Good sign: NTI’s Rett Syndrome Trial Gets Extended

In a positive update, our biotech Investment Neurotech International (ASX: NTI) revealed that the company’s Phase I/II clinical trial on Rett Syndrome has been extended for an additional 52 weeks.

All 14 patients in the trial will remain on NTI’s biopharmaceutical NTI164 for this time period, which we interpret as a potential leading signal of the efficacy and safety of the treatment.

The reasoning here is straightforward, parents caring for children with the rare paediatric neurological condition (Rett Syndrome) may have seen positive results from the treatment, and felt comfortable with their children’s toleration of the treatment.

The Human Research Ethics Committee also had to sign off on the extension.

Of course however, we must wait for the clinical trial results to get a clearer picture on the safety and efficacy of NTI’s treatment for Rett Syndrome, which are just around the corner.

Importantly, this is not the first time NTI has had one of its clinical trials extended.

NTI has now had the following trials extended:

✅TODAY: Autism Spectrum Disorder (ASD) Phase II/III trial - results due in “coming weeks”

✅Autism Spectrum Disorder (ASD) Phase I/II trial - now going for 90 weeks

✅Phase I/II PANDAS/PANS - strong data at 24 weeks

That’s a very good pattern for NTI to establish.

We have high hopes that NTI may be able to improve upon the safety profile of Neuren Pharmaceuticals Rett Syndrome treatment - something we covered in our latest NTI note.

The results shape as a major catalyst for NTI, we see a lot of momentum in NTI’s clinical trial program, especially after NTI announced that it intends to start a Phase I/II trial for Cerebral Palsy in the first half of this year as well.

Bottom line for us: NTI has kicked a lot of goals in a short period of time, and we think the company has plenty more “shots on goal” to come.

What’s next for NTI?

Phase I/II Rett Syndrome clinical trial due in “coming weeks” 🔄

We are hoping NTI can show that its treatment is safer and has similiar (we hope better) efficacy to $2.6BN capped Neuren’s Daybue treatment.

Phase II/III trial for Autism Spectrum Disorder (results in Q1/Early Q2 2024) 🔄

After promising Phase I/II trial results, we see this trial as important from a commercialisation perspective.