NTI’s autism data is still improving…big implications

Our biotech Investment, Neurotech International (ASX: NTI) reported positive secondary endpoint data from its Phase II/III autism trial today.

Children receiving NTI's drug candidate NTI164 showed statistically significant improvements in anxiety, depression and mood at 8 weeks compared to placebo.

We see this as a further encouraging sign that NTI164 could become an important new treatment option for the 40-50% of autistic children who experience clinically significant anxiety, and the 10-20% who suffer from depression.

With this positive 8-week data in hand, we will be keenly awaiting the full results from the 16-week treatment period of the Phase II/III autism trial.

We’ve said before that further positive efficacy and safety data could help position NTI164 for successful regulatory submissions and partnering discussions down the track.

There were two particularly pleasing elements of the results released today:

• NTI164 led to a clinically meaningful 19-point improvement in the Anxiety, Depression and Mood Scale (ADAMS) total score versus placebo at 8 weeks (p<0.001)
• 62% of NTI164 patients were already on existing anxiety/depression medications at trial start, yet still saw added benefits from NTI164

On the first point, that is an exceptionally low p-score (p<0.001), meaning a very strong indication that NTI is having the desired effect for patients and the results can be attributed to NTI’s treatment.

On the second point, and this is particularly interesting, the fact that NTI is helping with anxiety and depression shows a potential added benefit from the treatment.

Namely, that NTI164 could be favoured by regulators for its potential ability to reduce the reliance of large portions of ASD patients on anxiety and depression medications.

It is commonly known that these medications have (in the case of anxiety medications) an association with dependence or (in the case of depression medications) such as SSRI’s, a range of undesirable side effects.

How does this news impact our NTI Investment Memo?

Promising efficacy data at treating Autism, large addressable market
If NTI’s treatment is proven safe and effective for Autism Spectrum Disorder (ASD), we think it could become an important part of the overall care for ASD sufferers, which is 1 in 100 children.

Source: 18 September 2023 NTI Investment Memo

We see this as really strong data, and further supports NTI in its pursuit of a large addressable market opportunity for an autism treatment.

We think regulators will look even more favourably on NTI’s treatment after today's results. There are currently no approved treatments for ASD, in Australia, the NDIS is haemorrhaging billions of dollars on autism, and off-label treatments for ASD have poor side effect profiles.

What’s next for NTI?

• FDA response on Orphan Drug Designation expected in ~3 months
• Advancing NTI164 through clinical pipeline across multiple rare neurological disorder indications (including Cerebral Palsy)

For a full rundown of what we want to see next from NTI, read our latest note:

NTI’s trial of Autism treatment in children shows further patient improvement after 12 weeks