NewAmsterdam's Amgen castoff cuts LDL cholesterol in phase 2, opening new front in race to market

NewAmsterdam Pharma has advanced its bid to write a new chapter in the story of an Amgen castoff by linking the CETP inhibitor to reductions in LDL cholesterol when used as an adjunct to high-intensity statin therapy. The Dutch biotech plans to start a phase 3 trial on the strength of the data.

Amgen acquired the CETP inhibitor, obicetrapib, for $300 million upfront in its 2015 takeover of Dezima Pharma. By 2017, the Big Biotech had given up on the drug. The failure of Merck & Co.’s rival candidate drove the final nail into the coffin by seeming to confirm doubts raised by earlier setbacks at Eli Lilly, Pfizer and Roche. Yet, key players in the Dezima story kept the faith, leading them to license obicetrapib from Amgen and pump money into further development.  

Now, operation save obicetrapib has delivered more phase 2 data. The placebo-controlled trial tested the CETP inhibitor as a monotherapy and in combination with ezetimibe, a cholesterol-lowering drug that Merck sold as Zetia. All participants stayed on high-intensity statin therapy after enrolling in the study.

The trial enrolled 119 patients with LDL cholesterol above 70 mg/dL and triglycerides below 400 mg/dL. After 12 weeks of daily dosing, LDL levels in the obicetrapib-ezetimibe combination cohort had fallen by 59%, compared to a 6% drop in the placebo arm. 

NewAmsterdam is yet to share data on the patients who received obicetrapib as a monotherapy. A meta-analysis of earlier clinical trials linked the addition of single agent ezetimibe to high-intensity statin therapy to a 14% reduction in LDL cholesterol. The meta-analysis suggests obicetrapib made a significant contribution to the 59% drop but the current lack of monotherapy data leaves questions unanswered. Obicetrapib lowered median LDL by 51% when given without ezetimibe in an earlier clinical trial.

The biotech plans to release full data in a publication or presentation at an upcoming medical meeting but has privately seen enough to commit to further development of the combination, as CEO Michael Davidson, M.D., said in a statement.

“We are now focused on selecting a formulated fixed-dose combination tablet of obicetrapib plus ezetimibe to advance into a definitive bioequivalence study and a phase 3 trial,” Davidson said. “If positive, the results from these trials, coupled with our ongoing BROADWAY, BROOKLYN and PREVAIL studies, if also positive, have the potential to support an application for approval of obicetrapib as an adjunct to maximally tolerated statin therapy and in a fixed-dose combination with ezetimibe.”

BROADWAY, BROOKLYN and PREVAIL are phase 3 trials that are testing obicetrapib as a monotherapy. The current phase 3 program consists of two assessments of the effect on LDL cholesterol of giving the CETP inhibitor as an adjunct to maximally tolerated lipid lowering therapy, and one evaluation of its effect on the risk of major adverse cardiovascular events. The first phase 3 data drops are due next year.