The data establish GKT831 as an attractive therapeutic option in a broad PBC population and support its development in multiple fibrotic diseases including NASH and IPF.
GKT831 achieved even greater GGT reductions (29%, p1.5XULN) and elevated gamma glutamyl transpeptidase (GGT; >1.5 XULN).
The primary efficacy endpoint is change in GGT at week 24. Key secondary endpoints include additional markers of liver and bile duct injury, markers of inflammation, non-invasive markers of liver fibrosis including liver stiffness assessed by transient elastography, the Enhanced Liver Fibrosis score, and circulating collagen fragments including proC3. In addition, indicators of quality of life including pruritus and fatigue will be assessed. Markers of bile acid metabolism and immune activation will be also investigated.
A total of 111 patients have been allocated according to a 1: 1: 1 randomization ratio to three groups: UDCA plus placebo, UDCA plus GKT831 400mg once a day and UDCA plus 400mg twice a day. This trial is one of the largest phase 2 PBC trials conducted to date.
The interim efficacy analysis was conducted when 92 patients completed 6 weeks of treatment. At baseline, mean ALP levels were respectively 304, 282, and 350 IU/L in the placebo, 400mg GKT831 OD, and 400mg GKT831 BID groups.
Mean baseline GGT levels were respectively 224, 215, and 237 IU/L in the placebo, 400mg GKT831 OD, and 400mg GKT831 BID groups.
Changes in the primary efficacy endpoint GGT, a marker of liver and bile duct injury, were: -7% in the placebo, -12% in the 400mg OD, and -23%, in the 400mg BID groups (p
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