Galectin Therapeutics says GR-MD-02 is efficacious in preclinical model of fatty liver disease with fibrosis

NORCROSS, Ga.—In early summer, Galectin Therapeutics Inc., a developer of therapeutics that target galectin proteins to treat fibrosis and cancer, announced data from a preclinical study in a mouse model of non-alcoholic steatohepatitis (NASH), or fatty liver disease, demonstrating that oral administration of the company's lead galectin-3 inhibitor, GR-MD-02, resulted in significant disease improvement.

Diabetic mice, fed a high-fat diet to induce NASH, were treated after the development of disease with either a vehicle control or GR-MD-02 administered orally five days out of seven for a total of four weeks. The liver weight, liver-to-body weight ratio and plasma triglyceride levels were significantly reduced in the animals treated with GR-MD-02 as compared to vehicle control animals. Blood indicators of liver damage, including plasma aspartate aminotransferase (AST), plasma alanine aminotransferase (ALT) and plasma total bilirubin (TB) also demonstrated a statistically significant reduction following oral treatment with GR-MD-02 and, in fact, levels were reduced back to near normal levels, Galectin reported.

AST, ALT and TB in normal animals were increased in the NASH animals treated with vehicle and were significantly reduced with oral GR-MD-02 treatment.

Finally, fibrosis of the liver was significantly reduced with treatment with GR-MD-02, as indicated by the liver hydroxyproline content, a biochemical marker of collagen in the liver. Liver hydroxyproline content in the normal liver was increased in the NASH animals treated with vehicle and was significantly reduced with oral GR-MD-02 treatment.

“Oral activity of GR-MD-02 in this established preclinical model of NASH represents an important step in the development of galectin inhibitors and complements our ongoing clinical program of intravenous administration in patients with NASH with advanced fibrosis,” said Dr. Peter G. Traber, president, CEO and chief medical officer of Galectin. “We have evaluated various established technology platforms and are currently developing oral formulations with a contracted firm with the goal of developing an oral formulation for human studies as a follow-on to our current clinical development program.”

The company also has an ongoing clinical trial of GR-MD-02 titled, “A Multi-Center, Partially Blinded, Maximum Tolerated Multiple Dose Escalation, Phase 1 Clinical Trial to Evaluate the Safety of GR-MD-02 in Subjects with Non-Alcoholic Steatohepatitis (NASH) with Advanced Hepatic Fibrosis.” In 2013, Galectin Therapeutics received Fast Track designation from the U.S. Food and Drug Administration for this clinical development program.