Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (September 2014)
50 slides from Lisata Therapeutics's investor presentation, Sep 8, 2014
Slide 1: Investor Presentation September 2014: Transforming Medicine
INVESTOR PRESENTATION NASDAQ: NBS SEPTEMBER 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: Cell Therapy
NASDAQ:NBS | www.neostem.com > 2 CELL THERAPY Using cells to prevent or treat disease and modulate the immune system Holds the promise to dramatically transform the course of medicine Improve clinical outcomes Reduce overall healthcare costs
Slide 4: About NeoStem
NASDAQ:NBS | www.neostem.com > 3 ABOUT NEOSTEM Leader in the emerging cellular therapy industry developing novel proprietary cell therapy products as well as generating revenue through a contract development and manufacturing organization that we believe will benefit from the growth of this industry
Slide 5: NeoStem Has an Integrated Business Model
NASDAQ:NBS | www.neostem.com > 4 NEOSTEM HAS AN INTEGRATED BUSINESS MODEL Therapeutic development around a significant IP portfolio and a revenue-generating service business allows for cost effective in- house product development and immediate revenue
Slide 6: Development Highlights: Multiple Platform Technologies
DEVELOPMENT HIGHLIGHTS: MULTIPLE PLATFORM TECHNOLOGIES NASDAQ:NBS | www.neostem.com > 5 DEVELOPING A PORTFOLIO OF CELL THERAPY PRODUCTS THAT LEVERAGES THE BODY’S NATURAL ABILITY TO HEAL AND FIGHT DISEASE TARGETED CANCER IMMUNOTHERAPY PROGRAM Using DC/TC Technology ISCHEMIC REPAIR PROGRAM Usi
Slide 7: Targeted Cancer Immunotherapy Program Rationale
TARGETED CANCER IMMUNOTHERAPY PROGRAM RATIONALE NASDAQ:NBS | www.neostem.com > 6 Cancer initiating (stem) cells isolated from patient tumor provide potent signature antigens to educate and direct the immune system Immunotherapy product uniquely targets the patient’s cancer initiating (stem) cell
Slide 8: Targeted Cancer Immunotherapy Treatment Process
TARGETED CANCER IMMUNOTHERAPY TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 7 STEP 1: Creation of the 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 the
Slide 9: Features of Our Targeted Cancer Immunotherapy Program
FEATURES OF OUR TARGETED CANCER IMMUNOTHERAPY PROGRAM NASDAQ:NBS | www.neostem.com > 8 OUR IMMUNOTHERAPY OVERCOMES Presents the entire spectrum of patient-specific cancer initiating (stem) cells for the immune system to target Limited antigen targeting Targets the cancer initiating (stem) cells that
Slide 10: First Target Indication: Melanoma
FIRST TARGET INDICATION: MELANOMA NASDAQ:NBS | www.neostem.com > 9 BASICS OF MELANOMA Most lethal form of skin cancer Most often caused by unrepaired DNA damage to skin cells from UV radiation 120,000 new cases per year in U.S.1 Kills an estimated 8,790 in U.S. annually2 SURVIVAL RATE Stag
Slide 11: Other Therapeutics for Melanoma
OTHER THERAPEUTICS FOR MELANOMA NASDAQ:NBS | www.neostem.com > 10 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 inhibito
Slide 12: Phase 2 Results for DC/TC Product Candidate for Metastatic Melanoma
PHASE 2 RESULTS FOR DC/TC PRODUCT CANDIDATE FOR METASTATIC MELANOMA NASDAQ:NBS | www.neostem.com > 11 Dillman, et al. Journal Immunotherapy 2012 31% 0% 20% 40% 60% 80% Control Group (Irradiated Tumor Cells Only) DC/TC Treatment Group 2 YEAR OVERALL SURVIVAL 72% • No serious adverse events related to
Slide 13: INTUS Phase 3 Special Protocol Assessment Study Design
INTUS PHASE 3 SPECIAL PROTOCOL ASSESSMENT (SPA) STUDY DESIGN NASDAQ:NBS | www.neostem.com > 12 STUDY NAME TARGET Patients with Stage IV or recurrent Stage III metastatic melanoma LOCATION United States and potentially Australia & New Zealand, approximately 60 sites DESIGN Double blind, placebo contr
Slide 14: Ischemic Repair Program: Enhancing the Body's Natural Repair Mechanism
ISCHEMIC REPAIR PROGRAM: ENHANCING THE BODY’S NATURAL REPAIR MECHANISM ■ Ischemia occurs when supply of oxygenated blood in body is restricted ■ We seek to reverse this restriction through development and formation of new blood vessels ■ CD34/CXCR4 cells are a natural repair mechanism, following the
Slide 15: NBS10 (AMR-001) for Post-STEMI Treatment
NBS10 (AMR-001) FOR POST- STEMI TREATMENT ■ 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 heart functio
Slide 16: PreSERVE Phase 2 Study Treatment Process
PRESERVE PHASE 2 STUDY TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 15 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 separati
Slide 17: Phase 1 Results Point to NBS10 Potential
PHASE 1 RESULTS POINT TO NBS10 POTENTIAL NASDAQ:NBS | www.neostem.com > 16 Patients dosed ≥ the threshold dose of 10 million cells showed significant improvement in perfusion Increasing doses of CD34/SDF-1 mobile cells reduced the size of the infarct region as measured by CMR DSMB DETERMINED THAT TH
Slide 18: PreSERVE Phase 2 Study: Enrollment Completed
PRESERVE PHASE 2 STUDY: ENROLLMENT COMPLETED WITH ANTICIPATED DATA RELEASE 2014 NASDAQ:NBS | www.neostem.com > 17 TARGET Post-AMI patients KEY INCLUSION CRITERIA Confirmation of ST Elevation MI (STEMI); ejection fraction < 48% at day 4; state of the art care post stenting LOCATION AND NUMBER OF SUBJ
