Drug showed therapeutic effect on NASH patients in early trial

Phase 1 trial results indicated that an experimental galectin-inhibiting drug had a therapeutic effect on biomarkers that reflect fibrosis, inflammation and cellular injury in patients with fatty liver disease, according to a press release from Galectin Therapeutics.

Eight patients with nonalcoholic steatohepatitis (NASH) and advanced fibrosis were randomly assigned four doses of 2 mg/kg (80 mg/m²) of GR-MD-02, a galectin-inhibitor, for 42 days (n=6) or placebo (n=2), the release said. GR-MD-02 targets and binds to galectin-3 proteins and disrupts their function.

Patients in the first of three planned cohorts showed no serious adverse events after GR-MD-02 therapy, the drugmaker said. After single and multiple doses, the drug was well tolerated, and biomarkers of fibrosis and inflammation showed improvement after four doses. Patients with greater evidence of liver cell injury had a marked decrease in CK-18, a clinical biomarker of cell death, and galectin-3 blood levels remained unchanged after therapy.

“We are extremely pleased with the positive results … which suggest a role for GR-MD-02 in the treatment of patients with fatty liver disease with advanced fibrosis,” Peter G. Traber, MD, chief executive officer, president and chief medical officer at Galectin Therapeutics, said in the release. “Intervention with the intent of reversing the fibrosis is a potentially important therapeutic approach in fatty liver disease, a condition with significant unmet medical need.”

Galectin said eight participants with NASH and advanced fibrosis will be enrolled this month in the second phase 1 trial. GR-MD-02 will be increased to 4 mg/kg (160 mg/m²) in the cohort; results are expected in late summer.