Positive trial results
Yesterday, Gemphire Therapeutics (GEMP) announced that its Phase 2b INDIGO-1 trial evaluating investigational first-in-class oral therapy, gemcabene, achieved its primary endpoint of significantly reducing triglyceride levels in Severe Hypertriglyceridemia (or SHTG) patients with baseline serum triglyceride levels greater than 500 mg/dl. The drug also demonstrated a solid safety and tolerability profile and could be safely combined with other drugs. Gemcabene currently holds a method patent in SHTG indication valid until 2032. As a result of this announcement, the company’s share price almost doubled in the morning trade today.
Gemcabene also demonstrated improvement in the various lipid and inflammatory markers of patients in the INDIGO-1 trial, which supports further study of this drug for nonalcoholic fatty liver disease (or NALFD) and nonalcoholic steatohepatitis (or NASH), a type of NALFD.
Gemphire Therapeutics has also initiated two Phase 2 open-label proof-of-concept (or POC) studies to study gemcabene in pediatric NALFD/NASH and Familial Partial Lipodystrophy (or FLPL) indications. The company expects to release data from these trials in H2 2018 or H1 2019. Gilead Sciences (GILD) and Exelixis (EXEL) are also focused on developing therapies targeting the large opportunity in the NASH segment.
According to Gemphire Therapeutics, approximately 7 million children in the United States suffer from NALFD while 2 million suffer from NASH. Further, the company estimates that 20% of children in the age group of 12–19 years are obese while approximately 38% of obese children suffer from NASH. Despite the high prevalence of NALFD and NASH in the pediatric patient population, there are no FDA-approved therapies in the market. In this context, Gemphire Therapeutics has managed to rapidly recruit patients in its trials from a few medical sites. Plus, the company may also face fewer problems due to trial design since the enrolled patient population is more uniform.