Akero Therapeutics (AKRO) investor relations material

Key clinical insights and data

• Three phase III programs are underway, targeting MASH with a focus on both cirrhotic (F4) and pre-cirrhotic (F2/F3) populations.

• Efruxifermin, an FGF21 analog, demonstrated a 24% effect size in F4 patients and a 52% effect size in F2/F3 patients for one-stage fibrosis improvement in recent studies.

• Safety profile is consistent, with diarrhea and nausea most common; bone mineral density loss (~5%) is being managed proactively in phase III.

• Phase III SYNCHRONY program includes real-world, histology, and outcomes studies, with global enrollment and a more patient-friendly drug formulation.

• Amendments to trial protocols are being considered to optimize patient selection based on phase IIb data.

Regulatory and competitive landscape

• Two drugs (Rezdiffra and semaglutide) are now approved for MASH in the U.S., but only for F2/F3; no approved therapies exist for F4.

• Efruxifermin shows a higher effect size than approved agents (Rezdiffra ~11.5%, semaglutide ~14.5%) and is positioned for both F2/F3 and F4 populations.

• Accelerated approval is possible in Europe for F4 based on histology, but U.S. FDA guidance is less clear; regulatory timelines depend on ongoing enrollment and data.

• Other FGF21 programs are in development, but many have been discontinued; robust clinical data is seen as a key differentiator.

• Market opportunity is significant and growing, with only two products currently available and high unmet need, especially in F4.

Innovation and future outlook

• AI is being used for pathology analysis in clinical trials, showing high correlation with human pathologists, though not yet accepted by the FDA.

• The SYNCHRONY Real-World study will provide the largest safety dataset to date, with results expected in the first half of next year.

• Global phase III studies are ongoing, with no expected geographic differences in response.

• The company is monitoring innovation from China and sees the need for definitive clinical data before considering competition from emerging FGF21s.

• The market is expected to expand as more companies enter and as unmet needs in advanced fibrosis are addressed.