Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (May 2013)
37 slides from Lisata Therapeutics's investor presentation, May 9, 2013
Slide 1: Investor Presentation
Investor Presentation NYSE MKT: NBS May 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 develops therapies for chronic unmet medical needs
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 6: AMR-001 Brings Repair System to the Heart in Order to Preserve Function
AMR-001 Brings Repair System to the Heart in Order to Preserve Function CD34⁺CXCR4+ Cells are a natural repair mechanism 6
Slide 7: 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 8: 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 8
Slide 9: 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 10: 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 10 Patients d
Slide 11: 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 11 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 12: PreSERVE-AMI Phase 2 Study
PreSERVE-AMI Phase 2 Study 12 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 13: Expansion of Intellectual Property
Expansion of 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. Indicati
Slide 14: Scientific Advisory Board
Scientific Advisory Board 14 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 15: 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 15 1) American Heart Association 2) Study by Case Western Reserve University
Slide 16: Intracoronary Delivery of CD34+ Stem Cell Shows Durable Improvement
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 16
Slide 17: CD34+ Stem Cell Therapy Yields Meaningful 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 17
Slide 18: 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 graft-versus-host-disease (GVHD), autoimmune disorders, such as type 1 diabetes and multiple sclerosis, and allergic conditions, are a result of an imbalance betwee
Slide 19: 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 19
Slide 20: 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 21: Open Treg Trials
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+ Polyclonal Tregs (unpubli
Slide 22: Scientific Advisory Board
Scientific Advisory Board 22 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 23: 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 24: Human VSELs Accelerate Healing in a SCID 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 24
Slide 25: Human VSELs Injected into Mouse Sub-Retinal Space Integrate and Differentiate
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 25 Eminli, S. et
Slide 26: Academic Collaborators
Academic Collaborators 26 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 27: Progenitor Cell Therapy
27 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 28: Manufacturing Facilities and Track Record
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 28 • cGMP/GLP Accredited and Certified Facilities • Experience with over • 100 Clients Served and Growing • 30,000 Products Ma
Slide 29: 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 30: 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 31: 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 32: 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 33: Key Financial Metrics
Key Financial Metrics 33 Revenue $2.5m (1Q 2013) Cash $9.3m (as of March 31, 2013) Additional Cash $10.7m (net proceeds from common stock offering in May 2013) Total Stock and Equivalent Shares Outstanding (as of May 9, 2013) Common Shares 193.8m Warrants 55.0m (avg. warrant exercise price of $1.56)
Slide 34: Accessed $130 Million and Completed 5 M&A Transactions
Acquired Stem Cell Technologies, Inc. Acquired CBH (51% of Erye) Acquired Acquired Divested Began partnership with Vatican Phase III Medical Acquired NeoStem, Inc. Accessed $130 Million and Completed 5 M&A Transactions To Date 80% Acquired 34 2006 2007 2009 2010 2011 2012
Slide 35: NeoStem Milestones
NeoStem Milestones 35 • 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 tri
Slide 36: NeoStem is Built for Success in Regenerative Medicine
• Dynamic, experienced and nimble management with a proven track record of success with leadership that can execute • Clinical and preclinical pipeline of cell therapies around a strong IP portfolio o Cardiovascular disease o Autoimmune disorders o Regenerative medicine • Recognized, state-of-the ar
Slide 37: 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 v7 37