With new data in hand, Amgen plans to take heart drug into phase 3 to challenge Novartis

With fleshed-out phase 2 data, Amgen is now ready to launch a late-stage trial of its heart drug by the end of this year. The Big Pharma currently lags behind Novartis, which has already launched a fully-enrolled phase 3 in the niche cardiovascular space, though Amgen’s new data may give it a leg up on the Swiss drugmaker.  

The end-of-treatment data comes from Amgen’s phase 2 study of olpasiran, an interfering RNA treatment previously known as AMG 890. This summer, the company shared limited data from the trial, dubbed OCEAN(a)-DOSE, which found the therapy reduced levels of lipoprotein, or Lp(a)—a risk factor for cardiovascular disease. Research suggests that high levels of Lp(a) can contribute to stroke, peripheral arterial disease and heart attack.

The data deep dive into the RNA silencer has cemented previous findings. Presented at the American Heart Association’s Scientific Sessions 2022 and published Nov. 6 in the New England Journal of Medicine, the data assessed 281 adults with elevated levels of Lp(a) and evidence of atherosclerotic cardiovascular disease. It found that olpasiran cut Lp(a) levels by over 95% for patients receiving 75 mg or more every three months.

This sets Amgen apart in the niche space without any approved therapy that can consistently and significantly reduce Lp(a) levels. Other players include Novartis, whose pelacarsen is a phase 3 antisense/GalNAc conjugate licensed from Ionis in 2019 to reduce Lp(a) and is currently being evaluated among 8,325 patients in a trial expected to wrap up in May 2025. However, pelacarsen has only shown a 72% reduction in Lp(a) at a 60-mg monthly dose, with an 80% decline at 20 mg every week, analysts at SVB Securities pointed out.

U.K.-based Silence Therapeutics also has an investigational siRNA/GalNAc conjugate—similar to olpasiran—known as SLN360 that is being tested in the same indication. However, the company is a few strokes behind, with a phase 2 study in the works but not yet enrolling patients and an estimated completion date of mid-2024.

Olpasiran came to the forefront for Amgen in 2020 when it acquired the drug from Arrowhead Pharmaceuticals and canceled a partnership with Cytokinetics for the heart failure drug omecamtiv mecarbil after a disappointing phase 3 readout.  

Now, with the new olpasiran results in hand, Amgen plans to launch a phase 3 pivotal trial this December among 6,000 cardiovascular patients. The study’s main goal is to measure "time to [coronary heart disease] death, myocardial infarction or urgent coronary revascularization, whichever occurs first," according to ClinicalTrials.gov. The trial’s estimated completion date is set for December 2026.

Editor's note: This article was updated at 9 p.m. ET to include olpasiran's acquisition from Arrowhead Pharmaceuticals.