NewAmsterdam reveals positive phase 3 results for CETP inhibitor obicetrapib in HeFH

NewAmsterdam Pharma has announced encouraging findings from a pivotal study of its oral CETP inhibitor, obicetrapib, aimed at treating heterozygous familial hypercholesterolaemia (HeFH). HeFH is a genetic condition that results in extremely elevated cholesterol levels, which, if not properly managed, can lead to serious cardiovascular events like heart disease, heart attacks, or strokes. Affecting roughly one in 250 people worldwide, the condition is often resistant to existing lipid-lowering therapies.

The phase 3 BROOKLYN trial focused on adult patients whose low-density lipoprotein cholesterol (LDL-C) levels remained inadequately controlled despite being on the highest tolerated doses of current lipid-lowering treatments. The trial successfully met its primary endpoint, showcasing that obicetrapib, when added to maximally tolerated lipid-lowering therapies, achieved an average reduction in LDL-C of 36.3% at day 84 and 41.5% at the one-year mark, compared to a placebo.

In addition to lowering LDL-C, obicetrapib also resulted in significant reductions in other key biomarkers such as high-density lipoprotein cholesterol (HDL-C), non-HDL-C, lipoprotein(a), and apolipoprotein B. These results align with data from NewAmsterdam's earlier clinical trials, further supporting the efficacy of the drug.

John Kastelein, NewAmsterdam’s chief scientific officer, commented on the results, expressing that many HeFH patients have already exhausted existing treatment options. He expressed enthusiasm over the BROOKLYN trial's outcomes, emphasizing the potential for obicetrapib to offer a novel, once-daily, low-dose oral treatment that could revolutionize the current therapeutic landscape for these patients.

Echoing this excitement, NewAmsterdam’s chief executive officer, Michael Davidson, highlighted the promising data from the BROOKLYN trial. He believes that the results underline obicetrapib's potential to significantly lower LDL-C levels in a particularly challenging patient demographic over a period of one year. Davidson also mentioned the company's plans to build on these findings with additional data expected later in the year from the BROADWAY trial. This trial is evaluating obicetrapib in adults with established atherosclerotic cardiovascular disease, HeFH, or both.

Furthermore, results are anticipated in the first quarter of the next year from another phase 3 trial investigating obicetrapib as part of a fixed-dose combination tablet with ezetimibe, a non-statin oral LDL-lowering therapy. The enrolment for the TANDEM trial, which is evaluating this combination, was recently completed. Davidson noted that this milestone is a significant step forward in the company’s mission to advance obicetrapib through its late-stage clinical development.

Overall, the positive results from the BROOKLYN trial suggest that obicetrapib could become a critical new option for patients with HeFH, providing hope for better management of cholesterol levels and potentially reducing the risk of serious cardiovascular events. The forthcoming results from additional trials will further elucidate the drug's efficacy and its potential role in the treatment of high cholesterol linked to genetic conditions and other cardiovascular diseases.