Phase 2b TRANSFORM Trial of Setanaxib Meets Primary Endpoint

Phase 2b TRANSFORM Trial of Setanaxib Meets Primary Endpoint

By, Admin 29 July 2024

Calliditas phase 2b TRANSFORM trial of setanaxib in primary biliary cholangitis meets primary endpoint

Overview

Calliditas Therapeutics AB a Stockholm, Sweden-based biopharma company, announced that the phase 2b TRANSFORM trial met its primary endpoint, showing statistically significant improvement in ALP (alkaline phosphatase) for both doses tested versus placebo.  The trial evaluated setanaxib, a NOX enzyme inhibitor, in patients with primary biliary cholangitis (PBC) and elevated liver stiffness.

TRANSFORM trial

  • The TRANSFORM trial is a double-blind, randomized, placebo-controlled phase 2b study investigating the effect of setanaxib 800 mg AM + 400 mg PM, (“1200 mg arm”) and 800 mg BID (“1600 mg arm”) over 24 weeks of treatment. 
  • The basis for the analysis consisted of a dataset of 76 patients with primary biliary cholangitis (PBC) and elevated liver stiffness.

The Outcome

  • The treatment groups were relatively well-balanced with no clinically relevant differences between the groups observed at baseline. 
  • The result is particularly encouraging as over 40% of the trial population was on dual therapy, ie was receiving UDCA (ursodeoxycholic acid) and either Ocaliva (o beticholic acid) or Bezafibrate (PPAR agonist) 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. S
  • etanaxib treatment was generally well tolerated with overall number of TEAEs (treatment emergent adverse events), 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.

Words from Newcastle University

It is very encouraging to see a statistically significant treatment effect in this hard-to-treat population which is already on multiple medications in this relatively small study,” said Professor Dave Jones OBE; director, NHIP Academy; Director, Newcastle Centre for Rare Disease; Professor of Liver Immunology, Newcastle University; and Honorary Consultant Hepatologist, Newcastle upon Tyne Hospitals.

Words from Calliditas Therapeutics

• CEO:

These positive data provide further clinical evidence of the potential of setanaxib in multiple rare diseases, and we are very pleased that we now have additional positive clinical evidence in support of our unique, first in class NOX platform.  We also look forward to the read out of the investigator led study in IPF as well as the ongoing study in Alport syndrome in due course,” said CEO Renée Aguiar-Lucander.

• CMO: 

I am delighted that we have seen statistically significant and clinically meaningful improvements in ALP with encouraging trends in other outcomes in this population of patients with PBC. I’d like to extend my thanks to investigators, clinical trial site staff, and most importantly patients, who have all contributed to this important study.” said CMO Richard Philipson.

Clinical Trials with Setanaxib

  • The company is conducting additional clinical trials with setanaxib and is expecting the investigator led phase 2 trial in IPF (idiopathic pulmonary fibrosis) 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.

About PBC

  • PBC is a progressive and chronic autoimmune disease of the liver that causes immune injury to biliary epithelial cells, resulting in cholestasis and fibrosis. 
  • It is an orphan disease and, based on its known prevalence rates, we estimate that there are approximately 140,000 patients in the United States, where the annual incidence ranges from 0.3 to 5.8 cases per 100,000.

Calliditas Therapeutics

Calliditas Therapeutics is a biopharma company headquartered in Stockholm, Sweden, focused on identifying, developing, and commercializing novel treatments in orphan indications with significant unmet medical needs.

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