Capstone Therapeutics and its joint venture affiliate, LipimetiX Development, LLC announced the completion of and results for its investigational AEM-28 (Apo E mimetic peptide) Phase 1b/2a human clinical trial in cholesterol and lipid reduction. The top-line data from the Phase 1a (reported on September 2, 2014) and Phase 1b/2a blended protocol has been analyzed. The Medical Safety Committee, reviewing all safety-related aspects of the clinical trial, observed a generally acceptable safety profile.

As a first-in-man study, the primary endpoint was safety; yet, efficacy measurements analyzing pharmacodynamics yielded statistical significance in the pooled dataset favoring AEM-28 versus placebo in multiple lipid biomarker endpoints, which included: p < 0.05 favoring AEM-28 vs. placebo within the first 12 hours post infusion at the high dose tested of 3.54mg/kg in VLDL, equating to a maximum 76% drop in VLDL vs. baseline and a 56% net maximum reduction of VLDL vs.

placebo; p < 0.05 favoring AEM-28 vs. placebo within the first 12 hours post infusion at the 2 mg/kg dose in VLDL, equating to a maximum 70% drop in VLDL vs. baseline and a 41% net maximum reduction of VLDL vs.

placebo; p < 0.025 favoring AEM-28 vs. placebo within the first 12 hours post infusion at the high dose tested of 3.54 mg/kg in triglycerides, equating to a maximum 74% drop in triglycerides vs. baseline and a 55% average net maximum reduction of triglycerides vs.

placebo; p < 0.025 favoring AEM-28 vs. placebo within the first 12 hours post infusion at the 2 mg/kg dose in triglycerides, equating to a 71% drop in triglycerides vs. baseline and a 45% net maximum reduction of triglycerides vs.

placebo. The JV has a development plan, subject to continued favorable study results and funding availability, to pursue regulatory approval of AEM-28 and/or analogs as treatment for Homozygous Familial Hypercholesterolemia (granted Orphan Drug Designation by FDA in 2012), Acute Hypertriglyceridemic Pancreatitis and other Orphan indications in hyperlipidemia. The JV may possibly explore additional indications for its family of Apo E mimetic peptides including Acute Coronary Syndrome, Peripheral Artery Disease and mixed dyslipidemia Type 2 Diabetes.