Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Investor Presentation (February 2018)
21 slides from Brainstorm Cell Therapeutics's investor presentation, February 2018
Slide 1: BrainStorm Cell Therapeutics Company Update
בס"ד BrainStorm Cell Therapeutics NASDAQ: BCLI COMPANY UPDATE Chaim Lebovits, CEO February 2018
Slide 2: Forward Looking Statement
בס"ד Forward Looking Statement 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 implie
Slide 3: Company Overview
בס"ד Company Overview Business Description Summary Financials P&L • BrainStorm Cell Therapeutics (NASDAQ: BCLI) is a ($, 000') 2015 2016 Q3'17 LTM biotechnology company, which develops innovative adult stem Sales - - - cell therapeutic products for neurodegenerative disease R&D expenses (4,949) (2
Slide 4: NurOwn® Technology
brainstorm cell therapeutics NurOwn® Technology Bone Marrow containing self renewing stem cells Differentiation Isolation and expansion msc Differentiation MSC-NTF cells i (GDNF/BDNF secreting cells) Isolation v ALS and other Neuro-degenerative Diseases Expansion
Slide 5: NurOwn®: NTF and cytokine secretion compared to MSC cells of origin
בס"ד NurOwn®: NTF and cytokine secretion compared to MSC cells of origin Enhanced in-vitro neurotrophic-factor secretion differentiates NurOwn® from MSC cells of origin in ALS patients GDNF BDNF MSC Paired samples NurOwn® Individual participant Individual participant VEGF HGF Individual participan
Slide 6: NurOwn® successfully evaluated in preclinical models of neurodegenerative disease
בס"ד NurOwn® successfully evaluated Parkinson’s Disease in preclinical models of neurodegenerative disease Huntington’s Multiple Disease Sclerosis ALS Autism 6
Slide 7: NurOwn® ALS Development
בס"ד NurOwn® ALS Development NurOwn ALS ALS ALS 2007 Early 2010 Phase 1/2a 2014 US Phase 2 2017+ US Phase 3 Development 200 48 # of Study Participants 26 7 Phase 1/2 Phase 2 Phase 3
Slide 8: NurOwn® BCT-001-US Phase 2 Study Design
בס"ד NurOwn® BCT-001-US Phase 2 Study Design Pre-treatment ALSFRS-R slope Post-treatment slope (mean, responder analysis, excluding slow progressors) Screening BMA Transplantation End of Study (V1) (V4) (V5) (V10) -3-5 weeks 0 24-36 weeks -12-16 weeks CSF Collection (V5 and V6) ~ 9 - 10 months •48
Slide 9: Phase 2 ALS participant disposition and demographics
בס"ד Phase 2 ALS participant disposition and demographics: baseline characteristics balanced MSC-NTF Placebo All Participants Disposition (N=36) (N=12) (N=48) Analysis Populations n (%) n (%) n (%) Safety (ITT*) 36 (100.0) 12 (100.0) 48 (100.0) Study Completion Completed 33 (91.7) 10 (83.3) 43 (89
Slide 10: BCT-001-US ALS Clinical Trial: Key Endpoints
בס"ד NurOwn® BCT-001-US ALS Clinical Trial: Key Endpoints • Primary: – Safety and tolerability • Secondary: – ALSFRS-R – Slow vital capacity (SVC) • Exploratory: – Muscle strength (HHD) – CSF Biomarkers (added after the 1st 8 participants) 10
Slide 11: BCT-001-US ALS Clinical Trial: Treatment was Safe and Well-Tolerated
בס"ד NurOwn® BCT-001-US ALS Clinical Trial: Treatment was Safe and Well-Tolerated No deaths No treatment-related SAEs observed No dropouts related to AEs Most common adverse events (>15%; most mild/mod severity): Adverse Event NurOwn® (%) Placebo (%) Headache and Procedural Headache 80.6 6
