We have discussed previously the lymphocyte data and the importance of targeting lymphocyte migration into the muscle tissue. Professor Voit addressed the data and made a number of profound observations of clinical importance. When asked whether the down-regulation represented by the median change from baseline for immune cells was the right level following treatment with ATL1102, Voit said that it was in a good ball park range.
Yes. Yes. Yes.
Hark back to the disappointment when the FDA cleared ANP to proceed with a Phase 2b trial in MS subject to the trial restriction that infusions would be limited to 25 mg per week. It seemed like the kiss of death not only for MS but for demonstrating efficacy in DMD. Did the FDA themselves demonstrate profound insight? If not the right dosage we are almost certainly in the right ball park.
The reasons of course go the very heart of the amazingly brilliant working of the immune system complete with its checks and balances. As Voit said, you don't want to completely immobilise it just dampen the over-reaction i.e. the Goldilocks dose to bind to just enough CD49d to modulate the migration of lymphocytes from the blood to the muscle inflammation sites.
And that was only one point that he made. If its not lost on HC followers, guarantee it won't be lost on PPMD, Sarepta or any number of biotech and pharmaceutical groups who understand the significance of the mechanism of action for ATL1102. Yes, it is lost on the ASX but in the end they'll play catch up.
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- Prof Thomas Voit, DMD expert, interview with Wilsons advisory
Prof Thomas Voit, DMD expert, interview with Wilsons advisory, page-11
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