PBC Phase II Study Shows Hopeful Results

CymaBay, Inc. recently announced positive provisional results from its low-dose Phase II seladelpar clinical trial for primary biliary cholangitis (PBC) patients.

Once you get past the confusing part of that statement, there’s actually a lot to be excited about. Let me help clarify.

Primary biliary cholangitis, or PBC for short, is a chronic and progressive liver disease.

Usually, PBC results from the progressive destruction of the bile ducts in the liver. When these bile ducts are destroyed and broken down, the bile has no other option but to accumulate in the liver. Ultimately, this accumulation leads to inflammation and scarring.

Primary biliary cholangitis can eventually lead to cirrhosis and a plethora of other complications and difficulties, which is why treatment is all the more vital. CymbaBay, Inc. researched and developed the treatment seladelpar for this purpose.

The initial element of this low-dose seladelpar Phase II study tested resistant patients who were at an increased risk of PBC disease progression. Researchers observed these patients who had an insufficient response to ursodeoxycholic acid (UDCA), which is the most commonly used therapy to treat PBC. These patients had a continual increase in alkaline phosphatase (AP) despite taking UDCA, or they were simply intolerant to UDCA.

(To clarify, alkaline phosphatase (AP) is a sure marker of disease progression in PBC patients; thus, an increase in this substance is directly correlated with disease progression, and vice versa.)

This group of resistant patients received a minuscule 5 mg or 10 mg of seladelpar every day—hence, the “low-dose” study. An interim investigation of these two testing groups (those given 5 mg and those given 10 mg) found that, after 12 weeks of receiving treatment and being observed, there was a significant reduction in alkaline phosphatase (AP).

Specifically, there was a 39% and 45% reduction in AP for the 5 mg and 10 mg groups, respectively.
In addition to this encouraging information, patients in these testing groups showed significant declines in other markers of liver function, such as gamma glutamyl transferase and total bilirubin, that demonstrate the impairment of bile flow.
Even more excitingly, not only is there good news associated with this project, but there’s also no bad news. According to researchers, there were not any serious adverse effects or safety concerns at either (5 mg or 10 mg) dosage.
Given this, it’s no surprise that the FDA approved a request to research the seladelpar treatment for longer than 6 months in the same 5 mg and 10 mg doses.

CMO of CymaBay, Inc., Dr. Pol Boudes, is incredibly thankful for all the report that his team has since received for this project. In addition, he wanted to thank every stakeholder in this trial for all of the support they have given and exemplified great enthusiasm for the FDA’s approval of a longer research span.

In concluding remarks, he relayed that CymaBay, Inc. can now start focusing on planning the Phase 3 study program for seladelpar to further catapult these interim results.

The data surrounding CymaBay’s seladelpar is substantial and encouraging and reflects the potency of the drug against the effects of PBC. So far, the treatment has kept passing through all of its’ necessary checkpoints, so there’s a lot to look forward to in the near future of PBC medicines.

To learn more about the seladelpar studies from Global Newswire, click here!


To find out more about PBC, check out our partners the PBCers, here!

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