Palisade Bio, Inc. · NASDAQ:PALI
Palisade Bio — Neuralstem — Investor Presentation (April 2017)
25 slides from Palisade Bio's investor presentation, Apr 19, 2017
Slide 1: Neuralstem Inc
NEURALSTEM INC April 2017
Slide 2: Safe Harbor Statement
Safe Harbor Statement Although we believe that we have a reasonable basis for each forward - looking statement contained in this presentation, we caution you that forward - looking statements are not guarantees of future performance and that our actual results of operations, financial condition and
Slide 3: Key Highlights
Key Highlights Lead Program in Phase II Development – Novel neurogenic small molecule approach – NSI - 189: Positive, randomized placebo - controlled Phase 1b in MDD – Phase II Major Depressive Disorder (MDD) • Efficacy data expected in 3Q 2017 – Montgomery - Asberg Depression Rating Scale (MADRS) p
Slide 4: Scientific Advisory Board
Scientific Advisory Board Comprised of World Class Psychiatric, Clinical and Regulatory Experts 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
Slide 5: Management
Management 5 Richard Daly President & Chief Executive Officer Karl Johe , Ph.D. Chief Scientific Officer Co - founder Jonathan Lloyd Jones Chief Financial Officer Thomas Hazel, Ph.D. Senior Vice President, Research 20 years as Stem Cell Discovery Program Director
Slide 6: Neuralstem's proprietary technology uses regionally specific neural stem cells
Neuralstem’s proprietary technology uses regionally specific neural stem cells for the development of CNS therapies. 6 Small molecule development capability & regenerative medicine Utilizing Neural Stems Cells to Develop Novel CNS Therapies NSI - 566: Cell Therapy • Regionally specific stem cell - l
Slide 7: Pipeline
Pipeline 7 Compound Indication Preclinical Phase I Phase II Phase III Status Small Molecule: Lead Asset NSI - 189 Major Depression Disorder (MDD) Topline Results 3Q17 Supplementary Preclinical Program Angelman Syndrome Ongoing MCAO Stroke Type 1 & 2 Diabetes - related neuropathy Irradiation - induce
Slide 8: NSI-189: A New Molecular Entity for Major Depressive Disorder
NSI - 189: A New Molecular Entity for Major Depressive Disorder
Slide 9: Compelling Efficacy Demonstrated To-Date
Compelling Efficacy Demonstrated To - Date – Phase Ib in Major Depressive Disorder (MDD) randomized, double - blind data • New chemical entity • Large effect size • Cognitive benefit profile • Potentially disease modifying • Excellent safety profile Phase II ongoing in MDD : randomized, double - bli
Slide 10: Major Depressive Disorder Large, Unmet Medical Need
Major Depressive Disorder Large, Unmet Medical Need: 16M US Patients TRD II - Adjunct Rx - Rx Switch - Other 4 th / 5 th - New combos TRD I - Rx Switch - Adjunct Rx - SSRI / SNRI First Line - Monotherapy - SSRI/SNRI Large patient Rx turnover due to low efficacy Low barrier to entry due to continued
Slide 11: NSI-189 MDD Phase Ib trial 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 Drug free observational follow up: days 35, 42, 49, 56, 70, 84 NSI - 189 MDD Phase Ib trial design NSI - 189 Phase Ib double - blind, randomized, p
Slide 12: Phase 1b Results Demonstrate Trends in Efficacy & Durability
NSI - 189 Phase 1b Results Demonstrate Trends in Efficacy & Durability Across Physician & Patient Depression Scales 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
Slide 13: NSI-189 MDD Phase Ib clinical results
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 clinical results All: A Phase 1B, Randomized, Double - Bl
