NewAmsterdam Pharma Company (NAMS) Study Update summary

Disease background and study rationale

• Heterozygous familial hypercholesterolemia (HeFH) affects 1 in 250 people globally, leading to 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, randomized, 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 (obicetrapib:placebo), with 53% female, mean age 57 years, and mean baseline LDL-C of 123 mg/dL.

• Most patients were on statins (79.4–90%), 50–52.5% on ezetimibe, and 14% on PCSK9 inhibitors, representing a highly refractory population.

• 72% had genotyping-confirmed HeFH or definite diagnosis by established criteria.

• 95.8% of patients in the obicetrapib arm completed the study, compared to 93.2% in placebo.

Efficacy results

• Obicetrapib achieved a placebo-corrected LDL-C reduction of 36.3% at day 84 and 41.5% at day 365 (p<0.0001 for both).

• LDL-C lowering was rapid (43.5% at day 30) and sustained over 52 weeks.

• 77% of treated patients achieved LDL-C <100 mg/dL, 51% <70 mg/dL, and 24% <50 mg/dL.

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

• Efficacy was consistent across background therapies and aligned with prior studies.