Novartis and Ionis Pharmaceuticals announced that their experimental RNA drug pelacarsen failed a Phase 3 clinical trial of over 8,000 participants, failing to reduce cardiovascular risks despite successfully lowering lipoprotein(a) levels. The failure sent Novartis shares down 3.3% on September 7, 2026, wiping out a potential blockbuster with estimated peak sales of $3 billion to $6 billion. The setback raises the bar for rival lipoprotein(a) therapies from Amgen and Eli Lilly.
Pelacarsen Phase 3 trial failure
- ▪Novartis and Ionis Pharmaceuticals announced on September 4, 2026, that their RNA drug pelacarsen failed to reduce the risk of heart attacks, strokes, and death in a Phase 3 clinical trial
- ▪The Phase 3 clinical trial of pelacarsen included more than 8,000 participants who received either a combination of pelacarsen and standard medications or typical therapies and a placebo
Lp(a) reduction without cardiovascular benefit
- ▪In an earlier clinical trial, pelacarsen decreased lipoprotein(a) levels in study participants by as much as 80% after 24 weeks, according to William Blair analyst Myles Minter
- ▪Novartis Chief Medical Officer Shreeram Aradhye stated that pelacarsen failed to lower cardiovascular risks despite successfully dropping levels of lipoprotein(a) in treated participants
Rival Lp(a) drug programs
- ▪Amgen's experimental lipoprotein(a)-lowering drug reduced lipoprotein(a) levels by more than 95% in mid-stage clinical testing
- ▪Pharmaceutical companies Amgen, Eli Lilly, and Silence Therapeutics are developing alternative therapies to stop genes from making lipoprotein(a) particles or block their formation
- ▪The failure of pelacarsen raises the bar and increases risk for rival lipoprotein(a)-lowering drug candidates currently in late-stage trials by Amgen and Eli Lilly
Financial impact on Novartis pipeline
- ▪Financial analysts had estimated peak annual sales for pelacarsen to be between $3 billion and $6 billion
- ▪Shares of Novartis fell by 3.3% on the morning of September 7, 2026, following the announcement of the pelacarsen clinical trial failure
- ▪The failure of pelacarsen increases pressure on Novartis's upcoming clinical data for del-desiran, an experimental muscular dystrophy drug Novartis acquired for $12 billion in 2025
Lp(a) as cardiovascular risk factor
- ▪According to the American Heart Association, approximately 20% of people have high levels of lipoprotein(a), which cannot be altered through diet or exercise
- ▪Lipoprotein(a) is an inherited, fatty, cholesterol-carrying protein particle linked to heart disease and arterial plaque accumulation
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