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left2rightdoor 67 posts  |  Last Activity: Jun 29, 2016 11:11 AM Member since: Jul 16, 2012
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  • Reply to

    Disclosure.

    by beaverfan12345678 Jun 29, 2016 3:38 AM
    left2rightdoor left2rightdoor Jun 29, 2016 11:11 AM Flag

    The dystrophin data may not be disclosable because Sarepta doesn't know the level of dystrophin production that will trigger FDA approval .9% 1.5%, 3% etc. However it is a little odd that Sarepta notified the DMD community of the role of the Essence 45/53 changes and their role as confirmatory for eteplirsen, and then added safe harbor statements (uncertainty, unknown results, possible changes) without making a Regulation FD disclosure (general market dissemination) of its communications to the DMD people about the clinical trial changes. Of course, all of this is subject to the extreme volatility, so less disclosure until an actual FDA action is probably prudent. Because FDA has said they are moving as quickly past PDUFA date as possible, likely that FDA action is before end of July, maybe before a month-6weeks after announcement of dystrophin data request.

  • left2rightdoor left2rightdoor Jun 28, 2016 1:40 AM Flag

    Postmarket requirement and commitment studies and clinical trials occur after a drug or biological product has been approved by FDA. For more information, please read: "Report to Congress: Reports on Postmarketing Studies [FDAMA 130]" and the Guidance for Industry (PDF - 456KB)

  • Went from

    Percentage of dystrophin-positive fibers
    to

    Dystrophin protein expression

    (which is not western blot exclusively, so it probably means submission of both PDPF and WB data
    or even all of them rt-PCR as well, because the original workshop in Mar 2015 recommended
    using multiple means to identify and measure dystrophin induction
    This is probably a negotiated point for the PMR/PMC)

  • FDA has two types of post marketing information collection and submission categories: Post marketing requirments (which is what FDA can require) and post marketing commitments, which co agrees to. One type of post marketing "requirement" is for confirmatory studies, called Phase IV, after accelerated approval. This seems to be what is going on, tying the shoelaces before letting eteplirsen take the boys amenable to Exon 51 out for a walk, which hopefully will last several years. See the FDA website on post marketing requirements (PMR) and commitments

  • left2rightdoor left2rightdoor Jun 17, 2016 12:18 PM Flag

    "Morpholinos are also effective to modify pre-mRNA splicing and to inhibit miRNA (Morcos, 2007). These oligonucleotides offer the prospect of a safe and effective therapeutic for a broad range of previously 'undruggable' diseases that include Duchenne muscular dystrophy (Moulton et al., 2009), Huntington disease (Sun et al., 2014), cancer and cardiovascular diseases (Templeton, 2015), Ebola (Heald et al., 2014), etc. Many of the morpholino-based treatments are currently undergoing clinical trials and are showing impressive safety and pharmacokinetics results (Sanghvi, 2011). "

    Once platform validated with Exon 51 approval, other disease pipeline will be valued

  • Reply to

    fortune article ends with this:

    by pebble16x Jun 14, 2016 2:09 PM
    left2rightdoor left2rightdoor Jun 14, 2016 3:16 PM Flag

    What is the expected form of the transaction that maximizes shareholder value? Not just a sale, but a reverse spin that minimizes tax liability by providing a business purpose for the new shares to be distributed tax free with new bases determined based on relative value of assets represented??

  • Captain Mary Kremzner: Now Cat, exactly how does the FDA determine when an expedited approval is warranted?

    Commander Catherine Chew: Generally drugs are approved under Accelerated Approval if they have the potential to impact factors, things like survival, day‑to‑day function or the likelihood of stopping a disease from progressing and becoming an even more serious condition.

    Captain Mary Kremzner: Now Cat earlier you mentioned filling an unmet medical need. How would you define that?

    Commander Catherine Chew: An unmet medical need simply means providing a therapy when none exists or providing a new therapy that may be superior to or less toxic than an existing one.

    Captain Mary Kremzner: So without the standard clinical outcomes how does FDA determine if a treatment is potentially effective?

