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Sarepta Therapeutics, Inc. Message Board

  • ml_expert ml_expert Feb 14, 2014 12:52 AM Flag

    odds of possible outcomes and price predictions

    Possible Outcomes:
    1. FDA grants AA and requires post approval PIII
    2. FDA does not grant AA but allows for a single ARM PIII
    3. FDA does not grant AA and require placebo controlled, double blind PIII
    4. FDA does not grant AA, and does not allow for a PIII yet due to lack of evidence for defining reliable clinical outcomes.

    These are the four possible outcomes I can come up with. Am I missing anything? What do you think the odds of each outcome will be and how the stock will react to it?

    I start with my predictions:

    1. Odds 10%, Price: $100
    2. Odds 70%, Price: flat to up slightly
    3. Odds 20%, Price: $15-20 range
    4. Odds 0%, Price: $10

    Sentiment: Strong Buy

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    • Based on today's Northera approval and the following statement from FDA I am now more optimistic about option 1. I am revising my odds as follows. This is a huge change in the odds. I already put my money where my mouth is and tripled my position in SRPT this past week.

      1. Odds 50%, Price: $100
      2. Odds 40%, Price: flat to up slightly
      3. Odds 10%, Price: $15-20 range
      4. Odds 0%, Price: $10

      FDA announcement:

      The FDA is approving Northera under the accelerated approval program, which allows for approval of a drug to treat a serious disease based on clinical data showing that the drug has an effect on an intermediate clinical measure (in this case, short-term relief of dizziness) that is reasonably likely to predict the outcome of ultimate interest (relief of dizziness during chronic treatment). This program provides patient access to promising drugs while the company conducts post-approval clinical trials to verify the drug's clinical benefit, which for this approval is a long-term effect on patient symptoms in NOH, a chronic disease.

      Sentiment: Strong Buy

    • Interesting framing ml_expert.

      I'm in the #3 camp. I prefer this choice because it comports to reality.

      Here is the FDA's position-

      "...we believe that a placebo-controlled trial would be the most likely method for developing interpretable evidence of efficacy for eteplirsen, because efficacy endpoints in DMD are effort-dependent and susceptible to bias, and the natural history is highly variable and has recently improved with steroid use and advances in ancillary care."

      • 2 Replies to pasteur420
      • " has recently improved with steroid use and advances in ancillary care." Than what happened in the GSK phase 3 trial? That was a decade AFTER the steroids and ancillary care was introduced and those boys declined A LOT in 48 weeks. Must be God that had something against them, while the FDA tells us that our boys are doing much better now. The selection criteria for recent trials IS NOT INCLUDING outliers that produce the 'variablity', this is why a max 6MW of 400m +/- 10% is used. The FDA had access to GSK data, as well as met with Duchenne experts and discussed these 'issues' since that silly comment you posted.

      • This statement was from last November. I don't believe FDA is still in the same page.

    • I think you guys are underestimating the odds of AA - the political pressure on the FDA is going to be immense, which in the absence of clinical results would not mean much, but that is not the case here. The company and independent experts have addressed the key issues raised by the FDA in their Nov letter to the company and the general consensus is that the FDA completely missed the boat with their analysis of the data or they did not have the data in hand to warrant their conclusions. The chronic doubters will continually point to the issue of n=12, but the FDA cannot fall back on that - they obviously knew it when they advised the company to pursue an AA filing earlier in 2013 and it flies in the face of the accelerated approval regs and intent. The bottom line is the FDA doesn't have a leg to stand on if they try to argue against AA - they might be able to hide behind their disclosure rules and get away with it in some low profile disease that has alternative treatments available, but it won't work in DMD. Odds of AA are 50%+.

    • Maybe the "event" coming up with the FDA, should be rephrased as being "Trinary"........................with either a "yes", "no", or the FDA favorite "maybe" as possible ways for the agency to approach the AA/P3/endpoint response. Ya, ya........that makes more sense to me. WE ave an upcoming Trinary event, and "maybe" is for certain, an option at the FDA's fingertips.

    • I can assure you, that one important possibility was left out:

      5. The FDA does, what it does best, and decides to somehow kick the can yet again down the road, and basically doesn't provide answers the the AA question everyone is waiting to learn.

      More than once in the recent past ,I've been certain that the FDA would answer the question with either a "yes" or a "no", and more than once, I lost money and was wrong, as the FDA basically didn't do anything but push off making a decision to a later date, and/or said "maybe". Thus, there is always a possibility that the FDA finds a way to not answer the questions about AA, endpoints, P3 and continues to procrastinate. Would that be wrong? yes. Have they behaved this way previously? ABSO-freaking-lutely.

    • Here are my predictions:

      1. FDA grants AA and requires post PIII:
      Odds 10%, Price: $100
      2. FDA does not grant AA but allows for a single ARM PIII: (this is extremely positive for the stock price)
      Odds 30%, Price: $60
      3A. FDA does not grant AA and require placebo controlled, double blind PIII and 6MWT as primary endpoint:
      Odds 50%, Price: $25 (the current stock price assumes this outcome)
      3B. FDA does not grant AA and require placebo controlled, double blind PIII and FDA define a new primary endpoint: Odds 5%, Price: $15
      4. FDA does not grant AA, PIII is further delayed due to lack of evidence for defining reliable clinical outcomes.
      Odds 5%, Price: $15

      Sentiment: Strong Buy

 
SRPT
13.48-0.06(-0.44%)Apr 21 4:00 PMEDT