Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — Cell Series UK 2019 Conference

29 slides from Brainstorm Cell Therapeutics's conference presentation, Mar 22, 2018

  1. Slide 1: Stem Cell Therapeutic Approaches For ALS

    ‫בס"ד‬ Stem Cell Therapeutic Approaches For ALS BrainStorm Cell Therapeutics Chaim Lebovits NASDAQ: BCLI CEO & President BrainStorm Cell Therapeutics March 22, 2018

  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: Stem cells therapy for ALS

    ‫בס"ד‬ Stem cells therapy for ALS Mesenchymal Neural stem cells stem cells Immune cells 3 Abati et al Molecular Neurobiology (2019)

  4. Slide 4: MSC sources used in clinical trials

    ‫בס"ד‬ MSC sources used in clinical trials Taken from www.clinicaltrials.gov (2015) 4

  5. Slide 5: NurOwn Process

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

  6. Slide 6: NurOwn Technology Advantages

    ‫בס"ד‬ NurOwn® Technology Advantages Autologous MSCs derived from bone marrow prevent risk of immune reaction to MHC class II antigens Intermediate cryopreserved product allows for 3 years of treatment from a single bone marrow aspiration Multiple mechanism of action including immunomodulation and n

  7. Slide 7: Stem cells therapy for ALS

    ‫בס"ד‬ Stem cells therapy for ALS Mesenchymal Neural stem cells Immune cells stem cells 7 Abati et al Molecular Neurobiology (2019)

  8. Slide 8: NurOwn MOA- Focus on Neuroprotection and Immunomodulation

    ‫בס"ד‬ D NurOwn MOA- Focus on Neuroprotection 3 Placebo 3 MSC-NTF ALSFRS-R Delta Slope ALSFRS-R Delta Slope 2 p=0.4 2 p<0.01 and Immunomodulation 1 1 0 0 -1 -1 -2 -2 -3 -3 -4 -4 -5 -5 0 20 40 60 0 20 40 60 MCP-1 (pg/ml) MCP-1 (pg/ml)) NurOwn® ↑NTFs NurOwn® ↑miR 132 E Neuroprotection miR-132 Individu

  9. Slide 9: NurOwn Enhanced Neurotrophic Factor Secretion

    ‫בס"ד‬ NurOwn® Enhanced Neurotrophic Factor Secretion Paired Comparisons Pre-Post Differentiation* GDNF BDNF TSG-6 Individual participant Individual participant Individual participant VEGF HGF LIF Individual participant Individual participant Individual participant NurOwn® MSC 9 *in vitro data from

  10. Slide 10: NurOwn Mechanism of Action- Overview

    NurOwn Mechanism of Action- Overview Autophagy Glutamate Oxidative cytotoxicity stress MSC-NTF Cells Transmission of Enriched aggregated miRNA proteins 10

  11. Slide 11: Brainstorm at a Glance

    ‫בס"ד‬ Brainstorm at a Glance Clinical stage biotechnology company developing innovative autologous cellular therapies for neurodegenerative disease Fully enrolled Phase 3 clinical trial in ALS, an orphan disease with no existing cure and limited treatment options. Phase 2 progressive MS trial activ

  12. Slide 12: Clinical-Stage and Preclinical Programs

    ‫בס"ד‬ Clinical-Stage and Preclinical Programs ALS – Amyotrophic Lateral Sclerosis (Lou Gehrig’s Disease) Preclinical Phase 1 Phase 2 Phase 3 Progressive MS – Multiple Sclerosis Preclinical Phase 1 Phase 22 Phase Phase Phase 33 Autism Spectrum Disorder Preclinical Phase 1 Phase 2 Phase 3 Parkinson’s

  13. Slide 13: ALS

    on °, brainstorm pee’ cell therapeutics ALS

  14. Slide 14: ALS Incidence

    ‫בס"ד‬ ALS Incidence What is ALS? It is estimated that 450,000 people worldwide are living with ALS, with Amyotrophic lateral sclerosis (ALS), also known over 30,000 people in the U.S. suffering from this neurodegenerative as Lou Gehrig’s disease (1), is a progressive condition at any given time. ne

