How United Therapeutics Leverages Metabolomics to Get Closer to the Phenotype Increasing Confidence in Clinical Trial Decisions

Successful drug development hinges on multimillion-dollar decisions about mechanism of action, safety, dosage, and patient response. While genomics, transcriptomics, and proteomics are well-established tools in the drug development arsenal, these tools stop short of providing a real-time, definitive representation of the clinical phenotype.

Derek Solum, Ph.D., Associate Director, Product Development at United Therapeutics, and Brian Keppler, Ph.D., Director, Discovery and Translational Sciences, Metabolon, Inc., share how metabolomics has helped United Therapeutics enhance its multi-omics platform and improve decision-making across all phases of drug development.

In this talk you’ll learn:

• How United Therapeutics incorporates metabolomics into multi-omics workflow across pre-clinical, first-in-human, and late-stage clinical trials.
• How the broad-scale use of targeted and untargeted metabolomics has helped researchers increase confidence in translatability of animal models, provide a clearer explanation of a drug’s mechanism of action, and provide direct evidence of phenotypic response for in vivo confirmation of genomic, RNA-seq, and protein data.
• How metabolomics helped inform rapid decision-making in a pivotal Phase III study that made it possible to enhance product labeling based on the outcome of the study.