Neuren Pharmaceuticals Limited · ASX:NEU

Neuren Pharmaceuticals — Investor Presentation (September 2014)

14 slides from Neuren Pharmaceuticals's investor presentation, Sep 4, 2014

  1. Slide 1: Investor Presentation

    For personal use only INVESTOR PRESENTATION 4 September 2014

  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: Company Snapshot

    Company Snapshot For personal use only  Stock code ASX: NEU – market cap approximately A$150 million  Developing treatments for chronic and acute neurological conditions  Large markets with no therapies currently available  Potential for abbreviated regulatory pathways and orphan drug designatio

  4. Slide 4: Scientific Foundation

    Scientific Foundation For personal use only  IGF-1 is a naturally occurring growth factor in the brain  Glypromate (GPE) separates from IGF-1 in the brain  IGF-1 and GPE maintain and restore equilibrium in the brain  NNZ-2566 is a synthetic analogue of GPE with a longer half-life, better stabili

  5. Slide 5: Intellectual Property

    Intellectual Property For personal use only  Broad patent estate with no royalties payable  NNZ-2566 and other GPE analogues  8 issued US patents covering composition, oral formulation and methods of use  Remaining patent life between 8 and 16 years  Further patent applications pending  Additi

  6. Slide 6: Strategy

    Strategy For personal use only  Demonstrate the therapeutic benefit of NNZ-2566 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 comm

  7. Slide 7: Orphan drug designation

    “Orphan drug” designation For personal use only  FDA may grant “orphan drug” designation to a drug to treat a rare condition – provides 7 years of marketing exclusivity following approval, as well as other incentives  Neuren received orphan drug for Fragile X Syndrome and will apply for Rett Syndr

  8. Slide 8: NNZ-2566 Clinical Strategy

    NNZ -2566 Clinical Strategy For personal use only Moderate to Rett severe Traumatic Syndrome Brain Injury (TBI) Mild TBI Fragile X (Concussion) Syndrome

  9. Slide 9: NNZ-2566 in Rett Syndrome

    NNZ-2566 in Rett Syndrome For personal use only  Mutation in a gene on the X chromosome - 1 / 10,000 females (20,000 USA)  Most physically disabling of the autism spectrum disorders - symptoms include:  Intellectual disability, loss of speech and motor control  Compulsive hand movements  Disord

  10. Slide 10: NNZ-2566 in Fragile X Syndrome

    NNZ-2566 in Fragile X Syndrome For personal use only  Mutation on the X chromosome affecting both males and females - 1 / 4,000 males and 1 / 6,000 females (58,000 USA)  The most common inherited cause of intellectual disabilities and the most common known cause of autism - symptoms include:  Int

  11. Slide 11: NNZ-2566 in Traumatic Brain Injury (TBI)

    NNZ-2566 in Traumatic Brain Injury (TBI) For personal use only  > 1.5 million head injuries annually in the US alone; >75% are mild (Concussion)  Leading cause of death and disability, especially in young and elderly  Serious health and economic effects of Concussion in sporting codes  Partnersh

  12. Slide 12: Shareholdings and Financial Position

    Shareholdings and Financial Position For personal use only • Fully funded through to completion of Phase 2 trials in 4 different indications • A$22m cash reserves at 30 June 2014 Shares outstanding: 1.59 billion Options outstanding: 147 million (1.3 cents to 3.8 cents per share) Closing price 28 Aug

  13. Slide 13: Investment Summary

    Investment Summary For personal use only  Patented drug analogues of naturally occurring brain growth factors  Potentially applicable to both acute and chronic neurological conditions – large markets with no therapies currently available  Compelling pre-clinical efficacy data in TBI, Fragile X an

  14. Slide 14: Expected Milestones

    Expected Milestones For personal use only Top-line results for Rett Phase 2 Q4-2014 Complete enrollment in Fragile X Phase 2 H2-2014 Complete enrollment in INTREPID H1-2015 Top-line results for Fragile X Phase 2 Q2-2015 Top-line results for INTREPID H2-2015 Top-line results for Concussion Phase 2 H2

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