Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Investor Presentation (November 2018)
20 slides from Brainstorm Cell Therapeutics's investor presentation, November 2018
Slide 1: BrainStorm Cell Therapeutics
בס"ד BrainStorm Cell Prepared Therapeutics NASDAQ: BCLI March 22, 2018 November 2018
Slide 2: Forward Looking Statement
בס"ד Forward Looking Statement Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implie
Slide 3: Brainstorm at a Glance
בס"ד Brainstorm at a Glance Targeted, innovative, proprietary and validated autologous cellular technology platform for the treatment of neurodegenerative disease Actively recruiting ALS phase 3 Clinical Trial, an orphan disease with no existing cure and limited treatment options Large addressable
Slide 4: NurOwn Technology
בס"ד NurOwn® Technology 1 2 Manufacturing process begins with a bone Patient-derived MSCs secrete neurotrophic factors Isolation marrow aspiration to harvest adult MSCs Expansion and anti-inflammatory cytokines from the patient • Promotes neuronal survival through • Stem cells are isolated and the
Slide 5: NurOwn - Potential Clinical Indications
בס"ד NurOwn® - Potential Clinical Indications Parkinson’s Disease Global patients 10,000,000 US patients 1,000,000 Huntington’s Multiple Disease ALS Sclerosis Global patients 300,000 Global patients 220,000 Global patients 1,250,000 US patients 30,000 US patients 30,000 US patients 500,000 Autism
Slide 6: What is ALS? ALS at a Glance
בס"ד What is ALS? ALS at a Glance Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease* It is estimated that around 45,000 people worldwide are is a progressive, incurable living with ALS, with other 30,000 people in the USA disease of the nervous system. suffering fro the condition at any
Slide 7: NurOwn ALS Development
בס"ד NurOwn® ALS Development 2017 ALS US Phase 3 2014 ALS 2010 US Phase 2 2007 ALS Phase 1/2a NurOwn Early Development 200 # of Study Participants 48 26 Phase 1/2 Phase 2 Phase 3 7
Slide 8: ALS Phase 2 Trial Design
בס"ד ALS Phase 2 Trial Design Safety Design Study Timeline • 48 participants (16 per site) -12 to -16 Screening (V1) • Randomized 3:1 weeks • Blinded to allocation ~3 months Primary Outcomes -3 to -5 weeks BMA (V4) • No deaths or treatment Transplantation related SAEs CSF Week 0 (V5)* Collection •
Slide 9: Phase 2 Trial – Approach to Data Analysis
בס"ד Phase 2 Trial – Approach to Data Analysis ALS Rapid Progressor Subgroup Has Different Phase 2 Trial: 3 Types of Efficacy Analysis Characteristics 1. Mean change in ✓ Change is ALS function in rapid progressors slope more closely predicts survival and quality of life ✓ Higher response rate in
Slide 10: NurOwn Phase 2: ALSFRS-R slope improvement (mean slope change/month)
בס"ד 1. NurOwn® Phase 2: ALSFRS-R slope improvement (mean slope change/month) All participants (n=46) Rapid progressors (n=21) Post-treatment - pre treatment slope Post treatment – pre treatment slope ALSFRS-R LS mean change in slope ALSFRS-R LS mean change in slope 2 4 1 2 0 0 (1) (2) (1) (2) 2wk
Slide 11: NurOwn Phase 2: Responder Analysis
בס"ד 2. NurOwn® Phase 2: Responder Analysis: (≥1.5 points/month ALSFRS-R slope improvement) All participants (n=46) Rapid progressors (n=21) ALSFRS-R ≥ 1.5 points improvement/month in ALSFRS-R ≥ 1.5 points improvement in post-treatment slope post-treatment slope compared to pre-treatment slope com
Slide 12: Phase 2 ALS Trial: CSF NTFs Significantly Increased Post-Treatment
בס"ד NurOwn® Phase 2 ALS Trial: CSF NTFs Significantly Increased 2 Weeks Post- Treatment Compared to Baseline VEGF (1) HGF (1) LIF (1) Pg/ml Pg/ml Pg/ml 1,000 * 600 ** 18 *** NurOwn® 800 480 15 12 600 360 (n = 26) 9 400 240 6 200 120 3 0 0 0 V5 V6 V5 V6 V5 V6 Pre transplantation Post transplantati
Slide 13: Phase 2 ALS Trial: CSF Inflammatory Markers Significantly Decreased
בס"ד NurOwn® Phase 2 ALS Trial: CSF Inflammatory Markers Significantly Decreased 2 weeks Post- Treatment Compared to Baseline CRP MCP-1 SDF-1 MIP-1b Pg/ml Pg/ml Pg/ml Pg/ml 1,600 50 *** 250 ** 10 NurOwn® 1,280 40 200 8 960 30 150 6 (n = 26) 640 20 100 4 320 10 50 2 0 0 0 0 V5 V6 V5 V6 V5 V6 V5 V6
Slide 14: Phase 2 ALS Trial Summary
בס"ד Phase 2 ALS Trial Summary Secreted Reduced Functional Neurotrophic Inflammation Improvement Factors VEGF LIF MCP-1 Change from Post-treatment to pre- treatment slope Pg/ml Pg/ml Pg/ml ALSFRS-R LS mean slope change 1,000 * 18 50 *** 4.0 *** 750 14 38 2.5 500 9 25 1.0 250 5 13 (0.5) 0 0 0 (2.0)
Slide 15: NurOwn ALS Phase 3 Clinical Trial
בס"ד NurOwn® ALS Phase 3 Clinical Trial Pre-Treatment Treatment Outcomes • Inclusion criteria • N=200 patients • ALSFRS-R responder analysis ▪ Less than 60 years of age ▪ Enrollment completed by • Safety ▪ SVC > 65% mid-2019 • ALSFRS-R change from baseline ▪ ALS ≤ 2 years • 1:1 randomization • SVC
Slide 16: Thank You
בס"ד BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You
Slide 17: Appendix
pee’ cell therapeutics Appendix
Slide 18: Additional Professional Board Members
בס"ד Additional Professional Board Members June S. Almenoff MD PhD • Chief Operating Officer & Chief Medical Officer of Innovate Biopharmaceutical • Formerly President and CMO of Furiex Pharmaceuticals and Board member of Tigenix NV (TIG) Tony Polverino PhD • CSO - Kite Pharmaceuticals 18
Slide 19: Scientific Advisory Board
בס"ד Scientific Advisory Board Jerold Chun, M.D., PhD -Chair • Neuroscientist, Professor and Senior Vice President of Neuroscience Drug Discovery, Sanford Burnham Prebys Medical Discovery Institute, San Diego CA. Stanley H. Appel, M.D • Peggy and Gary Edwards Distinguished Endowed Chair for the Tr
Slide 20: IP Portfolio
בס"ד IP Portfolio Allowed Granted Patent Name/ Int. App. No. Pending Jurisdictions Expiry Date Jurisdictions Jurisdictions ISOLATED CELLS AND POPULATIONS COMPRISING SAME FOR THE TREATMENT OF CNS US Europe, US 2030 DISEASES/PCT/IL2006/000699 MESENCHYMAL STEM CELLS FOR THE TREATMENT OF Hong Kong US,
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