Slide 1: Transforming Personalized Medicine
1 Corporate Presentation NASDAQ: NBS Transforming Personalized Medicine David J. Mazzo, PhD Chief Executive Officer March 2015
Slide 2: Forward-looking statements
Forward-looking statements 2 This presentation contains “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 m
Slide 3: NeoStem financial metrics
NeoStem financial metrics 3 MARKET METRICS MARKET CAPITALIZATION1 $150.0 M STOCK PRICE2 $3.90 52 WEEK RANGE2 $3.08 - $7.75 FLOAT1 33.7M INSIDER HOLDINGS1 12.1% FINANCIAL METRICS REVENUE3 $17.9M (FY 2014) CASH4 $26.3M COMMON SHARES OUTSTANDING1 38.3 M WARRANTS1 3.5 M (avg. warrant exercise price of $
Slide 4: Transforming cells into therapies
Transforming cells into therapies 4
Slide 5: NeoStem's PCT: Industry-recognized single source premier services
NeoStem’s PCT: Industry-recognized single source premier services Center of excellence for process development, engineering and manufacturing 5 PRODUCT & PROCESS DEVELOPMENT MANUFACTURING CELL & TISSUE PROCESSING LOGISTICS STORAGE & DISTRIBUTION EXPERT CONSULTATION & REGULATORY SUPPORT ENGINEERING &
Slide 6: At a glance
At a glance 6 Highly experienced management and scientific team Proprietary platform technologies yielding a diversified, balanced pipeline targeting critical unmet needs in large global markets and near-term development milestones Immunotherapy platform applicable across multiple solid tumors with
Slide 7: Experienced executive team
Experienced executive team 7 David J. Mazzo, PhD Chief Executive Officer Over 30 years experience in all aspects of large and emerging global biotech/biopharma company operations and successful international drug development Robert S. Vaters, MBA President and Chief Financial Officer Over 25 years f
Slide 8: Robust, diversified and balanced pipeline
Robust, diversified and balanced pipeline 8 PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Acute myocardial infarction - STEMI : NBS10 Type I diabetes: NBS03D Metastatic melanoma: NBS20 (USAN = eltrapuldencel-T) Fast Track & Orphan Drug Designations with Special Protocol Assessment Granted
Slide 9: Robust, diversified and balanced pipeline
Robust, diversified and balanced pipeline 9 PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Metastatic melanoma: NBS20 (USAN = eltrapuldencel-T) Acute myocardial infarction - STEMI : NBS10 Type I diabetes: NBS03D
Slide 10: NBS20: Fast Track, Orphan Drug, Special Protocol Assessment
10 Fast Track designation Orphan Drug designation Special Protocol Assessment NBS20: (USAN = eltrapuldencel-T) Metastatic melanoma
Slide 11: Stage III recurrent/stage IV metastatic melanoma
Numerous non-synonymous mutations • Unique patient-specific antigenic fingerprints • Ideal target for autologous immunotherapy Stage III recurrent/stage lV metastatic melanoma 11 Brain Lung Bone Liver 1. American Cancer Society, 2014 SEER 2. For Stage IV metastatic melanoma - AJCC Cancer Staging 201
Slide 12: Current metastatic melanoma therapies insufficient
Current metastatic melanoma therapies insufficient 12 CURRENTLY APPROVED THERAPIES 2 Year Survival 5 Year Survival DTIC (GENERIC) JCO 1999, JCO 2000 9-20% 4% INTERLEUKIN-2 (PROMETHEUS LABS) JCO 2002 25% 15% IPILIMUMAB (BMS) NEJM 2010 24% 20% ANTI-PD11 (BMS AND MERCK) Clin Oncol 2014 40-50% N/A VEMUR
Slide 13: Preparation of NBS20 - stimulating immune response
13 Process times are approximate Isolation of cancer initiating cells Expansion and irradiation Incubate with irradiated tumor _ cells for antigen presentation Dendritic cell production Leukapheresis Monocyte isolation Surgical excision NBS20 uniquely targets cancer initiating cells by leveraging th
Slide 14: Phase 2: multiple trials; consistent, compelling data
0% 20% 40% 60% 80% 100% 0 10 20 30 40 50 60 P e rc ent S u rv iv in g Months Phase 2: multiple trials; consistent, compelling data 14 Dillman, et al. Journal Immunotherapy 2012 Historical control for distant metastases 0% 20% 40% 60% 80% Control Group Irradiated Tumor Cells Treatment Group 2-YEAR OV
Slide 15: The Intus study: phase 3 with SPA
