SEOVF TRADED ON The Path To A Regenerative Cure Bloom Burton - - PowerPoint PPT Presentation

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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

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Bloom Burton Healthcare Investor Conference

April 30 – May 1, 2019

Metro Toronto Convention Centre | Toronto, ON

The Path To A Regenerative Cure

TRADED ON

SEOVF

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SLIDE 2

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

  • bligation 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.

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SLIDE 3

Brief Overview

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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

  • f high risk type 1 diabetes with an unmet need called hypoglycemia unawareness

as a first approach for our therapeutic Cell Pouch technologies

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SLIDE 5

40% 32% 28%

Sernova’s Approach

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To inspire hope and contribute to health and well-being by providing the best care to every patient

Top Notch Doctors

A Total Regenerative Medicine Solution for the Therapeutic Treatment of Chronic Diseases

Cell Pouch™ Therapeutic Cells Immune Protection

Immune Protection

Technologies to protect therapeutic cells from immune system attack

Cell Pouch

An implantable medical device which provides a vascularized environment for therapeutic cells

Therapeutic Cells

Cells that produce and release missing (or needed) proteins or hormones into the bloodstream

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SLIDE 6

CELL POUCH CANDIDATE PRE- CLINICAL PHASE I PHASE II PHASE III DEVELOPMENT STAGE INDICATION

P H A S E I / I I I N I T I AT E D D E C 2 0 1 8 H Y P O G L Y C E M I A U N A W A R E N E S S H U M A N D O N O R I S L E T S , S Y S T E M I C I M M U N E 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 I S L E T S A N T I C I PAT E D 2 N D A P P R O VA L F O R D I A B E T E S H Y P O G L Y C E M I A U N A W A R E N E S S M I C R O E N C A P S U L A T E D S T E M C E L L D E R I V E D C E L L S A N T I C I PAT E D 3 R D A P P R O VA L F O R D I A B E T E S A L L I N S U L I N D E P E N D E N T D I A B E T I C P A T I E N T S P R E C L I N I C A L S E V E R E H E M O P H I L I A A P A T I E N T S C O R R E C T E D P A T I E N T C E L L S A L L O G R A F T I M M U N E P R O T E C T E D C E L L S E A R L Y D E V E L O P M E N T B R O A D E R H E M O P H I L I A A P A T I E N T S P R E - C L I N I C A L T H Y R O I D E C T O M Y 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 T H Y R O I D C E L L S

Sernova Pipeline

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TYPE 1 DI ABETES HEM O PHI LI A A THYRO I D DI SEASE

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Pre-Clinical Models

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Type 1 Diabetes: Pre-Clinical Models

Animal Models

Proof of Concept Study in Mice

  • Showed Islets survived
  • Animals became insulin independent
  • Cell Pouch removed and animals became diabetic

again

  • Can be applied to other indications
  • Hemophilia
  • Thyroid disease
  • And other rare diseases

Large Animal Study

  • Islet survival in the large animals
  • Control of blood sugar levels
  • First to show efficacy in two different large animal

models of diabetes First in Human Model *Health Canada

Study Design

  • 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

Cell Pouch™ and Islet Safety Met

  • Safety successfully met for the Cell Pouch™
  • Cell Pouch™ histology assessed by independent

pathologists blinded to the treatment

  • Islets housed within a natural tissue matrix
  • Islets are well-vascularized
  • Islet safety successfully met
  • Islets show evidence of insulin, somatostatin, & glucagon
  • Cell Pouch™ and islet biocompatibility met
  • Proof of islet protection from immune system attack

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SLIDE 9

Cell Pouch Vascularized Chambers

Consistent Across All Animal Models

Mouse Model Porcine Model Cynomolgus Monkey Model

vWF Nuclei

100um

vWF Nuclei

500um

vWF Nuclei

500um

500um 100um 100um

Tissue Chamber and Microvessel Development Conserved Across All Animal Models Evaluated

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Cell Pouch Vascularized Chambers

Consistent Across Multiple Implant Sites

Abdomen (NHP)

vWF Nuclei

Limb (NHP)

vWF Nuclei

Interscapular (NHP)

vWF Nuclei

Vascularized Tissue Chamber and Microvessel Development Consistent Across Multiple Implant Sites

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1 Month 6 Month 12 Month

Porcine Model: Long-Term Implantation Safety Study: Proof of Stable Vascularized Tissue Chambers

3x - Masson’s Trichrome 3x - Masson’s Trichrome 3x - Masson’s Trichrome vWF Nuclei 500um vWF Nuclei 500um vWF Nuclei 500um

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Cell Pouch Vascularized Chambers

12 Months Study

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SLIDE 12

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Able to be surgically implanted and incorporated across indications and drug regimes

Transplanted Cell Pouch ™: Immunosuppression and STZ Non-Transplanted Cell Pouch ™ Immunosuppression and STZ

