Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Investor Presentation (August 2020)
38 slides from Brainstorm Cell Therapeutics's investor presentation, August 2020
Slide 1: Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics August 2020 I NASDAQ: BCLI
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: Brainstorm At-a-Glance
Brainstorm At-a-Glance A leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases NASDAQ BCLI HEADQUARTERS New York, NY R&D CENTER Israel MANUFACTURING SITES Dana Farber Cancer Institute, City of Hope NUMBER OF EMPLOYEES 42 SHARES OUSTANDING 31
Slide 4: Brainstorm Senior Leadership Team
Brainstorm Senior Leadership Team Chaim Lebovits Ralph Kern, MD, MHSc Arturo Araya, MBA Chief Executive Officer President & Chief Medical Officer Chief Commercial Officer David Setboun, PharmD, MBA Preetam Shah, PhD, MBA Uri Yablonka EVP & Operating Officer EVP & Chief Financial Officer EVP & Chief
Slide 5: NurOwn: A unique cell therapy product
NurOwn® A unique cell therapy product Autologous and convenient Consistent and reliable Platform technology NurOwn® autologous cell therapy No animal proteins, antibiotics, Consistent biology observed in- uses the patient’s own cells genetic modifications or viral vitro and in-vivo • Safety and cell
Slide 6: Transforming the Treatment of ALS and Other Neurodegenerative Diseases
NurOwn® Transforming the Treatment of ALS and Other Neurodegenerative Diseases One Platform, Multiple Indications Flagship Program in ALS Diversified Clinical Pipeline NurOwn® autologous cell therapy Phase 3 fully enrolled Phase 2 progressive MS ongoing platform is broadly applicable • Top-line data
Slide 7: Brainstorm's Growing Pipeline
Brainstorm’s Growing Pipeline Indication Preclinical IND Enabling Phase 1 Phase 2 Phase 3 Next Milestones NurOwn® MSC-NTF Cells Platform Q4’20E ALS Top-line data Q4’20E Progressive MS Top-line data CTA: Q2’20E Alzheimer’s Disease Phase 2: 2H’20E Parkinson’s Disease Huntington’s Disease Autism Spectr
Slide 8: Technology Platform
NurOwn® Technology Platform Best-in-class autologous cell therapy 8
Slide 9: Manufacturing: Fast, Consistent and Reproducible
NurOwn® Manufacturing: Fast, Consistent and Reproducible 1. Harvesting: Outpatient procedure for collection of patient’s bone marrow sample 2. Isolation & Expansion: Mesenchymal stem cells (MSCs) isolated from the total bone marrow sample and MSCs expanded ex-vivo (12 days) 3. Cryopreservation: Crea
Slide 10: Effect on MSC-NTF Neurotrophic Factors
NurOwn® Effect on MSC-NTF Neurotrophic Factors n=28 ALS Patients GDNF BDNF BDNF (pg/106 cells) 5000 GDNF (pg/106 cells) 1800 1500 4000 1200 900 3000 600 300 2000 0 1000 -300 MSC MSC-NTF 0 MSC MSC-NTF VEGF HGF VEGF (ng/106 cells) 80 100 HGF (ng/106 cells) 60 80 60 40 40 20 20 0 0 MSC MSC-NTF MSC MSC-
Slide 11: Promotes Expansion of Anti-inflammatory Treg Cells
NurOwn® Promotes Expansion of Anti-inflammatory Treg Cells A 2.2 A 2.2 Post-transplant Post-transplant B 2.2 B 2.2 Post-transplant Post-transplant 2.0 2.0 2.0 2.0 1.8 1.8 1.8 1.8 1.6 1.6 1.6 1.6 %CD4+CD25+ lymphocytes %CD4+CD25+ lymphocytes %CD4+CD25+ lymphocytes %CD4+CD25+ lymphocytes 1.4 1.4 1.4 1
Slide 12: in vitro Induction of Regulatory T cells
NurOwn® in vitro Induction of Regulatory T cells PBMC co-cultured with PBMC only PBMC co-cultured with PBMC only PBMC only MSC-NTF PBMC co-cultured with MSC-NTF MSC-NTF T Regulatory Cells Increased In-vitro Representative FACS analysis of double positive FoxP3 and CD25 cells in a gate of CD4+cells s
Slide 13: MOA: Broadly Applicable Across Neurodegenerative Diseases
NurOwn® MOA: Broadly Applicable Across Neurodegenerative Diseases Underlying pathologies NurOwn MSC-NTF MOAs of ALS, MS, and AD Neuroprotection Neuronal Degeneration Reduces CSF Caspase-3 Astrocyte Dysfunction and Loss of Enhanced delivery of NTFs Neurotrophic Support Increases CSF VEGF, HGF, LIF, B
Slide 14: NurOwn for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
