Olezarsen Sodium

– TRYNGOLZA® (olezarsen) outperforms expectations, achieved 2025 preliminary U.S. net product sales of $105M * as first FDA-approved treatment for FCS ; sNDA for sHTG submitted for review – – Increasing annual TRYNGOLZA peak net sales guidance to > $2B for sHTG – – Positive topline results announced for pivotal Phase 3 program of bepirovirsen in chronic hepatitis B; first of five Phase 3 readouts from partnered programs expected this year – CARLSBAD, Calif. --(BUSINESS WIRE)--Jan. 12, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2025 achievements and provided additional updates on key milestones expected in 2026. Ionis will provide a business update at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13 at 8:15am PT ; the presentation is available today on the Ionis website here. “2025 was a defining year for Ionis, as we successfully executed our first two independent launches as a commercial stage biotech company. We expect 2026 to be another transformative year, poised for two additional independent launches of breakthrough therapies – olezarsen for severe hypertriglyceridemia, Ionis’ first launch in a large patient population, and zilganersen for Alexander disease, Ionis’ first independent launch from our leading neurology pipeline,” said Brett P. Monia , Ph.D., chief executive officer, Ionis. “This momentum is further bolstered by the promise of our late-stage Ionis owned and partnered programs, with a total of five Phase 3 readouts and four NDA submissions anticipated this year. With strong execution and multiple key catalysts expected, Ionis is well-positioned to deliver accelerating revenue growth to achieve cash flow breakeven in 2028.” Preliminary TRYNGOLZA® (olezarsen) 2025 Full Year U.S. Net Sales TRYNGOLZA outperformed expectations as the first U.S. Food and Drug Administration (FDA)-approved treatment for familial chylomicronemia syndrome (FCS), generating $105 * million in preliminary U.S. net product sales in 2025 2026 Anticipated Highlights Include: Ionis Owned Programs Potential approval and launch of olezarsen as the new standard of care for severe hypertriglyceridemia (sHTG) Submitted U.S. Supplemental New Drug Application (sNDA), following receipt of Breakthrough Therapy Designation Olezarsen achieved a highly statistically significant placebo-adjusted reduction of up to 72% in fasting triglycerides and an 85% reduction in acute pancreatitis events with favorable safety and tolerability in pivotal Phase 3 CORE and CORE2 studies Increasing annual olezarsen peak net sales guidance to > $2 billion from > $1 billion based on strong product profile and positive Phase 3 data Submitted U.S. Supplemental New Drug Application (sNDA), following receipt of Breakthrough Therapy Designation Olezarsen achieved a highly statistically significant placebo-adjusted reduction of up to 72% in fasting triglycerides and an 85% reduction in acute pancreatitis events with favorable safety and tolerability in pivotal Phase 3 CORE and CORE2 studies Increasing annual olezarsen peak net sales guidance to > $2 billion from > $1 billion based on strong product profile and positive Phase 3 data Olezarsen achieved a highly statistically significant placebo-adjusted reduction of up to 72% in fasting triglycerides and an 85% reduction in acute pancreatitis events with favorable safety and tolerability in pivotal Phase 3 CORE and CORE2 studies Continued early positive momentum with the launch of DAWNZERA™ (donidalorsen), the first RNA-targeted therapy for hereditary angioedema (HAE) Prescriptions written for all patient segments; growing number of repeat prescribers Expect European Medicines Agency (EMA) approval and launch in Q1 2026 Prescriptions written for all patient segments; growing number of repeat prescribers Expect European Medicines Agency (EMA) approval and launch in Q1 2026 Potential approval and launch of zilganersen for Alexander disease (AxD), the first and only investigational medicine to demonstrate clinically meaningful and disease-modifying impact U.S. Expanded Access Program (EAP) underway Planned NDA submission in Q1 2026 and launch later