Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (November 2014)
53 slides from Lisata Therapeutics's investor presentation, Nov 18, 2014
Slide 1: Investor Presentation November 2014: Transforming Medicine
INVESTOR PRESENTATION NASDAQ: NBS NOVEMBER 2014 TRANSFORMING MEDICINE
Slide 2: Forward-Looking Statements
NASDAQ:NBS | www.neostem.com > 1 FORWARD-LOOKING STATEMENTS This presentation contains “forward-looking” statements within the meaning of the Private Securities Litigation Reform Act of 1995, as well as historical information. Such forward-looking statements involve known and unknown risks, uncertai
Slide 3: NeoStem Company Overview
NASDAQ:NBS | www.neostem.com > 2 NEOSTEM COMPANY OVERVIEW Integrated biotechnology company with a strong pipeline based on multiple platform technologies, that includes Phase 2 and 3 assets, and a revenue- generating contract development and manufacturing service business Headquarters in New Yor
Slide 4: Our Value Proposition
OUR VALUE PROPOSITION NASDAQ:NBS | www.neostem.com > 3 A LATE STAGE CLINICAL PIPELINE AND A REVENUE-GENERATING SERVICE BUSINESS IN CELL THERAPY TARGET INDICATIONS INCLUDE: ■ Stage IV and recurrent Stage III melanoma ■ Acute myocardial infarction ■ Type 1 diabetes ■ Steroid resistant asthma
Slide 5: Management Highlights
Robert Dickey IV Chief Financial Officer Former investment banker (Lehman Brothers) Former CFO at StemCyte, a stem cell company Douglas W. Losordo, MD Chief Medical Officer Leader in cell therapy research and renowned cardiologist (Baxter, Northwestern University) Robert A. Preti, PhD Chief Sc
Slide 6: Development Highlights: Multiple Platform Technologies
DEVELOPMENT HIGHLIGHTS: MULTIPLE PLATFORM TECHNOLOGIES NASDAQ:NBS | www.neostem.com > 5 A PORTFOLIO OF CELL THERAPY PRODUCTS IN DEVELOPMENT THAT LEVERAGE THE BODY’S NATURAL ABILITY TO HEAL AND FIGHT DISEASE Using VSEL™ Technology and Stem Cell Derived Growth Factors Using DC/TC Technology Usin
Slide 7: Targeted Cancer Immunotherapy Program Rationale
TARGETED CANCER IMMUNOTHERAPY PROGRAM RATIONALE NASDAQ:NBS | www.neostem.com > 6 Once isolated from patient’s tumor, cancer initiating cells provide potent signature antigens to educate and direct the immune system Our immunotherapy program uniquely targets the patient’s cancer initiating cells
Slide 8: Basics of Melanoma
NASDAQ:NBS | www.neostem.com > 7 BASICS OF MELANOMA Most lethal form of skin cancer Most often caused by unrepaired DNA damage to skin cells from UV radiation 76,100 estimated new cases per year in U.S.1 Kills an estimated 9,710 in U.S. annually1 SURVIVAL RATE Stage IV metastatic melanoma
Slide 9: Melanoma: Standard of Care Suboptimal
NASDAQ:NBS | www.neostem.com > 8 THERAPY 2 YR OVERALL SURVIVAL SIDE EFFECTS ESTIMATED COST Proleukin (Interleukin-2) Prometheus Labs 25%1 Capillary Leak Syndrome Impaired Neutrophil Function Disseminated Infection Sepsis >$100,000 Yervoy (Ipilimumab) (CTLA-4 inhibitor) Bristol Myers – Squibb 28%2 En
Slide 10: Targeted Cancer Immunotherapy Treatment Process
NASDAQ:NBS | www.neostem.com > 9 STEP 1: Treatment begins with the surgical resection of the patient’s tumor STEP 2: (DAY 0 - WEEK 6) The cancer initiating (stem) cells from the tumor are isolated, expanded, and irradiated to render them inactive STEP 3: (PRIOR TO WEEK 6) Patient undergoes leukapher
Slide 11: Phase 2 Results for Eltrapuldencel-T for Metastatic Melanoma
NASDAQ:NBS | www.neostem.com > 10 Dillman, et al. Journal Immunotherapy 2012 31% 72% 0% 20% 40% 60% 80% Control Group Irradiated Tumor Cells Only Eltrapuldencel-T Treatment Group 2 YEAR OVERALL SURVIVAL (18 patients) TRIAL DESIGN: ■ Treatment group: Eltrapuldencel-T (autologous dendritic cells pulse
Slide 12: Features and Intended Effects of Targeted Cancer Immunotherapy Program
