Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (August 2013)
44 slides from Lisata Therapeutics's investor presentation, Aug 8, 2013
Slide 1: Investor Presentation: Transforming how we treat chronic disease
NASDAQ: NBS | 1 Investor Presentation NASDAQ: NBS August 2013 Transforming how we treat 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: Unsustainable Growth in US Health Care Costs
NASDAQ: NBS | 3 Unsustainable Growth in US Health Care Costs • $2.7 Trillion spent annually on health care1 • 80% associated with chronic conditions2 o Cardiovascular disease: $445B+ today, $1T+ by 20303 o Diabetes: $174B+ today, $300B+ by 20254 We need to move paradigm from treatment of chronic dis
Slide 4: Regenerative Medicine
NASDAQ: NBS | 4 Regenerative Medicine • Repair or replace damaged tissue and restore function • Novel regenerative therapies that hold possibility to: Improve clinical outcomes Reduce overall healthcare costs
Slide 5: Regenerative Medicine Platform
NASDAQ: NBS | 5 Regenerative Medicine Platform Autoimmune Disorders Cardiovascular Disease NeoStem Develops Therapies for Chronic Unmet Medical Needs • Significant IP Portfolio… • …and a revenue generating service division CDMO TM
Slide 6: The Revenue Side of the Business
NASDAQ: NBS | 6 The Revenue Side of the Business…
Slide 7: Progenitor Cell Therapy
NASDAQ: NBS | 7 Progenitor Cell Therapy • Recognized contract development and manufacturing organization (East and West Coast operations) • Development and delivery of high quality, cost-efficient, and effective therapeutics can be leveraged by state-of-the-art manufacturing and regulatory expertise
Slide 8: 15 Year Track Record of Success
NASDAQ: NBS | 8 Experience: • 100+ clients served and growing • 30+ tech transfers • 30,000+ products manufactured • 18,000+ products stored • 14,000+ products shipped for clinical use • 50+ US and EU regulatory filings successfully completed PCT is the only contract manufacturing organization to ha
Slide 9: Facilities: 15 Year Track Record of Success
NASDAQ: NBS | 9 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 • cGMP/GLP accredited and certified Facilities 15 Year Track Record of Success • Ability to serve clients and
Slide 10: Manufacturing Growth Plans
NASDAQ: NBS | 10 • Establish opportunities for early partnering relationships with goals of commercial manufacturing, equity participation and back-end royalties • Automation initiatives focused on lowering cost of goods and increasing gross profits • Initiatives being pursued to expand commercial m
Slide 11: Contract Manufacturing Engagement Types and Pricing
NASDAQ: NBS | 11 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 12: Building the Best in Class Cell Therapy Pipeline
NASDAQ: NBS | 12 Building the Best in Class Cell Therapy Pipeline…
Slide 13: Built for Success in Regenerative Medicine
NASDAQ: NBS | 13 Cardiovascular disease Autoimmune disorders Regenerative medicine platform Built for Success in Regenerative Medicine • Acute myocardial infarction – Phase 2 • Congestive heart failure – Preparing for Phase 1b/2a • Traumatic brain injury – Preclinical • Type 1 diabetes – Phase 2 rea
Slide 14: High Cost of Cardiovascular Disease
NASDAQ: NBS | 14 High Cost of Cardiovascular Disease $2.7 trillion dollars is spent annually on health care costs, currently 18% of US GDP Cardiovascular disease costs over $445 billion today, Projected to increase to $1 trillion by 2030
Slide 15: AMR-001 Brings Repair System to the Heart
NASDAQ: NBS | 15 AMR-001 Brings Repair System to the Heart in Order to Preserve Function After a STEMI CD34⁺CXCR4+ Cells are a natural repair mechanism
Slide 16: The Peri-Infarct Zone Becomes the Infarct
