Dr. Christie Ballantyne contributes to global study demonstrating the drug’s potential.

A newly published international study, “Safety and Efficacy of Obicetrapib in Patients at High Cardiovascular Risk”, co-authored by Christie Ballantyne, MD, professor of medicine and chief of cardiovascular research at Baylor College of Medicine and cardiologist at The Texas Heart Institute at Baylor College of Medicine, highlights the potential of obicetrapib, a selective cholesteryl ester transfer protein (CETP) inhibitor, as a powerful adjunct to current lipid-lowering therapy in high-risk cardiovascular patients.

In the BROADWAY study, clinical trials involving 2,530 patients across four continents, the efficacy and safety of 10 mg daily obicetrapib in individuals with established atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia were evaluated. All participants were already receiving maximally tolerated lipid-lowering therapy, including statins, ezetimibe, and in some cases, PCSK9 inhibitors.

At 84 days, patients receiving obicetrapib experienced a least-squares mean LDL cholesterol reduction of 29.9%, compared to a 2.7% increase in the placebo group. The between-group difference was –32.6 percentage points (P<0.001). In addition to lowering LDL-C, obicetrapib also reduced levels of non-HDL cholesterol, apolipoprotein B, and lipoprotein(a), while significantly increasing HDL cholesterol and apolipoprotein A1.

Importantly, the safety profile of obicetrapib was comparable to placebo, with no significant increase in adverse events, liver enzyme abnormalities, muscle symptoms, or glycemic deterioration.

“Obicetrapib offers a promising new tool for helping patients who remain above target despite intensive therapy,” said Dr. Ballantyne. “It’s an oral, once-daily agent that showed consistent LDL-lowering across diverse patient populations and maintained a favorable safety signal throughout the study period.”

CETP inhibitors have had a controversial history, with prior agents such as torcetrapib and evacetrapib failing due to off-target effects or lack of outcome benefits. However, obicetrapib’s high selectivity and hydrophilic structure appear to mitigate many of those historical concerns. A large cardiovascular outcomes trial (NCT05202509) is currently underway to determine whether the lipid changes observed with obicetrapib will translate into reduced cardiovascular events.

The study, funded by NewAmsterdam Pharma, was published in The New England Journal of Medicine on May 7, 2025.

As part of its mission to advance the prevention and treatment of cardiovascular disease, The Texas Heart Institute at Baylor College of Medicine continues to support translational research efforts that bring cardiovascular health to life.