International Stem Cell Corporation · OTC:ISCO
International Stem Cell — Investor Presentation (November 2019)
16 slides from International Stem Cell's investor presentation, Nov 27, 2019
Slide 1: Cells for Therapy and Research
Cells for Therapy and Research OTCQB ISCO www.internationalstemcell.com International Stem Cell Corporation bd@intlstemcell.com 5950 Priestly Drive 1-760-940-6383 Carlsbad CA 92008
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: Cells for Therapy and Research
Cells for Therapy and Research HLA-compatible stem cell platform Proprietary Robust pre-clinical Scalable pipeline addressing large unmet Ethical medical needs Neural stem cells Parkinson’s disease Stroke Commercially successful Liver cells biomedical business Crigler-Najjar syndrome α1-antitrypsin
Slide 4: UniStemCell
UniStemCell Unique stem cell platform technology Human parthenogenesis (unfertilized oocytes) PSC ESC iPS Adult Potential to Impractical Individual Individual Yes immune match each line is unique only only Fully proliferative/ Strong Varies Weak Economic source Strong Genes manipulated/ No No Yes No
Slide 5: Intellectual Property
Intellectual Property Freedom to operate in major jurisdictions Potential competitive advantages in EU 22 issued patents, 117 patent applications • Pluripotent cell development • Cell banking • Manufacturing methods • Cell differentiation • Treating diseases Numerous publications • Nature’s Scientif
Slide 6: Broad Pipeline of Targets
Broad Pipeline of Targets Clinical success unlocks significant value Pre- Indication IND Phase I/II clinical hpNSC Parkinson’s disease Ischemic stroke Spinal cord injury Metabolic liver diseases Retinal blindness Corneal blindness 6
Slide 7: cGMP Manufacturing of NSC
cGMP Manufacturing of NSC Highly optimized GMP process ‒ Virtually unlimited quantities of NSC ‒ Produces extremely pure cell populations ‒ Patented Differentiate Expand Thaw On-site QC Prep. Ideal product characteristics ‒ Cryopreservable ‒ Centralized manufacturing ‒ Easily scalable 7
Slide 8: Parkinson's Disease
Parkinson’s Disease # 2 Neurodegenerative disease Limited treatment options (symptomatic only) ‒ About 1 million people in US have PD ‒ Direct and indirect costs ~$25 billion ‒ Rarely diagnosed prior to 50 years of age ‒ Hallmark motor symptoms Bradykinesia (slow movement) Rigidity Tremor ‒ Mo
Slide 9: Summary of Non-clinical Studies
Summary of Non-clinical Studies Proof of Principle Studies 6 month in 6-OHDA rodents 3 month in primates Dose Escalating Efficacy Study Dose escalating study in 6-OHDA rodents Safety Studies Acute toxicity in immune deficient rodents 9 month GLP pharm/tox., biodistribution and tumorigenici
Slide 10: 12-Month MPTP-Lesioned Primate Data
12-Month MPTP-Lesioned Primate Data Control Treatment 10
Slide 11: 12-Month MPTP-Lesioned Primate Data
12-Month MPTP-Lesioned Primate Data Transplanted Cells Integrate Dopamine Fiber Innervation Control Treatment 11
Slide 12: 12-Month MPTP-Lesioned Primate Data
12-Month MPTP-Lesioned Primate Data Dopamine levels post mortem Stable Parkscores and Healthy Behavior for duration of the study Functional activity demonstrated in disease model primates 9/1/2015 12
Slide 13: Safety: 9-months GLP study
Safety: 9-months GLP study Necropsy Biodistribution Tissue Finding Analysis Organs Analyzed Human cells detected Cervical lymph node Normal Salivary gland Normal Liver Not detected Thyroid w/ parathyroid Normal Kidney Not detected Esophagus, Trachea, Normal Heart Not detected Larynx, Tongue Diaphrag
Slide 14: Phase 1 Clinical Trial – 2H 2015
Phase 1 Clinical Trial – 2H 2015 12 month single arm dose escalating study ‒ Doses based on primate data ‒ 12 subjects with idiopathic Parkinson’s disease ‒ UPDRS < 49 in off-phase ‒ Stable on medications Primary endpoints: ‒ Safety No worsening of symptoms Brain lesions (MRI) ‒ Efficacy Impro
Slide 15: Prior Clinical Trials
Prior Clinical Trials Freed, C. Transplantation of embryonic Olanow, C. W., et al. A double-blind controlled trial dopamine neurons for severe Parkinson's of bilateral fetal nigral transplantation in disease. N Engl J Med 344(10):710-719; Parkinson's disease. Annals of Neurology 54:403- 2001. 414; 2
Slide 16: 2015/2016 Milestones
2015/2016 Milestones Program 2H 2015 1H 2016 2H 2016 Phase I/IIa: • 6 months • Full clinical • Patient clinical data Parkinson’s disease enrollment data • US IND • Dosing (Phase II) • Animal • License Ophthalmology studies w/Rohto • New • New extra • Medical specialty strength Line products products
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