Gemphire: FDA asks company give additional data regarding partial clinical hold
Gemphire Therapeutics announced that the U.S. Food and Drug Administration has requested that the company produce data from a sub-chronic toxicology study to provide information to support lifting the partial clinical hold on gemcabene with respect to clinical trials of longer than six months in duration. 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. Gemphire's ongoing Phase 2a proof-of-concept studies investigating gemcabene as a treatment for familial partial lipodystrophy and for pediatric 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. In addition, the Company continues to be free to conduct clinical trials that do not extend beyond six months in duration. Beginning in 2004, the FDA began issuing partial clinical holds to all sponsors of PPAR agonists or agents deemed to have PPAR-like properties from preclinical studies. The FDA takes the position that PPAR agonists are potential liver toxins, but recognizes that rodent observations are often not relevant to humans. In 2004, the FDA determined that gemcabene has PPAR agonist properties and issued a partial clinical hold. In response the FDA has requested that, as part of a complete response, Gemphire must provide additional data including a subchronic (13 week) study in PPARalpha knock-out mice and PPAR transactivation assays using monkey and canine PPAR isoforms, to further understand the human relevance of the preclinical findings. The Company has initiated plans to conduct these required studies and expects to submit the additional results to the FDA in the second quarter of 2019. "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," said Dr. Steven Gullans, CEO of Gemphire. "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. "In the meantime, we are continuing to execute on our ongoing Phase 2a POC clinical trials of gemcabene in NAFLD/NASH. Gemphire is well capitalized, with $28 million cash on hand as of June 30 2018. Based on current projections, taking into account the delay of significant cash expenditures for clinical trials and manufacturing and the amended terms of the loan agreement with SVB, we believe we have sufficient resources to fund operations into the fourth quarter of 2019."