• Neurotech has a clear path for development and commercialisation of NTI164 to treat PANDAS/PANS
  • With no approved therapy for PANDAS/PANS Neurotech is pursing orphan drug designation for NTI164
  • Results of Neurotech’s phase 1/2 trial investing use of NTI164 in children with Rett Syndrome in Q1 2024

 

Neurotech International achieved a key milestone with positive results from its Phase I/II clinical trial into conditions where children go to bed perfectly normal but wake up with uncontrollable tic movements, become severely obsessive compulsive with heightened anxiety and depression.

Neurotech International (ASX:NTI) in October reported strong clinical results for the 15 patient, open-label Phase 1/2 clinical trial of NTI164 in children diagnosed with Paediatric autoimmune neuropsychiatric disorders associated with  streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).

PANDAS and PANS manifest due to some dysregulation from streptococcus or some other unknown pathogen that initiates an autoimmune response which sees inflammation in the brain.

NTI’s proprietary NTI164 drug formulation is derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN.

NTI164 is 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.

It is being developed as a therapeutic drug various neurological disorders in children where neuroinflammation is involved and is currently undergoing a randomised, double-blind, placebo-controlled Phase 2/3 clinical trial in Australia for autism spectrum disorder following earlier success.

It has also secured HERC approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) for investigations in treating female Rett Syndrome patients.

Executive director Dr Thomas Duthy discusses the latest results and its commercialisation strategy for NTI164.

Q: How important are the results of the PANDAS/PANS trial to the company?

A: “This is a significant milestone for the company,” Duthy says.

“This is also a huge step forward in Neurotech’s human clinical program as NTI is the only company in Australia that is carrying out a rigorous pharmaceutical development program using a biopharmaceutical that is derived from low THC medicinal cannabis, NTI164.

“Our world first trial has effectively reduced some of the symptoms associated with PANDAS/PANS, such as obsessive-compulsive behaviours, anxiety, tics, mood disturbances, cognitive difficulties, and other neurological symptoms.

“The treatment was also well-tolerated by the patients involved in the trial with no serious adverse events recorded.

“It is important to add that all 15 participants elected to remain on the therapy at the conclusion of the trial.

“PANDAS/PANS is a complex disorder with no approved therapy or specific drug, hence why NTI is pursuing its orphan drug approach.

“This presents an exciting potential commercial opportunity for Neurotech.

“The company has a clear path forward for the development and commercialisation of NTI164 as a treatment for PANDAS/PANS.

“Given the initial PANDAS/PANS results, NTI will look towards developing an orphan drug pathway with all the advantages that it offers and intends to progress with applications for an orphan drug designation for NTI164 associated with the treatment of PANDAS/PANS in the US, and EU.”

Q: What are the symptoms of PANDAS/PANS and how does NTI164 help?

A: “PANDAS/PANS are conditions that are characterized by sudden onset neuropsychiatric symptoms in children,” Duthy says.

“The main symptoms of PANDAS/PANS can include obsessive compulsive symptoms (OCD), where children may develop sudden and severe obsessions or compulsions that significantly interfere with their daily activities and routines.

“Motor and/or vocal tics, such as repetitive movements or sounds, may appear suddenly or worsen in intensity.

“Children may experience mood swings, irritability, anxiety, depression, or aggression that are out of character for them.

“Children may also regress and experience a sudden loss of previously acquired  skills, such as handwriting, toileting, or verbal communication.

“Children with PANDAS/PANS may exhibit excessive and irrational fears, as well as difficulty being separated from their caregivers.

“They may have sleep disturbances such Insomnia, frequent awakenings, night terrors, or bedwetting.

“Sensory issues where children become hypersensitive or hyposensitive to sensory stimuli, leading to difficulties with processing sensory information can be a symptom.

“In a world first, NTI successfully demonstrated that its lead biopharmaceutical product, NTI164 was able to reduce and alleviate some of  the main symptoms of PANDAS/PANS as described.

“These changes and improvements were deemed to considerably improve the quality of life of participants.

“NTI164 successfully improved OCD like behaviours, significantly reduced anxiety, improved emotional changes leading to outstanding patient outcomes when compared to where they were at the start of the trial.

Q: Were any major side effects experienced by patients during the trial?

A: “We were extremely pleased with the safety data from the trial,” Duthy says.

“There were some reported occurrences of nausea and vomiting from 3 of the 15 participants over 12 weeks of daily oral treatment, however none of these events were deemed to be severely adverse.

“It is likely that most of these occurrences are from the taste and volume of medication that the participants ingest.”

Q: Are you expecting any further results from the trial?

A: “Yes, we are awaiting further data from Professor R Dale who is undertaking a series of molecular mechanism of action studies,” Duthy says.

“He is conducting a world first study designed to provide evidence of cellular changes from baseline measures to after 12 weeks of PANDAS/PANS treatment with NTI164 to correlate and assist in identifying relevant biomarkers of the disorder.

“If successful, this could give great insight into the method of action of NTI164 which could significantly assist the company in its efforts to gain registration of the drug.

“These studies will also assist the company select the most appropriate disorders to treat.”

Q: How will securing orphan drug designation help Neurotech commericalise NTI164?

A: “Orphan drug designation is a specific regulatory mechanism that exists in the US and EU through the FDA and EMA to encourage investment and development of new drugs for rare diseases,” Duthy says.

“It was established to encourage research into rare diseases and encourage corporates to commercialise drugs for such indications.

“We believe that PANDAS/PANS meets the criteria of a rare disease under the FDA and EMA guidelines for an orphan drug.

“Orphan Drug Designation will also allow Neurotech to gain significant benefits, such as market exclusivity periods,  financial incentives, reduced clinical requirements and assistance in the drug development process.

“There  have  been  recent  ASX  examples  of  biotech  companies  successfully  demonstrating  the commercial significance of an Orphan Drug development strategy, including Neuren Pharmaceuticals (ASX:NEU).

“Neuren has successfully commercialised a drug called Trofinetide which secured multiple  orphan drug designations and subsequently achieved FDA approval in 2023. Neuren has a market capitalisation now of over $1.3 billion.”

Q: What is Neurotech’s strategy for commercialising NTI164?

A: “Neurotech’s short term strategy is to generate clinical evidence of safety and benefit for NTI164 across multiple paediatric  neurological disorders to add substantial value to our  NTI164  therapeutic platform,” Duthy says.

“In parallel, NTI continues to pursue a strategy of establishing strategic partnerships aimed at progressing FDA, TGA and EMA drug registrations in the US, Australia, and the EU.

“The clinical evidence of benefit generated from this successful PANDAS/PANS trial along with earlier long-term clinical results in ASD will continue to assist with this key strategic objective.

Q: What company catalysts can investors look forward to over the coming months?

A: “There are several key objectives and future milestones for the company,” Duthy says.

“There’s results of the landmark 54 patient Phase II/III double blinded placebo-controlled ASD trial  which is nearing completion of patient recruitment at Monash Children’s Hospital with results  anticipated in Q1 2024.

“There’s molecular mechanism of action studies being conducted under guidance of Professor Dale including a world first study designed to provide further evidence of genomic molecular changes from baseline measures to after 12 weeks of PANDAS/PANS treatment with NTI164 to correlate and assist in identifying relevant biomarkers of the disease.

“We’re aiming to apply for an Orphan Drug Designation for NTI164 associated with the treatment of PANDAS/PANS in the US and EU.

“We’re expecting results from our phase 1/2 trial investing the use of NTI164 in children with Rett Syndrome in Q1 2024.”

 

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.