What’s Next for Sarepta Therapeutics Inc (SRPT)?

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Either way, look for Sarepta to begin lining up patients and sites for additional testing in the second half of this year. Dosing of patients participating in the study would probably begin in the first quarter of 2014. Announcements of interim results could occur later in the year, although it seems likely that final results would be pushed back to early 2015.

If we assume the company goes down the phase 3 path, each announcement would potentially serve as another catalyst for Sarepta. Confirmation of efficacy and safety in a larger study should pump up shares.

3. More exons on the way
DMD is caused by low production of muscle protein dystrophin. The problem is that a section of the gene called an exon is deleted and the rest of the gene can’t be built as a result. Eteplirsen works by skipping the deleted exon and allowing dystrophin to be produced without it.

So far, Sarepta has focused primarily on skipping exon 51. Around 1,950 DMD patients in the U.S. could potentially be helped by skipping this exon. The company estimates that market potential to be between $600 million and $1 billion per year. Around 2,600 additional patients in Europe could also possibly be treated with eteplirsen.

However, the exon-skipping approach can also be used for other exons. Sarepta projects that four other targeted exons represents a potential market nearly twice the size of the population affected by exon 51. The company plans to have a Pre-Investigational New Drug Application meeting with the FDA in the second half of 2013 to discuss moving forward with clinical trials for these other exons.

Pathway to success
I’m not sure whether Sarepta will convince the FDA on the merits of accelerated approval for eteplirsen. If it does, shares will undoubtedly skyrocket. If not, shares will fall on the disappointment. However, that could present an excellent buying opportunity for investors. Good news on further clinical studies or progress on targeting other exons should bolster the stock.

What’s immediately next for Sarepta? We’ll see. I think that what’s ultimately next for Sarepta, though, should be a pathway to success.

The article What’s Next for Sarepta? originally appeared on Fool.com and is written by Keith Speights.

Fool contributor Keith Speights has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned.

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