17 slides from Brainstorm Cell Therapeutics's conference presentation, January 2023
Slide 1: Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics January 2023 I NASDAQ: BCLI Biotech Showcase
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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 implied by s
Slide 3: NurOwn: Autologous, consistent, reliable and convenient
NurOwn® Autologous, consistent, reliable and convenient Autologous and Convenient Consistent and Reliable Manufacturing Progress NurOwn® autologous cell therapy No animal proteins, antibiotics, Successful commercial technology uses the patient’s own cells genetic modifications or viral vectors trans
Slide 4: Brainstorm's Pipeline
Brainstorm’s Pipeline Indication Preclinical IND Enabling Phase 1 Phase 2 Phase 3 Milestones NurOwn® MSC-NTF Cells Platform Manuscript ALS completed Published Muscle & Nerve Dec 2021* Progressive MS completed Published in Multiple Sclerosis Journal Sept 2022** Alzheimer’s Disease Parkinson’s Disease
Slide 5: 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 Full
Slide 6: NurOwn Phase 3 results
NurOwn® Phase 3 results Advancing ALS treatment 6
Slide 7: Manufacturing Sites
Manufacturing Sites City of Hope Center for Biomedicine & Dana-Farber Harvard Cancer Center Genetics Cell Manipulation Core Treatment Centers Funding 7
Slide 8: Participants with baseline ALSFRS-R ≤25 and floor effect
Participants with NurOwn Phase 3 featured participants with advanced ALS disease baseline ALSFRS-R £ 25 are impacted by the resulting in a floor effect * floor of the scale ALSFRS-R % participants Edaravone J-19 Subscale with value of 0 Methycobalamin at baseline Lithium Bulbar 7% Proact Data Base E
Slide 9: NurOwn fails to reach statistical significance in primary endpoint in overall study
NurOwn fails to reach statistical significance in primary endpoint in overall study, suggests treatment effect in participants with less severe disease Primary Endpoint: % Response at week 28† Secondary Endpoint: Average Change from 40 p=0.453 p=0.305 baseline to Week 28, ALSFRS-R 35 32.6% 34.6% 0 -
Slide 10: Primary Endpoint as pre-specified: Percent Response by baseline cut-points
Primary Endpoint as pre-specified Baseline ALSFRS-R value by cut-points and their impact on the Primary Analysis Participants n (%) MSC-NTF placebo 40 included, by of total % NurOwn % Placebo ✱ ✱ ✱ baseline: participants: Response Response p-value# ≥35 n=58 (31%) 34.6% 15.6% 0.305 30 ≥34 n=68 (36%)
Slide 11: ALS Phase 3 Secondary Endpoint by Baseline ALSFRS-R >25
ALS Phase 3 Secondary Endpoint by Baseline ALSFRS-R > 25: NurOwn participants with less advanced disease had greater preservation of function compared to Placebo ALSFRS-R MMRM Change from Baseline to Week 28 Secondary Endpoint ALSFRS-R n (%) total MSC-NTF LS Placebo LS Delta p-value Baseline partici
Slide 12: Biomarker ALS Phase 3 study
Biomarker ALS Phase 3 study
Slide 13: 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, M
Slide 14: NurOwn Improves Biomarkers Across Multiple Pathways Over Time
NurOwn Improves Biomarkers Across Multiple Pathways Over Time, LS Mean (95%CI) MCP-1 (Pro-Inflammatory) LAP (Anti-inflammatory) LSMean (±95% CI) Change in MCP-1 LSMean (±95% CI) Change in LAP 6.5 5000 MCP-1 NurOwn® MCP-1 Placebo LAP NurOwn® LAP Placebo 6.4 4000 6.3 * * * * * 6.2 3000 6.1 6.0 * 2000
Slide 15: Biomarkers are not impacted by the floor effect of the ALSFRS-R
Biomarkers are not impacted by the floor effect of the ALSFRS-R MCP-1 LAP 6.6 B a s e lin e A L S F R S ≤ 2 5 B a s e lin e A L S F R S > 2 5 9.0 B a s e lin e A L S F R S ≤ 2 5 B a s e lin e A L S F R S > 2 5 6.4 8.5 * ** ** ** 6.2 * ** Estimate Estimate 8.0 6.0 * ** ** ** ** 7.5 5.8 * ** 5.6 * 7.0
Slide 16: Biomarkers are not impacted by the floor effect of the ALSFRS-R
Biomarkers are not impacted by the floor effect of the ALSFRS-R NfL VEGF-A 9.4 B a s e lin e A L S F R S ≤ 2 5 B a s e lin e A L S F R S > 2 5 4 Baseline ALSFRS ≤ 25 Baseline ALSFRS > 25 9.2 3 ** ** 9.0 * ** ** Estimate Estimate 8.8 ** ** ** 2 8.6 8.4 1 * 8.2 * 8.0 0 0 2 4 8 12 16 20 0 2 4 8 12 16 2
Slide 17: Thank you
Thank you 17