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Insmed Incorporated Message Board

  • fudfighter4 fudfighter4 Feb 10, 2013 3:05 PM Flag

    Pole position to treat the two billion with latent TB

    Rehdvm - apart from your ignoring the reality that the two billion worldwide estimated to be carrying latent Tuberculosis infection don't have any symptoms, and would have little interest in a therapeutic regimen involving months of nasty side effects from drug-associated blood toxicity - your observations on this topic suggest you are pretty clueless regarding how well suited as regards efficacy is a liposomal inhalation therapy to TB in particular.

    Re your -

    "And fud is quoting Estonia for justifying treating TB with Arikace instead of looking at the joint CDC and NIH recommendations for treating pulmonary TB. TB does leave the lungs and go systemically to the liver, other abdominal organs and, as recently mentioned in the scientific literature, sequesters in the bone marrow.

    Lets put it this way, the new drugs for treating pulmonary TB will include the mycobacterial metabolic and enzyme inhibitors before they will fall back on any antibiotic, like Arikace. Just read the CDC and NIH statements."

    - that last bit of advice was a tiresome bluff on your part which I'm happy to call. This isn't the first time you've made an attempt to deceive supported by the implication that your version would be corroborated by a visit to an 'official' site.

    From the CDC site -

    "TB is a disease caused by a bacterium called Mycobacterium tuberculosis. The bacteria usually attack the lungs, but TB bacteria can attack any part of the body such as the kidney, spine, and brain." "TB is spread through the air from one person to another. The TB bacteria are put into the air when a person with TB disease of the lungs or throat coughs, sneezes, speaks, or sings. People nearby may breathe in these bacteria and become infected."

    So (who would have guessed?) ..... the bacteria USUALLY attack the lungs.

    Why do you believe that these "metabolic and enzyme inhibitor" therapies would me more effective when utilising a delivery method which relies upon -

    achieving a concentration of drug in the bloodstream sufficient to induce a significant amount of the drug to move from the bloodstream into the interstitial fluid in the lungs, and then

    maintaining that concentration of drug in the bloodstream for as long as it takes for the majority of the tuberculosis bacteria, which are near the air interface some distance from the blood vessels, to be exposed to an effective concentration of the drug?

    In other words -

    why do you believe it would not make more sense to deliver these "metabolic and enzyme inhibitors" directly to the area of the lungs where the majority of the bacteria reside, utilising Insmed's liposomal inhalation platform?

    which of the two routes of delivery do you believe would allow the greater opportunity for drug-resistant strains to develop?

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    • Again...sheer nonsense from fud. Latent TB is not treated. It is like saying, "I have a latent headache, I'll take an aspirin." It doesn't work that way. Arikace is not prophylactic therapy.

    • Under the heading "Strawman in the WIND . . ." Rehdvm was kind enough back in 2011 to educate the board on the structure of the clinical trial process -

      "Phase I Trial - single dose safety in multiple subjects.

      Phase IIa - multiple dose safety in multiple subjects.

      Phase IIb - escalating multiple dose safety, Phase III enabiling.

      Phase III - clinical trial of efficacious dose and continued safety trial looking for transient, reversible side effects from clinical dose. Therapy with test drug more advantageous to patient than risk of side effects.

      NDA - New drug application based upon completion of Phase III clinical trial, demonstration of ability to produce three cGMP batches of drug that will meet specifications of test substance used in Phase III clinical trial and request for approval to market.

      Please read the ClinicalTrial.gov "Glossary." Or are you too busy to educate yorself?!?"

      Anybody here who doubts that Rehdvm is sufficiently stupid to post a complete fabrication which is easily exposed can quickly find his post with a search for the word "enabiling".

      Here are the definitions anybody would have actually seen had they taken the trouble to call his bluff by visiting the ClinicalTrial.gov Glossary -

      "PHASE I TRIALS: Initial studies to determine the metabolism and pharmacologic actions of drugs in humans, the side effects associated with increasing doses, and to gain early evidence of effectiveness; may include healthy participants and/or patients.

      PHASE II TRIALS: Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition under study and to determine the common short-term side effects and risks.

      PHASE III TRIALS: Expanded controlled and uncontrolled trials after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional information to evaluate the overall benefit-risk relationship of the drug and provide and adequate basis for physician labeling."

      Rehdvm invented that insulting pile of dog-poo because it suited his agenda for the investors who use this forum to believe that none of the data available for Arikace reflected efficacy.

      According to Rehdvm's pathetic fabrication efficacy is not assessed until Phase III. Everything before Phase III assesses safety only. But the truth is that efficacy is assessed as early as Phase I.

      Imagine how this bloviating charlatan must be feeling having seen the guidance I posted in this thread from the head of the FDA concerning the FDA's new resolve to "move drugs for serious diseases to market more quickly" -

      "We expect many of these would come available very quickly with Phase 1 data," Woodcock said.