FEATURES AND INTENDED EFFECTS OF TARGETED CANCER IMMUNOTHERAPY PROGRAM NASDAQ:NBS | www.neostem.com > 11 FEATURES: INTENDED EFFECTS: Designed to present the entire spectrum of patient-specific antigens that are expressed on cancer initiating (stem) cells for the immune system to target Designed to a
Slide 13: Melanoma Scientific Advisory Board
MELANOMA SCIENTIFIC ADVISORY BOARD Robert Dillman, MD SAB Administrative Co-Chairman Vice President, Oncology, NeoStem Andrew L. Pecora, MD SAB Administrative Co-Chairman Chief Visionary Officer, NeoStem Hackensack University Medical Center Michael B. Atkins, MD Georgetown-Lombardi Comprehensive Can
Slide 14: INTUS Phase 3 Special Protocol Assessment Study Design
INTUS PHASE 3 SPECIAL PROTOCOL ASSESSMENT (SPA) STUDY DESIGN NASDAQ:NBS | www.neostem.com > 13 STUDY NAME TARGET Patients with Stage IV or recurrent Stage III metastatic melanoma LOCATION United States and potentially Australia & New Zealand, approximately 50 sites DESIGN Double blind, placebo contr
Slide 15: Ischemic Repair Program Rationale
ISCHEMIC REPAIR PROGRAM RATIONALE: TO ENHANCE THE BODY’S NATURAL REPAIR MECHANISM ■ Ischemia occurs when the supply of oxygenated blood is restricted ■ Program seeks to reverse this restriction through development and formation of new blood vessels ■ CD34/CXCR4 expressing cells have been shown to be
Slide 16: First Target Indication: STEMI
FIRST TARGET INDICATION: STEMI ■ Following a heart attack, apoptosis and progressive cardiomyocyte loss leads to infarct expansion ■ ST segment Elevation MI (STEMI) patients are at a high risk of a progressive deterioration in heart muscle function that leads to worsening of cardiac output, morbidit
Slide 17: STEMI: Standard of Care
STEMI: STANDARD OF CARE INVASIVE, ASSOCIATED MORBIDITY & MORTALITY NASDAQ:NBS | www.neostem.com > 16 ■ Emergency care: ►Administration of antithrombotic therapy, aspirin, beta-blocker, nitroglycerin, and/or morphine ► Percutaneous coronary intervention - coronary angioplasty and stenting 1. AHA 2013
Slide 18: NBS10 Treatment Process
NBS10 TREATMENT PROCESS* NASDAQ:NBS | www.neostem.com > 17 DAY 1: Patient comes to emergency room with heart attack and receives stent DAY 4: Cardiac magnetic resonance to assess ventricular function DAY 4-9: Mini bone marrow procedure to harvest cells DAY 5-9: 6-8 hour cell separation process to is
Slide 19: PreSERVE Phase 2 Study: Initial Data Released
PRESERVE PHASE 2 STUDY: INITIAL DATA RELEASED NASDAQ:NBS | www.neostem.com > 18 TARGET Post-AMI patients KEY INCLUSION CRITERIA Confirmation of ST Elevation MI (STEMI); ejection fraction < 48% at day 4 by CMR; state of the art care post stenting LOCATION AND NUMBER OF SUBJECTS United States, 60 cent
Slide 20: Initial Phase 2 Results: Mortality Benefit and Dose-Dependent Relationship
NASDAQ:NBS | www.neostem.com > 19 INITIAL PHASE 2 RESULTS: 1 YEAR MORTALITY BENEFIT & DOSE-DEPENDENT RELATIONSHIP WITH DECREASE IN SERIOUS ADVERSE EVENTS & IMPROVEMENT IN EJECTION FRACTION ■ Results provide preliminary evidence that intracoronary administration of autologous CD34+ cells (NBS10) is:
Slide 21: Phase 2 Results: Mortality and Overall MACE
NASDAQ:NBS | www.neostem.com > 20 PHASE 2 RESULTS: MORTALITY AND OVERALL MACE MACE= DEATH, MI, CHF HOSPITALIZATION, REVASCULARIZATION Infusion through last follow-up visit Subjects with MACE through 6 month follow up of last patient; P-value reflects a z-test **P- value reflects a z-test of the null
Slide 22: CD34 Cell Dose-Dependent Reduction in MACE Incidence
NASDAQ:NBS | www.neostem.com > 21 CD34 CELL DOSE-DEPENDENT REDUCTION IN MACE INCIDENCE PHASE 2 RESULTS 14% 17% 10% 7% CD34 CELL DOSE-DEPENDENT INCREASE IN LVEF CHANGE FROM BASELINE 4.90% 3.10% 5.80% 10.20% 0% 10% 20% (N=83) (N=47) (N=31) (N=15) 1.0 1.1 0.4 0.3 0.0 0.2 0.4 0.6 0.8 1.0 1.2 Serious Adv
