Novartis' Cholesterol-Lowering Drug Pelacarsen Fails in Key Study

Novartis shares plummeted over 3% on Monday after its cholesterol-lowering drug pelacarsen failed in a crucial study, marking a significant setback for the company's pipeline. Analysts had predicted peak annual sales of between $3 billion and $6 billion if the drug proved successful.

The failure raises pressure on upcoming data from Novartis' RNA therapy del-desiran, which is being tested in patients with a rare muscle disease acquired through its $12 billion purchase of Avidity. Del-desiran's success is crucial to justify its high price tag, according to Barclays analysts.

Novartis' anti-inflammatory drug remibrutinib, meanwhile, has shown promise in treating multiple sclerosis patients and hidradenitis suppurativa, but its potential to lower cholesterol levels effectively remains uncertain. The company plans to present full results for pelacarsen at a medical meeting later this year.

The failure of Novartis' drug also casts doubt on the wider industry effort to target lipoprotein(a), or Lp(a), an inherited cardiovascular risk factor with no approved treatments. Amgen and Eli Lilly are running late-stage trials of rival experimental medicines, olpasiran and lepodisiran.