LG Chem Life Sciences announced the initiation of two phase 2 clinical trials of LB54640, the company’s novel oral small molecule melanocortin-4 receptor (MC4R) agonist, which is in development for the potential treatment of certain rare forms of obesity. In the upcoming phase 2 clinical trials, which will be conducted in multiple sites located in the US and Europe, LG Chem plans to assess the efficacy and safety of LB54640 in patients with obesity due to defects in the MC4R pathway.

The Phase 2 ROUTE trial is a single-arm, open-label study that will evaluate the safety, tolerability, pharmacokinetics, and weight loss efficacy of LB54640. The trial will enroll approximately five patients with pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency, each of which is a rare genetic disorder associated with severe, early-onset obesity and insatiable hunger, hyperphagia. Participants will receive LB54640 by oral administration once daily for up to 52 weeks. The primary endpoint of the study is the change from baseline in body mass index after 16 weeks of treatment.

The Phase 2 SIGNAL trial is a randomized, placebo-controlled, double-blind study that will evaluate the safety, tolerability, pharmacokinetics, and weight loss efficacy of three different doses of LB54640. The trial will enroll approximately 28 patients with acquired hypothalamic obesity. Participants will receive one of three doses of LB54640 by oral administration once daily for up to 52 weeks. The primary endpoint of the study is the change from baseline in body mass index after 14 weeks of treatment.

LB54640 previously received orphan drug designation from the US FDA for LEPR deficiency and POMC deficiency.