International Stem Cell Corporation · OTC:ISCO
International Stem Cell — Investor Presentation (October 2018)
19 slides from International Stem Cell's investor presentation, October 2018
Slide 1: Cells for Therapy and Research
Cells for Therapy and Research International Stem Cell Corporation OTCQB: ISCO 5950 Priestly Drive www.internationalstemcell.com Carlsbad, CA 92008 bd@intlstemcell.com 1-760-940-6383
Slide 2: Investment Highlights
Corporate Overview Investment Highlights Headquartered in California • Clinical-stage biotechnology company developing stem cell-based therapies • Three locations: GMP manufacturing lab in MD, GMP manufacturing lab in Oceanside, CA, headquarters in Carlsbad, CA • Unique stem cell platform that is pr
Slide 3: Therapeutic Programs
Corporate Overview Therapeutic Programs • Ongoing Phase 1/2a study in Parkinson’s disease with ISC-hpNSC • Conducting preclinical studies in traumatic brain injury and stroke with ISC-hpNSC • Conducting basic research in liver disease and osteoarthritis www.internationalstemcell.com 3
Slide 4: Market Opportunity
Corporate Overview Market Opportunity Indication Market Potential Current approach 60,000 new patients / year Parkinson’s disease1 No cure $2.5 billion / year 600,000 strokes / year Stroke2 3 hour treatment window $500 million / year 10 million new patients / year Traumatic Brain Injury3 worldwide N
Slide 5: Scientific Collaborators
Corporate Overview Scientific Collaborators Yale University Sanford Burnham Prebys Medical Eugene Redmond Jr, MD Discovery Institute Professor, Yale School of Medicine Evan Y. Snyder, MD, PhD Director, Stem Cells and Regeneration Program Royal Melbourne Hospital University of South Florida Andrew Ev
Slide 6: Intellectual Property
Corporate Overview Intellectual Property ✓ Freedom to operate in major jurisdictions ✓ Competitive advantages in EU ✓ 162 patents and licenses in 36 families ✓ 48 issued patents ✓ 114 patents pending in 36 families Significant peer-reviewed publications: • Scientific Reports by NPG • Cell Stem Cell
Slide 7: Human Parthenogenetic Stem Cells
Scientific Background Human Parthenogenetic Stem Cells No Life Possible Revazova, et al., Cloning Stem Cells. 2007;9(3):432-49 • ISCO developed and exclusively owns a new class of pluripotent stem cells known as human parthenogenetic stem cells (hpSC) hpSC • hpSC are derived from unfertilized eggs a
Slide 8: Human Parthenogenetic Stem Cells
Scientific Background Human Parthenogenetic Stem Cells • hpSC can be derived homozygous with identical copies of Human Leukocyte Antigen (HLA), making it easier to immune-match with patients • A single homozygous hpSC line can potentially match millions of patients with the same HLA type Histocompat
Slide 9: Rationale
ISC-hpNSC Rationale Degeneration of DA Neurons in Parkinson’s Disease Fetal tissue transplant 2nd Most Common Motor Symptoms: Neurodegenerative disease Bradykinesia, rigidity, tremors • Approved treatments do not stop disease progression or reverse symptoms • Progressive degeneration of nigrostriata
Slide 10: cGMP Manufacturing
ISC-hpNSC cGMP Manufacturing ISC-hpNSC has ideal Highly Optimized cGMP Process product characteristics • Virtually unlimited quantities of ISC-hpNSC • Extremely pure cell populations • Cryopreservable • Manufactured in-house in cGMP facilities • Centralized manufacturing • Patented • Scalable • Mult
Slide 11: hpSC-Derived Neural Stem Cells
ISC-hpNSC hpSC-Derived Neural Stem Cells • Cryopreserved Allogeneic Cell Population • Derived from Human Parthenogenetic Stem Cells (hpSC) • Characterized Composition of Cells: • Neural Stem Cells • Three Identified Functions: • Neurotrophic Support • Anti-Inflammation • Neuroregeneration • “Off the
Slide 12: Increases DA Neuron Innervation and Number
ISC-hpNSC Increases DA Neuron Innervation and Number Control ISC-hpNSC TH ISC-hpNSC increases DA neuron innervation in the striatum and the total number of DA neurons in the substantia nigra of PD monkeys compared to controls www.internationalstemcell.com 12
Slide 13: Safety & Efficacy Profile in Nonclinical Studies
ISC-hpNSC Safety & Efficacy Profile in Nonclinical Studies ✓ Survives in the Brain ✓ Differentiates into Neural Cell Types ✓ Improves Locomotor Activity • 10 Animal Studies ✓ Increases Dopamine Concentration • >300 Rodents and Monkeys ✓ Increases DA Neuron Innervation ✓ Increases DA Neuron Number ✓
Slide 14: First Clinical Trial to Establish Product Safety
ISC-hpNSC First Clinical Trial to Establish Product Safety • Open Label Phase I/IIa Study • Moderate PD, UPDRS Part III < 49 • 12 patients: 3 cohorts of 4 patients each Study Conducted at: • Dose Escalation: 30, 50, 70 million cells • Transplantation into Putamen, Caudate Nucleus and Substantia Nigr
Slide 15: Phase 1/2a PD Study Schema
ISC-hpNSC for Parkinson’s Disease Phase 1/2a PD Study Schema www.internationalstemcell.com 15
Slide 16: Delivery is Feasible and Safe
ISC-hpNSC for Parkinson’s Disease Delivery is Feasible and Safe 6 patients Completed 12 months 9 patients 2 patients Received ISC-hpNSC Completed 6 months 1 patient Completed 3 months • Successful Delivery of ISC-hpNSC to Striatum and Substantia Nigra Without Intra-operative Complications • Delivery
Slide 17: PDQ-39 Summary Index
ISC-hpNSC for Parkinson’s Disease Phase 1/2a Interim Results % OFF time PDQ-39 60 50 1st Cohort 50 45 1st Cohort PDQ-39 Summary Index 2nd Cohort 40 2nd Cohort 40 35 % OFF Time 30 30 25 20 20 15 10 10 5 0 0 0 100 200 300 400 0 100 200 300 400 Days Post-Transplantation Days Post-Transplantation The %
Slide 18: Traumatic Brain Injury Program
ISC-hpNSC Traumatic Brain Injury Program ISC-hpNSC® significantly improves cognitive performance, locomotion, and neurological function in rodents with TBI. The preclinical studies were conducted by the world renowned Prof. Cesar Borlongan. The results of these studies demonstrate the therapeutic po
Slide 19: Business Performance
Corporate Overview Business Performance Net Sales $3,500,000 $3,000,000 Upcoming Milestones $2,500,000 $2,000,000 December 2018 $1,500,000 ❑ Dosing of last patient in 3rd cohort $1,000,000 $500,000 January 2019 $0 Q2,2016 Q3,2016 Q4,2016 Q1,2017 Q2,2017 Q3,2011 Q4,2017 Q1,2018 Q2,2018 ❑ IND submissi
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