Neuren Pharmaceuticals Limited · ASX:NEU

Neuren Pharmaceuticals — Investor Presentation (July 2015)

16 slides from Neuren Pharmaceuticals's investor presentation, Jul 31, 2015

  1. Slide 1: Investor Presentation

    For personal use only Investor Presentation 31 July 2015 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: Company snapshot

    Company snapshot For personal use only  Stock code ASX: NEU – market cap approximately A$160 million  Developing treatments for chronic and acute neurological conditions  Large markets with no therapies currently available  Orphan drug designation and potential for abbreviated regulatory pathway

  4. Slide 4: Strategy

    Strategy For personal use only  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 c

  5. Slide 5: 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  Trofinetide is a synthetic analogue of GPE with a longer half- life, better sta

  6. Slide 6: Trofinetide current clinical strategy

    Trofinetide current clinical strategy For personal use only Moderate to Rett severe Traumatic syndrome Brain Injury (TBI) Mild TBI Fragile X (Concussion) syndrome 6

  7. Slide 7: Importance of "orphan" drug designation

    Importance of “orphan” drug designation For personal use only  FDA and EMA may grant “orphan” designation to a drug to treat a rare condition – provides marketing exclusivity following approval for 7 years in US and 10 years in EU  Orphan granted to Neuren in US and EU for both Fragile X syndrome

  8. Slide 8: Trofinetide in Rett syndrome

    Trofinetide 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  Dis

  9. Slide 9: Phase 2 trial highlights

    Phase 2 trial highlights For personal use only  Double-blind, placebo-controlled Phase 2 trial of 28 days of treatment with two dose levels (35 mg/kg and 70 mg/kg of body weight twice daily)  56 subjects aged 16 to 45 years randomized, with 53 subjects completing the trial  Both dose levels were

  10. Slide 10: Core efficacy measures that met target

    Core efficacy measures that met target For personal use only Motor Behavior Assessment Change Index Clinical Global Impression of Improvement Absolute value Caregiver Top 3 Concerns  Analysis of group mean values  Solid line is 70mg/kg, dotted line is placebo  The two different shaded areas indic

  11. Slide 11: Subject-level efficacy analysis

    Subject-level efficacy analysis For personal use only  Solid line is 70mg/kg, dotted line is Mean subject-level efficacy score placebo  The two different shaded areas indicate the treatment period and the period post-cessation of treatment  A positive value on the y-axis indicates clinical benefi

  12. Slide 12: Remaining development for Rett syndrome

    Remaining development for Rett syndrome For personal use only  Meaningful guidance in all areas of development program from recent FDA meeting  Neuren and FDA committed to reach agreement quickly on primary efficacy endpoint for pivotal trials, to be derived from the Motor Behavior Assessment (MBA

  13. Slide 13: Trofinetide in Fragile X syndrome

    Trofinetide 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: 

  14. Slide 14: Trofinetide in Traumatic Brain Injury (TBI)

    Trofinetide 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  Partne

  15. Slide 15: 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$18m cash reserves at 30 June 2015 Shares outstanding: 1.7 billion Options outstanding: 73 million (1.3 cents to 3.8 cents per share) Closing price 30 July

  16. Slide 16: Neuren 2015 expected milestones

    Neuren 2015 expected milestones For personal use only FDA meeting on remaining Achieved development for Rett Syndrome Orphan Drug designation in Europe for Achieved Rett syndrome and Fragile X syndrome Top-line results for Fragile X Phase 2 December 2015 Top-line results for INTREPID Phase 2 H2 2015

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