Investor Presentation
2024-04-17 · Neurotech International Limited · original investi.com.au ↗
Investor Presentation: Autism Phase II/III Clinical Trial Results Rett Syndrome Phase I/II Clinical Trial Top-Line Results Capital Raise Improving Lives 17 April 2024 Dr Tom Duthy Executive Director Disclaimer Purpose of presentation: This presentation (including this document, any related video or oral presentation, any question and answer session and any written or oral material discussed or distributed in relation to this presentation) has been prepared by Neurotech International Limited (ACN 610 205 402) (Neurotech or Company). It has been prepared for the sole purpose of providing general information on Neurotech and its business. Not an offer or solicitation: This presentation is not investment advice nor an offer to subscribe for securities or otherwise invest in Neurotech, and it should not be relied upon to make any investment decision. 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Professional advice: Recipients of this presentation should consider seeking appropriate professional financial, taxation and legal advice in reviewing the presentation and all other information with respect to Neurotech and evaluating its business, financial performance and operations. Proprietary information and copyright: This presentation and the information it contains is proprietary to Neurotech. Neurotech holds the copyright in this presentation. Except as permitted under the Copyright Act 1968 (Australia), this paper or any part thereof may not be reproduced without its written permission. IMPORTANTINFORMATION 2 Autism Spectrum Disorder (ASD) Goals 3 “The goals of treatment for ASD are to improve core deficits in social communication and social interactions and minimize the impact of restricted behaviours, with an overarching goal to help children develop greater functional skills and independence.” 1 1. Weitlauf AS, McPheeters ML, Peters B, et al. Therapies for Children With Autism Spectrum Disorder: Behavioural Interventions Update. Rockville (MD): Agency for Healthcare Research and Quality (US); 2014 Aug. (Comparative Effectiveness Review, No. 137.) Introduction. PREVALENCE OF ASD ~1 in 44 children in the US1 Current TreatmentMarket ASD is a serious neuro inflammatory developmental disorder that impairs the ability to communicate & interact Common symptoms; behavioural issues, agitation, repetitive movements, inability to focus & compulsive neurological patterns Clinical Trial Huge unmet medical need - patients need better treatment Current drugs have numerous side effects; weight gain, breast tissue development, nausea, dry mouth, anxiety, irritability, insomnia, stomach pain & movement disorders 2 Approved Drugs (* limited use) Risperidone, Aripiprazole 1. www.cdc.gov/ncbddd/autism/addm.html 2. https://www.fortunebusinessinsights.com/industry-reports/autism-spectrum-disorder-therapeutics-market-101207-CAGR-of-7-4.html Autism Spectrum Disorder (ASD) 4 2 TREATMENT MARKET SIZE US$2.0bn Initial Focus of NTI164 – A full spectrum, oral cannabinoid biopharmaceutical product Initial Phase I/II data positive at 4 weeks, 20 weeks and 52 weeks…safety past 90 weeks No FDA-approved drugs for the core symptoms of ASD (i.e. social communication, social interaction, restricted behaviours) SIGNIFICANT UNMET MEDICAL NEED 1. The Australian, 25 October, 2022- https://www.afr.com/politics/federal/how-the-ndis-will-blow-out-to-50b-in-four-charts-20221019-p5br1c Source: NDIS data 30 June 2023 ASD and the NDIS 5 The National Disability Insurance Scheme (NDIS) provides assistance to people with a disability, as well as their families and carers $35.5 Billion Cost of NDIS in 2022, to increase to $52 billion by 2026, $100 billion by 20331 There is a strong market need for an effective therapeutic intervention such as NTI164 to improve ASD symptoms & reduce healthcare costs 230,119 as at 31 December 2023 NTI164 ASD Program Highlights The Program 6 28 Day Data Released 8 July 2022 20 Week Data Released 26 October 2022 52 Week Data Released 17 March 2023 First in human Phase