Benzinga | Nov 12, 2021 08:23AM EST

Akero Highlights Presentation Of New Analysis Of Phase 2a BALANCED Study Data Showing Additional Qualitative Evidence Of Histological Improvement In EFX-treated NASH Patients After 16 Weeks Of Treatment

Most EFX-treated patients with end-of-treatment biopsies showed improvements in features of steatohepatitis (35 of 40; 87%) and/or fibrosis (32 of 40; 80%), after only 16 weeks

Histological improvements were evident across all types of patients, including those at higher risk of progressing to advanced stages of NASH

SOUTH SAN FRANCISCO, Calif., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (NASDAQ:AKRO), a cardio-metabolic biotechnology company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today announced a new, blinded, post-hoc analysis of its Phase 2a BALANCED study of efruxifermin (EFX) in biopsy-confirmed patients with non-alcoholic steatohepatitis (NASH), which will be presented in a poster at The Liver Meeting of the American Association for the Study of Liver Diseases (AASLD), Nov. 12-15, 2021.

The analysis, entitled "Characterization of Histologic Patterns of Improvement Following Treatment With Efruxifermin (EFX) in NASH Patients With Fibrosis," evaluated pre- and post-treatment biopsies in 40 EFX-treated patients from the BALANCED study. These post-treatment biopsies showed improvements in histological features of steatohepatitis in 87% (35 of 40) and fibrosis in 80% (32 of 40) of EFX-treated patients after 16 weeks of treatment.

Patterns of disease regression were evident in many patients who did not meet the categorical thresholds for either NASH resolution without worsening of fibrosis or at least a one-stage improvement in fibrosis without worsening of NASH. For example, some patients achieved resolution of hepatocyte ballooning without complete resolution of NASH, and some patients with bridging fibrosis (F3) showed evidence of features of fibrosis regression (e.g. interrupted septa) without complete reversion to a lower, non-bridging stage. These and other qualitative improvements, which are consistent with previously reported reductions in categorical scores, provide further evidence of the potential rapidity of EFX's disease modifying activity.

"More than 80% of biopsied patients treated with EFX showed signs of histological improvements after only 16 weeks of treatment with EFX. The observed trends highlight the potential to achieve higher response rates after longer periods of treatment, based on the categorical endpoints accepted for use in Phase 3 trials," said Kitty Yale, chief development officer of Akero. "We are eager to see the histology results after treatment for 24 weeks or more in the ongoing Phase 2b studies with EFX."

The BALANCED study was a randomized, placebo-controlled Phase 2a trial that enrolled 80 biopsy-confirmed, pre-cirrhotic NASH patients (F1 to F3 fibrosis stage) who received either placebo or EFX for 16 weeks as a weekly subcutaneous injection in one of three doses: 28 mg, 50 mg, or 70 mg. Of the 40 EFX-treated patients who received end-of-treatment biopsies, 48% achieved a one-stage improvement in fibrosis without worsening of NASH, and 48% achieved NASH resolution without worsening of fibrosis. These two endpoints remain the FDA-recommended endpoints for Phase 3 clinical trials in pre-cirrhotic NASH patients.

The analysis presented at AASLD was proposed by Cynthia A. Behling, M.D., Ph.D., a liver pathologist at Pacific Rim Pathology Medical Group in San Diego who was the central reader for the BALANCED study, based on her assessments of biopsies concurrent with categorical scoring and blinded to both treatment arm and biopsy sequence. The analysis provides a more detailed view of histological change than is reflected in the categorical, FDA-recommended scoring for NASH resolution and fibrosis stage. Moreover, two target populations at elevated risk of NASH progression--carriers of the PNPLA3 risk allele (I148M) and/or those with Type 2 diabetes--showed comparable qualitative and/or quantitative histological improvements to the rest of the patients in the BALANCED study.

"This qualitative analysis gives us even greater confidence in the emerging evidence of EFX's anti-fibrotic effects and the potential for a strong performance in reaching phase 3 study endpoints with longer treatment periods," said Andrew Cheng, M.D., Ph.D., president and chief executive officer of Akero. "Taken together, this and other analyses of our data provide compelling evidence that EFX has the potential to treat many of the complex causes of NASH, as well as to treat patients at greatest risk of progression to later stages of NASH-associated fibrosis and cirrhosis."