STRATEGY DISCUSSION



Investor Presentation‌‌‌‌‌‌‌‌‌‌‌‌‌‌

11 January 2016


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Forward looking statements


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 expectations will prove to be correct. Actual results could differ materially from those anticipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, risks associated with patent protection, future capital needs or other general risks or factors.



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Company snapshot

  • Stock code ASX: NEU - market cap approximately A$200 million

  • Developing treatments for chronic and acute neurological conditions

    • Large markets with no therapies currently available

    • Orphan drug designation and potential for abbreviated regulatory pathways

  • Lead drug candidate trofinetide

    • In development for Rett syndrome, Fragile X syndrome, Traumatic Brain Injury and Concussion

    • Excellent safety and tolerability profile to date

    • Pattern of clinical improvement observed in both Rett syndrome and Fragile X syndrome Phase 2 trials

    • Clinical effects consistent with its known biological actions on brain function

  • Key strategic relationships

    • US Army Medical Research & Materiel Command

    • International Rett Syndrome Foundation (Rettsyndrome.org)

    • FRAXA and National Fragile X Foundation


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  • Demonstrate the therapeutic benefit of trofinetide in human subjects in both acute and chronic conditions


  • Potential to establish a "gateway" to autism and other neurodevelopmental disorders


  • Criteria for selecting therapeutic targets

    • Significant unmet need and commercial opportunity with no approved drugs

    • Regulatory advantages - candidates for Fast Track, Orphan Drug, Breakthrough Therapy

    • Strong support from advocacy groups and other stakeholders


  • Realising value

    • Generate clinical data with trofinetide in Phase 2 clinical trials

    • Define optimum pathway towards New Drug Applications

    • Optimise manufacturing process for commercial product supply

    • Engage with potential partners


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Neuren Pharmaceuticals Limited issued this content on 2016-01-11 and is solely responsible for the information contained herein. Distributed by Public, unedited and unaltered, on 2016-01-10 22:42:19 UTC

Original Document: http://www.neurenpharma.com/IRM/PDF/1562/NeurenInvestorPresentation11January2016