I/II ASD paediatric study (open-label n=14) Commenced in May 2021 at Monash Children’s Hospital led by A/Prof. Michael Fahey 90 Week Safety Data Released 7 Feb 2024 Phase II/III ASD paediatric study (double-blind) n=54 Commenced in December 2022 at Monash Children’s Hospital led by A/Prof. Michael Fahey Completed recruitment in December 2023 Last patient, last visit in April 2024 Strength of data facilitated a cap raise of $9.0m to fund a larger study ASD Trial Design (NTIASD2) 7 1. DAYBUE is a trademark of Acadia Pharmaceuticals Inc 2. Pending data Primary Endpoint Secondary Endpoints • Clinical Global Impression – Severity of illness (CGI-S) Entourage Effect Neuroprotective Anti- Neuroinflammatory High potency, Broad Spectrum Cannabinoid Formulation in Oil, C. sativa L. (Plant Derived) • Vineland -3 (adaptive behaviours measure) • Clinical Global Impression – Improvement (CGI-I) • Social Responsiveness Scale, 2nd Edition (SRS-2), • Safety • Change in Anxiety, Depression and Mood Scale (ADAMS)2 Enrolment Placebo NTI164 Randomise / Double-Blind Unblind NTI164 Maximum Tolerated Dose Maximum Tolerated Dose Extension Phase -- Maximum Tolerated Dose Baseline Week 8 Week 12 Week 16 Week 18 Wash-Out Wash-Out Week 52 End of Study End of Study Wash-Out Week 54 End of Study Baseline Patient Characteristics 8 Characteristic Number (%) / Mean NTI164 (n=26) Placebo (n=28) P Value Age 12.4 years 12.0 years 0.442 Sex Male 14 (54%) 15 (54%) 0.982 Female 12 (46%) 13 (46%) CGI-S1 Overall Score 5.54 (100%) 5.21 (100%) 0.106 Mild Pts 1 (4%) 1 (4%) N/A Moderate Pts 2 (8%) 9 (32%) N/A Marked Pts 11 (42%) 5 (18%) N/A Severe Pts 12 (46%) 13 (46%) N/A 1. Clinical Global Impression (CGI)- is a physician/observer-rated scale synthesizing the clinician’s impression of the global state of an individual & frequently employed in clinical trials for neuropsychiatric disorders. CGI-S reflects clinician’s impression of severity of illness on a 7-point scale ranging from 1 = not at all to 7 = among the most extremely ill. N/A – not available A total of 54 patients evaluable for Primary Endpoint Summary of Efficacy Measures 9 Primary Endpoint CGI-S Key Secondary Endpoints Vineland-3 CGI-I Significant treatment effect 3.23 (95% CI; 0.44, 6.0) versus placebo at 8 weeks (p=0.024) Significant treatment effect -1.65 (95% CI; -2.3, -1.0) versus placebo at 8 weeks (p<0.001) Clinical Global Impression- Severity of Iillness (CGI-S)CGI-S is a single-item, 7-point scale by clinicians designed to assess global impression of severity (1=normal, 7 = severely ill). Clinical Global Impression – Improvement (CGI-I) is a 7−point scale that reflects experts' clinical judgment of the patient based on the clinician's total experience with the ASD population graded from 1 (very much improved) to 7 (very much worse). A decrease in CGI-I score indicates improvement. Vineland -3 is internationally recognised as a leading instrument for supporting the diagnosis of intellectual and developmental disabilities in ASD; specifically adaptive behaviour. Adaptive functioning, Social Responsiveness Scale (SRS -2) is internationally recognised as a leading instrument (65 items) for identifying the presence and severity of social impairment within the Autism spectrum Significant treatment effect -1.42 (95% CI; -2.0, -0.82) versus placebo at 8 weeks (p<0.001) SRS Significant treatment effect -3.064 (95% CI; -5.781, -0.348) versus placebo at 8 weeks (p=0.028) 1. www.cdc.gov/ncbddd/autism/addm.html 2. https://www.fortunebusinessinsights.com/industry-reports/autism-spectrum-disorder-therapeutics-market-101207-CAGR-of-7-4.html Expert Interpretation of Results 10 Professor Michael Fahey – Lead Investigator “The analysis so far of the trial, which compared NTI164 to placebo over 8 weeks of daily treatment, have demonstrated statistically significant and clinically meaningful improvements in the severity of illness and adaptive behaviours such as communication and socialisation without any significant side effects. Currently, there are no FDA or TGA- approved treatments that show clinically significant improvements in one or more of autism's three core symptom domains: communication, impaired social interaction, and restricted behaviours. Therefore, the NTIASD2 clinical trial data look promising, given the substantial unmet market need for safe and effective therapies for autism, like NTI164.” 