On September 8, Ionis Pharmaceuticals fell 9.78% overnight, trading at $52.41/share, with turnover of $57,300. The decline was triggered by partner Novartis announcing on September 4 that pelacarsen, a drug discovered and initially developed by Ionis, failed to meet the primary endpoint in its Phase 3 Lp(a)HORIZON cardiovascular outcomes trial.

According to the announcement, while pelacarsen significantly reduced lipoprotein(a) levels, the reduction did not translate into a meaningful lowering of cardiovascular event risk — a critical disconnect that undermined the drug's commercial thesis. This marks the second major Phase 3 setback for Ionis in recent months, following the failure of eplontersen in the Cardio-TTRansform ATTR-CM trial with AstraZeneca in July, which had already sent shares down over 20%.

Despite these pipeline headwinds, Ionis has secured recent wins including FDA approval of Zanvastro for Alexander disease and Tryngolza for severe hypertriglyceridemia, and reported Q1 revenue of $246 million, well above the $195.6 million estimate. The company guided full-year revenue of $875-900 million versus the FactSet estimate of $808.5 million.

Ionis Pharmaceuticals is a fully integrated commercial-stage biotechnology company with seven marketed drugs and a pipeline spanning neurology, cardiometabolic diseases, and other therapeutic areas.

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