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Regenerative Therapies for Neurological Disorders

Regenerative Therapies for Neurological Disorders. Overview. Company. Company Background. Mission Statement. SanBio develops regenerative therapies addressing unmet medical needs and creating value for stakeholders. Quick Facts. Clinical-stage regenerative medicine company

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Regenerative Therapies for Neurological Disorders

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  1. Regenerative Therapiesfor Neurological Disorders

  2. Overview Company

  3. 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 • Headquartered in Mountain View, Calif. • Funding to Date: $53 million • 35 patents issued, 41 patents pending

  4. Japan/U.S. Hybrid Innovation Clinical Trial & Development Innovative Science & Technology Financial Support Innovative Business Infrastructure

  5. Neurological Disorders: Market Size Damien – Our team was unable to find updated product sales. Perhaps this is something your commercial team has? Neurological Disorder Sector is Growing Rapidly, Faster Than Other Markets 1American Heart Association; Alzheimer’s Association; National Parkinson Foundation; Multiple Sclerosis Foundation; The Foundation for Spinal Cord Injury, Prevention, Care & Cure

  6. Potential Market Value Valuation Summary North America* Worldwide *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.

  7. Competitive Overview

  8. Management Team Keita Mori, MBA Co-CEO, Chairman, Co-Founder Damien Bates MD, PhD, FRACS, MBA Chief Medical Officer,Interim Head, R&D Toru Kawanishi Co-CEO, Co-Founder Michael P. McGrogan, Ph.D. Senior Vice President, Production Development

  9. Advisors Scientific Advisory Board Senior Advisors

  10. Partnerships Clinical Trial Partners • Corporate Partners InnovationAwards

  11. Approach to Regeneration Damien – Please let us know which image you’d like to use for the Allogeneic Cell Transplant section Allogeneic Cell Transplant Regenerative Effect on Recipient Cells Donor Bone Marrow Stem Cells are Harvested Body Function Restored

  12. Bone Marrow Donation & Stem Cell Isolation Modification Production Products Technology SB623 Bone Marrow SB618 Proprietary Transfection and Selectionand/or Growth Factor Treatment SB308

  13. Pipeline Product

  14. SanBio Stem Cell Technology Platform – Application SB623 SB618 SB308 Bone Marrow-Derived Muscle Regenerative Cells Bone Marrow-Derived Neuroregenerative Cells Enhanced Marrow Stromal Cells (eMSC) Stroke, Traumatic Brain Injury, Spinal Cord Injury, Retinal Disease(Dry Age-Related Macular Degeneration), Parkinson’s Disease, Alzheimer’s Disease Multiple Sclerosis, Peripheral Nerve, Spinal Cord Injury Muscular Dystrophy

  15. SB623

  16. SB623: Therapy Overview • Regenerates neural tissue • Single allogeneic donor cell can be used to treat thousands of patients • Shown to improve function in animal models of stroke, traumatic brain injury, Parkinson’s disease and retinal disease • Clinical stage development for chronic stroke and traumatic brain injury

  17. SB623: Product Preparation Scalable Production Methods Reduced to Practice • 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 San Bio Incorporated. (2014). Investigator's drug brochure [Brochure]. Mountain View

  18. SB623: Multiple Modes of Action

  19. SB623: Product Characteristics Drug Product Specifications San Bio Incorporated. (2014, May 15). Type B meetings background/briefing materials [SB623 Cells]. Mountain View

  20. SB623: Identity and Purity of SB623 Cells Cells are Positive for MSC Markers and Negative for Hematopoietic Markers CD29 = Beta-1 Integrin CD90 = Thy-1 CD105 = Endoglyn CD31 = PECAM-1 CD34 = Hematopoietic Progenitor Marker CD45 = Leukocyte Common Antigen San Bio Incorporated. (2014). Investigator's drug brochure [Brochure]. Mountain View

  21. SB623 Derived ECM Promotes Neural Cell Growth Aizman. I, J Neurosci Res. 2009, 87(14):3198-206

  22. SB623 Secreted Proteins Decrease Cell Death In Vitro Model of Ischemia Tate CC. Cell Transplant. 2010,19(8):973-84

  23. SB623 Action Mediated by FGF2 and BMP4 Comparison of The Neuropoietic Activity Aizman I, Stem Cell Res Ther. 2014, 5(1):29

  24. SB623 Cells Recruit Host Neural Progenitor Cells to Sites of Injury SB623 Supports Robust Migration and Proliferation of Endogenous Stem Cells in Traumatic Brain Injury Model Tajiri, N. PLoS ONE 2013, 8(9):e74857

  25. Dose Response of SB623 Conditioned Medium on NPC Migration 300 250 200 150 100 50 * * in millions San Bio Incorporated. (2014, May 15). Type B meetings background/briefing materials [SB623 Cells]. Mountain View

  26. SB623: Safety Cells Are Not Tumorigenic SB623 Cells Do Not Exhibit Signs of Tumorigenic Transformation Absence of Growth on Brain Slices San Bio Incorporated. (2014). Investigator's drug brochure [Brochure]. Mountain View

  27. SB623: Safety Transient Persistence of SB623 Cells In Vivo and No Migration Away From Site of Implantation Rapid Disappearance of SB623 Cells In Vivo San Bio Incorporated. (2014). Investigator's drug brochure [Brochure]. Mountain View

  28. And Data Clinical Trials

  29. SB623: Clinical Trial Overview Stable Stroke Traumatic Brain Injury Dry AMD Retinitis Pigmentosa Parkinson’s Disease Spinal Cord Injury Alzheimer’s Disease

  30. SB623: Stroke Phase 1 & 2 Clinical Trials 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 San Bio Incorporated. Data on file

  31. SB623: Statistically Significant Efficacy Endpoints San Bio Incorporated. Data on file

  32. SB623: Clinical Safety • Safety Parameters • 17 patients had at least one treatment-emergent adverse event (TEAE) – No trends observed with dose level and 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 • SB623- and surgical-related adverse events using WHO toxicity criteria • Adverse changes imaged by head MRI • Serum chemistry and hematology • Immunology (anti-HLA antibodies; mixed lymphocyte reaction; TNF-α; IL-6; IFN-γ) • 2 yrs. post-implant follow-up • Analysis of Safety San Bio Incorporated. Data on file

  33. SB623 Injection Decreases Zones of Injury Test Control San Bio Incorporated. Data on file

  34. SB623: Traumatic Brain Injury Preclinical Efficacy SB623 Stimulates Neural Progenitor Cell Proliferation & Migration “Biobridge” Concept San Bio Incorporated. Data on file

  35. SB623: Traumatic Brain Injury Clinical Study Design Overall Design • Open Label • 12 Traumatic Brain Injury Patients • 1 Year Patient Population • Male, Female, Age 18-75 • Patients with traumatic brain injury who have remained stable during the prior 3 weeks • Between 12 and 36 months post-traumatic brain injury with focal injury and fixed motor deficit Endpoints • Safety determined during 1 year post-implant • Efficacy endpoints will be determined at 6 months post-implant; measures will also be at 1 week, and months 1, 2 and 12 San Bio Incorporated. Data on file

  36. SB623: Retina Preclinical Program SB623 Tested in RCS Rat: Subretinal Transplantation San Bio Incorporated. Data on file

  37. SB623: Retina Preclinical Efficacy Significant Vision Preservation in RCS Rat Model Azide Response @ 8 Wks Post Transplantation San Bio Incorporated. Data on file

  38. Q&A

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