A new experimental treatment may help improve signs of disease in people living with primary biliary cholangitis (PBC), according to a press release from biotechnology company COUR Pharma.
The company released one-year results from a Phase 2a study evaluating its investigational therapy called CNP-104. The drug is designed to retrain the immune system to tolerate specific proteins involved in the autoimmune attack that drives the bile duct inflammation and progressive liver damage of PBC.
According to the company, the findings showed durable clinical effects following two doses of the therapy administered one week apart. The one-year results indicated improvements across several measures associated with liver health and disease progression.
Specifically, those who received CNP-104 were more likely to achieve a composite biochemical response used in PBC studies compared to a placebo. This included having lower levels of alkaline phosphatase (ALP), a liver enzyme linked to disease activity, and normal levels of total bilirubin, another marker of liver function.
The results also showed stabilization of liver stiffness compared with placebo. Liver stiffness, measured using transient elastography with FibroScan, is considered an indicator of disease progression in people with PBC.
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Additionally, researchers observed improvements in albumin levels, a marker of liver synthetic function, as well as improvements in the UK-PBC prognostic risk score, a tool used to estimate long-term disease outcomes. The score includes measures such as ALP, bilirubin, alanine aminotransferase, albumin and platelet counts.
The trial enrolled adults between the ages of 18 and 75 who had PBC but had not responded to standard treatments such as ursodeoxycholic acid or obeticholic acid.
Earlier results from the study’s primary 120-day analysis showed that CNP-104 reduced antigen-specific Th17 T cells and produced statistically significant differences in liver stiffness between the treatment and placebo groups.
“The one-year data confirms continued stabilization in liver stiffness measurements and validated prognostic risk scores, while newly demonstrating achievement of established composite biochemical response criteria at twelve months,” said Dannielle Appelhans, chief executive officer of COUR Pharma. “Together, these results spanning both positive fibrotic and cholestatic biomarkers support the potential of CNP-104 as a novel therapeutic option of PBC and inform our ongoing evaluation of the program’s development strategy, including potential collaboration with a strategic partner.”
The therapy also showed a favorable safety and tolerability profile during the one-year follow-up period, with only mild or moderate treatment-related side effects and no severe adverse events.
Researchers plan to present the full one-year results from the study at future scientific meetings as development of the therapy continues.
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