Ionis Pharmaceuticals IncPelacarsen, Ionis's partnered Lp(a)-lowering drug, failed its Phase III cardiovascular outcomes trial, removing a major potential growth driver and heightening concentration risk.
Pelacarsen, an experimental Lp(a)-lowering drug from Novartis AG and partner Ionis Pharmaceuticals, Inc., failed to reduce heart attacks, strokes and related cardiovascular events in the Phase III Lp(a)HORIZON trial, which enrolled more than 8,000 patients, Reuters reported on September 5, 2026. The drug did lower Lp(a) levels as expected, but that reduction did not translate into fewer real-world cardiac events. Novartis shares fell 5% and Ionis shares fell 12% in aftermarket trading. Novartis called the results a disappointment but said they still advance scientific understanding of the Lp(a) pathway. The failure removes a major potential growth driver for both companies and raises the burden of proof for other Lp(a) drugs, with Novartis now under greater pressure to replace revenue as Entresto and other products approach patent challenges. Ionis faces greater concentration risk because pelacarsen mattered more to the smaller company, though its broader RNA portfolio and newly approved Zanvastro for Alexander disease provide some diversification.
Ionis Pharmaceuticals IncPelacarsen, Ionis's partnered Lp(a)-lowering drug, failed its Phase III cardiovascular outcomes trial, removing a major potential growth driver and heightening concentration risk.
Novartis AGNovartis's pelacarsen failed the Phase III Lp(a)HORIZON trial, eliminating a key growth driver and increasing pressure to replace revenue as Entresto faces patent challenges.
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