I have often wondered this exact thing and was talking to someone about it recently. It would make sense to stick with the Orphan indications initially (given pricing challenges) and then as further data and proof comes in over time, alongside some anecdotal stories from off-label treatments, look to run a broader trial for "Autism" and all the unique aspects that fall within that diagnosis.
Running trials for Autism would be a gargantuan task and beyond our capabilities I would think. The size and scale is one thing, the variations in Autism Spectrum Disorders is another altogether and trying to manage that would require a company with significantly more resources and expertise - in my opinion.
I think your statement about PMS, PHS, AMS and PWS being the "easier paths" to commercialisation is spot on, in that they serve as a proof of concept for a justified crack at Autism as a whole. I came across some information recently that would support that view, wherein the conditions we are currently trialling are not considered Autism Spectrum Disorders but are categorised as Autism Related Disorders. I am not a doctor and don't know if that is a medically valid distinction but the point is made that each of the genetic conditions have symptoms commonly seen in Autism, alongside their own condition specific symptoms -
https://autism.org/related-disorders/#williams-syndrome.
It would stand to reason that if you can treat the Related Disorders, you are a good chance of being able to treat the Spectrum Disorders and there are an enormous and rapidly increasing number of people with them. In 200, 6.7 in 1,000 children were diagnosed with ASD, which rose to 27.6 in 1,000 children by 2020. This means that currently, 1 in 36 children in the U.S. get diagnosed with ASD.
If you take a step back and really consider the implications of that, it really is remarkable - and those figures don't include the ROW prevalence, which are not quite as high as I understand it, but trending in that direction.
It therefore won't surprise to learn that I very much agree with Sillazze's comments above about Autism being the end game here and
THE significant value driver. It really is remarkable to contemplate the possibility that ASD could be the significant value driver here, when considering the value that KJT and others have shown that can be derived from just two of the 4 conditions we are trailing/treating.
I could be totally wrong, as could other posters, but I just have a feeling that this drug is very special, a once in a generation drug and management know it. It's not the drug you sell for a quick 200% profit. This is very possibly the Meta/Google/Apple equivalent in the pharmaceutical world, that you hold onto long term, because you know the true value and understand that it changes the landscape and has a commensurate value.
(20min delay)