Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — Technical Presentation (November 2023)

14 slides from Brainstorm Cell Therapeutics's technical presentation, Nov 17, 2023

  1. Slide 1: NurOwn for ALS

    NurOwn for ALS Biomarker exploration of NurOwn multimodal mechanism of action on neuroinflammation, neuroprotection and neurodegeneration Bob Dagher, MD Executive Vice President Chief Development Officer November 17, 2023

  2. Slide 2: Complex & Multi-faceted Mechanisms Underlying ALS

    Complex & Multi-faceted Mechanisms Underlying ALS  Neurodegeneration may be linked to deficient neuroprotection and neuroinflammation1  Stem cell treatment potential to synergistically tackle interrelated pathological mechanisms  MSCs play key role in immunomodulation 1. Chen, 2020

  3. Slide 3: Stem Cells Exhibit Multiple Physiological Actions

    Stem Cells Exhibit Multiple Physiological Actions Function of NSC’s fundamental physiological role¹ Neuroprotection & trophic support via diffusible factors, gap junctions & exosomes  Scavenging ROS & excitotoxins  Reducing inflammation & scarring  Promoting angiogenesis  Repairing the blood-bra

  4. Slide 4: Stem Cell Therapy Success Lessons

    Stem Cell Therapy Success Lessons  Treat as early in the disease process as possible¹ o Protecting established neural circuits/networks much more tractable than reconstructing or replacing them  Transplanted stem cell to participate in homeostatic processes of target organ (brain & spinal cord) 

  5. Slide 5: NurOwn (MSC-NTF): A Novel Stem Cell Therapy

    NurOwn (MSC-NTF): A Novel Stem Cell Therapy  Modulates neuroinflammatory and neurodegenerative disease processes o Promotes neuronal survival o Improves neurological function  Autologous cells recognized as individual’s own cells o Safer choice in avoiding unwanted immune responses Ref: FDA CMC Re

  6. Slide 6: BCT-002-US: NurOwn Phase 3 Trial in ALS

    BCT-002-US: NurOwn Phase 3 Trial in ALS Bone Marrow Aspiration NurOwn (N = 95) R 1:1 Placebo (N = 94) Baseline 20-week pre-treatment B 28-week treatment & follow-up Week Week Week Week Week Week Week Week Week Week -18 to -20 -6 to -9 0 4 8 12 16 20 24 28 Dose-1 Dose-2 Dose-3 Biomarkers Sampling (CS

  7. Slide 7: BCT-002-US: Biomarkers from Different Pathways

    BCT-002-US: Biomarkers from Different Pathways Biological Biomarkers Pathway Neurodegeneration NfL, pNfH, DR6, Caspase-3, TWEAK, UCH-L1, miR-142-5p, Tau Neuroinflammation Anti-inflammatory LAP (TGF-β1), Fetuin-A, IL-37, MSR1, miR-146a-5p, miR-146b-5p MCP-1, CHI3L1/YKL-40, Chitotriosidase-1, IP-10, S

  8. Slide 8: Significantly Lowers NfL vs Placebo

    NurOwn Significantly Lowers NfL vs Placebo Neurodegeneration 7,000 Stable from Baseline 6,000 NfL Benefit pg/mL (95% CI) 5,000 4,000 * 11% Reduction 3,000 0 2 4 8 12 16 20 Weeks Dose-1 Dose-2 Dose-3 * p < 0.05

  9. Slide 9: Positive Impact on Neurodegeneration Biomarkers

    NurOwn Positive Impact on Neurodegeneration Biomarkers NurOwn pNfH: Neurodegeneration DR6: Neurodegeneration (N = 95) Placebo (N = 94) Benefit pNfH DR6 pg/mL pg/mL Benefit (95% CI) (95% CI) Weeks Weeks Dose-1 Dose-2 Dose-3 Dose-1 Dose-2 Dose-3 * p < 0.05

  10. Slide 10: Significantly Impacts Inflammatory Biomarkers

    NurOwn Significantly Impacts Inflammatory Biomarkers NurOwn Anti-inflammatory Pro-inflammatory (N = 95) 45% Placebo Increase 5,000 700 (N = 94) * * * Stable from 2% 9% 4,000 * * Increase 600 Baseline Reduction TGF-β1 3,000 MCP-1 500 Benefit Benefit pg/mL pg/mL (95% CI) 2,000 (95% CI) 400 * * 1,000 1

  11. Slide 11: Significantly Impacts Neuroprotection Biomarkers

    NurOwn Significantly Impacts Neuroprotection Biomarkers Galectin-1 VEGF-A NurOwn (N = 95) Neuroprotection Neuroprotection 52% Placebo 11,000 Increase (N = 94) * 10,000 9,000 13% Increase 8,000 Galectin-1 VEGF-A pg/mL 7,000 pg/mL Benefit (95% CI) (95% CI) 6,000 Benefit 5,000 2% 4,000 Increase 7% Redu

  12. Slide 12: Totality of Evidence Supports NurOwn MOA

    Totality of Evidence Supports NurOwn MOA Average Z-score = 2.24 NurOwn p < 0.0001 Placebo NfL MCP-1 7,000 600 6,000 500 Value pg/mL 5,000 400 (95% CI) * * 4,000 * 300 * * * * 3,000 200 0 2 4 8 12 16 20 Weeks 0 2 4 8 12 16 20 Weeks TGF-β1 Galectin-1 5,000 * * * 11,000 * 4,000 * * 9,000 Value 3,000 pg

  13. Slide 13: NurOwn Phase 3 Trial in ALS: Data Summary

    NurOwn Phase 3 Trial in ALS: Data Summary  Clinical results in overall population did not reach significance  Results in subgroups with less advanced disease show consistent treatment effect of NurOwn across endpoints and over time  Supportive results from biomarkers data  Data collected support

  14. Slide 14: Thank you

    Thank you

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