Novel NASH drug fails to meet endpoints in short-dose study

Results from the NASH-FX clinical trial involving GR-MD-02 show the drug met neither its primary endpoint of improving inflammation and fibrosis nor the secondary endpoint of improved liver stiffness with a short course of treatment, manufacturer Galectin Therapeutics said in a press release.

“Although there was no apparent improvement in the three non-invasive tests for assessment of liver fibrosis in this 4-month pilot trial, inhibition of galectin-3 with GR-MD-02 remains promising for treatment of [nonalcoholic steatohepatitis] fibrosis,” Stephen A. Harrison, MD, principal investigator of NASH-FX and medical director of Pinnacle Clinical Research in San Antonio, said in a conference call with reporters. “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.”

According to Galectin, researchers assessed fibrosis using LiverMultiScan (Perspectum Diagnostics) and evaluated liver stiffness via magnetic resonance elastography and FibroScan (Echosens). Thirty patients were included in the study, 15 treated with GR-MD-02 and 15 received placebo. The endpoint was an adjustment of baseline mean value of corrected T1 value as determined by LiverMultiScan, according to Galectin.

“The NASH-FX trial was designed to follow up on limited data from a phase 1 study in NASH with advanced fibrosis, which suggested that FibroScan measurements may have improved with just four doses of drug,” Harrison said in the press release. “However, as we have witnessed in other liver fibrosis trials, the relatively short treatment duration of only 4 months assessed in the NASH-FX was inadequate to see an efficacy response.”

Through NASH-FX and an ongoing NASH-CX study, more than 1,600 doses of GR-MD-02 (Galectin Therapeutics) have been administered with no serious adverse effects. NASH-FX’s failure to meet its endpoints means researchers will use results from the year-long NASH-CX study in patients with NASH cirrhosis to advance GR-MD-02’s treatment potential, according to the release.

“It is critical that we complete the longer therapy, much larger NASH-CX trial,” Harrison said in the press conference.

Results from the NASH-CX trial are expected by December 2017. – by Janel Miller

Disclosure: Healio.com/Hepatology was unable to determine Harrison’s relevant financial disclosures at time of publication.