Phase 2 study results of Ionis' novel antisense treatment for hereditary angioedema to be presented at ACAAI annual meeting
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" will be presented on
About Hereditary Angioedema
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
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. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.
To learn more about Ionis, visit www.ionispharma.com and follow us on twitter @ionispharma.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of Ionis' technologies, donidalorsen 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 the impact COVID-19 could have on our business, and including but not limited to 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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