Ionis Pharmaceuticals Inc (IONS)

Ionis announces license agreement with Recordati for zilganersen in Alexander disease (AxD) in all countries outside the U.S.June 25, 2026 2:59 AM
Business Wire - Recordati brings deep rare disease expertise and proven capabilities in commercialization, access and country-specific regulatory environments - - Ionis to independently commercialize zilganersen in the U.S. assuming FDA approval - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Recordati (“Recordati”), under which Recordati obtained exclusive rights to develop and commercialize zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD), in all countries outside the U.S. Ionis will maintain sole commercial responsibility for zilganersen in the U.S. and will continue to lead development globally. Recordati will be responsible for regulatory filings and commercialization outside the U.S., including country-specific support for early access pathways based on local regulations and access dynamics. "Recordati combines proven rare disease and global commercialization expertise with deep experience navigating regional and local regulatory environments and the infrastructure needed to reach patients outside the United States, making them the right partner to deliver zilganersen to patients with urgency. They recognize, as we do, the high unmet need for this rare, serious and often fatal neurodegenerative disease,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. "Together, we are committed to working closely to help bring zilganersen to people living with Alexander disease outside the United States and enable broader access to this important treatment.” Ionis plans to independently launch zilganersen in the U.S, which is currently under FDA review with a Prescription Drug User Fee Act (PDUFA) action date of September 22. As the potential first and only disease modifying treatment for AxD, zilganersen would also mark Ionis’ first independent commercial launch in neurology, advancing the company’s goal of bringing transformational medicines to people with serious neurological diseases. Rob Koremans, Chief Executive Officer, Recordati, commented, “We are very pleased to partner with Ionis on the development and commercialization of zilganersen outside the United States. The addition of zilganersen further strengthens our Rare Diseases portfolio with a potentially transformative therapy for patients affected by Alexander disease, a devastating and progressive neurological disorder with significant unmet medical need. By combining Ionis' innovation and development expertise with Recordati's global rare disease capabilities, we believe we are well positioned to maximize the potential of zilganersen and bring a much-needed treatment option to patients with Alexander disease." After disclosing positive top-line results, Ionis recently announced additional positive data from the pivotal study of zilganersen, highlighting potential treatment benefit across multiple AxD symptom domains and reinforcing zilganersen’s positive impact on people living with AxD. The study met its primary endpoint in individuals ≥ 5 years of age, with zilganersen 50 mg demonstrating statistically significant and clinically meaningful stabilization of gait speed as assessed by the 10-Meter Walk Test (10MWT), a commonly used measure of gross motor function in neurologic disease, compared to control at Week 61. Secondary and exploratory endpoint results from patient/caregiver- and clinician-reported outcome assessments consistently favored zilganersen. Zilganersen demonstrated a favorable safety and tolerability profile, with most adverse events (AE) mild or moderate in severity. Serious treatment-emergent adverse events (TEAEs) occurred less frequently in the zilganersen group compared to control (37.5% zilganersen 25 mg or 50 mg; 47.1% pooled control). Under the terms of the agreement, Ionis receives an upfront payment of $30 million, and is eligible to receive additional payments based on achievement of milestones and a tiered royalty of up to the mid-20% range on annual net sales. About Zilganersen Zilganersen is an investigational antisense oligonucleotide medicine being evaluated as a treatment for people with Alexander disease (AxD). Zilganersen is designed to inhibit production of excess glial fibrillary acidic protein (GFAP) that accumulates because of disease-causing variants in the GFAP gene. The U.S. Food and Drug Administration (FDA) granted zilganersen Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations for AxD. In addition, the European Medicines Agency (EMA) granted zilganersen Orphan Drug designation for AxD. About Alexander Disease (AxD) AxD is a rare, progressive and often fatal neurological disease that occurs in approximately 1 per 1 to 3 million people worldwide and affects a type of cell in the brain called astrocytes. Astrocytes have multiple roles in the brain to support neurons and oligodendrocytes, including maintenance of the myelin sheath that protects nerve fibers. AxD is caused by disease-causing variants in the glial fibrillary acidic protein (GFAP) gene and is generally characterized by progressive neurological deterioration resulting in loss of functional mobility, loss of independence and the inability to control muscles for large movements, swallowing and airway protection, though symptoms can vary depending on age of onset. AxD usually leads to death within 14-25 years after symptom onset. There are no approved disease-modifying medicines. About Ionis Neurology Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA® (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 13 investigational medicines, of which six are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Alexander disease, Angelman syndrome, prion disease, multiple system atrophy and Huntington’s disease, as well as more common conditions such as Alzheimer's disease. About Ionis Pharmaceuticals, Inc. For more than 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 zilganersen, our commercial medicines, 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® is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUA® is a registered trademark of the AstraZeneca group of companies. View source version on businesswire.com: https://www.businesswire.com/news/home/20260624743930/en/ Ionis Investor Contact:
D. Wade Walke, Ph.D.

