Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Investor Presentation (September 2018)
16 slides from Brainstorm Cell Therapeutics's investor presentation, September 2018
Slide 1: BrainStorm Cell Therapeutics
בס"ד BrainStorm Cell Ralph Kern MD MHSc. Therapeutics Chief Operating Officer and Chief Medical Officer September 2018 NASDAQ: BCLI March 22, 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: Successfully Leveraging Core Capabilities
בס"ד Successfully Leveraging Core Capabilities Manufacturing Excellence Strong Science • NurOwn Cryopreservation • Cell and Disease biology • Manufacturing Capabilities • Industry CNS experience Strategic Partnerships Patient Focus • California Institute for • Patient Advocacy Leadershi Regenerati
Slide 4: Addressing High Unmet Need Across Neurodegenerative Disorders
בס"ד Addressing High Unmet Need Across Neurodegenerative Disorders Amyotrop Parkins Progressiv Alzheimer’ Huntingt Autism hic on’s e Multiple s Disease on’s Spectrum Lateral Disease Sclerosis (AD) Disease Disorder (ASD) Sclerosis (PD) (MS) (HD) (ALS) Global patients 220,000 10,000,000 1,250,000 47
Slide 5: NurOwn Preclinical Studies
בס"ד Parkinson’s NurOwn® Disease- 6OHDA Preclinical model Studies Multiple Huntington’s Sclerosis- Disease-QA EAE model ALS- model SOD1 model Autism- Mouse BTBR model 5
Slide 6: Global Prevalence of ALS is Increasing (2015-2040)
בס"ד Global Prevalence of ALS is Increasing (2015-2040) Nature Communications, 2016.
Slide 7: Global Incidence of ALS is Increasing
בס"ד Global Incidence of ALS is Increasing (2015- 2040) Nature Communications, 2016.
Slide 8: NurOwn Manufacturing Process
בס"ד NurOwn® Manufacturing Process NurOwn® MSC derived No donor antigens from self Single bone marrow MSCs are Thawing MSC to injection aspirate yields cryopreserved 2-3 years of MSC-NTF 7 days dosing NurOwn® process is Manufacturing and shipping validated processes validated 8
Slide 9: NurOwn: Mesenchymal Stem Cells Secreting Neurotrophic Factors (MSC-NTF)
NurOwn®: Mesenchymal Stem Cells בס"ד Secreting Neurotrophic Factors (MSC- NTF) CD44 CD73 M SC Preserved MSC cell surface markers CD M SC-NTF markers Enhanced Neurotrophic T regulatory cells factor secretion (Tregs) VEGF CD3 CD14 CD19 Consistent Immuno- MSC modulation
Slide 10: NurOwn ALS Development
בס"ד NurOwn ALS ® Development • NurOwn® • ALS • ALS • ALS Early • Phase • US Phase 2017 • US Phase 2007 2010 2014 Developm + 1/2a 2 3 ent Repeat dosing Rapid progressors N=200 N=48 # of Study N=26 Participants Phase 1/2 Phase 2 Phase 3 10
Slide 11: NurOwn Phase 2 Study: Met Primary Safety Outcomes
בס"ד NurOwn Phase 2 Study: ® Met Primary Safety Outcomes Screening BMA Transplantation End of Study (V1) (V4) (V5)* (V10) -3-5 weeks 0 24-36 weeks -12-16 weeks CSF Collection (~ 3 months) (V5 and V6) (~ 9-10 months) Study Design • No deaths or treatment related SAEs Efficacy • N=48 participants (1
Slide 12: NurOwn ALS Phase 3 Study Schema
בס"ד NurOwn® ALS Phase 3 Study Schema Inclusion • ≤ 60 years MSC-NTF (n=100) Outcomes • SVC > 65% • ALSFRS-R responder • ALS ≤ 2 years Bone Marrow • Rapid Randomization analysis Aspiration • Safety progressors Placebo (n=100) • ALSFRS-R change from Exclusion baseline • Edaravone • SVC • Ventilatio
Slide 13: NurOwn ALS Phase 3 Study Sites
בס"ד NurOwn® ALS Phase 3 Study Sites Mayo Clinic (Rochester, MN) MGH (Boston, MA) California Pacific Medical Center, (San Francisco, CA) UMass (Worchester, MA) Cedar Sinai (Los Angeles, CA) UC Irvine (Los Angeles, CA) 13
Slide 14: NurOwn ALS Phase 3 Study Timeline
בס"ד NurOwn® ALS Phase 3 Study Timeline Study Status N=200 patients 1:1 randomization Enrollment completed by Mid-2019 * Supported by $16M CIRM CLIN-2 grant ClinicalTrials.gov Identifier: NCT03280056
Slide 15: NurOwn Strong IP Portfolio
בס"ד NurOwn® Strong IP Portfolio Allowed Granted Pending Expiry Patent Name/ Int. App. No. Jurisdictio Jurisdiction Jurisdictions Date ns s ISOLATED CELLS AND POPULATIONS COMPRISING SAME FOR THE TREATMENT US Europe, US 2030 OF CNS DISEASES/PCT/IL2006/000699 MESENCHYMAL STEM CELLS FOR THE Hong Kong
Slide 16: Thank You!
בס"ד BrainStorm Cell Therapeutics Prepared NASDAQ: BCLI Thank You! March 22, 2018
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