NewAmsterdam Pharma Maps Obicetrapib Strategy as PREVAIL Interim Nears

Key Points

• PREVAIL is the key near-term catalyst: NewAmsterdam expects to reach its interim event target by year-end, with an independent review planned for the first quarter of 2027. The trial could continue through 2027 if interim data do not support early stopping.
• The company reported encouraging supporting data for obicetrapib, including LDL reductions of up to 43.5% alone and 63% with ezetimibe in prior studies, while advancing trials in coronary plaque, metabolic disease and potential Alzheimer’s prevention.
• NewAmsterdam is preparing for potential commercialization, including a U.S. launch and a European rollout with Menarini. The company expects an EMA decision in early fourth quarter and has $678 million in cash to fund preparations.

NewAmsterdam Pharma NASDAQ: NAMS used its annual Investor Day to outline its cardiovascular outcomes strategy for obicetrapib, provide updates on additional clinical programs and discuss commercial preparations ahead of a potential European launch.

Chief Executive Officer Michael Davidson said the company’s central focus remains the PREVAIL cardiovascular outcomes trial, while emphasizing that obicetrapib is being studied for effects beyond LDL cholesterol reduction. The company also highlighted a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, or CHMP, for obicetrapib, which Davidson described as the first regulatory recognition for a CETP inhibitor from a major regulatory body.

PREVAIL trial and interim analysis

PREVAIL is evaluating obicetrapib in approximately 10,000 patients with established atherosclerotic cardiovascular disease. Davidson said the trial was initially designed to assess a 20% relative risk reduction in a four-point major adverse cardiovascular event endpoint, or MACE, and was subsequently expanded to support evaluation of a 15% reduction.

The company amended the trial’s primary endpoint from urgent revascularization to total revascularization, alongside cardiovascular death, non-fatal myocardial infarction and stroke. Davidson said the change aligns PREVAIL with definitions used in several recent cardiovascular outcomes trials and adds events that could improve the study’s statistical power.

NewAmsterdam expects to reach its interim-analysis event target by the end of the year, with the independent data safety monitoring board expected to review the data in the first quarter of 2027. The company said a positive interim result would need to meet statistical requirements for both four-point MACE and the three-point MACE secondary endpoint. If the interim analysis does not result in an early stop, the company plans to continue the study through the end of 2027 to obtain additional events and preserve power for the final analysis.

Davidson said blinded event rates in PREVAIL have tracked closely with rates observed in the company’s BROADWAY phase 3 study during the first year. In BROADWAY, the company previously reported a 21% relative risk reduction in four-point MACE over one year. NewAmsterdam said it believes the PREVAIL event-rate trends and the BROADWAY results provide encouragement, though it acknowledged that PREVAIL remains blinded and that the final outcome is not yet known.

Additional clinical programs

Chief Scientific Officer John Kastelein presented analyses intended to support the company’s view that CETP inhibition could affect several lipid-related biomarkers, including LDL cholesterol, non-HDL cholesterol, ApoB, lipoprotein(a), small dense LDL particles and HDL cholesterol.

In an on-treatment analysis from the BROADWAY and BROOKLYN studies, Kastelein said obicetrapib reduced LDL cholesterol by 43.5% at week four and 41.1% at week 12. The company’s fixed-dose combination of obicetrapib and ezetimibe reduced LDL cholesterol by 63% at week four and 61% at week 12, according to the presentation.

NewAmsterdam is also advancing several named studies intended to broaden the evidence base for obicetrapib:

• REMBRANDT: A 323-patient trial comparing the obicetrapib-ezetimibe fixed-dose combination with placebo over 18 months. The study is evaluating non-calcified coronary plaque volume using coronary CT angiography. Enrollment is complete across 50 sites in seven countries, with completion expected in 2027.
• RUBENS: A 300-patient study in people with type 2 diabetes or metabolic syndrome. It includes obicetrapib, the fixed-dose combination and placebo groups, and is evaluating LDL cholesterol, small dense LDL particles and lipoprotein(a). NewAmsterdam expects data near the end of the year.
• SPINOZA: A planned Alzheimer’s disease prevention study in APOE4 carriers. The company said the trial will examine biomarkers including p-tau217, as well as cognition.

Kastelein highlighted an analysis from BROADWAY in which p-tau217 levels declined in APOE4 homozygotes treated with obicetrapib and increased in the comparison arm over one year. The company characterized the finding as a rationale for prospective research but did not state that obicetrapib has been proven to prevent Alzheimer’s disease.

Commercial and European preparations

Chief Commercial Officer BJ Jones said NewAmsterdam is building its commercial, medical affairs, market-access and analytics capabilities in preparation for a potential U.S. launch. He said the company sees a large opportunity among patients with hyperlipidemia who are untreated or remain above LDL cholesterol targets despite statin therapy.

Jones said NewAmsterdam’s market research indicated that two-thirds of surveyed healthcare professionals expressed a high propensity to prescribe obicetrapib or its fixed-dose combination, citing the product’s potential efficacy, oral dosing, tolerability profile and broader lipid effects. Those survey results reflect physician perceptions and do not establish future prescribing behavior.

In Europe, NewAmsterdam is working with partner Menarini, which is expected to commercialize obicetrapib following a potential approval. The company said it anticipates an EMA decision in early fourth quarter and, if approved, expected launches in the United Kingdom and Germany later in the fourth quarter.

Davidson said NewAmsterdam had $678 million in cash and believes its balance sheet can support launch preparations. He concluded that the company’s near-term milestones include RUBENS data, the PREVAIL interim review, the REMBRANDT readout in 2027 and the initiation of SPINOZA.

About NewAmsterdam Pharma (NASDAQ:NAMS)

NewAmsterdam Pharma, Inc is a clinical‐stage biopharmaceutical company focused on discovering and developing novel small‐molecule therapies for cardiometabolic diseases. The company’s research efforts are aimed at addressing key unmet needs in metabolic syndrome, obesity and type 2 diabetes by modulating pathways involved in glucose regulation, energy homeostasis and lipid metabolism.

The company’s development pipeline features multiple small‐molecule candidates at various stages of preclinical and clinical evaluation.

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