The next time a biotech team decides to run a quick proof-of-concept study to back up their clinical quest on a drug designed to fight a big-market disease, they might want to carefully consider what can go wrong.
Take Galectin $GALT, for example.
Developing a new NASH contender, a hot field for drug developers these days, the small Norcross, GA-based biotech decided to recruit 30 patients for a short “pilot” trial to run a few months, with a biomarker on liver fibrosis in focus.
Not only did the Phase IIa study fail on the primary endpoint, it also flopped on secondaries. Galectin’s shares were crushed — despite the company’s best efforts to explain it all away — plunging by half in overnight trading.
The biotech recruited 30 NASH patients with advanced fibrosis for the study, providing four months of treatment with GR-MD-02. But in the end, an MRI failed to produce the necessary improvements on inflammation and fibrosis. Secondaries on liver fibrosis using stiffness as a surrogate also failed.
The lead investigator, Stephen Harrison, said there was “no apparent improvement” among patients. Then he suggested that the brief, four month treatment period was too short. The ongoing NASH-CX study, a Phase IIb trial, will treat 162 patients for a year.
There’s also a different primary endpoint — hepatic venous pressure gradient. And liver biopsies that couldn’t be done safely in the IIa will be used in this trial to look at secondary goals.
Investors, though, didn’t respond well to the “pilot” error, cutting the company’s small market cap in half.
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John Carroll, Editor and Co-Founder
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