Gemphire Therapeutics Enrolls First Patient in the COBALT-1 Trial Investigating Gemcabene in Homozygous Familial Hypercholesterolemia
Phase 2b Study to Determine Safety and Tolerability of Gemcabene in HoFH
Topline Data Readout Currently Anticipated in First Half -2017
“We are pleased to begin patient enrollment in the COBALT-1 trial for HoFH,” said
The open-label Phase 2b trial, “Efficacy and Safety of Gemcabene in Patients with Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)” will enroll up to eight adult patients at clinical sites in
Gemphire currently anticipates the 12-week study to complete enrollment and all patient follow-up visits in the first half of 2017, with top-line data readout expected in
Additional information on the trial design, including eligibility criteria and site locations, can be found at www.clinicaltrials.gov, using the NCT Identifier NCT02722408.
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 Homozygous Familial Hypercholesterolemia (HoFH)
HoFH is a rare genetic disease that is usually caused by a mutation in both alleles of the LDL receptor gene. The LDL receptor is responsible for removing LDL from the blood. As a result, HoFH patients exhibit severely high LDL‑C levels, are at very high risk of experiencing premature cardiovascular events, such as a heart attack or stroke, and develop premature and progressive atherosclerosis. LDL‑C levels in HoFH patients are typically in the range of 500 mg/dL to 1,000 mg/dL, compared to a normal target range of 70 mg/dL to 100 mg/dL. Unless treated, most patients with HoFH do not survive adulthood beyond 30 years of age. There are approximately 300 to 2,000 HoFH patients in
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). 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," 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 (734)-245-1700