Calliditas announces positive TRANSFORM Phase 2b topline data in primary biliary cholangitis

Core Insights - The TRANSFORM trial demonstrated that setanaxib significantly improved alkaline phosphatase (ALP) levels in patients with primary biliary cholangitis (PBC) compared to placebo, meeting its primary endpoint [3][4][7] Group 1: Trial Details - The TRANSFORM trial is a double-blind, randomized, placebo-controlled Phase 2b study involving 76 patients with PBC and elevated liver stiffness [1] - Two treatment arms were tested: 1200 mg (800 mg AM + 400 mg PM) and 1600 mg (800 mg BID) over 24 weeks [1] - The treatment groups were well-balanced with no significant differences at baseline [2] Group 2: Efficacy Results - Patients receiving setanaxib showed a statistically significant improvement in ALP levels: 19% in the 1600 mg arm and 14% in the 1200 mg arm [2][3] - Positive trends were observed in liver stiffness assessed by FibroScan® at 24 weeks [2] Group 3: Safety and Tolerability - Setanaxib treatment was generally well tolerated, with similar rates of treatment-emergent adverse events (TEAEs) between active treatment and placebo [2] - However, the frequency of TEAEs leading to study discontinuation was higher in the active treatment group [2] Group 4: Future Developments - The company is conducting additional clinical trials with setanaxib, including a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) expected to report data in Q4 2024/Q1 2025 [5] - An ongoing Phase 2 proof of concept trial in Alport syndrome is anticipated to deliver data in 2025 [5]