Ann: Percheron appoints Dr Cathryn Clary as Chief Medical Advisor, page-62

@waynesworld

Hi Wayne,

I believe they will not do MS. Too much competition now. Also, and very importantly, ATL1102 will sell for US$300k for DMD. The price for ATL1102 for MS patients would not be like DMD, approximately $40k. DMD patients would buy it ‘off label’ and get the MS price. Impossible to police.
In tying in with the above, muscular dystrophies are where the focus will be. DMD, Becker, LGMD.. they can move straight into late stage trials with the same trial timelines for DMD. The price for the drug will be the same. Huge margins, similar clinicians, similar advocacy groups/strategies.

What does the new CMO know?? She knows what we all know but is waiting for it to be confirmed in trial results like all of us. As you mentioned, the science is settled. Some further anecdotal evidence to share.

On the trial. I have heard from both previous and current management on these matters.Firstly.. Dr Voit said there will be zero placebo effect in this trial. The non ambulant boys muscles are too far gone and no amount of hope will make them more functional. Some have 70% fat in the legs, 30% muscle. After hearing this i had a look at Capricor’s trial results in Non Ambulant boys.. zero placebo effect.

Post the PH2a.. the principal investigator told Diamond that he could ‘visibly see the boys deteriorate after they stopped the treatment of ATL1102. This is going to be a key bit of data to show the regulator. Good luck punters