Delving into the Latest Updates on NTI-164 with Synapse

Overview

Basic Info

Drug Type

Herbal medicine

Synonyms

NTI 164, NTI164

Target-

Action-

Mechanism-

Therapeutic Areas

Immune System DiseasesNervous System DiseasesCongenital Disorders+ [1]

Active Indication

Autism Spectrum DisorderAutistic DisorderRett Syndrome+ [1]

Inactive Indication-

Originator Organization

Fenix Innovation Group Pty Ltd

Neurotech International Ltd.

Active Organization

Neurotech International Ltd.Fenix Innovation Group Pty Ltd

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States)

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The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design. RTT is a devastating rare genetic condition affecting females and involves debilitating physical and intellectual symptoms. NTI164 is an oil which has demonstrated efficacy in reducing symptoms in several paediatric neurological conditions, including RTT, autism spectrum disorder (ASD), and paediatric acute-onset neuropsychicatric syndrome (PANS). A Phase I/II clinical trial of NTI164 in RTT (FENRTT1/NTIRTT1) showed NTI164 is safe in this population and significantly improved overall clinical severity of illness, as well as core RTT symptoms, including anxiety, mental alertness, communication skills, socialisation/eye contact, and attentiveness. The FENRTT2 study will investigate NTI164 in a larger number of patients, and compare NTI164 to a placebo control. Research tests on patient blood will also be included to further investigate how NTI164 works in the body.

Start Date01 Jul 2026

Sponsor / Collaborator

Fenix Innovation Group Pty Ltd

[+1]

NCT07257939

/ Not yet recruitingPhase 3

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

This is a double-blind, randomised, placebo-controlled study investigating the efficacy of a full-spectrum medicinal cannabis plant extract on core and associated ASD symptoms over placebo. Participants will be randomly allocated to either NTI164 or placebo at a 1:1 ratio and blood samples will be collected and surveys completed at baseline and Week 16. This study will expand efficacy and safety data of NTI164 and provide additional mechanism of action data of NTI164 in this patient cohort.

Start Date01 Jul 2026

Sponsor / Collaborator

Fenix Innovation Group Pty Ltd

[+1]

NCT06713447

/ Active, not recruitingPhase 1

A Pharmacokinetic Study of NTI164 in Healthy Adult Volunteers

This study is looking at how a new cannabis compound with extremely low THC (0.08%), NTI164, is metabolised in the body and how it is excreted in urine over time (up to 28 days) in healthy adult volunteers. NTI164 will be delivered twice daily, at a concentration of 20mg/kg/day. There will be 2 parts to this study, Part A and Part B. Part A involves only one day of taking NTI164, whereas Part B involves taking NTI164 for 7 consecutive days. Urine samples and blood samples will be collected at certain time points after NTI164 dosing to help characterise how the drug is being broken down in the body and over what time frame.

Start Date01 Feb 2024

Sponsor / Collaborator

Fenix Innovation Group Pty Ltd

[+2]

100 Clinical Results associated with NTI-164

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100 Translational Medicine associated with NTI-164

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100 Patents (Medical) associated with NTI-164

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2

Literatures (Medical) associated with NTI-164

Biomolecules

Evaluation of the Efficacy of a Full-Spectrum Low-THC Cannabis Plant Extract Using In Vitro Models of Inflammation and Excitotoxicity

Article

Author: Ross-Munro, Emily ; Fleiss, Bobbi ; Isikgel, Esra

Evidence has accumulated that Cannabis-derived compounds have the potential to treat neuroinflammatory changes present in neurodevelopmental conditions such as autism spectrum disorder. However, research is needed on the specific brain health benefits of strains of whole Cannabis extract that are ready for commercial production. Here, we explore the anti-inflammatory and neuroprotective effects of NTI-164, a genetically unique high-cannabidiol (CBD), low-Δ9-tetrahydrocannabinol extract, and also CBD alone on BV-2 microglia and SHSY-5Y neurons. Inflammation-induced up-regulation of microglial inflammatory markers was significantly attenuated by NTI-164, but not by CBD. 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 OF PAEDIATRICS AND CHILD HEALTH

Full‐Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) Improves Symptoms of Rett Syndrome: An Open‐Label Study

Article

Author: Y. Ogru ; K. Lichkus ; M. C. Fahey ; T. G. Duthy ; C. Ellaway ; L. Douglas ; B. A. Keating ; E. Isikgel

ABSTRACT:

Aim:

The aim of this Phase I/II open‐label study was to assess the safety and efficacy of NTI164, a novel full‐spectrum medicinal cannabis plant extract 0.08% Δ‐9‐tetrahydrocannabinol (THC), in Rett syndrome (RTT).

