Research Project
10080262
Blue Oak Pharmaceuticals is developing the next generation of drugs for the treatment of acute bipolar depression and long-term maintenance (BPD), a mental disorder that affects ~6 million adult Americans. Existing drug classes are relatively ineffective and very few new modes-of-action have been developed in the past 40+ years. This is due, in part, to the biopharma industry?s focus on drugging single molecular targets, and the lack of predictive animal models for bipolar disorder. To overcome these significant hurdles, Blue Oak?s drug discovery paradigm combines custom-designed privileged chemotypes with a proven deep behavioral profiling method. Successful Phase I studies using this novel, but previously validated, approach resulted in the discovery and optimization of a novel lead compound for the treatment of BPD. The therapeutic utility of drug candidates was confirmed using molecular target profiling, ex vivo imaging of the forebrain circuits implicated in BPD and predictive translational medicine biomarkers. Phase II studies are focused on advancing the lead compound through Investigational New Drug (IND)-enabling studies, the necessary next step for FDA review and approval of human studies. Aims include: (1) quantification of the preclinical therapeutic (safety) margin for the lead, or superior back-up molecule, including analyses of antidepressant activity; (2) confirmation of an enhanced EEG gamma band as a reliable translational biomarker of BPD drug activity and efficacy in non-human primates; (3) manufacture and qualification of cGLP drug product suitable for IND-enabling studies; and (4) completion of preclinical studies necessary for an IND filing with the FDA, including standard absorption, distribution, metabolism, excretion, and toxicity (ADME/TOX) preclinical studies and preclinical dose escalation studies necessary prior to first-in-human clinical studies. The successful completion of these aims will result in a Type B meeting to obtain guidance from the FDA and the filing of an IND package. The Blue Oak Pharmaceuticals team includes experienced ?drug hunters? with expertise in systems neurobiology, medicinal chemistry and informatics, and advisors with proven track records in drug development and commercialization. The internal team is supported by trusted partners in preclinical research, development and validation of translational medicine biomarkers, clinical site management, and clinical development. If successful, this program will deliver a new drug candidate for bipolar depression that will improve the quality of life of patients and their families.
