Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (May 2014)
48 slides from Lisata Therapeutics's investor presentation, May 8, 2014
Slide 1: Investor Presentation May 2014: Transforming Medicine
INVESTOR PRESENTATION NASDAQ: NBS MAY 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 CANCER TREATMENT – TARGETED IMMUNOTHERAPY PROGRAM ISCHEMIC REPAIR – CD34 CELL PROGRAM IMMU
Slide 7: Targeted Immunotherapy Program Rationale
TARGETED 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) cells, whic
Slide 8: Targeted Immunotherapy Treatment Process
TARGETED 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 them inact
Slide 9: Features of Our Targeted Immunotherapy Program
FEATURES OF OUR TARGETED 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 expres
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 Results for Melapuldencel-T
PHASE 2 RESULTS FOR MELAPULDENCEL-T NASDAQ:NBS | www.neostem.com > 11 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 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 AND NUMBER OF SUBJECTS United States and Europe, multicenter, 250 patients* DESIGN Double blind, placebo control
Slide 14: 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 15: PreSERVE Phase 2 Study Treatment Process
PRESERVE PHASE 2 STUDY TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 14 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 16: Features and Benefits of AMR-001
FEATURES AND BENEFITS OF AMR-001 NASDAQ:NBS | www.neostem.com > 15 ■ 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 angiogenesi
Slide 17: Phase 1 Results Point to AMR-001 Potential
PHASE 1 RESULTS POINT TO AMR-001 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
Slide 18: Migratory Capacity of Administered CD34 Cells and Event-Free Survival
MIGRATORY CAPACITY OF ADMINISTERED CD34 CELLS ASSOCIATED WITH EVENT- FREE SURVIVAL POST AMI NASDAQ:NBS | www.neostem.com > 17 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 19: PreSERVE Phase 2 Study: Enrollment Completed
PRESERVE PHASE 2 STUDY: ENROLLMENT COMPLETED WITH ANTICIPATED DATA RELEASE 2H 2014 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; state of the art care post stenting LOCATION AND NUMBER OF S
Slide 20: 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 > 19 Adapted from Vrtovec et al, Circ Res publi
Slide 21: T Regulatory Cell Program: Potential to Limit Autoimmunity
T REGULATORY CELL PROGRAM: POTENTIAL TO LIMIT AUTOIMMUNITY NASDAQ:NBS | www.neostem.com > 20 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 22: Features of Our Treg Program
FEATURES OF OUR TREG PROGRAM NASDAQ:NBS | www.neostem.com > 21 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 23: Diabetes Mellitus Type-1 (T1D)
DIABETES MELLITUS TYPE-1 (T1D) NASDAQ:NBS | www.neostem.com > 22 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 24: Economic Impact of T1D
ECONOMIC IMPACT OF T1D NASDAQ:NBS | www.neostem.com > 23 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 25: Treg Immunotherapy Works in Model of T1D
NASDAQ:NBS | www.neostem.com > 24 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 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: Regulatory T Cells Preserve Beta Cell Function in T1D in Children
NASDAQ:NBS | www.neostem.com > 26 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 28: Market Opportunity in Asthma
MARKET OPPORTUNITY IN ASTHMA NASDAQ:NBS | www.neostem.com > 27 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 29: Tissue Regeneration Programs
TISSUE REGENERATION PROGRAMS NASDAQ:NBS | www.neostem.com > 28 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 30: Intellectual Property
NASDAQ:NBS | www.neostem.com > 29 INTELLECTUAL PROPERTY TARGETED IMMUNOTHERAPY PROGRAM (CANCER TREATMENT) ■ 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 pu
Slide 31: 2014 Outlook: Clinical Milestones
2014 OUTLOOK: CLINICAL MILESTONES NASDAQ:NBS | www.neostem.com > 30 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 by Dr. Jeffrey Bluestone (University of Californi
Slide 32: 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 33: Contract Manufacturing Is a Significant Opportunity
CONTRACT MANUFACTURING IS A SIGNIFICANT OPPORTUNITY NASDAQ:NBS | www.neostem.com > 32 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 34: 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 35: 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 36: Key Metrics
KEY METRICS NASDAQ:NBS | www.neostem.com > 35 MARKET METRICS MARKET CAPITALIZATION1 $203M STOCK PRICE2 $5.98 52 WEEK RANGE2 $5.00 - $9.89 FLOAT1 29.8M INSIDER HOLDINGS1 11.9% FINANCIAL METRICS REVENUE3 $4.1M (First Quarter) CASH4 $41.4M COMMON SHARES OUTSTANDING1 33.9M WARRANTS1 4.0M (avg. warrant e
Slide 37: Unique Business Model
UNIQUE BUSINESS MODEL NASDAQ:NBS | www.neostem.com > 36 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 38: Contact Information
CONTACT INFORMATION NASDAQ:NBS | www.neostem.com > 37 NEOSTEM, INC. NASDAQ: NBS WWW.NEOSTEM.COM ROBIN SMITH, MD, MBA CHAIRMAN & CEO PHONE: (212) 584-4174 EMAIL: RSMITH@NEOSTEM.COM
Slide 39: Appendix
NASDAQ:NBS | www.neostem.com > 38 APPENDIX
Slide 40: 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 > 39
Slide 41: 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 42: Athelos Scientific Advisory Board
ATHELOS SCIENTIFIC ADVISORY BOARD NASDAQ:NBS | www.neostem.com > 41 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 43: VSEL Technology Academic Collaborators
VSEL™ TECHNOLOGY ACADEMIC COLLABORATORS NASDAQ:NBS | www.neostem.com > 42 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 44: T1D Is on the Rise
T1D IS ON THE RISE NASDAQ:NBS | www.neostem.com > 43 NEW CASES OF T1D (0-14 YEARS) PER 100,000 CHILDREN, 2013: CONCENTRATION IN DEVELOPED MARKETS Dabelea, Lancet, 2009
Slide 45: Human VSELs Accelerate Healing in a SCID Mouse Tail Wound Model
HUMAN VSELS™ ACCELERATE HEALING IN A SCID MOUSE COMPLEX TAIL WOUND MODEL NASDAQ:NBS | www.neostem.com > 44 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 46: VSELs Could Be Used to Treat Macular Degeneration
VSELS™ COULD BE USED TO TREAT MACULAR DEGENERATION NASDAQ:NBS | www.neostem.com > 45 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 47: NeoStem Oncology Intellectual Property
NEOSTEM ONCOLOGY INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 46 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 progenitor ce
Slide 48: CD34 Cell Program Intellectual Property
CD34 CELL PROGRAM INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 47 ■ 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