Pelacarsen missed its late-stage cardiovascular endpoint in more than 8,000 patients, lowering Lp(a) but failing to significantly cut heart attacks, strokes or other cardiovascular outcomes.
The setback is the first major outcomes failure for the Lp(a) field, casting doubt on whether lowering the genetically driven cholesterol marker alone can deliver clinical benefit.
Novartis shares fell 3% on Monday after analysts said the drug appears to have fallen well short of the roughly 13% benefit needed for statistical significance; full data will be presented later.
Amgen, Eli Lilly and other rivals now face heavier scrutiny, with analysts saying deeper Lp(a) reductions or different trial designs may be needed to revive a market once modeled at $4 billion to $5 billion for pelacarsen alone.