• Established new corporate responsibility strategic pillars and actionable goals
• Received approval for two Ionis-discovered medicines in 2023, and continued to advance a broad pipeline poised to bring a steady cadence of important new medicines to people with serious diseases

CARLSBAD, Calif., April 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), today published its 2023 Corporate Responsibility Report: Action for a Healthier Future. The report details Ionis' relentless passion to create better futures for people touched by serious disease as well as other stakeholders. In 2023, Ionis extended its commitment to corporate responsibility (CR) through the establishment of three strategic pillars with actionable goals. Ionis also committed to continuing to transparently communicate the Company's progress over time.

"At Ionis, we are focused on bringing a steady cadence of new medicines to people with serious diseases and believe our long-standing commitment to operating responsibly and sustainably is core to achieving this mission," said Brett Monia, Ph.D., chief executive officer of Ionis. "The actions outlined in our 2023 report are a testament to the extraordinary work our colleagues do every day to innovate for patients, maintain the highest standards of integrity, and ensure we continue to deliver on our corporate responsibility priorities for many years to come."

In 2023, Ionis completed its first CR materiality assessment, which informed the new CR pillars and goals. The company also formalized board-level oversight of the responsibility program and expanded its CR Steering Committee to include representation from across the Company. The 2023 Report includes the following highlights:

Innovate to improve the lives of people with serious diseases:

• Celebrated the U.S. approval of WAINUA™ (eplontersen) for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) and the accelerated approval of QALSODY® (tofersen) for superoxide dismutase type 1 amyotrophic lateral sclerosis (SOD1-ALS).
• Continued to advance a broad clinical pipeline, which now includes nine medicines in Phase 3 development for 11 serious diseases, as well as expanded
  technology capabilities.
• Worked closely with nearly 100 patient advocacy groups to support patients and caregivers across the globe.

Empowering our employees and communities:

• Continued to foster a diverse, equitable and inclusive workplace culture by launching a new corporate DEI site and employee VisION Awards, recognizing outstanding employees making an impact through championing innovation, community service and modeling Ionis' culture and core principles.
• Remained committed to volunteerism and philanthropy focused in the areas of adaptive experiences for people with neurological diseases, science, technology, engineering and math (STEM) education and bolstering our local communities.

Operating responsibility and sustainably:

• Disclosed multi-year environmental data and continued to manage our environmental impact as our operations grew, including breaking ground on a new state-of-the-art research facility on our Carlsbad, California campus, which is designed to achieve LEED Gold certification.
• In preparation for our first independent commercial launch, continued to focus on maintaining strong corporate governance, compliance, and a culture of integrity, while increasing transparency around company policies regarding the environment and clinical trials.

For more information on the 2023 Corporate Responsibility program, please visit: https://www.ionispharma.com/about/corporate-responsibility/

For more information about QALSODY, visit https://www.qalsody.com/. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

INDICATION for WAINUA™ (eplontersen)

WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen)

WARNINGS AND PRECAUTIONS

Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS

Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA.

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other 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 Ionispharma.com and follow us on X (Twitter) and LinkedIn. 

Forward-looking Statements

This press release may include forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of Ionis' commercial medicines, additional medicines in development and technologies. 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 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. 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 Dec. 31, 2023, which is on file with the SEC. Copies of this and other documents are available at www.ionispharma.com.    

Definition of Materiality in This Report

The discussion of topics included in this report and our other corporate responsibility and sustainability disclosures should not be read as implying that such topics are "material" in the context of the U.S. federal securities laws, Delaware General Corporation Law or any other regulatory framework, even where we use words such as "material" or "materiality. Our approach to sustainability and other corporate responsibility disclosures is informed by sustainability reporting frameworks, that involve broader definitions of materiality than used for purposes of our compliance with SEC disclosure obligations. As a result, "materiality" for purposes of our corporate responsibility reporting includes impacts on communities, the environment and stakeholders such as employees, patients and suppliers, and the inclusion of topics in such reporting, even when described as "material," does not indicate that such topics are material to the Company's business, operations or financial condition.

Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. QALSODY® is a registered trademark of Biogen. WAINUA^TM is a registered trademark of the AstraZeneca group of companies.

Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. - This email address is being protected from spambots. You need JavaScript enabled to view it. - 760-603-2331; Ionis Pharmaceuticals Media Contact: Hayley Soffer - This email address is being protected from spambots. You need JavaScript enabled to view it. - 760-603-4679