Shares of yet another NASH hopeful are rocketing north this morning, with Tel Aviv-based Galmed posting news on its Phase IIb fatty liver disease drug. Although the data are exciting some investors, the results were also distinctly mixed, raising some doubts about the sudden enthusiasm.
The company, which reported a flop for the rollout of its last NASH trial, today reported top-line, 52-week results from a global study testing its drug Aramchol in a high dose (600 mg) and lower dose (400 mg), alongside a placebo. The goal was to cut down liver fat, with the secondary endpoints shooting for a reduction in scarring (fibrosis) and NASH resolution. The lower dose met the primary endpoint, the company said, posting a statistically significant reduction in liver fat vs. placebo (p=0.0450). The high dose did not.
Interestingly, though, the FDA’s approvable endpoints for NASH do not include a reduction in liver fat alone. For a Phase III trial, which Galmed hopes to enter with this drug, the FDA recommends NASH resolution as an endpoint, and/or at least one-point improvement in fibrosis with no worsening of NASH.
Galmed says its high dose achieved NASH resolution in 19.2% of patients (p=0.0462), compared to 7.5% in the placebo group.
It appears this is what Galmed is rallying around — the chance that its higher dose can achieve NASH resolution in a Phase III trial.
“Aramchol 400 mg is probably sufficient for fat reduction but, biologically, a higher dose is needed for achieving more stringent histological endpoints such as NASH resolution and fibrosis reversal,” said principal investigator Vlad Ratziu in a statement. “NASH is a chronic disease with complex comorbidities and Aramchol’s favorable safety and tolerability profile support long-term treatment.”
Galmed’s stock $GLMD is up 205% as of press time, opening around $25 per share Tuesday morning. That follows Monday night’s close at $7.05 per share. Biotwitter included some skeptical notes as the stock soared, but analysts Edward Nash and Fang-Ke Huang from Suntrust sent me their latest note on Galmed, which came off cautiously optimistic.
“Our first take on the data is positive as the side effect profile was very benign and efficacy was demonstrated in both fibrosis and NASH in the two dose cohorts combined. While not all measurements achieved statistical significance, there was a clear trend in improvement which we believe is impressive.”
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