Drug Repositioning Strategies for Refueling the Pipeline

SUMMARY

Introduction:

Drug companies have historically found new indications for their existing drugs serendipitously. Now, however, to refuel product pipelines and improve profi t margins, many companies are turning to low-risk strategies such as drug repositioning. Biotech companies such as Dynogen, BrainCells, Gene Logic, and Xenogen are developing technology platforms that aid companies in the development of systematic approaches to this low-risk strategy.

Questions Answered in This Spectrum Report:

• Many new in vivo, ex vivo, in vitro, and in silico drug repositioning approaches are just now emerging. Which approach is likely to have the greatest success in fi nding unexpected new indications for drugs?
• The pharmaceutical industry is beginning to adopt technologies based on metabolomics-the comprehensive analysis of metabolites. Which companies are pursuing metabolomics as their primary approach to drug repositioning?
• Gene Logic's Phase R Program covers the up-front costs of drug discovery for companies with which it partners. Has Gene Logic's risky strategy been successful in attracting partners? What other partnering strategies are repositioning companies actively pursuing?
• CombinatoRx has developed a high-throughput screening system to evaluate pairs of known compounds for specifi c diseases. What successes has CombinatoRx had in identifying new combinations of known drugs to be used in specifi c diseases?
• An essential part of the business strategy for repositioning compounds is ensuring a strong patent estate. What repositioned drugs are most likely to achieve the strongest patent protection?

Scope:

• In vivo technologies: real-time imaging, zebra fi sh modeling, and multiplexing.
• Ex vivo technologies: kinetic markers and metabolomics.
• In vitro cell-based technologies: genetically engineered cell lines and high-throughput screening.
• In silico technologies: computer simulations and database analyses.
• Repositioning approaches: technology platforms enabling a systematic approach to fi nding new uses for drugs; use of animal models, cell-based assays, computer simulations, and database analyses; pathway-based approaches; the "hypothesis-free" approach.
• Strategies in drug repositioning: trends in repositioning efforts; intellectual property strategies; business models; licensing and collaborative partnerships.
• Outlook: Big Pharma's interest in drug repositioning; the promise of in vivo models; hopes for a powerful technology toolbox; repositioning of branded versus patented drugs.

TABLE OF CONTENTS

• Executive Summary
  • Strategic Considerations
  • Stakeholder Implications
• Introduction
• In Vivo Technologies
  • Real-Time Imaging in Animal Models
  • Zebra Fish Disease Models
  • In Vivo Multiplexing
• Ex Vivo Technologies
  • Kinetic Markers
  • Metabolomics
    • Metabolon
    • Gene Logic
• In Vitro Cell-Based Technologies
  • Bionaut Pharmaceuticals
  • CombinatoRx
  • Gene Logic
• In Silico Technologies
  • Arachnova
  • Inpharmatica
  • Off-label.com
  • Sosei
  • Gene Logic
• Repositioning Within a Therapeutic Area
  • Dynogen
  • Jenken Biosciences
  • Vicus Therapeutics
  • BrainCells
• Strategies in Drug Repositioning
  • Technology Strategies
  • Intellectual Property Strategies
  • Business Models
• Outlook for Drug Repositioning

Tables

• 1. Select Technologies for Drug Repositioning
• 2. Select Companies with Drug Repositioning Technologies or Programs
• 3. Select Collaborations in Drug Repositioning

Figures

• 1. Number of FDA-Approved New Molecular Entities (1997-2006) and Total Global R&D Spending, PhRMA Members (1997-2005)
• 2. In Vivo and Ex Vivo Technologies for Drug Repositioning
• 3. In Vitro, Cell-Based Assays for Drug Repositioning
• 4. In Silico Technologies for Drug Repositioning