Calliditas Therapeutics announces TRANSFORM trial met primary endpoint

Calliditas Therapeutics announced that the Phase 2b TRANSFORM trial met its primary endpoint, showing statistically significant improvement in ALP for both doses tested versus placebo. The trial evaluated setanaxib, a NOX enzyme inhibitor, in patients with primary biliary cholangitis and elevated liver stiffness. The TRANSFORM trial is a double-blind, randomized, placebo-controlled Phase 2b study investigating the effect of setanaxib 800 mg AM + 400 mg PM, and 800 mg BID over 24 weeks of treatment. The basis for the analysis consisted of a dataset of 76 patients with primary biliary cholangitis and elevated liver stiffness. The treatment groups were relatively well-balanced with no clinically relevant differences between the groups observed at baseline. Over 40% of the trial population was on dual therapy, ie was receiving UDCA and either Ocaliva or Bezafibrate as base therapy and 13% were receiving all three therapies during the study, reflecting setanaxib having clinically relevant incremental benefit beyond existing standard of care. Patients treated with setanaxib showed statistically significant improvements in the primary endpoint of ALP of 19% in the 1600mg arm and 14% in the 1200mg arm and showed positive trends on liver stiffness assessed by FibroScan at 24 weeks. Setanaxib treatment was generally well tolerated with overall number of TEAEs, as well as serious TEAEs, being similar between active treatment and placebo. The frequency of TEAEs leading to study discontinuation was higher in patients receiving active treatment compared to placebo. The company is conducting additional clinical trials with setanaxib and is expecting the investigator led Phase 2 trial in IPF to provide top line data in Q4 2024 / Q1, 2025. There is also an ongoing Phase 2 proof of concept trial in Alport syndrome, which is expected to deliver top line data in 2025.