Seladelpar (MBX-8025) is a potent, selective, orally active peroxisome proliferator-activated receptor δ (PPARδ) agonist in development for the treatment of patients with the autoimmune liver disease, primary biliary cholangitis (PBC). It has received Breakthrough Therapy Designation (FDA) and PRIME status (EMEA); Orphan Drug Designation in U.S. and Europe. Backed by an extensive body of clinical evidence and global patient experience through its Phase 2 and Phase 3 (ENHANCE) studies of seladelpar in Primary Biliary Cholangitis (PBC), CymaBay initiated an international pivotal Phase 3 study (RESPONSE) in early 2021.
We continue to evaluate development opportunities for seladelpar.
Seladelpar is uniquely suited as a potential treatment for inflammatory liver diseases. In the liver, PPARδ is expressed in multiple cell types including: hepatocytes, cholangiocytes, Kupffer cells and stellate cells. Preclinical and clinical data support its effect on regulating genes involved in bile acids synthesis, inflammation, fibrosis and lipid metabolism, storage and transport.
PBC primarily affects women; 1 in 1000 women over the age of 40 lives with PBC. It is rare, chronic, progressive autoimmune disease of the liver characterized by impaired bile flow and accumulation of toxic bile acids. Accompanying inflammation and destruction of the intrahepatic bile ducts may cause progression to fibrosis, cirrhosis and liver failure. Other clinical symptoms of PBC include fatigue and pruritus, which can be quite disabling in some patients.