Slide 23: Migratory Capacity of Administered CD34 Cells and Event-Free Survival Post AMI
MIGRATORY CAPACITY OF ADMINISTERED CD34 CELLS ASSOCIATED WITH EVENT- FREE SURVIVAL POST AMI NASDAQ:NBS | www.neostem.com > 22 Assmus, B., et al. (2014) Long-term clinical outcome after intracoronary application of bone marrow-derived mononuclear cells for acute myocardial infarction: migratory capac
Slide 24: Features and Intended Effects of NBS10
NASDAQ:NBS | www.neostem.com > 23 FEATURES: INTENDED EFFECTS: CD34/CXCR4 cells are designed to target viable tissue surrounding the infarcted myocardium (peri-infarct zone) after administration and persist Mobile cells migrate to targeted tissues Autologous cells take up residence in the peri-infarc
Slide 25: Immune Modulation Program Rationale
IMMUNE MODULATION PROGRAM RATIONALE NASDAQ:NBS | www.neostem.com > 24 TREG THERAPY REPRESENTS A NOVEL APPROACH FOR RESTORING IMMUNE BALANCE BY ENHANCING T REGULATORY CELL NUMBER AND FUNCTION1 1. Chai, Jian-Guo et al, Journal of Immunology 2008; 180;858-869
Slide 26: First Target Indication: Diabetes Mellitus Type-1
FIRST TARGET INDICATION: DIABETES MELLITUS TYPE-1 (T1D) NASDAQ:NBS | www.neostem.com > 25 Autoimmune destruction of insulin- producing (beta cells) of the pancreas Diabetes is leading cause of kidney failure, new cases of adult blindness, and non- traumatic lower-limb amputations Results in to
Slide 27: T1D: Standard of Care
T1D: STANDARD OF CARE LIFETIME INSULIN DEPENDENCY, COMORBIDITIES NASDAQ:NBS | www.neostem.com > 26 There is no treatment for T1D – only lifelong insulin therapy to help avoid complications 2 or more injections daily $2 billion estimated market size for insulin sales in 2017 for T1D alone1 Co
Slide 28: T1D Treg Treatment Process
T1D TREG TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 27 DAY 1: Screening and enrollment DAY 5-7: Blood draw from patient DAY 7-21: Manufacturing including expansion DAY 21: Infusion of Treg therapy to patient
Slide 29: Regulatory T Cells Preserve Beta Cell Function in Children with T1D
■ First human evidence of therapeutic effect of autologous Treg therapy protection of pancreatic function in new onset T1D in children ■ One year follow-up: evidence that Treg therapy preserves function of pancreatic islets cells ►C-peptide levels stabilized ►Reduction of insulin requirements ■ 20%
Slide 30: Regulatory T Cells Appear Safe in Adults with Established T1D
ADMINISTRATION OF REGULATORY T CELLS* APPEARS TO BE SAFE IN ADULTS WITH ESTABLISHED T1D NASDAQ:NBS | www.neostem.com > 29 Gitelman et al, American Diabetes Association Abstract, 2014 Preliminary data indicates safety and tolerability Infused Tregs detected in peripheral circulation for over 6 mo
Slide 31: Features and Intended Effects of Immune Modulation Program
FEATURES: INTENDED EFFECTS Tregs are natural part of immune system Potential for positive safety profile Tregs shown in pre-clinical studies to be important in modulating autoimmune disorders and allergic conditions Platform may be applicable to steroid resistant asthma, rheumatoid arthritis, lupus,
Slide 32: Tissue Regeneration Programs
TISSUE REGENERATION PROGRAMS NASDAQ:NBS | www.neostem.com > 31 VSEL™ TECHNOLOGY: POTENTIAL TO REPAIR DAMAGED TISSUE ■ Evaluating therapeutic potential of very small embryonic- like stem cells (VSELs™) ■ Research suggests multipotency and multi-lineage differentiation into all basic cell types (mesod
Slide 33: Intellectual Property
NASDAQ:NBS | www.neostem.com > 32 INTELLECTUAL PROPERTY TARGETED CANCER IMMUNOTHERAPY PROGRAM ■ 5 issued patents and 35 pending patents in the U.S. and OUS with coverage including: ► Stem cell growth medium and methods of making and using same; Antigen-presenting cancer vaccines; Individualized high
