JPMorgan Healthcare Conference Highlights: Sarepta Therapeutics

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 (NASDAQ: SRPT  ) 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 (NYSE: SNY  ) and BioMarin Pharmaceuticals' (NASDAQ: BMRN  ) Aldurazyme in the results column, and as an orphan drug -- much like Alexion Pharmaceuticals' (NASDAQ: ALXN  ) Soliris or Vertex Pharmaceuticals' (NASDAQ: VRTX  ) 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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  • Report this Comment On January 10, 2013, at 12:35 PM, redplate wrote:

    One point that is a bit misleading is this. Tou state correctly that 28/36 pts made dystrophin. However that 'low number' is a result of the nature of dose escalation studies, where a number of pts recieved what are quite low doses in early UK trials. However since safety was observed and doses were indeed escalated those results are better stated as 20 out of 20 patients that were dosed at 10mg or higher made dystrophin. There have been no dose limiting effects seen at 30 or even 50mg doses, and ALL of these patients have shown a significant response and all of these patients individually and as a whole have met the defined endpoint for the studies. Respose rates are 100%

    Not a trivial note.

  • Report this Comment On January 12, 2013, at 11:00 AM, MEDS4HEALTH wrote:

    Kimberly Lee, an analyst at Janney Montgomery Scott LLC says Sarepata probably won’t receive accelerated approval based on current results in so few patients and likely will be required to run a larger, pivotal study. I would like to present an alternative view. Sarepata is seeking results in a new area of drug therapy which is RNA based Orphan drugs used to treat rare diseases presently are DNA based.

    Several drugs on the market today such as Cerezyme for treatment of Gaucher’s disease, Fabrazyme for Fabry disease and Myozyme for Pompes disease come to mind. These drugs were given FDA approval based on very similar study groups to Sarepata’s eteplirsen.

    Eteplirsen has been proven to have little sign effects in all patent’s receiving the medication The positive results far out weigh the chances of adverse effects since Duchenne disease is fatal in most cases by 25 to 30 years of age. All of the above medications have side effects and often times present adverse effects that must be reported to the FDA. Myozyme even has a black box FDA warning to health care professionals.

    It is clear that Kimberly Lee has not sat with a child with one of these diseases and seen the terror in the eyes of a mother or father hoping for something positive to happen to save their child from a dreaded disease. Parents, physicians, pharmacists and nurses are joining advocacy groups to petition their congressional representatives to ask the FDA to fast tract eteplirsen. In the coming weeks and months, the pressure will build to approve the drug and than increase the sample size. At least more children can be treated while compiling more data. Also funds can be provided to Sarepata to implement full scale production.

    Let us depend on our financial analyst to recommend or not recommend stocks based on solid financial statements. In this case, let us look to medical research personal to look at medical data to conclude what the FDA may do or not do.

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