The United States is void of treatments for liver fibrosis, a disease that starts by fat accumulating in liver cells and eventually leads to scarring (fibrosis) and can result in severe scarring (cirrhosis). Norcross, Georgia-based Galectin Therapeutics (GALT) is looking to one day fill that void with its focus on galectin proteins in the development of drugs for fibrosis and cancer. On Monday, the company’s stock got a significant lift after announcing that the U.S. Food and Drug Administration has granted Fast Track designation for its complex carbohydrate drug candidate GR-MD-02 in the treatment of patients with non-alcoholic steatohepatitis, or “NASH,” with hepatic fibrosis. GR-MD-02 targets galectin-3, a protein known to play a key role in the pathogenesis of fatty liver disease and fibrosis, binding to it the protein and disrupting its function.
Fatty liver disease with advanced fibrosis, the more common phrase for NASH with hepatic fibrosis, is growing in prevalence with rising obesity rates in the United States. The latest stats from the Centers for Disease Control and Prevention show that 35.7 percent of all adult Americans are clinically obese with many having fatty liver disease to some degree. According to Galectin, up to 15 million people in the U.S. are affected by NASH with no FDA-cleared therapies.
Early in July, Galectin submitted its application to the FDA requesting the Fast Track designation for GR-MD-02 for the specific indication. The company has a Phase 1 clinical trial ongoing with the first patients being dosed with GR-MD-02 late last month. Up to 40 patients will be enrolled in the clinical trial across six centers in the U.S.
In laboratory studies, GR-MD-02 demonstrated “robust treatment effects in reversing fibrosis and cirrhosis,” said Dr. Peter Traber, a man who wears many hats at Galectin as president, CEO and chief medical officer, in a statement today.
In pre-clinical research, Galectin induced diabetes in mice and then fed high-fat diets, conditions similar to the human disease. After four weeks of treatment with GR-MD-02, significant reductions in fat accumulation, hepatocyte degeneration and inflammation in the liver histology were documented. Collagen levels in the liver returning to normal levels were also demonstrated. Additional lab research reiterated the effects ahead of clinical trials.
A Fast Track designation is designed to expedite the development process of drugs that are for serious diseases that lack sufficient (if any) treatments today. Many tactics are used to achieve this, including additional meetings with FDA officials and the submittal of data for review when collected, rather than all at once as customary.
Galectin says that GR-MD-02 has also delivered promising results in pre-clinical studies to reverse fibrosis in scarring of the lungs and kidneys.
Shares of GALT rose as high as $7.95 in early trading after the news hit the wire, marking the highest level since June 2011. The company has been garnering attention this year as its Phase II trial of GM-CT-01 for melanoma moves ahead and now the developments with GR-MD-02. Shares are up more than 200 percent in 2013.
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