this year; received Breakthrough Therapy Designation First anticipated launch from Ionis owned neurology portfolio U.S. Expanded Access Program (EAP) underway Planned NDA submission in Q1 2026 and launch later this year; received Breakthrough Therapy Designation First anticipated launch from Ionis owned neurology portfolio Continued progress of Phase 3 REVEAL study of ION582, a promising investigational medicine for Angelman syndrome (AS) Expect to complete enrollment in 2026 with data expected in 2027 Expect to complete enrollment in 2026 with data expected in 2027 Multiple Phase 2 data readouts from neurology pipeline Partnered Programs Potential approval and launch of bepirovirsen, an Ionis-discovered investigational medicine for chronic hepatitis B (CHB) (GSK) Planned regulatory submissions to health authorities worldwide in 2026 based on positive topline results from pivotal Phase 3 B-Well 1 and B-Well 2 studies, in which bepirovirsen demonstrated a statistically significant and clinically meaningful functional cure rate Full data presentation and publication planned Planned regulatory submissions to health authorities worldwide in 2026 based on positive topline results from pivotal Phase 3 B-Well 1 and B-Well 2 studies, in which bepirovirsen demonstrated a statistically significant and clinically meaningful functional cure rate Full data presentation and publication planned Results from Phase 3 Lp(a) HORIZON cardiovascular outcomes study of pelacarsen, an Ionis-discovered investigational medicine for lipoprotein(a) (Lp(a))-driven cardiovascular disease (CVD) (Novartis) Results expected in H1 2026 Planned NDA submission Results expected in H1 2026 Planned NDA submission Results from Phase 3 CARDIO-TTRansform study of eplontersen, Ionis’ co-commercialized investigational medicine with AstraZeneca for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) Results expected in H2 2026 Planned sNDA submission Results expected in H2 2026 Planned sNDA submission Additional Phase 3 readouts from partnered programs: Results from IMAGINATION study of sefaxersen for IgA nephropathy (IgAN) (Roche) Results from FUSION study of ulefnersen for Fused in Sarcoma (FUS) Amyotrophic Lateral Sclerosis (ALS) (Otsuka) Results from IMAGINATION study of sefaxersen for IgA nephropathy (IgAN) (Roche) Results from FUSION study of ulefnersen for Fused in Sarcoma (FUS) Amyotrophic Lateral Sclerosis (ALS) (Otsuka) Multiple Phase 2 data readouts, including IONIS-MAPTRx (BIIB080) in Alzheimer’s disease (AD) (Biogen) and new Phase 3 clinical trial initiations *Indicates preliminary unaudited results. INDICATION for TRYNGOLZA® (olezarsen) TRYNGOLZA® (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur. ADVERSE REACTIONS The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia. Please see full Prescribing Information for TRYNGOLZA. INDICATION for DAWNZERA™ (donidalorsen) DAWNZERA (donidalorsen) is indicated for prophylaxis to prevent attacks of hereditary angioedema ( HAE) in adult and pediatric patients 12 years of age and older. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy. ADVERSE REACTIONS Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort. Please see full Prescribing Information for DAWNZERA. 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 and the therapeutic and commercial potential of TRYNGOLZA, DAWNZERA and additional medicines in development and technologies, and our expectations regarding development and regulatory milestones. Any statement describing Ionis' goals, expectations, financial or other projections or guidance, 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, 2024 , and most recent Form 10-Q, which are on file with the SEC . Copies of these and other documents are available at www.Ionis.com. Ionis Pharmaceuticals ® and TRYNGOLZA® are registered trademarks of Ionis Pharmaceuticals, Inc. DAWNZERA™ is a trademark of Ionis Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20260111381308/en/ Ionis Investor Contact: D. Wade Walke , Ph.D. IR@ionis.com 760-603-2331 Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679 Source: Ionis Pharmaceuticals, Inc.