NurOwn® for the Treatment of Amyotrophic Lateral Sclerosis (ALS) 14
Slide 15: ALS: High Unmet Medical Need
ALS: High Unmet Medical Need Disease and Progressive neurological disorder with many symptoms including progressive muscle weakness, difficulty Symptoms breathing, and death. Severity of collective symptoms is measured via the ALSFRS-R functional score Life 3 - 5 years from diagnosis; median surviva
Slide 16: NurOwn in ALS: 10+ Years Clinical Development
NurOwn® in ALS: 10+ Years Clinical Development # of Study Participants 200 48 26 Phase 1/2 Phase 2 Phase 3 Q4 2020 ALS US Phase 3 Topline Data 2007 2010 2014 2017 2019 Readout NurOwn ALS ALS ALS ALS Preclinical Phase 1/2a US Phase 2 US Phase 3 US Phase 3 Development Open label Randomized trial Rando
Slide 17: Primary Endpoint ALS Functional Rating Scale - Revised (ALSFRS-R)
Primary Endpoint ALS Functional Rating Scale - Revised (ALSFRS-R) Validated and Regulatory Accepted Measure of ALS Function 48-point scale with 4 domains • Respiratory, fine motor, bulbar, gross motor Bulbar Function • 12 different items (scored 0-4) - rate of disease progression • Speech Respirator
Slide 18: NurOwn ALS Phase 2 Trial Design
NurOwn® ALS Phase 2 Trial Design Patients completing follow up Collect CSF, Collect Treatment CSF 43 Bone Marrow Aspiration MSC-NTF Treated (N=33) Primary Endpoint • CSF collection pre-treatment and 2 weeks post-treatment Safety & Tolerability RR • Follow up visits on week 4, 8, 16 N = 48 3: 3:1 • P
Slide 19: Phase 2 Outcomes: Greater Efficacy Observed in Rapid Progressors
Phase 2 Outcomes: Greater Efficacy Observed in Rapid Progressors Phase 3 has enrolled only rapidly progressing patients Single dose NurOwn® slowed the rate of disease progression by improving the ALSFRS-R LS mean change in slope vs. pre-treatment slope A Change from post-treatment to pre-treatment s
Slide 20: Phase 2 Responder Analysis: Improved Outcomes in Rapid Progressors
ALSFRS-R LS mean change i ALSFRS-R LS mean change 2 2 2 2 * * 1 1 Phase 2 Responder Analysis: Improved Outcomes in Rapid Progressors 1 1 0 0 Responder defined as ≥1.5 point/month ALSFRS-R 0 slope improvement 0 -1 -1 -1 -1 -2 -2 -2 -2 2wks2wks 4wks4wks8wks8wks12wks 12wks16wks 16wks24wks 24wks 2wks 4w
Slide 21: Phase 2 Biomarker Analysis
Phase 2 Biomarker Analysis CSF Profile 2 Weeks Post Treatment Demonstrates Delivery of NTFs VEGF HGF LIF 1000 VEGF VEGF VEGF VEGF VEGF 600 HGF HGF HGF HGF 18 LIF LIF LIF LIF 1000 10001000 1000 900 1000 600 600 600 600 600 ** 18 18 18 18 18 *** ****** *** 16 900 900 900 900 * 16 16 *** *** *** * ****
Slide 22: Phase 2 Results in ALS
Phase 2 Results in ALS MCP-1 (an inflammatory mediator) is Significantly Reduced 2 Weeks Post-Treatment MCP-1 SDF-1 50,000 CHIT-1 50 MCP-1 250 SDF-1 CHIT-1 *** ** 45,000 50,000 4550 250 * 4045 *** 200 ** 40,000 45,000 35,000 40,000 * 3540 200 NurOwn® 3035 150 MCP-1 Reduction (40%) 30,000 35,000 pg/m
Slide 23: Phase 2 CSF Biomarker Results in ALS
Phase 2 CSF Biomarker Results in ALS Post-Treatment CSF MCP-1 Shows Inverse Correlation with CSF VEGF and with ALSFRS-R slope improvement CSF MCP-1 inverse correlation with CSF VEGF CSF MCP-1 inverse correlation with ALSFRS-R Slope Improvement Berry et al Neurology 2019; Nagata Neurol Res 2007 23
Slide 24: NurOwn ALS Phase 3 Trial Design
NurOwn® ALS Phase 3 Trial Design Topline Data Expected Q4 2020 Phase 3 was designed based on safety, efficacy and durability data from Phase 2 Primary Endpoint Treatment Treatment Treatment A responder analysis of the rate of decline as assessed by Bone Marrow Screening MSC-NTF Treated (N=100) ALSFR
Slide 25: ALS Phase 3 Primary Endpoint Illustration – Responder Analysis
ALS Phase 3 Primary Endpoint Illustration – Responder Analysis Slope represents the rate of functional decline per month for each patient Patient 1 (NurOwn®) 39 Pre-treatment Post-treatment 37 Diff. in Post Pre-trt Post-trt Responder 35 Patient Treatment vs. Pre-trt Slope = 0.4 Pre-trt. Slope Slope
