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Sarepta Therapeutics, Inc. Message Board

hardfocus 16 posts  |  Last Activity: Jul 11, 2014 8:47 AM Member since: Nov 26, 2003
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  • Reply to

    consider these questions:

    by simp08801 Jul 11, 2014 7:49 AM
    hardfocus hardfocus Jul 11, 2014 8:47 AM Flag

    Add 2 more Assumptions:
    1 - increased pulmonary function will assure lifetime use of etep
    2 - muscle enhancing drugs (e.g., idebenone, dantrolene, Cialis, Viagara, etc.) will be added to the treatment regime after the the dystrophin stimulating drugs (Etep) take hold. This "cocktail approach" is already being planned and the individual myogenic drugs mentioned above all are in trials now. Remember, most chronic or life threatening diseases require multi-targeted treatment (e.g., diabetes, hypertension, cancer, AIDS, etc.).

  • Reply to

    Cocktails anyone?

    by jim_himmel Jul 10, 2014 11:34 PM
    hardfocus hardfocus Jul 11, 2014 12:20 AM Flag

    Combination of dystrophin stimulating drug followed later with a muscle stimulant (myogenic) drug has been well discussed in the medical literature. Several studies are in progress (e.g., UCLA studying etep + dantrolene). Many other studies are looking at 6PDEI drugs (Viagara, Cialis, etc.). The 144-wk etep data suggests to me and other clinicians that adding a myogenic drug after induction with Exon-skipper drug will make sense. PS- Just about every disease is muti-drug (cancer, diabetes, hypertension, AIDS, etc.).

  • Reply to

    Merck b/o Idenix for $3.5B

    by hardfocus Jun 9, 2014 9:53 AM
    hardfocus hardfocus Jun 9, 2014 10:35 AM Flag

    Short % of Float for IDIX was 30.9% and for SRPT is 30.2%.

  • Todays buyout of IDIX ($3.5B) by Merck for 3x closing price on Fri gives you an idea of how desperate Big Pharma is for pipeline. Only 2 weeks ago, Motley Fool posted a review of IDIX that was less than flattering. It seems SRPT should now be worth well over this amount.

  • Public scrutiny finally coming to fruition. Yesterday, the U.S. House of Representatives Appropriations Committee, while marking up an appropriations bill for 2015 FDA funding, expressed concern that FDA has "underutilized" its authority to expand the use of Accelerated Approval under the FDA Safety and Innovation Act." The committee directed "FDA to report on the way it has used this authority since 2012, its plans to use it in the future, and a justification for using this authority for diseases that are not life-ending." It also requested the U.S. Government Accountability Office (GAO) produce a report on compassionate access to investigational drugs, including a "review of how the FDA is working with all stakeholders to accelerate the approval of innovative, safe and effective medicines, and how FDA takes into account safety and efficacy data from expanded access programs." Special thanks to Rep. Michael McCaul (R-Texas) who pushed for this.

  • Reply to

    May Be Of Interest To Some.

    by cigan1956 May 28, 2014 7:36 PM
    hardfocus hardfocus May 30, 2014 6:19 AM Flag

    After Eisai's blockbuster drug (ARICEPT -donepezil) for Alzheimers disease went generic last year year, they have been in a tailspin. If they were really smart, they would partner with SRPT to refuel their R&D and next wave of growth.

  • ...regarding provider input just published. Helps the case for AA of drugs like eteplersen. Must read .....it's an Open Access article, so Google any of the below text to retrieve:

    Clinical Therapeutics Volume 36, Number 5, May 2014

    A Community-Engaged Approach to Quantifying Caregiver
    Preferences for the Benefits and Risks of Emerging Therapies
    for Duchenne Muscular Dystrophy

    Holly L. Peay, MS
    Ilene Hollin, MPH
    Ryan Fischer, BA
    and John F.P. Bridges, PhD

  • hardfocus hardfocus May 23, 2014 9:37 PM Flag

    Nothing sinister here....they're just informing you that the shelf originally opened in 2011 (of which they only tapped slightly more than $10M but could have gone up to $100M) will expire on July 26, 2014.

  • This deserves some attention from Tuesday (May 20) ....

