Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — 37th Congress of ECTRIMS 2021

14 slides from Brainstorm Cell Therapeutics's conference presentation, October 2021

  1. Slide 1: Multicenter Phase 2 Safety and Efficacy Study of MSC-NTF Cells (NurOwn) in Progressive MS

    Multicenter Phase 2 Safety and Efficacy Study of MSC-NTF Cells (NurOwn®) in Progressive Multiple Sclerosis J. Cohen, F. Lublin, C. Lock, D. Pelletier, T. Chitnis, M. Mehra, Y. Gothelf, R. Aricha, S. Lindborg, C. Lebovits, R. Kern 37th Congress of ECTRIMS October 2021

  2. Slide 2: Disclosures

    Disclosures Study funded by Brainstorm Cell Therapeutics Inc. Jeffrey Cohen: Received personal compensation consulting for Adamas, Atara, Bristol-Myers Squibb, Convelo, MedDay, and Mylan; Editor of Multiple Sclerosis Journal Fred Lublin: Consultant to and received research funding from Brainstorm Ce

  3. Slide 3: MSC-NTF Cells (NurOwn) Modulate Neuroinflammatory and Neuroprotective Pathways in Progressive MS

    MSC-NTF Cells (NurOwn®) Modulate Neuroinflammatory and Neuroprotective Pathways in Progressive MS NurOwn MOA Neuroinflammation↓ Osteopontin MCP-1 CHIT-1 SDF-1 sCD-27 Neuroprotection↑ VEGF HGF Fetuin-A NCAM-1 Follistatin Baecher-Allan et al, Neuron 2018

  4. Slide 4: NurOwn Progressive MS Phase 2 Trial Design

    NurOwn® Progressive MS Phase 2 Trial Design Primary Outcome Phase 2 study to evaluate the safety and efficacy of NurOwn® in Progressive MS Safety Secondary Outcomes 16-week treatment period, 12-week follow-up period, 8 weeks between treatments 6 weeks apart Biomarkers (CSF & Serum) Changes from base

  5. Slide 5: BCT-101 Demographics and Baseline Characteristics

    BCT-101 Demographics and Baseline Characteristics NurOwn Baseline Parameters (N=18) Mean Age: Years(SD) 47 (9.6) Females N(%) 10 ( 56%) EDSS Score: Mean(SD) 5.4 (1.3) T25FW Score (ft./sec.): Mean (SD) 2.4 (1.6) 9-HPT Score (sec.) – Combined Average: Mean(SD) 35.2 (15.7) 9-HPT Score (sec.) – Dominant

  6. Slide 6: CONSORT Diagram

    CONSORT DIAGRAM Assessed for eligibility (n=23) Screen failed (n=3) Bone marrow aspiration (n=20) Inadequate cell growth (n=2) Allocated to NurOwn(n=18) Received all 3 allocated interventions (n=17) Follow-up (n=18) Lost to follow-up (n=0) Analyzed observed cases (n=18) Discontinued from Study (n=2)

  7. Slide 7: Safety: SAEs, Frequently Occurring TEAEs

    Safety: SAEs, Frequently Occurring TEAEs Two subjects had SAEs of Arachnoiditis and one of these discontinued the study. AE Preferred Term # of Subjects (% out of 18) Headache 16 ( 88.9) Back pain 15 ( 83.3) Urinary tract infection 6 ( 33.3) Musculoskeletal pain 5 ( 27.8) Injection site pain 4 ( 22.

  8. Slide 8: CSF Neuroinflammatory Biomarkers Decreased

    CSF Neuroinflammatory Biomarkers Decreased Chitotriosidase-1 in CSF Over Time MCP-1 in CSF Over Time 7.0 9 CHIT-1 Log Concentration (mean ±95%CI) MCP-1 Log Concentration (mean ±95%CI) 6.5 8 6.0 7 5.5 6 Wk0 Wk8 Wk16 Wk0 Wk8 Wk16 Time (weeks) Time (weeks) Osteopontin in CSF Over Time SDF-1a in CSF Ove

  9. Slide 9: CSF Neuroprotection Biomarkers Increased

    CSF Neuroprotection Biomarkers Increased VEGF-A in CSF Over Time HGF in CSF Over Time 5.0 VEGF-A 6.5 HGF Log Concentration (mean ±95%CI) Log Concentration (mean ±95%CI) 4.5 6.0 4.0 5.5 3.5 3.0 5.0 Wk0 Wk8 Wk16 Wk0 Wk8 Wk16 Time (weeks) Time (weeks) Fetuin-A in CSF Over Time Follistatin in CSF Over T

  10. Slide 10: Demographics and Baseline Characteristics: NurOwn vs. CLIMB vs. SPRINT-MS

    Demographics and Baseline Characteristics: NurOwn vs. CLIMB1 vs. SPRINT-MS2 BCT-101 CLIMB Registry NN-102 (SPRINT-MS) Baseline Parameters NurOwn Observed Patients Ibudilast Placebo (N=18) (N=48) (N=121) (N=123) Mean Age: Years(SD) 47 (9.6) 55 (6.7) 55 (7.7) 57 (6.5) Females N(%) 10 ( 56 %) 32 (67 %)

  11. Slide 11: Responder Analysis, Over Weeks: NurOwn vs. CLIMB vs. SPRINT-MS

    Responder Analysis, Over 28 Weeks: NurOwn vs. CLIMB1 vs. SPRINT-MS2 CLIMB Registry NN-102 (SPRINT-MS) BCT-101 Observed Patients Ibudilast Placebo NurOwn (n=18) Outcome (n=48) (n=121) (n=123) T25W OR 9-HPT (≥25% improvement) 3/16 (19%) 2/47 (4%) 19/117 (16%) 13/121 (11%) T25W (≥25% improvement) 2/14

  12. Slide 12: Average Change from Baseline Over 28 Weeks, NurOwn vs. CLIMB vs. SPRINT-MS

    Average Change from Baseline Over 28 Weeks, NurOwn vs. CLIMB vs. SPRINT-MS BCT-101 CLIMB Registry NN-102 (SPRINT-MS) Outcome NurOwn (N=18) Observed Patients (N=48) Ibudilast (N= 121) Placebo (N=123) T25FW Speed (feet/seconds) 0.10 (n=14) -0.07 (n=43) 0.04 (n=114) -0.06 (n=118) 9-HPT Time, Combined A

  13. Slide 13: Conclusions

    Conclusions • Repeated intrathecal administration of MSC-NTF cells (NurOwn®) was generally safe and well-tolerated • CSF biomarkers at 3 consecutive time points ( 2 post-treatment) showed increases in neuroprotective biomarkers and decreases in neuroinflammatory biomarkers. • After 28-week follow-up

  14. Slide 14: Acknowledgments: Manufacturing, Treatment Centers, Funding, Data Access

    Manufacturing Dana-Farber Harvard Cancer Center Cell Manipulation Core Treatment Centers Funding Data Access CLIMB Registry Data: Tanuja Chitnis, MD 14 NN102 SPRINT-MS Study Data: NIH/NINDS. PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic. Original Publication

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