Results from NTI’s PANDAS PANS Phase I/II trial are due in September Pic: Getty Images.
Health & Biotech
Neurotech International has concluded its last patient last visit (LPLV) at the Children’s Hospital at Westmead in Sydney for its groundbreaking phase I/II trial of NTI164 into the treatment of orphan disorder PANDAS/PANS.
Neurotech International (ASX:NTI) has announced successful completion of the LPLV for the Phase I/II clinical trial of NTI164 in children diagnosed with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).
NTI executive director Dr Thomas says a total of 15 paediatric patients have now successfully completed daily oral treatment with NTI164 over the initial 12 weeks of the trial with 100% of patients continuing to receive treatment under the extension phase (54 weeks) of the trial protocol.
Duthy says three patients have now turned 18 years of age and continue to receive NTI164 under revised Human Research Ethics Committee (HREC) approval granted on May 30, 2023.
NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN.
The drug has been exclusively licenced for neurological applications globally with pre-clinical studies demonstrating a 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.
Duthy says the trial is a world first with a broad-spectrum cannabinoid therapy, with results forecast for release to the market in mid to late September.
“We warmly congratulate the Co-Principal Investigators Professor Russell Dale and Professor Michael Fahey and their respective clinical teams on the very rapid enrolment into this important clinical trial in PANDAS/PANS patients,” Duthy says.
PANDAS stands for Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcus Infections. PANS stands for Pediatric Acute-onset Neuropsychiatric Syndrome.
“Collectively PANDAS PANS is a disorder where children go to bed perfectly normal but wake up with uncontrollable tic movements in their hands and legs and become severely obsessive compulsive with heightened anxiety and depression,” Duthy said.
“The reason that disease manifests are there’s some dysregulation from streptococcus or some other unknown pathogen that initiate’s an autoimmune response which sees inflammation in their brain.”
Duthy says PANDAS/PANS are classified as rare orphan disorders, where approved drug therapies are lacking, and clinical trials are desperately needed.
“NTI164 has very demonstrable and positive impacts on neuroinflammatory processes in the brain and also has ability to protect neurons in the brain so that dual mechanism of action is why we are targeting these kinds of disorders in children,” he says.
“It is testament to the clinical interest in NTI164 as a treatment for certain paediatric neurological disorders characterised by persistent neuroinflammation and the evidence of benefit we have shown in our reported autism spectrum disorder trial.
“We look forward to reporting the clinical efficacy and safety of NTI164 in these patients over the 12-week study period.”
NTI is a clinical-stage biopharmaceutical development company focused predominately on paediatric neurological disorders.
The company’s primary focus to date has been autism spectrum disorder (ASD) and it’s currently running a Phase 2/3 clinical trial in Australia in 54 ASD patients.
Following in the footsteps of 2023 biotech market darling Neuren Pharmaceuticals (ASX:NEU), NTI in July announced HREC approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) to investigate the use of NTI164 in female Rett Syndrome patients.
Rett Syndrome is the second leading cause of intellectual disability in girls, with an urgent medical need to develop safe and effective therapies to treat this progressive neurological disease.
In March this year NEU announced that its large US pharma partner Arcadia Pharmaceuticals (NASDAQ:ACAD) had received FDA approval for its drug trofinetide, now marketed under the name Daybue.
PANDAS stands for Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcus Infections. PANS stands for Pediatric Acute-onset Neuropsychiatric Syndrome.
“Collectively PANDAS PANS is a disorder where children go to bed perfectly normal but wake up with uncontrollable tic movements in their hands and legs and become severely obsessive compulsive with heightened anxiety and depression,” Duthy said.
“The reason that disease manifests are there’s some dysregulation from streptococcus or some other unknown pathogen that initiate’s an autoimmune response which sees inflammation in their brain.”
Duthy says PANDAS/PANS are classified as rare orphan disorders, where approved drug therapies are lacking, and clinical trials are desperately needed.
“NTI164 has very demonstrable and positive impacts on neuroinflammatory processes in the brain and also has ability to protect neurons in the brain so that dual mechanism of action is why we are targeting these kinds of disorders in children,” he says.
“It is testament to the clinical interest in NTI164 as a treatment for certain paediatric neurological disorders characterised by persistent neuroinflammation and the evidence of benefit we have shown in our reported autism spectrum disorder trial.
“We look forward to reporting the clinical efficacy and safety of NTI164 in these patients over the 12-week study period.”
The trial is assessing both efficacy and safety in the patient population based on standard clinician assessed measures of severity of illness, improvement and anxiety and depression measures over 12 weeks of the trial, compared to baseline (day 0).
Duthy says several other important assessments will also be included. In addition, NTI is undertaking a significant genomic analysis to examine how NTI164 effects certain biomarkers, relative to baseline measures.
It is testament to the clinical interest in NTI164 as a treatment for certain paediatric neurological disorders characterised by persistent neuroinflammation and the evidence of benefit we have shown in our reported autism spectrum disorder trial.
Neuroinflammation is well characterised in PANDAS/PANS, where approved drug therapies are lacking, and clinical trials are desperately needed.
This article was developed in collaboration with Neurotech International, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
Health & Biotech
Health & Biotech
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