Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (October 2013)
25 slides from Neuren Pharmaceuticals's investor presentation, Oct 9, 2013
Slide 1: Investor Presentation
For personal use only INVESTOR PRESENTATION 9 October 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 ORIGINS • Formed in 2001 • IP rights acquired from the University of Auckland • Listed on the ASX in February 2005 • Partnership established with the US Army in 2009 KEY STRATEGIC RELATIONSHIPS • US Army Medical Research & Materiel Command • International R
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 high d
Slide 7: NNZ-2591
NNZ‐2591 For personal use only Synthetic analog ‐ cyclic dipeptide 50 30 mg/kg IV NNZ-2591 (µg/ml) 40 100% orally bioavailable 30 mg/kg Oral 30 Testing in Fragile X model completed; 20 results comparable to NNZ‐2566 at 10 1/3rd the dose 0 2 15 30 60 90 120 US Army working on mechanism of Tim
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: Pre-Clinical Studies
For personal use only Pre‐Clinical Studies
Slide 10: Dendritic Spines - NNZ-2566 in Fragile X (fmr1) model
Dendritic Spines ‐ NNZ‐2566 in Fragile X (fmr1) model For personal use only Dendritic spine morphology
Slide 11: Behaviour - NNZ-2566 in Fragile X (fmr1) model
Behaviour ‐ NNZ‐2566 in Fragile X (fmr1) model For personal use only NS p < 0.01 NS P < 0.001 4.5 4.0 Entries Open Arm 3.5 3.0 2.5 2.0 1.5 1.0 0.5 0.0 N = 10 per group Vehicle NNZ-2566 Vehicle NNZ-2566 Wild Type frm1 KO fmr1 KO mice show a significant difference to Wild Type controls NNZ‐2566 tr
Slide 12: Enlarged testes - NNZ-2566 in Fragile X (fmr1) model
Enlarged testes ‐ NNZ‐2566 in Fragile X (fmr1) model For personal use only p < 0.001 p < 0.001 25.0 NS NS 20.0 Tests Weight (mg) 15.0 10.0 5.0 0.0 Vehicle NNZ-2566 Vehicle NNZ-2566 Wild Type fmr1 KO fmr1 KO mice show an increase in testis weight, as do human Fragile X Syndrome patients After NNZ
Slide 13: Key Clinical Programmes
For personal use only Key Clinical Programmes
Slide 14: NNZ-2566 Clinical Strategy
NNZ ‐2566 Clinical Strategy For personal use only Traumatic Rett Brain Injury Syndrome (TBI) Concussion Fragile X (mild TBI) Syndrome
Slide 15: TBI and Concussion
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, especially in young people and th
Slide 16: Traumatic Brain Injury (INTREPID)
Traumatic Brain Injury (INTREPID) For personal use only Phase II 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
Slide 17: Concussion (mTBI)
Concussion (mTBI) For personal use only Phase II 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 with
Slide 18: 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 19: Rett Syndrome Phase II (RTT-001)
Rett Syndrome Phase II (RTT‐001) For personal use only Phase II randomized (2:1), placebo‐controlled, dose escalation, safety and efficacy study Up to 60 (48 complete) female subjects ages 16‐40 years Subjects stratified 1:1 based on mutation (correlates with severity) NNZ‐2566 oral solution
Slide 20: 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 21: Fragile X Syndrome Phase II (FXS-001)
Fragile X Syndrome Phase II (FXS‐001) For personal use only Phase II 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 Endpoint
Slide 22: Strategy
Strategy For personal use only Demonstrate the therapeutic benefit of NNZ‐2566 in human subjects Expand treatments to include both acute and chronic conditions Potential to establish a “gateway” to autism and other neurodevelopmental disorders Criteria for selecting therapeutic targets Sig
Slide 23: Shareholdings and Financial Position
Shareholdings and Financial Position For personal use only Shares outstanding: 1.25 billion Options outstanding: 255 million Closing price 4 Oct 2013 10.5 cents 52 week range: 2.2 cents ‐ 12.5 cents Market capitalization 4 Oct 2013 $131 million Cash at 30 June 2013 NZ$4.1 million Top 20 shareholders
Slide 24: 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 – with large unserved markets. Compelling pre‐clinical efficacy data in TBI, Fragile X and Rett Syndrome models
Slide 25: Expected News Flow
Expected News Flow For personal use only Initiate Fragile X Phase 2 2H‐2013 Complete enrollment in Rett 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‐line results for Fragile X Phase 2 1H‐2015 To
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