September 28, 2016
By Alex Keown, BioSpace.com Breaking News Staff
NORCROSS, Ga. – Shares of Galectin Therapeutics plummeted more than 43 percent this morning after the company announced its Phase IIa study of its lead product candidate, GR-MD-02 evaluating 30 non-alcoholic steatohepatitis (NASH) with advanced fibrosis failed to meet primary endpoints.
This morning the company announced the trial did not meet its primary biomarker endpoint as measured by LiverMultiScan (LMS, Perspectum Diagnostics), a magnetic resonance imaging test that evaluates inflammation and fibrosis. Nor did the trial meet its secondary endpoint that measured liver stiffness as a surrogate for fibrosis. The results of the IIa trial looked at the drug’s efficacy over a short-term period of four months. However, Galectin said it remains convinced that GR-MD-02 remains an effective treatment for NASH fibrosis. GR-MD-02 is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of fatty liver disease and fibrosis.
“Although there was no apparent improvement in the three non-invasive tests for assessment of liver fibrosis in this four month pilot trial, inhibition of galectin-3 with GR-MD-02 remains promising for treatment of NASH fibrosis,” Stephen Harrison, principal investigator of the trial, said in a statement. “In regard to the potential activity of GR-MD-02, it is encouraging that there is an important clinical effect in moderate-to-severe psoriasis, suggesting the compound has activity in a human disease that can occur in association with NASH.”
Harrison said early data in a Phase I trial suggested that four treatment of GR-MD-02 could offer improvements of patients based on FibroScan measurements.
“However, as we have witnessed in other liver fibrosis trials, the relatively short treatment duration of only four months assessed in the NASH-FX was inadequate to see an efficacy response. Therefore, we look forward to additional results from the NASH-CX trial in which patients with NASH cirrhosis are treated for one year,” Harrison said.
Galectin is engaged in a year-long 162-patient trial studying GR-MD-02 in patients with NASH cirrhosis. Top line results of that trial are expected in December 2017, the company said. In the four month trial, GR-MD-02 was found to be safe and well tolerated among the patient population with no serious adverse events, the company said.
Although the four-month trial was a bust, Galectin said it received a $1.5 million investment from a single source, Richard Uihlein, chairman and chief executive officer of Uline Inc., to support the one-year NASH trial.
NASH is a progressive form of fatty-liver disease that has been directly associated with diabetes and obesity. Excessive accumulation of fat in the liver induces chronic inflammation, which causes progressive fibrosis, cirrhosis and eventually end-stage liver disease. The prevalence of NASH is increasing worldwide in part due to the increase of obesity and diabetes diagnoses. According to the National Institute of Diabetes and Digestive and Kidney Diseases, NASH affects 2 to 5 percent of people in the U.S. There are currently no specific treatments aside from weight loss, increased physical activity, and avoiding alcohol and unnecessary medications. NASH is projected to become the leading indication for liver transplant by 2020.
Shares of Galectin are trading at $1.08 as of 11:40 a.m.
NASH treatments are being explored by other companies too, including San Diego-based Akarna Therapeutics, Ltd., which was recently acquired by Allergan .
