Slide 1: Investor Presentation
Investor Presentation NYSE MKT: NBS March 2013
Slide 2: Forward-Looking Statements
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 factors which may
Slide 3: Unsustainable Growth in US Health Care Costs
Unsustainable Growth in US Health Care Costs 3 • $2.7 Trillion Dollars is spent annually on health care costs (currently 18% of US GDP)1 • 80% of health care costs are associated with chronic conditions2 o Cardiovascular disease costs over $445B today Projected to increase to over $1T by 20303 o Dia
Slide 4: Regenerative Medicine
Regenerative Medicine • Repairing or replacing damaged tissue and restoring function • Novel regenerative therapies hold the promise of transforming clinical outcomes and reducing overall healthcare costs • The regenerative medicine market is estimated to grow to $88 billion by 20141 4 1) According
Slide 5: NeoStem is Built for Success in Regenerative Medicine
• Dynamic, experienced and nimble management team • Recognized, state-of-the art contract development and manufacturing organization (East and West coast operations) • Advancing clinical and preclinical pipeline of cell therapies o Cardiovascular disease o Autoimmune disorders o Regenerative medicin
Slide 6: Transaction Timeline Through 2012
Acquired Stem Cell Technologies, Inc. Acquired CBH (51% of Erye) Acquired Acquired Divested Began partnership with Vatican Phase III Medical Acquired NeoStem, Inc. Transaction Timeline Through 2012 Accessed $120 Million 80% Acquired 6 2006 2007 2009 2010 2011 2012
Slide 7: Deloitte's 2012 Technology Fast 500 - #7 Nationwide
7 Deloitte's 2012 Technology Fast 500™ - #7 Nationwide NeoStem Ranked #1 Fastest Growing Company in the New York Tri-State Region 1. Tesla Motors 2. Palo Alto Networks 3. Sagent Pharmaceuticals, Inc. 4. FireEye, Inc. 5. Aerohive Networks, Inc. 6. Avail-TVN 7. NeoStem Technology Fast 500™, conducted
Slide 8: Developing Therapies on a Foundation of Manufacturing Expertise
Autoimmune Disorders Regenerative Medicine NeoStem develops therapies for chronic unmet medical needs around a significant IP portfolio… Developing Therapies on a Foundation of Manufacturing Expertise Cardiovascular Disease …and operates a revenue generating service division with expertise in contra
Slide 9: Cochrane Collaboration Review: Bone Marrow Derived Cells
Cochrane Collaboration Review Bone Marrow Derived Cells: Likely Safe and Positive Impact on Mortality Clifford et al, Cochrane Library 2012 9
Slide 10: AMR-001 Brings Repair System to the Heart
AMR-001 Brings Repair System to the Heart in Order to Preserve Function CD34⁺CXCR4+ Cells are a natural repair mechanism 10
Slide 11: The Peri-Infarct Zone Becomes the Infarct
• A consequence of inadequate perfusion (microvascular insufficiency) is apoptosis and progressive cardiomyocyte loss in the peri-infarct zone, leading to infarct expansion • STEMI patients are at risk of a progressive deterioration in heart muscle function that leads to arrhythmia, recurrent myocar
Slide 12: 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 13: Phase 1 Trial Results Summary
Phase 1 Trial Results Summary 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 -20.9 DSMB determined that no adverse events were related to therapy 13 Patients d
Slide 14: Effect of AMR-001 on Left Ventricular Function
Effect of AMR-001 on Left Ventricular Function: Results of Phase 1 Trial Quyyumi AmHtJ 2011 and data on file 14 0 20 40 60 Control 5 10 15 Treatment Group (control vs. millions of CD34+ cells) % of individuals with decrease in left ventricular ejection fraction Threshold
Slide 15: PreSERVE-AMI Phase 2 Study
