Bristol-Myers Squibb’s investigational liver disease candidate has hit its targets in a mid-stage trial, supporting further research on the drug as a potential treatment for Nonalcoholic Steatohepatitis (NASH).
The firm reported Phase II data showing that BMS-986036, an investigational pegylated analogue of human fibroblast growth factor 21 (FGF21), a key regulator of metabolism, significantly reduced liver fat in patients confirmed to have the condition.
At Week 16, both dosing regimens of BMS-986036 (10mg daily or 20mg weekly) significantly reduced liver fat compared to placebo: 6.8 percent and 5.2 percent, respectively, versus. 1.3 percent.
Statistically significant improvements were also seen in pre-specified exploratory endpoints including biomarkers of fibrosis, metabolic parameters and markers of liver injury, the firm said.
“These data suggest that BMS-986036 may be effective in patients with NASH, many of whom will experience disease progression due to the lack of available treatment options,” said Arun Sanyal, professor, Departments of Medicine, Physiology, and Molecular Pathology, Virginia Commonwealth University.
“The results of this study show that BMS-986036 had beneficial effects on three important components in the treatment of NASH: liver fat, liver injury and fibrosis.”
“These data, along with previously announced Phase II data in patients with type 2 diabetes, support further clinical research of BMS-986036 as a potential treatment for NASH,” added Mike Burgess, head of Cardiovascular, Fibrosis and Immunoscience Development, at BMS. “We look forward to sharing these data with health authorities to determine next steps for further study of this asset.”
NASH is a serious liver disease resulting from metabolic dysfunction that causes toxic build-up of fat in the liver (steatosis) that can lead to inflammation, hepatocellular injury, progressive fibrosis and cirrhosis.