TRYNGOLZA® (olezarsen) approved by the FDA as the first and only treatment to reduce triglycerides and the risk of acute pancreatitis in patients with severe hypertriglyceridemia (sHTG)June 24, 2026 2:37 PM
Business Wire – Proven to deliver significant and robust triglyceride reductions – – Only treatment for sHTG indicated to reduce the risk of acute pancreatitis – – Ionis’ first independent commercial launch in a prevalent condition – – Ionis to host webcast today at 3:30 p.m. ET – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA® (olezarsen) as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG greater than or equal to 500 mg/dL). TRYNGOLZA is available in a 50 mg or 80 mg dose and is self-administered once monthly via an autoinjector. sHTG is characterized by an increased risk of acute pancreatitis, which causes debilitating abdominal pain that often leads to repeated and prolonged hospitalization, permanent organ damage and can be life-threatening. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260624119051/en/TRYNGOLZA (olezarsen) product image “The approval of TRYNGOLZA marks an historic advance for people who have long struggled to control their dangerously high triglycerides, providing the only approved therapy for sHTG to dramatically lower triglyceride levels and significantly reduce acute pancreatitis events,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “TRYNGOLZA reflects the strength of Ionis’ innovative science and our commitment to transforming patients’ lives. As our first independent launch in a prevalent disease, this milestone builds on our success in familial chylomicronemia syndrome, a rare form of sHTG, and marks a defining moment for Ionis as we bring our groundbreaking medicines to even more patients in need. We are deeply grateful to the clinical trial participants, investigators, the Ionis team and many others whose dedication made this achievement possible.” “As a physician, I have seen firsthand how challenging it can be for patients with sHTG to lower their triglycerides below 500 mg/dL, despite background lipid-lowering therapies and lifestyle changes, which leaves them at risk of a devastating and potentially life-threatening acute pancreatitis attack,” said Archna Bajaj, M.D., assistant professor of clinical medicine, University of Pennsylvania. “TRYNGOLZA is a transformational new therapy that showed unprecedented, clinically meaningful outcomes for sHTG, with the potential to redefine the treatment paradigm.” The FDA approval was based on positive results from the Phase 3 CORE and CORE2 studies, which were published in The New England Journal of Medicine. In the CORE and CORE2 studies, TRYNGOLZA demonstrated rapid and consistent triglyceride control, lowering fasting triglyceride levels by up to 72% compared to placebo at six months and sustaining those reductions at 12 months. Additionally, TRYNGOLZA significantly reduced acute pancreatitis events by up to 91%. Among patients treated with TRYNGOLZA with baseline and 12-month data, 86% achieved triglyceride levels below 500 mg/dL, a critical threshold for reducing acute pancreatitis risk. The number needed to treat (NNT) over one year to prevent one episode of acute pancreatitis was 20 in the overall cohort and four in patients with triglycerides ≥880 mg/dL and a prior history of acute pancreatitis, indicating a strong clinical benefit across the spectrum of sHTG patients and an exceptional clinical benefit in the highest risk subgroup.1 Across the clinical program, TRYNGOLZA demonstrated a favorable safety and tolerability profile. The most common adverse reactions in patients with sHTG (incidence ≥2% higher than placebo) were injection site reactions and liver enzyme increases. "With limited options to lower triglycerides, people living with sHTG often face a constant and real fear that a debilitating acute pancreatitis attack could strike at any time without warning,” said Emily Draud, interim executive director, National Pancreas Foundation. “The availability of TRYNGOLZA for sHTG represents an important new option for this community, offering hope for people who have been waiting for a new treatment to reduce the risk of acute pancreatitis by significantly lowering their triglyceride levels. It also underscores the urgent need for continued innovation and improved care for patients living with this serious condition." Ionis is committed to helping people access the medicines they are prescribed and will offer a full suite of services for people prescribed TRYNGOLZA through Ionis Every Step™. Ionis Every Step offers personal support, including nutrition information and injection training, insurance support and financial assistance programs. Visit TRYNGOLZA.com for more information. TRYNGOLZA will be available for sHTG in the U.S. in July. Webcast Ionis will hold a webcast today at 3:30 p.m. ET to discuss the FDA approval. Interested parties may access the webcast here. A webcast replay will be available for a limited time. INDICATIONS
Familial Chylomicronemia Syndrome (FCS)
TRYNGOLZA (olezarsen) is indicated as an adjunct to diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS). Severe Hypertriglyceridemia (sHTG)
TRYNGOLZA is indicated as an adjunct to diet to reduce TG and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL). 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. Liver Enzyme Abnormalities
TRYNGOLZA can cause increases in liver enzymes and hepatic fat in adults. Increases in liver enzymes were more frequently reported with the 80 mg dose as compared with the 50 mg dose. Consider liver enzyme testing before TRYNGOLZA initiation or an increase in dosage and when clinically indicated thereafter. If persistent elevations in liver enzymes occur, consider dose interruption and/or dose reduction. If serious hepatic injury with clinical symptoms and/or hyperbilirubinemia or jaundice occurs, promptly discontinue TRYNGOLZA. ADVERSE REACTIONS
Adverse Reactions for FCS
Most common adverse reactions in patients with FCS (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count, and arthralgia. Adverse Reactions for sHTG
Most common adverse reactions in patients with sHTG (incidence ≥2% higher than placebo) were injection site reactions and liver enzyme increases. Please see full Prescribing Information for TRYNGOLZA. Please see Important Safety Information throughout and full Prescribing Information for TRYNGOLZA. About the CORE and CORE2 Studies CORE (NCT05079919; n=617) and CORE2 (NCT05552326; n=446), conducted with The TIMI Study Group, are Phase 3 global, multicenter, randomized, double-blind, placebo-controlled trials investigating the safety and efficacy of olezarsen for severe hypertriglyceridemia (sHTG). Participants aged 18 and older with triglyceride levels ≥500 mg/dL were enrolled. Participants were required to be on standard of care therapies for elevated triglycerides. At baseline, 47% and 37% of participants had fasting triglycerides ≥880 mg/dL in CORE and CORE2, respectively. Participants were randomized to receive 50 mg or 80 mg of olezarsen or placebo every four weeks via subcutaneous injection for 12 months. The CORE and CORE2 studies met the primary endpoint, with both 50 mg and 80 mg monthly TRYNGOLZA doses. TRYNGOLZA demonstrated a statistically significant reduction in fasting triglycerides of 49%-63% (50 mg) and 55%-72% (80 mg) compared to placebo at six months. Additionally, TRYNGOLZA demonstrated a statistically significant 91% (50 mg) and 76% (80 mg) reduction in acute pancreatitis events at 12 months, with a pooled reduction of 85%. 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 TRYNGOLZA® (olezarsen) TRYNGOLZA® (olezarsen) is an RNA-targeted therapy designed to lower the body's production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. TRYNGOLZA is approved in the United States as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG greater than or equal to 500 mg/dL). TRYNGOLZA is also approved in the United States, European Union and other countries for adults with familial chylomicronemia syndrome (FCS), a rare form of sHTG. Visit TRYNGOLZA.com for more information. 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, Ionis' technologies and other products in development. 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. _____________  1 What are poor, acceptable, and great Number Needed to Treat (NNT) figures? Dr. Oracle. AI. Updated December 13, 2025. Accessed May 8, 2026. https://www.droracle.ai/articles/612269/what-are-poor-acceptable-and-great-number-needed-to   View source version on businesswire.com: https://www.businesswire.com/news/home/20260624119051/en/ Ionis Investor Contact:
D. Wade Walke, Ph.D.

