PER 1.27% 8.0¢ percheron therapeutics limited

Is Capricor cap1002 a competitor to Avicursen re Dmd, page-9

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    Here's another interesting bit comparing us to CAP. This is a statement from Capricor's Principal Investigator, Dr. Craig McDonald. He says he doesn't see a pathway to CAP1002 ever being dosed in a home-setting. It will always be dosed in a hospital or an infusion center due to the risk of allergic reaction:

    At the 52 minute mark:
    Pat Furlong: Craig as you well know some of the folks that are currently taking advantage of an antisense drug are often dosed at home on antisense, using home health nurses, do you foresee that this could get there as well?

    Dr. Craig McDonald: Yeah I think that, I I think given just the theoretic risk of an allergic reaction to DMSO, which is the preservative in the therapeutic, you know, the need for the pre-treatment regiment with H2 blockers, H1 blockers, and the steroids, I think we're we're a ways off before we would feel comfortable enough for a patient to actually get homeinfusion. There's, you know, there's usually from the DMSO, patients will get a little bit of metallic taste in their mouth, the nurses are monitoring patients, you know, very carefully for any signs of anallergic reaction. And, you know, if you did have an allergic reaction, you know, you may have to treat that with, you know, some some degree of medical intervention. You know, it's sort of sort of like an allergy to a bee sting or something like that, and so I I don't necessarily foresee that we'd be able to rely completely on home infusions. But fortunately, this is only once every three months, so the the burden on the families, you know, is not, you know, to the same degree as the currently FDA approved Exxon skipping drugs.

    So that is certainly a comparative advantage for PER, which can be dosed at home. CAP1002 must be dosed in a hospital, and the boys come in in the morning and leave mid-afternoon, and are treated with high dose steroids and H2 and H1 blockers to prevent an allergic reaction from CAP1002. And the risk of allergic reaction is a risk that could give the FDA or EMA pause in approving CAP1002.
 
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