H&E 1.7x Sernova Confidential SH1-P047 H&E 10x Sernova Confidential SH1-P047 Blood vessel

Non-Transplanted Cell Pouch™ Haemophilia A Model

Cell Pouch Vascularized Chambers

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  • 95% insulin independence in diabetic animals
  • Efficacy achieved using a marginal islet mass

200 islets/mouse

Mouse Model: 3rd Party Independent Efficacy

Cell Pouch™ Small Islet Dose: Insulin Independence*

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

Islets in tissue Matrix with microvessels Islet stained for beta cells and insulin

*Insulin staining

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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 following Cell Pouch™ removal Chamber space ready for transplant

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Healthy Islets in the Cell Pouch™ Micro-vessels

Sernova Corp

12 Weeks Post Cell Pouch™ and Islet Transplant Islets showing insulin & Other pancreas hormones

Insulin Glucagon Nuclei Insulin Somatostatin Nuclei Insulin vWF Insulin vWF Nuclei

50um

C-peptide Nuclei Insulin Nuclei

These images are the same section, showing co- localization of both insulin and C-peptide

Porcine Autograft Islet Transplant

Proof of Islet Survival and Function - Cell Pouch™ Islet Transplant in large animal Diabetes Model

Insulin and C-peptide co-localized

50um 50um 50um

Islets showing insulin & supporting blood vessels

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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

2-3 weeks 4-8 weeks 2-4 weeks 1 week

Tx Cell Pouch™ Removal Cell Pouch™ Removal

Fasting Blood Glucose (mM) Post-Cell Pouch™ removal Fasting Blood Glucose (mM) Daily Insulin Requirements (U) *Marginal Islet dose 2,500 IEQ/kg; Standard human islet transplant is 10,000 IEQ/kg

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Safety and Efficacy in Animal Models

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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
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SLIDE 18

Cell Pouch Human Clinical Evaluation

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SLIDE 19

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“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 of epinephrine a/k/a the symptoms of hypoglycemia (Palpitations, Anxiety,

Excessive Sweating, Light Headedness)

  • Patients live in constant fear of their next diabetic episode

Type 1 Diabetes: 1st Clinical Indication

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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 of this treatment

  • ption
  • Graft-versus-host-disease
  • Post-transplant lymphoproliferative disorder
  • Transplant Rejection
  • Survival of Islets
  • Risks around portal vein implantation
  • Low number of cells available
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Cell Pouch Human Clinical Studies

  • First-In-Human Safety Study (Canada)
  • Chicago Phase I/II Study (USA)
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Study Design

  • 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

Cell Pouch™ and Islet Safety Met

  • Safety successfully met for the Cell Pouch™
  • Cell Pouch™ histology assessed by independent

pathologists blinded to the treatment

  • Islets housed within a natural tissue matrix
  • Islets are well-vascularized
  • Islet safety successfully met
  • Islets show evidence of insulin, somatostatin, & glucagon
  • Cell Pouch™ and islet biocompatibility met
  • Proof of islet protection from immune system attack

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Type 1 Diabetes : First-in-Human Study

2015

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Type 1 Diabetes : First-in-Human Study

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FDA Cleared Phase I/II Trial

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Phase I/II U.S. FDA Cleared Study

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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 antirejection medication activity

Primary Objective: To demonstrate the safety and tolerability of islet transplantation into the Cell Pouch for the

treatment of TID in subject with hypoglycemia unawareness and a history of severe hypoglycemic episodes.

Secondary Objectives: To establish islet release criteria that accurately characterize the islet product and are

predictive of clinical transplant outcomes into the Cell Pouch, which will be demonstrated through defined efficacy measures

  • Survival of endocrine tissue in the Cell Pouch
  • Proportion of subjects with a reduction in severe hypoglycemic events
  • Proportion of subjects with a reduction in HbA1c >1mg%
  • Over 20 additional endpoint analyses will occur

Status: US IND Cleared by FDA and IRB and patient enrolment initiated; Medtronic Minimed, Northridge, CA CGM is

supplying patients in Sernova’s U.S. regenerative medicine clinical trial of its Cell Pouch ; Next step: Interim safety and efficacy results

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2nd Islet Transplant

(increase dose)

Phase I/II Timeline

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Cell Pouch™ Implantation 1st Islet Dose Transplant Immuno Suppression Introduced

Feb 2019 Day180 Day365

3-4 weeks

Primary Endpoint: Initial Topline Safety Readout Small

(sentinel)

Pouches Removed Survival of Endocrine Tissue & Identification of Hormones Reduction in hypoglycemic events Reduction in HbA1c Secondary Endpoints:

Apr 2020

90 Days 3weeks

Immunosuppression Stabilized Cell Pouch Vascularization

90 Days

Additional Safety Assessments Completed Days 30, 60, 270 Post-Transplant

90 Days 90 Days

Day365 Day 0 Day180 Day0 Nov 2020

Safety Efficacy

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Future Developments / Additional Programs