PreSERVE-AMI Phase 2 Study 15 Indication Post-AMI preservation of cardiac function Design Double blinded, placebo controlled, randomized (1:1) Primary Endpoint Change in cardiac perfusion (RTSS by SPECT) from baseline to 6 months Other Endpoints Secondary endpoints to determine preservation of cardi
Slide 16: Expansion of Intellectual Property
Expansion of Intellectual Property • Broad and growing patent portfolio supports cardiac and other ischemic conditions • 3 issued US composition of matter and methods patents: • U.S. 7,794,705: Issued 9/14/2010. Indication: Cardiac: Post AMI early and late • U.S. 8,088,370: Issued 1/3/2012. Indicati
Slide 17: Scientific Advisory Board
Scientific Advisory Board 17 Andrew L. Pecora, MD, FACP, CPE SAB Administrative Chairman Chief Medical Officer, NeoStem Hackensack University Medical Center Eugene Braunwald, MD, FRCP Brigham & Women’s Hospital Bernard J. Gersh, MD, ChB, DPhil, FRCP The Mayo Clinic Dean J. Kereiakes, MD, FACC The Ch
Slide 18: What's Next? Congestive Heart Failure
What’s Next? Congestive Heart Failure US: Incidence – 660,000, Prevalence 5.8 million1 Worldwide: Prevalence – 20-23 million2 18 1) American Heart Association 2) Study by Case Western Reserve University
Slide 19: Intracoronary Delivery of CD34+ Stem Cell: Durable to 5 Years
Intracoronary Delivery of CD34+ Stem Cell Shows That Improvement in Physiologic and Clinical Status is Durable (to 5 years) Adapted from Vrtovec et al, Circ Res published online Oct 12, 2012 Stem Cell Group Controls 19
Slide 20: CD34+ Stem Cell Therapy Yields Clinical Benefits in DCM
CD34+ Stem Cell Therapy Yields Meaningful Clinical Benefits in DCM Adapted from Vrtovec et al, Circ Res published online Oct 12, 2012 Stem Cell Group Controls 20
Slide 21: Treg Cells to Restore Immune Balance
Treg Cells to Restore Immune Balance • Partnership with Becton Dickinson, which owns 20% of Athelos • Immune-mediated diseases, such as GVHD, autoimmune disorders and allergic conditions, are a result of an imbalance between T-effector cells and T-regulatory cells (Treg) • Treg therapy represents a
Slide 22: Ex vivo Expanded Human Tregs Show Safety and Potential Efficacy
Ex vivo Expanded Human Tregs Show Safety and Potential Efficacy in Early Clinical Trials CD4 SS C CD25 CD 1 2 7 Post-sort nTreg : >90% T effector to nTreg ratio P re -s o rt P o s t- s o rt 22
Slide 23: Published Treg Clinical Trials
Published Treg Clinical Trials • Trzonkowski et al., First-in-man clinical results of the treatment of patients with graft versus host disease with human ex vivo expanded CD4+CD25+CD127- T regulatory cells . Clin. Immunol. 2009 • Di Ianni et al., Tregs prevent GVHD and promote immune reconstitution
Slide 24: Open Treg Trials
Open Treg Trials • Laport and Negrin, Stanford (NCT01660607,unpublished pers comm) Phase I/II MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell • Gitelman and Bluestone, UCSF (NCT01210664, unpublished pers comm) T1DM Immunotherapy Using CD4+CD127lo/-CD25+ Poly
Slide 25: Scientific Advisory Board
Scientific Advisory Board 25 Robert A. Preti, PhD, SAB Administrative Chairman Progenitor Cell Therapy Jeffrey Bluestone, PhD University of California, San Francisco, Diabetes Center David A. Horwitz, MD University of Southern California Robert Korngold, PhD Hackensack University Medical Center Robe
Slide 26: VSELs – Adult Stem Cells
VSELs – Adult Stem Cells heart neuron pancreas VSEL™ • Very small embryonic-like (VSELsTM) stem cells are believed to be naturally pluripotent • Animal models have demonstrated that highly enriched human VSELs are able to integrate, differentiate and potentially regenerate • Treatment indications be
Slide 27: Human VSELs Accelerate Healing in a Mouse Wound Model
