The Development and Commercialization of New Technologies Discovery Lecture Series Purdue University...

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


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


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



throughput


exposure



TargetDiscovery


LeadOptim. ADMET

ClinicalDevelop.

Regis-tration.

2-3 yr 0.5-1 yr 1-3 yr 1-2 yr 5-6 yr 1-2 yr






3. 4 Things Every Start-Up Must Get Right

1. Technology

2. Strategy

3. Team

4. Money


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”


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


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!


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


2. Strategy: Drug Repositioning

Ashburn & Thor, Nature Reviews Drug Discovery, Aug, 2004, pg 673-683

Idea! Drug




throughput




exposure

TargetDiscovery


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




•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


1-2 yr

Develop-ment

Regis-tation.


“Been there, done that,” a big plusLeadershipVC’s want Wow! Leadership Teams

3. Team


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


4. Money: Midwest-Focused VC’s

Teri F. Willey

[email protected]

Firm/Meeting Contact

Mina Patel [email protected]

Scott R. Naisbitt, M.D., [email protected]

Michael [email protected]

www.investmidwestforum.com


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

The Development and Commercialization of New Technologies Discovery Lecture Series Purdue University...

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