Slide 1: Biotech Showcase Corporate Presentation
January 12, 2016 Biotech Showcase Corporate Presentation
Slide 2: Safe Harbor Statement
NEURALSTEM, INC. Safe Harbor Statement Safe Harbor statements under the Private Securities Litigation Reform Act of 1995 : This presentation contains forward - looking statements as defined in Section 27 A of the Securities Act of 1933 as amended, and section 21 E of the Securities Exchange Act of 1
Slide 3: Overview
Overview 2 • Founding science utilizing regionally specific stem cells • Novel small molecule drug screening platform targeting neurogenesis; identified multiple compounds • Phase II lead compound (NSI - 189) targeting major depressive disorder (MDD) • Novel neurogenic, synaptogenic MOA • Clinical s
Slide 4: Dual Platform Pipeline
Dual Platform Pipeline 3 Lead Small Molecule Asset Targeting Depression Cell therapy targeting high unmet medical need populations
Slide 5: Small Molecule Overview
NSI - 189 Small Molecule Overview 4 NSI - 189 Small Molecule • Novel MOA increasing synaptic connections • Patent composition of matter (US Pat.No . 7,560,553) till 2029 NSI - 189 Phase II Major Depressive Disorder (MDD) • Commencement early 2016 • Data 2H 2017 Compelling early clinical data • Rando
Slide 6: Differentiation
NSI - 189 Differentiation 5 N ovel neurogenic MOA • I ncreasing synaptic connections in the hippocampus • Neurogenesis in the hippocampus is linked to depression and other psychiatric and neurologic disorders No major s ide affects; better than current depression therapies • No metabolic or sexual d
Slide 7: Phase Ib study design
Cohort 1 N=8 (6 drug, 2 placebo) 40 mg QD Cohort 2 N=8 (6 drug, 2 placebo) 40 mg BID Cohort 3 N=8 (6 drug, 2 placebo) 40 mg TID Acute treatment: 28 days Follow up: Days 35, 42, 49, 70, 84 (End - of - study) NSI - 189 Phase Ib NSI - 189 Phase Ib double - blind, randomized, placebo - controlled, multi
Slide 8: MDD Phase Ib results
NSI - 189 MDD Phase Ib 7 p=0.02 d=0.90 Study Day -20 0 20 40 60 80 100 Symptoms of Depression Questionnaire 2.0 2.2 2.4 2.6 2.8 3.0 3.2 3.4 3.6 3.8 Placebo NS-189 NS-189 1x per day NS-189 2x per day NS-189 3x per day p=0.03 d=1.10 Day 84 Day 28 Symptoms of Depression Questionnaire (SDQ) p=0.09 d=0.9
Slide 9: MDD Phase Ib cognitive and physical functioning
p=0.01 d=0.94 Study Day -20 0 20 40 60 80 100 Cognitive and Physical Functioning Questionnaire 2.0 2.5 3.0 3.5 4.0 4.5 5.0 Placebo NS-189 NS-189 1x per day NS-189 2x per day NS-189 3x per day p<0.01 d=1.20 Day 28 Day 84 NSI - 189 MDD Phase Ib 8 All: A Phase 1B, Randomized, Double - Blind, Placebo -
Slide 10: NSI-189 Biomarkers
NSI - 189: Biomarkers 9 Quantitative EEG ( qEEG ) biomarker: • Statistically significant change in brain waves over hippocampus and prefrontal cortex Blood biomarker panel : • Consistent with therapeutic effects shown by clinical measures MRI: • Trend for increase volume in left hippocampus High - f
Slide 11: Phase II Trial Design
NSI - 189 Phase II Trial Design 10 Study Objectives • Primary: Montgomery - Asberg Depression Rating Scale ( MADRS ) • Secondary: response and remission rates, durability effect Principal Investigator: Maurizio Fava, M.D. Slater Family Professor of Psychiatry at Harvard Medical School, Massachusetts
Slide 12: Cell Therapy Overview
Biological Activity: • Multiple mechanisms of action: neuroprotection , bridging , regeneration • Definitive evidence of long - term graft survival without immunosuppression in ALS patients NSI - 566 spinal cord stem cell - derived programs • Phase Ib Chronic Spinal Cord Injury ( cSCI ) – Funded by
Slide 13: Neuralstem Catalysts
Neuralstem Catalysts 12 Program Milestones • Phase II MDD FDA Approval 1Q16 • Sept 2015 FDA protocol submission • Phase I cSCI d ata 1Q16 • Ongoing Phase I/II stroke trial 2016 • Phase II MDD d ata 2H17 Other Initiatives • Exploring additional NSI - 189 possible indications • Secure grant money for