NewAmsterdam Pharma has shared positive results from a late-stage study of its oral CETP inhibitor in heterozygous familial hypercholesterolaemia (HeFH), an inherited genetic disorder that causes dangerously high cholesterol levels.
The phase 3 BROOKLYN trial has been evaluating the candidate, obicetrapib, in adults whose low-density lipoprotein cholesterol (LDL-C) is not adequately controlled despite being on maximally tolerated lipid-lowering therapy.
Affecting approximately one in 250 people globally, HeFH can lead to heart disease, heart attack or stroke if left untreated. However, many patients are unable to achieve guideline-recommended LDL-C levels with currently available treatment options.
BROOKLYN met its primary endpoint, with obicetrapib on top of maximally tolerated lipid-modifying therapies demonstrating mean reductions in LDL-cholesterol of 36.3% and 41.5% compared to placebo at day 84 and one year, respectively.
Statistically significant reductions in biomarkers, including high-density lipoprotein cholesterol (HDL-C), non-HDL-C, lipoprotein(a) and apolipoprotein B, were also observed and consistent with data reported from the company’s prior clinical trials.
NewAmsterdam’s chief scientific officer, John Kastelein, said: “Many [HeFH patients] have exhausted available treatment options and we are delighted by the top-line data from BROOKLYN… Our goal is to provide physicians and patients with a novel, once-daily, low-dose oral option that can potentially transform the treatment landscape.”
Sharing a similar sentiment, NewAmsterdam’s chief executive officer, Michael Davidson, said: “We are very excited about the results from our BROOKLYN trial and believe they support obicetrapib’s potential to significantly reduce LDL-C in a challenging patient population over a duration of one year.”
Davidson added that the company is expecting to build on the data later this year with top-line results from the late-stage BROADWAY trial, which is evaluating obicetrapib in certain adults with established atherosclerotic cardiovascular disease, HeFH or both.
Results are also expected in the first quarter of next year from a phase 3 trial assessing the candidate as part of a fixed-dose combination tablet with ezetimibe, a non-statin oral LDL-lowering therapy. Enrolment in the TANDEM trial was completed last month, which Davidson said marked an “important step in [the company’s] mission of advancing obicetrapib through late-stage clinical development”.