Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Investor Presentation (January 2020)
17 slides from Brainstorm Cell Therapeutics's investor presentation, January 2020
Slide 1: BrainStorm Cell Therapeutics
בס"ד BrainStorm Cell Therapeutics Prepared NASDAQ: BCLI January 2020 March 22, 2018 1
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: Brainstorm at a Glance
בס"ד Brainstorm at a Glance CLINICAL STAGE ADVANCED STAGE CLINICAL LARGE ADDRESSABLE BIOTECHNOLOGY COMPANY PROGRAMS MARKETS Potential to be first-in-class ALS autologous stem cell Repeat Dose Phase 3 ALS trial 30,000 patients (US) therapy for ALS and other fully enrolled 450,000 (Worldwide) neurod
Slide 4: Brainstorm's Pipeline
בס"ד Brainstorm’s Pipeline ALS – Amyotrophic Lateral Sclerosis (Lou Gehrig’s Disease) Preclinical Phase 1 Phase 2 Phase 3 Progressive MS – Multiple Sclerosis Preclinical Phase 1 Phase 2 Phase Phase 33 Parkinson’s Disease Preclinical Phase 1 Phase 2 Phase 3 Autism Spectrum Disorder Preclinical Phas
Slide 5: NurOwn® - A Platform Technology Across CNS
בס"ד NurOwn® - A Platform Technology Across CNS Parkinson’s Disease Global patients 10,000,000 US patients 1,000,000 Progressive Huntington’s ALS Multiple Disease Sclerosis Global patients 300,000 Global patients 450,000 Global patients 1,250,000 US patients 30,000 US patients 30,000 US patients 5
Slide 6: NurOwn® Technology Process
בס"ד NurOwn® Technology Process 1 2 Isolation 12 Days Manufacturing process begins with a bone marrow aspiration to harvest Expansion Intermediate MSC product is cryopreserved adult MSCs from the patient Harvesting Cryopreservation Transplantation The proprietary culture medium The cells are injec
Slide 7: NurOwn® ALS Development
בס"ד NurOwn® ALS Development 2H 2020 ALS US Phase 3 Topline Data Readout 2017 2019 ALS ALS US Phase 3 US Phase 3 2014 Randomized trial Fully Enrolled ALS US Phase 2 2010 Randomized trial 2007 ALS Phase 1/2a NurOwn Open label Preclinical Development 200 26 48 # of Study Participants Phase 1/2 Phase
Slide 8: NurOwn® ALS Phase 2 Double-Blind Placebo-Controlled Trial
בס"ד NurOwn® ALS Phase 2 Double-Blind Placebo- Controlled Trial 48 participants (16 per site); Randomized 3:1 -12 to -16 weeks Screening Safety (V1) ~3-4 months 3 month run in period BMA -3 to -5 weeks (V4) ••No deaths or treatment CSF Week 0 Transplantation (V5)* related SAEs Collection Week 2 (V
Slide 9: ALSFRS-R Efficacy Outcomes
בס"ד ALSFRS-R Efficacy Outcomes All n=46 Rapid Progressors n=21 A Change from post-treatment to pre-treatment slope B Change from post-treatment to pre-treatment slope 4 4 ALSFRS-R LS mean change in slope * 3 3 2 2 * Mean ALSFRS-R 1 1 Post-pre slope change 0 0 MSC-NTF -1 -1 Placebo -2 -2 2wks 4wks
Slide 10: Phase 2 CSF Biomarker Analyses
בס"ד Phase 2 CSF Biomarker Analyses 10
Slide 11: CSF Neurotrophic Factors Increased Post-Treatment
בס"ד CSF Neurotrophic Factors Increased Post-Treatment VEGF HGF LIF 1000 600 18 900 ** *** * 500 16 800 14 700 400 12 pg/ml 600 NurOwn® 10 500 300 8 400 (n = 26) 300 200 6 4 200 100 V5 -Pre transplantation 100 2 0 0 0 V5 V6 V5 V6 V5 V6 V6 - Post transplantation 1000 600 18 900 16 500 800 14 700 Pl
Slide 12: CSF Inflammatory Biomarkers Reduced Post-Treatment
בס"ד CSF Inflammatory Biomarkers Reduced Post-Treatment MCP-1 SDF-1 50,000 CHIT-1 50 250 *** ** 45,000 45 40,000 * 40 200 35 35,000 NurOwn® 30 150 30,000 pg/ml 25 25,000 (n = 26) 20 100 20,000 15 15,000 10 50 10,000 V5 -Pre transplantation 5 5,000 0 0 0 V6 - Post transplantation V5 V6 V5 V6 V5 V6
Slide 13: NurOwn® ALS Phase 3 Study Design
בס"ד NurOwn® ALS Phase 3 Study Inclusion Outcomes Bone Marrow Aspiration • ≤ 60 years • ALSFRS-R responder MSC-NTF analysis Randomization • SVC > 65% (n=100) • Safety • ALS ≤ 2 years • ALSFRS-R change from • Rapid progressors baseline Exclusion Placebo • SVC (n=100) • Tracheostomy-free survival •
Slide 14: Phase 3 ALS Status
בס"ד Phase 3 ALS Status • Phase 3 enrollment completed in October 2019 • DSMB safety review October 2019 – Trial to continue – No protocol changes • Top line data Q4, 2020 14
Slide 15: Phase 2 Progressive MS Clinical Trial Status
בס"ד Phase 2 Progressive MS Clinical Trial Status • IND granted December 2018 • Repeat intrathecal dosing MSC- NTF cells every 2 months • Validated clinical endpoints and biomarkers • 5 clinical trial sites in US • First subjects enrolled March 2019 • DSMB first review completed Dec 2019 • Top Lin
Slide 16: Brainstorm Senior Leadership Team
בס"ד Brainstorm Senior Leadership Team Ralph Kern, Chaim Lebovits, Arturo Araya, Preetam Shah, MD, MHSc Uri Yablonka President & MBA PhD, MBA Chief Operating EVP & Chief Chief Executive Chief Commercial EVP & Chief Officer & Chief Business Officer Officer Officer Financial Officer Medical Officer
Slide 17: BrainStorm Cell Therapeutics
בס"ד BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You 17
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