  15. Slide 15: Amyotrophic Lateral Sclerosis (ALS)

    ‫בס"ד‬ Amyotrophic Lateral Sclerosis (ALS) A rapidly progressive disease that leads to degeneration of upper and lower motor neurons Clinically, the effect is progressive muscle weakness leading to death, usually from respiratory failure There is no known cause and no effective treatments Familial A

  16. Slide 16: NurOwn ALS Development

    ‫בס"ד‬ NurOwn® ALS Development 2H 2020 US Phase 3 Topline Data 2017 Readout 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 16

  17. Slide 17: ALS Phase 2 Trial Design and Safety Outcomes

    ‫בס"ד‬ ALS Phase 2 Trial Design and Safety Outcomes Safety Design Study Timeline • 48 participants (16 per site) -12 to -16 weeks Screening (V1) • Randomized 3:1 • Blinded to allocation ~3-4 months Primary Outcomes -3 to -5 weeks BMA (V4) • No deaths or treatment Transplantation related SAEs CSF Wee

  18. Slide 18: ALS Functional Rating Scale–Revised (ALSFRS-R)

    ‫בס"ד‬ ALS Functional Rating Scale–Revised (ALSFRS-R) Cedarbaum, JM. et al. J Neurol Sci. 1999;169:13-21. 18

  19. Slide 19: 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 Mean change in ✓ Decline in ALS function in rapid progressors ALSFRS-R slope better predicts survival and quality of life ALSFRS-R Responder ✓ High

  20. Slide 20: NurOwn Phase 2: ALSFRS-R slope improvement

    ‫בס"ד‬ 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) 2wks 4wks

  21. Slide 21: NurOwn Phase 2: Responder Analysis

    ‫בס"ד‬ 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 compared

  22. Slide 22: Phase 2 ALS Biomarkers: CSF NTFs Significantly Increased

    ‫בס"ד‬ NurOwn® Phase 2 ALS Biomarkers: 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 transpla

  23. Slide 23: Phase 2 ALS Biomarkers: CSF Inflammation Significantly Decreased

    ‫בס"ד‬ NurOwn® Phase 2 ALS Biomarkers: CSF Inflammation 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 Pre

  24. Slide 24: Strong correlations between post-treatment NTF increase and inflammatory factor decrease

    ‫בס"ד‬ Strong correlations between post-treatment NTF increase and inflammatory factor decrease Treated Placebo Pre-transplant Post-transplant Pre-transplant Post-transplant Correlation p value Correlation p value Correlation p value Correlation p value VEGF/MCP-1 -0.017 0.9326 -0.564 0.0027 -0.005

  25. Slide 25: Correlation between reduction in MCP-1 and clinical improvement

    ‫בס"ד‬ NurOwn® Phase 2: Strong correlation between reduction in MCP-1 (2 weeks) and clinical improvement (ALSFRS-R slope improvement at 12 weeks) MSC-NTF cells Placebo 25

  26. Slide 26: Phase 2 ALS Trial Summary

    ‫בס"ד‬ Phase 2 ALS Trial Summary • Intrathecal NurOwn treatment was safe and well tolerated • Multiple doses will be needed to sustain efficacy • Rapid progressor subgroup showed higher efficacy signal • Responder analysis highlights clinically important response • A significant increase in neurotro

  27. Slide 27: ALS Phase 3 Trial Design & Assessments

    ‫בס"ד‬ ALS Phase 3 Trial Design & Assessments 27

  28. Slide 28: NurOwn ALS Phase 3 Clinical Trial

    ‫בס"ד‬ NurOwn® ALS Phase 3 Clinical Trial Study Timelines Site Locations ▪ Enrollment completed October 2019 • Study duration: Mayo Clinic MGH • 11.5 months total (Rochester, MN) (Boston, MA) California Pacific Medical Center, • Seven months post-first (San Francisco, CA) transplantation Cedars Sina

  29. Slide 29: BrainStorm Cell Therapeutics

    ‫בס"ד‬ BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You

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