OrsoBio has announced the publication in Nature Medicine of Phase I clinical trial data for TLC-2716, an oral liver-targeted inverse agonist of Liver X Receptor (LXR) developed for the treatment of obesity and related metabolic disorders.
The publication highlights TLC-2716’s favourable safety profile and robust lipid-lowering capabilities observed in healthy volunteers.
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The results were consistent with human genetic data and observations from studies involving dysmetabolic rodent models, non-human primates, and human liver organoids, demonstrating effective translation of liver-restricted LXR inhibition from preclinical research to human clinical trials.
Currently, TLC-2716 is under evaluation in a Phase IIa trial for patients with metabolic dysfunction-associated steatotic liver disease (MASLD) and severe hypertriglyceridemia (SHTG), with final data expected by mid-2026. Together, these conditions affect more than 80 million Americans.
Liver X receptors play a crucial role in regulating triglyceride and cholesterol homeostasis. TLC-2716 is designed to modulate plasma triglycerides and cholesterol through various mechanisms such as suppression of hepatic de novo lipogenesis and enhanced clearance of triglyceride-rich lipoproteins.
Preclinical studies reported improvements in lipid and metabolic parameters across disease-relevant models. Specifically, the treatment reduced plasma cholesterol and enhanced glucose tolerance in dysmetabolic rodents, while maintaining peripheral LXR activity.
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By GlobalDataThe placebo-controlled, double-blind, randomised Phase I trial involved 100 healthy participants. It evaluated the pharmacodynamics, pharmacokinetics, tolerability, and safety of TLC-2716. Over 14 days, treatment resulted in dose-dependent reductions in multiple atherogenic lipid parameters. No serious adverse events were reported.
OrsoBio CEO Mani Subramanian said: “The positive results from our first-in-human study validate LXR as a therapeutic target and highlight the unique liver and gut-restricted pharmacology of TLC-2716 for the treatment of diseases driven by excessive lipid production.
“By improving key lipid parameters and reducing hepatic fat, TLC-2716 has the potential to address severe hypertriglyceridemia, elevated remnant cholesterol, and MASH—major contributors to cardiovascular morbidity and mortality.”