Methods:

Eleven female participants (5–16 years) with a pathogenic variant in the MECP2 gene were recruited to this study, receiving NTI164 twice daily for 12 weeks. The primary outcome measure was the Clinical Global Impression‐Improvement (CGI‐I) Scale, with secondary outcomes measured using the CGI‐Severity (CGI‐S), RTT Behaviour Questionnaire (RSBQ), RTT‐Symptom Index Score (RTT‐SIS), RTT‐Domain‐Specific Concerns‐Visual‐Analog Scale (RTT‐DSC‐VAS), Impact of Childhood Neurological Disability/Quality of Life (ICND+QoL), and RTT‐Caregiver Burden Inventory (RTT‐CBI). Paired‐samples t‐test was used to assess significance between baseline and Week 12.

Results:

Improvements were seen in the total CGI‐I score (p = 0.028), with improvements in communication skills (p = 0.003), mental alertness (p = 0.033), socialisation/eye contact (p = 0.0004), attentiveness (p = 0.001), and anxiety (p = 0.004). CGI‐S also demonstrated better outcomes after NTI164 administration (p = 0.008). RSBQ showed improvements in total score (p = 0.0005), general mood (p = 0.0003), breathing problems (p = 0.041), repetitive face movements (p = 0.004), and fear/anxiety (p = 0.006). RTT‐DSC‐VAS showed positive developments in abilities to communicate choices (p = 0.041). ICND total score was improved (p = 0.003), as well as cognition (p = 0.027) and Quality of Life (p = 0.0002). Total score on the RTT‐CBI was improved (p = 0.006).

Conclusion:

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.

100 Deals associated with NTI-164

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Autistic Disorder	Phase 3	Australia	Fenix Innovation Group Pty Ltd	01 Jul 2026
Rett Syndrome	Phase 3	Australia	Fenix Innovation Group Pty Ltd	01 Jul 2026
Autism Spectrum Disorder	Phase 3	Australia	Neurotech International Ltd.	30 Nov 2022
Pediatric Acute-Onset Neuropsychiatric Syndrome	Phase 2	Australia	Neurotech International Ltd.	02 Jun 2023
Pediatric Acute-Onset Neuropsychiatric Syndrome	Phase 2	Australia	Fenix Innovation Group Pty Ltd	02 Jun 2023

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06621043 (NTIRTT1, Company_Website) Manual	Phase 1/2	Rett Syndrome	14	NTI164	aaxkfngiiq(uqcoxhjfcy) = zvhikbqout bktybweofs (qtewwsrtpn ) View more	Positive	01 Dec 2024
NCT06621888 (NTIPANS1, Company_Website) Manual	Phase 1/2	Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections	15	NTI164	zzhjjlwtxj(dohdixusis) = Primary endpoint of anxiety and depression (RCADS-P) met (p=0.016) with a 30% improvement in overall symptoms from high severity at baseline to low severity from week 4 onwards. 45% improvement versus baseline. jaowgrwvim (tbuojthphn ) Met View more	Positive	01 Dec 2024
NCT05626959 (NTIASD2, Company_Website) Manual	Phase 2/3	Autism Spectrum Disorder	54	NTI164	diwvmjnqkb(yzmqhpepqr): Difference = -1.65 (95% CI, -2.3 to -1.0), P-Value = <0.001 Met View more	Positive	01 Dec 2024
NCT05626959 (NTIASD2, Company_Website) Manual	Phase 2/3	Autism Spectrum Disorder	54	Placebo		Positive	01 Dec 2024