    At least two stakeholders called for greater use of accelerated approval by FDA at a Tuesday hearing held by the U.S. House of Representatives Energy and Commerce Committee as part of the committee's 21st Century Cures initiative. Frank Sasinowski, director of the National Organization for Rare Disorders, said FDA should increase the use of accelerated approval by considering it for each new therapy and recommended greater use of intermediate clinical endpoints for accelerated approval. Sarah Radcliffe, EVP for health for the Biotechnology Industry Organization, called on FDA to clarify the process for validating novel endpoints and to discuss with sponsors the use of surrogates or intermediate clinical endpoints earlier in development.

    Radcliffe also stressed that the President's Council of Advisors on Science and Technology (PCAST) urged FDA to use accelerated approval more broadly, beyond HIV/AIDS and oncology, which could result in "fewer, smaller, or shorter clinical trials." The E&C committee held Tuesday's hearing to review recommendations from PCAST for FDA included in a 2012 report for the agency (see BioCentury Extra, Sept. 25, 2012).

    The E&C committee is holding a series of roundtables and hearings and is releasing white papers on ways to accelerate the development and approval of drugs and devices under its 21st Century Cures initiative, which the committee unveiled last month.

  • hardfocus hardfocus May 5, 2014 12:30 AM Flag

    Vuk - sorry to break this to YOU, but FDA is accountable to Congress and the American people. When FDA acts erratically or mismanages applicable legislation, such as Congress passage of the AA law in July 2012, then corrective measures shall be taken. FDA has been remanded many times, e.g, AIDS activists in the past.

  • hardfocus hardfocus Apr 26, 2014 7:06 AM Flag

    You sound like Putin, except forgot to mention that Russian capital flight was over $60B in 1Q2014 (more than all of 2013) and expected to to go to $150B by end of yr. Russian tycoons know their money is really safe & wisely invested in EU, Cyprus, and USA. ....and yes, Russians are well invested in SRPT.

  • Reply to

    Austin

    by greyzone513 Apr 23, 2014 8:55 PM
    hardfocus hardfocus Apr 25, 2014 6:59 PM Flag

    Pasteur- lower extremity muscles may be too fibrotic by early teens for DMD boys like Austin to walk again, but upper thoracic (diaphragm, lungs, heart) might still have enough salvageable dystrophin fiber. Recall, SRPT data has shown favorable PFTs and that is why FDA wants an older & non-ambulatory group to be studied. Ultimately, the clinical benefits in the approved drug label may different for age & preexisting severity of the DMD patient.

  • hardfocus hardfocus Apr 22, 2014 9:39 PM Flag

    What if a big pharma uses this to establish a minority stake in SRPT? ALNY is a good example: 2 mos ago, Sanofi bought 12% stake. In prior years, Novartis bought 13% and Roche 5% stakes....and bought bailed out later when the failed ALNY tech became obvious.

  • Reply to

    Quote from Hoffman from WP yesterday

    by likeafox7210 Apr 22, 2014 11:22 AM
    hardfocus hardfocus Apr 22, 2014 3:45 PM Flag

    So Hoffman is insinuating other DMD experts like Drs. Wilton, Mutoni, MacDonald, Kunkel, and Mendell (principal investigator for eteplersen) are Bullies? His arrogance is over the top....and yes, he has personal friends in nearby FDA to his WDC hospital. It is the ultimate in DC insider politics.

  • As I noted in earlier post, SRPT has a superior RNA antisense chemistry than ALNY (whose current market cap is $3.5B) and none of the issues of inability to penetrate cells, toxicity, etc. Plus ALNY recently guided no expected drug to market until 2017. SRPT has none of these issues and drug to market now expected for 2015. CONCLUSION: SRPT deserves a $3.5B market cap now (and will get it when b/o comes) with this benchmark.

  • One has to wonder why SRPT market cap not in same range as ALNY $3.5B. Last week, Novartis terminated collaboration with ALNY and this follows similar terminations by Merck and Roche. ALNY admitted no new drug to market expected until 2017 and that drug delivery to cells is a challenge they're struggling. Surprisingly, Sanofi then bought equity shares @ $65. Remember, SRPT has superior chem and advantages in other competing issues. Just a matter of time.....

SRPT
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