Akero Therapeutics (AKRO) Announces Positive Histological Improvements in Cirrhotic NASH (F4) Patients after 16 Weeks in Extension Cohort C

March 22, 2021 4:03 PM EDT

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33% of patients treated with efruxifermin (EFX) (4 of 12) improved by one fibrosis stage without worsening of NASH --

-- 25% of EFX patients (3 of 12) showed NASH resolution --

-- Rapid fibrosis improvement in cirrhotic patients after only 16 weeks of EFX treatment, the highest rate reported publicly to date, suggests direct anti-fibrotic effects --

Akero Therapeutics, Inc. (Nasdaq: AKRO), a cardio-metabolic biotechnology company developing transformational treatments for non-alcoholic steatohepatitis (NASH), today announced results of an expansion cohort of a 16-week Phase 2a clinical trial, Cohort C, evaluating efruxifermin (EFX) in the treatment of adult patients with cirrhotic nonalcoholic steatohepatitis (NASH) (compensated stage 4 fibrosis, Child-Pugh Class A). Of the 17 confirmed compensated cirrhosis (F4) study subjects who volunteered to have end-of-treatment biopsies, 4 of 12 patients (33%) treated with EFX achieved a one-stage improvement in fibrosis without worsening of NASH. Another 3 of 12 EFX patients (25%) achieved NASH resolution. In total, 7 of 12 EFX patients (58%) showed histological improvements. None of the 5 placebo patients (0%) achieved either one-stage improvement in fibrosis without worsening of NASH, or resolution of NASH. In addition, statistically significant improvements in glycemic control and lipoprotein profile, and a trend toward weight loss, were also observed.

“I believe these data are unprecedented,” said Stephen Harrison, M.D., medical director of Pinnacle Clinical Research. “Today’s data in cirrhotic patients, who have the highest unmet need, show clear signals of fibrosis improvement without worsening of NASH and NASH resolution, supported by compelling, statistically significant results for non-invasive fibrosis measures. These results set EFX apart.”

Cohort C is an expansion of the Phase 2a BALANCED study evaluating EFX in the treatment of F4 NASH patients, Child-Pugh Class A. Thirty cirrhotic NASH subjects with a historical biopsy-confirmed fibrosis score of F4 were randomized 2:1 to receive either 50mg of EFX or placebo for 16 weeks. A total of 27 subjects were subsequently confirmed by the central reader to have F4 fibrosis at baseline. The primary objective of Cohort C was to assess the safety and tolerability of EFX in NASH patients at greatest risk of progressing to end-stage liver disease, including liver failure and liver cancer. Secondary objectives included assessments of liver stiffness by Fibroscan and serum markers of liver fibrosis, such as the Enhanced Liver Fibrosis (ELF) score and Pro-C3. The trial design was amended to allow voluntary end-of-treatment biopsies.

Summary of Biopsy Results and Non-Invasive Fibrosis Measurements

Histology Endpoint (% responders)Placebo(n=5)50mg (n=12)
Improvement in at least one stage of fibrosis without worsening of NASH,%1,2033
Resolution of NASH, %1,2025
Non-invasive measurement (LS Mean)Placebo(n=10)50mg (n=20)
Liver Stiffness, kPa3-1.9-5.7††
Pro-C3, μg/L4-3.4-9.0*
ELF Score4+0.3-0.4*

1 Study not powered to assess statistical significance of changes in histological endpoints2 Liver Biopsy Evaluable Analysis Set (all patients who had baseline and end-of-treatment liver biopsy results)3 Liver Stiffness Analysis Set (all subjects with a week 16 FibroScan)4 Biomarker Analysis Set (all subjects with a post baseline interpretable measure of ELF or pro-C3, respectively)†† p<0.01, versus baseline (ANCOVA)* p<0.05, versus placebo (ANCOVA)

EFX was reported to be generally well-tolerated. The most common adverse event in the EFX group was mild or moderate diarrhea. There were two discontinuations, one in the placebo group and one in the EFX group. There was one serious adverse event in the placebo group and no deaths in either group.

“The promising results in cirrhotic NASH patients reported today build on the strong results previously reported for patients with F1-F3 fibrosis,” said Andrew Cheng, M.D., Ph.D., president and CEO of Akero. “We believe EFX has the potential to be a foundational NASH monotherapy for cirrhotic patients as well as patients with earlier-stages of fibrosis. We look forward to continuing the development of our Phase 2b HARMONY study in patients with F2-F3 fibrosis started in February 2021, and our planned Phase 2b SYMMETRY study in cirrhotic patients (F4 fibrosis), which we plan to initiate in the second half of this year. We remain extremely grateful to all of our study patients and investigators, particularly given that this study cohort was conducted during the COVID-19 pandemic.”

Conference Call / Webcast DetailsThe company will host a conference call and webcast with slide presentation at 4:30 p.m. ET (1:30 p.m. PT) today, March 22. The webcast will be made available on Akero’s website at www.akerotx.com under the Investors tab in the Events, Presentations & Webcasts section. To access the call, please dial 1-877-282-0556 (U.S. toll free) or 1-270-215-9899 (international) five minutes prior to the start time, and provide Conference ID #1885464. Following the live audio webcast, a replay will be available on the company's website for 90 days.

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