Novartis announced that its pelacarsen drug, developed with Ionis Pharmaceuticals, did not significantly improve cardiovascular outcomes despite reducing harmful cholesterol levels in a late-stage trial.
This setback is significant as it marks the first major failure in the race to develop effective treatments for Lp(a), which affects approximately one in five people globally and currently lacks approved therapies. Following the announcement, Novartis' stock fell 3%, reflecting investor concerns about the implications for the broader market, which analysts had estimated could be worth billions.
The failure has also negatively impacted shares of competitors Amgen and Ionis, with declines of about 5% and 10%, respectively. Analysts from Citi noted that while the Lp(a) hypothesis is weakened, it is not entirely disproven, and further data is needed to understand the trial's results.
Novartis' chief medical officer emphasized the importance of the findings for future cardiovascular risk management. The trial involved over 8,000 patients already receiving optimized care, and Jefferies analysts pointed out that improving standards of care may complicate the demonstration of additional benefits from new treatments.
The potential market for pelacarsen was projected at $4 billion to $5 billion annually, which would have been crucial for Novartis as it faces significant patent expirations.
The trial's outcome raises the stakes for Amgen and Eli Lilly, whose competing drugs are still in development, with analysts suggesting that the failure places greater pressure on their upcoming studies to show meaningful cardiovascular benefits