Agreed KJK with this post and the former - very much looking forward to your analysis on HIE and have been waiting for baited breath for your DCF now that we know the parameters of the P3 PMS trial.
Not sure if you have any views on this (or whether the forum at large does) but I have been thinking about the nature of HIE in comparison to PMS etc.
Given the recurrent prevalence (6,000 per annum in the USA alone), it may be possible that running a trial for HIE is significantly easier and faster than for our current conditions. Unlike with our current conditions that require genetic testing and/or complex screening and diagnostic processes to simply identify patients, HIE seems to be much easier to identify and much more prevalent. You could (theoretically) identify the top 100-200 hospitals in the USA where there are high incidences of children born with HIE and have arrangements ready to go so that parents could provide consent quickly for participants to enter the trial and have the drug available on site. Given the advantages of dose size and lack of cold storage that 2591 has over Trofinetide as an example, this would be possible.
That being the case, you could imagine a scenario where once all the traditional "barriers" are removed (or lowered), you could really move the trial quite quickly and if it is a 13 week trial like PMS, with similar patient numbers, then I think it is possible that the trial takes closer to 1 year and not 2, to complete.
I could be wrong and missing nuance with the trial process that won't speed it up.
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Agreed KJK with this post and the former - very much looking...
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