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
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 information does not constitute any advice, promise or obligation of Sernova Corp. and does not necessarily represent the most current source of company information. Sernova Corp. cannot, and does not, guarantee or ensure either the accuracy, completeness, or authenticity of this presentation’s contents and may make changes and revisions to the information on this presentation at any time and without notice. The information is presented and stored on an "as is" basis and the use of the presentation to collect information is completely at your own risk. This presentation contains information about third-parties merely as a convenience. The inclusion of such information does not imply that Sernova Corp. endorses or accepts any responsibility for the content or use of such information. For more information on Sernova Corp, investors should review filings available at www.sedar.com. 2
Brief Overview
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 currently conducting a U.S. Phase I/II clinical trial targeting an indication of high risk type 1 diabetes with an unmet need called hypoglycemia unawareness as a first approach for our therapeutic Cell Pouch technologies
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 Therapeutic Cells Immune Protection Top Notch Doctors An implantable medical device Cells that produce and release Technologies to protect To inspire hope and contribute to health and well-being by providing which provides a vascularized missing (or needed) proteins or therapeutic cells from immune the best care to every patient environment for therapeutic cells hormones into the bloodstream system attack 5
Sernova Pipeline CELL POUCH PRE- DEVELOPMENT CANDIDATE CLINICAL PHASE I PHASE II PHASE III STAGE INDICATION TYPE 1 DI ABETES H U M A N D O N O R I S L E T S , P H A S E I / I I I N I T I AT E D H Y P O G L Y C E M I A S Y S T E M I C I M M U N E D E C 2 0 1 8 U N A W A R E N E S S P R O T E C T I O N M I C R O E N C A P S U L A T E D A N T I C I PAT E D 2 N D H Y P O G L Y C E M I A I S L E T S A P P R O VA L F O R U N A W A R E N E S S D I A B E T E S M I C R O E N C A P S U L A T E D A N T I C I PAT E D 3 R D A L L I N S U L I N S T E M C E L L D E R I V E D A P P R O VA L F O R D E P E N D E N T D I A B E T I C C E L L S D I A B E T E S P A T I E N T S HEM O PHI LI A A S E V E R E H E M O P H I L I A A P R E C L I N I C A L C O R R E C T E D P A T I E N T P A T I E N T S C E L L S E A R L Y A L L O G R A F T I M M U N E B R O A D E R H E M O P H I L I A P R O T E C T E D C E L L S D E V E L O P M E N T A P A T I E N T S THYRO I D DI SEASE T H Y R O I D E C T O M Y P R E - C L I N I C A L T H Y R O I D C E L L S P A T I E N T S F O L L O W I N G H Y P E R T H Y R O I D I S M 7
Pre-Clinical Models
Type 1 Diabetes: Pre-Clinical Models Large Animal Study Proof of Concept Study in Mice Animal Models • Islet survival in the large animals • Showed Islets survived • Control of blood sugar levels • Animals became insulin independent • First to show efficacy in two different large animal • Cell Pouch removed and animals became diabetic models of diabetes again • Can be applied to other indications • Hemophilia • Thyroid disease • And other rare diseases First in Human Study Design Cell Pouch™ and Islet Safety Met Model • Diabetes subjects with hypoglycemia unawareness • Safety successfully met for the Cell Pouch™ *Health Canada • Open-label; single-arm • Cell Pouch™ histology assessed by independent pathologists blinded to the treatment • Donor islet transplantation 2-24 weeks post Cell Islets housed within a natural tissue matrix o Pouch™ implantation Islets are well-vascularized o • Primary endpoint Islet safety successfully met o Islets show evidence of insulin, somatostatin, & glucagon o • Safety post Cell Pouch™ implantation and 1 month post Cell Pouch™ and islet biocompatibility met o islet transplantation Proof of islet protection from immune system attack o 9
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 100um 500um 100um 500um 100um 500um vWF vWF vWF Nuclei Nuclei Nuclei 10
Cell Pouch Vascularized Chambers Consistent Across Multiple Implant Sites Vascularized Tissue Chamber and Microvessel Development Consistent Across Multiple Implant Sites Interscapular (NHP) Abdomen (NHP) Limb (NHP) vWF Nuclei vWF Nuclei vWF Nuclei 11
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 500um vWF 500um 500um vWF vWF Nuclei Nuclei Nuclei 12
Cell Pouch Vascularized Chambers Able to be surgically implanted and incorporated across indications and drug regimes Transplanted Cell Pouch ™: Non-Transplanted Cell Pouch ™ Non-Transplanted Cell Pouch™ Immunosuppression and STZ Immunosuppression and STZ Haemophilia A Model H&E 1.7x Sernova Confidential SH1-P047 H&E 10x Blood vessel Sernova Confidential SH1-P047 13
Mouse Model: 3 rd Party Independent Efficacy 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 Syngeneic Islet Transplantation Using a Subcutaneous Cell Pouch Device. Transplantation, 2015 200 islets/mouse Islet stained for Islets in tissue beta cells and insulin Matrix with microvessels *Insulin staining 14
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 1 day 4-7 days 4-8 weeks 8-12 weeks 1 week Sugar levels rise Chamber space following Cell ready for Pouch™ removal transplant 15
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 vWF Nuclei Sernova Corp Islets showing insulin & Other pancreas hormones Insulin and C-peptide co-localized These images are Insulin Insulin C-peptide Insulin the same section, Somatostatin Glucagon Nuclei showing co- Nuclei Nuclei Nuclei localization of both insulin and C-peptide 50um 50um 50um 50um 16
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 Glucose (mM) Post-Cell Pouch™ removal *Marginal Islet dose 2,500 IEQ/kg; Fasting Blood Glucose (mM) Standard human islet transplant is 10,000 IEQ/kg Daily Insulin Requirements (U) 17
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 the Human-Scaled Cell Pouch™ • Safety Evaluation of the Cell Pouch™ Prior to Cell Transplantation • Histological Assessment of the Cell Pouch™ In a Porcine Animal Model • Assessment of Neovascularization of the Cell Pouch™ in the Pig • Cell Pouch™ Cynomolgus Primate Safety Study Safety and Efficacy of the Cell Pouch™ in Diabetic Pigs Following Autograft and Allograft Islet Transplantation • In vitro Islet Assessment as a Predictor of Long-Term Graft Function • Establishment of a Large Animal Porcine Autograft Diabetes Model • Cell Pouch™ Assessment in a Porcine Diabetes Model: Assessment of Safety and Efficacy for Autograft Islet Transplantation • Establishment of a Large Animal Porcine Diabetes Model Suitable for Allograft Transplantation • Porcine Allograft Safety and Efficacy 18
Cell Pouch Human Clinical Evaluation
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