Ionis partner GSK announces bepirovirsen achieves unprecedented functional cure rates with potential to redefine treatment for chronic hepatitis BMay 28, 2026 4:45 AM
Business Wire – Pivotal B-Well data show significant 19% functional cure rate in the overall study population and 26% in patients with lower viral activity, compared to 0% with standard of care – – 49% of bepirovirsen recipients achieved a surface antigen level of ≤100 IU/mL one year after end of treatment in exploratory analysis – – A loss in surface antigen is associated with a significant reduction in risk of liver cancer – – Over 240 million people worldwide live with chronic hepatitis B – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) partner GSK today announced positive pivotal data for bepirovirsen, an investigational antisense oligonucleotide (ASO) for the treatment of chronic hepatitis B (CHB). Results from the two Phase 3 trials, B-Well 1 and B-Well 2, were simultaneously published in the New England Journal of Medicine and presented at the European Association for the Study of the Liver (EASL) congress. GSK licensed bepirovirsen from Ionis and the two companies have collaborated on its development. Pooled data from both trials showed that six-month treatment with bepirovirsen achieved a statistically significant and clinically meaningful 19% functional cure response rate (233 of 1,220 vs. 0 of 614 in the placebo group, with p

Ionis partner Biogen announces topline results from Phase 2 CELIA study of diranersen (BIIB080): first study to show reduction in tau pathology and cognitive benefit in patients with early Alzheimer’s diseaseMay 14, 2026 7:05 AM
Business Wire - Based on the strength of the biomarker and efficacy data, Biogen plans to advance diranersen to registrational development; CELIA did not meet its primary endpoint assessing dose response - - Robust reductions in tau pathology were observed across all studied doses, with results generally consistent with those observed in the Phase 1b study - - Pre-specified analyses of cognitive endpoints demonstrated slowing of clinical decline across all studied doses, particularly at the lowest dose - - The safety and tolerability profile of diranersen was generally consistent with the Phase 1b study - - Data will be presented at the Alzheimer’s Association International Conference (AAIC) 2026 and other upcoming scientific congresses - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that its partner, Biogen, shared compelling topline results from the Phase 2 CELIA study evaluating diranersen (IONIS-MAPTRx/BIIB080), an investigational antisense oligonucleotide (ASO) therapy targeting tau, in individuals with early Alzheimer’s disease (AD). The CELIA results provide the first evidence from a randomized Phase 2 study of a tau-directed therapy demonstrating both robust biomarker impact and cognitive benefit in early AD. “We are highly encouraged by the topline results from CELIA, which underscore the potential of targeting tau to meaningfully impact patient outcomes in early Alzheimer’s disease,” Holly Kordasiewicz, Ph.D., executive vice president, chief development officer, Ionis. “These findings represent an important advancement for the field and underscore the impact of Ionis’ growing neurology portfolio, which now includes 13 medicines in clinical development including six that are wholly owned. We’re proud to have discovered diranersen and are deeply grateful to everyone who made this research possible.” Pre-specified analyses of cognitive endpoints demonstrated slowing of clinical decline across all studied doses, particularly in participants receiving the lowest dose of diranersen, 60 mg administered every 24 weeks. Diranersen also demonstrated robust reductions in both cerebrospinal fluid (CSF) tau and tau pathology, as measured by positron emission tomography (PET), across all studied doses, with reductions maintained throughout the dosing period. CELIA did not meet its primary endpoint assessing dose response for change from baseline on the Clinical Dementia Rating–Sum of Boxes (CDR-SB) at Week 76. “In CELIA, we believe we have seen an unprecedented and compelling confluence of efficacy and biomarkers results from a tau-directed agent in a randomized early Alzheimer’s disease study,” said Priya Singhal, M.D., M.P.H., Executive Vice President and Head of Development at Biogen. “We are excited by these Phase 2 data, which give us the confidence to advance diranersen to registrational development. We look forward to engaging with