Gemphire Therapeutics Inc. (NASDAQ: GEMP) shares were more than halved on Tuesday after the company provided an update on the clinical hold on gemcabene with respect to clinical trials of longer than six months in duration.
Essentially the U.S. Food and Drug Administration (FDA) has requested that Gemphire produce data from a sub-chronic toxicology study to provide information to support lifting the partial clinical hold.
The FDA specifically requested that, as part of a complete response, Gemphire must provide additional data including a subchronic (13 week) study in PPARα knock-out mice and PPAR transactivation assays using monkey and canine PPAR isoforms, to further understand the human relevance of the preclinical findings.
So far, Gemphire has initiated plans to conduct these required studies and expects to submit the additional results to the FDA in the second quarter of 2019.
The FDA also informed the company that the end of Phase 2 meeting, and consequently the initiation of Phase 3 trials investigating gemcabene in dyslipidemia indications and long-term safety exposure trials needed for registration, will not take place until the partial hold has been lifted.
It’s worth pointing out that Gemphire’s ongoing Phase 2a proof-of-concept studies investigating gemcabene as a treatment for familial partial lipodystrophy and for pediatric nonalcoholic fatty liver disease (NAFLD) are not affected by the FDA’s request for additional data, and the company continues to expect that these studies will produce top-line interim data in late 2018 and in the first half of 2019, respectively.
Dr. Steven Gullans, CEO of Gemphire, commented:
We are working closely with the FDA to release the partial clinical hold on gemcabene, with the goal of proceeding to an End of Phase 2 meeting and reaching agreement on the design of a Phase 3 clinical program. Our confidence in gemcabene’s safety profile is supported by the fact that it has been observed to be safe in nearly 1,200 human subjects in 24 Phase 1 and 2 clinical trials. In fact, gemcabene’s safety performance in previous human clinical provided the basis for the FDA to allow the agent to be evaluated in a multi-center, investigator-led ongoing NAFLD trial in pediatric patients.
Shares of Gemphire were last seen down about 52% at $3.51, with a consensus analyst price target of $27.40 and a 52-week range of $3.50 to $11.43.