Ionis Pharmaceuticals publishes positive results from OASISplus study, donidalorsen shows significant HAE attack rate reduction and patient preference.

Ionis Pharmaceuticals announced results from the Phase 3 OASISplus study of donidalorsen for hereditary angioedema. Patients who switched to donidalorsen from prior treatments showed a 62% further reduction in mean monthly HAE attack rate from baseline, with 84% of patients preferring donidalorsen. The new drug application for donidalorsen is under review with the FDA, with a target action date of August 21.

Ionis Pharmaceuticals, Inc. (IONS) has announced positive results from the Phase 3 OASISplus study of donidalorsen, an investigational RNA-targeted medicine for hereditary angioedema (HAE). The study, published in The Journal of Allergy and Clinical Immunology (JACI) In Practice, showed that patients who switched to donidalorsen from prior prophylactic treatments experienced a 62% further reduction in mean monthly HAE attack rate from baseline. Additionally, 84% of patients surveyed reported a preference for donidalorsen over their previous treatment [1].

HAE is a rare and potentially life-threatening genetic condition characterized by recurrent attacks of severe swelling (angioedema) in various parts of the body. The study included an open-label extension (OLE) cohort of patients from the OASIS-HAE trial, as well as a prospective cohort of patients switching from oral and injectable long-term prophylactic treatments to donidalorsen. The OASISplus switch cohort evaluated the safety and efficacy of donidalorsen administered every four weeks in patients previously treated with lanadelumab, C1-esterase inhibitor, or berotralstat for at least 12 weeks before entering the study [2].

After 16 weeks of treatment with donidalorsen, patients experienced a 62% overall reduction in mean HAE attack rate compared to baseline. The attack rate decreased by 65%, 41%, and 73% for patients switching from lanadelumab, C1INH, and berotralstat, respectively. The majority of patients (84%) reported a preference for donidalorsen, citing better disease control, less time to administer, and less injection site pain or reactions. Quality-of-life measures also improved regardless of prior therapy. Donidalorsen was well-tolerated, with no serious treatment emergent adverse events (TEAEs) related to donidalorsen [1].

The New Drug Application (NDA) for donidalorsen to prevent attacks of HAE in adult and pediatric patients 12 years of age and older is currently under review with the U.S. Food and Drug Administration (FDA), with a target action date of August 21, 2025. If approved, donidalorsen could represent a significant advancement in the treatment of HAE, offering patients a more effective and convenient prophylactic option.

References:
[1] https://www.businesswire.com/news/home/20250721310278/en/Phase-3-OASISplus-data-demonstrating-benefit-of-donidalorsen-for-HAE-patients-who-switched-from-other-prophylactics-published-in-JACI-In-Practice
[2] https://www.marketscreener.com/news/ionis-pharmaceuticals-inc-announces-positive-results-from-the-phase-3-oasisplus-prospective-switch-ce7c5cdcd981f02c