NewAmsterdam Pharma Reports Positive Phase 3 BROOKLYN Trial Results for Obicetrapib in Heterozygous Familial Hypercholesterolemia

NewAmsterdam Pharma Company N.V., a clinical biopharmaceutical entity focused on developing non-statin oral medications for cardiovascular disease (CVD) patients with elevated low-density lipoprotein cholesterol (LDL-C), announced positive topline results from its Phase 3 BROOKLYN clinical trial. The study evaluated the drug obicetrapib in patients with heterozygous familial hypercholesterolemia (HeFH) whose LDL-C levels were not adequately controlled despite being on maximally tolerated lipid-lowering therapy.

The BROOKLYN trial achieved its primary endpoint, showing a statistically significant reduction in LDL-C levels. The mean LDL-C reduction was 36.3% at day 84 and 41.5% at day 365 compared to placebo. Other biomarkers, including high-density lipoprotein cholesterol (HDL-C), non-HDL-C, lipoprotein(a) (Lp(a)), and apolipoprotein B (ApoB) also showed significant reductions, aligning with previous clinical trial data from the company.

Dr. John Kastelein, Chief Scientific Officer of NewAmsterdam, expressed optimism about the data, noting that 51% of patients achieved LDL-C levels below 70 mg/dL. The goal is to provide a novel, once-daily, low-dose oral treatment option that can transform the current therapeutic landscape for these patients.

The safety profile of obicetrapib was promising, with observed tolerability comparable to placebo and no increase in blood pressure. The rate of treatment discontinuation was lower in the obicetrapib group (7.6%) compared to the placebo group (14.4%). The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs) were similar between obicetrapib and placebo groups.

Michael Davidson, Chief Executive Officer of NewAmsterdam, highlighted the significance of the results for HeFH patients and the broader CVD community. He noted that despite existing therapies, many patients still do not meet their LDL-C targets, making the need for additional treatment options crucial. The BROOKLYN trial results reinforce obicetrapib’s potential to reduce LDL-C significantly over one year for a challenging patient population. Davidson also noted that there was no difference in blood pressure, liver enzymes, hs-CRP, or renal function between the obicetrapib and placebo groups.

Stephen Nicholls, Director of the Monash Victorian Heart Institute, echoed the enthusiasm, stating that the results suggest obicetrapib could become a new oral option for difficult-to-treat patients if approved. He emphasized that HeFH patients often require multiple therapies to achieve safe LDL-C levels, and obicetrapib could provide another efficacious oral option.

Katherine Wilemon, Founder and CEO of the Family Heart Foundation, pointed out the impact of HeFH on individuals, noting that many cannot attain guideline-recommended LDL-C levels despite available treatments. The foundation is encouraged by the potential of obicetrapib to offer another effective oral therapy.

NewAmsterdam plans to present the full results of the BROOKLYN trial at an upcoming medical conference and publish the data in a major medical journal. The 52-week trial was a global, randomized, double-blind, placebo-controlled multicenter study involving 354 patients from North America, Europe, and Africa. Participants were randomized to receive either 10 mg obicetrapib or placebo once daily, with or without food.

The primary endpoint was the percentage change in LDL-C from baseline at day 84. Secondary endpoints included changes in HDL-C, non-HDL-C, ApoB, and Lp(a). The study also evaluated the safety and tolerability profile of obicetrapib.

NewAmsterdam’s global Phase 3 clinical program includes four studies involving over 12,250 patients. The company is also conducting additional Phase 3 trials, BROADWAY and TANDEM, to further evaluate obicetrapib’s efficacy and safety. The BROADWAY trial began in January 2022, and the TANDEM trial started in March 2024. The company completed enrollment for the BROADWAY trial in July 2023 and expects to complete the TANDEM trial enrollment by July 2024.