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Cell Pouch Technologies: Next Steps

Immune Protected Cell Pouch™

(No Need for Immuno-suppression)1

Immune Protected Cell Pouch™ with Pluripotent Stem Cells

  • Unlimited supply of cells
  • Worldwide exclusive rights to UHN

(University Health Network) diabetes stem cell technology

  • CCRM (Center for Commercialization of

Regenerative Medicine) successfully conducted tech transfer, optimize cell production process and produce cells for testing within the Cell Pouch

  • Robust cell production process has been

developed where cells consistently reach or exceed release criteria

  • Local Immune Protection Technologies
  • (i.e.) Microencapsulated stem cell

derived technology

1 Immune protection technology not disclosed yet

Next Generation

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  • All Type 1 diabetic patients and 30% of

Type 2 diabetes who convert to insulin use

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Hemophilia Program

Patient Population

  • Hemophilia A ≈ 20,000 NA/EU ($15B Orphan Indication)

Hemophilia Therapy

  • Factor VIII Gene corrected cells within Cell Pouch – produce constant

therapeutic Factor VIII levels

  • Patient corrected cells (autologous)
  • Stem cell derived technology and local immune protection (allograft)

Therapeutic Goals:

  • Improved efficacy with prophylactic treatment reduced cost; improved

patient QOL; reduction of disease side effects Sernova’s Product Approach

  • Corrected own patient cells into the Cell Pouch (Horizon 2020 Grant)
  • Status: Corrected patient cells survive and produce Factor VIII in

hemophilia model

  • Treatment for all patients
  • Stem cell releasing Factor VIII product
  • Status: in development

Sernova’s Cell Pouch™ with Factor VIII releasing cells:

  • Reduce/eliminate Factor VIII infusions
  • Maintain constant blood levels of

Factor VIII

  • Reduce joint bleeds
  • Improve long-term efficacy
  • Improve QOL

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Hemophilia Program

Preclinical Safety and Efficacy of the Hemophilia Cell Therapy in the Cell Pouch™ Concept Proven

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T P

Non-transplanted Cell Pouch™ Awaiting Cells

T – Transplant area of Cell Pouch™ P – Peritoneum

T P Cell Pouch™ Transplanted with Cell Therapy

40×

Confirmed release tested BOECs

Human stained (red) HLA-ABC FVIII DAPI

40x-zoomed

Hemophilia not corrected Transplanted FVIII corrected BOECs

Dots represent collected droplet every 30sec

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 The patient’s own corrected BOECs transplanted into the Cell Pouch™ improved clotting in Hemophilia A, providing supporting documentation for next step development.

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Thyroid Disease Program

Estimated Market $2,2 Billion Thyroid Therapy (Current Standard of Care) Oral – Intravenous – Others Targeted Thyroid Disorders (Thyroidectomy)

  • Grave’s disease
  • Thyroid Nodules (Hyperthyroidism)
  • Hashimoto disease

Therapeutic Goals: Improved efficacy with prophylactic treatment reduced cost; improved patient QOL; reduction of disease side effects Sernova’s Product Approach

  • Thyroidectomy patient cells transplanted into the Cell Pouch
  • Status: Preclinical assessment: Corrected patient cells survive and

produce thyroid hormone Sernova’s Cell Pouch™ Thyroid releasing cells:

  • Reduce/eliminate daily life long thyroid

medications

  • Recover natural feedback loop of

thyroid hormones

  • Reduce side effects from low thyroid

hormone levels

  • Improve long-term efficacy
  • Improve QOL

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Thyroid Disease Program

Preclinical Safety and Efficacy of the Thyrioid Tissue Therapy in the Cell Pouch™

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Cell Pouch™ Transplanted with Cell Therapy

+

T P

Thyroglobulin IHC-4x 10x larger image 20x larger image

Human Thyroid Tissue

Mouse Thyroid

Human Thyroid Tissue

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SLIDE 33

Manufacturing & International Patent Protection

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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 Regulation MDR 2017/745
  • US FDA Quality System Regulations (QSR) 21 CFR 820
  • Canadian Medical Device Regulation (CMDR)

Two year real time Cell Pouch™ product stability and package integrity International (North/South American, Europe, Asia) patents and patent applications portfolio in 10 patent families: Composition and use of medical devices for delivery and cell transplantation

  • Composition and use of medical devices for delivery and cell transplantation
  • Glucose responsive insulin secreting stem cell technologies
  • Local immune protection technologies
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Confirmed Collaborations

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President and CEO

  • Dr. Philip Toleikis

1-519-858-5126 Info@Sernova.com Corporate Communications Dominic Gray 1-519-858-5126 Dominic.Gray@Sernova.com

TRADED ON

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Investor Relations Ray Matthews 1-604-818-7778 Ray@raymatthews.ca