Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — NeoStem — Investor Presentation (October 2013)
35 slides from Lisata Therapeutics's investor presentation, Oct 17, 2013
Slide 1: Investor Presentation: Transforming the Treatment of Chronic Disease
NASDAQ: NBS | 1 Investor Presentation NASDAQ: NBS October 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, impr
Slide 6: Our Contract Development and Manufacturing Organization (CDMO) Business
NASDAQ: NBS | 6 Our Contract Development and Manufacturing Organization (CDMO) Business
Slide 7: 15-Year Track Record of Success
NASDAQ: NBS | 7 • 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 8: Manufacturing Growth Plans
NASDAQ: NBS | 8 • 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 9: CDMO Contract Types and Pricing
NASDAQ: NBS | 9 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 1
Slide 10: Building a Best in Class Cell Therapy Pipeline
NASDAQ: NBS | 10 Building a Best in Class Cell Therapy Pipeline
Slide 11: Built for Success in Regenerative Medicine
NASDAQ: NBS | 11 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 12: High Cost of Cardiovascular Disease
NASDAQ: NBS | 12 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 13: AMR-001 Brings Repair System to the Heart
NASDAQ: NBS | 13 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 14: PreSERVE-AMI Phase 2 Trial Design
NASDAQ: NBS | 14 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 15: Phase 1 Results Point to AMR-001 Potential
NASDAQ: NBS | 15 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 16: Intellectual Property
NASDAQ: NBS | 16 • 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 17: What's Next? Congestive Heart Failure
NASDAQ: NBS | 17 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 18: CD34+ Stem Cell Therapy Improves Event Free Survival in Dilated Cardiomyopathy
NASDAQ: NBS | 18 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 Significantly Improves Event Free Survival at 5 Years in Patients with Dilated Cardiomyopathy Pump Failure Deaths Sud
Slide 19: Using Treg Cells to Restore Immune Balance
NASDAQ: NBS | 19 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 20: Recent Advancements in the Treg Program
NASDAQ: NBS | 20 • Type 1 diabetes affects over 34 million worldwide • Advancing to Phase 2 study in 2014 through collaboration with Drs. Jeffrey Bluestone and Qizhi Tang (UCSF) • Severe asthma affects 60 million worldwide • Designing protocol for Phase 1b/2a steroid resistant asthma study with Drs.
Slide 21: VSELs Regenerative Medicine Potential
NASDAQ: NBS | 21 • 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 22: Human VSELs Accelerate Healing in a SCID Mouse Wound Model
NASDAQ: NBS | 22 • 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 23: Human VSELs Injected into Mouse Sub-Retinal Space Show Differentiation Potential
NASDAQ: NBS | 23 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 24: Key Executives
NASDAQ: NBS | 24 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 25: Board of Directors
NASDAQ: NBS | 25 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 26: Key Metrics
NASDAQ: NBS | 26 Market Metrics Market Capitalization(1) $189M Enterprise Value(1) $177M Recent Price(2) $7.00 52 Week Range(2) $5.00 - $9.89 Float(1) 23.8M Insider Holdings(2) 11.6% 1) As of October 15, 2013, based on 26.9 million shares outstanding and a $7.00 share price 2) As of October 15, 2013
Slide 27: NeoStem Milestones
NASDAQ: NBS | 27 • 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 trials - Advancing Treg cell program towards Phase 2 trial in type 1 di
Slide 28: Contact Information
NASDAQ: NBS | 28 Contact Information NeoStem, Inc. NASDAQ: NBS www.neostem.com Robin Smith, MD, MBA Chairman & CEO Phone: (212) 584-4174 Email: rsmith@neostem.com
Slide 29: Appendix
Appendix 02. NeeStem NASDAQ: NBS | 29
Slide 30: Since 2006, Accessed Over $183 Million and Completed Multiple M&A Transactions
NASDAQ: NBS | 30 Since 2006, We Have Accessed Over $183 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 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 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 Hackensa
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
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