Slide 14: NSI-189: Excellent Safety Profile with No Serious SAEs
NSI - 189: Excellent Safety Profile with No Serious SAEs 14
Slide 15: NSI-189: Ongoing Phase II MDD Trial, Topline Results Expected 3Q 2017
NSI - 189: Ongoing Phase 2 MDD Trial, Topline Results Expected 3Q 2017 15 Study Objectives – Primary: Montgomery - Asberg Depression Rating Scale (MADRS ) – Secondary*: SDQ, HAMD17, CGI - S, CPFQ, SFI – Exploratory: Cogscreen Battery, Cogstate Brief Battery Innovative Study Design – Randomized, doub
Slide 16: NSI-189: Preclinical Data Support Neurogenesis, Cognitive Enhancement & Durability
NSI - 189: Preclinical Data Support Characteristics of Neurogenesis, Cognitive Enhancement & Durability 16
Slide 17: Preclinical Overview
Preclinical Overview 17 • Treatment of a rat model for ischemic stroke shows a durable effect in promoting behavioral recovery that corresponds with increased neurogenesis • Type 1 & 2 diabetic neuropathy reversal and prevention • Prevention of cognitive deficit in irradiated mice • MOA: Enhances lo
Slide 18: Orphan Opportunity in Angelman Syndrome
Orphan Opportunity in Angelman Syndrome 0 50 100 150 200 250 -10 -5 0 5 10 15 20 25 30 35 40 AS WT-vehicle AS-NSI189 EPSP slope (% of baseline) Time (min) NSI - 189 restores LTP in Angelman Syndrome Mice Confirmatory model in a genetic disease By courtesy of Yan Liu 1 , Xiaoning Bi 1 , Michel Baudry
Slide 19: NSI-189 Enhances LTP Magnitude
NSI - 189 Enhances LTP Magnitude 19 TBS after 2.5 h incubation 0 50 100 150 200 250 300 350 -10 -5 0 5 10 15 20 25 30 35 40 WT-Vehicle WT-NSI189 Vehicle EPSP slope (% of baseline) TBS after 3.5 h incubation 0 50 100 150 200 250 300 350 -10 -5 0 5 10 15 20 25 30 35 40 WT-Vehicle WT-NSI189 Vehicle Tim
Slide 20: Stem Cell Development Efforts & Strategy
Stem Cell Development Efforts & Strategy
Slide 21: Cell Therapy: Biological Activity Across Three Indications
Cell Therapy : Biological Activity A cross T hree I ndications 21 – ALS Phase 1 & 2 follow - up evaluation – Phase I stroke completed dosing all 9 patients and currently evaluating safety – cSCI is currently evaluating 4 Phase 1 thoracic patients; Phase 1 trial recruiting additional ( Group B) 4 cer
Slide 22: Lead Product: NSI-566
Lead Product: NSI - 566 Chemically defined culture system – No serum, no feeder cells, no particulates, no unknown raw material – Fully tested for potential pathogens; validated SOPs Efficient expansion – Multi - tiered cell banks for maximum efficiency – Scalable expansion – Relatively small infras
Slide 23: NSI-566: Preliminary Data in Indications
NSI - 566: Preliminary Data in 3 Indications PROGRAM OVERVIEW - Transplantation into spinal cord of ALS - Phase 1 & Phase 2a dose - escalation, safety studies completed - 30 subjects with 2 - 6 years of safety data KEY TAKEAWAYS - Procedure and treatment is well - tolerated - L ong - term cell graft
Slide 24: Continued Execution of New Corporate & Clinical Development Strategy
Continued Execution of New Corporate & Clinical Development Strategy 24 2016 x Jan : Refocused clinical development strategy on NSI - 189 x Feb : Rich Daly joins as the new CEO x May : Initiation of Phase 2 MDD study for NSI - 189 x $9mn institutional capital raise x Corporate reorganization to alig
Slide 25: Key Highlights
Key Highlights Lead Program in Phase II Development – Novel neurogenic small molecule approach – NSI - 189: Positive, randomized placebo - controlled Phase 1b in MDD – Phase II Major Depressive Disorder (MDD) • Efficacy data expected in 3Q 2017 – Montgomery - Asberg Depression Rating Scale (MADRS) p