Slide 26: Phase 3 ALS Trial: Two Manufacturing Sites, Six Treatment Centers
Phase 3 ALS Trial: Two Manufacturing Sites, Six Treatment Centers Manufacturing Sites City of Hope Center for Biomedicine & Dana-Farber Harvard Cancer Center Genetics Cell Manipulation Core Treatment Centers 26
Slide 27: NurOwn for the Treatment of Progressive Multiple Sclerosis
NurOwn® for the Treatment of Progressive Multiple Sclerosis
Slide 28: Progressive MS: High Unmet Medical Need
Progressive MS: High Unmet Medical Need Progressive neurological disorder with the brain’s inability to control one’s body. Symptoms include pain, Disease and toxto electric shock sensations, muscle weakness, trouble walking, vision problems, numbness, dizziness, Symptoms balance etc. Life 5-10 year
Slide 29: NurOwn Progressive MS Phase 2 Trial Design
NurOwn® Progressive MS Phase 2 Trial Design Topline Data Expected Q4 2020 Phase 2 study to evaluate the safety and efficacy of NurOwn® in Progressive MS 16-week treatment period, 12-week follow-up period, Primary Outcome 8 weeks between treatments 6 weeks apart Safety Bone Marrow Collect CSF, Collec
Slide 30: NurOwn for the Treatment of Prodromal to Mild Alzheimer's Disease
NurOwn® for the Treatment of Prodromal to Mild Alzheimer’s Disease
Slide 31: Alzheimer's Disease: High Unmet Medical Need
Alzheimer’s Disease: High Unmet Medical Need Progressive central nervous system disorder. Symptoms include difficulty learning new information, mood Disease and toxto changes, severe disorientation, memory loss, behavior changes, and difficulty speaking, swallowing and Symptoms walking Life On avera
Slide 32: NurOwn-induced decrease in MCP-1 may address AD pathology
NurOwn® induced decrease in MCP-1 may address AD pathology NurOwn® treatment decreases CSF MCP-1, which increases with disease severity in AD patients CSF MCP-1 is increased in AD1 Increased CSF MCP-1 correlates with cognitive decline MCP-1 in CSF Annual Decrease in MMSE Score Annual Decrease in MMS
Slide 33: Delivery of miR-132 may address the pathology of AD
NurOwn® delivery of miR-132 may address the pathology of AD NurOwn MSC-NTF cells express and deliver miR-132, which is downregulated in AD miR-132-3p Expression Log2 Fold Change Normalized Relative FC = +4.06 of mir-132-3p miR-132 is miR-132 is Quantiles downregulated in upregulated in AD1 MSC-NTF c
Slide 34: Phase 2 AD trial enrollment will be driven by biomarkers
Phase 2 AD trial enrollment will be driven by biomarkers Biomarker driven approach facilitates patient selection and improves chances of trial success CSF amyloid and tau are linked to cognitive decline in AD Trial biomarkers are predictive of clinical decline CSF: Cerebrospinal fluid; MMSE: Mini-me
Slide 35: Rationale for NurOwn in Alzheimer's
Rationale for NurOwn® in Alzheimer’s • Targeting neuroinflammation offers an exciting and not yet fully evaluated therapeutic approach in Alzheimer’s disease • Robust effect on CSF biomarkers in Phase 2 ALS study ▪ Robust effect on MCP-1/CCL2 o MCP-1/CCL2 is associated with faster clinical progressi
Slide 36: Phase 2 AD Trial: Led by Two World Renowned Investigators
Phase 2 AD Trial: Led by Two World Renowned Investigators Principal Investigator French National Coordinator Philip Scheltens, MD, PhD (Principal Investigator) Bruno Dubois, MD, PhD Professor of Cognitive Neurology: Amsterdam UMC Professor of Neurology: Salpétrière University Hospital Director of th
Slide 37: BCT-201-EU, AD Phase 2a Trial Design
BCT-201-EU, AD Phase 2a Trial Design 52 week clinical trial 16-week treatment period, 26-week follow-up period, Primary Objective 8 weeks between treatments 12 & 14 weeks apart To evaluate safety and tolerability of 3 intrathecal Collect CSF, Collect CSF, In person administrations of Bone Marrow Col
Slide 38: Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics NASDAQ: BCLI www.brainstorm-cell.com Linkedin Twitter
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