Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — 12th Annual California ALS Research Summit (January 2021)
17 slides from Brainstorm Cell Therapeutics's conference presentation, Jan 27, 2021
Slide 1: Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics Ralph Kern MD MHSc 12th Annual California ALS Research Summit January 27, 2021
Slide 2: NurOwn in ALS: 10+ Years Clinical Development
NurOwn® in ALS: 10+ Years Clinical Development ALS Clnical Studies 200 48 26 Phase 1/2 Phase 2 Phase 3 2007 2010 2014 2017 2019 2020 NurOwn ALS ALS ALS ALS ALS US Phase 3 Preclinical Phase 1/2a US Phase 2 US Phase 3 US Phase 3 Topline Data Development Open label Randomized trial Randomized trial Ful
Slide 3: BCT-002 had a unique range of ALS disease severity
BCT-002 had a unique range of ALS Disease severity (very late) Mean Baseline ALSFRS-R Score in Phase 2 & 3 Clinical Trials Clinical Trials 3 Mean ALSFRS-R Score
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 First Participant Screened: 28 August 2017. Database Lock: 30 October 2020 Designed to exclude slow progressors. Inclusion criteria specified: • ALSFRS-R ≥ 25 at Screening Visit (Not at Baseline).
Slide 5: Baseline Disease Characteristics (mITT Population)
Baseline Disease Characteristics (mITT Population) More NurOwn-treated participants had ALSFRS-R baseline scores <35 NurOwn Placebo Total N=95 N=94 N=189 Males, n (%) 68 (71.6) 59 (62.8) 127 (67.2) Pre-treatment slope, Mean (SD) -1.7 (0.75) -1.6 (0.82) Baseline ALSFRS-R, Mean (SD) 30.3 (6.5) 31.4 (6
Slide 6: Patient Safety- Overall Treatment Emergent AEs
Patient Safety- Overall Treatment Emergent AEs Safety Population NurOwn (N=95) Placebo (N=94) Participants (%) Participants (%) TEAEs 94 (98.9) 92 (97.9) TEAEs Related to Study Drug(1) 76 (80.0) 66 (70.2) Serious TEAEs 23 (24.2) 17 (18.1) Serious Related TEAEs 1 (1.1) 1 (1.1) (1) Treatment related T
Slide 7: Responder Analysis by Baseline ALSFRS-R <35 or ≥35
Responder Analysis by Baseline ALSFRS-R <35 or ≥35 Primary endpoint re-analyzed by baseline ALSFRS-R score, pre-specified subgroup Baseline ALSFRS-R ≥35 Primary endpoint: % Responders Percent Responders at Week 28: NurOwn 34.6% (9/26) Placebo 15.6% (5/32) p=0.288 Primary endpoint: Baseline ALSFRS-R
Slide 8: Baseline ALSFRS-R cut-points and impact on Primary Analysis
Primary Endpoint as pre-specified Baseline ALSFRS-R value by cut-points and their impact on the Primary Analysis NurOwn Placebo 40 35 30 25 % Response 20 15 10 5 0 ≥16 ≥17 ≥18 ≥19 ≥20 ≥21 ≥22 ≥23 ≥24 ≥25 ≥26 ≥27 ≥28 ≥29 ≥30 ≥31 ≥32 ≥33 ≥34 ≥35 % data: 100% 99% 98% 97% 96% 95% 93% 88% 86% 83% 77% 73%
Slide 9: ALS Phase 3 Secondary Endpoint by Baseline ALSFRS-R
ALS Phase 3 Secondary Endpoint by Baseline ALSFRS-R > 25 Consistent preservation of function across baseline thresholds ALSFRS-R MMRM Change from baseline to Week 28 Participants 0 included, n (%) NurOwn Placebo LS Mean Change from Baseline Delta by baseline: of total patients: LS mean (SE) LS mean
Slide 10: NurOwn MOA: Lessons learned from Phase 2, Replicated in Phase 3
NurOwn MOA: Lessons learned from Phase 2, Replicated in Phase 3 Neuroinflammation, Neurodegeneration, and Neuroprotection Biomarkers Underlying pathologies NurOwn MSC-NTF MOAs of ALS, pMS, and AD Neuroinflammation and Reduced Neuroinflammation Microglial Activation Reduces CSF MCP-1, SDF-1, S100b, N
Slide 11: Neuroinflammatory Biomarker Concentration in CSF Over Time
Neuroinflammatory Biomarker Concentration in CSF Over Time, LSMean (95%CI) NurOwn significantly lowers neuroinflammation biomarkers over time compared to placebo MCP-1: Statistically significant reduction with NurOwn SDF-1a: Statistically significant reduction with at each visit, and significant ove
Slide 12: Neurodegenerative Biomarker Concentration in CSF Over Time
Neurodegenerative Biomarker Concentration in CSF Over Time, LSMean (95%CI) NurOwn lowers neurodegenerative biomarkers over time compared to placebo NfL: NurOwn values lowered 82% compared to DR6: Statistically significant reduction with NurOwn, Placebo at week 20 significant DR6 overall treatment ef
Slide 13: Neuroprotective Biomarker Concentration in CSF Over Time
Neuroprotective Biomarker Concentration in CSF Over Time, LSMean (95%CI) NurOwn significantly elevates Neuroprotection biomarkers over time compared to placebo VEGF: Statistically significant increase with NurOwn at each BDNF: Statistically significant increase with visit, and significant overall tr
Slide 14: Statistical Analysis of Biomarker data through Principal Components
Statistical Analysis of Biomarker data through Principal Components NurOwn decreases Neuroinflammation and Neurodegeneration, and increases Neuroprotection Trend with individual biomarkers confirmed across biomarkers with PCA • Neuroinflammation decreased with NurOwn Neuroinflammation treatment over
Slide 15: Final Model identifies Biomarkers predictive of Primary Endpoint
Final Model identifies Biomarkers predictive of Primary Endpoint with high accuracy (82.5%) Statistically significant treatment effect on Primary Endpoint (p=0.003) ROC Curve for Selected Model Area Under the Curve = 0.8250 Stepwise Post baseline 1.00 logistic model ENCALS Model* ü MCP-1 ü, Δ 0.75 N
Slide 16: Update
Update Increased understanding of clinical Recent Updates outcomes related to baseline New biomarker analyses subgroups • Phase 3 biomarkers presented at • BCT-002 enrolled a more • Individual CSF biomarkers MND, Dec. 2021 by Dr Robert advanced ALS population consistently demonstrate Brown, UMass •
Slide 17: Manufacturing Sites
Manufacturing Sites City of Hope Center for Biomedicine & Dana-Farber Harvard Cancer Center Genetics Cell Manipulation Core Treatment Centers Funding 17
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