Release Details
New Analyses from Phase 3 Study 009 of Translarna™ (ataluren) in Patients with Cystic Fibrosis Presented at 39th European Cystic Fibrosis Conference
For more information, visit https://www.ecfs.eu/basel2016.
Poster Presentations
- Data regarding lung function (ePoster WS13.1.1) and exacerbations (ePoster WS13.1) from the Phase 3 study, as well as data regarding the natural history of patients with cystic fibrosis carrying nonsense mutations (Poster 264), are being presented during poster sessions beginning
June 9 th, 2016.
Oral Presentation
- Clinical benefits of ataluren in patients with CF and nonsense mutations,
Friday, June 10 th, 2016,3:00 p.m. -4:30 p.m. presented byJ. Davies ,Royal Brompton and Harefield NHS Imperial College London,London, UK
ABOUT TRANSLARNA™ (ATALUREN)
Translarna, discovered and developed by
ABOUT
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:
+1 (908) 912-9167
jbaj@ptcbio.com
MEDIA:
+1 (908) 912-9551
jomalley@ptcbio.com
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.
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 timing and outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), including whether PTC is required to perform additional clinical and non-clinical trials at significant cost and whether such trials, if successful, may enable FDA review of a new drug application (NDA) submission; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the EEA, including whether the EMA determines that the risk-benefit balance of Translarna supports continuation of our marketing authorization in the EEA on a conditional basis, a full
basis, or at all; whether other regulators agree with PTC's interpretation of the results of ACT DMD; the EMA's determinations with respect to PTC's variation submission which seeks to add Translarna for the treatment of nonsense mutation cystic fibrosis to PTC's marketing authorization in the EEA; the scope of regulatory approvals or authorizations for Translarna (if any), including labeling and other matters that could affect the availability or commercial potential of Translarna; PTC's ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna, including whether final guidance from
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.
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