On September 8, Ionis Pharmaceuticals fell 12.19% in pre-market trading, trading at $50.95/share, with turnover of $4.99 million.

On the news front, partner Novartis announced on September 4 that pelacarsen, a drug discovered and initially developed by Ionis, failed to meet the primary endpoint in the Phase 3 LpHORIZON cardiovascular outcomes trial. While the drug significantly reduced lipoprotein(a) levels, it failed to translate into a meaningful reduction in cardiovascular event risk. Pelacarsen had been considered a potential blockbuster targeting elevated Lp(a), a well-established risk factor for cardiovascular disease.

This marks the second major late-stage clinical setback for Ionis in recent months. In July, eplontersen, developed in partnership with AstraZeneca for transthyretin-mediated amyloid cardiomyopathy, also failed to meet its primary efficacy endpoint in the Cardio-TTtransform Phase 3 trial, which previously sent shares down more than 20%.

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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