8 Week Safety Data 11 NTI164 Exhibits Excellent Safety Over 8 Weeks Conclusion: NTI164 exhibits an excellent safety profile and minimal patient-specific side-effects No serious adverse events (SAEs) recorded for NTI164 & placebo, across entire period (8 weeks) Adverse Events (AEs) were tolerated and manageable (total of 11 AEs across 7 patients for both arms) A total of 54 patients evaluable at 8 weeks Nausea/Vomiting Diarrhoea Kidney/Liver Function Vital Signs Blood Chemistry • 2 pts (8%) (NTI164) • 3 pts (11%) (Placebo) • 0 pts (0%) (NTI164) • 2 pts (8%) (Placebo) Normal Primary Endpoint: CGI-S CGI-Severity of illness versus placebo at 8 weeks1 (p <0.001) 12 Clinical Interpretation • Placebo group showed no improvement at week 8 (1.8% worse) • 28% improvement for NTI164 v placebo at 8 weeks, 32% v baseline • Significant down-staging of patient’s illness severity – 88% pts markedly/severely ill at baseline in the NTI164 arm 1. Clinical Global Impression (CGI)- is a physician/observer-rated scale synthesizing the clinician’s impression of the global state of an individual & frequently employed in clinical trials for neuropsychiatric disorders. CGI-S reflects the clinician’s impression of severity of illness on a 7-point scale ranging from 1=Normal to 7=among the most extremely ill. 5.21 5.25 5.54 3.77 Baseline Week 8 Mean Severity of Illness (n=54) Placebo NTI164 1 2 3 4 5 6 7 Severity of illness Scale (CGI-S) Baseline (NTI164) Week 8 (placebo) Week 8 (NTI164) Baseline (placebo) Mean difference: –1.48 Treatment effect: -1.65 (p<0.001) Secondary Endpoint: CGI-I CGI-Global improvement 1 13 1. Clinical Global Impression (CGI) - is a physician/observer-rated scale synthesizing the clinician’s impression of the global state of an individual & frequently employed in clinical trials for neuropsychiatric disorders. Clinical Global Impression – Improvement (CGI-I) is a 7−point scale that reflects experts' clinical judgment of the patient based on the clinician's total experience with the ASD population graded from 1 (very much improved) to 7 (very much worse). A decrease in CGI-I score indicates improvement. Clinical Interpretation • 1.42 mean improvement between NTI164 and placebo at 8 weeks (36%) • 46% of NTI164 patients very much or much improved v 4% for placebo 8%4% 38%39% 38% 29% 15% 14% 7% 7% Placebo NTI164 Very much Improved Much Improved Minimally Improved No Change Minimally Worse Much Worse Very Miuch Worse 1 2 3 4 5 6 7 - 1(4%) 11 (39%) 8 (29%) 4 (14%) 2 (7%) 2 (7%) 2 (8%) 10 (38%) 10 (38%) 4 (15%) - - - Very Much Improved Much Improved Minimally Improved No Change Minimally Worse Much Worse Very Much Worse Scale Placebo (week 8) Clinical Global Impression – Improvement (CGI-I) is a 7−point scale that reflects experts' clinical judgment of the patient based on the clinician's total experience with the ASD population graded from 1 (very much improved) to 7 (very much worse). A decrease in CGI-I score indicates improvement. NTI164 (week 8) 4.0 2.62 4.0 4.04 2.5 3.0 3.5 4.0 4.5 Baseline Week 8 CGI-I (n=54) NTI164 Placebo Improved Worse CGI-I at 8 weeks (p<0.001) Week 8 CGI-I Mean difference: –1.42 Treatment effect: -1.42 (p<0.001) Secondary Endpoint: Vineland -3 Vineland -31 14 1. Vineland -3 is internationally recognised as a leading instrument for supporting the diagnosis of intellectual and developmental disabilities in ASD; specifically adaptive behaviour. Adaptive functioning, which are skills people need to function independently at home, at school and in the community is an important factor in predicting long-term outcomes for people with ASD. Improving adaptive abilities in patients is therefore a desirable treatment goal. The adaptive behaviour composite consists of (a) communication, (b) daily living skills & (c) socialisation. Clinical Interpretation • No Secondary