Neurotech International Ltd. · ASX:NTI
Neurotech International — Investor Presentation (May 2024)
27 slides from Neurotech International's investor presentation, May 6, 2024
Slide 1: Rett Syndrome Clinical Trial Results
For personal use only Improving Lives Rett Syndrome Clinical Trial Results Dr Tom Duthy Executive Director 6 May 2024
Slide 2: Disclaimer
Disclaimer IMPORTANT INFORMATION Purpose of presentation: This presentation (including this document, any related video or oral These events are, as at the date of this presentation, expected to take place, but there cannot be any guarantee that such will occur as anticipated, or at all, given that
Slide 3: Neurotech Four Core Strategies
Neurotech Four Core Strategies For personal use only Focus On Rare Focus on Paediatric Patients Neurological Disorders with Neuroinflammation Focus on Partnering with Focus On Drug Product Key Opinion Leaders / Development Clinicians 3
Slide 4: Clinical Pipeline – 2024
Clinical Pipeline – 2024 Pre-Clinical Phase I/II Phase II/III For personal use only NTI164 NTI164 NTI164 Combination Therapies Prednisone, Diclofenac, Other Cerebral Palsy ASD Other Licensed NTI164 Strains PANDAS / PANS1 Pipeline (2020/1) NTI164 ASD NTI164 Combination Therapies (90 week+ open label
Slide 5: Rett Syndrome Phase I/II Trial
Rett Syndrome Phase I/II Trial “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 c
Slide 6: Rett Syndrome Trial Design (NTIRTT1)
Rett Syndrome Trial Design (NTIRTT1) Primary Endpoint For personal use only • Clinical Global Impression – Improvement (CGI-I) All patients completed 12 weeks i.e. no adverse events requiring withdrawal from the study Secondary Endpoints • Rett Syndrome Behaviour Questionnaire (RSBQ) All patients en
Slide 7: Baseline Patient Characteristics
Baseline Patient Characteristics Characteristic Number (%) / Mean Age 8.8 years For personal use only Weight 27.5 kg Sex Female 14 (100%) Mean 4.6 (100%) A total of 14 female patients CGI-S Moderate (4) 7 (50%) with Rett Syndrome Marked (5) 5 (36%) participated Severe (6) 2 (14%) Mean 44.6 (100%) RS
Slide 8: NTI164 Exhibits Excellent Safety Over 12 Weeks
12 Week Safety Data A total of 14 patients NTI164 Exhibits Excellent Safety Over 12 Weeks evaluable at 12 weeks One serious adverse event (SAE) recorded (Urticaria-hives) Across all doses, across entire period (12 weeks) For personal use only Adverse events (AEs) were tolerated and manageable 11 AEs
Slide 9: Summary of Efficacy Measures
Summary of Efficacy Measures CGI-I Co-primary endpoints used for FDA Primary Endpoint approval in trofinetide Phase 3 trial RSBQ CGI-I Improvement of 10% from Baseline at 12 weeks (p=0.009) – 9 exploratory Rett-Specific Anchors Improvement of 23% from Baseline at 12 weeks (p=0.001) – 4 core Rett Anc
Slide 10: Clinician Expert View on Results
Clinician Expert View on Results Associate Professor Carolyn Ellaway – Lead Investigator For personal use only “The NTIRTT1 clinical trial is the first time a broad-spectrum cannabinoid drug therapy (NTI164) has demonstrated significant patient improvements in Rett Syndrome using validated clinical
Slide 11: Caregiver Testimonials
Caregiver Testimonials Caregiver #1 “She seems much more in tune to what’s going on around her, e.g. patting the dog (has NEVER done this before)” For personal use only Caregiver #2 “She uses eye pointing, sometimes brings food / drink to an adult - this has not happened before” Caregiver #3 “Since
Slide 12: Primary Endpoint: CGI-I
Primary Endpoint: CGI-I 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 Rett syndrome population graded from 1 (very much improved) to 7 (very much worse). A decrease in CGI
Slide 13: Primary Endpoint: CGI-I
Primary Endpoint: CGI-I 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 Rett syndrome population graded from 1 (very much improved) to 7 (very much worse). A decrease in CGI
Slide 14: CGI-I: Specific Rett Anchor Analysis
CGI-I: Specific Rett Anchor Analysis • As a first in human trial of NTI164 in Rett patients, Neurotech examined nine (9) anchors/sub-domains to further understand what domain benefits NTI164 could target for registration-directed trials • Only 2-3 sub-domains are typically examined in Phase 3 trials
