Slide 1: Transforming the Treatment of Chronic Disease
NASDAQ: NBS | 1 Investor Presentation NASDAQ: NBS September 2013 Transforming the Treatment of Chronic Disease
Slide 2: Forward-Looking Statements
NASDAQ: NBS | 2 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, uncertainties and other f
Slide 3: Regenerative Medicine
NASDAQ: NBS | 3 Regenerative Medicine •Repair or replace damaged tissue and restore function •Novel regenerative therapies with potential to: Improve clinical outcomes Reduce overall healthcare costs
Slide 4: NeoStem Has an Integrated Business Model
NASDAQ: NBS | 4 NeoStem Has an Integrated Business Model • Develops therapies for chronic unmet medical needs around a significant IP portfolio • Benefits from growth of the regenerative medicine industry through development and manufacturing contracts
Slide 5: Corporate Goals
NASDAQ: NBS | 5 Drive shareholder value through… 1) Growing a successful global cell therapy contract development and manufacturing business 2) Developing cutting edge therapies around a strong IP portfolio in the treatment of chronic diseases, becoming a global leader in regenerative medicine and d
Slide 6: Since 2006, We Have Accessed Over $138 Million and Completed Multiple M&A Transactions
NASDAQ: NBS | 6 Since 2006, We Have Accessed Over $138 Million and Completed Multiple M&A Transactions and One Divestiture Acquired Stem Cell Technologies, Inc. Acquired CBH (51% of Erye) Divested Began partnership with Vatican Acquired 80% Acquired Acquired Phase III Medical, Inc. Acquired NeoStem,
Slide 7: Our Contract Development and Manufacturing Organization (CDMO) Business
NASDAQ: NBS | 7 Our Contract Development and Manufacturing Organization (CDMO) Business
Slide 8: 15-Year Track Record of Success
NASDAQ: NBS | 8 • Currently serve more than 35 clients • Demonstrated regulatory expertise having successfully completed 50+ EU and US regulatory filings and worked with a client through all phases of clinical trials, to BLA submission, and product approval by FDA • Provide established, high quality
Slide 9: Manufacturing Growth Plans
NASDAQ: NBS | 9 • Initiatives focused on lowering cost of goods and increasing gross profits through innovation, engineering and automation • Pursue commercial expansion of manufacturing in the US and internationally • Establish opportunities for early partnerships with goals of commercial manufactu
Slide 10: Contract Complexity, Duration and Value by Product Type
NASDAQ: NBS | 10 Low Complexity Product Medium Complexity Product High Complexity Product Pre-clinical Drug Discovery Contracts Phase 2 Clinical Trial Manufacturing Contract Phase 1 Clinical Trial Manufacturing Contract Phase 3 Clinical Trial Manufacturing Contract Commercial Manufacturing Contract
Slide 11: Building a Best in Class Cell Therapy Pipeline
NASDAQ: NBS | 11 Building a Best in Class Cell Therapy Pipeline
Slide 12: Built for Success in Regenerative Medicine
NASDAQ: NBS | 12 Cardiovascular disease* • Acute myocardial infarction – PreSERVE Phase 2 Study • Congestive heart failure – Preparing for Phase 1b/2a • Traumatic brain injury – Preclinical * These cells (AMR-001) are autologous and not expanded Autoimmune disorders • Type 1 diabetes – Phase 2 IND p
Slide 13: High Cost of Cardiovascular Disease
NASDAQ: NBS | 13 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 2
Slide 14: AMR-001 Brings Repair System to the Heart After a STEMI
NASDAQ: NBS | 14 AMR-001 Brings Repair System to the Heart in Order to Preserve Function After a STEMI • CD34⁺CXCR4+ Cells are a natural repair mechanism • A consequence of inadequate perfusion (microvascular insufficiency) after a heart attack is apoptosis and progressive cardiomyocyte loss in the
Slide 15: PreSERVE-AMI Phase 2 Trial Design
NASDAQ: NBS | 15 Indication 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 Number of Subjects United States, 60 centers, 131 of 160 patients infused as of 9/6
Slide 16: Phase 1 Results Point to AMR-001 Potential
NASDAQ: NBS | 16 Phase 1 Results Point to AMR-001 Potential Dose Response Correlated with Mobile CD34+ Cells RTSS (Hypoperfusion) Cohort Base Line 6 months Delta % Change Control 259.0 273.5 +14.5 +5.6 5M Cells 714.2 722.0 +7.8 +1.1 10M Cells 998.6 635.8 -362.8 -36.4 15M Cells 584.0 462.0 -122.0 -20
Slide 17: Intellectual Property
NASDAQ: NBS | 17 • Broad and growing patent portfolio supports cardiac and other ischemic conditions • Amorcyte’s patent claims cover a pharmaceutical composition that contains a therapeutic concentration of non-expanded CD34+ CXCR4+ stem cells that move in response to SDF-1, together with a stabili
Slide 18: What's Next? Congestive Heart Failure
NASDAQ: NBS | 18 What’s Next? Congestive Heart Failure Incidence 660,000 Prevalence 5,800,000 American Heart Association 23,000,000 Prevalence 20,000,000 Case Western University
