Sarepta Looks to be Placing all its bets on Eteplirsen
Mumbai, India -- (SBWIRE) -- 10/16/2013 -- Sarepta Therapeutics, Inc. (SRPT), which has been in the news for its drug eteplirsen for the treatment of DMD would be hosting a conference call to provide updates on its DMD program on the 17th of October 2013 at 12 PM Eastern. Sarepta plans to submit its NDA for eteplirsen to the Food and Drug Administration (FDA) in the first half of 2014.
Sarepta had received feedback from the FDA in July that they were open to considering an NDA filing based on eteplirsen’s Phase II data prior to a confirmatory study, which they intend to start in the first quarter of next year. The only factor holding them back was the FDA wanting more information related to their methodology and verification of dystrophin quantification.
So far this year, Sarepta has been consistently reporting results from its Phase IIb open-label extension study titled Study 202 of eteplirsen in patients with DMD. Results from walking tests conducted this year from 74, 84 and 96 weeks have demonstrated that eteplirsen does lead to continued stability in walking ability in DMD patients.
While presenting at the R.W. Baird 2013 Healthcare Conference in September, CEO Chris Garabedian spoke on three key areas they needed to see success in. He believes Sarepta has unprecedented favorable data in all these areas.
The first area: safety. An important measure to keep an eye on would be toxicity levels. As the pediatric population who would be using eteplirsen for DMD would be taking it for life, it should not cause any long-term toxicity, or for that matter other issues which could arise later on. On safety of eteplirsen, Chris Garabedian stated that “the safety profile we've seen to date has been very favorable and conducive to long-term chronic administration in a pediatric population”.
The second area would be showing that the mechanism of action would work with a bio-chemical response, showing the presence of good uniformity and good response across a population. Over here, they saw dystrophin production at very high levels consistently, not only across samples within patients but also across the patient population. All the patients had between 30% to 60% dystrophin positive fibers after 48 weeks of treatment, placebo crossover after 24 weeks of treatment had more than 28% dystrophin positive fibers on average. Chris Garabedian says the biochemical response seen was critical in showing that eteplirsen was working by the mechanism and Watson-Crick pairing to target that specific genetic sub-type.
The third area would be observing clinical benefit. What Sarepta showed with patients was that, although they had two patients, twins, who lost ambulation by 24 weeks before they were able to confirm dystrophin in their muscle biopsy, the other 10 patients in the study all maintained their walking ability with no deterioration. None of them declined less than 3% from the last time point before they confirmed dystrophin in their muscles.
He felt Sarepta was hitting on all these key criteria that they were looking for when they actually embarked on the eteplirsen study.
At the Baird conference, Chris Garabedian also provided an interesting overview on how DMD was passed down. He stated that the reason Duchenne exists in boys without of frame deletions was they were unable to produce an essential muscle protein. Sarepta's technology helps to skip over the deletion to restore the reading frame or turn an out of frame deletion to an inframe deletion.
Looking at the positive walking test results provided thus far and the data shared, Sarepta Therapeutics looks all set to see positive news coming from the FDA next year. Investors would be keenly hearing CEO Chris Garabedian and Chief Medical Officer Edward Kaye’s update this Thursday and also hear their response to questions on eteplirsen.
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