GSK has shared promising results from a late-stage study of its investigational oral ileal bile acid transporter inhibitor linerixibat in patients with the rare autoimmune liver disease primary biliary cholangitis (PBC).

The phase 3 GLISTEN trial has been evaluating the candidate in adults with PBC and cholestatic pruritus, or relentless itch, a key symptom of the condition that can cause sleep disturbance, fatigue and impaired quality of life.

Results presented at this year’s European Association for the Study of the Liver (EASL) Congress demonstrated that linerixibat significantly improved itch compared to placebo over 24 weeks, meeting the trial’s primary endpoint. Patients randomised to receive linerixibat experienced an average placebo-adjusted improvement of 0.72 points on a ten-point rating scale for the worst itch (WI-NRS).

Key secondary endpoints were achieved, with significant itch improvements seen as early as week two. GSK’s drug also led to significant benefits in itch-related sleep interference, and 56% of linerixibat-treated patients achieved clinically meaningful itch improvement, defined as at least a three point reduction on the WI-NRS, compared to 43% in the placebo group at week 24.

Expected to affect 510,000 people worldwide by 2030, PBC is a life-long condition that leads to irreversible scarring of the liver and the destruction of bile ducts. Pruritus can occur at any stage of the disease and is experienced in varying degrees of severity by up to 90% of patients.

Linerixibat is designed to inhibit bile acid re-uptake and has already been granted orphan drug designations by the US Food and Drug Administration and the European Medicines Agency to treat PBC-associated cholestatic pruritus.

Kaivan Khavandi, senior vice president, global head, respiratory, immunology and inflammation research and development, GSK, said: “With linerixibat, we are one step closer to addressing the high unmet need of itch and its related sleep interference that are critically important to patients but historically under-treated.”