Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (April 2014)
49 slides from Lisata Therapeutics's investor presentation, Apr 18, 2014
Slide 1: Investor Presentation April 2014: Transforming Medicine
INVESTOR PRESENTATION NASDAQ: NBS APRIL 2014 TRANSFORMING MEDICINE
Slide 2: Forward-Looking Statements
NASDAQ:NBS | www.neostem.com > 1 FORWARD-LOOKING STATEMENTS This presentation includes “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, uncerta
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: Acquisition of California Stem Cell, Inc. (CSC)
NASDAQ:NBS | www.neostem.com > 4 ACQUISITION OF CALIFORNIA STEM CELL, INC. (CSC) ■ Merger agreement signed April 11, 2014 ■ Requisite number of CSC stockholders have consented to adoption of the merger ■ Closing anticipated May 2014, subject to satisfaction of closing conditions ■ This presentation
Slide 6: NeoStem Has an Integrated Business Model
NASDAQ:NBS | www.neostem.com > 5 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 7: Development Highlights: Multiple Platform Technologies
DEVELOPMENT HIGHLIGHTS: MULTIPLE PLATFORM TECHNOLOGIES NASDAQ:NBS | www.neostem.com > 6 DEVELOPING A PORTFOLIO OF CELL THERAPY PRODUCTS THAT LEVERAGES THE BODY’S NATURAL ABILITY TO HEAL AND FIGHT DISEASE CANCER – TARGETED IMMUNOTHERAPY PROGRAM* ISCHEMIC REPAIR – CD34 CELL PROGRAM IMMUNE MODULA
Slide 8: Targeted Immunotherapy Program Rationale
TARGETED IMMUNOTHERAPY PROGRAM RATIONALE NASDAQ:NBS | www.neostem.com > 7 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) cells, whic
Slide 9: Targeted Immunotherapy Treatment Process
TARGETED IMMUNOTHERAPY TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 8 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 them inact
Slide 10: Features of Our Targeted Immunotherapy Program
FEATURES OF OUR TARGETED IMMUNOTHERAPY PROGRAM NASDAQ:NBS | www.neostem.com > 9 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 expres
Slide 11: First Target Indication: Melanoma
FIRST TARGET INDICATION: MELANOMA NASDAQ:NBS | www.neostem.com > 10 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 Sta
Slide 12: Other Therapeutics for Melanoma
OTHER THERAPEUTICS FOR MELANOMA NASDAQ:NBS | www.neostem.com > 11 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 13: Phase 2 Results for Melapuldencel-T
PHASE 2 RESULTS FOR MELAPULDENCEL-T NASDAQ:NBS | www.neostem.com > 12 Dillman, et al. Journal Immunotherapy 2012 31% 0% 20% 40% 60% 80% Control Group (Irradiated Tumor Cells Only) Melapuldencel-T Treatment Group 2 YEAR OVERALL SURVIVAL 72% • No serious adverse events related to immunotherapy • Minor
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 AND NUMBER OF SUBJECTS United States and Europe, multicenter, 250 patients* DESIGN Double blind, placebo control
Slide 15: CD34 Cell Program: Enhancing the Body's Natural Repair Mechanism
CD34 CELL PROGRAM: ENHANCING THE BODY’S NATURAL REPAIR MECHANISM ■ 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 wor
Slide 16: PreSERVE 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: Features and Benefits of AMR-001
FEATURES AND BENEFITS OF AMR-001 ■ 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 (development and formation of ne
Slide 18: Phase 1 Results Point to AMR-001 Potential
PHASE 1 RESULTS POINT TO AMR-001 POTENTIAL NASDAQ:NBS | www.neostem.com > 17 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
Slide 19: Migratory Capacity of CD34 Cells Associated with Event-Free Survival Post AMI
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: PreSERVE Phase 2 Study: Enrollment Completed, Data Release 2H 2014
PRESERVE PHASE 2 STUDY: ENROLLMENT COMPLETED WITH ANTICIPATED DATA RELEASE 2H 2014 NASDAQ:NBS | www.neostem.com > 19 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 S
Slide 21: Recent Data Supports CD34 Stem Cell Therapy in Chronic Heart Failure
RECENT DATA SUPPORTS CD34 STEM CELL THERAPY IN CHRONIC HEART FAILURE NASDAQ:NBS | www.neostem.com > 20 Adapted from Vrtovec et al, Circ Res published online 10/12/2012 Note: 110 patients (open label, 55 treated with cells and 55 standard of care) CD34 STEM CELL THERAPY SIGNIFICANTLY IMPROVES EVENT F
Slide 22: T Regulatory Cell Program: Potential to Limit Autoimmunity
T REGULATORY CELL PROGRAM: POTENTIAL TO LIMIT AUTOIMMUNITY NASDAQ:NBS | www.neostem.com > 21 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 23: Features of the Treg Program
FEATURES OF THE TREG PROGRAM NASDAQ:NBS | www.neostem.com > 22 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 permits the T ef
Slide 24: Diabetes Mellitus Type-1 (T1D)
DIABETES MELLITUS TYPE-1 (T1D) NASDAQ:NBS | www.neostem.com > 23 Diabetes is leading cause of kidney failure, new cases of adult blindness, and non-traumatic lower-limb amputations Results in total insulin deficiency At time of diagnosis, there are still insulin-secreting beta cells in islets
