Sernova Biotherapeutics Inc · TSX:SVA
Sernova Biotherapeutics — Sernova — Bloom Burton Healthcare Investor Conference 2019
35 slides from Sernova Biotherapeutics's conference presentation, May 1, 2019
Slide 1: The Path To A Regenerative Cure
SEOVF TRADED ON The Path To A Regenerative Cure Bloom Burton Healthcare Investor Conference April 30 – May 1, 2019 Metro Toronto Convention Centre | Toronto, ON
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation may contain forward looking statements. Forward-looking statements address future events and conditions and therefore involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. The in
Slide 3: Brief Overview
Brief Overview
Slide 4: Our Mission
Our Mission Sernova is a clinical stage regenerative medicine therapeutics company developing a Cell Pouch implantable device with therapeutic cells. Our primary focus is development of treatments for patients with insulin-dependent diabetes (T1), hemophilia A and thyroid disease Sernova is currentl
Slide 5: Sernova's Approach
Sernova’s Approach Cell Pouch™ Therapeutic Cells A Total Regenerative 28% Medicine Solution for the 40% Therapeutic Treatment of Chronic Diseases 32% Immune Protection Cell Pouch Top Notch Doctors Therapeutic Cells Immune Protection An implantable To inspire hopemedical device and contribute to Cell
Slide 6: Sernova Pipeline
Sernova Pipeline CELL POUCH PRE- DEVELOPMENT CANDIDATE CLINICAL PHASE I PHASE II PHASE III STAGE INDICATION TYPE 1 DIABETES HUMAN DONOR ISLETS, P H A S E I / I I I N I T I AT E D HYPOGLYCEMIA SYSTEMIC IMMUNE DEC 2018 UNAWARENESS PROTECTION MICROENCAPSULATED A N T I C I PAT E D 2 ND ISLETS HYPOGLYCEM
Slide 7: Pre-Clinical Models
Pre-Clinical Models
Slide 8: Type 1 Diabetes: Pre-Clinical Models
Type 1 Diabetes: Pre-Clinical Models Animal Models Proof of Concept Study in Mice Large Animal Study • Showed Islets survived • Islet survival in the large animals • Animals became insulin independent • Control of blood sugar levels • Cell Pouch removed and animals became diabetic • First to show ef
Slide 9: Consistent Across All Animal Models
Cell Pouch Vascularized Chambers Consistent Across All Animal Models Tissue Chamber and Microvessel Development Conserved Across All Animal Models Evaluated Mouse Model Porcine Model Cynomolgus Monkey Model 500um 100um 100um 100um 500um 500um vWF vWF vWF Nuclei Nuclei Nuclei 10
Slide 10: Cell Pouch Vascularized Chambers
Cell Pouch Vascularized Chambers Consistent Across Multiple Implant Sites Vascularized Tissue Chamber and Microvessel Development Consistent Across Multiple Implant Sites Abdomen (NHP) Limb (NHP) Interscapular (NHP) vWF Nuclei vWF vWF Nuclei Nuclei 11
Slide 11: Cell Pouch Vascularized Chambers
Cell Pouch Vascularized Chambers 12 Months Study Porcine Model: Long-Term Implantation Safety Study: Proof of Stable Vascularized Tissue Chambers 1 Month 6 Month 12 Month 3x - Masson’s Trichrome 3x - Masson’s Trichrome 3x - Masson’s Trichrome vWF 500um vWF 500um vWF 500um Nuclei Nuclei Nuclei 12
Slide 12: Cell Pouch Vascularized Chambers
Cell Pouch Vascularized Chambers Able to be surgically implanted and incorporated across indications and drug regimes Non-Transplanted Cell Pouch™ Non-Transplanted Cell Pouch ™ Transplanted Cell Pouch ™: Haemophilia A Model Immunosuppression and STZ Immunosuppression and STZ H&E 1.7x Sernova Confide
Slide 13: Mouse Model: 3rd Party Independent Efficacy
Mouse Model: 3 Party Independent Efficacy rd Cell Pouch™ Small Islet Dose: Insulin Independence* • 95% insulin independence in diabetic animals • Efficacy achieved using a marginal islet mass Glucose levels rise upon Cell Pouch™ removal *Diabetes Is Reversed in a Murine Model by Marginal Mass Syngen
Slide 14: Porcine Diabetes Autograft Transplant
Porcine Diabetes Autograft Transplant Proof of Safety and Efficacy: Cell Pouch™ Islet Transplant in Large Animals Cell Pouch™ implant Pancreatectomy Diabetes Induction (STZ) Islet Transplant Cell Pouch™ Removal 4-8 weeks 1 day 4-7 days 8-12 weeks 1 week Chamber space Sugar levels rise ready for foll
Slide 15: Porcine Autograft Islet Transplant
Porcine Autograft Islet Transplant Proof of Islet Survival and Function - Cell Pouch™ Islet Transplant in large animal Diabetes Model 12 Weeks Post Cell Pouch™ and Islet Transplant Islets showing insulin & supporting blood vessels Insulin vWF Healthy Islets in the Cell Pouch™ Insulin Micro-vessels v
Slide 16: Porcine Diabetes Allograft Transplant
Porcine Diabetes Allograft Transplant Proof of Safety and Efficacy: Glucose Control with 25% Standard Human Dose of Islets Cell Pouch™ implant Diabetes Induction (STZ) Islet Transplant Cell Pouch™ Removal 4-8 weeks 2-3 weeks 2-4 weeks 1 week Tx Cell Pouch™ Removal Cell Pouch™ Removal Fasting Blood G
Slide 17: Safety and Efficacy in Animal Models
