Drug repurposing companies
Rather than chasing new compounds, pharmaceutical companies can reduce risk and costs by finding new uses for existing products
Daniel Grau, M.Phil, and George Serbedzija, PhD
Grau is vice president of commercial operation and Serbedzija is associate director of corporate development for CombinatoRx Inc., Boston.
In recent years, an increasing number of biotechnology companies have been focusing on drug repurposing, the development of novel uses for existing drugs. Although repurposing is not new to the pharmaceutical industry—large companies using classical life-cycle management strategies often extend drug use into new indications to preserve or extend the value of a pharmaceutical brand—the emergence of companies founded exclusively on repurposing reflects a general trend evident in biotechnology today that seeks to reduce the risks of drug development. Three of the most prevalent strategies for seeking to reduce the risks of drug development are (a) developing new formulations of existing drugs, (b) in-licensing of clinical stage programs, frequently the core strategy of so-called NRDO (no research, development only) companies, and (c) repurposing of existing drugs or discontinued compounds to identify novel uses or therapeutic properties.
Repurposing companies fit naturally within the risk reduction model because often they start with approved compounds with established safety and bioavailability profiles, proven formulation and manufacturing routes, and well-characterized pharmacology. Theoretically, repurposed compounds can enter clinical testing more rapidly, and at less cost, than new chemical entities. But all risk reduction strategies are not created equal. There is a striking difference between formulation companies that develop new deliveries for old drugs, or those that in-license clinical stage programs from pharmaceutical companies, and today's newest repurposing groups. In contrast to new delivery companies, some of today's repurposing companies are distinguished by their use of proprietary discovery engines and their focus on discovering new therapeutic properties and mechanisms. In contrast to NRDO companies, leading repurposing companies leverage a research-based lead generation capability. This capacity can involve wet biology or entirely in silico methods. Although the precise mix of research tools may vary, several repurposing companies have created proprietary discovery engines capable of bringing new drug candidates into clinical development on an ongoing basis. By virtue of their research-based discovery engines, these repurposing companies have the potential to expand and refresh their product portfolios.
click the image to enlarge
A matrix representation of the drug development landscape as a function of risk. (Source: CombinatoRx)
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