ROYAL-1 is a Phase 2b Study to Determine Safety and Tolerability of Gemcabene in Hypercholesterolemia Not Adequately Controlled on High-Intensity or Moderate-Intensity Stable Statin TherapyTopline Data Readout Anticipated in Second Half 2017 LIVONIA, Mich., Nov. 28, 2016 (GLOBE NEWSWIRE) -- Gemphire Therapeutics Inc. (Nasdaq:GEMP), a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for the treatment of dyslipidemia, a serious medical condition that increases the risk of life threatening cardiovascular disease, and NAFLD/NASH (nonalcoholic fatty liver disease), today announced enrollment of its first patients in ROYAL-1, a Phase 2b trial designed to investigate gemcabene in the treatment of patients with hypercholesterolemia not adequately controlled on high-intensity or moderate-intensity stable statin therapy. It is designed to enroll a broad patient population, including patients with heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD), who have baseline LDL-C ("bad cholesterol") values = 100 mg/dL. "The patient groups we plan to enroll in the ROYAL-1 trial, including patients with HeFH and patients with ASCVD, particularly those with diabetes, represent some of the more prevalent hypercholesterolemia conditions," said Mina Sooch, President and Chief Executive Officer of Gemphire. "If we are successful in obtaining approval for gemcabene in these populations, we expect to be able to reach a large market of over 10 million patients just in the U.S. that are currently unable to attain their LDL-C goals despite current statin and ezetimibe therapies. Gemcabene, if approved, may offer a competitive and complementary profile as compared with anti-PCSK9 monoclonal antibodies (MAbs), a recently approved class of LDL-C lowering drugs." The open-label Phase 2b trial, " A 12-Week, Phase 2 Study of Gemcabene in Hypercholesterolemia Patients on Stable Moderate and High-Intensity Statins (ROYAL-1)" will enroll up to 104 adult patients at clinical sites in the United States. Patients meeting eligibility requirements are being randomized in a 1:1 ratio to 600mg of gemcabene or placebo. The primary endpoint is the percent change from baseline of LDL-C at 12 weeks. Secondary endpoints include the change from baseline in non-HDL-C, total cholesterol, triglycerides, ApoB, and hsCRP at the 12-week time point. "We have seen significant interest in the study from principal investigators in the first week of enrollment which reinforces this large unmet patient need despite current available therapies," shared Dr. Lee Golden, Chief Medical Officer. "Gemcabene is an oral once-daily drug candidate with broad lipid-lowering and anti-inflammation attributes in patients on background stable statin therapy that has been previously studied in a Phase 2 trial and summarized in a recently published paper in the Journal of Clinical Lipidology ." Gemphire currently anticipates the 12-week study to complete enrollment and all patient follow-up visits in the second half of 2017. Additional information on the trial design, including eligibility criteria and site locations, can be found at www.clinicaltrials.gov, using the NCT Identifier NCT02634151. About GemcabeneGemphire's product candidate, gemcabene (CI-1027), is a novel, once-daily, oral therapy that may be suitable for patients who are unable to achieve normal levels of LDL-C or triglycerides with currently approved therapies, primarily statins. Gemcabene's mechanism of action is designed to enhance the clearance of very low-density lipoproteins (VLDLs) in the plasma and inhibit the production of cholesterol and triglycerides in the liver. The combined effect for these mechanisms has been observed to result in a reduction of plasma VLDL-C, LDL-C, and triglycerides, as well as markedly lowering C-reactive protein. Gemcabene is liver-directed and reduces apoC-III mRNA and plasma levels and may also inhibit acetyl-CoA carboxylase (ACC) which has applications in NASH/NAFLD. Gemcabene has been tested as monotherapy and in combination with statins and other drugs in 895 subjects across 18 Phase 1 and Phase 2 clinical trials and has demonstrated promising evidence of efficacy, safety and tolerability. About Heterozygous Familial Hypercholesterolemia (HeFH)The HeFH patient population is generally comprised of individuals who have one defective gene that leads to elevated LDL-C levels between 190 mg/dL and 500 mg/dL. These patients are prone to premature cardiovascular events. The incidence of patients with HeFH is estimated to be one in 200 and one in 500, and accordingly, Gemphire estimates there are approximately 0.5 to 1.5 million patients with HeFH in the United States and 15 to 30 million in the rest of the world. Current approved treatments for HeFH include statins, ezetimibe, bile acid sequestrants and the recently approved injectable PCSK9 inhibitors. Despite the availability of various treatments, many patients are still unable to achieve recommended LDL-C levels. In addition, patients, physicians and payors may prefer more convenient, cost-effective, oral drugs.