Ionis announces positive donidalorsen late-stage clinical progress in HAE
- Two-year treatment with donidalorsen in the Phase 2 open label study resulted in consistent, sustained protection from HAE attacks
- Completed enrollment in the Phase 3 OASIS-HAE study
- Topline Phase 3 results expected in H1 2024
"By completing enrollment in the Phase 3 study, we are one step closer to bringing a potentially transformative and differentiated prophylactic treatment to HAE patients," said
The two-year Phase 2 OLE results will be presented at an upcoming medical congress.
In the Phase 2 study, through week 17, donidalorsen 80 mg monthly demonstrated a 90% reduction in angioedema attacks compared with placebo after the first dose, and a 97% reduction in angioedema attacks starting with the second dose. The Phase 2 results also showed a significant improvement in quality of life as assessed by the Angioedema Quality of Life Questionnaire (AE-QoL), in the patients treated with donidalorsen. Donidalorsen continues to demonstrate a favorable safety and tolerability profile with added two-year OLE data.
OASIS-HAE is a multi-center, double-blind, randomized, placebo-controlled study of monthly and bimonthly subcutaneous injections of donidalorsen or placebo in 84 participants, age 12 and above, with Type 1 and Type 2 hereditary angioedema (HAE). The study is designed to evaluate the safety and efficacy of donidalorsen in participants with HAE and the effect of donidalorsen on the severity and pattern of HAE attacks and its impact on quality of life (QoL). Participants were randomized in a 2:1 ratio to Cohort A (donidalorsen or placebo every four weeks) or Cohort B (donidalorsen or placebo every eight weeks), respectively. Within each cohort, participants were randomized in a 3:1 ratio to receive donidalorsen or matching-placebo. The primary endpoint is the time-normalized number of investigator-confirmed HAE attacks from week one to 25. A key secondary endpoint is change in the Angioedema Quality of Life Questionnaire (AE-QoL) total score at week 25. Additional information about OASIS-HAE (NCT05139810) and the Phase 2 open-label extension study (NCT04307381) may be found at ClinicalTrials.gov.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially fatal genetic disease characterized by severe and potentially fatal swelling of the arms, legs, face and throat1,2. HAE is estimated to affect more than 20,000 patients in the
Donidalorsen is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target the prekallikrein, or
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.
To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of donidalorsen, Ionis' technologies and other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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1 Cicardi M, et al. Allergy. 2012;67(2): 147-157.
2 Zuraw BL. N Engl J Med. 2008;359(10): 1027-1036.
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