NTI164 / Neurotech International - LARVOL DELTA

Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder (clinicaltrials.gov) - P2/3 | N=54 | Completed | Sponsor: Fenix Innovation Group | Trial completion date: Nov 2023 ➔ May 2026

Trial completion date • Autism Spectrum Disorder • Genetic Disorders

NTI164 Human PK Study (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed

Trial completion

CannaPANS: Investigating The Effects Of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) On Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) (clinicaltrials.gov) - P1/2 | N=18 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed

Trial completion • Pediatrics • Psychiatry

Safety and Efficacy of Oral Full-Spectrum Medicinal Cannabis Plant Extract in Children With Autism Spectrum Disorder. (clinicaltrials.gov) - P1/2 | N=18 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed | Trial completion date: Sep 2022 ➔ Aug 2026

Trial completion • Trial completion date • Autism Spectrum Disorder • Genetic Disorders

HarmonyPlus: NTI164 in Autism Spectrum Disorder (clinicaltrials.gov) - P3 | N=98 | Not yet recruiting | Sponsor: Fenix Innovation Group | Initiation date: Jul 2026 ➔ Sep 2026

Trial initiation date • Autism Spectrum Disorder • Genetic Disorders

Evaluating the Safety and Efficacy of Full-Spectrum Medicinal Cannabis (FEN164) in Children with ASD. (ANZCTR) - P1/2 | N=20 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed

Trial completion • Autism Spectrum Disorder • Genetic Disorders

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) (clinicaltrials.gov) - P1/2 | N=14 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed

Trial completion • Developmental Disorders • Movement Disorders

Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder (clinicaltrials.gov) - P2/3 | N=54 | Completed | Sponsor: Fenix Innovation Group | Active, not recruiting ➔ Completed

Trial completion • Autism Spectrum Disorder • Genetic Disorders

NTI164, a novel medicinal cannabis extract, improves core symptoms of autism spectrum disorder: Results from a double-blind, randomised, controlled trial (The Harmony study). (PubMed, Neurotherapeutics) - "NTI164 significantly improved core and associated symptoms of ASD compared to placebo. Consistent benefits reported by both clinicians and caregivers in both open label and double-blind contexts supports further clinical development of NTI164 in ASD."

Journal • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Genetic Disorders • Mental Retardation • Pediatrics • Psychiatry

Neurotech International has secured Human Research Ethics Committee (HREC) approval to commence its Beyond Harmony phase III clinical study of lead asset NTI164 in autism spectrum disorder (ASD) Levels 2 and 3. (Herald Sun) - "Neurotech is now advancing NTI164 into a pivotal registration pathway to support potential submissions to the Therapeutic Goods Administration (TGA) in Australia and the US Food and Drug Administration (FDA)."

New P3 trial • Autism Spectrum Disorder

Towards treatments targeting the gut to improve behavioural outcomes in autism spectrum disorder. (PubMed, J Neural Transm (Vienna)) - "Repurposed medications such as pimavanserin (an atypical antipsychotic) and the antiparasitic suramin are being trialled for treating irritable behaviours and impaired social interaction, respectively, in autism. NTI164 is a medicinal cannabis-derived biopharmaceutical undergoing clinical safety and efficacy trials for improving social communication and similarly, ML-004 is an investigational drug being assessed for treating social communication deficits. Two other repurposed medications previously utilised for schizophrenia; brexpiprazole and lumateperone, as well as AB-2004, a microbial metabolite sequestering agent (with proposed actions on gut function), are undergoing clinical trials to assess impacts on irritability associated with autism. We also outline emerging findings from clinical studies on the use of gut-targeted small molecules and bacteriophage therapy, prebiotics, probiotic supplementation and faecal microbiota transplantation (FMT), and their potential..."

Journal • Review • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Gastrointestinal Disorder • Genetic Disorders • Mental Retardation • Psychiatry • Schizophrenia • Transplantation

Medicinal cannabis plant extract (NTI164) modifies epigenetic, ribosomal, and immune pathways in paediatric acute-onset neuropsychiatric syndrome. (PubMed, Neurotherapeutics) - "NTI164 shows promise as a disease-modifying therapeutic for PANS. Multi-omics reveal broad epigenetic and immune dysregulation in patients, which was modified by NTI164, presenting epigenetic machinery as a therapeutic target in PANS."