Human VSELs Accelerate Healing in a SCID Mouse Complex Tail Wound Model Days Post-wounding VSELs vs. MSCs % R e -e p it h e li a li z a ti o n p < 0.05 2,500 human VSELS 500,000 human MSCs Fibrin Control 27
Slide 28: Human VSELs Injected into Mouse Sub-Retinal Space Integrate
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 Human VSELs Injected into a Mouse Sub- Retinal Space Integrate and Show Differentiation Potential in situ 28 Eminli, S. et
Slide 29: Academic Collaborators
Academic Collaborators 29 Vincent Falanga, MD Roger Williams Medical Center Kameran Lashkari, MD Schepens Eye Institute, Harvard Medical School Song Li, PhD University of California, Berkeley Mariusz Ratajczak, MD, PhD, DSci University of Louisville Russel Tacihman, DMD, DMSc University of Michigan
Slide 30: Progenitor Cell Therapy
30 Progenitor Cell Therapy Development and delivery of high quality, cost-efficient, and effective therapeutics can be leveraged by state-of-the-art manufacturing and regulatory expertise
Slide 31: Facilities and 15 Year Track Record of Success
Mountain View, California (25,000 ft2) ISO Class 7 / Class 10,000 suites Allendale, New Jersey (30,000 ft2) ISO Class 7 / Class 10,000 suites ISO Class 6 / Class 1,000 suite 31 • cGMP/GLP Accredited and Certified Facilities • Experience • Over 100 Clients Served and Growing • 30,000 Products Manufac
Slide 32: Business Model & Capabilities
• Large and small companies in the cell therapy space outsource services for all or part of their manufacturing needs to improve efficiencies and profitability and to reduce capital investment: • PCT supports NeoStem’s cell therapy development programs with o Lower costs for internal cell therapy de
Slide 33: Contract Engagement Types and Pricing
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 12 to 18 Month En
Slide 34: NeoStem Key Executives
NeoStem Key Executives Robin Smith, MD, MBA CEO & Chairman of the Board MD – Yale; MBA – Wharton 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 Diseases (through November
Slide 35: NeoStem Board of Directors
NeoStem Board of Directors Robin Smith, MD, MBA CEO & Chairman of the Board MD – Yale; MBA – Wharton 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 Lif
Slide 36: Key Financial Metrics (as of December 31, 2012)
Key Financial Metrics (as of December 31, 2012) 36 2012 Revenue $14.3m (43% increase over 2011) Cash $13.7m Total Stock and Equivalent Shares Outstanding Common Shares1 162.7m Warrants 54.7m (avg. warrant exercise price of $1.58) Options 21.7m (avg. option exercise price of $1.29) 36
Slide 37: Achievements Since March 2012
• Divested of Suzhou Erye, adding $12.3m to the balance sheet and removing over $33m of debt • Received positive 6 and 12 month DSMB safety evaluations for Phase 2 PreSERVE clinical trial • Expanded global breadth of Amorcyte IP to all areas of vascular insufficiency • Increased PCT annual revenues
Slide 38: NeoStem Milestones
Product/Service Expansion Transaction(s) NeoStem Milestones Q1-13 Q2-13 Q3-13 Q4-13 Q1-14 Q2-14 Q3-14 Q4-14 C o mme rc ia l O p e rat io n s T h e ra p e u ti c P ip e li n e Manufacturing Expansion into Europe Complete Phase 2 STEMI Trial with 6 Month Data Readout Commence Enrollment Phase 3 STEMI
Slide 39: Why Invest Now?
Why Invest Now? • Proven track record of success with leadership that can execute • Exciting proprietary cell therapy pipeline with near term data • Strong IP portfolio • Growth in revenue generating contract development and manufacturing business with plans for further expansion 39
Slide 40: Contact Information
Contact Information NeoStem, Inc. NYSE MKT: NBS www.neostem.com Robin Smith, MD, MBA Chairman & CEO Phone: (212) 584-4174 Email: rsmith@neostem.com v6 40