Full Press Release Details
SOUTH PLAINFIELD, N.J. , June 10, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ) today announced that new analyses from the company's previous Phase 3, double-blind, placebo-controlled, 48-week 009 trial of Translarna™ (ataluren) for the treatment of nonsense mutation cystic fibrosis (caused by at least one nonsense mutation) are being presented at the 39 th European Cystic Fibrosis Conference, June 8-11, 2016 , in Basel, Switzerland .
For more information, visit https://www.ecfs.eu/basel2016 .
Poster Presentations
Oral Presentation
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. Translarna is licensed in the European Economic Area 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 PTC THERAPEUTICS, INC . 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 is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. 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 .
FOR MORE INFORMATION:
INVESTORS: Jane Baj +1 (908) 912-9167 [email protected]
MEDIA: Justine O'Malley +1 (908) 912-9551 [email protected]
FORWARD LOOKING STATEMENTS: All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including statements regarding the future expectations, plans and prospects for PTC; the clinical utility and potential advantages of Translarna; the rate and degree of market acceptance of Translarna; the timing, results and conduct of PTC's clinical trials and studies of Translarna; PTC's strategy, future operations, future financial position, future revenues or projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "will," "work towards," "anticipate," "believe," "estimate," "expect," "intend," "may," "predict," "project," "plan," "potential," "would," "could," "should," "continue," and similar expressions.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that Translarna will receive full regulatory approval in any territory or maintain its current marketing authorization in the EEA, or prove to be commercially successful in general, or specifically with respect to the treatment of nmDMD.
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 press release except as required by law.
SOURCE PTC Therapeutics, Inc.