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Revolutionizing Vaccines Dr. J. Joseph Kim President & CEO

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September 2013 Corporate Presentation

Transcript of Inovio sep13pres

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Revolutionizing Vaccines

Dr. J. Joseph Kim President & CEO

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Forward Looking Statement

Our commentary and responses to your questions may contain forward-looking statements, including comments concerning clinical trials and product development programs, evaluation of potential opportunities, the level of corporate expenditures, the assessment of Inovio’s technology by potential corporate partners, capital market conditions, timing of events, cash consumption and other subjects. Information concerning factors that could cause actual results to differ materially from those set forth in our Annual Report on Form 10-K for the year ended December 31, 2012, our Form 10-Q for the quarter ended June 30, 2013 and other regulatory filings from time to time.

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Overview

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Inovio’s Technology

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Plasmids

Electroporation device

• DNA vaccines

• Electroporation delivery

• Best-in-class immune responses

• Favorable safety profile

• Over 400 patents

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Products

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• Targeting cancers and infectious diseases

• Multi-billion dollar healthcare markets

• Lead program for

HPV-caused disease in phase II

• First efficacy data in mid-2014

• Multiple clinical trials in phase I

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• Collaborating with a global leader in innovative cancer drugs • Develop and commercialize Inovio’s prostate cancer (INO-

5150) and hepatitis B (INO-1800) immunotherapies • $10 million up-front payment • $412.5 million milestone payments for certain development

and commercial events • Roche may pay other development milestone payments if it

pursues other indications with INO-5150 or INO-1800 • Roche to fund all ongoing development costs • Up to double-digit royalties on sales of a marketed product

Roche Partnership

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Validation

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• Advancing partnering discussions with large pharma

• Gates-funded malaria program

• US gov’t $25M grant for HIV vaccine development

• NIH Director: “Transformational Research” grant • Almost $60 million in

non-dilutive grants in past few years

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Strategy

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• Establish product proof-of-principle with phase I and II clinical trials

• Spread cost/risk & advance development & commercialization: o R&D grants

o “Sponsored”

clinical trials

o Partnerships

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Why Revolutionize Vaccines?

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Stimulating the Immune System: A Powerful Legacy

• 1776: concept of “modern” vaccination

• Effective vaccines

against 20+ diseases cowpox anthrax

polio

measles Edward Jenner Louis Pasteur Maurice Hilleman

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#1 Medical Invention

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INFANT MORTALITY RATE

WORLDWIDE LIFE EXPECTANCY

• Reduced child mortality

• Increased life span

• Protected billions from sickness and death

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Extending the Legacy

• Concept of stimulating immune system as relevant today as ever

• Can we create 21st century technology to fight today’s cancers & challenging infectious diseases?

• Yes!

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How do we Revolutionize Vaccines?

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Immune Stimulation in the 21st Century

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• Synthetic • Not a

weakened, killed, or part of a virus

• Therapeutic

• Not preventive only

• Universal

• Not protective against only a single, matched strain

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Inovio’s Synthetic DNA Vaccines

• Contain DNA code for target disease antigen(s)

• Body produces antigen

• Cannot replicate

• Closest to body’s natural

immune response

• Preventive antibodies • Therapeutic T-cells

• Formulated in water • Stable at room temp

Immune response to last century conventional vaccine

antibodies

Immune response to 21st century DNA vaccine

T-cells

antibodies

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Novel Consensus Design

• Use gene sequences from multiple strains or types of target disease antigen

• Create new antigen DNA

sequence to help the body recognize: • “Self” made cancer

cells

• Break tolerance

• Similar but unmatched strains

• Universal, cross-strain protection

• Novel DNA sequences

patentable

Differentiate cancer cells from “self”

Multi-strain protection within Pathogen families

New synthetic consensus sequence

Multiple unique strains

Break Tolerance Universal Protection

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Efficient DNA Vaccine Manufacturing

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• DNA plasmid production

• Bacterial fermentation process

• Efficient, fast, cost effective, scalable

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DNA Delivery: Electroporation

Electric fields applied Vaccine injection

• Overcome two decade hurdle of DNA delivery

• Millisecond electric pulses create pores in cells

• Increase vaccine

uptake 1000X

• Enables cells to produce target antigen

• Widest and deepest global patent estate

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Cellular vaccine uptake Cell produces coded antigen

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Raising the Bar: Best in Class Immune Responses

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Functional T-Cell Responses

• Highest magnitude of T-cell responses

• 83% response rate in highest dose group

• 92% of responders showed 9 month durability

• 91% of responders showed killing effect against target cells

• HIV study: 89% response rate with robust T-cells

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Universal Immune Responses

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• Protective HAI titers in humans against 9 unmatched strains of H1N1 flu from last 100 years

• Strong HAI titers in humans against 6 unmatched strains of deadly H5N1 flu

• Protection against

newly emergent, pandemic-potential H7N9 influenza in mice challenge study

Conventional vaccine: single matched strain only

DNA vaccine: multiple unmatched strains

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Broad Medical and Market Opportunities

Product Name

INTERNALLY FUNDED

Indication Preclinical Phase I Phase II

Cervical dysplasia Vgx-3100

Prostate cancer Ino-5150

Breast/lung cancers Ino-1400

EXTERNALLY FUNDED

hiv pennvax®

influenza Ino-3510

Hepatitis C Ino-8000

Hepatitis B ino-1800

malaria MaV-12

Milestones

Therapeutic

Therapeutic

Therapeutic

Therapeutic

Preventive

Preventive

Preventive/Therapeutic

Therapeutic

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1H 2014 Initiate phase I/IIa

