Boston Pharmaceuticals announced positive Phase 2 study results for efimosfermin alfa, a long-acting FGF21 analogue, in patients with stage F2/F3 fibrosis due to metabolic dysfunction-associated steatohepatitis (MASH). The once-monthly treatment showed significant fibrosis improvement without worsening MASH, and MASH resolution without worsening fibrosis over 24 weeks, alongside a favorable tolerability profile. These findings will be presented at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting in November 2024.
The randomized, double-blind, placebo-controlled study involved 84 participants with biopsy-confirmed F2 or F3 MASH. Results demonstrated that 45.2% of the efimosfermin group achieved fibrosis improvement of at least one stage without MASH worsening, compared to 20.6% in the placebo group. MASH resolution without fibrosis worsening was observed in 67.7% of the efimosfermin group versus 29.4% of the placebo group. Although not statistically significant, 38.7% of the efimosfermin group saw both fibrosis improvement and MASH resolution, compared to 17.6% in the placebo group.
Beyond histological endpoints, efimosfermin also led to rapid and significant improvements in fibrosis biomarkers and reductions in non-invasive markers of liver injury and liver fat. Participants with type 2 diabetes experienced a clinically meaningful improvement in HbA1c values. Importantly, efimosfermin demonstrated a low discontinuation rate due to adverse events, with a low incidence of gastrointestinal side effects and injection site reactions. The 24-week treatment data will inform future regulatory discussions, and the company plans to advance the clinical program to late-stage development in 2025. An open-label extension study is ongoing, providing an additional 24 weeks of treatment to evaluate long-term safety and efficacy, including immunogenicity.
Efimosfermin targets the core components of hepatic disease, impacting fibrosis improvement, a critical aspect of MASH management. It also offers potential cardiometabolic benefits, including liver fat reduction and improved glycemic control, which are particularly relevant given the high prevalence of obesity and diabetes in MASH patients. The upcoming results from the extension study and a separate advanced fibrosis study will contribute to a comprehensive data package for regulatory discussions prior to pivotal studies.
Efimosfermin alfa is a long-acting FGF21 analogue administered via once-monthly subcutaneous injection. It works by regulating metabolic pathways to reduce liver fat, lessen liver damage and inflammation, and reverse liver fibrosis in MASH patients. MASH, formerly known as non-alcoholic steatohepatitis (NASH), is a growing global health concern linked to obesity and type 2 diabetes. Affecting millions worldwide, MASH is a progressive disease characterized by liver scarring and is associated with various cardiometabolic risk factors. Untreated, it can lead to liver failure, liver cancer, or death. Boston Pharmaceuticals is focused on developing differentiated therapies for severe liver diseases, with MASH as the primary focus for efimosfermin.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
