In the orphan drug meeting in Boston Dr. Pall will talk about...

In the orphan drug meeting in Boston Dr. Pall will talk about "Biophysical Characteristics of ATH434, a Unique Iron-Targeting Drug for Treating
Friedreich’s Ataxia".
I would think that the presentation is mostly about biophysical characteristics and not yet anything related that it would be tested in FA patients, but perhaps in the FA cells of these patients.
It is an important study also regarding other degenerative diseases but at this phase "not price sensitive".

It is not evident that ATH will give any "price sensitive" announcements in any scientific meeting. IMO the open study results will be announced as separate announcements. The first open-study patient was recruited 11 months ago but it took until February to get all 15 patients (?). So it is impossible to get all the results before the randomized study which ends in November. IMO, we will get the 6-month results in June but only for the first 10 (?)patients. This would be enough to see the trend if ATH434 works in these patients. There will be an MRI scan after 6 months. So, we get an answer if ATH434 reduces iron content in the brain as in experimental MSA and PD studies. Iron content is the "primary outcome measure" in both studies, so I think this is what we are waiting for.

They already have the collected blood and spinal fluid analyses as well as questionnaire results from many patients but when the primary outcome measure is still missing, they wait before they can be sure that the trend is seen. ATH434 is a "Unique Iron-Targeting drug" with special biophysical characteristics as Dr. Pall will tell us next week.