Neurotech applies for ethics approval to trial cannabis as a treatment for Rett Syndrome

About one in 9,000 females in Australia are diagnosed with Rett Syndrome at birth. In the US, about 15,000 girls and women suffer from it, and globally, this number grows to 350,000 females. The significance of its prevalence is nothing compared to its impact. The disorder can have a detrimental effect on the lives of the affected individuals and families, and right now, only one treatment exists.

Rett Syndrome is a rare genetic disorder that affects brain development, primarily in females. It is caused by a mutation of the MECP2 gene and can result in a range of symptoms, including loss of speech and hand use, breathing difficulties, and repetitive hand movements.

Through its cannabis-based treatment NTI164, biopharma development company Neurotech International (ASX: NTI) hopes to become a panacea. The Company has launched a new clinical program in Rett Syndrome, comprising an upcoming filing for Human Research Ethics Committee (HREC) approval. After that, it will start a Phase II clinical trial that will investigate the use of NTI164 in female patients. 

The trial will be conducted across two centres in Australia, with co-principal investigators Dr Giuliana Antolovich from the Department of Neurodevelopment & Disability, Royal Children’s Hospital, and Professor Michael Fahey, the Head of the Paediatric Neurology Unit at Monash Medical Centre, Director of Neurogenetics.

Executive Director of Neurotech International, Dr Thomas Duthy, said, “We have carefully examined the design and subsequent results of several late-stage Phase III clinical trial results in Rett Syndrome, which has provided invaluable information relating to important regulatory endpoints that have now been proven as acceptable to global regulators, notably the US Food and Drug Administration, with the very recent approval of DAYBUE™ (trofinetide).” 

Trofinetide is a drug developed by Neuren Pharmaceuticals for the treatment of Rett Syndrome, a rare genetic disorder that affects brain development primarily in females. It got FDA approval in March 2023, becoming the first-ever treatment for Rett Syndrome.

The NTI164 proposed Phase II clinical trial will evaluate the effects of daily oral treatment of NTI164 on Rett Syndrome patients. The trial aims to recruit approximately 15 patients and will last for 12 weeks, with its primary endpoints being the Rett Syndrome Behaviour Questionnaire, Clinical Global Impression Scale-Improvement, and CGI severity of illness. The trial will also analyze the safety and adverse events associated with NTI164, as well as the effects on hand function, motor skills, communication, and quality of life. In addition, the trial will evaluate the effects of NTI164 on various biomarkers associated with neuronal function and neuroinflammation in Rett Syndrome. 

If all goes well with the HREC approval, the trial will be followed by a 14-week double-blind, randomized, placebo-controlled Phase II involving 34 participants to further determine efficacy and safety.

Duthy added, “We believe the neuroprotection shown by NTI164 with improvements in neuronal function and strong anti-inflammatory effects in brain-derived neuronal and microglial cells could translate to improved clinical outcomes in Rett Syndrome patients. When overlaid with NTI164’s excellent safety profile, this new clinical focus in Rett Syndrome where only one recently approved therapy now exists.”

Plus, the syndrome offers an annual market opportunity worth around $3 billion, allowing Neurotech to boost shareholder value. 

Neurotech expects to receive approval from the Human Research Ethics Committee (HREC) and clearance from the Therapeutic Goods Administration (TGA) for the Phase II clinical trial of NTI164 by June 2023. The patient recruitment for the trial is expected to start in the second half of 2023. The preliminary results of the trial, which will involve 15 patients, are expected to be available in the first half of 2024.