NewAmsterdam Pharma Company (NAMS) Study Update summary

Disease background and study rationale

• Heterozygous familial hypercholesterolemia (HeFH) affects 1 in 250 people globally, causing high LDL-C from birth and increased risk of premature atherosclerotic cardiovascular disease if untreated.

Study design and patient population

• BROOKLYN was a global, pivotal, 52-week, double-blind, placebo-controlled Phase III trial evaluating 10 mg obicetrapib in adults with HeFH inadequately controlled on maximally tolerated lipid-lowering therapy.

• 354 patients were randomized 2:1 to obicetrapib or placebo; mean baseline LDL-C was 123 mg/dL, 53% were female, mean age 57, and BMI averaged 29.

• Most patients were on statins (up to 90%), 50–52.5% on ezetimibe, and 14% on PCSK9 inhibitors; 72% had genotyping-confirmed or definite HeFH.

• BROOKLYN is the first of four pivotal Phase III studies for obicetrapib, with additional trials (BROADWAY, TANDEM, PREVAIL) ongoing.

• The primary endpoint was percent change in LDL-C at day 84; secondary endpoints included HDL-C, non-HDL-C, ApoB, Lp(a), and safety.

Efficacy results

• Obicetrapib achieved a placebo-corrected LDL-C reduction of 36.3% at day 84 and 41.5% at day 365, with rapid onset and sustained effect over one year (p<0.0001).

• 77% of patients on obicetrapib achieved LDL-C <100 mg/dL, 51% <70 mg/dL, and 24% <50 mg/dL, all statistically significant versus placebo.

• 34% of treated patients had >50% LDL-C reduction at day 84.

• Statistically significant reductions were also observed in HDL-C, non-HDL-C, Lp(a), and ApoB, consistent with prior trials.

• The LDL-C lowering effect is expected to translate into a 22–25% reduction in major adverse cardiovascular events (MACE) based on established regression models.