regenerative therapies for neurological disorders
DESCRIPTION
Regenerative Therapies for Neurological Disorders. Company Overview. Company Background. Mission Statement SanBio develops regenerative therapies addressing unmet medical needs and creating value for stakeholders. Quick Facts Clinical-stage regenerative medicine company Founded in 2001 - PowerPoint PPT PresentationTRANSCRIPT
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Regenerative Therapiesfor Neurological Disorders
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Company Overview
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Company Background
Quick Facts• Clinical-stage regenerative medicine company• Founded in 2001• Headquartered in Mountain View, Calif.• Funding to Date: $53 million• 35 patents issued, 41 patents pending
Mission StatementSanBio develops regenerative therapies addressing unmet medical needs
and creating value for stakeholders.
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Financial Support
Clinical Trial & Development
Innovative Business Infrastructure
Innovative Science & Technology
Japan/U.S. Hybrid Innovation
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Neurological Disorders: Market SizeNeurological Disorder Sector is Growing Rapidly, Faster Than Other Markets
Disease Prevalence in U.S. (2010)
U.S. Product Sales (2010)
Stroke 6.8MM
Parkinson’s Disease 1.6MM
Multiple Sclerosis 400,000
Spinal Cord Injury 300,000 N/A
We are researching the product sales and will add
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Potential Market Value
Valuation Summary
Expected
Launch
Year
Target
patients
SB623
market
share
Patients
treatedPrice
Peak
sales
Peak
salesENPV
'000( ) (%) '000( ) $/ treatment( ) MM)($ MM)($ MM)($
Stroke 2017 102 80% 81 25,000 2,032 10,377 4,585
Traumatic brain injury 2019 74 80% 60 25,000 1,493 6,781 2,298
Parkinson's disease 2019 63 80% 51 25,000 1,267 5,757 1,939
Spinal cord injury 2019 26 80% 21 25,000 528 2,399 765
Retinal disease 2019 92 80% 74 25,000 1,843 8,375 2,854
Total 12,441
* Indications limited to the ones with efficacy confirmation in animal model. Do not include anticipated indications such
as Alzheimer's or multiple sclerosis.
** Target patients, SB623 market share and patients treated are all taken at the peak sales year.
Indication
North America* World wide
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Competitive Overview
• Neural Deficit Recovery™ (NDR) patented drug discovery platform
• Bone marrow-derived cells• Industrial scale expansion• Therapeutic potential
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Management TeamKeita Mori, MBA
Co-CEO, Chairman, Co-Founder
Toru Kawanishi
Co-CEO, Co-Founder
Damien Bates MD, PhD, FRACS, MBA
Chief Medical Officer
Michael P. McGrogan, Ph.D.
Senior Vice President, Production Development
Placeholder: Headshot
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Senior AdvisorsDonald Kennedy Former President of Stanford University, Commissioner of the United States Food and Drug Administration, and
Editor-in-Chief of Science
Mario Rosati Partner at Wilson Sonsini Goodrich & Rosati. Managing partner of WS Investments, an investment partnership composed of the partners and associates of the firm
Charles Garvin Principal, Palisades Associates, a merchant banking firm. A founding principal of The Beta Group, a Silicon Valley business development organization
Gary Snable Founder, Ex CEO, Layton Bioscience
George Martin Former Scientific Director, National Institute of Aging (NIH); SVP Fibrogen
Arnold Caplan Professor, Case Western Reserve
Martha Bohn Professor, Northwestern University, Past Member of RAC
Cesario Borlongan Professor, University of Southern Florida
Krys Bankiewicz Professor, UCSF
Yoichi Nabeshima Former Professor, Kyoto University; Director, IBRI
Scientific Advisory Board
Advisors
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Partnerships
• Clinical Trial Partners
• Corporate Partners
• Innovation Awards
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Approach to Brain Regeneration
Donor bone marrow stem cells
are harvested
Allogeneic Cell Transplant
Regenerative effect on recipient cells
Brain & body function restored
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NDR™ Technology Platform
BoneMarrow
SB623
SB618
SB308
Products(“Cells-in-a-bottle”)
Production
...
