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author：focus 2025-09-30 00:05:09 - browse：2126

The headquarter of Biotechnology company Ionis Pharmaceuticals, consists of glass windows, pale yellow exteriors and plants outside the building — biotech coverage from STAT — courtesy Ionis Pharmaceuticals

Ionis Pharmaceuticals, a California company that pioneered a whole class of RNA-targeted medicines, reported on Friday that a pair of late-stage trials showed that its experimental therapy for hereditary angioedema benefitted patients — including those taking drugs currently on the market.

In one of the Phase 3 trials, dubbed OASIS, patients with hereditary angioedema, a rare disease characterized by severe bouts of swelling, were given injections of donidalorsen either monthly or every two months. Those in the former group had an 81% lower monthly rate of swelling attacks after 25 weeks than patients given a placebo, while the reduction compared to placebo was 55% for those treated every two months.

The other study, OASISplus, found that donidalorsen’s benefits deepened and converged after up to an additional year of treatment, with swelling attack rates dropping by 92% to 93% regardless of how often patients were dosed. Notably, this study also tracked patients who switched over to donidalorsen from existing therapies meant to prevent swelling attacks, including drugs sold by Takeda and BioCryst Pharmaceuticals. Patients who switched to the Ionis drug saw their swelling attack rates drop by 62% compared to previous treatment.

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