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Akero Therapeutics Reports Histological Data From All Efruxifermin Dose Groups In 16-Week Phase 2a BALANCED Study In NASH Patients

Akero Therapeutics, Inc. (NASDAQ:AKRO) today announced results of a 16-week analysis of secondary and exploratory endpoints in its Phase 2a BALANCED study of efruxifermin (EFX), formerly known as AKR-001, in patients

Benzinga · -

Akero Therapeutics, Inc. (NASDAQ:AKRO) today announced results of a 16-week analysis of secondary and exploratory endpoints in its Phase 2a BALANCED study of efruxifermin (EFX), formerly known as AKR-001, in patients with nonalcoholic steatohepatitis (NASH). Notably, of the 40 treatment responders who had end-of-treatment biopsies, we observed that 48% achieved at least a one-stage improvement in fibrosis without worsening of NAFLD activity score (NAS) and 28% achieved at least a two-stage improvement in fibrosis. In addition, 48% of responders achieved NASH resolution with no worsening of fibrosis. Improvements in glycemic control and dyslipidemia, as well as weight loss, were also observed across all dose groups. Treatment with EFX was generally reported to be well tolerated.

Akero Therapeutics, Inc. Logo (PRNewsfoto/Akero Therapeutics, Inc.)

"These substantial improvements observed in multiple measures of liver health, particularly the one- and two-stage improvements in fibrosis, are extremely encouraging and among the strongest biopsy results reported in NASH to date," said Stephen Harrison, M.D., medical director of Pinnacle Clinical Research. "I believe Efruxifermin continues to set itself apart as one of the most promising drug candidates in NASH, with impressive histology results after just 16 weeks of treatment."

Summary of Week 16 Biopsy Endpoints1

Measure (Mean)

Placebo

(N=2)

All EFX

(N=40)

28mg

(N=13)

50mg

(N=13)

70mg

(N=14)

Improvement in at least one stage of fibrosis
without worsening NAS (%)2

0

48

46

62

36

Improvement in at least two stages of
fibrosis (%)2

0

28

31

38

14

Resolution of NASH without worsening of
fibrosis (%)2

50*

48

46

54

43

Combination of improvement in at least one
stage of fibrosis and NASH resolution (%)2

0

28

31

39

14

NAS Reduction ≥2 points without worsening
of fibrosis (%)2

50*

78

77

77

79

1 Secondary and exploratory histological endpoints were not powered for statistical significance
2 Liver Biopsy Evaluable Analysis Set (all patients who had Baseline and end-of-treatment liver biopsy results)
* A single placebo responder lost 25 pounds over 16 weeks (11% weight reduction)

The BALANCED study underscored EFX's potential to address multiple important NASH comorbidities. We observed that all dose groups had mean weight loss over the 16-week study, with the 70 mg dose group achieving a statistically significant 3.7kg (about 8 pounds) reduction in body weight at Week 16. Clinically meaningful improvements in glycemic control were observed, including significant reductions in HbA1c in the 50 and 70 mg dose groups of 0.4 and 0.5, respectively. EFX also improved dyslipidemia, including significant increases in HDL cholesterol and significant decreases in triglycerides observed across all EFX dose groups.

Summary of Cardio-Metabolic Biomarkers

Measure (Mean Change From Baseline)

Placebo

(N=21)

28 mg

(N=19)

50 mg

(N=20)

70 mg

(N=20)

Body Weight (kg)1

+0.1

-0.3

-2.3

-3.7*

HbA1C (%, absolute)1

+0.1

-0.1

-0.4*

-0.5**

Triglycerides (%)1

+8

-37***

-45***

-43***

HDL Cholesterol (%)1

0

+32***

+40***

+40***

Non-HDL Cholesterol (%)1

0

-20***

-13*

-15**

LDL Cholesterol (%)1

+1

-14*

0

-3

1 Full Analyses Set (all patients randomized into the study)
*p<0.05, **p<0.01, ***p<0.001, versus placebo

EFX was reported to be generally well tolerated. There were no deaths in the study, and there were two Serious Adverse Events, one of which occurred prior to dosing. Across EFX groups, the most frequent AEs were grade 1 or 2 gastrointestinal events, which were transient in nature. There were no discontinuations due to treatment-emergent adverse events in the 50 mg dose group and no discontinuations due to the most common adverse event, diarrhea. There were no treatment-related effects on blood pressure, heart rate or bone mineral density.

"We believe the BALANCED study data, which exceeded our expectations, demonstrate the strong potential of efruxifermin to be a foundational monotherapy for the treatment of NASH," said Andrew Cheng, M.D., Ph.D., president and CEO of Akero. "We look forward to the continued development of efruxifermin and are working diligently to deliver this potentially leading treatment to patients. We are extremely grateful to all of our investigators and study patients, particularly given that this study cohort was completed amidst the COVID-19 pandemic."

The BALANCED study is an ongoing randomized, double-blind, placebo-controlled study in NASH patients. The company previously reported that each of the 28, 50 and 70 mg EFX dose groups met the primary endpoint compared to placebo, with absolute reductions of 12, 13 and 14 percent of liver fat, respectively, compared with 0.3 percent for placebo, and relative reductions of 63, 71 and 72 percent, compared to 0 percent for placebo. All of these results were highly statistically significant at p<0.001.