Gemphire's gemcabene shows treatment effect in mid-stage HoFH study

Jan. 30, 2017 11:41 AM ETGemphire Therapeutics Inc. (GEMP)By: Douglas W. House, SA News Editor
  • On a preliminary basis, Gemphire Therapeutics' (NASDAQ:GEMP -0.3%) gemcabene demonstrated a significant treatment effect after eight weeks of treatment in a Phase 2b study in patients with genetic high cholesterol called homozygous familial hypercholesterolemia (HoFH).
  • Interim data from the trial, called COBALT-1, showed two patients who received 300 mg of gemcabene for four weeks followed by 600 mg for four weeks experienced reductions in LDL-C ("bad cholesterol") of 23% and 28%, respectively. Both were on a background of high-intensity statin therapy before receiving gemcabene.
  • Orphan Drug-tagged gemcabene is a once-daily orally available medication for patients unable to achieve normal levels of LDL-C or triglycerides with currently available therapies. It has a dual mechanism of action that blocks the production of hepatic triglyceride and cholesterol synthesis and enhances the clearance of VLDL (very low density lipoprotein). Specifically, it inhibits a liver protein called apolipoprotein C-III and may inhibit a liver enzyme called acetyl-CoA carboxylase. Gemphire licensed it from Pfizer in April 2011.

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