regulators and the broader Alzheimer’s disease community on next steps. I would like to thank the patients, families, investigators, and study teams who participated in this pioneering study.” The safety and tolerability profile of diranersen across all studied doses was generally consistent with the Phase 1b study and the known profile of diranersen to date. The incidence of adverse events (AEs) was comparable across dose groups, with a higher incidence of serious adverse events (SAEs) observed at the highest dose studied. CELIA is a pioneering study evaluating diranersen, a first-in-class investigational ASO designed to reduce the production of tau protein at its source in early AD. While tau plays an important role in the normal function of brain cells, in AD, abnormal tau can accumulate and form intracellular tangles that contribute to neurodegeneration and cognitive decline. Unlike many investigational approaches that have focused on targeting extracellular tau, diranersen is designed to reduce both extracellular and intracellular tau. About diranersen (IONIS-MAPTRx/BIIB080) Diranersen (IONIS-MAPTRx/BIIB080) is an investigational antisense oligonucleotide (ASO) therapy designed to target microtubule-associated protein tau (MAPT) mRNA to reduce the production of tau protein. Abnormal accumulation of tau in the brain is a hallmark of Alzheimer’s disease (AD) associated with neurodegeneration and cognitive decline. Diranersen is being investigated as a potential treatment for early AD. In 2025, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to diranersen for the treatment of AD. In December 2019, Biogen exercised a license option with Ionis Pharmaceuticals and obtained a worldwide, exclusive, royalty-bearing license to develop and commercialize diranersen. Diranersen was discovered by Ionis. About the CELIA Study CELIA is a global Phase 2 randomized, double-blind, placebo-controlled, dose-ranging study evaluating the efficacy, safety and tolerability of diranersen in individuals with early Alzheimer’s disease (AD). The study enrolled 416 participants with mild cognitive impairment due to AD or mild AD dementia. All participants enrolled in CELIA had not previously received anti-amyloid therapy. The study evaluated three doses of diranersen administered intrathecally over a 76-week placebo-controlled treatment period: 60 mg every 24 weeks, 115 mg every 24 weeks and 115 mg every 12 weeks. The primary endpoint of CELIA was assessment of dose response for change from baseline on the Clinical Dementia Rating–Sum of Boxes (CDR-SB) at Week 76. Secondary and exploratory endpoints included additional clinical, biomarker and imaging measures, including cerebrospinal fluid tau biomarkers and tau positron emission tomography (PET). Additional information on the CELIA study design is available in the ClinicalTrials.gov listing for the CELIA study. An ongoing long-term extension (LTE) study is continuing to evaluate the long-term safety, tolerability and durability of diranersen in early AD. About Ionis Neurology Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA® (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 13 investigational medicines, of which six are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Angelman syndrome, prion disease, multiple system atrophy, Huntington’s disease and Alexander disease, as well as more common conditions such as Alzheimer's disease. 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 diranersen (IONIS-MAPTRx/BIIB080), our commercial medicines, 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 and most recent Form 10-Q for the year ended December 31, 2025, 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® is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUA® is a registered trademark of the AstraZeneca group of companies. View source version on businesswire.com: https://www.businesswire.com/news/home/20260514986673/en/ Ionis Investor Contact:
D. Wade Walke, Ph.D.

Ionis to host 2026 virtual Annual Meeting of StockholdersMay 4, 2026 7:05 AM
Business Wire
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2026 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 4, 2026.


The agenda for the event is as follows:



5:00 p.m. – 5:15 p.m. ET (2:00 p.m. – 2:15 p.m. PT) – Virtual Annual Meeting of Stockholders


All stockholders of record at the close of business on April 7, 2026, are invited to participate in the virtual Annual Meeting webcast, which will be broadcast live at www.virtualshareholdermeeting.com/IONS2026.