    Commander Catherine Chew: Great question Mary. Accelerated Approvals use what we call a surrogate end point in clinical trials. These are markers or a physical sign of sorts used as an indirect measurement to predict a clinically meaningful outcome like survival or symptom improvement.

    Captain Mary Kremzner: So exactly how does the surrogate end point save time in the drug approval process?

    Commander Catherine Chew: Well for example, instead of having to wait to learn if a drug prolonged survival in cancer patients the FDA has approved drugs based on evidence that they shrink tumors. As a surrogate end point, tumor shrinkage is reasonably linked to real clinical benefit.

    Captain Mary Kremzner: Okay, so let me make sure I have this. Let’s say a drug goes through Accelerated Approval and gets to market, how do we know if it really provides that clinical benefit?

    Commander Catherine Chew: Any approvals given based on surrogate end points are on the condition that the sponsor will conduct post-marketing clinical trials to verify the anticipated benefit. If these trials, called Phase 4 Confirmatory Trials, shows the drug does in fact provide a clinical benefit then

  • Sites are currently being initiated into the study. Initiation of approximately 39 planned sites in the United States is expected to be completed by June 2016. The initiated sites can be found in the "Contacts and Locations" section of this posting in addition to a listing of the city and states of sites the investigators are working to initiate. T

  • Reply to

    i am buying more tomorrow.

    by renegade259er Jun 13, 2016 11:56 PM
    left2rightdoor left2rightdoor Jun 14, 2016 12:19 AM Flag

    FDA needs to post the transcript - now about 7 weeks, even for an extended day, should be available.
    I want to see the text where one of the AdCom members says I don't want to have a standoff between the Committee and the FDA and the Chair, Caleb A. says, I think you meant between the committee and the applicant, when what the member accurately really meant was, we don't like the FDA's approach and questions.

  • Sarepta has been supposedly been negotiating Pediatric Investigation Protocols with Europe for sometime. when are those finalized?

  • but won't give them any for shorts must be hard to find

  • left2rightdoor by left2rightdoor Jun 1, 2016 3:45 PM Flag

    Approve Eteplirsen, stabilize Exon-51 amenable patients, proceed with 45, 53, other exons
    This will delay progressive decline, stabilize patients for a few years
    Develop a better PPMO, third/fourth generation AONs,
    Develop a myostatin to restore, not just stabilize muscle.

    Art Krieg knows his biotechnology
    Art Krieg ‏@ArtKrieg 2h2 hours ago
    $SRPT from Ed k: if all continues well, PPMO could be in human DMD in ’17. Etep works, 0.9% dys helps; I believe PPMO should give 10X dys

  • Reply to

    Broker wants me to lend my shares

    by left2rightdoor Jun 1, 2016 10:08 AM
    left2rightdoor left2rightdoor Jun 1, 2016 11:54 AM Flag

    Who said it was a margin account. My fully paid accounts don't let shares be borrowed without my permission. Go back to tweeting about your $150 profits from a successful fade. Titan will be at $30 before its at $5 again.

  • left2rightdoor by left2rightdoor Jun 1, 2016 10:08 AM Flag

    to facilitate short sales - no way

  • left2rightdoor by left2rightdoor May 31, 2016 12:32 AM Flag

    16-02 Associate Director, Medical Writing Cambridge, Massachusetts, United States 5/20/2016
    16-03 Director/Sr. Director, Medical Writing Cambridge, Massachusetts, United States 5/20/2016
    16-01 Senior Director, Biometrics Cambridge, Massachusetts, United States 5/20/2016

  • left2rightdoor by left2rightdoor May 29, 2016 3:15 PM Flag

    Lack of a treatment for Exon 51 amenable skipping DMD patients
    FDA reluctance to use patient registries for comparative information
    FDA reluctance to use AA for diseases outside of HIV and Cancer
    FDA insistence on bureaucratic control of patient access to new precision therapies
    FDA lack of use of biomarkers for AA unless validated biomarkers (years of proof)
    FDA contribution to high drug prices by extending valley of death for over-extensive clinical trials
    Lack of treatment for 80 percent of DMD patients amendable to skipping other exons.

