Strong patent protection is essential for a start-up biotechnology company and can be a valuable company asset. However, it is also expensive, with costs ranging from tens to hundreds of thousands of dollars over time. This session will focus on how to get the most out of your patent dollars. This session presentation is available in audio format here: http://www.marsdd.com/bioent/dec4

1. Intellectual Property
What Do Investors Look For?
Dr. Andre Uddin
VP Strategic Development
Transition Therapeutics Inc.
Page 1 of 17

2. Criteria for
Biotech Investment
! Excellent Management
! Strong Balance Sheet (2 years of cash)
! Products Targeting Large or Untapped Markets
! Strong Pipeline of Products
! Solid Intellectual Property
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3. Other
Considerations
! Clinical Data ! Diseases
! Clinical Trials ! Health Care Savings
! Competitors ! Manufacturing
! Product Development program ! Technology Platform
Page 3 of 17

4. Only the Paranoid Survive
“ When business andofthe old ways of competing,structure, from the old ways of
the balance forces shifts from the old “
doing to the new.
- Andrew S. Grove
Business goes onto
new heights
Disruptive forces
Value
Creation
Business declines
Time
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5. The Pharmaceutical Industry
Strategic Inflection Points 1820 to
present
1820~1880 Natural Products have active principles
1880~1930 Isolation of chemicals from plants, dyes and organic chemistry
1930~1960 Large scale synthesis
1960~1980 Determination of Mechanisms of Action
Chemical Transmitters
Biological Receptors
1980~2000 Advent of Molecular Biology
Monoclonal Antibodies
Drug Delivery
Genomics
2000~Present Convergence of Pharmaceuticals/Medical Devices
RNA interference (RNAi)
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6. Pharmaceutical
Life Cycle
Patent Expiration
Generics
Launch
les
a
S
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7. Building
A Patent Estate
Typical Pharma Patent Portfolio
High
NCE Patents
Mechanism of Action Patents
VALUE
Method of Treatment Patents
Formulation Patents
NCE Salt/Solvate/Polymorph/Crystalline habit Patents
Process Patents
Gene Patents
Low
Source: GlaxoSmithKline
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8. Typical Modern Pharma
R&D Process
Source: GlaxoSmithKline
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9. Successful Pharmaceuticals
with Exclusivity/Method of Use Patents
Company Drug Use Sales (USD)
BMS Taxol Cancer > $1B/yr (peak)
GSK AZT AIDS $700M/yr (peak)
Astellas Adenocard (adenosine) Cardiac $285M/yr (2004)
Celgene Thalidomide Cancer $418M/yr (current annual run rate)
KOS Pharma Niaspan (niacin) Cholesterol $441M/yr (2005)
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10. Regulatory
Exclusivity
A fixed period of time within which a regulatory authority will not allow approval
of a generic abbreviated/abridged application of an approved drug
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11. A Cautionary Tale
“ In science the world, goestotothe man towho the
the credit the man “
convinces not whom
idea first occurs.
- Sir William Osler
! Dr. Horwitz, synthesized AZT in 1964 as a potential anticancer agent -
didn’t work
! Developed and sold under a patent once owned by GlaxoSmithKline
! AZT became a highly successful anti-HIV drug (peak sales of US$700M)
! Dr. Horwitz nor his university ever saw a penny
“Missed Chances” Goldie Blumenstvk, Chronicle of Higher Education
Chances” Blumenstvk,
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12. Anything is Possible
Highly Valued Patents
“ It is harddeserves timeworn impact of PCR…
to exaggerate the
actually superlatives like
“
quot;revolutionaryquot; and quot;breakthrough.”
- Tabitha M. Powledge
Case Study: PCR
! 1983 Dr. Kary Mullis at Cetus Corporation conceives of Polymerase Chain Reaction
(PCR). PCR is a simple yet elegant process that enables the production of virtually
unlimited copies of genetic material in the laboratory.
! 1985 Cetus files first PCR patent application. First publication of PCR by Cetus
appears in Science.
! 1987 Cetus is awarded fundamental patents for PCR.
! 1989 Cetus agree to begin joint development of diagnostic applications for PCR.
! 1991 PCR patent and related technology sold by Cetus to Roche for US$300M
! 1993 Dr. Kary Mullis shares Nobel Prize in Chemistry for conceiving PCR technology
Source: Roche Page 12 of 17

13. Anything is Possible
Highly Valued Patents
Case Study: RNAi
! 2005 Sirna Therapeutics first company to advance RNAi compound
into human trials
! April 3, 2006 Sirna Therapeutics Granted First Broad siRNA Patent
for a Gene Target in the United States
! Present - Sirna has 52 issued patents and more than 270 pending
patents in the area of RNAi
! October 30, 2006 Merck purchases Sirna for US$1.1 BN
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14. Patent
Enforcement
“ A manofhad better his family by influenza, to him in thisdispute
have anything happen world
short losing all than have a
“
about a patent.
- 1892 Quote from Master of the Rolls (Head of English C of A)
“ Irather as a casino. the law courts not as a cathedral but
have come to regard
“
- 1977 Quote from Editor of Private Eye
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15. Anything is Possible
Highly Valued Patents
Angiotech/Boston Scientific’s Paclitaxel coated stent
! 1998 Angiotech licenses paclitaxel-coated stents to
Cook/Boston Scientific
! 2004 Angiotech/Boston Scientific launched TAXUS in US
- peak sales of US$2.6BN
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16. Anything is Possible
Highly Valued Patents
Case Study: Getting around Angiotech’s
Paclitaxel coated stent patents
! 1999 Conor founded to develop stents which have drug reservoirs in their struts
! February 1, 2005 BSX/Angiotech sues Conor for patent infringement
! February 18, 2005 Conor Medsystems Files lawsuit to revoke Angiotech patent
! February 17, 2006 Conor receives CE mark for its CoStar paclitaxel Drug-Eluting Stent
! February 24, 2006 U.K. High Court of Justice ruling invalidates Angiotech Patent on
Paclitaxel Coated Stents
! November 16, 2006 JNJ announces the purchase of Conor Medsystems for
US$1.4BN
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17. Key Rules
for Patenting
1. Avoid early public disclosure. File first, then disclose.
2. Do not publish interim results
3. Research the market and technical novelty of an invention before patent filing
4. If you cannot protect the patent from infringement, don’t file
5. File in the US first
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