• Neurotech’s NTIRTT1 becomes first clinical trial to show statistically significant clinical improvement in Rett Syndrome patients with a broad-spectrum cannabinoid drug
  • Company’s NTI164 compares favourably to DAYBUE, the only FDA-approved drug for Rett Syndrome
  • Third neurological disorder in children where NTI164 has shown a statistically significant clinical benefit, alongside autism and PANDAS/PANS


Special Report: Neurotech International has reported positive top-line Phase 1/2 Rett Syndrome clinical trial results with its NTI164 being the first broad-spectrum cannabinoid drug therapy to show a statistically significant clinical improvement in Rett Syndrome patients.

Clinical-stage biotech Neurotech International (ASX:NTI) has reached a milestone, reporting the initial findings of its Phase 1/2 clinical trial (NTIRTT1), showcasing the efficacy and safety outcomes of NTI164 in treating Rett Syndrome, the treatment of which is a US$2bn market.

The trial involved 14 female paediatric patients who underwent a 12-week daily oral treatment regimen with NTI164.

Results indicate a statistically significant improvement in Clinical Global Impression

(CGI) scores, a key measure of clinical symptoms associated with Rett Syndrome, compared to baseline measures.

The CGI scale is a tool used by clinicians to assess the overall state of an individual, commonly used in clinical trials for neuropsychiatric disorders.

CGI-Improvement (CGI-I) is a component of this scale, employing a 7-point rating system to gauge the degree of improvement or worsening of a patient’s condition compared to a baseline state.

Initial primary endpoint analysis has shown a statistically significant improvement in CGI-1 at 12 weeks versus baseline measures with a mean difference of -0.3 (p=0.04). A decrease in CGI-I score indicates improvement.

NTI’s latest results demonstrate Rett Syndrome is the third neurological disorder in children where NTI164 has shown a statistically significant clinical benefit, alongside autism and PANDAS/PANS.

The company is focused predominately on paediatric neurological disorders.


Following in the footsteps of Neuren

NTI is following in the footsteps of Neuren Pharmaceuticals (ASX:NEU) which became the 2023 ASX biotech market darling after its large pharma partner Acadia (NASDAQ:ACAD) announced that the US FDA had approved trofinetide for the treatment of Rett syndrome.

Now dubbed DAYBUE, trofinetide is the first approved treatment for Rett syndrome with its official launch in the US by Acadia in April 2023 sending NEU’s share price soaring.

NTI says in the Phase 3 trial called LAVENDAR of trofinetide CGI-I served as a co-primary endpoint.

Results showed a CGI-I improvement of -0.3 (8% improvement) in the trofinetide arm compared to placebo (p=0.003) with NTI164 comparing favourably to the only FDA-approved treatment.

All 14 girls taking part in the trial will take part in the extended study of 52 weeks.


Need for more Rett treatments

NTI executive director Dr Thomas Duthy says Rett Syndrome is a rare genetic neurological and developmental disorder and is almost exclusively the result of a mutation or mutations in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.

Duthy says Rett Syndrome occurs almost exclusively in girls, with an incidence of one in 10,000 female live births with the prevalence ~15,000 girls and women in the US and 350,000 globally.

He says the Rett Syndrome market is estimated at more than US$2bn annually.

“We warmly congratulate our principal investigator Associate Professor Carolyn Ellaway and her Rett Syndrome patients and families who took part in the trial,” he says.

Ellaway is the senior staff specialist NSW Genetic Metabolic Disorders Service, the Sydney Children’s Hospital Network and Metabolic Genetics at The Children’s Hospital at Westmead.

“There remains an urgent need for more safe and effective therapies in Rett Syndrome,” Duthy said.

“Data analysis and interpretation continues, and we are very much looking forward to finalising and reporting further detailed clinical findings over the next two to four weeks and thereafter presenting this data at a major scientific meeting.”



This article was developed in collaboration with Neurotech International, a Stockhead advertiser at the time of publishing.  

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.