A Serial Entrepreneur’s Journey from Concept

to Customers

George Jackowski, Ph.D., KCTJ

October 19th, 2015

“Maintaining the Heartbeat”

Chairman of NAVA Corp.

BIOGRAPHY

Academic Appointments Held:

• University of California, San Francisco (USA)

• Cardiovascular Research Institute, San Francisco (USA)

• University of Toronto, Department of Laboratory Medicine & Pathobiology (Canada)

• Research Institute, Hospital for Sick Children (Toronto, Ontario, Canada)

• Florida Atlantic University, The Centre for Molecular Biology & Biotechnology (Boca Raton, Florida, USA)

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HISTORY

• Over the last 35 years in the Healthcare/Biotech sector raised over $150 Million from the public capital markets

• Federal Government support (IRAP): approximately $2 Million

• Over 170 US Patents issued and filed

• Created over 1,000 jobs in a hard-to-fund sector

• Created numerous new biotech companies with company valuations in the range of millions to $500 Million

• Over 25 products cleared through the United States Food & Drug Administration (FDA) and commercialized globally

• >$100 Million in transactions with Big Pharma→ Baxter, Dade, J&J, Ortho, Roche, Abbott, Fisher, Becton-Dickenson,

Genzyme, Nanogen, Schering-Plough

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COMMERCIAL FOCUS OF COMPANIES CREATED

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Commercial Focus

Cardiac Panel PCR Diagnostics/ Therapeutics

Biomarker, Proteomics, Diagnostics Pain Therapeutics

BioWrap Oral DNA Therapy

Cancer Antibodies Reprofiling

Biogenerics Stroke Diagnostics

Blood Purification Alzheimer’s Disease

Biological Chips/Wireless transmission/

Safety ProfilesHeart Failure Diagnostic

Point-of-Care DeviceSpecialty Pharma – Omega-3 Therapies

COMMERCIAL POTENTIAL

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Need to know how to identify and when to go forward with basic laboratory discoveries that have potential for commercial development.

CURRENT STATE OF DRUG DISCOVERY

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• The worldwide pharmaceutical market is tracking to come in at US $1,100 Billion (Growth of 3-6% over the last five years)

• By 2020 ~ $3 Trillion US

• To achieve this growth pharma needs fundamentally new drugs to market – blockbusters

• Dependent:

– Identifying novel drug targets

– Embracing new technologies

• Genomics & Proteomics based drug discovery• Screening unique cell lines• Vaccines• Hormone based cancer therapies• Immunotherapies

– Propelling the related therapeutics to market

• Changing market conditions – previously only Phase II or greater deals, now looking at preclinical data

Cancer Therapeutics (1970 – 80’s):

• EGF/Receptor

• TGF/Receptor

• VEGF/Receptor

• Nuclear lamins

• cis and trans acting factors

• Ribonucleoprotein

• Heat shock proteins

• Erythropoietin (Epogen)

• Herceptin

• Neupogen/Neulasta

FOR OUR CURRENT DRUGS AND DIAGNOSTICS THE BASIC SCIENCE COMMERCIALIZED GOES BACK APPROXIMATELY 30 YEARS

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FOR OUR CURRENT DRUGS AND DIAGNOSTICS THE BASIC SCIENCE COMMERCIALIZED GOES BACK APPROXIMATELY 30 YEARS

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Cardiovascular (1970-80’s):

• Histone acetylase – PPARS (glitazones)

• Protein kinase C (Calcium channel blockers)

• Brain natriuretic peptides

• Lipid regulators – cholesterol-lowering statin drug

• Cardiac enzymesSource: https://thernhangout.wordpress.com/

FOR OUR CURRENT DRUGS AND DIAGNOSTICS THE BASIC SCIENCE COMMERCIALIZED GOES BACK APPROXIMATELY 30 YEARS

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Proteomics:

• Mass spectrophotometers – chemist in the 70’s

• Gels: Laemmli (1970) Nature

O’Farrell (1975) J. Biol. Chem

Genetics (1990-2000’s):

• Cancer phenotypes and mutated genes (BRAC1 & BRAC2)

Source: http://www.surgery.hku.hk/images/br01.jpg

WHY?