Slide 19: Migratory Capacity of Administered CD34 Cells Associated with Event-Free Survival
MIGRATORY CAPACITY OF ADMINISTERED CD34 CELLS ASSOCIATED WITH EVENT- FREE SURVIVAL POST AMI NASDAQ:NBS | www.neostem.com > 18 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 20: Features and Benefits of NBS10
FEATURES AND BENEFITS OF NBS10 NASDAQ:NBS | www.neostem.com > 19 ■ CD34/CXCR4 cells home to the viable tissue surrounding the infarcted (dead) myocardium (peri-infarct zone) after administration and persist ■ Autologous cells take up residence in the peri-infarct zone, likely promoting angiogenesis
Slide 21: 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 > 20 Adapted from Vrtovec et al, Circ Res publi
Slide 22: Recent Data Supports CD34 Stem Cell Therapy in Critical Limb Ischemia
NASDAQ:NBS | www.neostem.com > 21 RECENT DATA SUPPORTS CD34 STEM CELL THERAPY IN CRITICAL LIMB ISCHEMIA PROBABILITY OF AMPUTATION-FREE SURVIVAL 12 MONTHS High Dose Low Dose Control Log Rank Test Control vs. Low Dose, p=0.187 Control vs. High Dose, p=0.014 Losordo et al. (2012) A Randomized, Controll
Slide 23: Immune Modulation Program: Potential to Limit Autoimmunity
IMMUNE MODULATION PROGRAM: POTENTIAL TO LIMIT AUTOIMMUNITY NASDAQ:NBS | www.neostem.com > 22 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 24: Features of Our Immune Modulation Program
FEATURES OF OUR IMMUNE MODULATION PROGRAM NASDAQ:NBS | www.neostem.com > 23 FEATURES OF TREGS: ■ Natural part of immune system ■ Regulate activity of T effector cells (responsible for protection from viruses and foreign antigens) ■ In autoimmune disease it is thought that deficient Treg activity per
Slide 25: First Target Indication: Diabetes Mellitus Type-1
FIRST TARGET INDICATION: DIABETES MELLITUS TYPE-1 (T1D) NASDAQ:NBS | www.neostem.com > 24 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 26: T1D Treg Treatment Process
T1D TREG TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 25 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 27: Administration of Regulatory T Cells Preserves Beta Cell Function in Children
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 28: 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 > 27 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 29: Market Opportunity in Asthma
MARKET OPPORTUNITY IN ASTHMA NASDAQ:NBS | www.neostem.com > 28 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
Slide 30: Tissue Regeneration Programs: VSEL Technology
TISSUE REGENERATION PROGRAMS NASDAQ:NBS | www.neostem.com > 29 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 31: Intellectual Property
NASDAQ:NBS | www.neostem.com > 30 INTELLECTUAL PROPERTY TARGETED CANCER IMMUNOTHERAPY PROGRAM ■ 1 issued patent and 28 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 32: Clinical Timelines
CLINICAL TIMELINES NASDAQ:NBS | www.neostem.com > 31 1. An abstract for the PreSERVE AMI study has been accepted for presentation at the American Heart Association's Scientific Sessions being held November 15-19, 2014 although we anticipate results of the study will be released earlier 2. Subject to
Slide 33: 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 PCT PROVIDES OUTSOURCED MANUFACTURING CAPABILITIES T
Slide 34: Contract Manufacturing Is a Significant Opportunity
CONTRACT MANUFACTURING IS A SIGNIFICANT OPPORTUNITY NASDAQ:NBS | www.neostem.com > 33 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 COMP
Slide 35: Management Team
Robert Dickey IV, MBA – Chief Financial Officer 15+ years management experience at life science companies, including cell therapy experience as CFO of StemCyte, following a career as an investment banker at Lehman Brothers Andrew L. Pecora, MD – Chief Visionary Officer Chief Innovations Officer
Slide 36: 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 37: Key Metrics
KEY METRICS NASDAQ:NBS | www.neostem.com > 36 MARKET METRICS MARKET CAPITALIZATION1 $203M STOCK PRICE2 $5.75 52 WEEK RANGE2 $4.56 - $9.89 FLOAT1 31.1M INSIDER HOLDINGS1 12% FINANCIAL METRICS REVENUE3 $4.5M (Second Quarter) CASH4 $33.8M COMMON SHARES OUTSTANDING1 35.3M WARRANTS1 3.6M (avg. warrant ex
Slide 38: Unique Business Model: Late Stage Clinical Pipeline and Revenue-Generating Service Business
UNIQUE BUSINESS MODEL NASDAQ:NBS | www.neostem.com > 37 COMBINATION OF A LATE STAGE CLINICAL PIPELINE AND A REVENUE-GENERATING SERVICE BUSINESS CANCER TREATMENT – TARGETED IMMUNOTHERAPY PROGRAM ■ Stage IV and recurrent Stage III melanoma – Intus Phase 3 study approved, initiating 2H 2014 ISCHEMIC RE
Slide 39: Contact Information
CONTACT INFORMATION NASDAQ:NBS | www.neostem.com > 38 NEOSTEM, INC. NASDAQ: NBS WWW.NEOSTEM.COM ROBIN SMITH, MD, MBA CHAIRMAN & CEO PHONE: (212) 584-4174 EMAIL: RSMITH@NEOSTEM.COM
Slide 40: Appendix
NASDAQ:NBS | www.neostem.com > 39 APPENDIX
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: CD34 Cell Program Intellectual Property
CD34 CELL 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 cells t