endpoints were statistically powered for this trial • Adaptive behaviour improvement is a treatment goal in ASD • Statistical significance reached for adaptive behaviour composite and all three sub-domains Standardised measure of adaptive behaviour Norm-based: adaptive functioning compared to others of same age Excellent test, re-test reliability & between rater (clinician, parent) Vineland-3 Domain 8 week measure Treatment Effect P-value Adaptive behaviour composite 3.23 0.0240 Communication 2.92 0.0467 Daily living skills 3.56 0.0213 Socialisation 3.47 0.0475 Data Comparison & Context - Risperidone 15 1. Kent, et al. Risperidone Dosing in Children and Adolescents with Autistic Disorder: A Double-Blind, Placebo-Controlled Study. Journal of autism and developmental disorders. 2012. 43. 10.1007 2. A Study to Evaluate the Efficacy and Safety of Risperidone (R064766) in Children and Adolescents With Irritability Associated With Autistic Disorder, 2015 3. Ghaeli P et al. Effects of risperidone on core symptoms of autistic disorder based on childhood autism rating scale: an open label study. Indian J Psychol Med. 2014 Jan;36(1):66-70. 4. McDougle CJ, et al.. Risperidone for the core symptom domains of autism: results from the study by the autism network of the research units on pediatric psychopharmacology. Am J Psychiatry. 2005 Jun;162(6):1142-8 CGI-Severity of illness RISPERIDONE NTI164 Phase I/II (n=11) Vineland -31 CGI- Improvement Vineland -3 Safety • (n=96): -1.0 from baseline at 12 months1 • (n=38): -0.7 from baseline at 48 weeks2 • -1.1 change at 20 weeks (p=0.005), 26% improvement • -1.3 change at 52 weeks (p=0.032) • ~40% of subjects markedly or severely ill at baseline – 0% from week 4 onwards • At 20 weeks, mean result: 100% mildly ill • -1.48 change v placebo at 8 weeks, 28% improvement • Treatment effect of -1.6 (p<0.001) • 88% of subjects markedly or severely ill at baseline – 27% at 8 weeks • 19% borderline ill at 8 weeks • (n=15): CGI-I changes after 8 weeks from baseline3 • 27% - very much improved • 47% - much improved • 20% - minimal improved • 6.6% - no change • 100% of active patients showed improvement after 20 weeks of daily treatment with NTI164 • 100% patients much Improved at 20 weeks • 90% of patients much Improved at 52 weeks 10% very much improved) • 86% of patients showed improvement at 8 weeks of daily treatment with NTI164 v 43% placebo • 46% of NTI164 patients very much or much improved v 4% for placebo • Near absence of RCTs examining Vineland noted in the medical literature • No impact on social interaction and communication4 • Adaptive behaviour mean difference of 3.8 (p=0.0005) at 20 weeks and mean difference 6.4 at 52 weeks (p=0.028) • Highly significant improvement • Highly significant improvements also in domains of communication, daily living, socialisation at 20 weeks and 52 weeks (ex-socialisation) • Adaptive behaviour treatment effect 3.23 v placebo (p=0.024) • Highly significant improvement • Highly significant improvements also in domains of communication, daily living, socialisation by 8 weeks • Significant weight gain Increase in BMI by 0.621 • Weight gain2 • Increase in appetite, sedation3 • No change to weight • No change to appetite • Mild nausea, stomach pain • Nausea / Vomiting (8% pts) • No diarrhoea RCT- randomised controlled trial; BMI – Body Mass index “The goals of treatment for ASD are to improve core deficits in social communication and social interactions and minimize the impact of restricted behaviours, with an overarching goal to help children develop greater functional skills and independence.” NTI164 Phase II/III (n=54) Secondary Endpoint: SRS 16 Clinical Interpretation • Clinically meaningful and statistically significant treatment effect at 8 weeks • Reinforces positive impacts of NTI164 on an important core symptom of ASD - social behaviours, including communication SRS -21 1. Social Responsiveness Scale (SRS -2) is internationally recognised as a leading instrument (65 items) for identifying the presence and severity of social impairment within the Autism spectrum and differentiates