Less than a year after Gemphire shares $GEMP were doused by poor LDL numbers for its lead drug gemcabene, investors rallied to the cause with a fresh dose of positive Phase IIb results for the same therapy among patients with severe hypertriglyceridemia — or at least the way they defined the disease.
On Thursday evening the stock more than doubled in value after the biotech reported that their treatment — at the high dose — was able to significantly reduce the amount of triglycerides in the blood relative to a placebo. The key readout for the company:
Starting from a median TG baseline of 637 mg/dL, patients receiving 600 mg of gemcabene experienced a median 47% decrease in TGs compared to a median 27% decrease for placebo patients, who started at a median of 658 mg/dL (P=0.0063).
But did they set the bar high enough?
SHTG is associated with the threat of acute pancreatitis and cardio issues, so the need is there. But the literature suggests that the severe form of the disease is defined by triglyceride levels above 1,000 mg/dL. Six years ago the Endocrine Society set out diagnosis standards that defined 200 to 999 mg per dL (2.3 to 11.3 mmol per L) as a moderate case. The next thousand mg/dL was severe and above 2000 is very severe.
Akcea, which is majority owned by Ionis, recently passed muster with the majority of experts on an FDA committee with volanesorsen, but only after demonstrating efficacy in a patient population suffering from hereditary familial chylomicronemia syndrome, where the average level was 2,209 mg/dL.
Akcea/Ionis’ drug was also linked with several cases of thrombocytopenia, which raised some serious safety issues for the developers.
Amarin, meanwhile, is finishing up a huge Phase III outcomes study called REDUCE-IT, where they’re testing the ability of Vascepa, purified ethyl ester of eicosapentaenoic acid, to reduce serious cardio issues. Their top-line data are due out in the next few months. That drug was approved after demonstrating a 33% reduction in triglycerides starting with a baseline number of 680 — the same field Gemphire is playing in.
Gemphire says it is now shooting for a Phase III for their drug — a Pfizer hand-me-down — which may now get more scrutiny. They are also making plans for a proof-of-concept study in NAFLD/NASH — an ever popular arena for small biotechs.
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