NASDAQ: NBS | 16 • A consequence of inadequate perfusion (microvascular insufficiency) after a heart attack 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 tha
Slide 17: Phase 1 Trial Design for AMR-001
NASDAQ: NBS | 17 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
Slide 18: Phase 1 Trial Results Summary
NASDAQ: NBS | 18 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 Patients dosed ≥ the thres
Slide 19: Effect of AMR-001 on Left Ventricular Function
NASDAQ: NBS | 19 Effect of AMR-001 on Left Ventricular Function: Results of Phase 1 Trial Quyyumi AmHtJ 2011 and data on file 0 20 40 60 0 5 10 15 CD34+ cells (millions) % of individuals with decrease in left ventricular ejection fraction (LVEF) Threshold No individuals showed a decrease in LVEF
Slide 20: PreSERVE-AMI Phase 2 Study
NASDAQ: NBS | 20 PreSERVE-AMI Phase 2 Study 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 preserv
Slide 21: Intellectual Property
NASDAQ: NBS | 21 Intellectual Property • Broad and growing patent portfolio supports cardiac and other ischemic conditions • 4 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. Indi
Slide 22: Scientific Advisory Board
NASDAQ: NBS | 22 Scientific Advisory Board Andrew L. Pecora, MD, FACP, CPE SAB Administrative Chairman Chief Scientific Officer, Amorcyte Hackensack University Medical Center Eugene Braunwald, MD, FRCP Brigham & Women’s Hospital Bernard J. Gersh, MD, ChB, DPhil, FRCP The Mayo Clinic Dean J. Kereiake
Slide 23: What's Next? Congestive Heart Failure
NASDAQ: NBS | 23 What’s Next? Congestive Heart Failure CHF 660,000 5,800,000 American Heart Association 23,000,000 20,000,000 Case Western University Incidence Prevalence Prevalence
Slide 24: CD34+ Stem Cell Therapy in Dilated Cardiomyopathy
NASDAQ: NBS | 24 CD34+ Stem Cell Therapy Yields Meaningful Clinical Benefits in Dilated Cardiomyopathy Adapted from Vrtovec et al, Circ Res published online 10/12/12
Slide 25: Treg Cells to Restore Immune Balance
NASDAQ: NBS | 25 Treg Cells to Restore Immune Balance • Partnership with Becton Dickinson (20% ownership of Athelos) • Immune-mediated diseases, such as graft-versus-host-disease (GVHD), autoimmune disorders, such as type 1 diabetes and multiple sclerosis, and allergic conditions, are a result of an
Slide 26: Treg Cells to Restore Immune Balance (cont.)
NASDAQ: NBS | 26 Treg Cells to Restore Immune Balance (cont.) • Ongoing collaboration with Drs. Jeffrey Bluestone and Qizhi Tang (UCSF) for treatment of: • type 1 diabetes • steroid resistant asthma • organ transplant rejection • Exclusive rights to more than 20 issued patents covering: • isolation
Slide 27: Scientific Advisory Board
NASDAQ: NBS | 27 Scientific Advisory Board 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 Korngold, PhD Hackensack Univ
Slide 28: Regenerative Medicine Potential: VSELs
NASDAQ: NBS | 28 • Our early stage research has identified cells in human blood and bone marrow that show evidence of the potential for multilineage differentiation (very small embryonic-like (VSELTM) stem cells) • Preliminary data in pre-clinical animal models have indicated that highly enriched hu
Slide 29: Regenerative Medicine Potential: Grants and Patents
NASDAQ: NBS | 29 • Pre-clinical work financed largely by grants and DOD funding • NeoStem has 7 families of patents pending for method of treatment claims that dovetail with the indications that we are pursuing o Total Active Grants Awarded: $4,596,676 o Total Grants Pending: $150,000 o Additional g
Slide 30: Human VSELs Accelerate Healing in a SCID Mouse Wound Model
