Neurizon Therapeutics Limited · ASX:NUZ

Neurizon Therapeutics — Technical Presentation (February 2024)

16 slides from Neurizon Therapeutics's technical presentation, Feb 27, 2024

  1. Slide 1: Phase 1 MEND Study Top-Line Results

    ASX: PAA Phase 1 MEND Study Top-Line Results February 2024 Dr Michael Thurn

  2. Slide 2: Disclaimer

    Disclaimer This disclaimer applies to this presentation and the information contained in it (This presentation has been prepared by PharmAust Limited (ASX: PAA) (the “Company”). It does not purport to contain all the information that a prospective investor may require in connection with any potentia

  3. Slide 3: Phase 1 Executive Summary

    Phase 1 Executive Summary Positive Phase 1 study results showing monepantel has an excellent safety profile and the ability to slow the progression of MND/ALS Highlights Primary Objectives Excellent safety and tolerability profile Slows the rate of progression by 58% (Cohort 2 Preliminary efficacy d

  4. Slide 4: Phase 1 Study Design

    Phase 1 Study Design PATIENT POPULATION: INTERVENTION: STUDY OBJECTIVES: 11 Men, 1 Woman 12 patients randomised • Adults with Familial or Sporadic ALS/MND Open label, 24 hour escalating single dose PK study, • Tolerability and safety of monepantel with a goal of • 1st symptoms occurred < 3 years pri

  5. Slide 5: Safety and Tolerability Summary

    Phase 1 Safety and Tolerability Summary No deaths, no Serious Adverse Events related to treatment and a very low incidence of Adverse Events Incidence of Adverse Event n Dose 1 (2 mg/kg) Dose 2 (4 mg/kg) Dose 3 (6 mg/kg) Dose 4 (10 mg/kg) Total Adverse Events 29 6 12 9 56 Related to Treatment 2 1 -

  6. Slide 6: Preliminary Efficacy: ALSFRS-R Score

    Phase 1 Preliminary Efficacy Amyotrophic Lateral Sclerosis Function Rating Scale – Revised (ALSFRS-R) ALSFRS-R is a predictor of survival time in ALS patients.1 The speed at which ALS progresses, measured by the rate of decline in a patient’s ALSFRS-R score over time, can be used to confidently pred

  7. Slide 7: Preliminary Efficacy: ALSFRS-R Score

    Phase 1 Preliminary Efficacy Amyotrophic Lateral Sclerosis Function Rating Scale – Revised (ALSFRS-R) There were no significant differences in ALSFRS-R scores between predose and end of treatment for all 12 patients Cohort 1, and Cohort 2 suggesting that treatment with monepantel over 8 – 12 months

  8. Slide 8: Preliminary Efficacy: ALSFRS-R Score

    Phase 1 Preliminary Efficacy Amyotrophic Lateral Sclerosis Function Rating Scale – Revised (ALSFRS-R) Treatment with monepantel for up to 12 months slowed the progression of MND/ALS in all 12 patients by 39% when compared to matched controls from the PRO-ACT database1 ALSFRS-R Domains Assessed MPL –

  9. Slide 9: Preliminary Efficacy: ALSFRS-R Score

    Phase 1 Preliminary Efficacy Amyotrophic Lateral Sclerosis Function Rating Scale – Revised (ALSFRS-R) Dose response observed between Cohort 1 and 2. Rate of disease progression in Cohort 2 was slowed by an impressive 58% compared to the PRO-ACT database matched controls FDA Timeframe for accelerated

  10. Slide 10: Exploratory Endpoints

    Phase 1 Exploratory Endpoints No significant difference in ALS Quality of Life Questionnaire, Edinburgh Cognitive and Behavioural ALS Screen, and Slow Vital Capacity between predose and end of treatment ALSSQOL-R Edinburgh Cognitive and Behavioural Slow Vital Capacity Quality of Life Questionnaire A

  11. Slide 11: Exploratory Biomarkers

    Phase 1 Exploratory Biomarkers No significant difference in biomarkers serum neurofilament /light chain (NfL) and urinary p75ECD levels between pre-dose and end of treatment Neurofilament Light Chain Neurofilament Light Chain Urinary p75ECD/Creatinine Serum Cerebrospinal Fluid 100 14 90 10000 80 12

  12. Slide 12: Pharmacokinetics and Target Engagement (mTOR Pathway)

    Phase 1 Pharmacokinetics and Target Engagement (mTOR Pathway) Cohort 1 (2 & 6 mg/kg) Cohort 2 (4 & 10 mg/kg) Pharmacokinetics Concentrations of MPL Sulphone, the active metabolite of MPL, increased somewhat proportionally with higher doses of MPL. MPL and MPLS was found in the cerebrospinal fluid. T

  13. Slide 13: Comparison to Leading FDA-Approved Product

    Phase 1 Comparison to Leading FDA-Approved Product Monepantel displays a superior safety, tolerability and preliminary efficacy profile to the leading FDA approved drug Relyvrio® Estimated Additional Life Expectancy for MPL Estimated rate Slope Change Additional Life Slowing in of ALSFRS-R from PRO-

  14. Slide 14: Conclusion

    Phase 1 Conclusion Positive Phase 1 study results showing monepantel has an excellent safety profile and the ability to slow the progression of MND/ALS Primary Objectives Excellent safety and tolerability profile Elows the rate of progression by 58% (Cohort 2 Preliminary efficacy data High Dose) Mon

  15. Slide 15: Thank you

    Thank you 15 Phase 1 MEND Study Top-Line Results

  16. Slide 16: For more information, please contact

    ASX: PAA For more information, please contact Matthew Baker, Corporate Advisor, Blue Ocean Equities +61 (0) 418 830 064, matthewbaker@boeq.com.au Matthew Wright, Investor Relations, NWR Communications +61 (0) 451 896 420, matt@nwrcommunications.com.au Registered Address: Phone: +61 (8) 9202 6814 Sui

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