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Repurposing Strategies for Therapeutics


Drug repurposing refers to the development of existing drugs for new indications. These drugs may have (i) failed to show efficacy in late stage clinical trials, without safety issues; (ii) stalled in development for commercial reasons; (iii) passed the point of patent expiry; or (iv) are being explored in new geographical markets. Over the past 5 years, pressure on the industry caused by the ‘innovation gap’ of rising development costs and stagnant product output have become major reasons for the growing interest in drug repurposing. Companies have emerged offering a variety of broad platforms for identifying new indications, and some have been successful in building their own pipelines of candidates for which the risks and timelines associated with further clinical development are reduced. The business models and platforms offered by these companies will be validated if they are able to generate positive proof-of-concept clinical data for their repurposed compounds. In the current economic climate, two factors may continue the flow of compounds into repurposing projects. First, the need to expand product pipelines with new projects has become more acute, especially with projects for which an element of the risk has been removed. Second, the pool of potential compounds abandoned for strategic reasons is growing. These factors may continue to provide opportunities for academia as well as a small number of successful indications discovery companies. Moving forward, the pharma industry is looking to explore the full potential of their compounds at a much earlier stage, perhaps even in late preclinical development, offering additional opportunities for these companies.

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The authors thank the following representatives of industry and academia who gave their time through interviews during the preparation of this article: Andrew Reaume, Chief Executive Officer, Melior Discovery; Jason Levin, Chief Business Officer, Brain Cells Inc.; and Kate Marusina, Manager, Research Facilitation and Industry Alliance, Clinical and Translational Science Center, University of California Davis School of Medicine.

No sources of funding were used to assist in the preparation of this article. The authors have no conflicts of interest that are directly relevant to the content of this article.

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Correspondence to Cheryl L. Barton.

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Sleigh, S.H., Barton, C.L. Repurposing Strategies for Therapeutics. Pharm Med 24, 151–159 (2010).

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