I have just come across this paper:This is really interesting as...

I have just come across this paper:

This is really interesting as the FDA provided advice for the IXC phase III trial to adopt a clinical endpoint (headache/eye sight) as a primary end point rather than CSF/intracranial pressure measurements (which was what UK/EU/AU regulators wanted and what IXC management decided was the least risk approach to approval).

This paper below confirms that there is a strong correlation between heightened CSF readings and headache days. Therefore, if Presendin reduces CSF pressure, the MOA will almost certainly reduce headache days over a larger Phase III sample, as it did in Phase II.

If this occurs with undeniable statistical strength, alongside papers defining CSF reduction's strong correlation with lowered headache days, alongside US trial centres, I wonder if the FDA will come around to the idea of approving it without the need for a second phase III trial.

Would love to know others thoughts on this - I think we may have to include a % likelihood of a surprise to the upside in form of FDA approving without a second trial, and its hard to see that as 0% for a repurposed drug, for an orphan indication, with papers showing a CSF endpoint indicating clear correlation to a clinical endpoint benefitting patients quality of life. Maybe 15-20% likelihood?


https://pubmed.ncbi.nlm.nih.gov/34620087/




AND THE KEY POINT: