Gemcabene trial for pediatric patients with non-alcoholic fatty liver disease (NAFLD) is terminated due to unanticipated problems.- Gemphire Therapeutics.

Gemphire Therapeutics Inc., announces that the Data and Safety Monitoring Board (DSMB) at Emory University School of Medicine overseeing the investigator-led open label Phase IIa 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.