A new experimental cholesterol drug, Obicetrapib, has shown potential as a groundbreaking treatment for reducing heart attack risk, according to a Phase 3 clinical trial led by Monash University researchers in Australia. The study, published in The New England Journal of Medicine, found that the once-daily pill significantly lowered levels of LDL cholesterol (often referred to as ‘bad cholesterol’) by 32.6% and lipoprotein(a) (Lp(a)) – a genetically inherited risk factor for heart disease – by 33.5% in participants who were already receiving maximum cholesterol-lowering therapy.
The drug is considered a promising option for patients who have not achieved sufficient cholesterol reduction with current treatments, as it is well-tolerated and offers a convenient daily dosage. However, researchers caution that while the initial results are encouraging, long-term studies are needed to confirm its impact on heart attack and stroke prevention, particularly in patients with very high Lp(a) levels.
The clinical trial involved over 2,500 participants, mostly individuals over the age of 65 who had either been diagnosed with heart disease or had genetically high cholesterol. All participants were on maximum tolerated doses of cholesterol-lowering medications, and one group received Obicetrapib while the other received a placebo. After 12 weeks, the group taking the drug showed significant reductions in both LDL and Lp(a), indicating potential for a new therapeutic approach for patients who have exhausted traditional treatment options.
Professor Stephen Nicholls, the lead researcher and director of Monash University’s Victorian Heart Institute, emphasized the importance of the findings, noting that patients at high risk for heart attacks and strokes often struggle to achieve optimal cholesterol levels with existing treatments. ‘Obicetrapib offers a promising new option— not only did it lower LDL cholesterol by over 30%, but we also saw a reduction in Lp(a), which is much harder to treat and has been linked to increased heart disease risk,’ he stated in a university press release.
Despite the encouraging results, the study had its limitations. The researchers acknowledged that the participants were not specifically selected for high Lp(a) levels, and the study did not measure actual heart attack or stroke outcomes. Additionally, long-term follow-up data is needed to fully assess the drug’s efficacy and safety in a broader population. Researchers from the study have called for more diverse and longer-term trials to ensure Obicetrapib’s effectiveness across different patient groups and to monitor its real-world impact on cardiovascular health.
The drug was developed by NewAmsterdam Pharma, a Dutch biotech company that specializes in lipid-lowering therapies. The study was funded by this company, and while the results are promising, the researchers remain cautious and advise that further clinical studies are necessary before the drug can be widely prescribed. The findings were presented at the European Atherosclerosis Society Congress and are now available in the prestigious medical journal, The New England Journal of Medicine, underscoring the significance of the research in the field of cardiovascular medicine.