Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (January 2016)
21 slides from Neuren Pharmaceuticals's investor presentation, Jan 11, 2016
Slide 1: Investor Presentation
For personal use only Investor Presentation 11 January 2016 1
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
Slide 3: Company snapshot
Company snapshot For personal use only 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 pathway
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
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
Slide 6: Current trofinetide development programs
Current trofinetide development programs For personal use only Moderate to severe Rett syndrome Traumatic Brain Injury (TBI) Phase 2 trial completed Phase 2 trial results due April 2016 Mild TBI (Concussion) Fragile X syndrome Phase 2 trial enrolling Phase 2 trial completed 6
Slide 7: Trofinetide in Rett syndrome
Trofinetide in Rett syndrome For personal use only Non-inherited mutation in a gene on the X chromosome - 1 / 10,000 females Most physically disabling of the autism spectrum disorders - symptoms include: Intellectual disability, loss of speech and motor control Compulsive hand movements Di
Slide 8: 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
Slide 9: 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
Slide 10: 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 improv
Slide 11: Remaining development for Rett syndrome
Remaining development for Rett syndrome For personal use only Meaningful guidance in all areas of development program from 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) MBA
Slide 12: Trofinetide in Fragile X syndrome
Trofinetide in Fragile X syndrome For personal use only Inherited mutation on the X chromosome – 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 com
Slide 13: 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) 70 subjects aged 12 to 45 years received treatment, with 68 subjects completing the trial Both dose leve
Slide 14: Core efficacy measures that met target
Core efficacy measures that met target For personal use only Fragile X Syndrome Rating Scale Fragile X Domain Specific Concerns Aberrant Behavior Checklist (ABC) Total Score Analysis of mean clinical responses at end of treatment for each treatment group A negative value on the y-axis indicates
Slide 15: Subject-level efficacy analysis
Subject-level efficacy analysis For personal use only Mean of individual subject scorecards A positive value on the y-axis indicates clinical improvement Changes in all 5 core efficacy measures at end of treatment were combined in an efficacy score for each subject Mean efficacy scores were th
Slide 16: 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
Slide 17: Other development activities for trofinetide
Other development activities for trofinetide For personal use only Manufacturing process for pivotal trials, New Drug Application (NDA) and commercial supply Optimisation and scale-up of API synthesis Commercial finished drug product Investment will be completed in H2 2016 Chronic toxicity
Slide 18: Trofinetide commercial exclusivity
Trofinetide commercial exclusivity For personal use only Issued composition of matter patents for trofinetide owned by Neuren US – expires 2022, potential to extend to 2026 Europe – expires 2022, potential to extend to 2027 Exclusivity periods from orphan drug designation for trofinetide in
Slide 19: Second drug candidate: NNZ-2591
Second drug candidate: NNZ-2591 For personal use only Like trofinetide, NNZ-2591 has demonstrated potential utility across a broad range of neurological conditions Cyclic dipeptide with higher oral bioavailability, improved stability and potential for oral solid dosage form Shown efficacy in p
Slide 20: Shareholdings and financial position
Shareholdings and financial position For personal use only Cash reserves at 30 November 2015: A$17.6m Shares outstanding: 1.76 billion Options/EPR’s outstanding: 79 million Closing price 8 January 2016: 11.5 cents 52 week range: 7.4 cents – 18.5 cents 12% Retail 11% 58% Substantial Board/management
Slide 21: Neuren milestones for 2016
Neuren milestones for 2016 For personal use only Commence Rett syndrome pediatric Phase 2 trial Q1 2016 Top-line results for INTREPID Phase 2 trial April 2016 FDA meeting on Fragile X syndrome remaining development H1 2016 Complete Rett syndrome pediatric Phase 2 trial Q4 2016 Complete commercial ma
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