Ann: Investor presentation, 2 February 2017, page-4

Having read this presentation I am left wondering about a particular question that I posed many months ago on this forum which no answer was forthcoming. If none are readily available again, it is probably time for me to ask this question to the right person at NEU. Is there anyone who could suggest a name to ask the following -

Why does NEU have to prove that Trofinetide is curative of a particular disease to allow commercialisation?

As the presentation outlines again Trofinetide has -

“…a broad range of clinical effects consistent with known normalising actions on brain function.”

These include -

1. Inhibits neuroinflammation
2. Normalises function of microglia
3. Normalises inter-neuronal communication


My query revolves around the medications such as paracetamol which decrease pain and temperature by its physiological effect of the active ingredient.
Similarly Aspirin reduces fever, reduces pain, helps with inflammation and also help reduce arterial blockages.

However, I can’t think of any disease that these medications cure.

I’m not sure by what avenue they received FDA approval, however I can only imagine it would have been by showing its consistent and reproducible physiological effects.

So why can’t Trofinetide be approved purely via it's recognised physiological effect as a neuroinflammatory reducer?
If it is shown to reduce inflammation in neural tissue and neural inflammation is known to be pathological then why can’t it be prescribed as the ‘aspirin of neural tissue’
You might argue that reducing inflammation of neural tissue is not curative, I’d argue it's physiological effect is enough, but even if not being curative is enough, well neither is panadol or aspirin.
So, as I said who could I pose this question to at NEU?