Ann: Positive Interim Data from ATH434-202 Phase 2 Clinical Trial, page-45

This is promising ...

Well, these results have certainly brought a smile to my face.

1. It’s safe. No SAEs. More important than anything. Just ask the FDA.

2. Yes, it is a small sample. This is to be expected because of the challenging nature of tests each patient and their family must undertake. Not a walk in the park.

3. MSA is a SoaB ... awful, and if you receive this diagnosis, you are only going one way ... Or are you? Historically you are on a very slippery slope but here we have almost 30% of the patients that have stabilised or improved. Seriously? I haven’t seen data like this before and I will be surprised if anyone here can show me any. This trial gives more than a little hope for sufferers.

4. The Clinical responders showed reduced iron accumulation in three critical areas of the brain. Again, I have not seen this before in a clinical trial, not to mention stable NFL (so, no further axonal injury in neurons). It seems we have a very effective little iron chelator here.

5. The “clinical responders, brain volume assessed by the MSA-AI was stable between Month 6 and Month 12”. Personally, I wasn’t counting on seeing this but we have it.

6. The Clinical Responders were those with less disease, so what might we see with the 201 trial? ATH434-201 has enrolled patients with early-stage MSA. Think about this ... it looks like ATH434 will work better the earlier you commence the treatment. Nice.

7. The selected bio-markers and clinical endpoints seem to be right on the money, thus giving us a much greater chance of success in the randomised, double-blind, placebo-controlled 201 trial. The FDA will like this. Big Pharma likewise.

So many aspects of these data point to the fact that ATH434 is working with MSA sufferers ... certainly if we catch them early enough.

What does this mean? Well, if these results are reflected in the ATH434-201 trial we may be sitting on a breakthrough neurological treatment. What will that be worth? I’ll let others play with those numbers.

IMO, this is a wonderful result. Others will have to make up their own mind. Enough from me for while.

As always, opinion only.