Surprising MS Drug Approval: Time to Buy?

PTC Therapeutics  (NASDAQ: PTCT  ) shares soared recently after the EU issued a preliminary approval for the company's Duchenne muscular dystrophy (DMD) drug, Translarna. This caught the market by surprise, which explains the 59% gains over the course of 3 days. Is their still upside in Translarna, or would investors be better served taking a long look at one of its competitors?

The other two stocks attmepting to treat DMD are using a different approach from PTC and have had mixed results. Prosensa's (Nasdaq: RNA) drug failed a phase 3 trial and its big pharma partner walked away, while Sarepta (Nasdaq: SRPT) has promising enough mid-stage results that it is pushing for an accelerated approval from the FDA.

In this episode of The Motley Fool's health-care show Market Checkup, analysts David Williamson and Michael Douglass talk about Translarna's surprising approval and what it means for investors in PTC Therapeutics, Sarepta, and Prosensa. 


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  • Report this Comment On June 02, 2014, at 12:04 AM, worddownunder wrote:

    MS drug? Actually Duchenne muscular dystrophy - unrelated to MS.

  • Report this Comment On June 02, 2014, at 1:04 AM, scientificman wrote:

    The problem with Serepta is Big, because Pulmonary data at 120 weeks was not statistically significant in 12 patients. "Serepta data demonstrated a 14.6% mean increase in MIP and a 15.0% mean increase in MEP. Analyses of MIP percent predicted (MIP adjusted for weight) and MEP percent predicted (MEP adjusted for age) showed a mean increase in MIP percent predicted to 95.2% from 90.2% in MIP percent predicted and a slight mean increase in MEP percent predicted to 79.6% from 79.3%. In addition, there was a mean increase in forced vital capacity (FVC) of 8.7% from baseline to Week 120 and FVC percent predicted (FVC adjusted for age and height) was maintained above a mean of 90% through Week 120 with 101% at baseline and 93% at Week 120." So Only MEP data were statistically significant. It is not good predictions for Serepta next phase, which has to be much bigger than 12 patients and with placebo arm…

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