  • Reply to

    margin calls?

    by renegade259er May 25, 2016 11:39 PM
    left2rightdoor left2rightdoor May 26, 2016 12:34 AM Flag

    Something like this, depending on the broker
    You need under Reg T something like a 50% initial margin. All this and the following depends on the broker. Then additional margin at say 30% will be required based
    upon the changing value of the shorted stock. So say you short 1000 shares of Sarepta at 20, you would
    have to have in the margin account $20,000 plus a 50% margin or $30,000 with $10K margin to start.
    If the price goes up $5, to 25, the margin requirement is $25,000, plus 30% of 25, or $7,500, for a total
    of $32,500, so you get a margin call for $2500 - you had $30K to start with, remember. Maybe you can come up with another $2500, so you have total account plus margin of $32,500. But it goes up another $10 per share.
    Now the total value of the short position is $35,000 (1000 shares at 35) plus 30% or $10,500, so your total
    margin account requirement is not 32,500, but $45,500, or another $13,000. If you don't come up with that
    when requested, the broker will sell you out. The 30% maintenance margin reqt could be higher or lower depending on broker. Have a hunch some brokers will be raising the 30%. So little guys will not survive, only big deep pockets will be able to push a lot of chips in on the table as the price goes up, unless they decide to get out. Do your own diligence, but this is not a stock to short for small hands and bet against a safe, effective treatment for a significant unmet medical need which will MakeDuchenneHistory.

  • inadequate resources for 2 drug reviews, snow, missing adcom members, opaque and leading questions, 100 members of Congress, 24 Senators, major amendment with positive 4 year data, reports of CRO benefits from study 204 on falls. ADL, 36 experts, 6 doctors and therapists reporting patients benefitted, Adcom comments "it ameliorates the DMD phenotype" "from everything I heard today the drug works, but that wasn"t the way question was phrased". Be Reasonable

  • Chad Onyike asks, to what extent are we supposed to incorporate the testimony of the patients and others
    the wording makes it seem a statistical question
    Woodcock; We are supposed to incorporate patient viewpoints
    Temple; You heard testimony from patients that tests didn't affect their level of effort
    Unger: Unprecedented to have basically all the patients here. We're hearing patients improving doing
    things they didn't do last year.
    Romitti Q: We have 2 different groups, are we supposed to consider dystrophin results
    Woodcock: this is the full approval question, not based on surrogate endpoint
    (a mistaken description - dystrophin could be confirmatory evidence of single arm HC control trial)
    Temple, Historical trials can be adequate and well controlled, some obvious, some not so clear.
    Dunn: thats why we discussed historical controls, evidence from trials plus info from patients can be substntial evidence:
    (So for 10 minutes they discuss, what does this question mean, can we consider patient and expert testimony....obviously the question unclear and basis for voting was not well defined)
    Voting explanations
    Foley: Yes -clearly ameliorated the DMD phenotype.
    Ovbiagele: No, but from everything I heard, this drug works
    Hoffman, Green, Romittee: abstain, don't want to make a type 2 error, fearful of stalemate between
    FDA and panel
    Gunvalson, Dupree: Yes-can't reconcile difference between testimony boys are improving
    Kryscio, Nuckolls, Kesselheim Onyike, No, not well controlled, but there seems to be more informatio from further studies showing benefits not included, use CROs like dementia (but they had 5-6 clinicians testify?)
    Dunn FDA final comments: have listened very carefully, seen AdCom highly influenced by testimony, will consider very seriously
    (So vote was again 6-7 with 3 abstainers sympathetic but restrained by "statistical" question-w Ovbiagele
    saying drug works, basically 7 in favor of approving drug if asked based on all heard from patients & doctors)

  • Reply to

    Their shorting again....losers

    by makebucks89 May 23, 2016 12:11 PM
    left2rightdoor left2rightdoor May 23, 2016 12:33 PM Flag

    Definition of a bear trap: Stock goes up in anticipation of approval, shorts pile on for quick .50 drop they hope, get stuck with FDA sudden decision and lose $30 per share short.

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