• 12-week Phase I/II clinical trial to treat Rett Syndrome with NTI164 broad spectrum cannabinoid drug therapy extended to 52 weeks
  • All 14 female patients to receive NTI164 under care of Associate Professor Carolyn Ellaway
  • Phase I/II 12 week data expected late Q1 to earl Q2 CY2024
  • The Rett Syndrome market is estimated at US$2 billion per annum


Special Report: Health tech business Neurotech International has extended its Phase I/II clinical trial to explore the usage of its lead NTI164 compound to treat kids with Rhett Syndrome.

After recently announcing approval and clearance from the Human Research Ethics Committee (HREC) and Therapeutic Goods Administration (TGA) respectively for a 12-week program, Neurotech International (ASX:NTI) has now received an additional HREC to allow paitents to continue to receive NTI164 beyond the initial 12 weeks of the clinical trial for a total of 52 weeks under the supervision of the ead investigator of the clinical trial, Professor Carolyn Ellaway.

NTI164 is a compound derived from a unique cannabis strain with low THC. The cannabinoids include CBDA, CBC, CBDP, CBDB and CBN.

Pre-clinical studies have demonstrated its efficacy and have been approved for clinical trials for a range of neurological studies in children – and Rett Syndrome has been targeted for NTI164’s application. Neurotech has already demonstrated significant clinical data in paediatric patients with autism and PANDAS/PANS.  Rett Syndrome will be the third clinical trial to shortly report results.

NTI164 has been exclusively licenced for neurological applications globally.


Trial extension

All 14 Rett Syndrome patients are female and will have the biotech company’s NTI164 drug treatment quadrupled from 12 weeks to 52 weeks under its Phase I/II clinical trial to investigate its use and efficacy.

Neurotech executive director Dr Thomas Duthy says 52 weeks of daily oral treatment with NTI164 was approved by Westmead’s Human Research Ethics Committee under the clinical trial protocol.

“It is certainly pleasing that under the supervision and care of Associate Professor Carolyn Ellaway at The Children’s Hospital at Westmead that all patients and their families have elected to continue treatment with NTI164 for a period of one year,” Duthy says.

“We eagerly await the clinical findings from this world-first cannabinoid drug therapy trial in Rett Syndrome over the coming weeks.

“Rett Syndrome is a rare genetic neurological and developmental disorder and is almost exclusively the result of a mutation(s) in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.

“Rett Syndrome occurs almost exclusively in girls, with an incidence of one in 10,000 female live births. The prevalence is approximately 15,000 girls and women in the US and 350,000 globally.

“The market is estimated at over US$2 billion annually.”


This article was developed in collaboration with Neurotech, 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.