Gemphire Plunges After Investigational Drug Worsens Liver Fat Levels in Pediatric NAFLD Trial Patients

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Shares of Gemphire Therapeutics have crashed 73 percent in premarket trading after the company revealed its mid-stage non-alcoholic fatty liver disease (NAFLD) treatment for pediatric patients actually worsened the disease in some patients.

The Phase IIa trial was halted early due to what has been termed “unanticipated problems.” Essentially, the company said the first three patients who underwent the 12-week course of treatment with gemcabene in February of this year all experienced an increase in liver fat content. Additionally, those patients also saw increases in serum alanine transaminase (ALT), which is an enzyme that serves as a biomarker of liver functions. The increase in liver fat was considered a sign of disease worsening. Following a review by the Data and Safety Monitoring Board at Emory University School of Medicine that was overseeing the study, Livonia, Mich.-based Gemphire halted the trial after it was determined that the medication was the likely contributor to the worsening of symptoms.

Other patients currently enrolled in the trial have now been taken off the lipid-altering gemcabene and early termination visits are being scheduled, Gemphire said this morning. Additional follow-ups with patients will be conducted to gather further safety data, the company added.

Seven Gullans, chief executive officer of Gemphire, said patient safety is the company’s primary concern and it will work closely with investigators to analyze the early results of the trial and identify the reasons the drug went wrong.

“Previously, Gemcabene had been administered to nearly 1,200 adult subjects across 25 Phase I and Phase II 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,” Gullans said in a statement.

The Phase IIa trial is not the only NAFLD or nonalcoholic steatohepatitis (NASH, a type of NAFLD) trial where Gemphire is assessing gemcabene. The company is conducting a Phase IIa study in patients with familial partial lipodystrophy (FPL) who have elevated triglycerides and NASH. The company noted that an initial review of the first three patients in that trial has not revealed any issues.

Gemcabene has hit the mark in previous studies, such as the Phase IIb Cobalt-1 trial assessing gemcabene as a treatment for homozygous familial hypercholesterolemia (HoFH) patients. The drug has also disappointed before. Last year, gemcabene failed to impress analysts in the Phase IIb Royal-1 trial of gemcabene. The trial met statistically significant endpoints in targeting LDL cholesterol levels, but the company acknowledged that the lower levels were less than observed in prior studies.

Gemphire said it has a cash balance of about $28 million, which should be sufficient to maintain current company operations through the fourth quarter of 2019. Shares of Gemphire are trading at 84 cents as of 9:37 a.m. The stock closed at $3.27 per share on Thursday.

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