(Reuters) - Galectin Therapeutics Inc said its drug for the complex progressive fatty liver disease NASH led to some clinically meaningful results in patients with cirrhosis, but failed to achieve the main goal of a mid-stage trial, sending its shares plunging 48 percent on Tuesday.
The primary goal of the study of 162 patients with NASH that had progressed to cirrhosis was a significant change in hepatic venous pressure gradient, or HVPG, a measure of high blood pressure in vessels that serve the liver also known as portal hypertension, which is a consequence of chronic liver disease.
Despite a positive trend Galectin said its drug, GR-MD-02, failed to achieve statistical significance versus placebo due to variability of results in patients with esophageal varices - abnormal, enlarged veins in the tube that connects the throat and stomach, common in people with serious liver diseases.
For the half of patients in the study who did not have esophageal varices, Galectin said its drug yielded a statistically significant and clinically meaningful reduction of HVPG.
In addition, GR-MD-02, led to a statistically significant improvement in hepatocyte ballooning, or liver cell death, a key factor in the underlying disease process in NASH, Galectin said.
There was also a statistically significant reduction in the development of new esophageal varices in patients without them at the start of the trial, the company reported.
GR-MD-02’s success in reducing ballooning and preventing development of esophageal varices “can’t be brushed under the rug”, one of the trial’s lead investigators said on a conference call on Tuesday.
NASH, formally known as non-alcoholic Steatohepatitis, is seen as one of the fastest growing diseases in the developed world with no approved treatments. Many companies are trying to develop drugs to tackle various aspects of the condition, so far with minimal success.
The disease is poised to become the leading cause of liver transplants by 2020 with the eventual market forecast to be $20 billion to $35 billion as populations with fatty diets increasingly fall victim to the condition.
GR-MD-02 targets galectin-3, a protein seen as critical in development of fatty liver disease and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs, such as cirrhosis, which can lead to liver failure.
The drug developer’s shares recouped some losses and were down 44 percent at $1.38 in morning trading.
Reporting by Bill Berkrot in New York and Divya Grover in Bengaluru; Editing by Richard Chang and Sriraj Kalluvila