Journal • ADHD (Impulsive Aggression) • Attention Deficit Hyperactivity Disorder • CNS Disorders • Genetic Disorders • Infectious Disease • Mood Disorders • Movement Disorders • Obsessive-Compulsive Disorder • Pediatrics • Psychiatry

TRANSCEND: Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome (clinicaltrials.gov) - P2/3 | N=40 | Not yet recruiting | Sponsor: Fenix Innovation Group

New P2/3 trial • Developmental Disorders • Movement Disorders

HarmonyPlus: NTI164 in Autism Spectrum Disorder (clinicaltrials.gov) - P3 | N=98 | Not yet recruiting | Sponsor: Fenix Innovation Group

New P3 trial • Autism Spectrum Disorder • Genetic Disorders

Neurotech International has passed an important regulatory milestone with the US Food and Drug Administration (FDA) granting Rare Paediatric Disease Designation (RPDD) for its lead drug candidate NTI164 for the treatment of Rett syndrome. (The Australian)

FDA event • Rare Diseases

A Phase I/II study to assess the safety and efficacy of full-spectrum medicinal cannabis plant extract 0.08% THC (NTI164) in the treatment of Rett syndrome (RTT) (ANZCTR) - P1/2 | N=14 | Active, not recruiting | Sponsor: Fenix Innovation Group | Recruiting ➔ Active, not recruiting

Enrollment closed • Developmental Disorders • Movement Disorders

Investigating the effects of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on paediatric acute-onset neuropsychiatric syndrome (PANS): An open-label study. (ANZCTR) - P1/2 | N=15 | Active, not recruiting | Sponsor: Fenix Innovation Group | Not yet recruiting ➔ Active, not recruiting

Enrollment closed • Pediatrics • Psychiatry

Evaluating the Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in Young People with Autism Spectrum Disorder, a Double-Blind, Randomised and Controlled-to-Open-Label Study. (ANZCTR) - P2/3 | N=54 | Active, not recruiting | Sponsor: Fenix Innovation Group | Not yet recruiting ➔ Active, not recruiting

Enrollment closed • Autism Spectrum Disorder • Genetic Disorders

Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study. (PubMed, J Paediatr Child Health) - "NTI164 demonstrated safety and improved some clinical and functional outcomes in RTT. These improvements justify ongoing research into NTI164, which may be a potential adjunct therapy in RTT."

Journal • CNS Disorders • Developmental Disorders • Inflammation • Mood Disorders • Movement Disorders • Psychiatry • Respiratory Diseases

NTI164 Human PK Study (clinicaltrials.gov) - P1 | N=12 | Active, not recruiting | Sponsor: Fenix Innovation Group | Not yet recruiting ➔ Active, not recruiting | Initiation date: Dec 2024 ➔ Feb 2024

Enrollment closed • Trial initiation date

NTI164 Human PK Study (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Fenix Innovation Group

New P1 trial

Evaluation of the Efficacy of a Full-Spectrum Low-THC Cannabis Plant Extract Using In Vitro Models of Inflammation and Excitotoxicity. (PubMed, Biomolecules) - "NTI-164 promoted undifferentiated neuron proliferation and differentiated neuron survival under excitotoxic conditions. These effects suggest the potential for NTI-164 as a treatment for neuropathologies."

Journal • Preclinical • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Genetic Disorders • Inflammation • Psychiatry

CannaPANS: Investigating The Effects Of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) On Paediatric Acute-onset Neuropsychiatric Syndrome (PANS) (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: Fenix Innovation Group

New P1/2 trial • Pediatrics • Psychiatry

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) (clinicaltrials.gov) - P1/2 | N=14 | Active, not recruiting | Sponsor: Fenix Innovation Group

New P1/2 trial • Developmental Disorders • Movement Disorders

A Phase I/II study to assess the safety and efficacy of full-spectrum medicinal cannabis plant extract 0.08% THC (NTI164) in the treatment of Rett syndrome (RTT) (ANZCTR) - P1/2 | N=14 | Recruiting | Sponsor: Fenix Innovation Group | Not yet recruiting ➔ Recruiting

Enrollment open • Trial initiation date • Developmental Disorders • Movement Disorders