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• Medical progress tends to move along at a slow and steady pace

• Impact of big advances on the art and practice of medicine is usually incremental

E.G. CHOLESTEROL-LOWERING STATIN DRUG (HMG-COA REDUCTASE INHIBITORS)

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• Discovered by Prof. Akira Endo ~1971 seeking a natural substance to block crucial enzyme involved in the body’s production of cholesterol

• At the time, many scientists were skeptical about the safety of lowering the amount of cholesterol

• Merck &Co. was the first to commercially market statins (Lovastatin – beginning research in 1976)

• 1980 demonstrated that statin lowered LDL or “bad” cholesterol levels

• Lovastatin was approved by the US FDA in 1987

• Many players have entered the market – E.g. AstraZeneca, Bristol-Myers Squibb, Novartis, Pfizer

• Merck loses patent protection in 2006

• Most widely prescribed drug for the prevention of heart attacks and stroke – approximately $30 Billion per year

• Prof. Akira Endo was awarded Japan Prize (2006) for his work on the development of statins and the Lasker Foundation Prize (2008)

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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Intellectual Property:

• Patents: US patents vs. National

• Freedom-to-Operate

• Invalidation

• Patent Strategy

• Pioneer patents and picket fence

• Sleeper/Submarine patents

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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The Inventor, The Science:

Breakthrough (Revolutionary Advances) vs. Innovated

• Paradigm shifts

• Simple application of technology that has worked

• Me too

Source: The Economist, Aug. 8, 2015

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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The Product:

• True market

• Competition

• Cost of manufacturing

• Regulatory hurdles – strategy

• Distribution channels

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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The Structure of the Company:

• Funding and Taxes

The Exit Strategy:

• Valuations and Making Money

The Team:

• Beyond the Inventor

• Different people for the different stages of the Company

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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Regulatory, Preclinical and Clinical Trials:

• Contract Research Organization

• Contract Manufacturing

• Contract Trial Organizations

• Marketing and Commercialization

• Payor Strategy

CREATING AN EDGE FOR COMMERCIALIZATION IN REAL-TIME

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Key Elements:

• Dramatic result in a well characterized population of patients

• Dramatic results with well defined endpoints

• Tissue specific biomolecules

• Collaborate (Preclinical models, contract manufacturing/

development)

• Don’t fall in love with your technology or approach to

commercialization

• Go as far as you can within the academic research

environment

TECHNIQUES TO EXPEDITE THE CLINICAL IMPACT OF DISCOVERY

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• Antisense

• iRNA

• Proteomics – protein – protein interaction – pathway elucidation

• Patient profiling

• Genomics

• Knock outs/in

• Models mimicking man

• Laser scanning microscopy:

– receptor interaction - pathway delineation

• Clinical samples, biopsies, serum

IMPACT OF BASIC RESEARCH

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Fundamental Discovery• Decades to commercialization• Protein pathways, new concept, cutting edge

E.g. Nanotechnology, Statins

Applied Discovery• Decade to commercialization• Disease related biomolecules

E.g. Cardiac Panel CK-MB, Myoglobin and Troponin

Platform• Years to commercialization• Screening drugs against series of protein targets

E.g. Bioactivity Assay

ACCEPTANCE

STANDARD OF CARE

FACILITATE DRUG SCREENING

YOUR MOTIVATION FOR GOING DOWN THE COMMERCIAL ROAD

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• Notoriety for the discovery – “Golden Hand Cuffs”

• Seeing your discovery (product) in the market and people benefiting from it

• Money – Short-term or long-term

• Control/willing to share

QUESTIONS TO BE ASKED BY YOU

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1. Can a patent portfolio be built around the technology?

2. Is the technology robust with well defined endpoints?

3. Can it be manufactured at a low cost of goods?

4. Regulatory strategy for commercialization?

5. Does a market exist or does it have to be created?

KEY TO COMMERCIAL DEVELOPMENT

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• Network, Network, Network – Collaborate

• Move the discovery forward as quickly as possible

Commercialization

R & D$

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Thank you

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