      And Arikace isn't just a therapy for serious diseases - it's a potential weapon in the wider strategy of the FDA and EMEA in taking proactive action against the public health threat of drug-resistant strains of Gram-negative pathogens.

      • 1 Reply to fudfighter4
      • Fud, you do make it tough to get a good feel for what to expect from this company, and it's
        stock by coming up with these long winded overviews. Your attacks on Red are somewhat
        unwarranted. He has offered in his latest post nothing more than a professional overview
        from a firm that spends countless hours coming to their much researched conclusions. You
        then come back and provide not one, or two, but three consecutive posts that provide your
        flowered illogical view of things. I for one would love to see you be 100% correct, but I doubt
        you will be. You really should tone down both your pumping, and your negativity to Red. What's
        more amazing than anything is you are both purported longs, but take each other to task.
        Good thing you folks aren't in the legislature of this country..We wouldn't ever get anything
        accomplished. .....Wait! maybe your are.!!!!!!!!!

    • While we're waiting for Rehdvm to have the good manners to respond .....

      From the Bloomberg article Blase picked up on -

      "Drugmakers may win approval from U.S. regulators for "breakthrough" therapies after a single round of studies, rather than three, in an effort to speed them to patients, a Food and Drug Administration official said.

      The agency assigned three experimental medicines the new status to try to reduce the time needed to get them to market against deadly diseases, Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said yesterday in an interview before a Bloomberg health conference in New York. Vertex Pharmaceuticals Inc. said last month that two of its drugs for cystic fibrosis had won the designation.

      Breakthrough status means the companies will have closer communication with top FDA staff to move drugs for serious diseases to market more quickly, potentially with data from an expanded Phase 1 trial, Woodcock said. Improved understanding of diseases is leading to more candidates for this status.

      "We expect many of these would come available very quickly with Phase 1 data," Woodcock said."

      One suspects that the "Breakthrough" designation is intended for drug candidates which have far less clinical data behind them than do Arikace and iPlex. But the FDA's new 'sooner rather than later' approach does seem (to an optimist such as myself) to offer the prospect of the following developments -

      1. approval of Arikace as a therapy for all susceptible pulmonary Gram-negative infections - supported by data from the original injected-amikacin studies, the Cystic Fibrosis studies and the 2009 Non-CF bronchiectasis Phase II study.

      2. approval of Arikace as a therapy for all susceptible pulmonary mycobacterial infections - supported by data from all of the studies mentioned above plus the NTM Phase II study.

      3. approval of PremIplex as a preventative therapy for the complications of pre-term birth - supported by data from all of the original iPlex studies plus the Premacure Phase II study.

      There would be a wealth of clinical data available for all three (most importantly, data on safety) - and all three warrant a fast-lane approach because they target serious unmet medical need.

      • 1 Reply to fudfighter4
      • Here's the relevant passage from the new legislation -

        "The Secretary shall, at the request of the sponsor of a drug, expedite the development and review of such drug if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. (In this section, such a drug is referred to as a `breakthrough therapy'.)"

        It's beyond reasonable doubt that the complications from preterm birth qualify as "a serious or life-threatening disease or condition".

        It's beyond reasonable doubt that Premiplex therapy, if effective, will demonstrate "substantial improvement over existing therapies" when one considers that the existing therapy for ROP is retinal laser ablation.

        The third eligibility criterion for a "breakthrough therapy" designation is "substantial treatment effects observed early in clinical development".

        Bo recently posted the first results from the ongoing Premiplex study in Sweden, from five very pre-term babies treated with Premiplex for just one week -

        "None of the infants developed severe ROP or any degree of cerebral intraventricular hemorrhage, nor any evidence of intracranial hypertension as detected by clinical signs or increased ventricular size at ultrasound examinations."

        That initial phase of the study was intended primarily to demonstrate safety - a small cohort on therapy for a relatively brief period.

        From Insmed's 2011 annual report -

        "We have been informed by Premacure that eight infants have been recruited into the trial as of the end of January 28, 2012 and we understand that Premacure plans to accrue up to 95 patients to get 80 evaluable patients in the trial."

        The primary data collection point occurs at the point when the baby would have reached full-term had the baby not been born pre-term. The recent update to the clinical trial record, extending the Estimated Primary Completion Date to December 2013, would seem to suggest that Premacure now has data on multi-week therapy for at least fifty babies.

        From the Bloomberg article -

        Breakthrough status means the companies will have closer communication with top FDA staff to move drugs for serious diseases to market more quickly, potentially with data from an expanded Phase 1 trial, Woodcock said. Improved understanding of diseases is leading to more candidates for this status.

        "We expect many of these would come available very quickly with Phase 1 data," Woodcock said."

        Can anybody here suggest a good reason why Premacure would not apply for a "breakthrough therapy" designation provided the overwhelming majority of the babies treated since that initial short-therapy phase have evidenced complication-free development of eyes, brain, heart, lungs etc?

 
INSM
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