Neuren Media and Analyst Coverage, page-1229

The additional indications Neuren will be pursuing is an interesting question. As I have mentioned in previous posts, neurodevelopmental disorders are increasingly regarded as lying on a spectrum or continuum, which is why they are often times difficult to diagnose resulting in children and parents enduring a years long diagnostic odyssey before receiving a diagnosis, which even then may not be correct.

For this reason, with the rapid advancement of genomic and genetic testing technologies, diagnosis is shifting from phenotypic assessment towards genomic and molecular diagnosis. An interesting paper here on the evolution of NDD diagnosis for anyone interested -

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7365295/

For these reasons, all of Neuren's chosen indications to date have a known genetic aetiology which can be identified by molecular testing. This has the added advantage of enabling preclinical studies in animal and in vitro models that recapitulate the genetic defect.

So, NDDs that have a known genetic aetiology, have an intellectual disability element, and a prevalence that sits below the ODD threshold might include:

Di George Syndrome - 1 in 4000 - 6000 births
Cornelia de Lange syndrome - 1 in 10,000 - 50,000 births
Sotos syndrome - 1 in 10,000 - 14,000 births
Smith-Lemli-Opitz syndrome - 1 in 20,000 - 60,000 births
Smith Magenis syndrome - 1 in 15,000 - 25,000 births
Tuberous Sclerosis with AHD - 1 in 6,000 - 12,000 births
CHD7 CHARGE - 1 in 8,000 - 15,000 births

This list is obviously far from exhaustive. As an aside, when universal newborn whole genome sequencing is introduced the diagnostic rate for all of these NDDs will increase significantly. This has important implications for the market sizes of Daybue and NNZ-2591.

Apologies if this comes out in a weird font - some issues with HC posting so had to copy and paste