Sernova Corp
Sernova Corporate Presentation Q1, 2013
P.M. Toleikis, Ph.D., President and CEO
Founded 2006, London Ontario; TSX(v); SVA.V
Sernova Corp
Cautionary and Forward-Looking Information
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.
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Sernova Company Overview A Clinical Stage Company in Regenerative Medicine/Cell Therapy:
Technologies Cell Pouch ™ - Implantable medical device for therapeutic cells;
Therapeutic cells - Donor, stem cells, xenogeneic cells
Sertolin ™ - Local immune protection;
First Product Focus: Insulin-Dependent Diabetes Patient population - 9M NA
Current Products: insulin injections, insulin pumps
Sernova’s Goal: eliminate insulin injections; reduce diabetes side effects Improved patient QOL, reduction in disease side effects, efficacy
Sernova’s Proof and Development Status Cell Pouch™ Proven safe environment for cells
Sertolin™ Proven Local immune protection Therapeutic Cells Proven safety/efficacy for diabetes Status: Phase I/II Ongoing Clinical study – interim results: 2013
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Sernova’s Patent Portfolio
Over 20 issued and pending patents in 5 families:
Composition and use of medical devices for delivery and cell transplantation
Methods for predicting long-term therapeutic cell function
Use of sertoli and genetically modified sertoli cells for treatment of disease
Use of combinations of sertoli cells and other cell types for treatment of disease
In vitro co-culture of mammalian cells with sertoli cells
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DIABETES Sernova’s Cell Pouch™ First Clinical Indication
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6
9M
14.6M
US Market 23.6M Patients
10%
63%
27%
Diabetes Market Diabetes Epidemiology*
Type 1 Insulin-dependent (10%)
Type 2 Insulin-resistant (63%)
Type 2 Insulin-dependent; (27%)
Insulin therapy: 37% (9M US/34M Asia)
Diabetes side effects: heart, kidney, nerve
disease, stroke, amputations, blindness
*American Diabetes Association, 2007
Diabetes Healthcare Costs $174 billion: US cost of diagnosed diabetes in 2007 $116 billion for direct medical costs $58 billion for indirect costs (disability, work loss, premature mortality)
Diabetes Treatments Are Inadequate
Artificial management of blood glucose
Insulin injections, pumps, etc.
Often painful leading to poor compliance (side effects)
Insulin levels spike and decay
Low patient QOL
“Natural” management of blood glucose
Portal vein transplant of islets (Edmonton Protocol)
No compliance issues; normal glucose control
Expensive; Limited in application
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The Current Approach to Islet Transplantation:
The Edmonton Protocol
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Portal vein islet transplantation for patients with hypoglycemia unawareness A study of 36 patients using the Edmonton Protocol: 44% were insulin independent at 1 year 15% insulin independent at 5 years (Shapiro et al. NEJM 355:13, 2006)
Edmonton Protocol - Gap
73%
0 12 24 36 48 60 72 84 96 108
0
20
40
60
80
100
C-peptide positive
Insulin Independence
Corrected HbA1C and absence of hypoglycemia
15%
% S
urv
iva
l
Time (Months)
Improving the Standard of Care of Islet Transplantation
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Catherization laboratory Loss of 60-75% of islets immediately-IBMIR Portal hypertension, thrombosis No possible Islet imaging Expensive procedure Life long systemic antirejection drugs No potential to use unlimited sources of cells such as stem cells/xenogeneic cells
Placed under the skin Outpatient procedure; minimally invasive No IBMIR No Portal hypertension or thrombosis Islet imaging capable Reduced cost Potential for local immune protection Ideal location for stem cell and xenogeneic technologies
Edmonton Protocol Cell Pouch System™
Cell Pouch™ Preclinical Summary
Cell Pouch™ contract manufactured (ISO 13485) Cell Pouch™ is biocompatible (ISO 10993 studies) Safety confirmed in 4 animal models rodent, pig, non-human primate Efficacy Confirmed: isograft, autograft, allograft diabetes models Sertoli cells protect therapeutic cells
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Cell Pouch™: A Natural Environment for Islets
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Subcutaneous Cell Pouch™ Post-Implant
Transplant Site Islet Transplantation into Cell Pouch™
8 Wks Post Cell Pouch™Implant 9 Wks Post Islet Transplant
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Cell Pouch™: Healthy Islets 12 Weeks Post-Transplant
Micro-vessel, Insulin Stain & Glucagon Post Tx
Fluorescence images of a chamber within the Cell Pouch™ stained for micro-vessels (green) and insulin (red) post-transplant. IHC of glucagon DAB staining
Tissue and Micro-Vessel Incorporation
Cell Pouch™ infiltrated with micro-vessels and collagen
Negative Glucagon
Cell Pouch™
Robust Allograft Efficacy Results with a
Marginal Islet Dose
Cell Pouch™ Removal
Tx Cell Pouch™
Removal
*Marginal Islet dose 2500 IEQ/kg
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Healthy Islets within the Cell Pouch™
Post-Allo Tx and Antirejection Regimen
