Neuren Media and Analyst Coverage, page-1709

Although I was always aware that NNZ-2591 regulates the level of IGF-1, because everything I read always referred to the deficiency in IGF-1 levels observed in ASD, particularly in younger children, I only ever thought about NNZ-2591's benefit through increasing IGF-1 kevels.

I have never read before the suggestion that IGF-1 levels are excessive in the autistic population in that first year or so of life and that this is what might cause the “irreversible structural connectivity abnormalities specific to ASD”. I’m interested to know, has anyone else?

Because, if this is the case, given that IGF-1 levels can be measured through a blood test, wouldn’t reducing IGF-1 to normal levels during that first year or so of life make sense as a way to avoid incurring those irreversible abnormalities?

And if NNZ-2591 can effectively either dial up or down IGF-1 levels (that is, provide a “Goldilocks dose”, wouldn’t it make a prime candidate for treating ASD at this early stage?

Obviously, we are talking about patient numbers here well beyond an orphan indication. For example, it is estimated that ~ 100,000 children with autism were born in the United States last year. While that is beyond Neuren’s focus and capability, it could certainly be of great interest to large pharma.