Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (November 2013)
22 slides from Neuren Pharmaceuticals's investor presentation, Nov 19, 2013
Slide 1: Investor Presentation
For personal use only INVESTOR PRESENTATION 19 November 2013 Dr Richard Treagus – Executive Chairman Jon Pilcher – Chief Financial Officer
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$175 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: Leadership
Leadership For personal use only BOARD OF DIRECTORS MANAGEMENT Richard Treagus (Executive Chairman) Larry Glass (CSO) Former CEO Acrux Limited 9 years with Neuren Trevor Scott Jon Pilcher (CFO) Former CFO Acrux Limited Head of Audit Committee James Shaw (COO) Bruce Hancox Forme
Slide 5: Scientific Foundation
Scientific Foundation For personal use only IGF-1 is a naturally occurring growth factor in the brain • Produced following brain injury and stress • One of the brain’s self-repair mechanisms Glypromate (GPE) is considered the active part of the molecule NNZ-2566 is a synthetic analogue of Glyp
Slide 6: NNZ-2566
NNZ-2566 For personal use only Pre-clinical efficacy has been demonstrated in animal models of TBI, stroke, cognition, Fragile X and Rett Syndromes Administered intravenously or orally 40-50% orally bioavailable, with or without food Crosses the blood brain barrier Well tolerated at therap
Slide 7: NNZ-2591
NNZ-2591 For personal use only Synthetic analog - cyclic dipeptide 50 100% orally bioavailable 30 mg/kg IV NNZ-2591 (µg/ml) 40 30 mg/kg Oral 30 Testing in Fragile X model completed; results comparable to NNZ-2566 at 20 1/3rd the dose 10 US Army working on mechanism of 0 action under Cooperat
Slide 8: Intellectual Property
Intellectual Property For personal use only Broad patent estate with no royalties payable NNZ-2566 and other GPE analogues 7 issued patents covering composition, oral formulation and methods of use 7 pending applications Remaining patent life between 9 and 15 years Additional market excl
Slide 9: 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 10: Key Clinical Programmes
For personal use only Key Clinical Programmes
Slide 11: NNZ-2566 Clinical Strategy
NNZ -2566 Clinical Strategy For personal use only Traumatic Rett Brain Injury Syndrome (TBI) Fragile X Concussion Syndrome
Slide 12: Rett Syndrome
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 Many girls live into adulthood, requiring total 24-hr-day care Profound disability and financial burden for >50,000 patients
Slide 13: Rett Syndrome Phase II (RTT-001)
Rett Syndrome Phase II (RTT-001) For personal use only Phase 2 randomized (2:1), placebo-controlled, dose escalation, safety and efficacy study Up to 60 (48 complete) female subjects ages 16-40 years NNZ-2566 oral solution or placebo administered for 28 days following 3 days of dose titration
Slide 14: Fragile X Syndrome
Fragile X Syndrome For personal use only Mutation on the X chromosome affecting both males and females The most common inherited cause of intellectual disabilities and the most common known cause of autism 1 / 4,000 males and 1 / 6,000 females (58,000 USA) Symptoms include: Intellectual di
Slide 15: Fragile X Syndrome pre-clinical efficacy
Fragile X Syndrome pre-clinical efficacy For personal use only NNZ-2566 and NNZ-2591 were separately tested in a model of Fragile X Syndrome by FRAXA Drug Validation Initiative “Wild type” (normal) mice and “fmr1 knockout” mice (bred with the Fragile X chromosome defect) were dosed with Neuren d
Slide 16: Fragile X Syndrome Phase II (FXS-001)
Fragile X Syndrome Phase II (FXS-001) For personal use only Phase 2 randomized (1:1:1), placebo-controlled, fixed dose, safety and efficacy study Up to 72 (60 complete) male subjects ages 16-40 years NNZ-2566 oral solution or placebo administered for 28 days at 35 or 70 mg/kg b.i.d Endpoints
Slide 17: Traumatic Brain Injury (TBI) and Concussion
Traumatic Brain Injury (TBI) and Concussion For personal use only NNZ-2566 improves functional recovery, preserves cognitive function, inhibits post-injury seizures > 1.5 million head injuries annually in the US alone; >75% are mild or concussion Leading cause of death and disability, especial
Slide 18: Traumatic Brain Injury (INTREPID)
Traumatic Brain Injury (INTREPID) For personal use only Phase 2 randomized (2:1), placebo-controlled, fixed dose escalation, safety and efficacy study 260 subjects, ages 16-75 with moderate to severe TBI (Glasgow Coma Scale 4-12) NNZ-2566 IV solution or placebo administered within 8 hours of i
Slide 19: Concussion
Concussion For personal use only Phase 2 randomized (1:1:1), placebo-controlled, fixed dose safety and efficacy study 132 subjects, 18-55 with mild TBI (Glasgow Coma Scale 13-15) and a pre-injury computerized neurocognitive assessment NNZ-2566 oral solution or placebo administered within 24 ho
Slide 20: 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$23m cash reserves at 15 November 2013 • A$5.4m expected from options through 2016 Shares outstanding: 1.5 billion Options outstanding: 221 million (1.3 cents
Slide 21: 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
Slide 22: Expected News Flow
Expected News Flow For personal use only Initiate Fragile X Phase 2 December 2013 Complete enrollment in Rett Phase 2 1H-2014 Initiate Concussion Phase 2 1H-2014 Top-line results for Rett Phase 2 2H-2014 Complete enrollment in Fragile X Phase 2 2H-2014 Complete enrollment in INTREPID 2H-2014 Top-lin
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