Sernova Biotherapeutics Inc · TSX:SVA
Sernova Biotherapeutics — Sernova — Cell and Gene Meeting on the Mesa 2020
26 slides from Sernova Biotherapeutics's conference presentation, October 2020
Slide 1: The Path To A Regenerative Medicine Cure
SEOVF TRADED ON The Path To A Regenerative Medicine Cure Cell and Gene Meeting on the Mesa October 12 - 16 , 2020 th th
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of applicable Canadian securities laws. Forward- looking statements in this presentation are statements that are not historical facts and are generally, but not always, identified by the words “expect
Slide 3: Sernova: Innovator & Leader
Sernova: Innovator & Leader Publicly traded, clinical-stage RM therapeutics solution innovator & leader: Cell therapy therapeutics solution platform treating chronic diseases & enhancing daily QOL Integrated RM therapeutic solution (Cell Pouch™ + therapeutic cells or tissue + immune-protection)
Slide 4: Sernova's Platform Approach
Sernova’s Platform Approach Integrated RM Therapeutic Solution for 28% 40% Treatment of Chronic Diseases 32% Cell Pouch Therapeutic Cells Immune Protection Implantable proprietary medical Human cells (donor / stem) & Technologies to protect device that provides vascularized tissues that produce & re
Slide 5: Worldwide IP / Patent Portfolio
Worldwide IP / Patent Portfolio International patents & patent applications portfolio in multiple patent families with broad application & continued expansion: Composition & use of medical devices for delivery & cell transplantation Glucose responsive insulin secreting stem cell technologies L
Slide 6: Sernova Pipeline
Sernova Pipeline CELL POUCH PRE- DEVELOPMENT CANDIDATE CLINICAL PHASE I PHASE II PHASE III STAGE INDICATION 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 T1D A N T I C I PAT E D 2 ND ALL INSULIN LOCALLY IMMUNE- A P P R O VA L F O R
Slide 7: Therapeutic Cells
Therapeutic Cells Therapeutic Cell Options Human donor cells enable early safety / efficacy testing in the clinic for indications & patients with unmet needs & advanced disease (i.e. diabetes HU patients). Supply is limited; however, they enable Cell Pouch validation in preparation for stem cell t
Slide 8: Immune Protection
Immune Protection Transplanted therapeutic cells must be protected from a natural immune system attack response. While established systemic transplantation immuno-suppression (anti-rejection) drugs can be effective, local immune protection (LIP) is optimal. LIP alternatives present different opp
Slide 9: Immune Protection
Immune Protection Conformal Coating Technologies: • Sernova’s proprietary cellular conformal coating technology - developed and optimized with years of research • It consists of a thin biocompatible porous polymer hydrogel coating surrounding therapeutic cells (islets, stem cells) • Proven to allow
Slide 10: Biologically Compatible Delivery Process
Biologically Compatible Delivery Process Cell Pouch Implantation & Therapeutic Cells Delivery Process Proprietary Cell Pouch is Therapeutics cells are Therapeutic cells release placed deep under the skin, transplanted directly into the missing proteins or allowing for vascularization & vascularized
Slide 11: Strategic Development Approach
Strategic Development Approach Device Therapeutic Immune Sernova Integrated Cells Protection RM Therapeutic Solution Immuno Portfolio Human Suppression Drugs Cell Therapeutic Donor (systemic) Pouch Cells Sernova Stem Cell Cell Encapsulation Proprietary Derived Conformal Coating Cell Pouch (iPSC / EP
Slide 12: Diabetes… Hope for a Functional Cure
Diabetes… Hope for a Functional Cure The Reality: Diabetes is one of the most prevalent diseases & pervasive medical problems impacting society & everyday quality of life (QOL) today 463 million affected worldwide and nearly 10% of these individuals have T1D(1) T1D represents a potential commerc
Slide 13: Diabetes Clinical Progress Summary
Diabetes Clinical Progress Summary Completion of first-in-human proof-of-concept study for diabetic condition HU Clinical protocol & regulatory package development for US Ph I / II clinical trial for Pre 2018 diabetic HU condition (T1D Study) FDA IND clearance to commence T1D Study T1D Study
