Gemphire's lead drug worsened disease, investigators conclude in ending trial
- Capping off a week of bad news, Gemphire Therapeutics revealed Friday morning it will end a Phase 2a trial of its only drug candidate because early results showed it was making patients with non-alcoholic fatty liver disease worse instead of better.
- Gemcabene, an oral therapy also being studied for dyslipidemia, continues to face a partial clinical hold preventing longer trials due to safety concerns with the drug's class.
- The setbacks pummeled Gemphire's stock, with shares opening Thursday morning at $1.03 apiece, down roughly 70% from the previous day's close.
Gemphire has only one drug candidate in gemcabene. On Monday, Gemphire revealed a two-year safety study on rats and mice failed to lift a partial clinical hold blocking clinical testing longer than six months. The Food and Drug Administration requested a 13-week safety study on monkeys and canines, for which the company said it expects to submit results in the second quarter of 2019.
Investors punished the stock Tuesday morning, with shares falling 50% to an all-time low.
The only positive spin CEO Steven Gullans could provide at the time was his confidence in gemcabene's demonstrated human safety, saying "it has been observed to be safe in nearly 1,200 human subjects in 24 Phase 1 and 2 clinical trials."
Yet the Phase 2a trial termination raises doubts about that sentiment. Gemphire said initial results showed an "unexpected consistent pattern of worsening the disease, rather than improvement, creating risks to the patients, which the investigator believed was likely due to the drug."
The recommendation to scrap the trial came from the Data and Safety Monitoring Board at Emory University's School of Medicine, which was overseeing the drug's use in pediatric patients with non-alcoholic fatty liver disease. The study began in early 2018.
Gemphire currently has $28 million in cash and equivalents on hand, which it believes will allow it to operate through 2019. However, the negative results, as well as the continued partial hold, make a path forward more difficult.
Gemphire says it intends to push on with its troubled drug, highlighting an ongoing Phase 2a trial at the University of Michigan for patients with familial partial lipodystrophy who have elevated triglycerides and NASH. An initial safety review of the first three patients did not uncover any safety concerns, the biotech said.
- BioPharma Dive Gemphire stock drops to all-time low as lead drug delayed
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