it from that which occurs in other disorders. The SRS-2 total score is the most reliable measure for social deficits related to ASD. SRS-2 is distinct from other measures in that it provides a continuous measure of social ability (from impaired to above average) instead of a categorical yes/no identification of ASD impairments. High scores are associated with more severe social impairments. SRS-2 is a valid and reliable quantitative measure of core ASD symptoms related to social impairment. SRS-2 Domain (8 week measure) Treatment Effect P-value Total Score -3.064 0.028 Children with autism spectrum disorder have difficulty with social interaction behaviours, including establishing and maintaining relationships, reciprocating social interaction, and communicating with others. SRS-2 is a validated measurement tool of assessing these factors Conclusions 1. Change in Anxiety, Depression and Mood Scale (ADAMS) not yet available 17 Met Primary Endpoint NTI64 has demonstrated a statistically significant and clinically meaningful improvement in ASD across multiple measures of assessments relating to severity of illness, drug-related improvement, adaptive behaviours and socialisation Met All Secondary Endpoints1 NTI164 Very Safe No serious adverse events. Small number of adverse events, relating to nausea/vomiting (formulation-related) and no diarrhoea observed in NTI164 arm. None of these adverse events were serious and were not considered to significantly interfere with the patient’s functioning and none of the adverse events required any additional medications (i.e. anti-nausea, anti-diarrhoea). Huge Unmet Need No FDA, TGA or EMA-approved drugs for the core symptoms of ASD (i.e. social communication, social interaction, restricted behaviours) Rett Syndrome Phase I/II Trial 18 “Caregivers of children with RTT experience the illness as being like an “obstacle course”, where they must continuously overcome hurdles. These include hindrances for finding responses to their symptoms and achieving a diagnosis, for managing the treatment and daily care, and for finding the essential financial resources to meet all the expenses generated by the illness.”1 1. Palacios-Ceña D, Famoso-Pérez P, Salom-Moreno J, Carrasco-Garrido P, Pérez-Corrales J, Paras-Bravo P, Güeita-Rodriguez J. “Living an Obstacle Course”: A Qualitative Study Examining the Experiences of Caregivers of Children with Rett S yndrome. International Journal of Environmental Research and Public Health. 2019; 16(1):41 About Rett Syndrome 19 First Ever Approval About Neuroinflammation • Neuren Pharmaceuticals (ASX:NEU) / Acadia Pharmaceuticals (NASDAQ:ACAD): FDA Approval 10 March 2023 • Sets benchmark for FDA accepted clinical endpoints, safety and tolerance • Rare genetic neurological and developmental disorder and is almost exclusively the result of a mutation(s) in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome: impaired brain development and function • Currently there is no cure for people with Rett syndrome and classified as a “rare/orphan disease” (by definition, less than 200,000 affected individuals in the US) by the Office of Rare Diseases of the National Institutes of Health • Numerous scientific reports support neuroinflammatory effects in Rett Syndrome • NTI164 shown to exhibit anti-neuroinflammation and neuroprotective effects in vitro 1. https://www.livewiremarkets.com/wires/a-de-risked-biotech-with-4x-upside 2. https://reverserett.org/about-rett-syndrome/ Rett Syndrome Market Dynamics 20 Source: Acadia Pharmaceuticals, Neuren Pharmaceuticals Nov 2023 Investor Presentation, International Rett Syndrome Foundation, Rett Syndrome Association of Australia, Livewire Markets, Company Analysis Significant Market • First FDA approved therapy (March 2023) • Est. drug cost to patient ~US$1,000 per day. US$87 million in Q4 CY2023 (US$177m in CY2023) net sales • Q3: 800 patient starts (4,500 registered with Rett, ~18% penetration) – strong demand highlights urgent market need • CY2024 sales est. US$370m – US$420m Single Approved Therapy Valuation/Pricing Benchmarks • Neuren (ASX:NEU) license