Seeking Alpha

Sarepta Therapeutics slides on dystrophin/ clinical benefit questions

  • Shares of Sarepta Therapeutics (SRPT -6.3%) are notably lower on the session.
  • The stock traded up on Friday as the market took a decidedly negative stance on data from a trial of Prosensa (RNA -1%) and GlaxoSmithKline's (GSK -0.2%) drisapersen (an eteplirsen competitor).
  • Today's weakness in SRPT shares may be attributable to a note from Cowen which suggests the drisapersen data highlights the debate regarding dystrophin as a surrogate endpoint.
  • The drisapersen data "underscores [the] point that no correlation can be drawn currently between dystrophin and clinical benefit," Cowen says.
  • Adam Feuerstein's take: "SRPT bears sticking to script. We knew they'd link poor drisapersen dystrophin data to eteplirsen."
  • Note: Cowen is still "bullish on SRPT shares."
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Comments (6)
  • Scrying Biotech
    , contributor
    Comments (1731) | Send Message
    Sarepta Investors: Hang in there! You're going to be fine.
    19 Aug 2013, 02:38 PM Reply Like
  • pat walsh
    , contributor
    Comments (268) | Send Message
    I guess those who shorted Sarepta will try to keep the stock down.When you own stock at $4.23 you really dont worry.I will enjoy the next few days watching the scramble.
    19 Aug 2013, 06:36 PM Reply Like
  • hyperal2
    , contributor
    Comments (4) | Send Message
    I'm a UK based investor. Invested in a UK AIM listed business called Summit plc.
    Many UK based SUMM investors are very surprised at the disparity between the market cap of USA listed companies with DMD solutions, and that of Summit PLC (SUMM). Particularly as Summits solution should help 100% of DMD boys.
    Surely the biotech sector is a global market, so why such large gaps in the market caps of companies working on solutions for the same medical conditions? is the investor community just not aware of what is going on outside their own country?
    19 Aug 2013, 08:44 PM Reply Like
  • hyperal2
    , contributor
    Comments (4) | Send Message
    UK based Summit Plc (SUMM) solution seems superior to both of the above because it offers a different approach.


    I quote from


    "Summit’s most advanced small molecule utrophin modulator is SMT C1100 which is currently in clinical trials. Discovered and developed in partnership with Professor Davies, SMT C1100 treatment increases and maintains utrophin transcription (production of utrophin RNA) which in turn is converted (translated) to utrophin protein. This enables functional utrophin to continually protect the dystrophin deficient muscle fibres. Dosing with SMT C1100 has demonstrated significant disease modifying potential in non-clinical efficacy studies. The results include improvement in whole muscle function in an in vivo study that is analogous to the six minute distance walk test, a primary end-point in patient clinical trials, while it also has a strong synergistic effect with the current standard of care, glucocorticoid steroids. These compelling data have been published in a peer-reviewed scientific journal in 2011.


    SMT C1100 has designated orphan drug status by the US Food & Drug Administration and the European Medicines Agency. Orphan drugs receive a number of benefits including extended market exclusivity following approval and additional regulatory support. In addition, orphan drug products often command premium pricing."


    Summits approach is works with 100% of DMD boys "A significant advantage of increasing or upregulating utrophin is that it will benefit all DMD patients, regardless of the specific genetic mutation that is causing the underlying disease."


    Because SUMM is not listed in the USA it's market cap is still a fraction of Sarepta and other USA listed competitors in this field. On top of this DMD solution, it also has a new type of antibiotic in the pipeline which is specific to C. Diff. and very specific!
    19 Aug 2013, 08:46 PM Reply Like
  • anosol
    , contributor
    Comments (2) | Send Message
    Just to add thy last week Summit PLC shared a panel session with Sarepta and Prosensa at Wedbush' annual biotech event. Clearly Wednush are starting to put them into a similar league.
    20 Aug 2013, 02:27 AM Reply Like
  • hyperal2
    , contributor
    Comments (4) | Send Message
    A copy of the Wedbush Securities Healthcare Conference presentation can be found here: -

    20 Aug 2013, 08:58 AM Reply Like
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