DUBLIN and BRIDGEWATER, N.J., Jan. 09, 2026 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ: AMRN), a company advancing the science of cardiovascular therapeutics worldwide, today commented on recent innovations in therapies for patients with elevated triglycerides (TG) and shared its perspective on how these developments stand to shape patient access and treatment strategies. Specifically, new therapies for these patients are likely to expand the use of existing, proven options over time, including Amarin’s VASCEPA®/VAZKEPA® (icosapent ethyl), an effective, safe, oral therapy that has been prescribed more than 25 million times to patients globally. In addition, through its approved indication for severe hypertriglyceridemia (sHTG), which is defined as TG ≥500 mg/dL, as well as strong clinical evidence, affordability, and broad reimbursement, VASCEPA/VAZKEPA aligns with current payor-driven step therapy programs, which require patients to try existing safe and efficacious treatment options before newer, more expensive sHTG alternatives. Celebrating Scientific Advancement Amarin applauds the groundbreaking work addressing sHTG and rare genetic disorders such as familial chylomicronemia syndrome (FCS). These advances help drive patient-doctor interactions and bring critical attention to patients with very high triglyceride levels, which has been shown to increase risk for pancreatitis. Aaron Berg, President and CEO of Amarin, stated: “Innovative therapeutics coupled with related FDA breakthrough therapy designations, underscore the renewed focus on recognizing and treating the risks in patients with elevated triglycerides. VASCEPA has been approved in the US to reduce triglycerides in patients with sHTG. VASCEPA/VAZKEPA has also been approved for cardiovascular risk reduction (CVRR) in over 50 countries and recognized for CVRR by more than 70 global medical societies and is therefore uniquely positioned to complement our industry’s progress and deliver further meaningful impact to patients worldwide. “ VASCEPA/VAZKEPA: Significantly Reducing Cardiovascular Events in a Global Population of High-Risk Patients The emergence of new ApoC-III–targeted injectables is a major step forward for patients, delivering transformative benefits for those at highest risk. Yet, beyond this small group of sHTG patients, there remains an unmet need among tens of millions of cardiovascular disease (CVD) patients worldwide above 150 mg/dL i who urgently need proven therapies to reduce their risk of a cardiovascular event. VASCEPA is FDA approved as an adjunct to diet to reduce TG levels in adult patients with sHTG. VASCEPA is also approved as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization, and unstable angina requiring hospitalization in adult patients with elevated TG levels. Based on the landmark results of the Company’s cardiovascular outcomes trial, REDUCE-IT, published in 2018, which included 8,179 patients, relative to placebo VASCEPA demonstrated a 25% decrease in cardiovascular (CV) events when used as an adjunct to statin therapy in patients with elevated TG levels. ii While the effect of VASCEPA on the risk of pancreatitis in patients with sHTG has not been determined, data from the Company’s pivotal MARINE study in patients with TGs between 500 and 2,000 mg/dL, published in 2011, demonstrated that approximately 50% of those treated orally with VASCEPA 4g/day achieved TG levels below 500 mg/dL at week 12. iii , iv Importantly, the reduction in TG levels observed with VASCEPA in the MARINE study was not associated with elevations in low-density lipoprotein cholesterol (LDL-C) levels relative to placebo, unlike other older and newer TG-lowering agents. v , vi , vii Elevated LDL-C is a well-established major CV risk factor. Unlike other TG-lowering agents such as fibrates, niacin and mixed omega-3 products, which have failed in prior cardiovascular outcomes trials, and unlike newer TG-lowering agents targeting ApoC-III protein, which have not yet been studied for cardiovascular outcomes, VASCEPA has already demonstrated robust efficacy in reducing CV events when added on top of statin therapy in the REDUCE-IT cardiovascular outcomes study. In 2025, Amarin further expanded the potential global reach of its VASCEPA/VAZKEPA franchise by securing an exclusive long-term license and supply agreement with Recordati S.p.A. to commercialize VAZKEPA across 59 countries, focused on Europe where patent protection extends until 2039. VASCEPA/VAZKEPA is now commercially available in more than 20 countries. Payor Driven Step-Therapy Programs Likely to Result in Broader Use of VASCEPA/VAZKEPA to Treat sHTG U.S. payors have already implemented step-therapy programs for new ApoC-III injectables within the