Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — Conference Presentation (February 2020)

23 slides from Brainstorm Cell Therapeutics's conference presentation, February 2020

  1. Slide 1: BrainStorm Cell Therapeutics

    ‫בס"ד‬ BrainStorm Cell Therapeutics Prepared NASDAQ: BCLI February 2020 March 22, 2018 1

  2. 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

  3. Slide 3: Brainstorm At-A-Glance

    ‫בס"ד‬ Brainstorm At-A-Glance CLINICAL STAGE ADVANCED STAGE CLINICAL LARGE ADDRESSABLE BIOTECHNOLOGY COMPANY PROGRAMS MARKETS Potential to be first-in-class ALS autologous stem cell Repeat Dose Phase 3 ALS trial 30,000 patients (US) therapy for ALS and other fully enrolled 450,000 (Worldwide) neurod

  4. Slide 4: Brainstorm's Pipeline

    ‫בס"ד‬ Brainstorm’s Pipeline ALS – Amyotrophic Lateral Sclerosis (Lou Gehrig’s Disease) Preclinical Phase 1 Phase 2 Phase 3 Progressive MS – Multiple Sclerosis Preclinical Phase 1 Phase 2 Phase Phase 33 Parkinson’s Disease Preclinical Phase 1 Phase 2 Phase 3 Autism Spectrum Disorder Preclinical Phas

  5. Slide 5: NurOwn® - A Platform Technology Across CNS

    ‫בס"ד‬ NurOwn® - A Platform Technology Across CNS Parkinson’s Disease Global patients 10,000,000 US patients 1,000,000 Progressive Huntington’s ALS Multiple Disease Sclerosis Global patients 300,000 Global patients 450,000 Global patients 1,250,000 US patients 30,000 US patients 30,000 US patients 5

  6. Slide 6: NurOwn® Process

    ‫בס"ד‬ NurOwn® Process 1 2 Isolation 12 Days Manufacturing process begins with a bone marrow aspiration (BMA) to Expansion Intermediate MSC product is harvest adult MSCs from the patient cryopreserved Harvesting Cryopreservation Transplantation The proprietary culture medium The cells are injected i

  7. Slide 7: NurOwn® (MSC-NTF Cells) Technology

    ‫בס"ד‬ NurOwn® (MSC-NTF Cells) Technology MSC Cell Identity (Surface Markers) CD44 CD73 CD90 CD105 MSC MSC-NTF CD3 Neuroprotection CD14 CD19 Immunomodulation CD34 CD45 HLA-DR (Fold Increase NTFs Co-culture MSC-NTF MSC-NTF vs. MSC) and PBMCs (% Reduction) MSC LIF 100 HGF MSC-NTF GDNF 50 BDNF VEGF 0 I

  8. Slide 8: NurOwn® increases Treg Cells

    ‫בס"ד‬ NurOwn® increases Treg Cells T Regulatory Cells Increased in Phase 1/2a T Regulatory Cells Increased In-vitro PBMC co-cultured with A 2.2 Post-transplant B 2.2 Post-transplant PBMC only 2.0 2.0 MSC-NTF 1.8 1.8 1.6 1.6 %CD4+CD25+ lymphocytes %CD4+CD25+ lymphocytes 1.4 1.4 1.2 1.2 1.0 1.0 0.8 0

  9. Slide 9: NurOwn® ALS Development

    ‫בס"ד‬ NurOwn® ALS Development 2H 2020 ALS US Phase 3 Topline Data Readout 2017 2019 ALS ALS US Phase 3 US Phase 3 2014 Randomized trial Fully Enrolled ALS US Phase 2 2010 Randomized trial 2007 ALS Phase 1/2a NurOwn Open label Preclinical Development 200 26 48 # of Study Participants Phase 1/2 Phase

  10. Slide 10: ClinicalTrials.gov identifier NCT02017912 Berry et al Neurology 2019

    ‫בס"ד‬ ClinicalTrials.gov identifier NCT02017912 Berry et al Neurology 2019 10

  11. Slide 11: Phase 2 Study Design

    ‫בס"ד‬ Phase 2 Study Design ClinicalTrials.gov identifier NCT02017912 Berry et al Neurology 2019 11

  12. Slide 12: Phase 2 Clinical Trial Demographics: Baseline Characteristics Balanced

    ‫בס"ד‬ Phase 2 Clinical Trial Demographics: Baseline Characteristics Balanced Berry et al Neurology 2019 12