Mid-2014 Phase II study data

2014 Initiate phase I/IIa

2013 Initiate PENNVAX – GP phase I study

2013 Phase I data reported

4Q 2013 Initiate phase I/IIa

2014 Prepare phase I/IIa

2014 Initiate phase I/IIa

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VGX-3100 • Capitalizes on Inovio’s ability to generate T

cells • Immunotherapy for pre-cancers & cancers

caused by human papillomavirus (HPV) • Phase II on-going: high grade cervical pre-cancers (CIN 2/3 dysplasia) • Projected efficacy data: mid-2014

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Broad Medical and Market Opportunities

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Cervical Cancer 12,357 new cases 3,909 deaths

CIN 2/3 dysplasia 300-400K new cases

CIN 1 dysplasia 1.4M new cases

Head & neck cancer (HPV Related) 20,000 new cases 7,922 deaths (oropharyngeal only)

Anogenital cancers (HPV related ) - Excluding cervical

7,931 new cases 2,396 deaths

U.S. Market opportunity ~ 70% of high risk cervical pre-cancers & cancers caused by HPV Type 16 and 18

Therapeutic HPV

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VGX-3100

Therapeutic HPV

• Diseases caused by HPV Type 16 and 18; Target antigens: E6 and E7

• 18 “healthy” patients with

prior CIN 2/3 dysplasia

• Robust immediate T-cell response, average across dose groups/78%

• Dose response

• 92% of responders showed 9 month durability

• 91% with killing effect

• Safe & well tolerated

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Phase I Trial

Results

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Next steps

Therapeutic HPV

Top-line efficacy data expected mid-2014

• Study protocol: minimum 148 patients with CIN 2/3

• Enrollment completed • Randomized, double-blinded,

placebo controlled

• More than 25 sites in 7 countries • Primary endpoint: regression to

CIN 1

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Phase II Trial

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HPV Product

Franchise Planning:

• CIN 2/3 phase III

• Other HPV-related indications: initiate phase IIs • Orphan designation potential

Therapeutic HPV

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power of our people

• Management • Board of Directors • Scientific Advisory Board

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Management

anthrax

polio

cowpox

Louis Pasteur

J.Joseph Kim, PhD President & CEO

• Decades of biotechnology/pharma management

• Merck: hepatitis A and B vaccines manufacturing; HIV vaccine (Ad5) R&D

Niranjan Y. Sardesai, PhD COO

• Extensive biotech management and product development experience

• Led development of diagnostics for mesothelioma, bladder cancer, and ovarian

cancer for Fujirebio Diagnostics

Peter Kies CFO • Ernst & Young • Experience with growth companies

Mark L. Bagarazzi, MD CMO • Clinical research experience incl. Merck • Led clinical/regulatory for shingles and rotavirus vaccines; DNA vaccine expert

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Board of Directors

anthrax

polio

cowpox

Louis Pasteur Simon X. Benito

• Former Senior Vice President, Merck Vaccine Division

Angel Cabrera, PhD • President, George Mason University

• Former President, Thunderbird School of Global Management

J.Joseph Kim, PhD • President & CEO, Inovio

Adel Mahmoud, PhD • Professor, Princeton University • Former President, Merck Vaccines • Responsible for Gardasil®, Zostavax®, Proquad® and Rotateq®

Avtar Dhillon, MD Chairman, BOD

• Former President & CEO, Inovio Biomedical

Morton Collins, PhD • General Partner, Battelle Ventures and Innovations Valley Partners

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Scientific Advisory Board

anthrax

polio

cowpox

Louis Pasteur Thomas S. Edgington, MD

• Founded multiple biotech companies; extensively published

• Emeritus Professor, Scripps Research Institute

Anthony W. Ford-Hutchinson, PhD • Former SVP, Vaccines R&D, Merck

• Oversaw development: Singulair®, Januvia®, Gardasil®, Zostavax®, Proquad® and Rotateq®

Stanley A. Plotkin, MD • Developed rubella and rabies vaccines • Oversaw Sanofi flu vaccine • Emeritus Professor, Wistar Institute & University of Pennsylvania

David B. Weiner, PhD Chairman

•“Father of DNA vaccines” • Dept. of Pathology & Laboratory Medicine,

University of Pennsylvania

Philip Greenberg, MD • Expert in T-cell immunology • Head, Immunology Program, Fred Hutchinson Cancer Research Center

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Stock Price

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Financial Information

Cash, cash equivalents & short-term investments1 $ 23.6 M

Debt1 0 M

Cash runway 3Q 2015

Issued & outstanding shares2 190.8 M

Recent price3 $2.33

Market cap3 $444.6 M

NYSE MKT: INO

1June 30, 2013 3 Sept. 19, 2013

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8/19/2013 9/19/2013

Additional cash raised2

$ 11.4 M

2Aug. 9, 2013

Roche up-front payment $ 10 M

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Investor Highlights • Break-through immune therapy with the power to save lives and maximize shareholder value

• Targeting broad range of diseases and billion dollar markets • Best-in-class T cells to prevent, treat & cure cancers and infectious diseases

• Phase II efficacy data coming

• Validating partnership with Roche

The Opportunity

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Bernie Hertel Senior Director, Corporate Communications 858-410-3101 [email protected]

Investor Contact

investor contacts

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