NASH, or non-alcoholic steatohepatitis, is a significant health concern affecting millions worldwide. Characterized by inflammation and liver cell damage, NASH can progress to liver fibrosis, cirrhosis, or liver cancer if left untreated. There is currently a pressing need for effective treatment options for NASH with liver fibrosis. The MAESTRO-NASH phase 3 trial, a recently conducted study, offers hope in this regard, highlighting the potential of Resmetirom as a treatment option.
Understanding the MAESTRO-NASH Phase 3 Trial
The MAESTRO-NASH phase 3 trial focused primarily on evaluating the efficacy of Resmetirom in treating NASH patients with liver fibrosis. This trial involved 966 adults who had biopsy-confirmed NASH and a fibrosis stage of F1B, F2 or F3. The patients were randomly assigned to receive either 80 mg or 100 mg of Resmetirom or a placebo, once daily. The primary endpoints at week 52 were NASH resolution and an improvement in fibrosis.
Trial Findings: Resmetirom Shows Potential
As per the findings, NASH resolution with no worsening of fibrosis was achieved in 25.9% of patients in the 80 mg Resmetirom group and 29.9% in the 100 mg Resmetirom group, compared to 9.7% in the placebo group. Also, improvement in fibrosis by at least one stage with no worsening of the NAFLD activity score was noted in 24.2% of the patients in the 80 mg Resmetirom group and 25.9% in the 100 mg Resmetirom group, as opposed to 14.2% in the placebo group. These findings underscore the potential of Resmetirom in treating NASH with liver fibrosis.
Resmetirom: A Liver-Directed Therapy
Resmetirom is an oral, liver-directed, thyroid hormone receptor beta-selective agonist under development for the treatment of NASH with liver fibrosis. It is designed to target key underlying causes of NASH. The drug also led to reductions in levels of a broad range of atherogenic lipids and lipoproteins, including LDL cholesterol, nonâHDL cholesterol, triglycerides, apolipoprotein B, and lipoprotein(a).
Adverse Effects and Safety Concerns
The trial also noted the adverse effects of Resmetirom. More patients in the 100 mg group discontinued the trial due to adverse events compared to the 80 mg or placebo groups. Diarrhea and nausea were more frequently observed in the Resmetirom groups. However, the incidence of serious adverse events was similar across trial groups, indicating that the drug is generally safe.
Towards FDA Approval
Resmetirom is currently under review to become the first approved medicine to treat patients with NASH with liver fibrosis. The drug has shown promising results in resolving NASH and improving liver fibrosis. However, there are concerns about the access and cost of the drug, especially for patients with stage F2 or higher fibrosis. The FDA is expected to decide on its approval by mid-March.
In conclusion, the MAESTRO-NASH Phase 3 trial has shed light on a potentially effective treatment for NASH with liver fibrosis. While further studies and investigations are needed to ascertain long-term safety and efficacy, the results so far are promising and pave the way for significant advances in NASH treatment.