NTI164 · program

NTI164 for Autism spectrum disorder

Phase 3RecruitingNeurotech International Limited (NTI)

Indications for NTI164: Autism spectrum disorder · Phase 3 Rett syndrome · Phase 1/2

NTI164, an oral CBDA-rich full-spectrum low-THC cannabis extract, for the core symptoms of autism spectrum disorder in children and young people (ASD Levels 2 and 3). This is Neurotech's lead program. After an open-label Phase I/II study (NCT05516407, 18 children) and the randomised, double-blind, placebo-controlled Phase II/III HARMONY study (NCT05626959, 54 children aged 2-17), which reported statistically significant improvements over placebo in overall clinical severity, adaptive functioning, social responsiveness and affective symptoms, Neurotech began the pivotal Phase 3 Beyond Harmony study (NCT07257939). Beyond Harmony is a multi-centre, randomised, double-blind, placebo-controlled study at six Australian sites, planned for up to 200 patients under an adaptive design, with Vineland-3 at Week 16 as the registered primary endpoint; the first patient was dosed on 2026-09-21. It is intended to support submissions to the Australian TGA and the US FDA, and the FDA cleared an IND for NTI164 on 2026-07-14 for a complementary US population-PK program. Neurotech says its August 2026 placement funds it through the Phase 3 topline.

Data current as of

Development timeline

Phase 3Sept 2026 – Jul 2029
  1. UpcomingClinicalTrials.gov estimated primary completion of the Phase 3 Beyond Harmony study of NTI164 in autism spectrum disorder: July 2029.
  2. All six Australian sites initiated and the first patient dosed in the Phase 3 Beyond Harmony study; enrollment target raised to up to 200 under the adaptive design.↗
  3. Trial startedFirst patient dosed in Phase 3 Beyond Harmony study of NTI164 in autism↗
Phase 2/3Nov 2022 – Aug 2026
  1. metHARMONY Phase II/III (54 children with Level II/III ASD, NTI164 up to 20 mg/kg/day vs placebo for 8 weeks) published: statistically significant improvements over placebo in overall clinical severity, adaptive functioning, social responsiveness and affective symptoms, with a favourable safety profile.↗
  2. FinancingNeurotech secures ~A$4.5 million placement to fund the NTI164 Phase 3 autism trial↗
  3. IND/CTA clearanceUS FDA clears IND for NTI164↗
  4. First Beyond Harmony site (Monash Children's Hospital) initiated and open for screening and recruitment; 150 patients planned at that time.↗
  5. Human Research Ethics Committee approval received to start the Beyond Harmony Phase 3 study in ASD Levels 2 and 3, led by Professor Michael Fahey; designed to support TGA and FDA submissions.↗
  6. Trial registeredPhase 3 Beyond Harmony study of NTI164 in autism registered on ClinicalTrials.gov
  7. Randomised, double-blind, placebo-controlled Phase II/III HARMONY study (NCT05626959, 54 participants) started per ClinicalTrials.gov; primary completion 2024-03.
Phase 1/2Apr 2021
  1. Open-label Phase I/II study of NTI164 (then FEN164) in children with ASD (NCT05516407, 18 participants) started per ClinicalTrials.gov; primary completion 2022-09.

Readouts

  • July 2029AnticipatedRegistry resultsNCT07257939

    ClinicalTrials.gov estimated primary completion of the Phase 3 Beyond Harmony study of NTI164 in autism spectrum disorder: July 2029.

