Calliditas reveals positive Phase 2b TRANSFORM data in primary biliary cholangitis

STOCKHOLM, July 26, 2024 -- Calliditas Therapeutics AB announced that their Phase 2b TRANSFORM trial has successfully achieved its primary objective. The trial demonstrated significant improvements in Alkaline Phosphatase (ALP) levels for both doses of setanaxib compared to a placebo. Setanaxib, a NOX enzyme inhibitor, was assessed in patients suffering from primary biliary cholangitis (PBC) with elevated liver stiffness.

The study, a double-blind, randomized, placebo-controlled Phase 2b trial, investigated setanaxib at two different dosages: 800 mg in the morning and 400 mg in the evening (1200 mg total daily) and 800 mg twice daily (1600 mg total daily). The treatment period spanned 24 weeks, and the analysis covered 76 patients diagnosed with PBC and increased liver stiffness.

Baseline results indicated that the treatment groups were well-matched without significant clinical differences. Notably, over 40% of participants were on dual therapy, receiving ursodeoxycholic acid (UDCA) in conjunction with either obeticholic acid or Bezafibrate. Furthermore, around 13% of patients were on a triple therapy regimen during the study. This highlights setanaxib's potential added benefit over existing treatments.

Patients treated with setanaxib showed statistically significant improvements in ALP levels, with a 19% reduction in the 1600 mg group and a 14% reduction in the 1200 mg group. Additionally, positive trends were observed in liver stiffness measurements obtained through FibroScan® after 24 weeks.

The treatment was generally well tolerated. The frequency of treatment-emergent adverse events (TEAEs) and serious TEAEs were similar between the active treatment and placebo groups. However, the rate of TEAEs leading to study discontinuation was higher among those receiving the active treatment compared to the placebo group.

Professor Dave Jones OBE from Newcastle University commented on the findings, highlighting the significance of seeing a statistically significant treatment effect in a challenging patient population already on multiple medications. He emphasized the promise these results show for setanaxib in treating various rare diseases.

Renée Aguiar-Lucander, the CEO of Calliditas, expressed satisfaction with the positive clinical evidence supporting their NOX platform and looked forward to future studies. Calliditas is anticipating results from an investigator-led Phase 2 trial in idiopathic pulmonary fibrosis (IPF) by the end of 2024 or early 2025. Additionally, a Phase 2 proof-of-concept trial for Alport syndrome is expected to yield results in 2025.

Chief Medical Officer Richard Philipson extended his gratitude to the investigators, clinical trial staff, and patients for their contributions to this significant study. He noted the clinically meaningful improvements in ALP and encouraging trends in other outcomes in patients with PBC.

Overall, the TRANSFORM trial results bring hope for new and effective treatment options for patients with primary biliary cholangitis and potentially other rare diseases, as further research and trials continue to explore the benefits of setanaxib.

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