Neural Deficit Recovery™
NDR™ Technology
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Product Pipeline
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Bone Marrow Derived Cells
Stroke, TBI, SCI,Retinal Disease,
PD, AD, etc.
Multiple Sclerosis,Peripheral Nerve,
SCI, etc.
Muscle Dystrophy, etc.
SB623 SB618 SB308
Bone Marrow-DerivedNeuroregenerative Cells
Enhanced Marrow Stromal Cells(eMSC)
Bone Marrow-DerivedMuscle Regenerative Cells
P-1/2 Nonclinical Research
Different image
Bone Marrow-Derived Regenerative Cell MedicinesDamien – Can you recommend an image for SB618?
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Indications Overview
Indication SB623 SB618 SB308
Stroke Recovery √
Parkinson’s Disease √
Spinal Cord Injury √ √
Multiple Sclerosis √
Muscular Dystrophy √
Trauma √ √ √
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SB623
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Therapy Overview
• Developed as treatment for chronic neurological deficits – stable stroke, advanced Parkinson’s disease, traumatic brain injury
• Reverses neural damage when injected into neural tissue
• Single allogeneic donor cell can be used to treat thousands of patients.
• Shown to restore function to damaged neurons associated with stroke, spinal cord injury and Parkinson's disease
SB623 Cells
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• Cells are expanded, formulated and cryopreserved in vials stored in vapor phase of liquid nitrogen
• Final product is a sterile frozen suspension containing at least 10 million viable cells per mL in a cryovial
• Shipped to clinical sites using a Dry Nitrogen Cryo shipper with adsorbed liquid nitrogen
• Cells will grow with a doubling time of 3 to 4 days after thaw
Scalable Production Methods Reduced to PracticeManufacturing Process & Product Preparation
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Multiple Modes of Action
SB623Modes of Action
Trophic Factors
Extracellular Matrix
Anti-inflammation
Stimulating Migration
Angiogenesis
Damien – Please note that we will have our creative team rework this slide to make it look more polished
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Product CharacteristicsBulk Substance Specifications
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Product CharacteristicsDrug Product Specifications
Attribute Specification
Purity and Impurities
Purity ≥ 80% PositiveCD29, CD90, CD105
Impurities ≤ 5% PositiveCD31, CD34, CD45
Quality
Cell Viability ≥ 70% viable
Total Viable Yield ≥ 8x106 viable cells/mL
Plating ≥ 50%
Cell Growth Rate Doubles in ≥ 1 to ≤ 5 days
Safety
Colony Growth in Soft Agar No Colonies
Sterility No Growth
Bacterial No Growth
Fungal No Growth
Endotoxin ≤ 5 EU/mL
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Loss of NICD with Continued PassagesDisappearance of The Plasmid During Expansion (prior to phase 2)
Plasmid pN-2 was measured using qPCR and expressed as copies per diploid genome. Passage number is represented as P1, P2, etc. The negative control is buffer alone.
Lanes 1, 2, 5 and 6 are unactivated/unstabilized negative controls. Lanes 3 and 4 show activated and stabilized samples and indicate the location of the endogenous NICD. Lane 7 shows high levels of NICD protein 3 days post transfection. Lane 8 shows no detectable NICD in the final product.
