This is from PBCers news feed. Sounds exciting.
PARIS -- Tropifexor, a non-bile acid FXR agonist, had a dose-dependent effect on gamma-glutamyl transferase (GGT) levels in patients with primary biliary cholangitis (PBC) compared with placebo, researchers said here.
In addition to a significant reduction in GGT levels, a reduction was observed in other liver tests, such as alkaline phosphatase (ALP), alanine aminotransferase (ALT), and aspartate aminotransferase (ASP) among patients who received the drug, reported Christopher Schramm, MD, of the University Medical Centre Hamburg-Eppendorf in Hamburg, Germany, and colleagues.
The drug was also well-tolerated, with an acceptable safety profile, they said at a late-breaking trial presentation at the International Liver Congress, the annual meeting for the European Association for the Study of the Liver (EASL).
Schramm noted that FXR is "a recognized therapeutic target for PBC," as it protects from bile acid accumulation in the liver and intestine, inhibits bile acid synthesis, and increases bile acid conjugation, transport and excretion. They added that tropifexor (LJN452) is a non-bile acid investigational compound that potently activates FXR, and was shown to reduce cholestasis and hepatocellular damage in pre-clinical studies.
The current research was part of an ongoing phase II study of patients with an inadequate response to ursodiol, a first-line therapy for primary biliary cholangitis. The interim phase II study examined both the effect of tropifexor on GGT, which the authors noted was chosen to avoid the confounding effect of FXR-mediated ALP gene induction, as well as the safety and tolerability of tropifexor. To participate, patients needed to be currently taking ursodiol, as well as meet several PBC diagnosis criteria and disease severity markers.