  • 2026-08-06ReportedFull resultsmetNCT05626959

    HARMONY Phase II/III (54 children with Level II/III ASD, NTI164 up to 20 mg/kg/day vs placebo for 8 weeks) published: statistically significant improvements over placebo in overall clinical severity, adaptive functioning, social responsiveness and affective symptoms, with a favourable safety profile. ↗

Clinical trials in Autism spectrum disorder

NCT07257939NTIASD3Phase 3Recruitingn=200

A Phase III Double-blind, Randomised, Placebo-controlled Study Investigating the Efficacy and Safety of NTI164 in Children and Young Adults With Autism Spectrum Disorder

Started Sept 2026· Primary completion Jul 2029· 📍 1 site across 1 country (Australia)

NCT05626959NTIASD2Phase 2/3Completedn=54

A Phase II/III Double-Blind, Randomised and Controlled-to-Open-Label Study Assessing the Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the Severity of Autism Spectrum Disorder in Young People

Started Nov 2022· Primary completion Mar 2024· 📍 1 site across 1 country (Australia)

NCT05516407FNX001-21Phase 1/2Completedn=18

A Phase I/II Open-Label Study to Evaluate the Safety and Efficacy of Orally Administered Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (FEN164) in Children With Autism Spectrum Disorder

Started Apr 2021· Primary completion Sept 2022· 📍 1 site across 1 country (Australia)

Formulations

FormulationRouteRegimenPharmacokinetics
NTI164 oral oil (weight-based, twice daily)
Oil-based oral extract. Rett Phase I/II: started at 5 mg/kg and titrated up to 20 mg/kg per day by tolerance, given twice daily for 12 weeks. Harmony ASD Phase II/III: up to 20 mg/kg/day for 8 weeks. PANS Phase I/II: 20 mg/kg/day for 12 weeks. Healthy-volunteer PK study: 20 mg/kg.
OralTwice daily—

Mechanism of action (compound-wide)

No single molecular target is claimed. NTI164 is a multi-constituent cannabis extract led by cannabidiolic acid (CBDA) with minor cannabinoids and very low THC; the sponsor attributes its activity to modulation of neuroinflammatory signalling, immune dysregulation and epigenetic pathways. In vitro it attenuated inflammation-induced up-regulation of microglial inflammatory markers in BV-2 cells (CBD alone did not) and promoted neuron survival under excitotoxic conditions. In an open-label PANS study, multi-omic blood analyses showed treatment-associated modulation of epigenetic, ribosomal and immune pathways in leucocytes.

TargetActionAffinity

← Full NTI164 compound page (identity, identifiers, all indications)

Sources

  1. Approval received for NTI164 Phase 3 Clinical Study in ASD (2026-02-02) — Neurotech International Limited (ASX announcement)
  2. Keating BA, et al. Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open-Label Study. J Paediatr Child Health. 2025 — Journal of Paediatrics and Child Health (Wiley) / PubMed
  3. Keating BA, et al. NTI164, a novel medicinal cannabis extract, improves core symptoms of autism spectrum disorder: results from a double-blind, randomised, controlled trial (The Harmony study). Neurotherapeutics. 2026;23(5):e01033 — Neurotherapeutics (Elsevier) / PubMed
  4. NCT05516407 (FNX001-21) - Phase I/II open-label study of full-spectrum medicinal cannabis plant extract 0.08% THC in children with autism spectrum disorder — ClinicalTrials.gov (U.S. National Library of Medicine)
  5. NCT05626959 (NTIASD2) - Phase II/III double-blind, randomised study of NTI164 in young people with autism spectrum disorder (Harmony) — ClinicalTrials.gov (U.S. National Library of Medicine)
  6. NCT07257939 (NTIASD3) - Phase III study of NTI164 in children and young adults with autism spectrum disorder (Beyond Harmony) — ClinicalTrials.gov (U.S. National Library of Medicine)
  7. Neurotech Doses First Patient in Phase 3 'Beyond Harmony' Autism Study (2026-09-21) — Neurotech International Limited (ASX announcement)
  8. Neurotech initiates first clinical site for Phase 3 'Beyond Harmony' trial (2026-03-17) — Neurotech International Limited (ASX announcement)
  9. Receipt of Binding Commitments for ~$4.5m Placement (2026-08-04) — Neurotech International Limited (ASX announcement)
  10. US FDA clears IND for NTI164, advancing global registration and commercialisation strategy (2026-07-14) — Neurotech International Limited (ASX announcement)