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Ionis Presents Positive Phase 2 Data from Donidalorsen Open-Label Extension Study at the 2022 ACAAI Annual Meeting | BioSpace

Interim Results Show Sustained Reduction in Hereditary Angioedema Attacks and No New Safety Signals in Patients Treated for One Year

CARLSBAD, Calif., Nov. 13, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported 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 completed 1 year of treatment in the study showed a sustained reduction in HAE attacks and no new safety signals after donidalorsen treatment. Treatment with donidalorsen resulted in an overall sustained mean reduction in HAE attack rates of 95% from baseline. For patients treated with donidalorsen, 99.6% of study days passed without an HAE attack.

The data was presented at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Louisville, Ky. Additional details can be found on the ACAAI website.

“Today’s data further enhances the profile of donidalorsen and its potential to provide significant long-lasting protection against stroke for people living with hereditary angioedema,” said Richard S. Geary, Ph.D., Executive Vice President and Chief Development Officer at Ionis. “The positive OLE Phase 2 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. There were 20 patients with HAE type 1 or type 2 in the phase 2 study, and 17 (85%) entered OLE. After a 13-week fixed-dose period in which participants received subcutaneous donidalorsen 80 mg every four weeks, eight patients were switched to subcutaneous donidalorsen 80 mg every eight weeks. Patients who remained on donidalorsen 80 mg every 4 weeks had an average 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. Patients receiving donidalorsen 80 mg every eight weeks had a mean attack rate reduction of 75.6% from baseline and the mean monthly attack rate was 0.28. Five of these patients remained seizure-free over the one-year duration of this analysis, and three patients returned to 80 mg every four weeks.

No serious adverse events were reported in the OLE study and no treatment-related adverse events (TEAEs) led to study discontinuation. There were no clinically relevant abnormalities in laboratory measurements.

About Hereditary Angioedema (HAE)

HAE is a rare and life-threatening genetic disease characterized by rapid and painful attacks of inflammation of 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 the larynx swells. In patients with frequent or severe attacks, doctors can use prophylactic treatment approaches to prevent and reduce the severity of HAE attacks.

About Donidorsen

Donidalorsen is an investigational antisense drug that uses Ionis’ advanced technologies LIthinking-VSconjugate 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 of 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.

About Ionis Pharmaceuticals, Inc.

For more than 30 years, Ionis has been the leader in RNA-targeted therapy, opening new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed drugs and a leading late-stage pipeline highlighted by industry-leading 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 a leading, fully integrated biotechnology company.

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

Ionis Forward-Looking Statements

This press release contains 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’ objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. These statements are subject to certain risks and uncertainties, including those related to the impact that COVID-19 may have on our business, and including, but not limited to, those related to our commercial products and pharmaceuticals. our pipeline, and in particular those inherent in the process of discovering, developing and commercializing safe and effective drugs for human therapeutic use, and in the effort to build a business around these drugs. Ionis’ forward-looking statements also involve assumptions which, if they never materialize or prove to be incorrect, 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, such statements are based solely on facts and factors currently known to Ionis. Accordingly, you are cautioned not to rely on these forward-looking statements. These and other risks relating to Ionis’ programs are described in more detail in Ionis’ Annual Report on Form 10-K for the fiscal year ended December 31, 2021, and most recent quarterly Form 10-Q filing. , which is filed with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

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

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

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SOURCEIonis Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:IONS

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