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Transient Transfection with NICD Causes Epigenetic ChangesCpG Methylation Status of 5 Different Genes for 3 Donors
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Homogeneity and Identity of SB623 CellsCells are Positive for MSC Markers and Negative for Hematopoietic Markers
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Proteomic Analysis of the Extracellular MatrixCharacterization of the ECM Produced by SB623
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Extracellular Matrix Produced by Bone Marrow Stromal CellsECM Promotes Neural Growth
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Extracellular Matrix Produced by Bone Marrow Stromal CellsSupports Growth of Different Neural Cell Types
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Comparison of The Neuropoietic ActivityDerived ECM Supports Nestin Growth; Mediated by TGM2
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Human Mesenchymal Stromal Cells and Their DerivativeMultiple Growth Factors Released
Placeholder: We are simplifying the table per your notes and will add in
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Human Mesenchymal Stromal Cells and Their DerivativeReduces Cell Death In Vitro Model Ischemia
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Quantitative Microplate Assay for StudyingMSCs Promote Neural Cell Growth and Differentiation
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Comparison of The Neuropoietic ActivitySB623 Promotes More Neural Cell Growth and Differentiation Than MSCs
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Comparison of The Neuropoietic ActivitySB623 Action Mediated by FGF2 and BMP4
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Comparing The Angiogenic PotencySB623 and MSCs Secrete Proangiogenic Factors
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Comparing The Angiogenic PotencySB623 Promotes More Branching in Aortic Ring Assay
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Comparing The Immunosuppressive PotencyDecreases Cell Mediated Immune Response
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Effect on Peripheral and Nervous System Innate Immune CellsSB623 Cells Reduce The Percentage of Pro-Inflammatory Peripheral Blood Monocytes
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Comparing The Immunosuppressive PotencySB623 Induces Regulatory T Lymphocytes
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Comparing The Immunosuppressive PotencySB623 Inhibits Differentation of Monocytes to Dendritic Cells
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Stem Cell Recruitment of Newly Formed Host CellsSB623 Supports Robust Migration and Proliferation of Endogenous Stem Cells in Traumatic Brain Injury Model
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Effect of SB623 on Neural Progenitor Cell MigrationDose Response of SB623 Conditioned Medium on NPC Migration
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SafetyCells Are Not Tumorigenic
Absence of Growth on Brain Slices SB623 Cells Do Not Exhibit Signs of Tumorigenic Transformation
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SafetyTransient Persistence of SB623 Cells In Vivo and No Migration Away From Site of Implantation
Rapid Disappearance of SB623 Cells In Vivo
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Safety
Differentiation Potential of MSCs and SB623
SB623 Cells Have Limited Differentiation Potential
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Clinical Trials and Data
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SB308
Muscle Degeneration
Development StagesDamien – Please note that we will have our creative team rework this slide to make it look more polished
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Research Non-clin. IND Approval
P1 P2 P3
Traumatic Brain Injury
Retinitis Pigmentosa
Dry AMD
Parkinson’s Disease
Spinal Cord InjuryAlzheimer’s Disease
Stable Stroke
SB623: Clinical Trial Overview
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SB623 Tested in Chronic Stroke Rat Model to Determine Functional RecoverySB623: Stroke Preclinical Program
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Overall Design– Open Label– 18 Stroke Patients– 6 Mo. Efficacy, 2-Year Follow-Up
Patient Population– 6-36 Months Post-Ischemic Stroke– Stable Deficits– Moderate to Severe Patients
Endpoints– Safety– Efficacy: Motor, Sensory, Cognitive– Brain Activity by PET
Trial Sites
12 Patients Enrolled&
Good Safety
SB623: Stroke Phase 1 & 2 Clinical Trials
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SB623: Stroke Phase 1 & 2 Clinical Trials
Stereotactic Frame Positioning
Needle tracks for cell implantation and suggest implant sites
Stereotactic Frame Positioning
Needle Tracks for Cell Implementation and Suggest Implant
Sites
Approach
Damien – We’ve reviewed all the materials and can’t find a better image. We can pull a stock image and then add the arrows to the sites if you’re okay with that
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NIHSS
Months Post-treatment
Ch
an
ge
fro
m B
ase
lin
e (
+SE
M)
ESS
Months Post-treatment
Ch
an
ge fr
om
Ba
seli
ne
(+S
EM
)
Fugl-Meyer
Months Post-treatment
Ch
an
ge
fro
m B
ase
lin
e (
+SE
M)
SB623: Statistically Significant Efficacy Endpoints
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Safety Parameters• SB623- and surgical-related adverse events using WHO toxicity criteria• Adverse changes imaged by head MRI• Serum chemistry and hematology• 2 yrs. post-implant follow-up
Analysis of Safety• 17 patients had at least one treatment-emergent adverse event (TEAE)
– No trends observed with dose level and majority of TEAEs
– Overall, assessed as mild or moderate
– Higher number of patients had TEAEs that were assessed as related to the surgical
procedure when compared with TEAEs related to study treatment
– Five patients experienced severe TEAEs
SB623: Clinical Safety
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“... This trial represents an important advance in bringing restorative therapeutics for neurologic disorders into the clinical arena. Three measures of efficacy (NIHSS, ESS, Fugl‐Meyer) all show a trend toward improvement. We are looking forward to the next clinical trial.”
