Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Therapeutic Area Partnerships Conference 2012
16 slides from Neuren Pharmaceuticals's conference presentation, Feb 11, 2016
Slide 1: Neuren presents at Therapeutic Area Partnerships Conference; announces positive Fragile X results
Neuren presents at Therapeutic Area Partnerships Conference; 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 today. The conference wa
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
Therapeutic Area Partnerships November 2012
Slide 4: Forward Looking Statement
Forward Looking Statement 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 expectations will prove to
Slide 5: Company Overview
Company Overview Larry Glass, Chief Executive Officer Joe Horrigan, MD, VP, Clinical Development & + 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 development; former Ass
Slide 6: NNZ-2566
NNZ-2566 • IGF-1 is essential for brain development, widely expressed in 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 response to injury • (
Slide 7: NNZ-2566
NNZ-2566 • Multiple mechanisms of action • 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 post-injury seizures N
Slide 8: NNZ-2566 (intravenous formulation)
NNZ-2566 (intravenous formulation) • Well-tolerated with no SAEs in 4 Phase I clinical trials • INTREPID-2566 trial underway • Randomized, double-blind, dose-escalation Phase II clinical trial • Fast Track designation granted by FDA • Exception from Informed Consent (EFIC) granted by FDA • >1/3 of s
Slide 9: NNZ-2566 (oral formulation)
NNZ-2566 (oral formulation) • Readily absorbed from the gut; crosses blood-brain barrier • 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 at highest dose 40 20
Slide 10: NNZ-2566 (oral): concussion
NNZ-2566 (oral): concussion • IND open • 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 neurocognitive assessment with Im
Slide 11: NNZ-2566 (oral): Rett Syndrome
NNZ-2566 (oral): Rett Syndrome • Neurodevelopmental disorder caused 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 Rett syndrome models •
Slide 12: NNZ-2566 (oral): Rett Syndrome
NNZ-2566 (oral): Rett Syndrome • Phase II trial of NNZ-2566 in adolescent and adult patients 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 mutation • 28 day tre
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 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, locomotor and social activ
Slide 14: Fragile X initial results
NNZ-2566: Fragile X initial results 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 Maze tests 10 • NNZ-25
Slide 15: NNZ-2566: Fragile X initial results
NNZ-2566: Fragile X initial results Social Behavior – Bouts of Sniffing 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 marble burying 4 and
Slide 16: Contact information
Contact information Larry Glass, CEO lglass@neurenpharma.com 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 Telephone: +64 (9) 3700 200
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