currently approved FCS indications. viii , ix This could result in increased utilization of existing drugs such as VASCEPA over time via their designation as a payor-preferred, first-line therapy prior to access to the newer, higher cost ApoC-III therapies. A relevant, recent comparison is the 2015 launch of premium priced PCSK9 inhibitors, during which U.S. payors implemented step-therapy requirements that drove significant growth for ezetimibe (also used to lower LDL or “bad” cholesterol). Despite a declining market in the years prior to PCSK9 inhibitors launch, over the past eight years, ezetimibe prescriptions have experienced sustained growth, averaging approximately 15% annually. x Taking into consideration both the PCSK9 inhibitors comparison and current step-therapy requirements for ApoC-III therapies, as treatable patient populations expand for ApoC-III therapies via future FDA approvals (e.g. sHTG), these financially driven payor requirements for ApoC-III therapies may increase demand and utilization of clinically proven, FDA-approved, cost-effective and broadly reimbursed agents, such as VASCEPA/VAZKEPA. About Amarin Amarin is a global pharmaceutical company committed to reducing the cardiovascular disease (CVD) burden for patients and communities and to advancing the science of cardiovascular health around the world. We own and support a global branded product approved by multiple regulatory authorities based on a track record of proven efficacy and safety and backed by robust clinical trial evidence. Our commercialization model includes a direct sales approach in the U.S. and an indirect distribution strategy internationally, through a syndicate of reputable and well-established partners with significant geographic expertise, covering close to 100 markets worldwide. Our success is driven by a dedicated, talented, and highly skilled team of experts passionate about the fight against the world’s leading cause of death, CVD. About VASCEPA®/VAZKEPA® (icosapent ethyl) Capsules VASCEPA (icosapent ethyl) capsules are the first prescription treatment approved by the U.S. Food and Drug Administration (FDA) comprised solely of the active ingredient, icosapent ethyl (IPE), a unique form of eicosapentaenoic acid. VASCEPA was launched in the United States in January 2020 as the first drug approved by the U.S. FDA for treatment of the studied high-risk patients with persistent cardiovascular risk despite being on statin therapy. VASCEPA was initially launched in the United States in 2013 based on the drug’s initial FDA approved indication for use as an adjunct therapy to diet to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. Since launch, VASCEPA has been prescribed more than twenty-five million times. VASCEPA is covered by most major medical insurance plans. In addition to the United States, VASCEPA is approved and sold in Canada, China, Australia, Lebanon, the United Arab Emirates, Saudi Arabia, Qatar, Bahrain, and Kuwait. In Europe, in March 2021 marketing authorization was granted to icosapent ethyl in the European Union for the reduction of risk of cardiovascular events in patients at high cardiovascular risk, under the brand name VAZKEPA. In April 2021 marketing authorization for VAZKEPA (icosapent ethyl) was granted in Great Britain (applying to England, Scotland and Wales). VAZKEPA (icosapent ethyl) is currently approved and sold in Europe in Sweden, Finland, England/Wales, Spain, Netherlands, Scotland, Greece, Portugal, Italy, Denmark and Austria. United States Indications and Limitation of Use VASCEPA is indicated: As an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (≥ 150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease. As an adjunct to diet to reduce TG levels in adult patients with severe (≥ 500 mg/dL) hypertriglyceridemia. The effect of VASCEPA on the risk for pancreatitis in patients with severe hypertriglyceridemia has not been determined. Important Safety Information VASCEPA is contraindicated in patients with known hypersensitivity (e.g., anaphylactic reaction) to VASCEPA or any of its components. VASCEPA was associated with an increased risk (3% vs 2%) of atrial fibrillation or atrial flutter requiring hospitalization in a double-blind, placebo-controlled trial. The incidence of atrial fibrillation was greater in patients with a previous history of atrial fibrillation or atrial flutter. It is not known whether patients with allergies to fish and/or shellfish are at an increased risk of an allergic reaction to VASCEPA. Patients with such allergies should discontinue VASCEPA if any reactions occur. VASCEPA was associated with an increased risk (12% vs 10%) of bleeding in a double-blind, placebo-controlled trial. The incidence of bleeding was greater