\u00a0<\/p>\n
Neurotech has a clear path for development and commercialisation of NTI164 to treat PANDAS\/PANS<\/strong> \u00a0<\/p>\n Neurotech International achieved a key milestone with positive results from its Phase I\/II clinical trial into <\/strong>conditions where children go to bed perfectly normal but wake up with uncontrollable tic movements, become severely obsessive compulsive with heightened anxiety and depression.<\/strong><\/p>\n Neurotech International (ASX:NTI)<\/strong><\/a> 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\u00a0 streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).<\/p>\n 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.<\/p>\n NTI\u2019s proprietary NTI164\u00a0drug formulation is derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN.<\/p>\n 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.<\/p>\n 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.<\/p>\n It has also secured HERC approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) for investigations in\u00a0treating female Rett Syndrome patients<\/strong><\/a>.<\/p>\n Executive director Dr Thomas Duthy discusses the latest results and its commercialisation strategy for NTI164.<\/p>\n Q: How important are the results of the PANDAS\/PANS trial to the company?<\/strong><\/p>\n A: \u201cThis is a significant milestone for the company,\u201d Duthy says.<\/p>\n \u201cThis is also a huge step forward in Neurotech\u2019s 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.<\/p>\n \u201cOur 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.<\/p>\n \u201cThe treatment was also well-tolerated by the patients involved in the trial with no serious adverse events recorded.<\/p>\n \u201cIt is important to add that all 15 participants elected to remain on the therapy at the conclusion of the trial.<\/p>\n \u201cPANDAS\/PANS is a complex disorder with no approved therapy or specific drug, hence why NTI is pursuing its orphan drug approach.<\/p>\n \u201cThis presents an exciting potential commercial opportunity for Neurotech.<\/p>\n \u201cThe company has a clear path forward for the development and commercialisation of NTI164 as a treatment for PANDAS\/PANS.<\/p>\n \u201cGiven 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.\u201d<\/p>\n Q: What are the symptoms of PANDAS\/PANS and how does NTI164 help? <\/strong><\/p>\n A: \u201cPANDAS\/PANS are conditions that are characterized by sudden onset neuropsychiatric symptoms in children,\u201d Duthy says.<\/p>\n \u201cThe 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.<\/p>\n \u201cMotor and\/or vocal tics, such as repetitive movements or sounds, may appear suddenly or worsen in intensity.<\/p>\n \u201cChildren may experience mood swings, irritability, anxiety, depression, or aggression that are out of character for them.<\/p>\n \u201cChildren may also regress and experience a sudden loss of previously acquired\u00a0 skills, such as handwriting, toileting, or verbal communication.<\/p>\n \u201cChildren with PANDAS\/PANS may exhibit excessive and irrational fears, as well as difficulty being separated from their caregivers.<\/p>\n \u201cThey may have sleep disturbances such Insomnia, frequent awakenings, night terrors, or bedwetting.<\/p>\n \u201cSensory issues where children become hypersensitive or hyposensitive to sensory stimuli, leading to difficulties with processing sensory information can be a symptom.<\/p>\n \u201cIn a world first, NTI successfully demonstrated that its lead biopharmaceutical product, NTI164 was able to reduce and alleviate some of \u00a0the main symptoms of PANDAS\/PANS as described.<\/p>\n \u201cThese changes and improvements were deemed to considerably improve the quality of life of participants.<\/p>\n \u201cNTI164 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.<\/p>\n Q: Were any major side effects experienced by patients during the trial?<\/strong><\/p>\n A: \u201cWe were extremely pleased with the safety data from the trial,\u201d Duthy says.<\/p>\n \u201cThere 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.<\/p>\n \u201cIt is likely that most of these occurrences are from the taste and volume of medication that the participants ingest.\u201d<\/p>\n Q: Are you expecting any further results from the trial?<\/strong><\/p>\n A: \u201cYes, we are awaiting further data from Professor R Dale who is undertaking a series of molecular mechanism of action studies,\u201d Duthy says.<\/p>\n \u201cHe 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.<\/p>\n \u201cIf 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.<\/p>\n \u201cThese studies will also assist the company select the most appropriate disorders to treat.\u201d<\/p>\n Q: How will securing orphan drug designation help Neurotech commericalise NTI164?<\/strong><\/p>\n A: \u201cOrphan 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,\u201d Duthy says.<\/p>\n \u201cIt was established to encourage research into rare diseases and encourage corporates to commercialise drugs for such indications.<\/p>\n \u201cWe believe that PANDAS\/PANS meets the criteria of a rare disease under the FDA and EMA guidelines for an orphan drug.<\/p>\n \u201cOrphan Drug Designation will also allow Neurotech to gain significant benefits, such as market exclusivity periods,\u00a0 financial incentives, reduced clinical requirements and assistance in the drug development process.<\/p>\n \u201cThere\u00a0 have\u00a0 been\u00a0 recent\u00a0 ASX\u00a0 examples\u00a0 of\u00a0 biotech\u00a0 companies\u00a0 successfully\u00a0 demonstrating\u00a0 the commercial significance of an Orphan Drug development strategy, including Neuren Pharmaceuticals (ASX:NEU)<\/strong><\/a>.<\/p>\n \u201cNeuren has successfully commercialised a drug called Trofinetide which secured multiple\u00a0 orphan drug designations and subsequently achieved FDA approval in 2023. Neuren has a market capitalisation now of over $1.3 billion.\u201d<\/p>\n Q: What is Neurotech\u2019s strategy for commercialising NTI164?<\/strong><\/p>\n A: \u201cNeurotech\u2019s short term strategy is to generate clinical evidence of safety and benefit for NTI164 across multiple paediatric\u00a0 neurological disorders to add substantial value to our\u00a0 NTI164\u00a0 therapeutic platform,\u201d Duthy says.<\/p>\n \u201cIn 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.<\/p>\n \u201cThe 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.<\/p>\n Q: What company catalysts can investors look forward to over the coming months?<\/strong><\/p>\n A: \u201cThere are several key objectives and future milestones for the company,\u201d Duthy says.<\/p>\n \u201cThere\u2019s results of the landmark 54 patient Phase II\/III double blinded placebo-controlled ASD trial \u00a0which is nearing completion of patient recruitment at Monash Children\u2019s Hospital with results\u00a0 anticipated in Q1 2024.<\/p>\n \u201cThere\u2019s 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.<\/p>\n \u201cWe\u2019re aiming to apply for an Orphan Drug Designation for NTI164 associated with the treatment of PANDAS\/PANS in the US and EU.<\/p>\n \u201cWe\u2019re expecting results from our phase 1\/2 trial investing the use of NTI164 in children with Rett Syndrome in Q1 2024.\u201d<\/p>\n \u00a0<\/p>\n This article was developed in collaboration with Neurotech International, a Stockhead advertiser at the time of publishing. <\/em><\/p>\n This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.<\/em><\/p>\n \u00a0<\/p>\n <\/span><\/p>\n The post Q+A: Neurotech\u2019s Dr Thomas Duthy on commercialising NTI164 as a treatment for PANDAS\/PANS<\/a> appeared first on Stockhead<\/a>.<\/p>","protected":false},"excerpt":{"rendered":"
\nWith no approved therapy for PANDAS\/PANS Neurotech is pursing orphan drug designation for NTI164<\/strong>
\nResults of Neurotech\u2019s phase 1\/2 trial investing use of NTI164 in children with Rett Syndrome in Q1 2024<\/strong><\/p>\n