Ionis to present new analyses from CORE and CORE2 pivotal studies supporting olezarsen for severe hypertriglyceridemia (sHTG) at upcoming congresses

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that new data analyses from the landmark Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG) will be presented during four oral presentations at upcoming medical conferences. Data will be presented at the 2026 Scientific Sessions of the American Diabetes Association® (ADA) in New Orleans (June 5-8), the National Lipid Association (NLA) Scientific Sessions in Chicago (June 11-14) and ENDO 2026, the Endocrine Society's annual meeting in Chicago (June 13-16).

"People living with sHTG are at risk of acute pancreatitis, a debilitatingly painful complication that can potentially be life-threatening and cause other serious health consequences. Standard lipid-lowering therapies only offer modest benefits, underscoring the urgent need for innovative new treatment options," said Sam Tsimikas, M.D., senior vice president, global cardiovascular development, Ionis. "The data that will be presented expand the understanding of how olezarsen impacts patients across different clinical and treatment backgrounds, and we look forward to sharing these results with the medical community."

Ionis previously reported results from the Phase 3 CORE and CORE2 studies, in which olezarsen demonstrated a highly statistically significant reduction in fasting triglycerides and acute pancreatitis events with favorable safety and tolerability.

The marketing application for olezarsen for sHTG is under Priority Review by the U.S. Food and Drug Administration, with a target action date of June 30, 2026.

Presentations will be available on the Ionis website once each embargo lifts.

Key Presentation Details

The ADA's 2026 Scientific Sessions

• APOC3 Inhibitor Olezarsen Markedly Reduces Triglycerides and Risk of Pancreatitis in Severe Hypertriglyceridemia Patients with Diabetes Mellitus: Insights from CORE-TIMI 72a and CORE2-TIMI 72b
  • Oral Presentation: Sat., June 6, 3:15 – 3:30 CT
  • Presenting Author: Yu Mi Kang, M.D., Ph.D.

NLA Scientific Sessions

• Olezarsen for Managing Severe Hypertriglyceridemia and Pancreatitis Risk
  • Oral Presentation: Fri., June 12, 1:20 – 1:50 CT
  • Presenting Author: Nicholas Marston, M.D., MPH

ENDO 2026

• Prevention of Acute Pancreatitis With APOC3 Inhibition in Severe Hypertriglyceridemia Across Triglyceride Levels and Prior Pancreatitis
  • Oral Presentation: Sun., June 14, 2:00 – 2:15 CT
  • Presenting Author: Filipe Moura, M.D., Ph.D.

• APOC3 Inhibition for Severe Hypertriglyceridemia in Patients with Versus without Baseline Fibrate Use
  • Oral Presentation: Sun., June 14, 2:45 – 3:00 CT
  • Presenting Author: Annabel Wang, M.D., Ph.D.

About Severe Hypertriglyceridemia

Severe hypertriglyceridemia (sHTG) is defined by very high triglycerides (≥500 mg/dL) and characterized by an increased risk of acute pancreatitis and other serious health complications. Considered a medical emergency, acute pancreatitis causes debilitating abdominal pain that often requires prolonged hospitalization, can lead to permanent organ damage and can become life-threatening. Preventing the first attack is key. In people with a history of acute pancreatitis episodes, the risk of future attacks is even greater. Current standard of care therapies for sHTG and lifestyle modifications (such as diet and exercise) do not sufficiently or consistently lower triglyceride levels or reduce the risks of sHTG in all patients. More than 3 million people are living with sHTG in the U.S., including approximately 1 million who are considered high risk. High-risk sHTG includes those with triglycerides ≥880 mg/dL or triglycerides ≥500 mg/dL and a history of acute pancreatitis or other comorbidities.

About Olezarsen

Olezarsen is an investigational RNA-targeted medicine being evaluated for the treatment of sHTG. Olezarsen is designed to lower the body's production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. Olezarsen is approved in the U.S., the European Union and other countries as TRYNGOLZA® for adults with familial chylomicronemia syndrome (FCS).

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiometabolic disease and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.

Ionis Forward-looking Statements

This press release includes forward-looking statements regarding Ionis' business, the therapeutic and commercial potential of our commercial medicines, olezarsen, additional medicines in development and technologies, and our expectations regarding development and regulatory milestones. 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 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. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. 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 December 31, 2025, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals^® and TRYNGOLZA^® are trademarks of Ionis Pharmaceuticals, Inc.

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