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The Development and Commercialization of New Technologies
Discovery Lecture SeriesPurdue University
Ted T. Ashburn, MD, PhDSenior Director, Corporate Development Genzyme [email protected]
November 9, 2007
2© 2007 Genzyme Corporation. All rights reserved.
Outline
1. Stages of Development for New Medicines
2. Genzyme Deal Criteria
3. 4 Things Every Start-Up Must Get Right
4. Key Takeaways
3© 2007 Genzyme Corporation. All rights reserved.
1. The Pharmaceutical Value Chain
Ashburn & Thor, Nature Reviews Drug Discovery, Aug, 2004, pg 673-683Gilbert, Henske & Singh, IN VIVO, Nov, 2003
• 10-17 years, $1.7 billion+ process• > 75 different disciplines• < 10% overall probability of success once a
candidate enters clinical trials!!!
Idea! Drug
• Testing starts at Phase I (Phase I/II for cancer)
• In vitro• Ex vivo• In vivo• In silico• High
throughput
• Bioavailability• Systemic
exposure
• Traditional Med. Chem.
• Rational drug design
TargetDiscovery
Discovery& Screening
LeadOptim. ADMET
ClinicalDevelop.
Regis-tration.
2-3 yr 0.5-1 yr 1-3 yr 1-2 yr 5-6 yr 1-2 yr
• U.S (FDA)• E.U. (EMEA)• Japan (MHLW)• Rest of World
• Expression analysis
• In vitro function• In vivo
validation• Bioinformatics
4© 2007 Genzyme Corporation. All rights reserved.
2. Genzyme Deal Criteria
Significant Unmet Medical Need– Rare diseases– New Standard of Care
Risk-reduced Opportunities– Human Proof of Concept or later– Clear Regulatory pathways
Focused Call Point(s)– Not PCP’s
Partnerships– Desire to work together to create value– Both Regional and worldwide
Idea! Drug
• Testing starts at Phase I (Phase I/II for cancer)
• In vitro• Ex vivo• In vivo• In silico• High
throughput
• Bioavailability• Systemic
exposure
• Traditional Med. Chem.
• Rational drug design
TargetDiscovery
Discovery& Screening
LeadOptim. ADMET
ClinicalDevelop.
Regis-tration.
2-3 yr 0.5-1 yr 1-3 yr 1-2 yr 5-6 yr 1-2 yr
• U.S (FDA)• E.U. (EMEA)• Japan (MHLW)• Rest of World
• Expression analysis
• In vitro function• In vivo
validation• Bioinformatics
5© 2007 Genzyme Corporation. All rights reserved.
3. 4 Things Every Start-Up Must Get Right
1. Technology
2. Strategy
3. Team
4. Money
6© 2007 Genzyme Corporation. All rights reserved.
1. Technology: Introduction
Either fills a current need (pain or greed) or creates a new market
VC’s like technologies that make you say Wow!
New technologies fall into 3 buckets:1. “Wow! Let’s build a company around this
technology!”2. “Interesting, you should try to license it to Big
Rx or one of our portfolio companies”3. “Thanks for coming in to see us today”
7© 2007 Genzyme Corporation. All rights reserved.
1. Technology: Patent Requirements
New or Novel: Two part test (US)– Cannot have been known, used or described by
others – Must not have been known more than one year prior
to filing the U.S. patent
Useful– In-vitro/vivo test results proving potential for
human application Non-obvious
– Prove the invention is something more than an ordinary practioner in the field would have been able to do
8© 2007 Genzyme Corporation. All rights reserved.
1. Technology: Intellectual Property
Issued patent allows you to exclude others from selling your invention (Barriers to Entry)– Composition of Matter (COM)– Method of Use (MOU)
Do you have the ability to sell your invention without infringing someone else's patent (Freedom to Operate)?
Key safety tip: Don’t disclose until you file!
9© 2007 Genzyme Corporation. All rights reserved.
Repositioning
Reformulating
In Licensing
De Novo
Small Markets*
• For example, rare diseases or diseases primarily incident in developing nations; government regulations have been enacted to reduce risk and/or raise potential reward for some small markets, e.g., orphan drug status
• Ashburn & Thor, Nature Reviews Drug Discovery, Aug, 2004, pg 673-683
Proteins
Risk ( Target Validity, Druglike Properties & the Development Pathway)
Reward ( Time to Market, Differentiability &
Revenue)
Low Hi
Hi
Low
2. Strategy: Risk vs. Reward Trade Offs
10© 2007 Genzyme Corporation. All rights reserved.
2. Strategy: Drug Repositioning
Ashburn & Thor, Nature Reviews Drug Discovery, Aug, 2004, pg 673-683
Idea! Drug
• Traditional Med. Chem.
• Rational drug design
• In vitro• Ex vivo• In vivo• In silico• High
throughput
• U.S (FDA)• E.U. (EMEA)• Japan (MHLW)• Rest of World
• Testing starts at Phase I (Phase I/II for cancer)
• Bioavailability• Systemic
exposure
TargetDiscovery
Discovery& Screening
LeadOptim. ADMET
Develop-ment
Regis-tation.
2-3 yr 0.5-1 yr 1-3 yr 1-2 yr 5-6 yr 1-2 yr
• Expression analysis
• In vitro function• In vivo
validation• Bioinformatics
•3-12 year process
•Reduced Safety & PK uncertainty
Drug
• Licensing• Novel I.P.• Both
• Targeted searches
• Novel insights• Specialized
Screening platforms
• Serendipity
• May start at Preclinical, Phase I or Phase II
• Ability to leverage existing data packages
AcquisitionIdentification
1–6 yr0–2 yr1–2 yr
• U.S (FDA)• E.U. (EMEA)• Japan (MHLW)• Rest of World
1-2 yr
Develop-ment
Regis-tation.
11© 2007 Genzyme Corporation. All rights reserved.
“Been there, done that,” a big plusLeadershipVC’s want Wow! Leadership Teams
3. Team
12© 2007 Genzyme Corporation. All rights reserved.
4. Money: Where & How Much?
Government–SBIR–STTR
FoundationsCorporationsVenture Capital
Must raise enough to reach next value step up and have 6-9 months of runway–Identification of a lead–I/POC/III/NDA/Launch
The higher quality the source, the better–VC’s want to be a part
of Wow! syndicates
13© 2007 Genzyme Corporation. All rights reserved.
4. Money: Midwest-Focused VC’s
Teri F. Willey
Firm/Meeting Contact
Mina Patel [email protected]
Scott R. Naisbitt, M.D., [email protected]
Michael [email protected]
www.investmidwestforum.com
14© 2007 Genzyme Corporation. All rights reserved.
4. Key Takeaways
1. Drug discovery & development is a long, expensive & arduous process
2. The Industry is Hungry for Innovation3. Every Start-Up must get 4 things right
• Technology• Strategy• People• $
The Development and Commercialization of New Technologies
Discovery Lecture SeriesPurdue University
Ted T. Ashburn, MD, PhDSenior Director, Corporate Development Genzyme [email protected]
November 9, 2007