According to a story from globenewswire.com, the drug development company Galectin Therapeutics, Inc., recently announced its plans to go forward with Phase 3 trials for GR-MD-02. The company is dedicated exclusively to developing treatments that act on galectin proteins. GR-MD-02 is classified as a galectin-3 inhibitor, and is in development for the treatment of cirrhosis caused by nonalcoholic steatohepatitis.
Nonalcoholic steatohepatitis (NASH) is the most severe type of nonalcoholic fatty liver disease. This most extreme form is generally rare. The disease is characterized by a build up of fat in the liver and inflammation of the organ. When treatment is delayed, Nonalcoholic steatohepatitis can progress to cirrhosis, and it is considered one of major causes of cirrhosis of unknown origin. Symptoms may not always be present, but they can include a general feeling of unwellness, fatigue, and abdominal pain on the right side. There have been no treatments introduced so far specifically to target nonalcoholic steatohepatitis. It is associated with metabolic syndrome, insulin resistance, and lysosomal acid lipase deficiency. To learn more about nonalcoholic steatohepatitis, click here.
The implementation of Phase 3 trials heralds a major step for the company. While there are many treatment routes available for cirrhosis in general, the approval of GR-MD-02 would be the first treatment specifically for cirrhosis that is caused by NASH. The trial design was developed in collaboration with the U.S. Food and Drug Administration (FDA), and after meetings between Galectin and the agency, the two groups have agreed on two specific endpoints.
These endpoints are the development of esophageal varices and changes to the hepatic venous pressure gradient (HVPG), which is a measure of blood pressure in the liver. These endpoints are based on results of the prior Phase 2 trial, in which GR-MD-02 was able to alter HVPG in a favorable manner and also appeared to delay or even prevent the development of esophageal varices.
The company is convinced that the results of the Phase 2 trial alone were sufficient to warrant the new therapy Breakthrough Therapy designation, which would impart significant benefits for the approval process, but the FDA has been hesitant, and wants to see if data from Phase 3 will reinforce the positive results from Phase 2. Hopefully, the new trial will continue the investigational drug’s intriguing track record so far.
