International Stem Cell Corporation · OTC:ISCO
International Stem Cell — Investor Presentation (January 2014)
22 slides from International Stem Cell's investor presentation, Jan 21, 2014
Slide 1: Cells for Therapy and Research
Cells for Therapy and Research ISCO.QB 1/21/2014 1
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation includes certain statements, estimates and projections with respect to the anticipated future business and performance of the Company, which are collectively referred to as forward-looking statements. Forward-looking statements reflect various assumptions
Slide 3: A Company with Substantial Revenues
RM Company with Substantial Revenues Stem cell platform technology targeting high value therapeutic indications: - First in man study in Parkinson’s disease - Secondary programs in metabolic liver disease and ophthalmology indications - Potential to unlock multi indications - Multiple partnering opp
Slide 4: Core Company Components
Core Company Components Stem Cell Human Cell Biomedical Cell Therapy Platform Manufacturing Products Programs • Unique Benefits • Research and Businesses • Large Unmet • Differentiated GMP grade • Strong Revenue Medical Needs • Multiple • Human Cells and Growth • Neural Stem Cells Indications Media
Slide 5: Platform Stem Cell Technology
Platform Stem Cell Technology Parthenogenesis. Human pluripotent stem cells from unfertilized eggs Clinically superior for allogeneic therapy Histocompatible cells (less rejection) Pluripotent One cell line can be used to treat millions Bypasses ethical concerns No viable embryo created or destroyed
Slide 6: Intellectual Property Investment
Intellectual Property Investment Large patent portfolio including stem cell creation and differentiation methods 130 patents and licenses in 14 families 90 patents pending in 5 families Significant peer-reviewed publications Nature’s Scientific Reports (2013) Cell Stem Cell (2011,2012) 1/21/2014 6
Slide 7: Manufacturing Capabilities
Manufacturing Capabilities Human cell manufacturing capabilities suitable for early-stage clinical trials 13,125 sq.ft laboratory including 5 GMP manufacturing suites Largest private cryobank of histocompatible human pSC 1/21/2014 7
Slide 8: R&D Targeting Multiple Diseases
R&D Targeting Multiple Diseases Validating the stem cell platform for allogeneic clinical therapy Program Target Pre-Clinical IND Track Phase I Human Neural Parkinson’s Stem Cells disease Hepatocyte-like Inherited cells metabolic liver diseases Corneal Corneal cell/tissue blindness Success unlocks s
Slide 9: Overview of Parkinson's Disease
Overview of Parkinson’s Disease 2nd most common neurodegenerative disease • 7-10 million people world-wide • 60k new case / year (Parkinson’s disease Foundation) • $2.5 billion in sales (2012) Oral medications Fetal tissue implants: • Dopamine replacement/ agonists • Ethically controversial • Blocke
Slide 10: hNSC for Allogeneic Therapy
hNSC for Allogeneic Therapy Self-renewing, multipotent human neural stem cells Derived from parthenogenetic stem cells 1. Practical, unlimited supply of pure cells 2. Not ethically controversial 3. May be less immunogenic Potential mechanism 1. Neuroprotection: Produce nerve growth factors that prot
Slide 11: hNSC Characterization and Manufacturing
hNSC Characterization and Manufacturing OOCYTE ICM STEM NSC DA CELL NEURON Scalable, low cost GMP protocol • Suitable for pre-clinical and clinical studies • Fully characterized for purity, safety and functionality Published in peer-reviewed literature 1/21/2014 11
Slide 12: hNSC Show Good Safety and Tolerability
hNSC Show Good Safety and Tolerability Body weight of mice and No visible tumors or pluripotent cells rats within normal ranges 27 Group 1: Mice Mean Body Weight (g) Vehicle 25 Group 2: 4k NSC 23 Group 3: 4k NSC 21 Group 4: 2k NSC 19 DAPI/STEM121/OCT4 17 Coronal section of the monkey brain Implantat
Slide 13: Primate Proof-of-Concept Data
Primate Proof-of-Concept Data Dopamine levels post mortem Stable Parkscores and Healthy Behavior for duration of the study Functional activity demonstrated in disease model (asymptomatic) primates 1/21/2014 13
Slide 14: World-class Partners
World-class Partners Dr. Mark Stacy, MD • Clinical PI Vice Dean for Clinical Research, Neurology at Duke • Neurologist University School of Medicine Dr. Evan Y. Snyder, MD PhD • Histology Chair of the FDA Advisory Committee for Cellular, Tissue and Gene Therapy • Data analysis UCSD and Director of S
Slide 15: Parkinson's Disease 2014 Targets
Parkinson’s Disease 2014 Targets Complete pre-clinical • Primate pharmacology, toxicology, and efficacy – Mid 2014 • Tumorigenicity study in SCID mice – Mid 2014 Submit IND and begin FIM study w/ Duke • Single arm, open label, dose escalating study • < 20 subjects with idiopathic PD, UPDRS < 50 • PI
Slide 16: Commercial Subsidiaries
Commercial Subsidiaries ISCO.QB 1/21/2014 16
Slide 17: Corporate Structure
Corporate Structure Core stem cell technology Intellectual Property Therapeutic Research and Development Biomedical research products Branded anti-aging skin care products Primary human cells Proprietary stem cell skin care Optimized cell culture media technology 1/21/2014 17
Slide 18: Commercial Operations
Commercial Operations Strong Revenue growth from Biomedical businesses 1.8 Quarterly / $million Annual / $million 1.7 7 1.6 6 1.5 5 1.4 4 1.3 3 1.2 2 1.1 1 1 0 2006 2008 2010 2012 2014 1/21/2014 18
Slide 19: Lifeline Cell Technology
Lifeline Cell Technology Vertically integrated human cell and media business • $2.3 billion world-wide market (bcc research 2009) • Over 130 cells and optimized culture media products • Branded and OEM products – Growing domestic sales force – International Distributors covering EU / Japan 1/21/2014
Slide 20: Lifeline Skin Care
Lifeline Skin Care Branded anti-aging stem cell skin care products Rosewood Four Seasons Resort Tucker’s Point Maui at Wailea Bermuda Marriott Palm Beach Jen Galardi Hollywood Fitness guru and yoga instructor Emme - Supermodel Woman of the year (Glamour Magazine) One of the 50 most beautiful people
Slide 21: A Company with Substantial Revenues
RM Company with Substantial Revenues Stem cell platform technology targeting high value therapeutic indications: - First in man study in Parkinson’s disease - Secondary programs in metabolic liver disease and ophthalmology indications - Potential to unlock multi indications - Multiple partnering opp
Slide 22: Questions?
Questions? ISCO.QB 1/21/2014 22
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