Novartis AG shares declined by nearly 4% on Monday after its experimental heart drug, pelacarsen, failed to meet its primary endpoint in a late-stage trial. The drug, which was designed to lower lipoprotein(a) (Lp(a)) levels, successfully reduced Lp(a) in patients but did not translate into a reduction in deaths, heart attacks, or strokes compared to a placebo. This marks a significant setback for Novartis, which had hoped pelacarsen would be a blockbuster drug with peak annual sales estimates ranging from $1.5 billion to $5.4 billion.
The failure of pelacarsen, which follows a temporary pause in trials for an experimental cell therapy due to patient deaths, casts doubt on the broader therapeutic approach of targeting Lp(a) to prevent cardiovascular disease. Jefferies analyst Michael Leuchten questioned whether any Lp(a)-lowering therapies can ultimately demonstrate a cardiovascular benefit, although Citi analyst Geoffrey Meacham suggested it might be too early to declare the mechanism entirely unsuccessful without full trial data.
The news also impacted other companies developing similar drugs, with Amgen's stock falling almost 7% in post-market trading. The setback puts additional pressure on Novartis's pipeline, particularly for its experimental muscular dystrophy drug, del-desiran, for which data is expected in the fourth quarter. Novartis had acquired the rights to del-desiran through a $12 billion deal.
Despite this disappointment, Novartis did experience some recent success with its multiple sclerosis pill, remibrutinib, which showed positive results in two late-stage trials last week, leading to a temporary increase in share value. However, the pelacarsen failure underscores the challenges Novartis faces in sustaining growth as some of its major products approach patent expiration.
