danleeg's  Instablog

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Pharmacist... Investing is my favorite hobby.
  • Gene Therapy Is Knocking At The FDA's Door 0 comments
    Aug 9, 2014 5:42 PM | about stocks: EIGR, KITE, NVS

    I still do a double-take when I review the few that are lucky enough to make the FDA's breakthrough therapy designation status. The list is impressive. So far that list has produced some serious treasure:

    Sovaldi, Kalydeco, Imbruvica, Zydelig

    Two on the list intrigue me the most:

    Celladon's Mydicar & CTL019 from Novartis. Both are essentially gene therapies & either one could change medicine forever.

    CLDN is eye-rubbing. Everything I can read shows it's miraculously effective (albeit n is understandably small). Yet it muddles around with a market cap of less than $200 million. CLDN isn't exactly swimming in cash right now, but JNJ, PFE, & NVS have fed this infant before, and I think they will do it again if CLDN gets thirsty.

    At 12 months, high dose Mydicar showed an 88% reduction of "major cardiovascular events such as death, need for LVAD or cardiac transplant, episodes of worsening heart failure and number of heart failure-related hospitalizations"

    2 more years after that? ... 82% reduction. At three years post-administration, there were six deaths in the placebo group and only 1 in the high-dose group. No significant side-effects & no safety concerns were noted during the three year follow-up period.

    (Ben Stein says "WOW").

    I feel Novartis is too big for CTL019 to move the needle. I'm holding on to some KITE instead. When CTL019 is approved, my KITE will fly.

    I suspect KITE & BLUE will join the FDA's list in due time.

    Disclosure: The author is long CLDN, KITE, GILD, BLUE.

    Stocks: EIGR, KITE, NVS
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