Gemphire Therapeutics Enrolls First Patients in the ROYAL-1 Trial Investigating Gemcabene in Hypercholesterolemia
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 Therapy
Topline Data Readout Anticipated in Second Half 2017
“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
The randomized, placebo-controlled, double-blind 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.
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 Gemcabene
Gemphire’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
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.
About Atherosclerotic Cardiovascular Disease (ASCVD)
ASCVD represents patients who have experienced or are at risk of a cardiovascular event and are unable to meet their LDL-C lowering goal of less than 70 mg/dL with maximally tolerated statin therapy. This population also includes many patients who, in addition to not being able to meet their LDL-C lowering goal, have elevated triglyceride levels greater than 150 mg/dL and less than 500 mg/dL, categorized as mixed dyslipidemia. Gemphire estimate that approximately 10 million patients in
Currently approved treatments for both primary hypercholesterolemia and ASCVD include statins, ezetimibe, bile acid sequestrants, niacin, fibrates and recently approved PCSK9 inhibitors. While these drugs have demonstrated efficacy in lipid-lowering in this population, they do not sufficiently address the patients with mixed dyslipidemia who need to lower both LDL-C and triglycerides.
About Gemphire
Gemphire is 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). Gemphire has initiated 3 clinical trials for HoFH, HeFH/ASCVD, and SHTG under NCT02722408, NCT02634151, and NCT02944383, respectively. Please visit www.gemphire.com for more information.
Forward Looking Statements
Any statements in this press release about Gemphire’s future expectations, plans and prospects, including statements about Gemphire’s financial prospects, future operations and sufficiency of funds for future operations, clinical development of Gemphire’s product candidate, expectations regarding future clinical trials and future expectations and plans and prospects for Gemphire and other statements containing the words "believes," "anticipates," "estimates," "expects," "intends," "plans," "predicts," "projects," "targets," "may," "potential," "will," "would," "could," "should," "continue," “scheduled” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the success and timing of Gemphire’s regulatory submissions and pre-clinical and clinical trials; regulatory developments; changes to Gemphire’s clinical trial designs and regulatory pathways; changes in Gemphire’s capital resource requirements; Gemphire’s ability to obtain additional financing; Gemphire’s ability to successfully market and distribute its product candidate, if approved; Gemphire’s ability to obtain and maintain its intellectual property protection; and other factors discussed in the "Risk Factors" section of Gemphire’s Quarterly Report on Form 10-Q for the quarterly period ended
Contact:Andrew McDonald , Ph.D.LifeSci Advisors, LLC (646) 597-6987Jeff Mathiesen , CFOGemphire Therapeutics Inc. (734)-245-1700