The JPMorgan Healthcare Conference currently under way in San Francisco is arguably the most important event of the entire year for the health care sector. This is one of the rarest opportunities for biotechnology, pharmaceutical, and medical device companies to open up about where they've been and where they're headed, so it pays to take notice.

Considering that from a high-to-low point Sarepta Therapeutics skyrocketed almost 1,400% at one point last year following the release of positive mid-stage data on eteplirsen, its Duchenne muscular dystrophy drug, I knew I had to keep my eyes peeled for what the company had to say at the JPMorgan Healthcare Conference.

The data, released in October, demonstrated not only a degradation of the disease, but actual improvement as evidenced by the 89 meter six-minute walking improvement over the placebo. As my Foolish colleague Brian Orelli noted, eteplirsen crushed Sanofi and BioMarin Pharmaceuticals' Aldurazyme in the results column, and as an orphan drug -- much like Alexion Pharmaceuticals' Soliris or Vertex Pharmaceuticals' Kalydeco -- it's eligible for patent protection, as well as accelerated review status since it targets clinically unmet needs. Not to mention, orphan drugs like Soliris and Kalydeco carry a hefty price tag.


As expected, the majority of Sarepta's presentation revolved around eteplirsen -- which isn't a bad thing. But, we also ascertained some milestone dates to keep in mind as it moves eteplirsen, and its remaining portfolio, forward.

The key points to Sarepta's eteplirsen study, if they haven't been touched on already, are that there weren't any treatment-related adverse events and that 28 of 36 patients enrolled in its studies showed increased dystrophin production. Sarepta is planning a confirmatory study which will begin enrolling later this year and begin dosing in the first quarter of 2014.

Sarepta will actually be stepping away from eteplirsen's studies for much of 2013 to prepare for the possibility of large-scale manufacturing of the drug. Since Sarepta considers manufacturing eteplirsen to manufacturing a small-molecule drug, the assumption is that purity and safety won't be an issue with the FDA with regard to the manufacturing process.

As I alluded previously, orphan drug pricing is often high, and based on Sarepta's presentation, it is expecting to price eteplirsen somewhere in the $300,000 to $500,000 price range -- consistent with comparable rare disease drugs -- and bring in between $600 million and $1 billion annually. By Sarepta's own figures, eteplirsen will target 13% of the 35,000 DMD patients in the U.S. and European Union.

Another key point worth mentioning is Sarepta's research on additional exons. In addition to exon 51, which is what eteplirsen targets, Sarepta is also developing treatments for exons 45, 50, and 53, which are currently in preclinical trials and are slated to be discussed before the FDA in a pre-IND meeting in the second-half of this year. Assuming all three translate into success, this would give Sarepta a chance to market to a third of all DMD patients.

In summary, we're still quite a ways away from actually seeing eteplirsen approved by the FDA, but we're going to see Sarepta's focus shift solely away from research and broaden into developing a commercial manufacturing process. Keep your eyes peeled for anything out of the ordinary in terms of the other exons in preclinical trials, and watch for a potential accelerated NDA filing much later this year. If that NDA doesn't come until next year, Sarepta could have a relatively quiet year.

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The article JPMorgan Healthcare Conference Highlights: Sarepta Therapeutics originally appeared on Fool.com.

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong. Motley Fool newsletter services have recommended buying shares of BioMarin Pharmaceutical and Vertex Pharmaceuticals. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

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