Markets

PTC Therapeutics Presents Phase III Data on Translarna

PTC Therapeutics, Inc.PTCT presented data from the phase III ACT DMD study on Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

Although Translarna was safe and generally well tolerated, the primary endpoint was not statistically significant in the overall intent-to-treat study population. However, pre-specified meta-analysis of combined ACT DMD study and phase IIb studies on Translarna demonstrated benefit across primary and key secondary efficacy endpoints.

We note that Translarna is approved in the EU for use in ambulatory nmDMD children in the age group of five years and above. The company intends to complete the rolling submission of a New Drug Application (NDA) for Translarna in the U.S by year end. In addition, the company plans to submit the results from the phase III ACT DMD study to the European Medicines Agency (EMA).

Translarna has orphan drug status in the U.S. and the EU for DMD, cystic fibrosis (CF), mucopolysaccharidosis I (MPS 1) and aniridia.

Apart from the ACT DMD study, Translarna is being studied in the phase III ACT CF study for the treatment of nonsense mutation cystic fibrosis (nmCF). The company intends to complete patient enrolment by the end of this year and present data by the end of 2016. We note that Translarna is under review in the EU for the treatment of nmCF for patients not taking chronic inhaled aminoglycoside antibiotics.

We remind investors that companies like BioMarin Pharmaceutical Inc. BMRN and Sarepta Therapeutics, Inc. SRPT are looking to bring their DMD treatment drug to market. Currently, BioMarin's drisapersen and Sarepta's eteplirsen are under FDA review for the treatment of DMD amenable to exon 51 skipping with an action date of Dec 27, 2015 and Feb 26, 2016, respectively.

PTC Therapeutics carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Nexvet Biopharma Public Limited Company NVET , sporting a Zacks Rank #1 (Strong Buy).

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days . Click to get this free report >>

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

BIOMARIN PHARMA (BMRN): Free Stock Analysis Report

PTC THERAPEUTIC (PTCT): Free Stock Analysis Report

SAREPTA THERAP (SRPT): Free Stock Analysis Report

NEXVET BIOPHARM (NVET): Free Stock Analysis Report

To read this article on Zacks.com click here.

Zacks Investment Research

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

In This Story

BMRNSRPTPTCT

Other Topics

Stocks

Latest Markets Videos

Zacks

Zacks is the leading investment research firm focusing on stock research, analysis and recommendations. In 1978, our founder discovered the power of earnings estimate revisions to enable profitable investment decisions. Today, that discovery is still the heart of the Zacks Rank. A wealth of resources for individual investors is available at www.zacks.com.

Learn More