We work at the intersection of groundbreaking science and patient need. Our research and development teams seek to identify and develop innovative drugs with the greatest promise of becoming first-in-class medicines for the treatment of chronic and life-threatening conditions such as anemia, idiopathic pulmonary fibrosis, pancreatic cancer, and Duchenne muscular dystrophy. Our product candidates leverage the body’s natural pathways, providing us with the scientific insight to investigate their potential across multiple diseases.
Roxadustat, our most advanced therapeutic, is an oral small molecule inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase, the enzyme that regulates HIF activity. By transiently inhibiting the enzyme, roxadustat induces the body’s natural coordinated process of producing red blood cells, increasing iron absorption, mobilization, and transport. We are developing roxadustat in partnership with AstraZeneca and Astellas for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. With Phase 3 clinical development well underway worldwide, roxadustat is currently approved in China for the treatment of anemia in CKD patients on dialysis and patients not on dialysis, and approved in Japan for the treatment of anemia in CKD patients on dialysis. Roxadustat is approaching new drug application (NDA) and marketing authorization application (MAA) submissions for use in anemia associated with CKD in multiple markets. Roxadustat is also in Phase 3 clinical development in the U.S. and Europe and in Phase 2/3 development in China for anemia associated with myelodysplastic syndromes (MDS), and in a Phase 2 U.S. trial for treatment of chemotherapy-induced anemia.
Pamrevlumab is a fully-human monoclonal antibody that inhibits the activity of connective tissue growth factor (CTGF). CTGF is a common factor in chronic fibrotic and proliferative disorders, characterized by persistent and excessive fibrous tissue which can lead to organ dysfunction and failure, and in cancer, characterized by promotion of tumor growth. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and for the treatment of locally advanced unresectable pancreatic cancer (LAPC), and in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (DMD). The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to pamrevlumab for the treatment of patients with IPF, LAPC, and DMD. Pamrevlumab also received Fast Track designation from the FDA for the treatment of patients with IPF and LAPC. Across all clinical studies to date, pamrevlumab has consistently demonstrated a good safety and tolerability profile.