Natural Product · NTI164

NTI164

Phase 3Neurotech International Limited (NTI)

NTI164 is an oral, full-spectrum medicinal cannabis plant extract with very low THC (registered as 0.08% THC; described in the Phase 3 registration as CBDA-dominant), developed by Neurotech International for paediatric neurodevelopmental and neuropsychiatric disorders. Neurotech describes it as a proprietary, multi-constituent, GMP-grade standardised formulation containing CBDA-rich extracts and select minor cannabinoids, with proposed effects on neuroinflammatory signalling and immune dysregulation. In cell models it reduced inflammation-induced microglial marker up-regulation (an effect not reproduced by CBD alone) and supported neuron survival under excitotoxic conditions. It is in a pivotal Phase 3 study in autism spectrum disorder (Beyond Harmony, first patient dosed September 2026); the US FDA cleared an IND for NTI164 in July 2026.

Also known as: NTI164, NTI-164, FEN164

Data current as of

Key facts

Modality
Natural product
Mechanism
Natural product
Highest phase
Phase 3
Lead indication
Autism spectrum disorder
Developer
Neurotech International Limited (NTI)
Trials
5 tracked · 1 recruiting · 5 sites
Next catalyst
October 2028 — Registry results (Rett syndrome)

Mechanism of action#

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

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—

Development timeline#

2022202420262028TodayPhase 1/21 Oct 2028 — upcoming — ClinicalTrials.gov estimated primary completion of the Phase II/III placebo-controlled crossover study of NTI164 in Rett syndrome: October 2028 (not yet recruiting; estimated start July 2027).1 Feb 2026 — Partnership — Neurotech and the University of Sydney collaborate on NTI164 for Rett syndrome2 Dec 2025 — Trial registered — Phase II/III study of NTI164 in Rett syndrome registered on ClinicalTrials.gov26 Jun 2025 — readout (met) — Open-label Phase I/II study of NTI164 in 11 girls with Rett syndrome published: improvement on CGI-I (p=0.028), CGI-S, RSBQ total score and caregiver burden after 12 weeks; well tolerated.20 Sept 2023 — phase change — Open-label Phase I/II study of NTI164 in Rett syndrome (NCT06621043) started per ClinicalTrials.gov.1 Apr 2021 — phase change — 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.Phase 2/36 Aug 2026 — readout (met) — 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.4 Aug 2026 — Financing — Neurotech secures ~A$4.5 million placement to fund the NTI164 Phase 3 autism trial14 Jul 2026 — IND/CTA clearance — US FDA clears IND for NTI16417 Mar 2026 — phase change — First Beyond Harmony site (Monash Children's Hospital) initiated and open for screening and recruitment; 150 patients planned at that time.2 Feb 2026 — phase change — 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.2 Dec 2025 — Trial registered — Phase 3 Beyond Harmony study of NTI164 in autism registered on ClinicalTrials.gov30 Nov 2022 — phase change — Randomised, double-blind, placebo-controlled Phase II/III HARMONY study (NCT05626959, 54 participants) started per ClinicalTrials.gov; primary completion 2024-03.Phase 31 Jul 2029 — upcoming — ClinicalTrials.gov estimated primary completion of the Phase 3 Beyond Harmony study of NTI164 in autism spectrum disorder: July 2029.21 Sept 2026 — phase change — 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.21 Sept 2026 — Trial started — First patient dosed in Phase 3 Beyond Harmony study of NTI164 in autism
Phase change Readout Event UpcomingHover a marker for details.
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.Autism spectrum disorder
  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.Autism spectrum disorder↗
  3. Trial startedFirst patient dosed in Phase 3 Beyond Harmony study of NTI164 in autismAutism spectrum disorder↗
Phase 1/2Apr 2021 – Oct 2028
  1. UpcomingClinicalTrials.gov estimated primary completion of the Phase II/III placebo-controlled crossover study of NTI164 in Rett syndrome: October 2028 (not yet recruiting; estimated start July 2027).Rett syndrome
  2. PartnershipNeurotech and the University of Sydney collaborate on NTI164 for Rett syndromeRett syndrome↗
  3. Trial registeredPhase II/III study of NTI164 in Rett syndrome registered on ClinicalTrials.govRett syndrome
  4. metOpen-label Phase I/II study of NTI164 in 11 girls with Rett syndrome published: improvement on CGI-I (p=0.028), CGI-S, RSBQ total score and caregiver burden after 12 weeks; well tolerated.Rett syndrome↗
  5. Open-label Phase I/II study of NTI164 in Rett syndrome (NCT06621043) started per ClinicalTrials.gov.Rett syndrome
  6. 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.Autism spectrum disorder
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.Autism spectrum disorder↗
  2. FinancingNeurotech secures ~A$4.5 million placement to fund the NTI164 Phase 3 autism trialAutism spectrum disorder↗
  3. IND/CTA clearanceUS FDA clears IND for NTI164Autism spectrum disorder↗
  4. First Beyond Harmony site (Monash Children's Hospital) initiated and open for screening and recruitment; 150 patients planned at that time.Autism spectrum disorder↗
  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.Autism spectrum disorder↗
  6. Trial registeredPhase 3 Beyond Harmony study of NTI164 in autism registered on ClinicalTrials.govAutism spectrum disorder
  7. Randomised, double-blind, placebo-controlled Phase II/III HARMONY study (NCT05626959, 54 participants) started per ClinicalTrials.gov; primary completion 2024-03.Autism spectrum disorder

