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Sarepta Therapeutics, Inc. Message Board

  • usagary1 usagary1 Apr 24, 2013 9:19 AM Flag


    JMP Securities Company Note

    April 24, 2013 / Comments Off

    Day 2 at MDA: The other side of the coin - patient advocacy in drug development;
    reiterate Market Outperform rating and $50 price target on Sarepta Therapeutics
    based on a risk-adjusted, discounted cash flow analysis. During day 2 of the Muscular
    Dystrophy Association (MDA) conference, Frank Sasinowski, attorney, former FDA
    member, and board member of NORD discussed changes in the 2012 PDUFA law
    that, for the first time, requires the input of patients into drug development. We have
    witnessed hints of this paradigm shift from patient advocates with whom we have
    interacted. Specifically in DMD, the FDA met with three mothers of boys with DMD, one
    the mother of twins, and feedback that Mr. Sasinowski has received from the FDA was
    that the women made a "powerful impression". In our view, this shifting FDA paradigm
    is the wild card in the case for accelerated approval of eteplirsen, as the FDA may be
    more receptive to a higher degree of uncertainty that they, on behalf of patients, are
    willing to take on for the accelerated approval of a potential life-saving therapy. At the
    conference, we also saw the 74-week data for the extension study of eteplirsen, which
    is consistent with the 62-week data and, in our view, are supportive of a drug effect.
    FDA evolution. The FDA is seeking input from patients, which is a paradigm shift, in
    our view. To this end, the Agency will conduct panels on 16 diseases in 2013-2015
    (Figure 1), focusing on important endpoints and outcomes for patients as well as what
    is acceptable to them as far as risk/benefit. One of the 16 disease areas covers inborn
    errors of metabolism, such as DMD. We believe the meetings between the FDA and
    Sarepta and between the FDA and patient advocacy groups are representative of this
    paradigm shift.
    74-week eteplirsen data. Data from the 74-week time point of the open label extension
    study was consistent with top-line data (Figure 2).

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