deal with Acadia (NASDAQ:ACAD) close to US$1 billion for trofinetide (*inc other indications) • 80% covered lives for DAYBUE from US payers within 6 months – rapid reimbursement adoption • Market approval via single Phase 3 clinical trial v placebo (“Lavender” – 187 pts), with open-label extension (“Lilac” – 154 pts) • 17-26k patients in USA, Europe, Japan, Australia • Est. US$2 billion annual market opportunity • Narrow range of Rett specialist clinicians: focused prescriber group • Concentrated market dynamics: 18 Rett Centres of Excellence in the US (3 in AU) • No approved Rett drugs in Europe, Japan and Australia (USA:1) 6,000-9,000 9,000-14,000 2,000-3,000 200 Safety Data of Interest 21 Safety Over 12 Weeks: Key Focal Points Weight Loss Nausea/Vomiting Diarrhoea Kidney/Liver Function Vital Signs Blood Chemistry All normal across Neurotech’s previous ASD (90 weeks+) and PANDAS/PANS (24 weeks) Studies 82% pts 29% pts 12% pts with >7% weight loss 1. DAYBUE data sourced from Acadia Pharmaceuticals Inc No significant weight change noted for Neurotech ASD & PANDAS/PANS Phase I/II trials 5% of pts for ASD (20 weeks) and 13% of pts for PANDAS/PANS (12 weeks) for NTI164 8% NTI164 v 11% placebo in Phase II/III ASD 0% reported in Neurotech ASD Phase I/II and Phase II/III (placebo 8%) and PANDAS/ PANS trials Rett Syndrome Trial Design (NTIRTT1) 22 1. DAYBUE is a trademark of Acadia Pharmaceuticals Inc & is not approved in Australia Primary Endpoint Secondary Endpoints • Clinical Global Impression – Improvement (CGI-I) • Rett Syndrome Behaviour Questionnaire (RSBQ) • Rett Syndrome: Symptom Index Score (RTT-SIS) • RTT- Clinician Domain Specific Concerns – Visual Analog Scale (RTT-DSC-VAS) • Communication and Symbolic Behaviour Scales Developmental Profile Infant-Toddler Checklist (CSBS-DP-IT Social) • Impact of Childhood Neurological Disability Scale (ICND) • RTT Caregiver Burden Inventory (RTT-CBI) • Overall Quality of Life Rating of the Impact of Childhood Neurological Disability Scale (ICND-QoL) • CGI-severity of illness (CGI-S) • Safety * No participants received DAYBUE (trofinetide)1 Entourage Effect Neuroprotective Anti- Neuroinflammatory High potency, Broad Spectrum Cannabinoid Formulation in Oil, C. sativa L. (Plant Derived) All patients completed 12 weeks i.e. no adverse events requiring withdrawal from the study * Available * Pending * Pending Top-Line Clinical Results 23 CGI-I Primary Endpoint Significantly Improved CGI-I versus baseline mean difference of -0.3 (95% CI -0.015, -0.56; p = 0.04) Data compares favourably to DAYBUE (trofinetide) Phase 3 data v placebo (mean difference -0.3, p=0.003)1 [DAYBUE CGI-I reported – 61% of patients “Unchanged”, 25% of patients “Minimally Improved”, 13% of patients “Much Improved”] 1. Neul, J.L. et al. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med 29, 1468–1475 (2023). Clinical Global Impression (CGI) - is a physician/observer-rated scale synthesizing the clinician’s impression of the global state of an individual & frequently employed in clinical trials for neuropsychiatric disorders. CGI- Improvement (CGI-I) is a 7-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention (1 – “Very Much Improved” 2 - “Much Improved” 3 - “Minimally Improved” 4 - “No Change” 5 - “Minimally Worse” 6 - “Much Worse” 7 - “Very Much Worse”) . A total of 14 patients evaluable at 12 weeks Data analysis and interpretation remains ongoing for further primary endpoint analysis, reporting of secondary endpoints (inc. RSBQ) and safety/tolerability Neurotech on-track to report further clinical trial data in the next 2-4 weeks Capital Raise 24 Pro-Forma Capital Structure 25 PRO-FORMA CAPITAL STRUCTURE - $10 Million Placement Current Shares on Issue 917.4M New Shares 100.0M Pro-Forma Shares on Issue 1017.4M Pro-Forma Cash1 $15.5M Pro-Forma Enterprise Value (undiluted)2 $86.2M 1. Cash at 31 December 2023 - $4.5 million, Option exercises - $1.5m; Placement of $10.0m minimum, transaction costs of $(0.5m) 2. Placement price shares on issue post placement less pro-forma net cash position post placement (no debt) 26 