Boston Pharmaceuticals has announced positive Phase II data for efimosfermin alfa in patients with metabolic dysfunction-associated steatohepatitis (MASH), showcasing significant improvements in liver fibrosis. The investigational therapy, a long-acting analog of fibroblast growth factor 21 (FGF21), is administered once-monthly, potentially offering a more convenient treatment option compared to other FGF21 analogs.
Mechanism of Action
Efimosfermin alfa works by regulating metabolic processes to reduce liver fat and prevent inflammation and damage. This mechanism allows the drug to decrease liver damage and potentially reverse fibrosis, while also improving metabolic health. The convenience of monthly dosing could provide an advantage in the competitive MASH treatment landscape.
Phase II Trial Results
The Phase II data, presented at The Liver Meeting of the American Association for the Study of Liver Diseases, revealed that 45.2% of patients treated with efimosfermin alfa experienced at least a one-stage improvement in fibrosis without worsening of MASH, compared to 20.6% in the placebo group (p=0.038). Furthermore, 67.7% of treated patients achieved MASH resolution without fibrosis worsening, versus 29.4% in the placebo group, also a statistically significant result.
Lead investigator Mazen Noureddin, director of the Houston Research Institute, emphasized the importance of treatment adherence for MASH patients, suggesting that the monthly dosing schedule of efimosfermin alfa could improve patient compliance and overall management of the disease.
Safety and Tolerability
Efimosfermin alfa demonstrated a favorable safety profile over the 24-week treatment period, with a low incidence of injection-site reactions and gastrointestinal toxicities. Dropouts due to side effects were uncommon, indicating good tolerability among patients.
Competitive Landscape
While efimosfermin alfa is still in mid-stage development, competitors like Akero and 89bio are further along with their FGF21 analogs, efruxifermin and pegozafermin, respectively, both in Phase III development with weekly dosing. However, the monthly dosing of efimosfermin alfa could differentiate it in the market.
Future Development
Boston Pharma plans to continue Phase II development with an open-label extension phase in patients with F2 and F3 fibrosis. CEO Sophie Kornowski stated that data from this extension, along with results from an advanced fibrosis study, will support discussions with regulatory authorities before entering pivotal studies.