Slide 34: PCT Provides Outsourced Manufacturing Capabilities to Cell Therapy Industry
MOUNTAIN VIEW, CALIFORNIA (25,000 ft2) ISO Class 7 / Class 10,000 suites Recent expansion of clean room space ALLENDALE, NEW JERSEY (30,000 ft2) ISO Class 7 / Class 10,000 suites ISO Class 6 / Class 1,000 suite Recent expansion of clean room space NASDAQ:NBS | www.neostem.com > 33 ■ High quality man
Slide 35: Financial Metrics and Market Metrics
FINANCIAL METRICS NASDAQ:NBS | www.neostem.com > 34 MARKET METRICS MARKET CAPITALIZATION1 $186M STOCK PRICE2 $5.26 52 WEEK RANGE2 $4.56 - $8.29 FLOAT1 31.2M INSIDER HOLDINGS1 11.9% FINANCIAL METRICS REVENUE3 $4.1M (Third Quarter) CASH4 $32.8M COMMON SHARES OUTSTANDING1 35.4M WARRANTS1 3.6M (avg. war
Slide 36: Future Growth Drivers
FUTURE GROWTH DRIVERS NASDAQ:NBS | www.neostem.com > 35 DEVELOP NOVEL PROPRIETARY CELL THERAPY PRODUCTS ■ Leverage unique capabilities for cost effective in-house product development ■ Partner select programs at key inflection points ■ Grow pipeline and capabilities through strategic acquisition EXP
Slide 37: Contact Information
CONTACT INFORMATION NASDAQ:NBS | www.neostem.com > 36 NEOSTEM, INC. NASDAQ: NBS WWW.NEOSTEM.COM ROBIN SMITH, MD, MBA CHAIRMAN & CEO PHONE: (212) 584-4174 EMAIL: RSMITH@NEOSTEM.COM
Slide 38: Appendix
NASDAQ:NBS | www.neostem.com > 37 APPENDIX
Slide 39: Board of Directors
Robin Smith, MD, MBA Chairman of the Board MD – Yale; MBA – The Wharton School Formerly President & CEO IP2M, EVP & CMO HealthHelp Experience - Trustee of NYU Medical Center; Chairman of the Board of NYU Hospital for Joint Diseases (through November 2009) and Chairman of Stem for Life Foundati
Slide 40: Examples of Contract Services Potential from Conception to Commercialization
NASDAQ:NBS | www.neostem.com > 39 EXAMPLES OF CONTRACT SERVICES POTENTIAL FROM CONCEPTION TO COMMERCIALIZATION* *Based on industry experience and estimated potential future commercial manufacturing in the industry LOW COMPLEXITY PRODUCT MEDIUM COMPLEXITY PRODUCT HIGH COMPLEXITY PRODUCT PRECLINICAL D
Slide 41: Since 2006, Accessed Over $193M and Completed Multiple M&A Transactions
SINCE 2006, ACCESSED OVER $193M AND COMPLETED MULTIPLE M&A TRANSACTIONS AND ONE DIVESTITURE NASDAQ:NBS | www.neostem.com > 40
Slide 42: Cardiovascular Scientific Advisory Board
CARDIOVASCULAR SCIENTIFIC ADVISORY BOARD Douglas W. Losordo, MD, FACC, FAHA SAB Administrative Chairman Chief Medical Officer, NeoStem Eugene Braunwald, MD, FRCP Brigham & Women’s Hospital Bernard J. Gersh, MD, ChB, DPhil, FRCP The Mayo Clinic Dean J. Kereiakes, MD, FACC The Christ Hospital Heart of
Slide 43: Immune Modulation Program Advisors
IMMUNE MODULATION PROGRAM ADVISORS NASDAQ:NBS | www.neostem.com > 42 The Company accesses these experts to advise in the areas of diabetes, asthma, and other autoimmune conditions for its Immune Modulation Program. Jeffrey Bluestone, PhD University of California, San Francisco, Diabetes Center Willi
Slide 44: VSEL Technology Academic Collaborators
VSEL™ TECHNOLOGY ACADEMIC COLLABORATORS NASDAQ:NBS | www.neostem.com > 43 Mariusz Ratajczak, MD, PhD, Dsci University of Louisville Russell Taichman, DMD, DMSc University of Michigan Vincent Falanga, MD Boston University Michael Young, PhD Schepens Eye Research Institute, Harvard Medical School Kame
Slide 45: Treg Immunotherapy Works in Model of T1D
NASDAQ:NBS | www.neostem.com > 44 TREG IMMUNOTHERAPY WORKS IN MODEL OF T1D Tregs effectively suppress diabetes 0 10 20 30 40 50 60 70 0 5 10 15 20 Weeks after treatment R emi s s io n ( % ) untreated Ag-specific Treg therapy Ag-specific Tregs reverse diabetes Tang, Bluestone, et al.
