Positive phase II study results of once-monthly efimosfermin alfa, in participants with stage F2/F3 fibrosis due to MASH
Boston Pharmaceuticals, a clinical-stage biopharmaceutical company developing differentiated molecules addressing serious liver diseases announced positive results from a Phase II study evaluating the safety and efficacy of once-monthly efimosfermin alfa (formerly known as BOS-580), a long-acting FGF21 analogue, in participants with stage F2/F3 fibrosis due to metabolic dysfunction-associated steatohepatitis (MASH)
Treatment with efimosfermin led to significant improvements in fibrosis ≥1 stage without worsening of MASH, and MASH resolution without worsening of fibrosis over 24 weeks. In the study, efimosfermin demonstrated a favorable tolerability profile. These topline results will be presented on Nov. 19 in a late-breaking oral presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting, Nov. 15-19, 2024, in San Diego.
The Phase II randomized, double-blind, placebo-controlled study (N=84) was designed to assess the efficacy and safety of once-monthly efimosfermin 300 mg dosed subcutaneously in participants with biopsy-confirmed F2 or F3 MASH over 24 weeks. The data provide a more comprehensive overview of the benefit-risk profile of the 300 mg dose and will inform future discussions with regulatory authorities. The company anticipates advancing the clinical program to late-stage development in 2025.
In addition, study participants treated with efimosfermin showed rapid and significant improvement in fibrosis biomarkers and significant reductions in non-invasive markers of liver injury and liver fat over the study period. Participants with type 2 diabetes also had significant and clinically meaningful improvement in HbA1c values. Over 24 weeks of treatment, efimosfermin-treated study participants had low discontinuation rates due to adverse events, and an overall low incidence of gastrointestinal side effects and injection site reactions.
“These data indicate that efimosfermin may effectively target the three main components of hepatic disease, thereby producing a meaningful impact on fibrosis improvement, which is a critical and often under-treated aspect of managing MASH,” stated Margaret Koziel, M.D., Chief Medical Officer at Boston Pharmaceuticals. “Furthermore, efimosfermin may offer important cardiometabolic benefits such as liver fat reduction and improved glycemic control in MASH patients who often struggle with co-morbidities such as obesity and diabetes.”
The Phase II clinical development program of efimosfermin is continuing with an open-label extension in F2 and F3 patients, providing an additional 24 weeks of treatment to assess long-term safety and efficacy which should offer further information on the low immunogenicity of efimosfermin.
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