Slide 14: Diabetes First Clinical Indication: HU
Diabetes First Clinical Indication: HU Hypoglycemia Hypoglycemia unawareness unawareness (HU), the(HU), most the most critical critical unmet needunmet needaffects in diabetes, in diabetes, 15% of T1D affects patients 15% (~240K of T1Dinpatients patients (~240 K patients in the US alone) the US alon
Slide 15: US Ph I/II Study Design
US Ph I/II Study Design US Ph I/II Safety, Tolerability, Efficacy Study Study Design: Company-sponsored IND. Open-label, single-arm study. Human donor islets are transplanted into the Cell Pouch after implantation & stable anti-rejection medication activity has been established. Primary Objective: T
Slide 16: US Ph I/II Case Study Early Findings
US Ph I/II Case Study Early Findings US Ph I/II Safety, Tolerability, Efficacy Study Safety Findings (90 days post-transplant) Incidence & severity of adverse events associated with Cell Pouch were monitored: No incidences of AEs determined to be probable or highly probable to the Cell Pouch Cel
Slide 17: US Ph I/II Case Study Early Findings
US Ph I/II Case Study Early Findings US Ph I/II Safety, Tolerability, Efficacy Study First Patient Observed Data Presented by Clinical Investigator* Early Efficacy Findings Islet Transplant Status: Before 3 Mo. After 90-day post-transplant glucose tolerance test (i.e. patient given a high sugar drin
Slide 18: US Ph I/II Case Study Early Findings
US Ph I/II Case Study Early Findings Improvement in ALL CGM Parameters Blood Glucose Over Time (mg/dL) Baseline: More excursions seen pre-transplant Parameter Baseline Post-transplant High Glucose Value (mg/dL) 285 231 Low Glucose Value (mg/dL) 50 66* # Glucose Excursions 15 3 # High Excursions 7 2
Slide 19: US Ph I/II 90-Days Post Transplant
US Ph I/II 90-Days Post Transplant Achievement of Secondary Endpoint “Survival of endocrine tissue in the Cell Pouch™ (defined by positive staining of islets during histological analysis) [ Time Frame: 90±5 days post-transplant for sentinel Cell Pouch™ ]” Islets Independent Pathologist reported: a
Slide 20: Earlier First-in-Human Study
Earlier First-in-Human Study First-in-World Successful Proof-of-Concept Study Design T1D subjects with HU & a history of severe hypoglycemic episodes Open-label, single-arm Donor islet transplantation 2 – 24 weeks post Cell Pouch implantation Primary Endpoint: Safety post Cell Pouch implanta
Slide 21: Hemophilia A Program
Hemophilia A Program Estimated ~20 K patients across North America & EU Market ~$10 B orphan indication Benefits of Sernova’s Cell Pouch with factor VIII releasing cells: Patients receive regular infusions of missing Standard clotting factors (i.e. factor VIII) Reduce / eliminate factor of C
Slide 22: Hemophilia A - Pre-Clinical Success
Hemophilia A - Pre-Clinical Success Pre-Clinical Safety & Efficacy of Hemophilia Cell Therapy in the Cell Pouch Confirmed Release-tested HLA-ABC BOECs FVIII DAPI Human stained (red) 40x Cell Pouch Transplanted with Cells Human corrected Human factor VIII BOECs transplanted corrected blood 40× into t
Slide 23: Thyroid Disease Program
Thyroid Disease Program Estimated 150,000 thyroidectomies performed annually in US ~$2.2 B market opportunity Market Benefits of Sernova’s Cell Pouch with Thyroid releasing cells: Patients require lifelong thyroid hormone Reduce / eliminate daily life replacement therapy Standard long thyroi
Slide 24: Thyroid - Pre-Clinical Success
Thyroid - Pre-Clinical Success Pre-Clinical Safety & Efficacy of the Thyroid Tissue Therapy in the Cell Pouch Human Thyroid Tissue Surviving Human Thyroid Tissue in Cell Pouch in mouse model ( 3 months post-transplant) Cell Pouch Transplanted with Healthy Human TG (green) TPO (red) Thyroid Tissue 4x
Slide 25: NEXT STEPS – Action Plan
NEXT STEPS – Action Plan Develop licensed/acquired local immune-protection technologies for Platform therapeutic cells Technology Advance local immune-protected diabetes stem cell technology in preparation for First-in-Man (FIM) study Expand existing strong worldwide multi-family patent portfo
Slide 26: 700 Collip Circle, Suite 114 London, Ontario Canada
700 Collip Circle, Suite 114 London, Ontario Canada N6G 4X8 [email protected] www.sernova.com