Webinar: Drug Repositioning Through Phenotypic Screening

Engineering Serendipity

The pharmaceutical industry is facing a crisis of unprecedented proportion. Approval rates for new medicines have been declining over the past several years despite increases in R&D spending, and, if recent data (2008 – 2010) are any indication of future performance, >80% of candidate agents entering Phase II clinical trials will fail. At that point, the sponsor will have invested ~$300 - $500 million in the development of that agent. Between 2009 and 2014, approximately one third of the total pharmaceutical industry’s revenue will lose patent protection. In light of these stressors, finding an alternate indication for failed clinical candidates is a strategy that might address many of these issues.

The majority of repositioning efforts and programs, however, rely on a hypothesis-based approach to finding new indications. Even though the biologic research of the past twenty years has yielded an overabundance of data, and entire companies have been formed to exploit those data and “connect the dots,” our understanding of the complex interactions between various pharmacologic targets remains relatively superficial, limiting the ability to predict alternative indications. A viable option to the target-based approach is the utilization of disease relevant, in vitro/in vivo assays, or phenotypic screening. Selecting a series of relatively quick test systems that are relevant to human diseases can provide not only a new indication, but the opportunity to discover new biology.

This presentation attempts to demonstrate the value of a non-biased, phenotypic screening strategy for drug repositioning. Several case studies are offered and some ideas are put forth for how this approach might be economically applied to failed clinical candidates.