It does appear that mixing orphan indications with non-orphan ones is not a good idea. This would indeed mean that considering scenarios where NNZ-2591 could be licensed out or sold for development in non-orphan disorders would not make sense.
However, I don't think this weakens the licensing vs 'go it alone' vs TO arguments, and in fact it probably strengthens them.
Firstly, if developing NNZ-2591 for non-orphan disorders is a bad move for Neuren, then it's also likely be a bad move for anyone who buys Neuren. If there isn't a good value opportunity in letting a licensing partner develop NNZ-2591 for neurodegenerative disorders then this value opportunity won't exist in a TO scenario either.
The reasons why many appear to be arguing for a TO by a large Pharma seem to be :
1. Neuren doesn't have the man-power to develop the drug for a huge number of indications.
2. Neuren doesn't have the infrastructure to market and sell at scale.
3. Neuren doesn't have enough capital to fund all the above.
If we were talking about non-ophan diseases such as Autism, Alzheimers, TBI, Parkinsons, etc then the above challenges are indeed enormous. The costs and size of trials required for non-ophan diseases are much higher, and the infrastructure needed to market and support a drug with millions of patients would be huge too, not something you could build overnight from just 20 staff.
But if we're only talking about orphan diseases now the story is different. Orphan drug trials are significantly shorter and lower cost. Marketing to and supporting an orphan disease with just 5,000 to 15,000 patients will be orders of magnitude easier too. And who says you can't employ a 3rd party to do this?
Finally, a TO is not the only way to solve the funding challenge. That's exactly where partners like Blackstone come in, or a big up front to a licensing partner to develop NNZ-2591 for other disorders (such as the dozen or so hottod has just listed), or both.
I think the 'go-it-alone' scenario is a red-herring. If by go-it-alone you mean that Neuren would do all the drug trials themselves, take the drugs to market themselves, and also fund everything themselves from existing cashflow, then you're right, that probably isn't sensible.
But I don't think that is something Neuren is even considering. Everything currently point to Neuren doing a hybrid model - using some of their own funds, supplemented with external funding or additional licensing fees. Doing some trials completely themselves, licensing some indications out to others. Then using a variety of partners to market and sell the product after approval, or to complete approval in other markets.
They got US$100m up front from Acadia recently just for ROW + a quid-pro-quo on Retts/NNZ-2591. In 3 years Neuren might get $1 billion from Acadia for a licensing an FDA approved treatment for Phelan McDermid (plus $billions in ongoing royalties and milestone payments).
I'm more and more convinced this is the path Neuren is considering. And there's no question in my mind this path would maximise long term shareholder value. Acadia analysts are still forecasting Acadia to reach peak sales of US$1 billion for Retts. Peak sales for the current 4 indications could be $5 billion (or $10 billion - refer my other post about the comparative market size vs Retts)
I must admit that after 8+ years holding Neuren the simplicity of a single big payout that forces me to finally take all my money off the table does have attraction.
. But still doesn't mean its the outcome for either shareholders or patients.
(20min delay)