Gary K. Steinberg, M.D., Ph.D.Chairman, Department of Neurosurgery
Stanford University School of Medicine
SB623: Results Presented at Key Industry Event
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Test
Control
SB623 Reduced TBI Core and Peri-Injury AreaSB623: Traumatic Brain Injury Preclinical Efficacy
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Test Control
Nestin
SB623 Increased Nestin+ Cells
SB623: Traumatic Brain Injury Preclinical Efficacy
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SB623 Increased MMP-9 Gelatinolytic Activity
SB623: Traumatic Brain Injury Preclinical Efficacy
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SB623 Stimulated New Cells to Migrate
SB623: Traumatic Brain Injury Preclinical Efficacy
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Elevated Body Swing Test BedersonRotarod
Significant Motor and Sensory Function Recovery in TBI Rat Model
SB623: Traumatic Brain Injury Preclinical Efficacy
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SB623: Traumatic Brain Injury Clinical Study Design
We reviewed the materials but may have missed this information. Can you let us know where to locate?
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SB623 Tested in RCS Rat: Subretinal Transplantation
SB623: Retina Preclinical Program
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Electroretinogram (a-wave, b-wave) in SB623 Intravitreal Administration
Significant Vision Preservation in RCS Rat Model
SB623: Retina Preclinical Efficacy
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Azide Response @ 8 wks post transplantation
Significant Vision Preservation in RCS Rat Model
SB623: Retina Preclinical Efficacy
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Retinal Structure @ 9 wks Post Transplantation
SB623 Preserved Outer Nuclear Layer
SB623: Retina Preclinical Efficacy
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Intravitreal Administration as Effective as Subretinal
Favorable Administration as Effective
SB623: Retina Preclinical Efficacy
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SB618
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• Potential to treat conditions that affect the myelin sheath of neurons
• Bone marrow-derived, Schwann-like cells• Allogeneic cells
SB618: Therapy Overview
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Myelin Sheaths Surrounding Regenerated Neurons
• SB618 cells labeled with Green Fluorescent Protein (GFP) and engrafted into transected sciatic nerve
• 6 months later, transverse sections of regenerated were stained for neuronal marker NF
• NF positive axons shown in the photomicrograph above staining red• GFP positive implanted Schwann-like cells shown as green• Green cells clearly surround red cells
SB618: Promising Therapy Approach
Mimura et al. (2004) J. Neurosurg. 101:806-812.
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Repair of Injured Peripheral Nerve
• Sections of the sciatic nerves of rats replaced with matrix-filled tubes of SB618 cells or vehicle control
• 6 months later, implant site sectioned transversely to count number of nerve cells (axons)
• Measure of hind limb function after spinal cord injury
• Animals were subjected to complete transection of spinal cord
• SB618-treated animals recovered more quickly and to a greater extent than untreated
Spinal Cord Injury Repair (SB618)
SB618: Promising Therapy Approach
* indicates p < .05. Mimura et al. J. Neurosurg. (2004) 101:806-812. * indicates p < .05. Kamada et al. (2005) J. Neuropathol. Expt. Neurol. 64:37.
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SB308
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• Extended core technology beyond nervous system• Bone marrow to stimulate muscle regeneration • Potential to treat conditions such as muscular dystrophy;
recovery from trauma and surgery• Tested in rat model with positive results• External funding required for development beyond animal
studies• Commercialization through larger pharmaceutical company
partnerships
Therapy Overview
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Scientific Publications
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Scientific Publications
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Q&A