Dive Brief:
- Galectin Therapeutics reported Tuesday its liver disease drug GR-MD-02 failed to meet either primary or secondary endpoints in a short, exploratory Phase 2a trial testing the compound among non-alcoholic steatohepatitis (NASH) patients with advanced fibrosis.
- The biotech company downplayed the significance of the four-month trial, saying it has higher hopes for positive results from a longer, larger-scale study of the drug in patients with NASH cirrhosis. Even so, Galectin shares dropped sharply in Wednesday morning trading, nearly halving in value.
- Allergan's acquisition of two NASH-focused companies last week had buoyed investor confidence in the NASH market, boosting share prices for a number of other biotechs in the space. For Galectin, at least, that enthusiasm quickly burst after news of the trial miss.
Dive Insight:
NASH has drawn interest as a potentially lucrative market, given the lack of treatment options and growing prevalence. The disease is hard to detect before progressing to more advanced stages, earning it descriptions as a "silent liver" disease or hidden epidemic.
The NASH-FX clinical trial did not meet its primary biomarker endpoint, or secondary endpoints that measure liver stiffness as a surrogate for fibrosis. Liver biopsies were not performed at the end of the study due to safety concerns.
Galectin had hoped the study would show fewer administrations of the drug could lead to improve measurements of liver stiffness, citing data from an earlier Phase 1 study which suggested that might be the case.
Company President and CEO, Peter Traber did his best to downplay the negative results. "We saw the effect in phase 1 and wanted to see the effect over a shorter period," he said, speaking on a call with investors. The major good news, he said, was that GR-MD-02 was found to be safe and well tolerated, with no serious adverse effects;
A larger one-year trial in 162 patients with NASH cirrhosis recently completed enrollment, setting up a top-line readout in December 2017, the company said.
That trial will be conducted with a primary endpoint (hepatic venous pressure gradient), which the company believes could be an acceptable surrogate for outcomes.
"Although there was no apparent improvement in the three non-invasive tests for assessment of liver fibrosis in this four month trial (NASH-FX), inhibition of galectin-3 with GR-MD-02 remains promising for treatment of NASH fibrosis," lead investigator Stephen Harrison said. "We look forward to additional results from the NASH-CX trial in which patient with NASH cirrhosis are treated for one year."
Galectin recently closed a private placement financing for $1.5 million to help fund the one-year trial.