Stockholders of record will receive an official proxy card or notice from their brokerage firm. Each proxy card/notice contains a 16-digit control number required to log-in, vote and submit questions during the webcast.



Ionis does not provide proxy cards or have access to proxy card information, including 16-digit control numbers. For help obtaining a proxy card, locating your control number or for instructions to access the webcast, stockholders of record should contact their brokerage firm ahead of the meeting.



A help line will be available on the registration page during the live webcast for participants requiring technical assistance to access or participate in the live event. There will not be a replay of this session.






5:30 p.m. ET (2:30 p.m. PT) – Virtual corporate update


Brett P. Monia, Ph.D., Ionis’ chief executive officer, will provide a general corporate update.



The presentation will be followed by a question-and-answer session. Instructions on how to submit questions will be shared at the beginning of the session.



All interested parties may access the webcast live at https://event.webcasts.com/starthere.jsp?ei=1753849&tp_key=89269291e9. An archived replay of the webcast will be posted for a limited time following the meeting at that same address.






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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260504500537/en/
Ionis Pharmaceuticals Investor Contact:

D. Wade Walke, Ph.D. –

Ionis to present at upcoming investor conferencesMay 1, 2026 7:05 AM
Business Wire
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences:



Bank of America Securities 2026 Health Care Conference on Tuesday, May 12, 2026



2026 RBC Capital Markets Global Healthcare Conference on Tuesday, May 19, 2026



Goldman Sachs 47th Annual Global Healthcare Conference on Wednesday, June 10, 2026



A live webcast of these presentations can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events. Replays will be available on the Ionis website within 48 hours and will be archived for a limited time.


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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260501002318/en/
Ionis Pharmaceuticals Investor Contact:

D. Wade Walke, Ph.D. –

Ionis partner GSK announces bepirovirsen accepted for Priority Review and granted Breakthrough Therapy Designation by U.S. FDA as a potential first-in-class medicine for chronic hepatitis BApril 28, 2026 2:06 AM
Business Wire
– Regulatory submission supported by Phase 3 B-Well trials demonstrating statistically significant and clinically meaningful functional cure rates in chronic hepatitis B -


– Breakthrough Therapy designation added to Fast Track Designation, recognizing potential for substantial improvement over existing treatments –


– Chronic hepatitis B is a leading cause of liver cancer globally –


– PDUFA Date of October 26, 2026 –


Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) partner GSK today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for bepirovirsen, an investigational antisense oligonucleotide (ASO) for the treatment of adults with chronic hepatitis B (CHB). The FDA has granted bepirovirsen Breakthrough Therapy designation and set a Prescription Drug User Fee Act (PDUFA) target action date of October 26, 2026.


“Over one million people in the U.S. are living with chronic hepatitis B and as of today, require lifelong antiviral therapy to suppress the virus,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “As the first medicine to deliver clinically meaningful functional cure rates, bepirovirsen is uniquely positioned to effectively treat CHB based on its potential to reduce the replication of hepatitis B virus, suppress hepatitis B surface antigen and stimulate the immune system. This milestone reflects the broad impact of Ionis’ science, and we look forward to the potential for bepirovirsen to help millions of people living with this devastating condition around the world.”


Breakthrough Therapy designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown preliminary clinical evidence indicating the potential for substantial improvement over available therapies. The designation enables greater FDA guidance on a medicine’s development program. Priority Review designation is granted to marketing applications for medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention or diagnosis of a serious condition, with the expectation of the FDA taking action within six months, compared to 10 months under standard review. Bepirovirsen also received Fast Track designation from the U.S. FDA in February 2024.


CHB is a major public health challenge, affecting over 250 million people worldwide and 1.7 million in the U.S. The current standard of care, typically nucleos(t)ide analogues, often requires lifelong therapy and the functional cure rates remain low, typically only 1%. Functional cure occurs when hepatitis B virus DNA and viral protein, hepatitis B surface antigen (HBsAg), are undetectable in the blood for at least 24 weeks after stopping all treatment, indicating that the disease can be controlled by the immune system without medication. Achieving functional cure is associated with significant reduction in the risk of long-term liver complications, including liver cancer.


The regulatory submission and Breakthrough Therapy designation were supported by positive results from the Phase 3 B-Well 1 and B-Well 2 trials, where bepirovirsen demonstrated statistically significant and clinically meaningful functional cure rates. Functional cure rates were significantly higher with bepirovirsen plus standard of care compared to standard of care alone across all ranked endpoints, including in patients with lower baseline HBsAg levels, where an even greater effect was observed. Bepirovirsen demonstrated an acceptable safety and tolerability profile consistent with previous studies. The data will be presented at the European Association for the Study of the Liver (EASL) 2026 Congress in May and submitted for simultaneous publication in scientific peer-reviewed journal in 2026.


