Neuren Pharmaceuticals Limited · ASX:NEU

Neuren Pharmaceuticals — AusBiotech Invest and Partnering 2018

11 slides from Neuren Pharmaceuticals's conference presentation, Nov 2, 2018

1. Slide 1: AusBiotech Invest and Partnering 2018

   For personal use only AusBiotech Invest and Partnering 2018 30 October 2018 1

2. Slide 2: Forward looking statements

   Forward looking statements For personal use only This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, Neuren can give no assurance that these expe

3. Slide 3: Neuren investment proposition

   Neuren investment proposition For personal use only  Two drugs (trofinetide and NNZ-2591) targeting broad impact on debilitating childhood disorders with urgent unmet need  Regulatory incentives – Orphan Drug, Fast Track, Priority Review  Strong support from advocacy groups and leading physicians

4. Slide 4: ACADIA partnership metrics

   ACADIA partnership metrics For personal use only  ACADIA funds all development costs for trofinetide in North America (including ~US$55 million for Rett syndrome)  Neuren retains all rights outside North America, with free use of all data generated by ACADIA in the US for registration outside Nort

5. Slide 5: Rett syndrome Phase 2 highlights

   Rett syndrome Phase 2 highlights For personal use only Clinical improvement measured by RSBQ Clinical improvement measured by CGI-I  RSBQ is a caregiver rating, reflecting the severity of the syndrome. Mean improvements for trofinetide and placebo were, respectively, 16% and 6%  CGI-I is a clinici

6. Slide 6: Rett syndrome Phase 3 program

   Rett syndrome Phase 3 program For personal use only  Continuing strong support and participation from leading Rett syndrome physicians and largest advocacy group (rettsyndrome.org)  Targeting underlying biology causing the disorder rather than treating one symptom  US Phase 3 program with single

7. Slide 7: Neuren's near-term priorities

   Neuren’s near-term priorities For personal use only  Actively support ACADIA in development of trofinetide for North America  Assist with preparations for Rett syndrome Phase 3 trial:  Manufacture of drug supplies  Finalise protocol, sites and logistics  Scientific Advisory Board  Optimise Fra

8. Slide 8: Appendix

   For personal use only Appendix 8

9. Slide 9: About Rett syndrome

   About Rett syndrome For personal use only  Seriously debilitating and life-threatening neurological disorder, with no approved medicines  Caused by non-inherited mutation on the X chromosome – estimated 1 in 10,000 to 15,000 live female births in all racial and ethnic groups  After apparently nor

10. Slide 10: About Fragile X syndrome

    About Fragile X syndrome For personal use only  Inherited X chromosome mutation – full mutation causes Fragile X syndrome  1 / 4,000 males and 1 / 6,000 females estimated to have full mutation  More severe in males, ~50% of females have some features of the syndrome  The most common inherited ca

11. Slide 11: Normal biology of IGF-1 in the brain

    Normal biology of IGF-1 in the brain For personal use only Produce essential growth factor Reversible binding IGF-1 IGF-1 regulates bioavailability IGF Binding Protein-3 Enzyme cleavage Regulates binding Truncated IGF-1 Enzyme cleavage GPE cGP Activates PI3K–Akt–mTOR and Ras–MAPK- ERK signalling pat

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