Innovative Technologies and Platforms: Fueling the Engines of Drug Discovery

Decision Resources, Inc, November 2011

Biotech companies are an important source of technological innovation for creating the next generation of new drugs. Despite a hostile economic environment, numerous biotech companies have emerged, offering exciting technology platforms that span an array of distinct technologies, from novel chemistries to computational approaches to transgenic animal models. In some cases, these companies have devised alternative business and partnering models that enable them to strike alliances to test and validate technology and that ensure a faster return on investment.

Questions answered in this report:

- Although the monoclonal antibody (MAb) marketplace is increasingly crowded, new technologies are emerging that will fuel new development. What technology platforms are emerging that can generate novel MAbs? What advantages do these technologies have over existing ones? Which companies are pursuing their development?

- Pharmaceutical companies are benefitting from innovative technology platforms through corporate partnerships. Which companies are striking deals with Big Pharma to develop their technology platform? What technologies are favored in these deals? How are the structures of these deals changing?

- Biotech companies have devised alternative business and partnering models that enable them to strike alliances that can test and validate technology. Which companies are pursuing alternative models for platform development and licensing? What technologies are involved? Which pharmaceutical partners are joining these efforts?

- Pharmaceutical companies continue to seek new methods of generating small-molecule drug candidates. Which companies are developing novel chemistry and computational technologies to support drug discovery efforts? What innovative biological platforms are supporting small-molecule discovery?

- Biotech companies are developing breakthrough, but yet unvalidated, technologies for drug development. What types of novel, unvalidated therapeutic technologies are emerging? Which companies are developing them?What is the potential of these technologies?

Scope:

- Industry landscape: Funding gap, industry challenges, patent cliff, strategy.
- Transgenic mouse platform: MAb technology, market development, novel platform, novel business and partnering structure.
- Yeast-based antibody discovery platform: Basic technology, novel platform, partnerships.
- Bispecific antibody platform: Technology description, development, partners.
- Epigenetics platform: Technologies, development, collaborations.
- Stapled peptide platform: Technologies, marketed products, collaborations.
- Kinase inhibitor development platform: Technologies, development strategy.
- Synthetic chemistry platform: Macrocyclic technology, development, collaborations.
- Computational chemistry platform: Company development, business model, financing.
- RNA targeted therapy platform: Locked nucleic acid chemistry, technology description, microRNA, partnerships.
- Novel vaccine platform: Virus-like particle (VLP) technology, recombinant vaccines, technology description, funding, development, joint venture.
- Stem cell platform: Induced pluripotent stem cells, technology description, development.
- Outlook: Value of technology platforms, funding gap, length of development, biotech-pharma partnering.

Executive Summary
- Strategic Considerations
- Stakeholder Implications

Introduction

Innovative Technology Platforms and Companies
- Novel Transgenic Mouse Platforms: Ablexis
- Yeast-Based Antibody Discovery Platform: Adimab
- Bispecific Antibodies: Micromet
- Epigenetics: Epizyme
- Stapled Peptides: Aileron Therapeutics
- Kinase Inhibitor Development: Ambit Biosciences
- Synthetic Chemistry: Ensemble Therapeutics
- Computational Chemistry: Nimbus Discovery
- RNA Targeted Therapies: Santaris Pharma
- Novel Vaccine Technologies: Novavax
- Induced Pluripotent Stem Cells: Fate Therapeutics

The Future of Innovative Platform Technology Development

About the Author

- Abgenix
- Ablexis
- Aileron Therapeutics
- Adimab
- Ambit Biosciences
- Amgen
- Astellas Pharma
- Bayer Schering
- Biogen Idec
- Boehringer Ingelheim
- Bristol-Myers Squibb
- Cadila Pharmaceuticals
- Cambridge Antibody Technologies
- Cephalon
- CPL Biologicals
- Ensemble Therapeutics
- Epizyme
- Eisai Pharmaceuticals
- Eli Lilly
- F-star
- Fate Therapeutics
- Fresenius Biotech
- Genentech
- Genzyme
- GlaxoSmithKline
- Human Genome Sciences
- MacroGenics
- Medarex
- Merck
- Merrimack Pharmaceuticals
- Micromet
- Nimbus Discovery
- Novartis
- Novavax
- Novo Nordisk
- Pfizer
- Regeneron
- Roche
- Sanofi-Aventis
- Santaris Pharma
- Schrödinger
- Shire
- Stemgent
- Takeda
- Teva
- Trion Pharma

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