H3 Biomedicine takes a target-centric approach to drug discovery through use of biological proof-of-concept milestones to enable project progression. Our research efforts focus on answering key scientific questions related to modulating a target that will have predictable therapeutic outcomes in patients. Each project starts with a therapeutic hypothesis for a group of patients that can be identified through testing with a companion assay to identify those patients likely to respond. Thus, our approach contrasts with the traditional and inefficient drug-candidate discovery process and ensures a higher likelihood of clinical success.
Our research team uses a deep understanding of cancer genomics and alterations that can impact various hallmarks of cancer to inform on drug target selection. Our drug discovery projects span a range of biological functions including targeting alterations in cancer driver genes, alterations leading to immune evasion and synthetic lethal approaches. We have a deep expertise in defining the role of changes in RNA homeostasis that contribute to cancer, with a particular focus on alterations in RNA splicing. The RNA splicing platform at H3 Biomedicine is an integral part of our research effort and explores approaches to modulate RNA splicing in cancer cells as a novel approach to cancer therapy. Our biological proof-of-concept approach relies on continuous testing of our therapeutic hypothesis in relevant model systems that enables identification of rationally designed drug candidates. We plan to advance our three lead cancer drug candidates into clinical development in 2016–17 in each case accompanied by a companion diagnostic to identify patients for clinical trials.