NewAmsterdam Pharma aced the first in a cascade of Phase 3 readouts, showing that its lead cardio medicine took down levels of bad cholesterol.
The positive results, if they bear out in future readouts, could shift the perception of CETP inhibitors, a class of drugs that held blockbuster potential but over the years has been troubled by problematic safety readouts or lackluster efficacy. Amgen, Lilly and Merck are among the names who have walked away from CETP drugs after disappointing results. NewAmsterdam’s candidate was previously cast aside by Amgen.
In Monday’s trial readout, obicetrapib was found to reduce LDL cholesterol by an average of 36.3% compared with placebo at the 12-week mark, a statistically significant difference. The difference grew to 41.5% by the end of one year. All patients in the trial were on maximally tolerated statins.
On the safety side, the rate of adverse events was lower in the drug arm than on placebo, and no severe side effects related to the drug arose in the treatment arm. There were three total deaths in the obicetrapib arm, which included a very sick patient who died on day six, a surfing accident, and another who died of COPD, CEO Michael Davidson said.
“In all my years of clinical trials and again, speaking as a clinician, safety is far more important than anything else,” Davidson said, calling the profile of the drug “excellent.”
Major data ahead, and maybe M&A
The results are the first is a handful of key data releases expected over the next six months that will likely set NewAmsterdam’s future.
But one question looming over NewAmsterdam as data roll in is whether it will even reach the market before a larger pharma comes calling. Davidson, who oversaw two prior acquisitions at other biotechs, confirmed NewAmsterdam had been in conversation with larger companies prior to the readout.
“We believe we will be highly sought after once we have our Phase 3 data,” he said. But he added that in his prior experience, being prepared to hit the market solo is critical leverage ahead of any sale.
“Getting this drug ready to launch is going to create a lot of value for anybody—for us or for a big pharma company,” he said.
Up next is a Phase 3 study testing obicetrapib in patients with atherosclerotic cardiovascular disease and/or heterozygous familial hypercholesterolemia, a genetic condition that results in elevated cholesterol levels. Then, in the first quarter of 2025, NewAmsterdam expects to have data on a fixed dose combo of obicetrapib and ezetimibe in patients with either of the same two conditions.
Once those two other trials are read out, NewAmsterdam will go to regulators. Davidson said the FDA has told NewAmsterdam it wants to see quality safety data before making a decision. He also said he wants to wait to file at a time when there would be cardiovascular outcome data available when the drug is hypothetically launched (its outcomes trial is expected to read out in late 2026).
Any label that includes a reduction in the risk of serious cardiac events would put NewAmsterdam squarely in competition with Novo Nordisk and Wegovy. Davidson called the GLP-1 class “a little bit of a headwind,” but felt more confident after Wegovy showed virtually zero improvements in LDL-C levels compared with placebo.
“We can, I think, fit very well into a GLP-1 kind of world,” Davidson said.
The company also expects to produce future data on how obicetrapib works in tandem with GLP-1s, with a growing share of enrolled patients on background therapy in NewAmsterdam’s Phase 3 studies.