GSK licensed bepirovirsen from Ionis in 2019 under a collaborative development and licensing agreement. Under the terms of the agreement, Ionis has received an upfront payment, license fee and development and regulatory milestone payments and is eligible to receive additional regulatory and sales milestone payments as well as tiered royalties of 10-12% on net sales of bepirovirsen.


Bepirovirsen is currently under regulatory review with the European Medicines Agency (EMA), the China National Medical Products Administration (NMPA) and Japan’s Ministry of Health, Labour and Welfare (MHLW). Bepirovirsen was also awarded Breakthrough Therapy Designation in China and SENKU Designation in Japan.


About B-Well 1 and B-Well 2


B-Well 1 (NCT05630807) and B-Well 2 (NCT05630820) trials are global multi-center, randomized, double-blind, placebo-controlled trials conducted in 29 countries. They assessed the efficacy, safety, pharmacokinetic profile, and the durability of functional cure in nucleos(t)ide analogue (NA)-treated participants with chronic hepatitis B and baseline surface antigen (HBsAg) ≤3000 IU/ml. The primary endpoint assessed the proportion of participants achieving functional cure in patients with baseline HBsAg ≤3000 IU/ml. A key secondary endpoint evaluated functional cure in participants with baseline HBsAg ≤1000 IU/ml. Functional cure is defined as HBsAg being undetectable in the blood for at least 24 weeks after stopping all treatment, indicating that the disease is controlled by the immune system without medication.


About Chronic Hepatitis B (CHB)


Hepatitis B is a viral infection that can cause both acute and chronic liver disease. Chronic hepatitis B occurs when the immune system is unable to clear the virus, resulting in long-lasting infection that affects more than 250 million people worldwide. The disease causes approximately 1.1 million deaths each year globally. Many patients often require lifelong antiviral therapy for viral suppression; making functional cure a critical goal in disease management.


About Bepirovirsen


Bepirovirsen is an investigational antisense oligonucleotide (ASO) designed to recognize and inhibit the production of the genetic components (i.e. RNA) of the hepatitis B virus that can lead to chronic disease, potentially allowing a person’s immune system to regain control. Bepirovirsen reduces the production of RNA and viral proteins associated with HBV, suppresses the level of hepatitis B surface antigen (HBsAg) in the blood, and stimulates the immune system to increase the chances of a durable and sustained response.


About Ionis Pharmaceuticals, Inc.


For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has marketed medicines and a leading pipeline in neurology, cardiometabolic 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 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 bepirovirsen, our commercial medicines, 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® is a trademark of Ionis Pharmaceuticals, Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260427721143/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


Original: Ionis partner GSK announces bepirovirsen accepted for Priority Review and granted Breakthrough Therapy Designation by U.S. FDA as a potential first-in-class medicine for chronic hepatitis B

Ionis to hold first quarter 2026 financial results webcastApril 15, 2026 7:05 AM
Business Wire
Webcast scheduled for Wednesday, April 29 at 8:30 a.m. Eastern Time


Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, April 29 at 8:30 a.m. Eastern Time to discuss its first quarter 2026 financial results and highlight progress on key programs.


The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay will be available for a limited time at the same address.


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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260415423973/en/
Ionis Investor Contact:

D. Wade Walke, Ph.D. –

Ionis to present at upcoming investor conferencesFebruary 19, 2026 7:05 AM
Business Wire
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences:



Oppenheimer 36th Annual Healthcare Life Sciences Virtual Conference on Thursday, February 26, 2026



TD Cowen 46th Annual Health Care Conference on Monday, March 2, 2026



Leerink Global Healthcare Conference on Monday, March 9, 2026



Barclays 28th Annual Global Healthcare Conference on Wednesday, March 11, 2026



Stifel 2026 Virtual CNS Forum on Tuesday, March 17, 2026



A live webcast of these presentations can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events. Replays will be available on the Ionis website within 48 hours and will be archived for a limited time.


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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260219233764/en/
Ionis Pharmaceuticals Investor Contact:

D. Wade Walke, Ph.D. –

Ionis updates time for fourth quarter and full year 2025 financial results webcastFebruary 17, 2026 2:58 PM
Business Wire
Webcast scheduled for Wednesday, February 25 at 8:30 a.m. Eastern Time


Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced a change in the start time for its fourth quarter and full year 2025 financial results webcast.


The live webcast, previously scheduled for 11:30 a.m. Eastern Time, will now be held at 8:30 a.m. Eastern Time on Wednesday, February 25. The date of the call remains unchanged.


The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay will be available for a limited time at the same address.


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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260217628310/en/
Ionis Investor Contact:

D. Wade Walke, Ph.D. –

Ionis to hold fourth quarter and full year 2025 financial results webcastFebruary 11, 2026 7:05 AM
Business Wire
Webcast scheduled for Wednesday, February 25 at 11:30 a.m. Eastern Time


Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 25 at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2025 financial results and highlight progress on key programs.


The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay will be available for a limited time at the same address.


