Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (January 2015)
20 slides from Neuren Pharmaceuticals's investor presentation, Jan 8, 2015
Slide 1: Investor Presentation
For personal use only Investor Presentation 8 January 2015 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$250 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
Slide 4: 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
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 NNZ-2566 is a synthetic analogue of GPE with a longer half-life, better stabili
Slide 6: 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 6
Slide 7: 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
Slide 8: Phase 2 trial outline
Phase 2 trial outline For personal use only Double-blind, placebo-controlled dose escalation Phase 2 trial at 3 US hospitals of two dose levels of NNZ-2566: 35 mg per kg of body weight twice daily 70 mg per kg of body weight twice daily 56 subjects aged 16 to 45 years randomized, with 53 sub
Slide 9: Results highlights
Results highlights For personal use only Achieved primary endpoint - both dose levels of NNZ-2566 were well tolerated after 28 days of treatment and no safety concerns were identified Higher dose (70mg/kg twice daily) exceeded the pre-specified criteria for improvement in core efficacy measures
Slide 10: Results - safety
Results - safety For personal use only Adverse events recording, laboratory measurements and physical observations Dose-dependent and time-dependent patterns were not observed in the adverse events reported during the trial No consistent dose-dependent trends in observations or laboratory meas
Slide 11: Results – core efficacy measures
Results – core efficacy measures For personal use only 6 core efficacy measures in 4 efficacy domains, including clinician assessments and caregiver assessments specific to Rett syndrome signs and symptoms The pre-specified criteria for evidence of clinical benefit in the analysis of the mean re
Slide 12: Results – core efficacy measures (continued)
Results – core efficacy measures (continued) For personal use only Lower dose (35mg/kg) showed improvement in core efficacy measures, but magnitude less than the pre-specified target Both doses showed trends of increasing effect with duration of treatment Clinical benefit encompassed core symp
Slide 13: Results – core efficacy measures that met target
Results – 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 Solid line is 70mg/kg, dotted line is placebo The two different shaded areas indicate the treatment peri
Slide 14: Results – core efficacy measures that met target
Results – core efficacy measures that met target For personal use only Mean subject-level efficacy score Solid line is 70mg/kg, dotted line is placebo The two different shaded areas indicate the treatment period and the period post-cessation of treatment A positive value on the y-axis indicate
Slide 15: “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 has applied for Rett synd
Slide 16: “Breakthrough Therapy” designation
“Breakthrough Therapy” designation For personal use only Intended to streamline drug development and review of innovative new medicines that address unmet medical needs for serious diseases or conditions The criteria for breakthrough therapy require preliminary clinical evidence indicating that
Slide 17: 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
Slide 18: 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
Slide 19: 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$21m cash reserves at 31 December 2014 Shares outstanding: 1.63 billion Options outstanding: 116 million (1.3 cents to 3.8 cents per share) Closing price 8
Slide 20: Neuren expected milestones
Neuren expected milestones For personal use only Outcome of Rett syndrome Orphan Drug and Breakthrough Therapy applications H1-2015 Rett Syndrome FDA meeting H1-2015 Top-line results for Fragile X Phase 2 H2-2015 Top-line results for INTREPID H2-2015 Top-line results for Concussion Phase 2 H2-2015 2
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