- Cempra announced Friday interim positive Phase 2 results for bacterial agent solithromycin to treat non-alcoholic steatohepatitis (NASH), a fatty liver disease that has no known cure and has drawn intense development interest by a growing number of drugmakers trying to score in a potentially lucrative market.
- In the study, six patients were shown to have reduced NASH activity after 90 days of treatment. While there were few patients in the study, Chapel Hill, NC-based Cempra said the findings show the drug was well-tolerated by patients, resulting in a good safety profile.
- NASH impacts up to 16 million Americans, or 2% to 5% of the population. According to the National Institutes of Health, another 10% to 15% of Americans have fatty liver. NASH can ultimately result in cirrhosis.
While drugmakers are examining different approaches in non-alcoholic fatty liver development, Cempra is focusing on solithromycin, described as a next generation macrolide, an anti-bacterial drug, against most macrolide resistant CABP (community acquired bacterial pneumonia) pathogens.
In the Phase 2 study, the company reported five of six patients had a reduction in their aspartate aminotransferase (AST) and the sixth patient had an AST that was changed and normal.
Cempra has been working on development of the drug for the past 15 years. The principal investigator of the study acknowledged that there were a small number of patients in the study, and more work to do. "That solithromycin was able to lower NASH by an average of 1.3 points in just 13 weeks with a good safety profile does provide a good measure of optimism," said Pierre Gholam from Case Western Reserve University of Medicine.
Based on the Phase 2 findings so far, Cempra said it plans to continue the study to obtain data on up to 15 patients. Enrollment is expected to be completed during the first quarter of 2017.
Reflecting the level of competition for anti-NASH drugs, Allergan recently made two moves on that front — announcing it is acquiring privately held Akarna Therapeutics for $50 million upfront. Earlier the same day, the specialty pharma paid $1.7 billion for Tobira Therapeutics.
Elsewhere, Galectin Therapeutics reported Tuesday its liver disease drug GR-MD-02 failed to meet Phase 2a trial goals, but is continuing development.