JPMorgan Healthcare Conference Highlights: Sarepta Therapeutics

The past four days have brought together pharmaceutical, biotechnology, and medical device makers all under one roof in what is arguably the most important health care conference of the year, the 2014 JPMorgan Healthcare Conference.

Just like the recently concluded Consumer Electronics Show in Las Vegas, this annual event gives health care companies a chance to demonstrate to investors and Wall Street where they've been and where they're headed. Because earnings guidance can be somewhat irrelevant for clinical-stage biotech and medical device companies, consider this event your chance to gain guidance from some 300 top health care companies.

Today, we're going to take a closer look atSarepta Therapeutics' (NASDAQ: SRPT) presentation, which was delivered Wednesday by President and CEO Chris Garabedian.

Sarepta Therapeutics' past year Like many of the companies we've chronicled this week, Sarepta's had a wild ride over the past year, flying high at one point on continued follow-up data of its relatively small midstage study of eteplirsen for Duchenne muscular dystrophy, or DMD, a disorder that results in muscle degeneration and early death in boys.

Shares of Sarepta climbed to a 52-week high of $55.61 in September shortly after an experimental rival drug, drisapersen, developed by Prosensa (NASDAQ: RNA) and GlaxoSmithKline (NYSE: GSK) , missed its primary end point by a mile in late-stage trials. However, Sarepta also tanked just weeks later after the Food and Drug Administration decided against supporting an accelerated drug approval for eteplirsen; the agency would not make the connection that increased dystrophin production led to its remarkable trial results.Also, given the recent failure of drisapersen in a phase 3 study, the FDA felt it pertinent that Sarepta engage in a broader study. Shares ultimately dipped as low as $12.12.

What Sarepta had to sayAs you might expect, with Sarepta being the leading DMD drug developer, Garabedian spent pretty much the entirety of his JPMorgan conference presentation discussing the benefits of eteplirsen and its potential superiority over other treatment possibilities. He also alluded to a number of new pipeline products.

The two factors that I found most intriguing from Sarepta's presentation were its new DMD and infectious disease ventures and the recently released 120-week data on eteplirsen.

Garabedian wasted no time by pointing out early that Sarepta exon-skipping technology would add another three exons to its clinical focus -- exon 52, exon 55, and exon 8 -- which are currently in the lead sequence identification process. DMD has many genotype variables, so this isn't a disorder where one therapy fixes all. Sarepta anticipates having the lead sequence selected for all three exons by the second quarter, and plans to file two or more investigational new drug applications for these compounds in the latter half of the year. In addition, it expects to have a pre-investigational new drug application meeting with the Food and Drug Administration over exon 53 sometime this quarter. All told, as you can see below, Sarepta is now targeting eight specific exons. Most importantly, it expects to dose the first patient in its critical phase 3 trial for exon 51 with eteplirsen in the second quarter.

Source: Sarepta Therapeutics.

Garabedian, toward the end of his presentation, also noted Sarepta's burgeoning infectious disease pipeline, with an influenza, Marburg virus, and Ebola virus all in early stage clinical trials, and additional infectious disease drugs in the discovery stage, including tuberculosis and dengue.

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JPMorgan Healthcare Conference Highlights: Sarepta Therapeutics