Slide 15: Secondary Endpoint: RSBQ
Secondary Endpoint: RSBQ The Rett Syndrome Behaviour Questionnaire (RSBQ) assesses the severity of neurobehavioral problems from the perspective of the caregiver and is one of the most widely used measures due to the specificity of its psychometric profile to the core features of Rett and is accepte
Slide 16: Secondary Endpoint: CGI-S
Secondary Endpoint: CGI-S Mean Severity of Illness (n=14) Severity of illness Scale (CGI-S) 5.0 4.6 4.5 4.3 For personal use only 1 2 3 4 5 6 7 4.2 4.0 Baseline 3.5 Week 4 Baseline Week 4 Week 12 Week 12 Clinical Interpretation CGI-Severity of illness1 (p = 0.009) • CGI-S reduced to a more moderate
Slide 17: NTI164, DAYBUE RSBQ – 12 weeks
NTI164, DAYBUE RSBQ – 12 weeks Rett Syndrome Behavioural Questionnaire (RSBQ) DAYBUE Placebo NTI164 For personal use only 0 Average Decrease from the Start of the Trial -2 -1.7 • Patients receiving NTI164 showed a 13.4 score decrease in average RSBQ total score from the start of the trial when compa
Slide 18: Other Secondary Endpoints NTI164
Other Secondary Endpoints NTI164 Statistical Significance Met Not Statistically Significant Not Measured For personal use only ICNDS RTT-SIS CSBS-DP-IT p=0.004 p=0.148 N/M RTT-DSC-VAS RTT-DSC-VAS ICNDS (QoL) Ambulation Hand Function p<0.001 p=0.374 N/M RTT-CBI RTT-DSC-VAS Communication Choices p=0.0
Slide 19: Conclusion
Conclusion NTI64 has demonstrated a statistically significant and clinically Met Primary Endpoint meaningful improvement in CGI-I (mean change, -0.4; p=0.009). When examining core domains, NTI164 showed 23% improvement at 12 weeks (p=0.001) and 93% of patients improved. For personal use only NTI64 h
Slide 20: Key Milestones – NTI164
Key Milestones – NTI164 1H CY2024 2H CY2024 • HREC/TGA Approval Cerebral Palsy Phase I/II Clinical Trial • Orphan Drug Designation USA – Rett Syndrome For personal use only • 24-week PANDAS/PANS Phase I/II Clinical Trial Data • Orphan Drug Designation USA – PANDAS/PANS • Rett Syndrome Phase I/II (14
Slide 21: Contact Details
For personal use only Contact Details Dr Tom Duthy Executive Director td@neurotechinternational.com +61 402 493 727 *This presentation has been authorised by the Board of Neurotech International Limited www.neurotechinternational.com Neurotech International Limited (ASX: NTI) 21
Slide 22: Appendices
For personal use only Appendices 22
Slide 23: Neurotech is a clinical-stage biopharmaceutical development company
Neurotech is a clinical-stage biopharmaceutical development company focused predominately on paediatric neurological disorders For personal use only NTI164 exclusive worldwide Novel oral Patents Pending – biopharmaceutical licence for neurological Use, Composition disorders cannabinoid platform (NTI
Slide 24: About Rett Syndrome
About Rett Syndrome About • 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 For personal use only • Currently there is no c
Slide 25: Rett Syndrome Market Dynamics
Rett Syndrome Market Dynamics Significant Market Single Approved Therapy Valuation/Pricing Benchmarks For personal use only 6,000-9,000 2,000-3,000 9,000-14,000 200 • 17-26k patients in USA, Europe, Japan, • First FDA approved therapy (March 2023) • Neuren (ASX:NEU) license deal with Acadia Australi
Slide 26: Therapeutic Agent: NTI164
Therapeutic Agent: NTI164 THC < 0.3% For personal use only Convenient 1x or 2x (split dose) High potency, Broad Spectrum Cannabinoid Formulation in Oil, C. Major constituent Cannabidiolic oral formulation in oil, ideal acid (CBDA) format for pediatric patients sativa L. (Plant Derived) 20mg/kg (CBDA
Slide 27: Our Target Markets
Our Target Markets Lack of effective therapies, significant unmet medical need Annual Drug Therapy Market opportunity US$2 billion* US$2 billion US$1.4 billion1 US$4.3 billion For personal use only Orphan Orphan Autism Spectrum Disorder (ASD) Rett Syndrome PANDAS/PANS Cerebral Palsy (CP) • Prevalenc
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