Slide 1: Investor Presentation March 2014: Transforming Medicine
INVESTOR PRESENTATION NASDAQ: NBS MARCH 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, uncertai
Slide 3: Cell Therapy
NASDAQ:NBS | www.neostem.com > 2 CELL THERAPY Using cells to prevent, treat, or cure disease by repairing and replacing damaged or aged tissue, cells, and organs in order to restore their normal function Holds the promise to dramatically transform the course of medicine: Improve clinical outcomes Re
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 ■ Combination of the development of therapies and a revenue-generating service business allows for cost effective in-house product development and immediate revenue and cash flow generation ■ Develops breakthrough products in
Slide 6: Company Overview
COMPANY OVERVIEW NASDAQ:NBS | www.neostem.com > 5 ■ Leader in cell therapy developing potentially transformative treatments for patients in multiple indications ■ Founded in 2006 ■ Integrated entity with 3 pipeline technology platforms and revenue generating contract development and manufacturing se
Slide 7: 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 8: PreSERVE Phase 2 Study Treatment Process
PRESERVE PHASE 1 STUDY TREATMENT PROCESS NASDAQ:NBS | www.neostem.com > 7 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 separatio
Slide 9: 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 10: Phase 1 Results Point to AMR-001 Potential
PHASE 1 RESULTS POINT TO AMR-001 POTENTIAL NASDAQ:NBS | www.neostem.com > 9 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 T
Slide 11: CD34 Cell Program Intellectual Property
CD34 CELL PROGRAM INTELLECTUAL PROPERTY NASDAQ:NBS | www.neostem.com > 10 ■ 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
Slide 12: PreSERVE Phase 2 Study: Enrollment Completed, Awaiting Data Release
PRESERVE PHASE 2 STUDY: ENROLLMENT COMPLETED, AWAITING DATA RELEASE NASDAQ:NBS | www.neostem.com > 11 TARGET Post-AMI preservation of cardiac function KEY INCLUSION CRITERIA Confirmation of ST Elevation MI (STEMI); ejection fraction < 48% at day 4; state of the art care post stenting LOCATION AND NU
Slide 13: What's Next? Chronic Heart Failure, Traumatic Brain Injury
WHAT’S NEXT? NASDAQ:NBS | www.neostem.com > 12 CHRONIC HEART FAILURE ■ Significant need - prevalence of over 23 million worldwide, 5.7 million US ■ Therapy would enable larger distribution (not limited to mapping systems) ■ Expect to initiate Phase 2 clinical trial in 2014 in Europe ■ Dilated cardio
Slide 14: Recent Data Supports CD34 Use in Chronic Heart Failure
RECENT DATA SUPPORTS CD34 USE IN CHRONIC HEART FAILURE NASDAQ:NBS | www.neostem.com > 13 Adapted from Vrtovec et al, Circ Res published online October 12, 201 Note: 110 patients (open label, 55 treated with cells and 55 standard of care) CD34 STEM CELL THERAPY SIGNIFICANTLY IMPROVES EVENT FREE SURVI
Slide 15: T Regulatory Cell Program: Potential to Limit Autoimmunity
T REGULATORY CELL PROGRAM: POTENTIAL TO LIMIT AUTOIMMUNITY NASDAQ:NBS | www.neostem.com > 14 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 16: Features of the Treg Program
FEATURES OF THE TREG PROGRAM NASDAQ:NBS | www.neostem.com > 15 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 17: Significant Market Opportunity in Initial Indications
SIGNIFICANT MARKET OPPORTUNITY IN INITIAL INDICATIONS NASDAQ:NBS | www.neostem.com > 16 TYPE 1 DIABETES ■ Also referred to as insulin dependent diabetes or juvenile diabetes ■ Affects >34 million worldwide or 1 in 300 children ■ Diabetes is leading cause of kidney failure, new cases of adult blindne
Slide 18: Treg Program Clinical Status
TREG PROGRAM CLINICAL STATUS NASDAQ:NBS | www.neostem.com > 17 ■ Phase 1 study for type 1 diabetes completed by Dr. Bluestone and Dr. Kevan Herold (Yale University) with results to be presented at American Diabetes Association Scientific Sessions in June 2014 ■ Advancing to initiate type 1 diabetes
Slide 19: VSEL Technology: Potential to Repair Damaged Tissue