NTI164 for Autism spectrum disorder#

Phase 3RecruitingAutism spectrum disorder indication →

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.

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. ↗

NTI164 for Rett syndrome#

Phase 1/2ActiveRett syndrome indication →

NTI164, an oral CBDA-rich full-spectrum low-THC cannabis extract, for Rett syndrome. An open-label Phase I/II study (NCT06621043) in 11 girls aged 5-16 with a pathogenic MECP2 variant, dosed twice daily for 12 weeks, reported improvement on the primary CGI-I measure and on the Rett Syndrome Behaviour Questionnaire, mood, anxiety, communication and quality-of-life measures, with NTI164 well tolerated; the study was uncontrolled. A Phase II/III double-blind, randomised, placebo-controlled crossover study (NCT07257978, 40 participants aged 4-25, primary endpoint RSBQ) is registered but not yet recruiting, with an estimated start of July 2027. In February 2026 Neurotech began a research collaboration with the University of Sydney to study NTI164's mechanism in human Rett neuronal models.

Readouts

  • October 2028AnticipatedRegistry resultsNCT07257978

    ClinicalTrials.gov estimated primary completion of the Phase II/III placebo-controlled crossover study of NTI164 in Rett syndrome: October 2028 (not yet recruiting; estimated start July 2027).

  • 2025-06-26ReportedFull resultsmetNCT06621043

    Open-label Phase I/II study of NTI164 in 11 girls with Rett syndrome published: improvement on CGI-I (p=0.028), CGI-S, RSBQ total score and caregiver burden after 12 weeks; well tolerated. ↗

Clinical trials#

NCT07257978NTIRTT2Phase 2/3Activen=40

A Phase II/III Double-blind, Randomised, Placebo-controlled, Crossover Study Investigating the Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

Started Jul 2027· Primary completion Oct 2028· 1 site across 1 country

Australia

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

NCT06621043NTIRTT1Phase 1/2Completedn=14

Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)

Started Sept 2023· Primary completion Dec 2025· 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

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. NCT06621043 (NTIRTT1) - Phase I/II study of NTI164 in Rett syndrome — ClinicalTrials.gov (U.S. National Library of Medicine)
  7. 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)
  8. NCT07257978 (NTIRTT2) - Phase II/III double-blind, randomised, placebo-controlled crossover study of NTI164 in Rett syndrome — ClinicalTrials.gov (U.S. National Library of Medicine)
  9. Neurotech Doses First Patient in Phase 3 'Beyond Harmony' Autism Study (2026-09-21) — Neurotech International Limited (ASX announcement)
  10. Neurotech initiates first clinical site for Phase 3 'Beyond Harmony' trial (2026-03-17) — Neurotech International Limited (ASX announcement)
Show all 13 sources
  1. Quarterly Activities Report for the period ending 31 March 2026 (2026-04-29) — Neurotech International Limited (ASX announcement)
  2. Receipt of Binding Commitments for ~$4.5m Placement (2026-08-04) — Neurotech International Limited (ASX announcement)
  3. US FDA clears IND for NTI164, advancing global registration and commercialisation strategy (2026-07-14) — Neurotech International Limited (ASX announcement)