Okay, these are my ‘first look’ thoughts regarding the presentation and the supporting data.
I believe that most neuroscientists at the presentation would agree that the data is most compelling. In this study (201) and in ‘early’ patients in the interim 202 figures, it is clear to anyone, that the drug works. I would love to know what questions flowed from the floor and I might ask Stamler or Claassen when I can (perhaps next week when David is here in Oz).
It is clear that ATH434 redistributes excess labile iron ... so reduces oxidative injury along with a reduction in a-syn. This is big and suggests to me it will be effective in other areas of PD.
Slide 8 - The UMSARS1 results are compelling, and I suspect this will carry considerable weight with the FDA.
Slide 9 – showing positive changes to Clinical Global Impression of Severity is also compelling.
Slide 10 – OHSA is super. Again, FDA should love this.
Slide 11 – Wearable sensors, while very positive, it still asks several questions that I’m sure the FDA would like answered. Why the ‘interesting’ differences between the 50mg and 75mg groups? To be honest, I would really like them answered too. I have my own theory, and I’m sure David is working hard to clarify.
A new analyses of neuroimaging data show target engagement of ATH434 on iron levels in MSA affected regions of the brain. Additional powerful proof for the FDA, so very positive.
And then there is slide 13. Clearly demonstrates that ATH434 has slows brain atrophy ... a big one, but again asks several questions.
I would expect that the presentation would have excited the science community, but perhaps not the average punter.
Personally, if Stamler can explain to the FDA why we are seeing differences in the 50 and 75 groups, then we could hear some very, very exciting feedback from the authority. If this comes, it may be quickly followed by a partnership or ...
Interesting times. The upcoming FDA meeting will be a big one. Fingers and toes crossed.
Of course, just the opinion of one poster.
(20min delay)