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Phase 2 study results of Ionis' novel antisense treatment for hereditary angioedema to be presented at ACAAI annual meeting
- Positive Phase 2 study results demonstrate significant efficacy of donidalorsen (formerly IONIS-PKK-LRx) in the reduction of hereditary angioedema attacks -
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"- Positive Phase 2 study results demonstrate significant efficacy of donidalorsen (formerly IONIS-PKK-LRx) in the reduction of hereditary angioedema attacks\n - Based on the results of the Phase 2 study, Ionis plans to initiate a Phase 3 program of donidalorsen in people with HAE\n\n\n CARLSBAD, Calif., Nov. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today that positive results from the Phase 2 study of its investigational antisense medicine, donidalorsen (formerly IONIS-PKK-LRx), will be presented at the American College of Asthma, Allergy & Immunology (ACAAI) Annual Scientific Meeting in New Orleans and via livestream, November 4-8. The Phase 2 study results support the clinical profile of donidalorsen as a potential, best-in-class prophylactic treatment for patients with hereditary angioedema (HAE), and underscore Ionis' commitment to advancing antisense technology to target the root cause of diseases.\n\n \n \n \n \n \n \n\n \nHAE is a rare and potentially fatal autosomal dominant disease that results in recurrent, painful attacks of swelling affecting the hands, feet, limbs, face, abdomen, larynx and trachea. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation of inflammatory mediators associated with acute attacks of HAE. Donidalorsen was developed using Ionis' advanced LIgand-Conjugated Antisense (LICA) technology.\nTopline results of the Phase 2 study, reported earlier this year, showed that donidalorsen met its primary and all secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with hereditary angioedema (HAE) compared to placebo. These data support advancing donidalorsen into Phase 3 development, which Ionis plans to initiate this year.\n\"A Phase 2 Study Evaluating an Antisense Oligonucleotide to Prekallikrein in Patients with Hereditary Angioedema\" will be presented on Sunday, Nov. 7 at 3:03 p.m. CT (recorded presentation) by Danny Cohn, M.D., Ph.D., Department of Vascular Medicine, Amsterdam Universities Medical Center, Amsterdam, The Netherlands. Dr. Cohn, a primary investigator in the study, will present results of the Phase 2 study during Oral Abstracts Session 2B (Angioedema/urticaria, food allergy,...