Slide 19: CD34+ Stem Cell Therapy Improves Event Free Survival at 5 Years in Dilated Cardiomyopathy
NASDAQ: NBS | 19 Adapted from Vrtovec et al, Circ Res published online 10/12/12 Note: 110 patients (open label, 55 treated with cells and 55 standard of care) CD34+ Stem Cell Therapy Improves Event Free Survival at 5 Years in Patients with Dilated Cardiomyopathy Pump Failure Deaths Sudden Cardiac De
Slide 20: Using Treg Cells to Restore Immune Balance
NASDAQ: NBS | 20 1) Chai, Jian-Guo et al, Journal of Immunology 2008; 180;858-869 • Treg therapy represents a novel approach for restoring immune balance by enhancing T-regulatory cell number and function1 • Partnership with Becton Dickinson (20% ownership of Athelos) • Immune-mediated diseases such
Slide 21: Recent Advancements in the Treg Program
NASDAQ: NBS | 21 • Type 1 diabetes affects over 340 million worldwide with over $174 billion economic cost in US alone • Advancing to Phase 2 study in 2014 through collaboration with Drs. Jeffrey Bluestone and Qizhi Tang (UCSF) • Severe asthma affects 60 million worldwide and costs US $56 billion pe
Slide 22: VSELs Regenerative Medicine Potential
NASDAQ: NBS | 22 • Preliminary data generated by third party collaborators in animal models have indicated that highly enriched human very small embryonic-like stem cells (VSELs™) are able to integrate, differentiate and potentially regenerate into all basic cell types (mesoderm, ectoderm, endoderm)
Slide 23: Human VSELs Accelerate Healing in a SCID Mouse Complex Tail Wound Model
NASDAQ: NBS | 23 • Preliminary data in a preclinical model of severe complex wounds suggest that VSELs™ may be more effective in accelerating healing than mesenchymal stromal cells % R e -ep it he lia liz at io n p < 0.05 Days Post-wounding VSELs vs. MSCs % R e -epi thel ia liz at io n 2,500 human V
Slide 24: Human VSELs Injected into Mouse Sub-Retinal Space Show Differentiation Potential
NASDAQ: NBS | 24 PKH-26/Recoverin PKH-26 positive cells co-labeled with Recoverin (400x). ONL INL GCL RPE PKH-26/rhodopsin PKH-26 positive cells co-labeled with rhodopsin (400x). ONL INL RPE GCL Eminli, S. et al. Exploring the use of human very small embryonic-like stem cells (VSELs) isolated from a
Slide 25: Key Executives
NASDAQ: NBS | 25 Key Executives Robin Smith, MD, MBA CEO & Chairman of the Board MD – Yale; MBA – The Wharton School Formerly President & CEO IP2M (HC multimedia), EVP & CMO HealthHelp (radiology management) Trustee of NYU Medical Center; Chairman of the Board of NYU Hospital for Joint Disease
Slide 26: Board of Directors
NASDAQ: NBS | 26 Board of Directors Robin Smith, MD, MBA CEO & 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 20
Slide 27: Key Metrics
NASDAQ: NBS | 27 Market Metrics Market Capitalization(1) $150M Enterprise Value(1) $138M Recent Price(2) $7.60 52 Week Range(2) $5.00 - $8.90 Float(1) 17.1M Insider Holdings(3) 15.8% Institutional Holdings(4) 6.6% 1) As of August 7, 2013, based on 20.3 million shares outstanding and a $7.40 share pr
Slide 28: NeoStem Milestones
NASDAQ: NBS | 28 • Therapeutic Pipeline - Complete enrollment PreSERVE-AMI Phase 2 trial - 1st data readout 6-8 months after last patient infused - Advancing towards Phase 1b/2a AMR-001 CHF trial - Advancing towards VSELTM human bone growth trials - Advancing Treg cell program towards Phase 2 trial
Slide 29: Contact Information
NASDAQ: NBS | 29 Contact Information NeoStem, Inc. NASDAQ: NBS www.neostem.com Robin Smith, MD, MBA Chairman & CEO Phone: (212) 584-4174 Email: rsmith@neostem.com
Slide 30: Appendix
Appendix 02. NeeStem NASDAQ: NBS | 30
Slide 31: Amorcyte Scientific Advisory Board
NASDAQ: NBS | 31 Amorcyte Scientific Advisory Board 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 Greater Cincinnati Douglas L. Mann, MD, FACC Washington University School of Medic
Slide 32: Athelos Scientific Advisory Board
NASDAQ: NBS | 32 Athelos Scientific Advisory Board Robert A. Preti, PhD SAB Administrative Chairman SO 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 Korngold, PhD Hackensac
Slide 33: VSEL Technology Academic Collaborators
NASDAQ: NBS | 33 VSEL™ Technology Academic Collaborators Mariusz Ratajczak, MD, PhD, Dsci University of Louisville Russell Taichman, DMD, DMSc University of Michigan Vincent Falanga, MD Boston University Kameran Lashkari, MD Schepens Eye Institute, Harvard Medical School Song Li, PhD University of C
Slide 34: Phase 1 Trial Design for AMR-001
NASDAQ: NBS | 34 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 fraction ≤ 50% 96 hour
Slide 35: Ex Vivo Expanded Human Tregs Show Safety and Potential Efficacy in Early Clinical Trials
NASDAQ: NBS | 35 T effector to nTreg ratio CD4 SS C CD25 CD 1 2 7 Post-sort nTreg : >90% P re -s o rt P o st -s o rt Ex vivo Expanded Human Tregs Show Safety and Potential Efficacy in Early Clinical Trials