Gemphire Therapeutics, a US based clinical-stage biopharmaceutical company focused on therapies for cardiometabolic disorders, including dyslipidemia and Nonalcoholic Steatohepatitis (NASH), has kicked off Phase 2a proof-of-concept (POC) clinical trial to see if gemcabene can be used as a treatment for pediatric nonalcoholic fatty liver disease (NAFLD).
The Detroit-headquartered Gemphire pointed out in its press release that NAFLD describes a continuum of disease from its mildest form of isolated fat infiltration of the liver, or steatosis, which can progress in severity to NASH, and is the leading cause of chronic liver disease in children and adolescents in the United States. The trial is one of two programs to develop gemcabene in NAFLD/NASH, and will go in parallel with the recently started trial for gemcabene in familial partial lipodystrophy (FPL). Gemphire said it expects top line results from NAFLD in 2019.
Gemphrie’s CEO Steven Gullans is confident in gemcabene will have a distinct competitive advantage, Gullans, Ph.D., Interim President and CEO of Gemphire, further said. “Given the paucity of clinical trials in pediatric NAFLD, and the large number of adolescents already identified with this condition, we expect to recruit pediatric patients faster than is currently possible in similar adult trials. We anticipate relatively little competition in this segment of the market over the next several years.”
In addition to the pediatric NAFLD study announced today, Gemphire is also evaluating gemcabene for treatment of adult Familial Partial Lipodystrophy (FPL), which is a NASH-related indication.
FPL is a rare genetic disorder and orphan disease characterized by an abnormal distribution of body fat, which can lead to a variety of metabolic abnormalities including NASH. FPL Patients currently don’t have FDA approved treatments. Gemphire noted it has started a Phase 2a study to study in this year two dosing regimens of gemcabene in up to eight FPL patients with elevated triglycerides and NAFLD.
Gemcabene’s safety profile demonstrated in more than 1000
Miriam Vos, Assistant Professor of Pediatrics at the Emory University School of Medicine and Primary Investigator of this study, noted: “Pediatric NAFLD, which often progresses to NASH, is a growing epidemic and can lead to significant morbidities in these children. When exploring new therapies in children, safety is a major concern and gemcabene’s safety profile as demonstrated in nearly 1100 adults provides a strong rationale to initiate this trial in pediatric patients with NAFLD. Additionally, the demonstrated effects of lowering triglycerides, inflammation, hepatic NAFLD Activity Score (NAS), intrahepatic fat and de novo lipogenesis in preclinical models, suggest that gemcabene may be beneficial in this patient population.“