NTI - last patient dosed for Phase II/III Autism trial

Today, our biotech Investment, Neurotech International (ASX: NTI) announced that the 54th and last patient had completed their last visit.

Which means that results should be due soon - NTI said today that they would come “early Q2 2024”.

As a reminder, NTI has TWO clinical trial results to come this quarter, the Rett Syndrome Phase I/II trial and the trial discussed today, an Autism Spectrum Disorder (ASD) Phase II/III trial.

Positive results from the Rett Syndrome trial may act as a major catalyst - given FDA approval for Rett Syndrome helped light a fuse under the Neuren Pharmaceuticals share price - it helped re-rate the stock ~2000%.

Meanwhile ASD clinical trial results could also be a major catalyst, given the dearth of effective treatments available to clinicians and caregivers.

Autism Spectrum Disorder (ASD) is estimated to affect around 1 in 100 children around the world.

Today’s NTI announcement referenced data that suggests - a prevalence rate of 1 in 25 for 7-14-year-olds with a primary diagnosis of autism in Australia, which is among the highest rate in the world

In Australia, 35% of the ~610,000 National Disability Insurance Scheme (NDIS) participants have ASD, and 78% are under 18 years old.

If NTI’s treatment is proven safe and effective for ASD, we think it could become an important part of the overall care for ASD sufferers, ease the burden on both carers and government programs, and as a result, make NTI’s treatment commercially attractive.

We see NTI’s ASD trial results as potentially opening up a large Total Addressable Market (TAM) commercialisation opportunity in Australia - with the rest of the world, in particular the lucrative US healthcare market to follow subsequently.

What’s next for NTI?

The two clinical trial results.

This is what we’re looking for:

🔄 Rett Syndrome Phase I/II trial -

• Bull case: NTI’s Rett Syndrome results demonstrate efficacy better than Neuren’s results, with no safety issues.
• Base case: NTI’s Rett Syndrome results demonstrate efficacy to around the same standard as Neuren’s results, with no safety issues.
• Bear case: NTI’s Rett Syndrome results don’t demonstrate efficacy to the standard of Neuren’s results, and/or there are safety issues.

🔄 Autism Spectrum Disorder (ASD) Phase II/III trial

• Bull case: NTI’s Phase II/III ASD results are broadly in line with its Phase I/II ASD results.
• Bear case: NTI’s Phase II/III ASD results are worse than its Phase I/II ASD results.