Repositioning Basimglurant to Treat Hermansky-Pudlak Syndrome Pulmonary Fibrosis

National Center For Advancing Translational Sciences

Investigators

Abstract

The development of an inhaled formulation of basimglurant would provide an alternative route of administration to more directly target pulmonary fibroblasts within the lungs. Such a formulation would enhance the safety margin by minimizing the therapeutic dose and increasing exposure within the lung. To date, synthesis of a pilot batch of basimglurant drug substance, including several salt forms, has been completed. The scaled-up production of bulk drug substance has begun, which will support the comprehensive drug product formulation development. In vivo pharmacokinetics studies are ongoing to determine the potency of basimglurant via inhaled administration versus the oral route. The team has begun to develop and validate the analytical methods to characterize the drug substance, and bioanalytical assays will be developed to evaluate the drug product in subsequent in vivo studies.