  13. Slide 13: ALSFRS-R Efficacy Outcomes

    ‫בס"ד‬ ALSFRS-R Efficacy Outcomes All n=46 Rapid Progressors n=21 A Change from post-treatment to pre-treatment slope B Change from post-treatment to pre-treatment slope 4 4 ALSFRS-R LS mean change in slope * 3 3 Mean ALSFRS-R 2 2 * Post-pre slope 1 1 change 0 0 MSC-NTF -1 -1 Placebo -2 -2 2wks 4wks

  14. Slide 14: Biomarkers

    48s. brainstorm pee’ cell therapeutics Biomarkers 14

  15. Slide 15: CSF Neurotrophic Factors Increased Post-Treatment

    ‫בס"ד‬ CSF Neurotrophic Factors Increased Post-Treatment VEGF HGF LIF 1000 600 18 900 ** *** * 500 16 800 14 700 400 12 pg/ml 600 NurOwn® 10 500 300 8 400 (n = 26) 300 200 6 4 200 100 V5 -Pre transplantation 100 2 0 0 0 V5 V6 V5 V6 V5 V6 V6 - Post transplantation 1000 600 18 900 16 500 800 14 700 Pl

  16. Slide 16: CSF Inflammatory Biomarkers Reduced Post-Treatment

    ‫בס"ד‬ CSF Inflammatory Biomarkers Reduced Post-Treatment MCP-1 SDF-1 50,000 CHIT-1 50 250 *** ** 45,000 45 40,000 * 40 200 35 35,000 NurOwn NurOwn®® 30 150 30,000 pg/ml 25 25,000 (n(n==26) 26) 20 100 20,000 15 15,000 10 50 10,000 V5 -Pre transplantation 5 5,000 0 0 0 V6 - Post transplantation V5 V6

  17. Slide 17: Post-treatment % reduction CSF MCP-1, Caspase-3 and SDF-1

    ‫בס"ד‬ Post-treatment % reduction CSF MCP-1, Caspase-3 and SDF-1 MCP-1 (40%) Caspase-3 (40%) SDF-1 (22%) MCP-1 Caspase-3 SDF-1 10 40 10 30 0 0 20 % Caspase-3 reduction % MCP-1 reduction % SDF-1 reduction p=0.001 10 -10 -10 p=0.0001 0 p=0.096 -20 -10 -20 -20 -30 -30 -30 -40 -40 -40 -50 -50 -60 -50 MS

  18. Slide 18: Phase 2 Clinical Trial: Biomarker Correlations

    ‫בס"ד‬ Phase 2 Clinical Trial: Biomarker Correlations CSF MCP-1 inverse correlation with VEGF CSF MCP-1 inverse correlation with ALSFRS-R Berry et al Neurology 2019; Nagata Neurol Res 2007 18

  19. Slide 19: NurOwn® ALS Phase 3 Study

    ‫בס"ד‬ NurOwn® ALS Phase 3 Study Inclusion Outcomes Bone Marrow Aspiration • ≤ 60 years • ALSFRS-R responder MSC-NTF analysis Randomization • SVC > 65% (n=100) • Safety • ALS ≤ 2 years • ALSFRS-R change from • Rapid progressors baseline Exclusion Placebo • SVC (n=100) • Tracheostomy-free survival •

  20. Slide 20: Phase 3 ALS Clinical Trial Status

    ‫בס"ד‬ Phase 3 ALS Clinical Trial Status • Phase 3 enrollment completed October 2019 • DSMB safety review conducted end of October 2019 – Trial to continue – No protocol changes • Agreement with FDA on potential regulatory pathways for approval in ALS- February 2020 • Seeking additional grant suppor

  21. Slide 21: Phase 2 Progressive MS Clinical Trial Status

    ‫בס"ד‬ Phase 2 Progressive MS Clinical Trial Status • IND granted December 2018 • Repeat intrathecal dosing MSC-NTF cells every 2 months • Validated clinical endpoints and biomarkers • 5 clinical trial sites in US • First subjects enrolled March 2019 • DSMB safety review conducted end of December 20

  22. Slide 22: Brainstorm Senior Leadership Team

    ‫בס"ד‬ Brainstorm Senior Leadership Team Ralph Kern, Chaim Lebovits, Arturo Araya, Preetam Shah, MD, MHSc Uri Yablonka President & MBA PhD, MBA Chief Operating EVP & Chief Chief Executive Chief Commercial EVP & Chief Officer & Chief Business Officer Officer Officer Financial Officer Medical Officer

  23. Slide 23: BrainStorm Cell Therapeutics

    ‫בס"ד‬ BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You 23

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