Brainstorm Cell Therapeutics Inc. · OTC:BCLI

Brainstorm Cell Therapeutics — 2021 ALS One Research Symposium

15 slides from Brainstorm Cell Therapeutics's conference presentation, Nov 30, 2021

  1. Slide 1: ALS Baseline Characteristics and Efficacy Outcomes in the NurOwn Phase 3 ALS Clinical Trial

    ALS Baseline Characteristics and Efficacy Outcomes in the NurOwn Phase 3 ALS Clinical Trial Cudkowicz M, Brown R, Windebank A, Goyal N, Berry J, Miller R, Burford M, Staff N, Katz J, Kern R, Gothelf Y, Lebovits C, Lindborg S 2021 ALS One Research Symposium November 30, 2021

  2. Slide 2: Disclosures

    Disclosures Study funded by Brainstorm Cell Therapeutics Inc The study was also supported by two grants: the California Institute for Regenerate Medicine and a grant from the ALS Association and I AM ALS Merit Cudkowicz: Received personal compensation consulting for Lilly Avexis, Pontifax, Orion, MT

  3. Slide 3: Responder Analysis, Improvement in Rate of Decline in ALSFRS-R

    Responder Analysis, Improvement in Rate of Decline in ALSFRS-R Primary Endpoint Analysis at Week 28, fails to reach statistical significance in overall study % Response by Treatment at week 28 % Response by Treatment at week 28 All Participants (n=189) Pre-specified subgroup, ALSFRS-R ≥35 (n=58) 40

  4. Slide 4: Phase 3 Trial of NurOwn in ALS Patients: Protocol

    Phase 3 Trial of NurOwn® in ALS Patients : Protocol Double-blind, Placebo-controlled, Randomized Trial Treatment Treatment Treatment Bone Marrow Aspiration NurOwn Treated (N=95) R • CSF 0, 2, 4, 8, 12, 16, and 20 weeks 1:1 • ALSFRS-R designed to be collected 13 times over study Placebo (N=94) Wk -18

  5. Slide 5: Phase 3 Trial of NurOwn in ALS Patients: Inclusion Criteria

    Phase 3 Trial of NurOwn® in ALS Patients : Inclusion Criteria Double-blind, Placebo-controlled, Randomized Trial Key Inclusion Criteria: • ALSFRS-R ≥ 25 at Screening Visit (Not at Baseline) • Decline in ALSFRS-R total score of 3 or more points in the 12 weeks before randomization* • Onset of ALS dis

  6. Slide 6: Phase 3 Trial of NurOwn in ALS Patients: Endpoints

    Phase 3 Trial of NurOwn® in ALS Patients : Endpoints Double-blind, Placebo-controlled, Randomized Trial Primary Endpoint Secondary Endpoints A responder analysis of the • Safety change in rate of decline as • ALSFRS-R change from baseline assessed by ALSFRS-R • Combined Analysis of Function and Surv

  7. Slide 7: Clinical Trials

    BCT-002 had a unique range of ALS Disease severity (severe ALS) Mean Baseline ALSFRS-R Score in Phase 2 & 3 Clinical Trials Clinical Trials 7 Mean ALSFRS-R Score

  8. Slide 8: ENCALS: Personalized Prognosis in ALS

    ENCALS: Personalized Prognosis in ALS Time to survival free of non-invasive ventilation  23 hours/day, tracheostomy or death • Rigorous model development, published 2019 in Lancet • 11,475 ALS patients from 14 European ALS centres across 9 countries participated. • Total follow-up was 40,016 years

  9. Slide 9: ENCALS model: NurOwn's Phase 3 trial enrolled a broad set of patients

    ENCALS model: NurOwn’s Phase 3 trial enrolled a broad set of patients NurOwn and Placebo treatment groups had patients across all 5 ENCALS categories ENCALS model prediction, Baseline distribution BCT-002 NurOwn Placebo Total prediction via ENCALS model Very short 8 (4.2%) 8 (4.2%) 16 (8.5%) Short 1

  10. Slide 10: Pre-Treatment Slope

    ENCALS categories and Baseline ALSFRS-R, Pre-treatment Slope Participants predicated to have the shortest survival have larger pre-treatment slopes and lower baseline ALSFRS-R scores Mean and 95% Confidence Interval, all participants Baseline ALSFRS-R Total Observations: • Participants predicted by

  11. Slide 11: Pre-Treatment Slope

    Relationship between ENCALS categories and Pre-treatment Slope There are more Placebo participants with larger pre-treatment slopes of those predicted by ENCALS to survive “Very Short” Pre-Treatment Slope Mean and 95% Confidence Interval, by treatment 11

  12. Slide 12: Phase 3, Primary Endpoint analyzed using ENCALS model

    Phase 3, Primary Endpoint analyzed using ENCALS model Inclusion of participants with severe ALS may have had a dilution effect, reducing the ability to show a treatment effect NurOwn participants Placebo participants Responder Responder Non-Responder Non-Responder Observations: • In participants pre

  13. Slide 13: Responder Analysis by Baseline ALSFRS-R ≥35 or <35

    Responder Analysis by Baseline ALSFRS-R ≥35 or < 35 Primary endpoint analyzed by baseline ALSFRS-R score, pre-specified subgroup 40% 34.60% 33.90% 35% 31.90% Percent Responders 30% p=0.288 p=0.744 25% 20% 15.60% 15% 10% 5% 0% Baseline ALSFRS-R≥35 Baseline ALSFRS-R <35 n=58 n=131 NurOwn Placebo Post-

  14. Slide 14: Conclusions

    Conclusions • BCT-002, NurOwn’s phase 3 trial included more participants (23%) with advanced ALS at baseline (ALSFRS-R at or below 25) compared to other trials • In participants that are predicted to have long to very long survival, there are more NurOwn responders versus Placebo (33% vs 14%) • The

  15. Slide 15: Manufacturing Sites, Treatment Centers, Funding

    Manufacturing Sites Confidential- not for external distribution City of Hope Center for Biomedicine & Dana-Farber Harvard Cancer Center Genetics Cell Manipulation Core Treatment Centers Funding 15

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