Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (May 2013)
18 slides from Neuren Pharmaceuticals's investor presentation, May 21, 2013
Slide 1: Investor Presentation May 2013
For personal use only Investor Presentation May 2013
Slide 2: Forward Looking Statement
Forward Looking Statement 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 expec
Slide 3: Agenda
Agenda For personal use only • Company snapshot • Investment thesis • Scientific foundation • Intellectual property • Strategy • Clinical trials • TBI trial • Rett Syndrome trial • Fragile X Syndrome trial • Concussion trial • Shareholding and financial position • Expected news flow 3
Slide 4: Company Snapshot
Company Snapshot For personal use only • Origins • Created in 2004 by merger of two companies formed around University of Auckland IP • Listed on the ASX in February 2005 • Leadership • Richard Treagus, Executive Chairman • Larry Glass, CEO & Managing Director • Joe Horrigan, VP for Clinical Develop
Slide 5: Investment Thesis
Investment Thesis • Lead clinical stage molecule has unique biology directly applicable to many For personal use only key CNS conditions • ~US$26m in non-dilutive funding from the US Army supports two clinical trials, funded development of the oral form of NNZ-2566 • Strong patent portfolio: composi
Slide 6: Scientific Foundation
Scientific Foundation • IGF-1 is one of the primary growth For personal use only NNZ-2566 factors in the CNS • Glypromate is a key part of the brain’s response to injury and stress • NNZ-2566 is a synthetic analogue of Glypromate • Addresses pathology underlying acute and chronic CNS conditions and
Slide 7: Intellectual Property
Intellectual Property • Robust patent estate For personal use only • No royalty burden on lead molecules • NNZ-2566 and other GPE analogues • 7 issued patents covering composition, oral formulation and methods of use • 7 pending applications covering composition, oral formulations and methods of use
Slide 8: Strategy
Strategy For personal use only • Build on the unique biology of NNZ-2566 to pursue multiple indications and commercial opportunities • Expand therapeutic focus to include both acute and chronic conditions • Criteria for selecting therapeutic targets • Significant unmet need with no approved drugs •
Slide 9: Key Programmes
For personal use only Key Programmes 9
Slide 10: TBI and concussion: rationale
TBI and concussion: rationale For personal use only • NNZ-2566 inhibits inflammatory cytokines, pathological microglial activation, apoptosis and necrosis • NNZ-2566 improves functional recovery, preserves cognitive function, inhibits post-injury seizures • > 1.5 million head injuries annually in th
Slide 11: TBI (INTREPID-2566)
TBI (INTREPID-2566) 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 injury as a 20
Slide 12: Concussion (TBI-003)
Concussion (TBI-003) 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 w
Slide 13: Neurodevelopmental disorders: rationale
Neurodevelopmental disorders: rationale • Rett Syndrome For personal use only • NNZ-2566 increases dendritic length and branching • NNZ-2566 improves signal transmission between neurons and improves survival • Cause of profound disability and huge financial burdens for >50,000 patients and families
Slide 14: 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 with a diagnosis of Rett Syndrome • Subjects stratified 1:1 based on mutation (correlates with
Slide 15: Fragile X Syndrome (FXS-001)
Fragile X Syndrome (FXS-001) For personal use only • Phase II randomized (1:1:1), placebo-controlled, fixed dose, cross- over, safety and efficacy study • Up to 60 (48 complete) male subjects ages 16-40 years with a diagnosis of Fragile X Syndrome • NNZ-2566 oral solution or placebo administered for
Slide 16: NNZ-2591
NNZ-2591 For personal use only • Lead preclinical molecule in cyclic dipeptide portfolio • Synthetic analog of naturally occurring neurotrophic factor • 100% orally bioavailable candidate for chronic, oral therapy • Manufacturing development for preclinical R&D nearing completion • US Army working o
Slide 17: Shareholding and Financial Position
Shareholding and Financial Position • Shares outstanding: 1.2b For personal use only • Options outstanding: 274m • Closing price (20 May 2013): $0.058 • Market capitalization: $69m • Current cash (30 April 2013): NZ$4.24m • Top 20 shareholders: 57% (approximate) • Institutions: 9%; directors: 6%; su
Slide 18: 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-2566 2H-2014 • Top-line results for Fragile X P
Neuren Pharmaceuticals — Investor Presentation (May 2013) on 6ix