Neurotech International admits final patient for NTI164 Phase II/III trial to treat autism

Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has confirmed that the last patient in a Phase II/III NTIASD2 clinical trial for children with autism spectrum disorder (ASD) has completed their final visit.

The child was the 54th patient to take part in the trial, which recruited individuals between the ages of 8 and 17 years with autism at Level 2 (needing substantial support) and Level 3 (needing the most support).

The patients completed eight weeks of daily treatment with cannabinoid-based drug formulation NTI164 (the randomisation period of the trial) at the Paediatric Neurology Unit within Melbourne’s Monash Medical Centre.

Regulatory talks

Executive director Dr Thomas Duthy said the trial would be used in talks with regulatory authorities.

“With an explosion in autism-associated costs under Australia’s National Disability Insurance Scheme, there is an urgent need for new and enabling treatments like NTI164, which has been shown in an earlier clinical trial to significantly improve adaptive behaviours, socialisation and quality of life,” he said.

“The results of this trial will inform our discussions with the Therapeutic Goods Administration to understand our pathway to market approval in Australia, where the prevalence of autism is estimated at 1-in-50 across the population and represents a 40-fold increase over the last 20 years.”

Efficacy and safety

NTIASD2 is a randomised, double-blind, placebo-controlled, Phase II/III clinical trial designed to determine the efficacy and safety of NTI164 versus placebo.

The study comprises two months of treatment period followed by a two-month open-label maintenance period and two-week “wash-out” period, during which no treatment is administered.

Participants who choose to continue receiving NTI164 beyond the duration of the study may do so for an additional 38 weeks.

At the end of the extension phase, they will undergo two weeks of down-titration, during which time the dose is gradually decreased.

Drug formulation

NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low tetrahydrocannabinol and a novel combination of cannabinoids.

The candidate has been exclusively licenced for neurological applications globally.

Pre-clinical studies have demonstrated potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.

NTI164 is being developed as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.

Fenix Innovation partnership

In addition, Neurotech announced today it had signed a binding term sheet with Melbourne-based Fenix Innovation Group for the development of NTI164 for paediatric neurological disorders.

As consideration for the partnership, Neurotech will issue 10 million ordinary shares to Fenix upon signing of a definitive contract and Fenix will voluntarily escrow them for a period of 12 months from the date of issue.

Fenix will also receive 50 million performance rights, with vesting conditions based upon the achievement of certain regulatory and commercialisation milestones which could result in value accretion for Neurotech.

The milestones include orphan drug designations, partnering transactions and approval of NTI164 by Australia’s Therapeutic Goods Administration over the next three years.

The issue of shares and the associated performance rights is subject to approval by Neurotech shareholders.

Existing relationship

Dr Duthy said the new agreement builds on an existing relationship between Neurotech and Fenix.

“Fenix has been integral to the development of NTI164 since 2019, efficiently overseeing the manufacturing and preparation of drug product, regulatory submissions, clinical trial protocol development, management of clinical trials and key opinion leader developments,” he said.

“We expect the alignment of [our] development objectives will result in the more timely and cost-effective execution of clinical and commercial development pathways.”

Fenix said NTI164 had potential to “significantly improve” clinical symptoms in paediatric patients across a range of neurological disorders involving persistent or progressive neuroinflammation.