World News: 11:00 GMT Friday 10th August 2018. [Gemphire Therapeutics Inc. via Globe Newswire via SPi World News]
LIVONIA, Mich., Aug. 10, 2018 (GLOBE NEWSWIRE) -- Gemphire Therapeutics Inc. (NASDAQ: GEMP), a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for cardiometabolic disorders, including dyslipidemia and nonalcoholic steatohepatitis (NASH), announces today that the Data and Safety Monitoring Board (DSMB) at Emory University School of Medicine overseeing the investigator-led open label Phase 2a proof-of-concept trial evaluating gemcabene in pediatric patients with non-alcoholic fatty liver disease (NAFLD) has recommended that the trial be terminated due to unanticipated problems.
This pediatric NAFLD trial was initiated in early 2018. Patients were treated with gemcabene at a dose of 300 mg once daily. The primary endpoint is a measure of the change in serum alanine transaminase (ALT), an enzyme that serves as a biomarker of liver function, from baseline to 12 weeks, and secondary endpoints include, among others, change in hepatic steatosis (liver fat) as measured by non-invasive magnetic resonance (MRI) imaging – proton density fat fraction (MRI-PDFF).
Data on the first three patients who underwent 12 weeks of treatment showed that all three experienced an increase in liver fat content, as measured by MRI-PDFF, and demonstrated increases in ALT. The increase in liver fat was deemed an unexpected problem by the trial investigator because it was an unexpected consistent pattern of worsening of the disease, rather than improvement, creating risk to the patients, which the investigator believed was likely due to the drug. Other patients currently enrolled in the trial have now been taken off gemcabene and early termination visits are being scheduled. The DSMB has recommended additional follow-up of the study subjects to gather additional safety data. The DSMB will provide Gemphire with a written report of their findings in the future once all the patient results have been collated and analyzed.
“Patient safety has always been our primary concern and we will work closely with the DSMB, the physicians at the clinical trial site, and other KOLs to analyze all the results and identify potential reasons for these unexpected events," said Dr. Steven Gullans, CEO of Gemphire. “Previously, Gemcabene had been administered to nearly 1,200 adult subjects across 25 Phase 1 and Phase 2 trials for up to 12 weeks with no drug-related serious adverse events (SAEs) reported. Gemcabene-related adverse events (AEs) in these adult subject trials were observed to be comparable to those seen with placebo treatment. We remain confident that gemcabene has the potential to be an effective therapy for a host of cardiometabolic patients and we intend to continue to develop gemcabene to address multiple indications.”
As part of Gemphire’s NAFLD/NASH program, gemcabene is also being studied in a Phase 2a study being conducted at the University of Michigan to assess the efficacy and safety of two dosing regimens of the drug in patients with familial partial lipodystrophy (FPL) who have elevated triglycerides and NASH. FPL is a rare genetic disorder and orphan disease characterized by an abnormal distribution of fatty (adipose) tissue, which can lead to a variety of metabolic abnormalities including NASH. An initial safety review of the first three patients in this study on a dose of 300 mg/day has not uncovered any safety or tolerability concerns nor was there a change in biomarkers that would indicate concerns about liver function. The principal investigator in the trial, Dr. Elif Oral, intends to closely monitor these patients while waiting for MRI-PDFF scans to be reviewed at an interim time point in the near future before dosing additional patients.
As previously reported, the Company’s cash balance at June 30, 2018 was $28 million and management believes that, based on current projections and taking into account the delay of significant cash expenditures for clinical trials and manufacturing, it will be sufficient to fund the Company’s operations into the 4 quarter of 2019.
Jeff Mathiesen, CFOGemphire Therapeutics Inc.(734) 245-1700
Globe Newswire: 11:00 GMT Friday 10th August 2018
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