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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260211190610/en/
Ionis Investor Contact:

D. Wade Walke, Ph.D. –

IONS 10Q due 4/30

IONS 10 Q due 2/19

https://www.reuters.com/business/healthcare-pharmaceuticals/lilly-pill-cuts-genetic-form-cholesterol-nearly-86-study-2024-11-18/?

Oops its a vice of mine not to give context...
So what I understood is special about Ionis compounds is that we can treat the bad LDL cholesterol and triglycerides for heart health but this LPA is genetically fixed and cannot reliably be lowered right now.
So this company can do shady business all they want if the results are right and the Novartis partnership hints at that.
I remember some analyst said that the medical societies will quickly get behind screening and treating it if their trials read out positively.

high?
High levels of lipoprotein (a) (Lp(a), pronounced “L-P-little-A”) increase your likelihood of having a heart attack, a stroke, and aortic stenosis, especially if you have familial hypercholesterolemia (FH) or signs of coronary heart disease.May

Making new lows

the Olezarsen/TRYNGOLZA NEJM paper claimed there were ZERO adverse events — perhaps a transient platelet decrease
Later the FDA label reveal 5 such events (12% of patients) in the same study.

All that will be forgotten quickly should the IONS / Novartis ph3 read out positively. Could be the only compound on the market for a while that can significantly lower lipoprotein a.
Lipoprotein a lowering might be one of the big biopharm stories in 2025.

IONS that awful Cathie Wood has been a recent buyer

IONS new 52 week high

IONS new 52 week high

$IONS short squeeze Ionis Pharmaceuticals Inc Nasdaq Ions Short Squeeze

New alert out https://msmoneymoves.com/2019/04/23/investor-alert-upcoming-earnings-and-catalyst/

What a beast. Just keeps going up

57 made in the shade. A little pause to regroup and popping 60. Shake, Rattle and Roll just watch the reaction to when this presentation hits the streets:
https://finance.yahoo.com/news/first-class-therapies-using-advanced-120000967.html

Headed to 57 I feel it in my bones!

Ready to Rocket!

Jimmy:
The FDA is seriously underfunded and being pulled in different directions by an ignorant public, ignorant legislators, big money lobbies, and loads of big pharma money.They are struggling.
They do the best they can.
The antisense platform is valid and has some FDA approvals and cured folks as proof.
The FDA is afraid of risk, even in the cases of folks with fatal diseases. The liability attorneys flock like hungry vultures.

The advisory committee recommended approval. A very small number of patents with deadly disease with no other options and the FDA issued a CRL instead of approval. Patients die short sellers get rich. What is wrong with the FDA? Corruption? Or is the Anti-sense platform a fraud? Get a rope. But who should be strung up IONS or the FDA? Right now it is the patients and long stock holders feeling the pain.

Exicure to Present Data at the Cure Spinal Muscular Atrophy Annual Conference in Dallas

Exicure’s three-dimensional spherical nucleic acid containing the nusinersen sequence prolongs survival and reduces toxicity compared to nusinersen in a spinal muscular atrophy mouse model

SKOKIE, Ill. – June 14, 2018 – Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA™) constructs, announced today that Exicure and its collaborators at The Ohio State University Wexner Medical Center will show preclinical data demonstrating the performance of Exicure’s SNA compound designed for use in spinal muscular atrophy (SMA). These data will be presented at the Cure SMA Annual Conference in Dallas, Texas on June 14, 2018.

“Exicure’s spherical nucleic acid version of nusinersen demonstrates increased survival and decreased toxicity in the translationally-relevant SMA mouse model,” said David Giljohann, PhD, CEO of Exicure. “We believe these results are important for developing improved treatments for patients with SMA. These data also suggest that Exicure’s technology platform could potentially create more potent therapies for other disorders of the central nervous system, including Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.”

At the meeting, Arthur Burghes, PhD, from Ohio State’s Wexner Medical Center, and Exicure will present data from preclinical studies in a SMA mouse model. The poster is titled “Nusinersen in spherical nucleic acid (SNA) format improves efficacy both in vitro in SMA patient fibroblasts and in ?7 SMA mice and reduces toxicity in mice.” The presentation will highlight that Exicure’s proprietary technology:

Prolonged survival by four-fold (maximal survival of 115 days compared to 28 days for nusinersen-treated mice);

Doubled the levels of healthy full-length SMN2 mRNA and protein in SMA patient fibroblasts when compared to nusinersen;

Doubled the quantity of healthy full-length SMN mRNA levels in spinal cord tissue compared to untreated mice;

Mitigated toxicity of nusinersen at the highest dose tested in mice.

In August 2017, Exicure and The Ohio State University established a collaboration to further validate and characterize the pharmacology of Exicure’s nusinersen-SNA compound in mouse models. This collaboration’s ongoing in vivo research is conducted by Dr. Burghes, an internationally known researcher, leading basic and clinical research on SMA and other genetic neuromuscular disorders.