in patients receiving concomitant antithrombotic medications, such as aspirin, clopidogrel, or warfarin. Common adverse reactions in the cardiovascular outcomes trial (incidence ≥3% and ≥1% more frequent than placebo): musculoskeletal pain (4% vs 3%), peripheral edema (7% vs 5%), constipation (5% vs 4%), gout (4% vs 3%), and atrial fibrillation (5% vs 4%). Common adverse reactions in the hypertriglyceridemia trials (incidence >1% more frequent than placebo): arthralgia (2% vs 1%) and oropharyngeal pain (1% vs 0.3%). Adverse events may be reported by calling 1-855-VASCEPA or the FDA at 1-800-FDA-1088. Patients receiving VASCEPA and concomitant anticoagulants and/or anti-platelet agents should be monitored for bleeding. FULL U.S. FDA-APPROVED VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT WWW.VASCEPA.COM . Europe For further information about the Summary of Product Characteristics (SmPC) for VAZKEPA ® in Europe, please visit: Globally, prescribing information varies; refer to the individual country product label for complete information. Forward-Looking Statements This press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including; our belief in the potential for the expanded use of VASCEPA/VAZKEPA and Amarin’s overall efforts to expand access and reimbursement to VAZKEPA across global markets; expectations regarding potential strategic collaboration and licensing agreements with third parties, including our ability to attract additional collaborators, as well as our plans and strategies for entering into potential strategic collaboration and licensing agreements and the overall potential and future success of VASCEPA/VAZKEPA and Amarin that are based on the beliefs and assumptions and information currently available to Amarin. All statements other than statements of historical fact contained in this press release are forward-looking statements. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in Amarin's filings with the U.S. Securities and Exchange Commission, including Amarin’s quarterly report on Form 10-Q for the period ending September 30, 2025 and annual report on Form 10-K for the fiscal year ended 2024. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. Amarin undertakes no obligation to update or revise the information contained in its forward-looking statements, whether as a result of new information, future events or circumstances or otherwise. Amarin’s forward-looking statements do not reflect the potential impact of significant transactions the company may enter into, such as mergers, acquisitions, dispositions, joint ventures or any material agreements that Amarin may enter into, amend or terminate. Investors and others should note that Amarin communicates with its investors and the public using the company website ( ), the investor relations website ( ) including but not limited to investor presentations and investor FAQs, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. Availability of Other Information About Amarin Amarin communicates with its investors and the public using the company website ( ) and the investor relations website ( investors.amarincorp.com ), including but not limited to investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Amarin posts on these channels and websites could be deemed to be material information. As a result, Amarin encourages investors, the media and others interested in Amarin to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Amarin’s investor relations website and may include social media channels. The contents of Amarin’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933. Amarin Contact Information Media Inquiries: Tegan Berry Amarin Corporation plc PR@amarincorp.com Investor Inquiries: Bob Burrows Western Avenue Advisers LLC Bob.burrows.ext@amarincorp.com Investor.relations@amarincorp.com i Fan W, Philip S, Granowitz C, Toth PP, Wong ND. Prevalence of US Adults with Triglycerides ≥ 150 mg/dl: NHANES 2007-2014. Cardiol Ther. 2020;9(1):207-213. ii Bhatt DL, Steg PG, Miller M, et al; REDUCE-IT Investigators. Cardiovascular risk reduction with icosapent ethyl for hypertriglyceridemia. N Engl J Med . 2019;380(1):11-22. doi:10.1056/NEJMoa1812792 iii Bays HE, et al. Am J Cardiol. 2011;108(5):682-90. doi: 10.1016/j.amjcard.2011.04.015. iv Chowdhury IN. Medical Review(s): Vascepa (Icosapent Ethyl) . Silver Spring, MD: US Food and Drug Administration; 2012:50. Application No. 202057Orig1s000. Accessed December 17, 2025. v vi vii Marston NA, Bergmark BA, Alexander VJ, et al.; CORE-TIMI 72a and CORE2-TIMI 72b Investigators. Olezarsen for managing severe hypertriglyceridemia and pancreatitis risk. N Engl J Med. 2025 Nov 8. doi: 10.1056/NEJMoa2512761. viii ix x Symphony METYS data on file. Accessed December 2025.