VSEL™ TECHNOLOGY: POTENTIAL TO REPAIR DAMAGED TISSUE NASDAQ:NBS | www.neostem.com > 18 ■ Evaluating therapeutic potential of very small embryonic-like stem cells (VSELs™) ■ Research suggests multipotency and multi-lineage differentiation into all basic cell types (mesoderm, ectoderm, endoderm) ■ Exp
Slide 20: 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 > 19 PRELIMINARY DATA IN A PRECLINICAL MODEL OF SEVERE COMPLEX WOUNDS SUGGEST THAT VSELS™ MAY BE MORE EFFECTIVE IN ACCELERATING HEALING THAN MESENCHYMAL STROMAL CELLS FIBRIN CONTROL (DAY 7) FIBRIN C
Slide 21: VSELs Could Be Used to Treat Macular Degeneration
VSELS™ COULD BE USED TO TREAT MACULAR DEGENERATION NASDAQ:NBS | www.neostem.com > 20 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 22: 2014 Outlook: Clinical Milestones
2014 OUTLOOK: CLINICAL MILESTONES NASDAQ:NBS | www.neostem.com > 21 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 23: 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 24: Contract Manufacturing Is a Significant Opportunity
CONTRACT MANUFACTURING IS A SIGNIFICANT OPPORTUNITY NASDAQ:NBS | www.neostem.com > 23 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 25: Management Team
Robert Dickey IV Chief Financial Officer Over 15 years management experience at life sciences companies, following a career as an investment banker Andrew L. Pecora, MD, FACP Chief Visionary Officer, CMO of PCT, CSO of Amorcyte Chief Innovations Officer at John Theurer Cancer Center at Hackensac
Slide 26: 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 Stem for Life F
Slide 27: Key Metrics
KEY METRICS NASDAQ:NBS | www.neostem.com > 26 MARKET METRICS MARKET CAPITALIZATION1 $199M RECENT PRICE2 $7.00 52 WEEK RANGE2 $5.00 - $9.89 FLOAT1 25.1M INSIDER HOLDINGS2 11.6% FINANCIAL METRICS REVENUE3 $14.7M (2013) CASH3 $46.1M ADDITIONAL CASH4 $6.9M COMMON SHARES OUTSTANDING1 28.5M WARRANTS2 4.5M
Slide 28: Investment Highlights
INVESTMENT HIGHLIGHTS NASDAQ:NBS | www.neostem.com > 27 DEVELOPING A PORTFOLIO OF CELL THERAPY PRODUCTS THAT LEVERAGES THE BODY’S NATURAL ABILITY TO HEAL AND FIGHT DISEASE. ISCHEMIC REPAIR – CD34 CELL PROGRAM* ■ Acute myocardial infarction – PreSERVE Phase 2 study (data avaibale 2H 2014) ■ Chronic h
Slide 29: Contact Information
CONTACT INFORMATION NASDAQ:NBS | www.neostem.com > 28 NEOSTEM, INC. NASDAQ: NBS WWW.NEOSTEM.COM ROBIN SMITH, MD, MBA CHAIRMAN & CEO PHONE: (212) 584-4174 EMAIL: RSMITH@NEOSTEM.COM
Slide 30: Appendix
NASDAQ:NBS | www.neostem.com > 29 APPENDIX
Slide 31: 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 > 30
Slide 32: Athelos Scientific Advisory Board
ATHELOS SCIENTIFIC ADVISORY BOARD NASDAQ:NBS | www.neostem.com > 31 Robert A. Preti, PhD SAB Administrative Chairman CSO of NeoStem and President of PCT Jeffrey Bluestone, PhD University of California, San Francisco, Diabetes Center David A. Horwitz, MD University of Southern California Robert Korng
Slide 33: VSEL Technology Academic Collaborators
VSEL™ TECHNOLOGY ACADEMIC COLLABORATORS NASDAQ:NBS | www.neostem.com > 32 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 Institute, Harvard Medical School Kameran Lashk
Slide 34: High Cost of Cardiovascular Disease
HIGH COST OF CARDIOVASCULAR DISEASE ■ $2.7 trillion dollars is spent annually on health care costs, currently 18% of US GDP1 ■ Cardiovascular disease costs over $445 billion today and projected to increase to $1 trillion by 20302 1. Center for Medicare and Medicaid, statistics for 2011 2. American H
Slide 35: Phase 1 Trial Design for AMR-001
PHASE 1 TRIAL DESIGN FOR AMR-001 INDICATION Post-AMI with LVEF ≤50% and wall motion abnormality in the myocardium of the IRA PRIMARY ENDPOINT Safety in post-AMI patients OTHER ENDPOINTS RTSS* (Perfusion); LVEF; ESV; SDF mobility KEY INCLUSION CRITERIA Confirmation of ST Elevation MI; Ejection fracti
Slide 36: Ex Vivo Expanded Human Tregs Show Safety and Potential Efficacy
EX VIVO EXPANDED HUMAN TREGS SHOW SAFETY AND POTENTIAL EFFICACY IN EARLY CLINICAL TRIALS NASDAQ:NBS | www.neostem.com > 35 EX VIVO EXPANDED TREGS SUPPRESS CD4+ T EFFECTOR CELLS % Suppression T effector to nT-reg ratio PRE-SORT POST-SORT Post-sort nT-reg: >90% BLT serum ALT serum-free CD25 CD127 CD4