I wonder how successful you can be if you're the second drug to market for an orphan disease?
I would assume insurance companys wouldnt be willing to fund someone using 2 drugs at the same time. You'd think all things being equal patients would stick with the drug they know if it's working - you'd be reluctant to take the risk on something new I'd think.
Therefore, I'd think the second drug to market could only succeed if either:
1. The first drug (Trof) didn't help, or had unacceptable side effects. You'd say 'that didn't work well, I'm happy to try something new'.
2. Or if the first drug is working well, then the new drug would have to offer a substantial improvement over Trof before you'd risk it, so the high bar for Trof has now been raised even further for success.
3. The new drug is substantially cheaper and so the insurance companies put lots of pressure on carers or patients to swap, even if second drug is not as good. I suspect there are protections to prevent insureres from doing this tho.
The above reasoning would suggest that the first mover advantage Neuren has is enormous. And if Trof works really well (and shows more improvement the longer you take it) then it's lead would become almost unassailable.
At the very least it will have raised the bar substantially. You not only have to clear the FDA criteria, you'll also have to do it with BETTER results than Neuren.
And of course, Neuren is still the only drug to pass a Ph 3 neurodedevelopment trial ever, so this suggests it's highly unlikely most of the current pipeline drugs will pass either.
Finally, now there is an approved and successful drug for Retts the investment case for new drug development in Retts must inevitably become weaker. Your forecasts now have to assume you will be sharing the market with Neuren, and there's a possibility you could pass FDA approval, but still end up with a small fraction of the market, even if you slash prices. Will it still be enough to justify the investment? Note that none of Neurens market share revenue forecasts to date have had to include a discount factor for market share.
In summary, I wonder if theres a good possibility, particularly if Trof and NNZ-2591 work well, that Neuren may end up with this whole market pretty much to themselves for the foreseeable future (or at least until gene therapy provides a final cure one dayl.
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