Safety and Efficacy in Animal Models Assessment of Cell Pouch™ in Small Animal Model of Diabetes • Cell Pouch™ Assessment in a Small Animal Model of Diabetes: Rat Isograft Study • Diabetes Reversal in a Murine Model by Syngeneic Marginal Mass Islet Transplantation into the Cell Pouch™ Assessment of
Slide 18: Cell Pouch Human Clinical Evaluation
Cell Pouch Human Clinical Evaluation
Slide 19: Type 1 Diabetes: 1st Clinical Indication
Type 1 Diabetes: 1 Clinical Indication st “Hypoglycemia unawareness” affects about 10% of Type 1 Diabetes patients or about 125k patients in the US • Clinically defined as a complication of diabetes in which the patient is unaware of a deep drop in blood sugar levels • Failure to trigger secretion o
Slide 20: T1D: Current Treatment Options
T1D: Current Treatment Options Insulin discovered in London, Ontario, in 1921 • Patent licensed by Novo Nordisk Islet donor transplants have been a verified as a successful treatment for Type 1 Diabetes since the Edmonton Protocol in the 1990s However, little has been done to progress the viability
Slide 21: Cell Pouch Human Clinical Studies
Cell Pouch Human Clinical Studies • First-In-Human Safety Study (Canada) • Chicago Phase I/II Study (USA)
Slide 22: Type 1 Diabetes: First-in-Human Study
Type 1 Diabetes : First-in-Human Study Study Design 2015 • Diabetes subjects with hypoglycemia unawareness • Open-label; single-arm • Donor islet transplantation 2-24 weeks post Cell Pouch™ implantation • Primary endpoint • Safety post Cell Pouch™ implantation and 1 month post islet transplantation
Slide 23: Type 1 Diabetes: First-in-Human Study
Type 1 Diabetes : First-in-Human Study 24
Slide 24: FDA Cleared Phase I/II Trial
FDA Cleared Phase I/II Trial
Slide 25: Phase I/II U.S. FDA Cleared Study
Phase I/II U.S. FDA Cleared Study Safety, Tolerability and Efficacy Study of Sernova’s Cell Pouch™ for Clinical Islet Transplantation Study design: A open-label, single-arm study of Sernova Cell Pouch implanted with islets. Islets are transplanted into the Cell Pouch after implantation and stable an
Slide 26: Phase I/II Timeline
Phase I/II Timeline Secondary Endpoints: Immuno Survival of Endocrine Tissue & Identification of Hormones Suppression Reduction in hypoglycemic events Introduced Small Reduction in HbA1c (sentinel) Pouches Cell Removed 1st Islet Dose Pouch™ Transplant Implantation Day 0 Day180 Day365 90 Days 90 Days
Slide 27: Future Developments / Additional Programs
Future Developments / Additional Programs
Slide 28: Cell Pouch Technologies: Next Steps
Cell Pouch Technologies: Next Steps Next Generation Immune Protected Cell Pouch™ (No Need for Immuno-suppression)1 1 Immune protection technology not disclosed yet • All Type 1 diabetic patients and 30% of Type 2 diabetes who convert to insulin use Immune Protected Cell Pouch™ with Pluripotent Stem
Slide 29: Hemophilia Program
Hemophilia Program Patient Population Sernova’s Cell Pouch™ with Factor VIII • Hemophilia A ≈ 20,000 NA/EU ($15B Orphan Indication) releasing cells: Hemophilia Therapy • Reduce/eliminate Factor VIII infusions • Factor VIII Gene corrected cells within Cell Pouch – produce constant therapeutic Factor
Slide 30: Hemophilia Program
Hemophilia Program Preclinical Safety and Efficacy of the Hemophilia Cell Therapy in the Cell Pouch™ Concept Proven Cell Pouch™ Transplanted with Cell Therapy T P Confirmed release tested BOECs HLA-ABC FVIII Human stained (red) DAPI + 40× 40x-zoomed Non-transplanted Cell Pouch™ Awaiting Cells Dots r
Slide 31: Thyroid Disease Program
Thyroid Disease Program Estimated Market Sernova’s Cell Pouch™ Thyroid $2,2 Billion releasing cells: Thyroid Therapy (Current Standard of Care) • Reduce/eliminate daily life long thyroid Oral – Intravenous – Others medications Targeted Thyroid Disorders (Thyroidectomy) • Recover natural feedback loo
Slide 32: Thyroid Disease Program
Thyroid Disease Program Preclinical Safety and Efficacy of the Thyrioid Tissue Therapy in the Cell Pouch™ Thyroglobulin IHC-4x Human Thyroid Human Tissue Thyroid Tissue Mouse Thyroid 10x larger image Cell Pouch™ Transplanted with Cell Therapy + T 20x larger image P 33
Slide 33: Manufacturing & International Patent Protection
Manufacturing & International Patent Protection Manufacture of the Cell Pouch™ in multiple sized is conducted GMP by a US contract manufacturer in a Class VII Clean Room Product and process development is conducted in accordance with manufacturer’s Quality System • ISO 13485 • EU Medical Devices Reg
Slide 34: Confirmed Collaborations
Confirmed Collaborations 35
Slide 35: SEOVF
President and CEO Dr. Philip Toleikis 1-519-858-5126 [email protected] Investor Relations Ray Matthews 1-604-818-7778 [email protected] Corporate Communications SEOVF Dominic Gray TRADED ON 1-519-858-5126 [email protected]
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