NASDAQ: NBS | 30 Human VSELs™ Accelerate Healing in a SCID Mouse Complex Tail Wound Model Preliminary data suggest that VSELs™ may be more effective in accelerating healing than mesenchymal stromal cells in a preclinical model of severe complex wounds % R e -ep it he lia liz at io n p < 0.05 Days Po
Slide 31: Human VSELs Injected into Mouse Sub-Retinal Space
NASDAQ: NBS | 31 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 Preliminary Data Suggest Human VSELs™ Injected into a Mouse Sub-Retinal Space Integrate and Show Differen
Slide 32: Academic Collaborators
NASDAQ: NBS | 32 Academic Collaborators Vincent Falanga, MD Boston University Kameran Lashkari, MD Schepens Eye Institute, Harvard Medical School Song Li, PhD University of California, Berkeley Mariusz Ratajczak, MD, PhD, DSci University of Louisville Russell Tacihman, DMD, DMSc University of Michig
Slide 33: Key Executives
NASDAQ: NBS | 33 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
Slide 34: Board of Directors
NASDAQ: NBS | 34 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 Ste
Slide 35: Key Metrics
NASDAQ: NBS | 35 Key Metrics Market Metrics Market Capitalization(1) $150M Enterprise Value(1) $138M Current Price(1) $7.40 52 Week Range(1) $5.00 - $8.90 Float(1) 17.1M Insider Holdings(2) 15.8% Institutional Holdings(2) 6.6% 1) As of August 7, 2013, based on 20.3 million shares outstanding 2) As o
Slide 36: Over Past 7 Years Accessed $134 Million and Completed 5 M&A Transactions
NASDAQ: NBS | 36 80% Acquired Acquired Stem Cell Technologies, Inc. Acquired CBH (51% of Erye) Acquired Acquired Divested Began partnership with Vatican Phase III Medical Acquired NeoStem, Inc. Over Past 7 Years Accessed $134 Million and Completed 5 M&A Transactions and 1 Divestiture 2006 2007 2009
Slide 37: Goal: Global Leader in Cell Therapy Regenerative Medicine
NASDAQ: NBS | 37 Goal Global Leader in Cell Therapy Regenerative Medicine Largest Cell Therapy Contract Manufacturer Global Leader in the Treatment of Ischemic Diseases Key Player in the Treatment of Autoimmune Diseases Proprietary Technology Platform / Market Changer Present Set Stage for Growth; N
Slide 38: NeoStem Milestones
NASDAQ: NBS | 38 • Therapeutic Pipeline o Complete enrollment PreSERVE-AMI Phase 2 trial o 1st data readout 6-8 months after last patient enrolled o Progress towards Phase 1b/2a AMR-001 CHF trial o Progress towards VSELTM human bone growth trials o Progress Treg cell program towards Phase 2 trial in
Slide 39: Contact Information
NASDAQ: NBS | 39 Contact Information NeoStem, Inc. NASDAQ: NBS www.neostem.com Robin Smith, MD, MBA Chairman & CEO Phone: (212) 584-4174 Email: rsmith@neostem.com v4
Slide 40: High Prevalence of Ischemic Heart Disease
NASDAQ: NBS | 40 High Prevalence of Ischemic Heart Disease Leading cause of death in the world Nearly 2X the next highest cause of death in high-income countries
Slide 41: Intracoronary Delivery of CD34+ Stem Cell Shows Durable Improvement
NASDAQ: NBS | 41 Intracoronary Delivery of CD34+ Stem Cell Shows Improvement in Physiologic and Clinical Status is Durable (to 5 years) Adapted from Vrtovec et al, Circ Res published online Oct 12, 2012 Controls Stem Cell Group
Slide 42: Ex vivo Expanded Human Tregs Show Safety and Potential Efficacy
NASDAQ: NBS | 42 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 st -s o rt
Slide 43: Open Treg Trials
NASDAQ: NBS | 43 Open Treg Trials • Laport and Negrin, Stanford (NCT01660607) Phase I/II MAHCT w/ TCell Depleted Graft w/Simultaneous Infusion Conventional and Regulatory T Cell (unpublished pers comm) • Gitelman and Bluestone, UCSF (NCT01210664) T1DM Immunotherapy Using CD4+CD127lo/- CD25+ Polyclon
Slide 44: Peer Reviewed Treg Publications
NASDAQ: NBS | 44 Peer Reviewed Treg Publications • 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 immu