10,000IEQ/Kg 5,000IEQ/Kg
2,500IEQ/Kg
• Insulin (red) and nuclei (blue) staining within one chamber of the Cell Pouch™ post-islet transplant at each transplant dose • Islet survival within the Cell Pouch™ for up to 6 weeks (length of study) while under immunosuppressive drug therapy
Local Sertolin™: Prevention of Graft Rejection
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A B
C D
A B
C D
Sertoli/Islets Islets Alone
Day 7
Day 21
Natural immune-privileged
environment protecting
cells from immune
destruction
A Phase I/II Study of the Safety and
Efficacy of Sernova’s Cell Pouch™ for
Therapeutic Islet Transplantation
Principal Investigator:
A.M. James Shapiro, M.D., Ph.D., FRCS (Eng), FRCSC
www.islet.ca
www.clinical trials.gov
Ongoing Sernova Cell Pouch™ Phase I/II Study Principal Investigator: Dr. A. J. Shapiro (Developer of Edmonton Protocol; U. Alberta)
Study Design 20 patients (unstable diabetes); open-label; single-arm; non-randomized
Donor islet transplantation 2-12 wks post Cell Pouch™ Implantation
Primary endpoint
Safety post Cell Pouch™ implantation and 1month post islet transplantation
Secondary endpoint
Efficacy, proportion of patients insulin independent at 3months post final islet transplant
Follow Up and Timeline
Patient follow-up for 3 years to assess long-term safety and efficacy
Health Canada Cleared – Patient Screening and Enrolment ongoing
Interim safety and efficacy expected - 2013
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Sernova Cell Pouch™ Clinical Development
Plan for international study (Canada, US, Europe, Asia)
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Product Product Strategy
Cell Pouch™, donor islets and state of the art antirejection protocol
POC Cell Pouch™; ≈$50M/yr
Cell Pouch™, donor islets and local immune protection (Sertolin™)
Local immune protection of islets; ≈$150M/yr
Cell Pouch™ and insulin producing stem cells
Unlimited source of insulin-producing cells; ≈$2.3B/yr (1%)
Cell Pouch™ Diabetes Products
Sernova’s Therapeutic Goal:
A Disruptive Approach to Treat Chronic Diseases
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A Natural Cell Environment:
Cell Pouch System™
Safe Prevention of Transplant Rejection Sertolin™
An Unlimited Source of Therapeutic Cells
Sernova
The Cell Pouch™: Product Pipeline
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Cell Pouch™
Diabetes
Type-2 Type-1
Parathyroid
Haemophelia Parkinson’s Disease
Liver Failure
Human Donor Cells Stem Cells; Xenogeneic Cells
2013 Goals and Value Drivers
• Interim clinical results from Phase I/II study • Preparation for a second clinical indication for the
Cell Pouch™, e.g. parathyroid gland • Development of an international regulatory strategy
for commercialization of the Cell Pouch™ for diabetes
• Development of Sertolin™ within the Cell Pouch™ for local immune protection to eliminate need for anti-rejection drugs
• Assessment of insulin producing stem cells and protected xenogeneic cells in the Cell Pouch™
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Management Team and BOD
Core Executive Management
President & CEO - Dr. Philip Toleikis, BA., MSc., Ph.D.
Joined 2009; Pharmaceutical/Device Consultant, V.P. R&D Pharmacology, Angiotech Pharmaceuticals (1996-2006)
Sr. Director R&D – Delfina Mazzuca-Siroen, BSc. Hon. Biochem., MSc.
20 yrs. R&D Mgmt. U. Western Ontario, Transplantation preclinical and clinical development
CFO – Bill Smethurst, CA
S.A.B. -Founding Scientist and Chair - Dr. David White, PhD., M.R.C.P., F.R.C.P., Professor Emeritus Medicine, Robarts Institute , U. of Western Ontario; S.A.B. Dr. James Shapiro, M.D., Ph.D. FRCS (ENG.)
Director of the Clinical Islet Transplantation Program at the University of Alberta and 5 additional members
Board of Directors
Dr. George Adams, MSc., Ph.D. (Chairman); CEO VentriPoint, Inc.
Jeffrey Bacha, BSc., MBA; CEO Del Mar Pharmaceuticals
James Parsons, CA ; CFO/advisory - DiaMedica, Stem Cell Therapeutics, Amorfix, Lorus Therapeutics, YM Biosciences, Astra Zeneca Canada
Bruce Weber, B.S., MBA; VP Clinical/Regulatory and QA Innovia LLC.
Dr. Philip Toleikis, BA., MSc., Ph.D.; President & CEO Sernova Corp.
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Sernova Company Summary A Clinical Stage Company in Regenerative Medicine/Cell Therapy:
Disruptive Technologies Cell Pouch ™ - Implantable medical device for Therapeutic Cells;
Therapeutic cells - donor, stem cells, xenogeneic cells
Sertolin ™ - Local immune protection;
First Product Focus: Insulin-Dependent Diabetes Patient population - 9M NA
Current Products: insulin injections, insulin pumps
Sernova’s Goal: eliminate insulin injections; reduce diabetes side effects Improved patient QOL, reduction in disease side effects, efficacy Clinical evaluation: Ongoing Phase I/II clinical study (Interim results: H1, 2013)
Sernova’s Depth
Experienced: management team, SAB, BOD
KOL Principal Clinical Investigator– Dr. James Shapiro Head of islet transplantation UA
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Contact Information
Dr. Philip Toleikis, President and CEO
Tel: (604) 961-2939
email: [email protected]
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