Mirum’s phase 2b clinical trial studying volixibat for the treatment of primary biliary cholangitis (PBC) showed positive preliminary results, according to a recently published press release.
PBC is characterized by the destruction of liver bile ducts by the patient’s own immune system. This leaves the liver without appropriate pathways to release bile into the intestines, which leads to bile accumulation in the liver (cholestasis). With time, cholestasis damages the liver enough to produce liver failure and scarring, known as cirrhosis.
Currently, there is no cure for PBC. Most treatments aim to delay the progression by preventing cholestasis. Available treatments like ursodeoxycholic acid (UDCA) increase bile flow out of the liver. Volixibat, on the other hand, inhibits bile acid transport into the liver by adhering to the ileal bile acid transporter (IBAT). The drug is thought to have beneficial effects for patients with PBC as well as for patients with nonalcoholic hepatic steatosis.
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The therapy is currently being investigated in the VANTAGE study, a phase 2b clinical trial. The preliminary results from the trial showed that patients receiving the drug experienced a statistically significant improvement in PCB symptoms such as itching and fatigue. Furthermore, over 70% of patients experienced a reduction in bile acid secretion of 50% or more.
The most common adverse effect in the studied population was mild to moderate diarrhea. Four patients reported severe adverse effects and one patients had to retire from the trial due to adverse effects.
Joanne Quan, M.D., chief medical officer at Mirum, called the results of the interim data “outstanding” in relation to other treatments of pruritis in PBC, and stated the company is looking forward to the next phase of the trial.
