NASH New Drug Reaches Phase 2 Clinical End Point, Liver Fat Decreases By 60%

- May 11, 2020-

On April 1, Akero Therapeutics announced that its main drug candidate AKR-001, in the Phase 2a clinical trial BALANCED for NASH patients, reached the end of the 12th week efficacy, reducing the liver fat level of patients by more than 60% compared with baseline , To achieve a significant reduction in statistical significance.

NASH is a serious non-alcoholic fatty liver disease (NAFLD), which refers to a pathological syndrome of excessive fat deposition in liver cells caused by factors other than alcohol. According to statistics, worldwide, NASH patients have exceeded 100 million. NASH causes inflammation and degeneration of liver cells. The progress of NASH will cause liver fibrosis, liver cirrhosis and liver failure, and may also induce liver cancer. As a type of metabolic inflammation, NASH patients are expected to exceed 350 million in 2030, and its occurrence is closely related to factors such as obesity, diabetes and abnormal blood lipid metabolism. Current treatments are still relatively scarce.

Akero's AKR-001 is a new type of long-acting fibroblast growth factor 21 (FGF21) analog. It combines the FGF21 polypeptide sequence with the Fc part of immunoglobulin and is considered a potential NASH therapeutic drug. Research in the past decade has shown that the endogenous hormone FGF21 plays a key role in human metabolism and signal regulation, and it can act on various organ systems including the liver. Usually it is naturally released to protect organs from stress and restore metabolic balance. Compared with natural FGF21, this drug can significantly improve the stability and half-life, and enhance the binding and activity of the receptor.

The study, called BALANCED, is a randomized, double-blind, phase 2a clinical trial with a placebo-controlled group. A total of 80 adult patients with NASH confirmed by biopsy participated. The test data showed that in the 12th week of treatment, the absolute liver fat levels of all patients in the AKR-001 dose group were significantly reduced from baseline, reaching the main endpoint of the trial. In addition, all AKR-001 dose groups reached the secondary endpoint of relative reduction in liver fat, and the 50 mg and 70 mg dose groups achieved a relative reduction of more than 70%.

"We are very pleased to receive these positive trial results, which support our continued development of AKR-001 for the treatment of NASH patients," said Andrew Cheng, MD, Akero President and CEO. "There are no FDA approved therapies available for NASH patients. AKR-001 has the potential to provide them with an important treatment option. We look forward to obtaining complete trial data to prepare for the next development of AKR-001. "