatory decisions are expected mid-2016.
Following the submission of a variation for Translarna for nonsense mutation cystic fibrosis to the EMA in 2015, PTC is responding to questions and expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) in mid-2016.
Pipeline Highlights:
Advancing PTC's 10 by 20 goal to expand Translarna's pipeline, investigational new drug applications (INDs) for aniridia and Dravet syndrome / CDKL5 have been filed.
PTC and collaboration partners Roche and the SMA Foundation expanded the SMA program to include RG7916, an additional SMN2 splicing modifier. A Phase 1 study evaluating RG7916 was recently initiated in healthy volunteers. Results will inform the development path forward for the SMA program.
Data expected in 2016 from Phase 1 oncology study in BMI1.
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.