The Intus study: phase 3 with SPA and orphan drug and fast track designations 15 DESIGN Single randomized, double blind, placebo controlled trial for stage III recurrent or stage IV metastatic melanoma for registration PRIMARY ENDPOINT Overall survival POWERING 80% power to detect 37.5% reduction in
Slide 16: Timeline to BLA
Timeline to BLA 16 1Q 2015 Initiate Screening 4Q 2017 Interim Results after 99 Deaths 2Q 2015 First Patient Randomized 2Q 2018 Projected BLA Submission based on successful interim analysis 2Q 2019 Final Study Results 4Q 2019 Projected BLA Submission 4Q 2016 Enrollment Completed 2015 2016 2017 2018 2
Slide 17: Multi-billion dollar lifecycle opportunity
Multi-billion dollar lifecycle opportunity Potential application across multiple solid tumor types 17 LUNG CANCER (Feasibility of cell lines from biopsies initiated) COLON CANCER (Feasibility of cell lines planned) OVARIAN CANCER (US FDA approved phase 2 protocol, cell line feasibility established)
Slide 18: Robust, diversified and balanced pipeline
Robust, diversified and balanced pipeline 18 PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Acute myocardial infarction - STEMI : NBS10 Type 1 diabetes: NBS03D Metastatic melanoma: NBS20 (USAN = eltrapuldencel-T)
Slide 19: NBS10: ST segment elevation myocardial infarction (STEMI)
19 NBS10: ST segment elevation myocardial infarction (STEMI)
Slide 20: Acute myocardial infarction (STEMI)
Acute myocardial infarction (STEMI) 20 1. Pedersen, Journal of the American College of Cardiology, 2014 and NeoStem modeling PREVALENCE AND UNMET MEDICAL NEED STEMI patients are at high risk of progressive deterioration in heart muscle function leading to Major Adverse Cardiac Events (MACE) ~65,000
Slide 21: NBS10 – Leveraging the body's natural repair mechanism
NBS10 – Leveraging the body’s natural repair mechanism to preserve heart muscle function 21 CD34/CXCR4 from bone marrow CD34 cell laying down new blood vessels CD34/CXCR4 SDF gradient CD34+ cells have been shown to induce the development of new blood vessels, preventing tissue death by improving blo
Slide 22: PreSERVE study: enrolled phase 2 study in follow-up
PreSERVE study: enrolled phase 2 study in follow-up 22 DESIGN Randomized (1:1), Phase 2, double blind, placebo controlled trial for post-AMI (STEMI) patients PRIMARY ENDPOINTS AND KEY SECONDARY ENDPOINT Change in cardiac perfusion from baseline to 6 months (exploratory) Incidence rates of SAEs and M
Slide 23: PreSERVE 12-month interim conclusions
PreSERVE 6 & 12-month interim conclusions* 23 CD34 cell dose-dependent trend in reduction of MACE — Signal for a mortality benefit (12 month data) Signal for reduction in frequency of SAEs in higher dose groups (12 month data) CD34 cell dose-dependent trend in improvement of left ventricular e
Slide 24: PreSERVE: MACE incidence
PreSERVE: MACE incidence (median follow-up 12 mos) 24 CD34 DOSE-DEPENDENT TREND IN REDUCTION OF MACE 14% 17% 10% 7% SIGNAL FOR A MORTALITY BENEFIT MACE= Death, MI, CHF Hospitalization, Revascularization. Control N=83 <14M N=47 >14M N=31 >20M N=15 0% 1% 2% 3% 4% 5% N=83 3.6% 0% (N=0) Pooled Treatment
Slide 25: PreSERVE: SAE incidence
1.2 1.1 0.6 0.7 0 0.2 0.4 0.6 0.8 1 1.2 1.4 PreSERVE: SAE incidence (median follow-up 18 mos) 25 SIGNAL FOR REDUCTION IN FREQUENCY OF SAES IN HIGHER DOSE GROUPS P ropo rtion of e ve n ts (n o rmali zed b y to tal s u b je cts ) P = 0.059 First time dose response demonstrated in this patient populati
Slide 26: PreSERVE: LVEF change from baseline
PreSERVE: LVEF change from baseline at 6 mos. 26 4.9% 3.1% 5.8% 10.2% Control <14M >14M >20M CD34 CELL DOSE-DEPENDENT TREND IN IMPROVEMENT OF LVEF LVE F chan g e f rom baseline ( % ) P < 0.05 First time dose response demonstrated in this patient population
Slide 27: PreSERVE: Infarct size change
-24% -16% -35% -41% -50% -40% -30% -20% -10% 0% PreSERVE: Infarct size change (baseline to 6 mos.) 27 Control <14M >14M >20M CD34 CELL DOSE-DEPENDENT TREND IN REDUCTION OF INFARCT SIZE % Me a n c ha n g e f rom baseli n e First time dose response demonstrated in this patient population
