- 65% of patients with clinically significant portal hypertension (CSPH) moved to lower risk categories after two years of treatment with Rezdiffra.
- The study achieved a statistically significant mean reduction of 6.7 kPa in liver stiffness among participants.
- 35% of patients showed potential cirrhosis reversal, with liver stiffness measurements consistent with F3 fibrosis.
Madrigal Pharmaceuticals (NASDAQ: MDGL) has announced promising two-year findings from its Phase 3 MAESTRO-NAFLD-1 trial, which investigated the effects of Rezdiffra™ (resmetirom) on patients with compensated MASH cirrhosis. The results reveal significant improvements across several clinical measures in this high-risk population, where effective treatment options remain scarce.
A key outcome of the trial was that 65% of patients with clinically significant portal hypertension (CSPH) at baseline transitioned to lower risk categories by the end of two years. This is significant, as CSPH is a major predictor of severe complications such as ascites and variceal bleeding, which increase mortality risk in cirrhosis patients.
The trial also recorded a mean reduction in liver stiffness of 6.7 kPa, which was statistically significant compared to baseline values. A substantial proportion of patients—51%—achieved at least a 25% improvement in liver stiffness, a mark associated with reduced progression to end-stage liver disease.
Additionally, 35% of the participants achieved liver stiffness measurements indicative of F3 fibrosis, suggesting potential reversal of cirrhosis.
Rezdiffra was generally well-tolerated, with the most common side effects being diarrhea, COVID-19, and nausea. There were two deaths during the trial; however, they were deemed unrelated to the treatment. Despite the positive results, the study's open-label design and lack of a placebo control group mean a larger, placebo-controlled study is needed to confirm these benefits. The ongoing Phase 3 MAESTRO-NASH OUTCOMES trial aims to provide more conclusive evidence.
