96-week data from the HARMONY Phase 2b trial show Akero Therapeutics’ efruxifermin (EFX) yielded a 49% achievement rate for at least one stage of fibrosis improvement (without worsening of MASH) in the 50mg cohort of patients with pre-cirrhotic (F2-F3) metabolic dysfunction-associated steatohepatitis (MASH). This compares favorably to the 19% rate observed in the placebo group. Secondary endpoints, including MASH resolution without worsening of fibrosis, were also met, with 37% and 40% of participants in the 50mg and 28mg EFX arms achieving this endpoint, respectively, compared to 19% in the placebo group.
The publication of these results in •The Lancet• solidifies EFX’s emerging profile as a potential treatment for MASH, a disease currently lacking approved therapies and representing a significant unmet medical need. The findings bolster Akero’s position as it moves EFX through Phase 3 trials. Demonstrating sustained efficacy and safety over 96 weeks is crucial, especially given the chronic nature of MASH and the likelihood of long-term treatment.
Akero’s strategy hinges on EFX’s ability to address both fibrosis and steatohepatitis, the core drivers of MASH progression. The data suggest EFX’s potential not only to halt but potentially reverse disease progression, particularly in patients with advanced fibrosis—a high-risk group with limited treatment options. This approach distinguishes EFX from other candidates that focus solely on inflammation or fibrosis reduction, potentially offering a more comprehensive therapeutic solution.
The trial’s success has a significant impact on multiple stakeholders. For patients, EFX represents a potential breakthrough, offering hope for disease reversal and improved quality of life. The data strengthens Akero’s position in discussions with regulatory agencies, potentially paving the way for accelerated approval pathways. For clinicians, EFX’s convenient once-weekly dosing and manageable safety profile simplify treatment administration and enhance patient adherence.
Looking ahead, the ongoing Phase 3 SYNCHRONY trial program will be critical in confirming these findings in a larger and more diverse patient population. Long-term safety data, particularly regarding the observed reduction in bone mineral density, will be closely scrutinized by regulators and clinicians. Furthermore, Akero will need to navigate the evolving MASH treatment landscape, which is witnessing increased competition from other promising drug candidates targeting various stages of the disease. The commercial viability of EFX will depend on demonstrating clear clinical benefits and establishing a competitive pricing strategy within a potentially crowded market. The continued development and eventual market entry of EFX could reshape the MASH treatment paradigm and influence future clinical trial design in this area.
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.
