Clinical Trial Update

Ionis Pharma Announces Positive Results From Phase 2 Study Of Antisense Medicine Donidalorsen

November 10,2021 09:58 AM
- By Admin

Ionis Pharmaceuticals, Inc., the leader in RNA-targeted therapies, announced that positive results from the phase 2 study of its investigational antisense medicine, donidalorsen (formerly IONIS-PKK-LRx). The results were 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.

HAE 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.

Topline 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.

"A phase 2 Study Evaluating an Antisense Oligonucleotide to Prekallikrein in Patients with Hereditary Angioedema" presented on Sunday, November 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, allergy diagnostics and immunotherapy).

HAE is a rare genetic disease that is characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea. HAE affects approximately 20,000 patients in the United States and Europe and can be fatal if swelling occurs in the larynx. In patients with frequent or severe attacks, doctors may use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks.

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology.