Brainstorm Cell Therapeutics Inc. · OTC:BCLI
Brainstorm Cell Therapeutics — Dawson James Securities 5th Annual Small Cap Growth Conference (October 2019)
18 slides from Brainstorm Cell Therapeutics's conference presentation, October 2019
Slide 1: BrainStorm Cell Therapeutics
בס"ד Dawson James Securities 5th Annual Small Cap Growth BrainStorm Cell Conference Therapeutics Prepared Preetam Shah, PhD, MBA NASDAQ: BCLI EVP and CFO BrainStorm Cell Therapeutics October 2019 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 - Potential Clinical Indications
בס"ד NurOwn® - Potential Clinical Indications 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 500
Slide 6: NurOwn Process
בס"ד NurOwn® Process 1 2 Isolation 3 Weeks 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 injected into th
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 (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 (V5 and V
Slide 9: ALSFRS-R mean change in slope
בס"ד ALSFRS-R slope improvement (mean pre-post LS mean slope change/month) All participants (n=46) Rapid progressors (n=21) Change from post-treatment to pre-treatment slope Change from post-treatment to pre-treatment slope 4 4 * ALSFRS-R mean change in slope ALSFRS-R mean change in slope 3 3 2 2
Slide 10: Responder Analysis: ≥1.5 point/month improvement ALSFRS-R Slope
בס"ד Phase 2 Responder Analysis: ≥1.5 point/month improvement ALSFRS-R Slope All participants (n=46) Rapid progressors (n=21) ALSFRS-R ≥1.5 points improvement/month ALSFRS-R 1.5 points improvement/month ALL Rapid progressors % of patients with ≥1.5 points change/month 90% % patients with ≥1.5 poin
Slide 11: NurOwn Phase 2 Increased CSF Neurotrophic Factors 2 Weeks Post-Treatment
בס"ד NurOwn® Phase 2 Increased CSF Neurotrophic Factors 2 Weeks Post-Treatment VEGF (1) HGF (1) (1) LIFLIF VEGF HGF Pg/ml 1000 * 600 18 ** *** 900 16 500 800 NurOwn® 14 700 400 12 600 (n = 26) pg/ml pg/ml pg/ml 10 500 300 8 400 200 6 300 200 100 4 Pre transplantation 100 2 0 0 0 Post transplantati
Slide 12: CSF Inflammatory biomarkers significantly reduced by NurOwn
בס"ד CSF Inflammatory biomarkers significantly reduced by NurOwn (2 weeks post treatment) CRP CRP MCP-1 MCP-1 SDF-1 SDF-1 MIP-1b MIP-1b 1600 50 250 10 *** ** 45 9 1400 40 200 8 1200 35 7 MSC-NTF 1000 30 150 6 pg/ml pg/ml pg/ml pg/ml 800 25 5 (n = 26) 600 20 15 100 4 3 400 10 50 2 200 5 1 0 0 0 0 P
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 conducted end of October 2019 – Trial to continue – No protocol changes • Top line data Q4, 2020 14
Slide 15: Phase 2 MS Clinical Trial Status
בס"ד Phase 2 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 • Top Line data mid 2020 15
Slide 16: Financial Highlights
בס"ד Financial Highlights $US in Thousands (except for Shares, Warrants and Options data) June 30, 2019 Cash and cash equivalents 1126 Short-term deposit 1575 Non-Dilutive Grants 3862* Total Liquidity 6563 Cap Structure Debt - Shares Outstanding (as of August 7, 2019) 22,550,442 Options / Warrants
Slide 17: 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 18: BrainStorm Cell Therapeutics - Thank You
בס"ד BrainStorm Cell Therapeutics NASDAQ: BCLI Thank You 18