Efimosfermin alfa

CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Boston Pharmaceuticals , a clinical-stage biopharmaceutical company developing differentiated molecules addressing serious liver diseases, today announced positive results from a Phase 2 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. “These new data demonstrate that efimosfermin may rapidly and significantly reduce disease progression in people diagnosed with moderate-to-advanced-stage liver disease, is well-tolerated and offers the convenience of monthly dosing – all factors that are important to this patient population,” said Mazen Noureddin, M.D., M.H.Sc., lead investigator and professor of medicine at Houston Methodist Hospital, and director of the Houston Research Institute. “For patients with MASH, maintaining their treatment regimen is essential for the overall effective management of this debilitating disease.” The Phase 2 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. Key treatment effects over 24 weeks with efimosfermin in biopsy-confirmed MASH patients with F2/F3 fibrosis showed: Histological Endpoints Efimosfermin alfa 300 mg (N=31) Placebo (N=34) p-value (vs. placebo) Fibrosis improvement ≥1 stage without worsening of MASH, n (%) 14 (45.2%) 7 (20.6%) 0.038 MASH resolution without worsening of fibrosis, n (%) 21 (67.7%) 10 (29.4%) 0.002 Fibrosis improvement ≥1 stage and MASH resolution, n (%) 12 (38.7%) 6 (17.6%) 0.066 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 2 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. “Efimosfermin has demonstrated significant efficacy across key liver and metabolic biomarkers, reinforcing its potential as a well-tolerated, once-monthly treatment for MASH,” said Sophie Kornowski, Chief Executive Officer, Boston Pharmaceuticals. “We believe these data, together with upcoming results from our extension study and our advanced fibrosis study, will enable us to build a comprehensive package for discussions with regulatory authorities ahead of our entry into pivotal studies.” Investor Webcast Boston Pharmaceuticals will host a conference call on Thursday, Nov. 21, 2024, at 4 p.m. ET. This virtual event will provide insights into the topline data including histology, changes from baseline in non-invasive markers, as well as safety and tolerability. Participants can register at the following link: https://events.q4inc.com/attendee/517931962 . An archived replay of the webcast will be available on the Boston Pharmaceuticals’ website. About efimosfermin alfa (BOS-580) Boston Pharmaceuticals’ lead investigational agent, efimosfermin alfa (BOS-580), is a once-monthly, subcutaneous injection of a long-acting variant of human fibroblast growth factor 21 (FGF21) that regulates metabolic pathways to decrease liver fat, ameliorate liver damage and inflammation, and reverse liver fibrosis in patients with metabolic dysfunction-associated steatohepatitis (MASH). Efimosfermin is manufactured in mammalian cells, leading to human-like glycosylation. Efimosfermin is currently in Phase 2 clinical trials for the treatment of MASH. In clinical studies, efimosfermin has previously demonstrated statistically significant reductions in liver fat content, markers of liver injury and fibrosis, and improved metabolic biomarkers, with a favorable safety profile in a 12-week Phase 2a study. About MASH Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), is a growing global epidemic fueled by the increasing prevalence of obesity and type 2 diabetes. It is estimated that MASH affects 115 million people worldwide, including 17 million in the U.S., and is expected to increase by 63% by 2030. MASH is a progressive disease staged by the degree of fibrosis (scarring) in the liver and is closely associated with multiple cardiometabolic risk factors. Left untreated, MASH could lead to liver failure, liver cancer or death. In the U.S., MASH is now a leading cause of liver transplantation. About Boston Pharmaceuticals Boston Pharmaceuticals, a portfolio company of B-FLEXION , is a clinical-stage biopharmaceutical company that leverages an experienced and committed drug development team to advance a portfolio of highly differentiated therapies that may address important unmet medical needs in severe liver diseases, with MASH as the focus of its lead asset. The Company has significant expansion opportunities through its portfolio of promising drug development candidates that were acquired through partnerships with proven, innovative biotechnology and pharmaceutical companies. Boston Pharmaceuticals applies rigorous decision-making to advance programs to deliver differentiated medicines to patients in need of new options while creating value for all parties involved in the journey. For more information, please visit www.bostonpharmaceuticals.com and follow us on LinkedIn .

Boston Pharmaceuticals succeeded on two closely watched efficacy endpoints for its Phase 2 MASH study, putting the company in contention with other biotechs seeking to treat the liver disease. The company reported Friday that its FGF21 analog, called efimosfermin alfa, spurred a statistically significant improvement in both fibrosis without worsening of MASH, and resolution of MASH without worsening of fibrosis. The new data assessed the once-monthly shot over 24 weeks in 84 patients with advanced stages of MASH, also known as metabolic dysfunction-associated steatohepatitis. The company said it plans to discuss the data with regulators and advance the drug into late-stage development next year. As is frequently the case in MASH studies, efimosfermin was considerably better at resolving MASH than improving fibrosis, a type of liver scarring that’s a hallmark of the disease. In the group treated with efimosfermin, 67.7% had their MASH disease improve without their fibrosis worsening, compared with 29.4% of patients given placebo. For the fibrosis endpoint, which uses a scale to measure liver scarring, 45.2% of patients getting the drug had at least a one-stage improvement without worsening of MASH, compared to 20.6% of patients on placebo. Boston didn’t report a statistically significant number of treated patients who had both MASH resolution and fibrosis improvement compared to placebo, a higher bar than the other two endpoints. The trial’s primary endpoints were safety and tolerability, according to a government database of trials. Patients receiving efimosfermin had low discontinuation rates, according to the company, and “an overall low incidence of gastrointestinal side effects and injection site reactions.” The data bode well for Boston’s prospects in the larger FGF21 race , though it still trails two other biotechs that are slightly ahead: Akero Therapeutics’ efruxifermin is in the middle of two Phase 3 studies, as is 89bio. All three companies are looking to carve out a slice of the market that Madrigal Pharmaceuticals currently has to itself. Madrigal’s treatment, Rezdiffra, became the first to win FDA approval to treat the underlying biology of MASH earlier this year and recently impressed investors with more than $62 million in third-quarter revenue. Novo Nordisk could become a competitor, too, after posting positive Phase 3 data earlier this month with plans to file for regulatory approval in the US and EU in the first half of 2025.