Slide 1: April 2016 Corporate Presentation
April 2016 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: Key Highlights
Key Highlights • Refocused Business Strategy: – New, experienced management team – Focus on lead program: NSI - 189 – Partner NSI - 566 programs • NSI - 189: Novel Neurogenic Small Molecule targeting MDD – Compelling early clinical data – I ntellectual property portfolio, protection expiring from 20
Slide 4: Management
Management 3 Richard Daly, CEO • President, AstraZeneca Diabetes, US; President, BMS Diabetes, US; Co - Founder & Partner, SagePath Partners; EVP , Takeda Pharmaceuticals (North and South America); VP, Commercial Strategy, TAP Pharmaceuticals • Boards: Catalyst Pharmaceuticals; Synergy Pharmaceutica
Slide 5: Scientific Advisory Board
NSI - 189 Scientific Advisory Board Dr. Maurizio Fava Harvard, MGH, Executive Vice Chair, Dept. of Psychiatry Principal Investigator: NSI - 189 Phase 2 MDD clinical trial Dr. Michael Thase Univ. of Pennsylvania, Chief. Division of Mood and Anxiety Disorders Treatment and Research Program Dr. Mark Fr
Slide 6: Pipeline Status
Pipeline 5 Status* (1) Phase II MDD clinical trial results to be provided 2H17 (2) Second Indication to be determined in 2Q16 (3) Ongoing preclinical studies in NSI - 189 and other undisclosed compounds. Multiple NSI - 189 POP publications to be submitted in 2016 (4) NSI - 566: Active efforts to pur
Slide 7: Intellectual Property
Intellectual Property S mall M olecule 11 US & 65 World Issued and Pending Patents – 16 Neurogenic compounds (including 189), composition of matter, US ( 7,560,553) to 2024, Patent Extension to 2029. – Assay method for screening neurogenic compounds, US ( 8,293,488) and Europe to 2023 – Synthesis me
Slide 8: MDD Market Opportunity
MDD Market Opportunity Patients First Line TRD I TRD II 4th line + % patients in given line of therapy 33% 17% 10% 40% % patients that fail given line of therapy 67% 75% 80% N/A Information regarding patient failure derived from: Rush AJ, Fava M, et al; Am J Psychiatry 163:11, November 2006 * Anxiet
Slide 9: Overview
NSI - 189 Overview • Unique New Chemical Entity (NCE) • Novel neurogenic MOA • Highly stable and well characterized • MDD Market Opportunity • U nsatisfied patient population* • High patient turnover rate in MDD* • Strong IP position through 2024 (2029 with patent term extension) • Efficacy: • Compe
Slide 10: Building the Case for MOA
Building the Case for MOA Extensive screening showed novel MOA vs. currently marketed therapies Screening: • 52 neurotransmitter related receptors/ion channels/ enzymes Novoscreen : Adenosine, GABA, Glutamate, Histamine, Muscarinic, Nicotinic, norepinephrine, opioid, or and serotonin receptors, Ca +
Slide 11: Clinical Results from MDD Phase Ib
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, 56, 70, 84 (End - of - study) Clinical Results from NSI - 189 MDD Phase Ib NSI - 189 Phase Ib double - blind, randomize
Slide 12: Clinical Results from MDD Phase Ib
Clinical Results from NSI - 189 MDD Phase Ib 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 Questionnair
Slide 13: Clinical Results from MDD Phase Ib
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 Clinical Results from NSI - 189 MDD Phase Ib All: A Phase 1B, Randomized, Double
Slide 14: Biomarker Results from MDD Phase Ib
Biomarker Results from NSI - 189 MDD Phase Ib Blood biomarker panel : • Blood panel analysis correlates to MADRS r esponse rate • MDD panel was developed based on SSRIs activity profile • Rapid and persistent efficacy 13 Response Rate Partial Responder (<14) +Responders ( ≥ 50%) Responder ( ≥ 50%) R
Slide 15: qEEG Biomarker Results from MDD Phase Ib
Topographs of High Frequency alpha (10 - 12 Hz): Day 28 from Baseline Left posterior temporal (T5) (t=2.45, p=0.02) Left parietal regions (P3) (t=3.31, p=0.004) qEEG Biomarker Results from NSI - 189 MDD Phase Ib Quantitative EEG ( qEEG ) biomarker : • Increases coherence activity between prefrontal
Slide 16: MDD Phase Ib: Adverse Events
NSI - 189 MDD Phase Ib : Adverse Events 15
Slide 17: Phase II MDD Trial Milestones
NSI - 189 Phase II MDD Trial Milestones: • First Patient enrolled anticipated 2Q16 • Phase 2 Results expected 2H17 Study Objectives • Primary: Montgomery - Asberg Depression Rating Scale (MADRS ) • Secondary: SDQ , HAMD17, CGI - S, CPFQ, SFI, Cogscreen Battery, Cogstate Brief Battery Principal Inves
Slide 18: Phase II MDD Trial Highlights
NSI - 189 Phase II MDD Trial Highlights • Experienced MDD trial sites (n=12) • Dual patient screening requirement – Secondary confirmatory screen: i ndependent, remote MADRS diagnosis by MGH • P lacebo - reducing prescreen process, re - randomization Study Design • Three arm: 40mg BID, 40mg QD, & pl
Slide 19: Preclinical Data
Preclinical Data • Orally active, neurogenic, neuroregenerative , compound for the treatment of depression, cognitive impairment, and neurodegeneration • A new chemical entity with novel mechanism of action, molecular target yet unknown, but not mediated by SSRI or SNRI or by BDNF release or via any
Slide 20: Pipeline Expansion
• Based on human neural stem cell differentiation in vitro • Through the use of our proprietary drug screening platform • Captures large window of neurodevelopment: neurogenesis to synaptogenesis • Multiple potential s ites of action d uring s tages of neurogenesis • Anticipated to identify addition
Slide 21: Key Highlights
Key Highlights • Experienced Management Team • Renowned Advisory Board • Lead Candidate: NSI - 189 novel neurogenic small molecule • Protected IP: 2024 (2029 with patent term extension) – New Chemical Entity • Near term milestones: – Phase II MDD trial; results expected 2H17 – Preclinical POP public