Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Therapeutic Area Partnerships Conference 2012
16 slides from Neuren Pharmaceuticals's conference presentation, Nov 30, 2012
Slide 1: Neuren presents at Therapeutic Area Partnerships Conference; Fragile X results
Neuren presents at Therapeutic Area Partnerships Conference; For personal use only announces positive results in Fragile X Syndrome model SYDNEY, Australia, 30 November 2012: Neuren Pharmaceutical Limited (ASX:NEU) presented at the Therapeutic Area Partnerships conference in Boston, Massachusetts to
Slide 2: About NNZ-2566, Rett Syndrome and Fragile X Syndrome
About NNZ-2566 NNZ-2566 is a synthetic analogue of a naturally occurring neuroprotective and neurotrophic molecule derived from IGF-1, a growth factor produced by brain cells as well as in other parts of the body. The intravenous form of NNZ-2566 is presently in a Phase II clinical trial in patients
Slide 3: Therapeutic Area Partnerships, November 2012
For personal use only Therapeutic Area Partnerships November 2012
Slide 4: 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 5: Company Overview
Company Overview Joe Horrigan, MD, VP, Clinical Development & For personal use only Larry Glass, Chief Executive Officer + 30 years of experience in management and Medical Affairs corporate development of life sciences 20+ years in academic medicine and drug companies; former CEO of CRO supporting d
Slide 6: NNZ-2566 and IGF-1 background
NNZ-2566 • IGF-1 is essential for brain development, widely expressed in For personal use only the CNS, upregulated following brain injury • Naturally occurring neurotrophic factor – one of the brain’s self- repair mechanisms • (1-3)IGF-1 or GPE is believed to be an active neuroprotective moiety in
Slide 7: NNZ-2566 mechanisms of action and animal models
NNZ-2566 • Multiple mechanisms of action For personal use only • Inhibits neuroinflammatory cytokine expression • Inhibits pathologic activation of microglia • Normalizes pro- and anti-apoptotic gene and protein expression • Enhances synaptic plasticity • Induces neuroblast proliferation • Inhibits
Slide 8: NNZ-2566 intravenous: traumatic brain injury
NNZ-2566 (intravenous formulation) • Well-tolerated with no SAEs in 4 Phase I clinical trials For personal use only • INTREPID-2566 trial underway • Randomized, double-blind, dose-escalation Phase II clinical trial • Fast Track designation granted by FDA • Exception from Informed Consent (EFIC) gran
Slide 9: NNZ-2566 oral formulation
NNZ-2566 (oral formulation) • Readily absorbed from the gut; crosses blood-brain barrier For personal use only • Targets: inflammation, synaptic function, and microglial activation 140 • Dose-dependent effects in animal models 120 100 80 with delayed administration 60 • No clinical toxicity in rats
Slide 10: NNZ-2566 (oral): concussion
NNZ-2566 (oral): concussion • IND open For personal use only • IGF-1 is upregulated in mild head injury • Inflammation, microglial activation are prominent • Phase II clinical trial preparations underway • 132 subjects; 2 doses plus placebo; 28 day follow-up • Subjects will have baseline neurocognit
Slide 11: NNZ-2566 (oral): Rett Syndrome
NNZ-2566 (oral): Rett Syndrome • Neurodevelopmental disorder caused For personal use only by mutation in MeCP2 gene • Synaptic dysfunction, microglial activation, neuroinflammation appear to play prominent roles in Rett symptomatology • Positive effects of IGF-1, (1-3)IGF-1 and NNZ-2566 observed in
Slide 12: NNZ-2566 (oral): Rett Syndrome Phase II trial
NNZ-2566 (oral): Rett Syndrome • Phase II trial of NNZ-2566 in adolescent and adult patients For personal use only scheduled to open in December 2012 • IND submitted in November following receipt of final Phase I study report • 48 subjects 16-40 years; two dose cohorts; randomized 2:1; stratified by
Slide 13: NNZ-2566: Fragile X Syndrome study
NNZ-2566: Fragile X Syndrome study • The effect of NNZ-2566 was investigated in Wild Type mice and the For personal use only fmr1 KO mouse model of Fragile X Syndrome • 100 mg/kg i.p. NNZ-2566 or vehicle administered for 28 days • 30 mg/kg dose level also tested – data being analysed • Anxiety, loco
Slide 14: Fragile X initial results
NNZ-2566: Fragile X initial results For personal use only Open Field - Rearing 35 Wild Type Vehicle 30 Wild Type NNZ-2566 • Anxiety and locomotor activity 25 fmr1 KO Vehicle fmr1 KO NNZ-2566 Rears / 3 min • fmr1 KO mice show hyperactivity in the open 20 field, Successive Alleys and Elevated Plus 15
Slide 15: Fragile X initial results: social behavior and morphology
NNZ-2566: Fragile X initial results Social Behavior – Bouts of Sniffing For personal use only p < 0.001 16 NS • Social behaviors and complex species 14 typical behaviors 12 10 NS • fmr1 KO mice show abnormal social interactions, 8 which are reversed by NNZ-2566 6 • fmr1 KO mice show abnormalities in
Slide 16: Contact information
Contact information For personal use only Larry Glass, CEO [email protected] US office 3 Bethesda Metro Center, Suite 700 Bethesda, MD 20814 USA Telephone: +1 301 941 1830 Facsimile: +1 435 518 7255 New Zealand office Level 1, 59 Wellington Street Freemans Bay 1011 Auckland, New Zealand Teleph
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