Abstract
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.
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