Two major pharmaceutical companies, Novartis and Novo Nordisk, have faced setbacks in their efforts to develop new heart disease treatments. Novartis's experimental drug, pelacarsen, failed to reduce deaths or prevent second heart attacks despite significantly lowering Lp(a), a genetic risk factor for heart disease. The trial, involving over 8,300 patients, showed no meaningful benefits after nearly five years of treatment. Dr. Steven Nissen of the Cleveland Clinic, who presented the results at the American Heart Association meeting, expressed surprise at the outcome, stating, "It didn’t do anything. It was completely neutral."
Similarly, Novo Nordisk's anti-inflammatory drug, ziltivekimab, also failed to meet its primary goals. Although the drug successfully reduced inflammation, it did not prevent major adverse cardiovascular events in high-risk patients. The trial involved 6,300 patients with heart disease, advanced kidney disease, and systemic inflammation. Dr. Paul Ridker of Harvard’s Brigham and Women’s Hospital suggested that the drug might have been studied too late in the disease progression to be effective.
Experts remain divided on the implications of these failures. Dr. Donald Lloyd-Jones of Boston University cautioned against abandoning Lp(a) as a therapeutic target, while Dr. Priscilla Hsue of UCLA noted that the ziltivekimab trial involved an extremely sick population. Other experimental drugs targeting Lp(a), such as Amgen’s olpasiran and Eli Lilly’s lepodisiran, are still in development, with results expected in early 2028 and 2029, respectively.
The setbacks raise questions about foundational theories regarding key factors underlying heart disease. Currently, there are no approved treatments for patients with high Lp(a), which affects roughly 20% of the global population. Researchers had hoped that lowering Lp(a) would yield benefits similar to statins, which reduce LDL cholesterol and lower the risk of heart attacks and strokes.