On September 8, Ionis Pharmaceuticals fell 7.76% in regular trading, trading near $54.40 per share, with turnover of approximately $98.08 million. The decline was driven by news that partner Novartis announced pelacarsen — discovered and initially developed by Ionis — failed to meet the primary endpoint in the Phase 3 LpHORIZON cardiovascular outcomes trial. Despite significantly reducing lipoprotein(a) levels, the drug did not translate that reduction into a meaningful decrease in cardiovascular event risk.
Notably, this marks the second major Phase 3 trial setback for Ionis within two months. In July, eplontersen — co-developed with AstraZeneca for transthyretin-mediated amyloid cardiomyopathy — also failed to meet its primary endpoint in the Cardio-TTtransform trial, which sent shares plunging over 20% at the time. The repeated late-stage clinical failures have significantly eroded investor confidence in the company's cardiovascular pipeline.
Ionis Pharmaceuticals is a commercial-stage biotechnology company with seven marketed drugs and a pipeline spanning neurology and cardiometabolic diseases. The company recently received FDA approval for Zanvastro for Alexander disease and has key partnerships with Biogen, AstraZeneca, and Novartis.
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