US-based Gemphire Therapeutics has reported positive top-line results from a Phase IIb clinical trial (COBALT-1) of gemcabene to treat patients with homozygous familial hypercholesterolemia (HoFH).
Gemcabene is an oral, once-daily product candidate being developed for patients who cannot achieve normal levels of LDL-C or triglycerides with existing therapies such as statins.
The product increases clearance of very low-density lipoproteins (VLDLs) in the plasma and inhibits cholesterol and triglyceride production in the liver.
Results showed that the trial has met its primary endpoint of decreasing low-density lipoprotein cholesterol (LDL-C) level.
Conducted in the US, Canada and Israel, the Phase IIb trial investigated the efficacy, safety and tolerability of various increasing doses of gemcabene from 300mg to 600mg, followed by 900mg every four weeks over 12-week duration.
The trial included eight subjects who were on stable maximally tolerated lipid-lowering therapies.
Gemphire Therapeutics interim CEO Steven Gullans said: "Gemcabene’s mean LDL-C reductions compare favourably with the LDL-C reductions, generally 15%-25%, observed for approved therapies to treat HoFH patients and are consistent with the LDL-C reductions seen in our prior hypercholesterolemia trials.
"This data continues to support gemcabene’s complementary mechanism of action, which is additive to existing lipid lowering therapies."
The primary endpoint was mean percent change in LDL-C and secondary endpoints were mean percent change in hsCRP, apoB, non-HDL-C, TG, VLDL-C and total cholesterol.
While 300mg gemcabene reduced LDL-C by a mean 25% at four weeks, 600mg dose lowered it by 30% at eight weeks and 900mg by 29% at 12 weeks in patients on low-fat and low-cholesterol diet.
The intensity of adverse events (AEs) during the trial was found to be mild to moderate without any serious AEs or AE withdrawals.