Slide 25: Economic Impact of T1D
ECONOMIC IMPACT OF T1D NASDAQ:NBS | www.neostem.com > 24 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 26: Treg Immunotherapy Works in Model of T1D
NASDAQ:NBS | www.neostem.com > 25 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 27: T1D Treg Treatment Process
T1D TREG TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 26 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 28: Regulatory T Cells Preserve Beta Cell Function in T1D in Children
NASDAQ:NBS | www.neostem.com > 27 ADMINISTRATION OF CD4+CD25highCD127- REGULATORY T CELLS PRESERVES BETA CELL FUNCTION IN T1D IN CHILDREN* Not Treated Time 0 Not Treated 4 mo 0 2 wk 2 mo 4 mo First human evidence of therapeutic effect of autologous Treg therapy protection of pancreatic function in
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
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 IMMUNOTHERAPY PROGRAM (CANCER) ■ 13 pending patents with coverage including: ► Individualized high purity carcinoma initiating (stem) cells for target indications, methods and use of same; and rapid methods to produce high purity cance
Slide 32: 2014 Outlook: Clinical Milestones
2014 OUTLOOK: CLINICAL MILESTONES NASDAQ:NBS | www.neostem.com > 31 1. The last patient primary endpoint follow-up for this study is expected in June followed by data lock and analysis with data available in 2H 2014. 2. It is expected that this study will be presented at the American Diabetes Associ
Slide 33: PCT Provides Outsourced Manufacturing Capabilities
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 Over 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 Offi
Slide 36: Board of Directors
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 Ste
Slide 37: Key Metrics
KEY METRICS NASDAQ:NBS | www.neostem.com > 36 MARKET METRICS MARKET CAPITALIZATION1 $183M RECENT PRICE2 $6.40 52 WEEK RANGE2 $5.00 - $9.89 FLOAT1 25.2M INSIDER HOLDINGS2 11.5% FINANCIAL METRICS REVENUE3 $14.7M (2013) CASH3 $46.1M ADDITIONAL CASH4 $6.9M COMMON SHARES OUTSTANDING1 28.6M WARRANTS2 4.0M
Slide 38: Unique Business Model
UNIQUE BUSINESS MODEL NASDAQ:NBS | www.neostem.com > 37 COMBINATION OF A LATE STAGE CLINICAL PIPELINE AND A REVENUE-GENERATING SERVICE BUSINESS CANCER – TARGETED IMMUNOTHERAPY PROGRAM ■ Stage IV and recurrent Stage III melanoma – Intus Phase 3 study approved, initiating 2H 2014 ISCHEMIC REPAIR – CD3
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 $189M and Completed Multiple M&A Transactions
SINCE 2006, ACCESSED OVER $189M AND COMPLETED MULTIPLE M&A TRANSACTIONS AND ONE DIVESTITURE NASDAQ:NBS | www.neostem.com > 40 * Closing expected in May 2014, subject to customary closing conditions *
Slide 42: Amorcyte Scientific Advisory Board
AMORCYTE SCIENTIFIC ADVISORY BOARD Andrew L. Pecora, MD, FACP, CPE SAB Administrative Chairman Hackensack University Medical Center Chief Scientific Officer, Amorcyte Eugene Braunwald, MD, FRCP Brigham & Women’s Hospital Bernard J. Gersh, MD, ChB, DPhil, FRCP The Mayo Clinic Dean J. Kereiakes, MD, F
Slide 43: Athelos Scientific Advisory Board
ATHELOS SCIENTIFIC ADVISORY BOARD NASDAQ:NBS | www.neostem.com > 42 Robert A. Preti, PhD SAB Administrative Chairman Chief Scientific Officer of NeoStem and PCT, President of PCT Jeffrey Bluestone, PhD University of California, San Francisco, Diabetes Center David A. Horwitz, MD University of Southe
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: T1D Is on the Rise
T1D IS ON THE RISE NASDAQ:NBS | www.neostem.com > 44 NEW CASES OF T1D (0-14 YEARS) PER 100,000 CHILDREN, 2013: CONCENTRATION IN DEVELOPED MARKETS Dabelea, Lancet, 2009
Slide 46: Human VSELs Accelerate Healing in a SCID Mouse Wound Model
HUMAN VSELS™ ACCELERATE HEALING IN A SCID MOUSE COMPLEX TAIL WOUND MODEL NASDAQ:NBS | www.neostem.com > 45 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 47: VSELs Could Be Used to Treat Macular Degeneration
VSELS™ COULD BE USED TO TREAT MACULAR DEGENERATION NASDAQ:NBS | www.neostem.com > 46 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 48: California Stem Cell Intellectual Property
CALIFORNIA STEM CELL INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 47 EIGHT ISSUED PATENTS WITH COVERAGE INCLUDING: Cardiomyocytes and methods of producing and purifying cardiomyocytes Stem cell growth medium and methods of making and using same Human late stage motor neuron progenito
Slide 49: CD34 Cell Program Intellectual Property
CD34 CELL PROGRAM INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 48 ■ 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