NewAmsterdam Pharma Company (NAMS) Jefferies London Healthcare Conference 2024 summary

Product overview and clinical need

• Obicetrapib, a CETP inhibitor, has advanced to late-stage development and demonstrates robust LDL, Lp(a), and small particle lowering, with strong tolerability and potential diabetes risk reduction.

• Fixed-dose combination with ezetimibe achieves 49% LDL reduction, simplifying lipid management and offering a PCP-friendly oral alternative to costly injectables.

• The drug addresses a significant unmet need, as 90% of 30 million US patients on lipid-lowering therapy remain above LDL goals.

Phase 3 clinical trial results and consistency

• The TANDEM phase 3 trial met all primary endpoints, showing a 49% LDL reduction with the fixed-dose combo and consistent efficacy across all trial phases.

• Safety profile is comparable to placebo, with fewer side effects reported, reinforcing the drug's tolerability.

• BROOKLYN and TANDEM trials both show consistent LDL and Lp(a) reductions, with mono therapy cutting Lp(a) by about 50%.

• Mean LDL reduction is emphasized for predicting MACE benefit, with LS mean used for regulatory labeling.

Upcoming data and regulatory outlook

• BROADWAY, the largest trial in high-risk patients, will provide key safety and efficacy data in Q4, with expectations of consistent LDL lowering and sufficient safety exposure for FDA requirements.

• MACE data from BROADWAY will be closely watched, though short-term results may not fully reflect long-term benefit.

• PREVAIL, the cardiovascular outcomes trial, is designed with higher baseline LDL, risk enhancers, and longer follow-up to maximize event reduction and avoid pitfalls of prior CETP trials.

• The trial is powered for a 20% MACE reduction, with recent outcome trials setting a benchmark of 13–15%.