Slide 12: ALSFRS-R Efficacy outcomes
בס"ד NurOwn® Phase 2 ALSFRS-R Efficacy outcomes: Mean change (pre-post) ALSFRS-R 1.5 point/month improvement- ‘reversal’ 100%+ improvement- ‘halt’ 12
Slide 13: Phase 2 ALSFRS-R outcomes: Mean ALSFRS-R slope improvement
בס"ד NurOwn® Phase 2 ALSFRS-R outcomes: Mean ALSFRS-R slope improvement All participants (n=46) Excluding Slow progressors (n=21) Pre-treatment Change from post-treatment to pre treatment slope Pre-treatment Change from Post-treatment to pre treatment slope 4 4 * 3 3 2 2 * LS means LS means 1 1 0
Slide 14: Responder Analysis: ≥1.5 points/month improvement
בס"ד NurOwn® Phase 2 Responder Analysis: ≥1.5 points/month improvement All participants (n=46) Excluding slow progressors (n=21) ALSFRS-R ≥1.5 points improvement/month in post- ALSFRS-R ≥1.5 points improvement excluding % of patients with ≥1.5 points change/month treatment slope compared to pre-tr
Slide 15: ≥1.5 point/month improvement - responder analysis
בס"ד ≥ 1.5 point/month improvement- responder analysis Group 12 weeks 12 weeks 16 weeks 16 weeks 24 weeks 24 weeks (n) (%) (n) (%) (n) (%) MSC-NTF 7/35 20 7/35 20 4/34 12 All Participants MSC-NTF 6/14 42 6/14 42 4/14 28 Fast Progressors Placebo 0/12 0 0/11 0 0/11 0 All Participants Placebo 0/6 0 0
Slide 16: Responder Analysis: >=100% improvement
בס"ד NurOwn® Phase 2 Responder Analysis: >=100% improvement All participants (n=46) Excluding slow progressors (n=21) ALSFRS-R ≥100% improvement in post-treatment ALSFRS-R ≥100%% improvement excluding slope compared to pre-treatment slope slow progressors 100% % of patients with 100% improvement 1
Slide 17: Phase 2 ALS Study: CSF NTFs Significantly Increased Post-Treatment
בס"ד NurOwn® Phase 2 ALS Study: CSF NTFs Significantly Increased 2 Weeks Post-Treatment Compared to Baseline VEGF VEGF VEGF HGF HGF HGF LIF LIF LIF 1000 1000 1000 600 600 600 18 18 18 * * * ** ** ** *** *** *** 900 900 900 16 16 16 500 500 500 800 800 800 14 14 14 700 700 700 400 400 400 12 12 12
Slide 18: Phase 2 ALS Study: CSF Inflammatory Markers Significantly Decreased
בס"ד NurOwn® Phase 2 ALS Study: CSF Inflammatory Markers Significantly Decreased Post-Treatment Pre transplantation CSF MCP-1, SDF- markers of neuroinflammation Post transplantation CRP CRPCRP CRP MCP-1 MCP-1 MCP-1 MCP-1 SDF-1 SDF-1 SDF-1 SDF-1 MIP-1b MIP-1b MIP-1b MIP-1b 1600 1600 1600 1600 50 50
Slide 19: NurOwn® Phase 3 ALS Study (BCT-002-US)
בס"ד NurOwn® Phase 3 ALS Study (BCT-002-US) • Randomized, double-blind, placebo-controlled, multi-dose trial • 200 patients; 1:1 randomization; 6 leading US ALS clinical sites • Actively recruiting and enrolling • Study design (total study duration ~11.5 months) – Pre-treatment: ~3 month baseline
Slide 20: Management Team
בס"ד Management Team Chaim Lebovits Yael Gothelf PhD. President and CEO VP, Scientific and Regulatory Affairs Uri Yablonka Ralph Kern, MD, MHSc. SVP, Chief Business Officer COO and CMO Eyal Rubin Yossef Levy PhD. EVP, CFO VP, Cell Production Mary Kay Turner VP, Patient Advocacy and Revital Aricha
Slide 21: BrainStorm Cell Therapeutics Thank You
בס"ד BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You
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