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Insmed Incorporated Message Board

  • fudfighter3 fudfighter3 Mar 18, 2014 1:03 PM Flag

    TV program last night on MDR-TB


    This was a ninety minute program - TB:Return of the Plague.

    "A deadly airborne disease is making a dramatic comeback. Passed on by a cough or a sneeze, TB is travelling fast."

    They claimed around half a million people a year are currently developing MDR-TB.

    I only watched the first half and hour or so, which focussed on a young girl (aged about 8 I think) in Africa who had just been diagnosed with MDR-TB.

    Started off with a health care lady breaking the news to her that, to protect her family, she would have to spend the next two years in a TB hospital.

    At the hospital she was told that the first six months of treatment would include a daily injection in the buttocks. She was warned about the danger of side effects - the most serious of which is that some patients lose their hearing. They told her to immediately report any ringing in the ears.

    An older girl who'd been there a while had been constantly sick from the tablets, to such an extent that her treatment had to be stopped for a while to allow her to regain weight.

    The tablets used to treat routine cases of TB also cause side effects such as nausea. The program attributed the MDR-TB epidemic to breaks in therapy for routine TB because of the side effects.

    The injections (though it wasn't mentioned) had to have been an aminoglycoside such as amikacin.

    But here WE are - possibly a matter of days away from clinical trial results proving that Arikace can deliver an effective concentration of aminoglycoside to a pulmonary mycobacterial infection (such as MDR-TB) without the risk of hearing loss.

    And we have an analyst suggesting that proof of efficacy may not be sufficient to warrant approval unless some of the patients were CURED by six months of therapy - when the minimum recommended period of therapy for a drug-resistant pulmonary mycobacterial infection is currently two years.

    Priceless :-)

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    • In case Arikace does indeed become available in Canada before the accelerated approval process makes it available in the US - it's worth noting that a 1991 study by the U.S. General Accounting Office found that one-third of all drug administrations to cancer patients were off-label.

      30,000 in the US with Cystic Fibrosis plus 50,000 treated for pulmonary NTM each year would generate substantial off-label use.

      The best antibiotic currently available for CF can only halt the relentless deterioration in lung function for a month or two at most. Data recently released show that Arikace halted deterioration for a year (and counting).

      As for NTM antibiotics, here's an excerpt from Dr. Ken Olivier's presentation at the 2013 European CF conference (thanks Blase) -

      [ M. avium complex may be easier to treat, using daily oral macrolide (preferably azithromycin), rifampin, and ethambutol, for clarithromycin-sensitive M. avium complex. ]

      Infections by Non-Tuberculosis Mycobacteria (such as MAC) and Mycobacterium Tuberculosis are treated with the same antibiotics - those which have evidenced efficacy in killing mycobacteria sheltering within the pulmonary macrophages (which usually digest bacteria).

      Here are the common side-effects -

      Amikacin ..... kidney and ear problems.
      Azithromycin ..... nausea, headaches, vomiting, diarrhea.
      Ciprofloxacin ..... nausea, vomiting, diarrhea.
      Clarithromycin ..... nausea, headaches, vomiting, diarrhea. Note: The maximum dose is 500 milligrams twice a day.
      Ethambutol ..... nausea, vomiting, vision problems.
      Rifabutin ..... rashes, nausea, anemia. Many drug interactions.
      Rifampin ..... fever, chills, muscle or bone pain; can turn urine, sweat, and saliva red-orange (may stain contact lenses); can interfere with birth control pills. Many drug interactions.

      Decide for yourselves the potential of Arikace if it does indeed deliver an effective concentration of amikacin without the systemic toxicity which causes those side-effects.

    • Who is that brilliant analyst for clarification purposes?

      • 1 Reply to blasedp31
      • The DHW has been claiming Leerink said that.

        The truth is it emanated from Apprentice Millionaire Portfolio -

        From Life Sciences — Biotechnology (JackBass team)



        Investment recommendation

        Reiterate BUY, raising PT to $30 on Arikace's increased potential in nontuberculous mycobacteria (NTM).

        INSM's lead drug Arikace is an inhaled liposomal form of potent, FDA-approved antibiotic amikacin. We expect positive data from a Ph2 US NTM trial in March.

        Our higher pNPV-based $30 target is driven by a higher chance of success.

        Investment highlights:

        Based on proprietary analysis/KOL talk, we think INSM's Ph2/3 NTM trial has a good chance of success with late March top-line data. The trial is powered to show a 1-point change on the 7-point semiquantitative scale of NTM infection burden. We think a 1-point benefit in these severe trial patients (whose baseline score we think will be ~5) would correlate with a benefit in QoL and be clinically meaningful.

        More confidence on data variance and noise: interim look gives us comfort on "neatness" of data and powering.

        INSM conducted an interim blinded look at the variance of the data to determine if the Ph2 was adequately powered. The look indicated data dispersion/ variance fell within trial powering assumption. We think it is unlikely INSM was able to see graphical representation of the data dispersion during variance calculation, which could have provided a "tell" on data.

        Unless we see cures in the Ph2/3, we think immediate US filing is unlikely, no matter what KOLs agitate FDA for.

        Based on our KOL conversations, we think FDA still has a lot to learn about NTM, having been previously hung up on TB-like "cure" endpoints. Given trial patients are end of the line, hard to treat, cures with only 3-mo Tx are unlikely.

        INSM may decide to focus initial registration on a subset of patients based on pre-specified stratification ...

25.09+1.70(+7.27%)3:59 PMEDT