PM Images Ionis Pharmaceuticals ( NASDAQ: IONS ) announced Friday that its investigational therapy donidalorsen generated positive results in two late-stage trials for hereditary angioedema (HAE), a rare genetic disorder characterized by body swelling. Citing data from its Phase 3 OASIS-HAE and OASISplus studies, the company said that donidalorsen, an RNA-targeted therapy, cut HAE attack rates after one year of treatment with monthly or every two-month-dosing. Based on 24-week data from its OASIS-HAE study involving 91 HAE patients aged 12 years and older, the Carlsbad, California-based biotech said donidalorsen as a subcutaneous therapy met its primary endpoint in terms of HAE attack rates.
According to the company, donidalorsen with Q4W and Q8W dosing was linked to an 81% and 55% lower monthly HAE attack rate than those on placebo from Week 1 to 25, respectively. Additionally, citing a February 28, 2024, data cut, Ionis said its OASISplus Study, a 52-week open-label trial for those who completed OASIS-HAE, indicated continuously improving HAE attack rates and safety results in line with the prior study. Based on these readouts, the company expects to seek regulatory approval for donidalorsen as a potential HAE therapy.
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