Slide 28: Next steps for ischemic repair program
Next steps for ischemic repair program 28 NEXT POTENTIAL DEVELOPMENT STEPS IN OTHER INDICATIONS • Phase 2 in chronic heart failure • Phase 2 in critical limb ischemia STEMI NEXT DEVELOPMENT STEPS • March 2015: • 2H 2015: • March 2016: • March 2017: One-year data Determine next development steps base
Slide 29: Multi-billion dollar lifecycle opportunity
Multi-billion dollar lifecycle opportunity Potential application across several cardiovascular indications 29 CHRONIC HEART FAILURE CRITICAL LIMB ISCHEMIA
Slide 30: Robust, diversified and balanced pipeline
Robust, diversified and balanced pipeline 30 PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Acute myocardial infarction - STEMI : NBS10 Type I diabetes: NBS03D Metastatic melanoma: NBS20 (USAN = eltrapuldencel-T)
Slide 31: NBS03D: Diabetes Mellitus Type-1 (T1D)
31 NBS03D: Diabetes Mellitus Type-1 (T1D)
Slide 32: Diabetes Mellitus Type-1: an autoimmune disease
Diabetes Mellitus Type-1 (T1D): an autoimmune disease 32 1. Hamman RF, et al. Diabetes Care. 2014; Sosenko JM, et al. Diabetes Care. 2008; Palmer JP. Diabetes/metabolism research and reviews. 2009 2. The DIAMOND Project Group. Diabetic Medicine. 2006;23:857-866. PREVALENCE AND UNMET MEDICAL NEED • 1
Slide 33: Preparation of NBS03D – restoring immune balance
33 NORMAL IMMUNE SYSTEM: IMMUNE BALANCE TREG INFUSION T regulatory cells T effector cells Blood draw from patient Manufacturing including expansion Infusion of Treg therapy to patient Preparation of NBS03D – restoring immune balance and function INFUSION of TREGs BALANCE REGAINED AUTOIMMUNITY: IMMUN
Slide 34: Regulatory T Cell therapy appears safe and well tolerated in adults
Regulatory T Cell therapy appears to be safe and well tolerated in adults1 34 1. Gitelman et al, American Diabetes Association Abstract, 2014 DESIGN U.S. UCSF/Yale open label Phase 1 study, 4-dose escalation cohorts PATIENTS 14 adult patients with established T1D RESULTS • Preliminary data indicates
Slide 35: Regulatory T Cell therapy preserves beta cell function in children
0 0.2 0.4 0.6 0.8 1 1.2 Day O Month 4 One Year 0% 20% 40% 60% 80% 100% Control Group Treatment Group Regulatory T Cell therapy preserves beta cell function in children1 35 Marek-Trzonkowska, N t al. Clinical Immunology 2014 1. Children aged 5-18 administered 1 (10 or 20 mil cells/kg) or 2 doses (tot
Slide 36: The Trutina study: phase 2 in adolescents with T1D
The Trutina study: phase 2 in adolescents with T1D1 36 DESIGN Double blind, placebo controlled, randomized (1:1:1) for adolescent patients with recent onset T1D ages 12 to 18 PRIMARY ENDPOINT Preservation of C-peptide at 52 weeks in comparison to placebo POWERING 80% power to detect 50% attenuation
Slide 37: Trutina study timeline
Trutina study timeline 37 Y1 Month 1 First subject first visit Year One Year Two Year Three Year Four Year Five Y1 Month 2 First subject randomized Y2 Q2 51st subject randomized* Y3 Q2 Last subject randomized Y3 Q3 Interim analysis topline results Y4 Q3 One-year topline results Y5 Q3 Two-year toplin
Slide 38: Multi-billion dollar lifecycle opportunity
Multi-billion dollar lifecycle opportunity Potential application across multiple autoimmune and allergic diseases 38 STEROID RESISTANT ASTHMA MULTIPLE SCLEROSIS (MS) CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD) INFLAMMATORY BOWEL DISEASE GRAFT VS. HOST DISEASE
Slide 39: Investment summary
Investment summary 39 Highly experienced management and scientific team Proprietary platform technologies yielding a diversified, balanced pipeline targeting critical unmet needs in large global markets and near-term development milestones Immunotherapy platform applicable across multiple soli
Slide 40: Transforming Personalized Medicine – Contact
Corporate Presentation NASDAQ: NBS March 2015 Transforming Personalized Medicine Contact: Eric Powers, Manager of Communications & Marketing Phone: 212.584.4173 Email: epowers@neostem.com Web: www.neostem.com