Novel Non-Bile FXR Agonist Promising in PBC... - PBC Foundation

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Novel Non-Bile FXR Agonist Promising in PBC Patients

JaneIng profile image

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.

4 Replies

What wonderful news!! It's nice to know they are working hard trying to find a cure.

Thank you for sharing Janelng.

Stella ❤

Thank you for posting this. I searched for the scientific journal on this & it is 69 pages. I’ll probably skim through it & ask my hepatologist what he thinks of this. I am sure he knows about this. He told me about OCA when it was in trials with Intercept.

Great news, thank you for sharing. Maybe with all this research going on the reason why may not be quite so far away!

I saw this a few months ago and spoke to them in regards to signing up for the trial. At that time, one of my liver enzymes was too high and they declined me due to that. Maybe I will look into it again since my numbers have come down

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