Slide 46: T1D Is on the Rise
T1D IS ON THE RISE NASDAQ:NBS | www.neostem.com > 45 NEW CASES OF T1D (0-14 YEARS) PER 100,000 CHILDREN, 2013: CONCENTRATION IN DEVELOPED MARKETS Dabelea, Lancet, 2009
Slide 47: Economic Impact of T1D
ECONOMIC IMPACT OF T1D NASDAQ:NBS | www.neostem.com > 46 THE ECONOMIC BURDEN OF T1D IN THE U.S. IS ESTIMATED AT $14.9 BILLION1 Average economic burden per person with diabetes is larger for T1D vs T2D PREVENTION IS KEY - MEDICAL COSTS ASSOCIATED WITH T1D INCREASE SUBSTANTIALLY WITH AGE AND DURATIO
Slide 48: Human VSELs Accelerate Healing in a SCID Mouse Complex Tail Wound Model
HUMAN VSELS™ ACCELERATE HEALING IN A SCID MOUSE COMPLEX TAIL WOUND MODEL NASDAQ:NBS | www.neostem.com > 47 PRELIMINARY DATA IN A PRECLINICAL MODEL OF SEVERE COMPLEX WOUNDS SUGGEST THAT VSELS™ MAY BE MORE EFFECTIVE IN ACCELERATING HEALING THAN MESENCHYMAL STROMAL CELLS (MSCs) FIBRIN CONTROL (DAY 7) F
Slide 49: VSELs Could Be Used to Treat Macular Degeneration
VSELS™ COULD BE USED TO TREAT MACULAR DEGENERATION NASDAQ:NBS | www.neostem.com > 48 Eminli, S. et al. Exploring the use of human very small embryonic-like stem cells (VSELs) isolated from adult peripheral blood for therapy of dry age-related macular degeneration (AMD). ISSCR 2012 Annual Meeting, Yo
Slide 50: Ischemic Repair Program Intellectual Property
ISCHEMIC REPAIR PROGRAM INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 49 ■ Broad and growing patent portfolio supports cardiac and other ischemic conditions ■ NeoStem’s patent claims cover a pharmaceutical composition that contains a therapeutic concentration of non-expanded CD34/CXCR4 stem c
Slide 51: Recent Data Supports CD34 Stem Cell Therapy in Chronic Heart Failure
Significant need - prevalence of over 23 million worldwide, 5.7 million U.S. Therapy would enable larger distribution (not limited to mapping systems) RECENT DATA SUPPORTS CD34 STEM CELL THERAPY IN CHRONIC HEART FAILURE NASDAQ:NBS | www.neostem.com > 50 Adapted from Vrtovec et al, Circ Res publi
Slide 52: Recent Data Supports CD34 Stem Cell Therapy in Critical Limb Ischemia
NASDAQ:NBS | www.neostem.com > 51 RECENT DATA SUPPORTS CD34 STEM CELL THERAPY IN CRITICAL LIMB ISCHEMIA PROBABILITY OF AMPUTATION-FREE SURVIVAL 12 MONTHS Losordo et al. (2012) A Randomized, Controlled Pilot Study of Autologous CD34+ Cell Therapy for Critical Limb Ischemia, Circulation Cardiovascular
Slide 53: Market Opportunity in Asthma
MARKET OPPORTUNITY IN ASTHMA NASDAQ:NBS | www.neostem.com > 52 ASTHMA ■ Affects 25 million in U.S. and 300 million worldwide ■ Asthma accounts for $56 billion in annual direct and indirect health care costs in U.S. ■ Steroid resistant asthma afflicts less than 5% of the total asthma population, but