CymaBay Therapeutics has initiated a Phase III study of seladelpar in patients with primary biliary cholangitis (PBC), a rare, chronic inflammatory liver disease.
The randomised, placebo-controlled Intended to Determine the Effects of seladelpar on the Normalisation of Alkaline Phosphatase (ALP) levels (IDEAL) study will enrol 75 PBC patients with an incomplete response or intolerance to ursodeoxycholic acid (UDCA).
Patients with ALP greater than the upper limit of normal (ULN) but less than 1.67xULN, and total bilirubin less than or equal to 2xULN are included in the study.
They will be randomised into a 2:1 ratio to receive 10mg of seladelpar once-daily orally or placebo for a duration of 52 weeks.
Normalisation of ALP at 52 weeks is the primary outcome measure while evaluating efficacy, including the percent change from baseline in ALP at 52 weeks, is the additional key outcome of the study.
Other key outcomes include evaluating the level of self-reported pruritus (itch) at six months in patients with moderate-to-severe symptoms at baseline, using a numerical rating scale and being recorded by an electronic diary.
CymaBay Therapeutics research and development chief scientific officer and president Charles McWherter said: “We see the IDEAL study as an opportunity to evaluate the potential benefits of seladelpar in a population that has a persistent elevation of ALP, but whose ALP levels are not above the 1.67 times upper-limit-of-normal recommended by clinical guidelines or that are used for entering clinical trials of new second-line agents.
“We remain fully committed to providing all patients the opportunity to have their diseases and unmet needs addressed, and the IDEAL study is emblematic of this endeavour.”
Seladelpar is an oral, selective peroxisome proliferator-activated receptor (PPAR) delta agonist or delpar.
It has received breakthrough therapy designation and orphan drug status from the US Food and Drug Administration (FDA) and priority medicines and orphan drug status from the European Medicines Agency (EMA).
