Ionis presents positive Phase 2 data from open label extension study of donidalorsen at 2022 ACAAI Annual Meeting

Interim results show a continued reduction in hereditary angioedema attacks and no new safety signals in patients treated for one year

CARLSBAD, Calif., November 13, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS ) today presented positive results from a phase 2 open-label extension (OLE) study evaluating the safety and efficacy of its investigational antisense drug, donidalorsen, in patients with hereditary angioedema (HAE), a rare and devastating inflammatory disease. Interim data after all patients had completed 1 year of study therapy showed a continued reduction in HAE attacks and no new safety signals after treatment with donidalorsen. Treatment with donidalorsen resulted in an overall sustained mean reduction in HAE attack rate of 95% from baseline. For patients treated with donidalorsen, 99.6% of study days were free of HAE attacks.

(PRNevsfoto/Ionis Pharmaceuticals, Inc.)

The data were presented at the annual scientific meeting of the American College of Allergy, Asthma and Immunology (ACAAI) in Louisville, Ky. Additional details can be found on the ACAAI website.

“Today’s data further improve the profile and potential of donidalorsen to provide significant sustained attack protection for people living with hereditary angioedema,” said Dr. Richard S. Geary, executive vice president and director of development at Ionis. “The positive Phase 2 OLE results are encouraging as we continue to evaluate donidalorsen, a potentially best-in-class drug, in the ongoing OASIS Phase 3 program.”

Patients who completed the Phase 2 study were eligible for enrollment in the OLE study. In the phase 2 study, there were 20 patients with type 1 or type 2 HAE, and 17 (85%) entered OLE. After a 13-week fixed-dose period where participants received subcutaneous donidalorsen 80 mg every four weeks, eight patients switched to subcutaneous donidalorsen 80 mg every eight weeks. Patients who remained on donidalorsen at a dose of 80 mg every 4 weeks had a mean reduction in attack rate of 95.3% and 98.3%, from week 1 (after the first dose) and week 5 (after the second dose), respectively. respectively. Patients receiving donidalorsen 80 mg every eight weeks had a mean seizure rate reduction of 75.6% from baseline, and the mean monthly seizure rate was 0.28. Five of these patients remained seizure-free during the one-year duration of this analysis, and three patients returned to 80 mg every four weeks.

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No serious adverse events were reported in the OLE study and no treatment-emergent adverse events (TEAEs) led to study discontinuation. There were no clinically significant abnormalities in any laboratory measurement.

About Hereditary Angioedema (HAE)

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

About Donidalorsen

Donidalorsen is an investigational antisense drug used by Advanced Ionis LIgand-Cconnected Antisense (LICA) and is designed to reduce the production of prekallikrein, or PKK, to treat patients with HAE. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. HAE is a rare genetic disease characterized by rapid and painful attacks of inflammation in the hands, feet, limbs, face, abdomen, larynx and trachea. HAE can be fatal if swelling occurs in the larynx. In patients with frequent or severe attacks, doctors can use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks.

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About Ionis Pharmaceuticals, Inc.

For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing the standard of care with its novel antisense technology. Ionis currently has three drugs on the market and a top late-stage pipeline highlighted by the industry’s leading cardiovascular and neurological disease franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become a leading, fully integrated biotechnology company.

To learn more about Ionis, visit and follow us on Twitter @ionispharma.

Yonis’ forward-looking statements

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 Jonis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered a risk-taking statement. Such statements are subject to certain risks and uncertainties, including those related to the impact that COVID-19 may have on our business, including, but not limited to, those related to our commercial products and medicines in our portfolio, and especially those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in an effort to build a business around such drugs. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove to be accurate, could cause its results to differ materially from those expressed or implied by such forward-looking statements.

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Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known to Ionis. As a result, you are cautioned not to place undue reliance on these forward-looking statements. These and other risks related to Ionis’ programs are described in more detail in Ionis’ Annual Report on Form 10-K for the year ended December 31, 2021 and its most recent quarterly filing on Form 10-K, which is on file with Charter as of values ​​and the Exchange Commission. Copies of these and other documents are available at the Company.

In this press release, unless the context otherwise requires, “Ionis,” “the Company,” “we,” “our,” and “us” refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

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SOURCE Ionis Pharmaceuticals, Inc.

Company codes: NASDAK-NMS:IONS


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