I did just speak to RH on the Phase 3 limitations.
There are many variations and complexities, including the following
1/ trial length is not set and depends on several factors, the main one as far as I could determine is the rate of decline in the natural history population. The faster they decline on average, the shorter the trial can be. So there’s a 1 or 2 year trial length implication straight away.
2/ There is also the double blinding nature of the trial, as you cant unblind the trial just because you want to, and whether the trial is in Europe or the US makes a difference as well, especially with overlap options.
3/ it sounds as if they have worked out the end points but they went over my head. But at least they are sorted. Mostly clinical.
the phase 3 trial may be able to start early if PYC get efficacy readouts on either of the 30ug or 90 ug cohorts on the Phase 1 trial, and we would know that some approx say 6 months after those dosings, possibly.
so, in summary, it’s complicated. But he does think revenue in late 26 is reasonable chance, all things considered.
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