Timetable* * Timetable is indicative and subject to change Settlement of Placement Shares & Unlisted Options 23 April 2024 Allotment of Placement Shares on ASX & Commencement of Trading 24 April 2024 Allotment of Unlisted Options 24 April 2024 Event Date Use of Funds 27 EVENT ALLOCATION OF FUNDS (PLACEMENT) Further human clinical trials NTI164 (Rett, PANS, Other) $5.5M Regulatory Development $0.5M IND Enabling Toxicology $2.4M Manufacturing / Production Expansion $1.0M Offer Costs / Other $0.6M TOTAL $10.0M * In addition, the Company is entitled to 43.5% to 48.5% of eligible R&D expenditure returned in cash through the Australian government R&D Tax Incentive Key Milestones – NTI164 28 1H CY2024 2H CY2024 • Orphan Drug Designation Europe – Rett Syndrome • Orphan Drug Designation Europe – PANDAS/PANS • Orphan Drug Designation USA – Rett Syndrome • Orphan Drug Designation USA – PANDAS/PANS • Commence Phase I/II Cerebral Palsy Clinical Trial • FDA IND / EMA2 toxicology • Presentation of Phase I/II Rett Syndrome data at international Rett meeting • HREC/TGA Approval Cerebral Palsy Phase I/II Clinical Trial • 24-week PANDAS/PANS Phase I/II Clinical Trial Data • Rett Syndrome Phase I/II (14 girls) 52-week Extension HREC Approval • Results of ASD Phase II/III Clinical Trial • Top-line Rett Syndrome Phase I/II Clinical Trial data • Results of Rett Syndrome Phase I/II Clinical Trial – full data • Meeting outcome – TGA1 Regulatory Advice • Publications for ASD Phase I/II + pre-clinical NTI164 results • Metabologenomic data from Phase I/II PANDAS/PANS Clinical Trial 1. Therapeutic Goods Administration (TGA) 2. Food and Drug Administration (FDA), European Medicines Agency (EMA) ENDS 29 Neurotech: Strategies, Pipeline, Milestones, Outlook 30 NTI164 exclusive worldwide licence for neurological disorders Patents Pending – Use, Composition Focus on Paediatric Patients Multiple Phase I/II and Phase II/III Clinical Trials Neurotech is a clinical -stage biopharmaceutical development company focused predominately on paediatric neurological disorders 31 Novel oral biopharmaceutical cannabinoid platform (NTI164) Supportive Efficacy & Safety Data in Children Neurotech Four Core Strategies 32 Focus on Partnering with Key Opinion Leaders / Clinicians Focus on Paediatric Patients Focus On Drug Product Development Focus On Rare Neurological Disorders with Neuroinflammation Therapeutic Agent: NTI164 33 High potency, Broad Spectrum Cannabinoid Formulation in Oil, C. sativa L. (Plant Derived) THC < 0.3% Major constituent Cannabidiolic acid (CBDA) Minor constituents include other cannabinoids: CBD, CBG, CBGA, other + terpenes Convenient 1x or 2x (split dose) oral formulation in oil, ideal format for pediatric patients 20mg/kg (CBDA) Entourage Effect Neuroprotective Anti- Neuroinflammatory NTI164 is not a low dose CBD oil to be sold over- the-counter Our Target Markets 34 Autism Spectrum Disorder (ASD) Rett Syndrome PANDAS/PANS Cerebral Palsy (CP) US$2 billion US$1.4 billion1 US$4.3 billionUS$2 billion* 1. Neurotech Estimate based on: Wald ER, et al. Estimate of the incidence of PANDAS and PANS in 3 primary care populations. Front Pediatr. 2023 Sep 21; EU/UK: 8,000 pts / US: 6,000 pts <18 years based on annual intravenous immunoglobulin (IVIG) cost of ~US$100k (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8019941/) • Prevalence of ~15,000 patients in the US • 1 Approved Drug • Trofinetide • Incidence of ~6,000 patients <18 yr. in the US1 • No FDA/EMA Approved Drug • Incidence of ~500,000 <18 yr. patients in the US • 2 Approved Drugs for spastic CP • Baclofen, Botox • Prevalence of ~2.0M <18 yr. patients in the US • 2 Approved Drugs (* limited use) • Risperidone, Aripiprazole Orphan Orphan Lack of effective therapies, significant unmet medical need Annual Drug Therapy Market opportunity Contact Details Dr Tom Duthy Executive Director [email protected] +61 402 493 727 www.neurotechinternational.com Neurotech International Limited (ASX: NTI) 35 *This presentation has been authorised by the Board of Neurotech International Limited
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