According to a story from GlobeNewswire, the biopharmaceutical company CymaBay Therapeutics, Inc. recently announced encouraging topline findings from a phase 3 clinical trial. This study was testing seladelpar as a treatment for primary biliary cholangitis, a rare disease that impacts the bile ducts. The findings suggest that seladelpar could be a useful therapy for patients living with this disease.
About Primary Biliary Cholangitis
Primary biliary cholangitis, less commonly referred to as primary biliary cirrhosis, is an autoimmune disease that affects the liver. It is most characterized by progressive damage to the bile ducts, which over time allows bile and other toxic substances to build up in the liver. The disease can ultimately progress to liver scarring and cirrhosis. The precise cause of the disease remains unknown; a genetic predisposition, perhaps involving the POGLUT1 gene, is a risk factor, as is the presence of another autoimmune disease. Women are also much more likely to get primary biliary cholangitis. Symptoms include reduced bone density, skin lesions, fatigue (sometimes severe), jaundice, abdominal swelling, hepatic encephalopathy, and enlarged spleen. Treatment for the disease may include the drug Ursodiol, vitamin supplementation, and liver transplant in severe cases. Some patients experience slowed disease progression to the extent that their lifespan and quality of life are not significantly affected. To learn more about primary biliary cholangitis, click here.
About the Trial
The trial involved a total of 265 patients who were given either placebo, 5 mg of seladelpar per day, or 10 mg of seladelpar per day. The primary endpoint was a normal level of bilirubin and a 15 percent drop from baseline serum alkaline phosphatase level (ALP) that was less than 1.67 times the upper limit of normal (ULN). This must be achieved after 52 weeks of treatment. Unfortunately, this study was ended early and the 52 week limit was modified to three months.
167 patients were able to reach the modified endpoint and a total of 78.2 percent of patients on the 10 mg dose achieved the composite primary endpoint, which was also achieved by 57.1 percent of patients in the 5 mg group. Only 12.5 percent in the placebo group reached the endpoint. In one month, the drug reduced ALP by 38 percent and 30 percent in the 10 and 5 mg dose groups respectively.
The therapy was also able to produce significant reductions in pruritus (itching) after three months. Seladelpar also displayed meaningful anti-inflammatory activity.
These results bode well for the future development of the drug and for the primary biliary cholangitis patient community.
