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Ionis Pharmaceuticals (Nasdaq: ions(Amazon) announced Friday that its investigational treatment dondalorsen has achieved positive results in two late-stage trials for hereditary angioedema (HAE), a rare genetic disorder characterized by body swelling.
Citing data from the phase III studies OASIS-HAE and OASISplus, the company Dundalorsen, an RNA-targeted treatment, reduced rates of hereditary angioedema after one year of treatment with monthly or bimonthly doses, he said.
Based on 24-week data from the OASIS-HAE study of 91 HAE patients aged 12 years and older, the Carlsbad, Calif.-based biotech said dondalorsen as a subcutaneous treatment met its primary endpoint in terms of HAE attack rates.
According to the company, use of donedalorsen at doses Q4W and Q8W was associated with an 81% and 55% reduction in monthly angioedema attack rates compared with those who took placebo from weeks 1 to 25, respectively.
Additionally, citing a data cutoff on February 28, 2024, Ionis said the OASISplus study, a 52-week open-label trial for those who completed the OASIS-HAE, indicated continued improvement in HAE attack rates and safety outcomes consistent with the prior study.
Based on these readings, the company expects to receive regulatory approval for dundallorsen as a potential treatment for hereditary angioedema.