About Exicure, Inc.
Exicure, Inc. is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure's proprietary 3-dimensional, spherical nucleic acid (SNA™) architecture unlocks the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address oncology, inflammatory diseases and genetic disorders. Exicure is based outside of Chicago, IL. For more information, please visit www.exicuretx.com

About Spinal Muscular Atrophy (SMA)
SMA is the most common genetic cause of death for infants. SMA results from the loss of the SMN1 gene and an inability of SMN2 to produce sufficient full-length protein to make up for the loss of SMN1. The SMN1 gene, in a healthy person, produces a full-length protein that is essential to the function of the nerves that control muscles. Without sufficient SMN protein, the nerve cells cannot properly function and eventually die. This leads to debilitating and even fatal muscle weakness.

About Nusinersen
Nusinersen, marketed as Spinraza® by Biogen, is a modified antisense oligonucleotide. In December of 2016, nusinersen was approved by the US FDA for the treatment of SMA in pediatric and adult patients.

Forward Looking Statements
This press release contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning the Company, the Company's technology, potential therapies, pre-clinical results, and other matters. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "plan," "believe," "intend," "look forward," and other similar expressions among others. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: that Exicure's pre-clinical programs do not advance into the clinic or result in approved products on a timely or cost effective basis or at all; regulatory developments; and the ability of Exicure to obtain sufficient funding for its programs and to protect its intellectual property rights. Exicure's pipeline programs are in various stages of pre-clinical and clinical development, and the process by which such pre-clinical or clinical therapeutic candidates could potentially lead to an approved therapeutic is long and subject to significant risks and uncertainties. Risks facing the Company and its programs are set forth in the Company's filings with the SEC. Except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Media Contact
Karen Sharma
ksharma@macbiocom.com
781-235-3060

So our new drug that gives a small population of doomed children a chance at life, only sold about as many doses as last quarter.
Everyone knew it was a small patient population and another important test of the success of our anti-sense platform.
As a reward, the SP plummets 10%!..A genuinely chicken-ship reaction from the market

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS): Stochastic Analysis

Technical Indicators and Signals

The stochastic oscillator indicator is the important indicator to compare the security price over a certain period of time. The stochastic %K of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) for the period of 9-Day stands at 91.68% while the stochastic %D for the same period stands at 90.12%.

The 7-Day Average Directional Index ADX (also known as Trend Strength Indicator) of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) signals “Buy” while other important signals like the 20-Day Moving Average Vs. Price signals “Buy”.

Another important signal of trend and momentum of a security is 20-50 Day MACD Oscillator or Moving Average Convergence Divergence signals “Buy” for Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) while 20-100 Day MACD Oscillator
“Buy”.

http://www.stocksmarketnews.com/ionis-pharmaceuticals-inc-nasdaqions-stochastic-analysis/

So, let's see....
IONS announces a new clinical trial series for a drug that might command enormous , unserved markets;
Collects a fat wad of dollars,
and the PPS sinks over # 3%.
Riiiight! Rational market indeed.

"CARLSBAD, Calif., Oct. 13, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that it has initiated a Phase 1/2a clinical study of IONIS-MAPTRx in patients with mild Alzheimer's disease (AD). Ionis earned a $10 million milestone payment from Biogen related to the initiation of this study."

Thanks Capecod.

If you want to be on a large board for ionis go to investor village and start posting on the public board there for ionis and then after a while ask if they will let you onto the private board. They let some people on and not others. You would be surprised how much posting they have on the private board and even the public one there has more than here.

That would explain the paucity of postings.
Please let me know if you find one Capecod.
( i'm not far from Big Sur on the other side of the continent.)

Is there a private board on this platform that has more ionis followers? There are a large number of followers and daily postings on a private board at investor village. Would like to join a similar board if there is one on this platform.

And between us we darn near own this board..grin.

I like this company too.

Thanks for the updates Thoro;
Ions has done quite nicely for me over the long run,
and with their promisingly fat pipeline
i'm sure to let it ride.

IONS, AKCA, Akcea and Ionis Announce Submission of Marketing Authorization Application for Volanesorsen to the European Medicines Agency

https://ih.advfn.com/p.php?pid=nmona&article=75321782

Anyone think this will be over $70 by the first of the year?

Ionis Pharmaceuticals reports phase 1/2 clinical study results with AKCEA-ANGPTL3-L

http://www.reuters.com/article/brief-ionis-pharmaceuticals-reports-phas-idUSFWN1IR0BI

Ionis Pharmaceuticals presents new data at peripheral nerve society meeting to support potential benefit of Inotersen

IONS

http://www.reuters.com/article/brief-ionis-receives-10-mln-milestone-pa-idUSASB0B97F

Ionis receives $10 mln milestone payment from Biogen

http://www.reuters.com/article/brief-ionis-receives-10-mln-milestone-pa-idUSASB0B97F

IONS

Reuters info on Ionis:

http://www.reuters.com/finance/stocks/overview?symbol=IONS.O

Another successful Ph 3 clinical trial result...

"The clinically meaningful benefit in quality of life bring a new energizing level of hope to the amyloidosis community around the world, a community whose critical needs have not been sufficiently addressed," said Isabelle Lousada, president and chief executive officer of the Amyloidosis Research Consortium (ARC)."

And again, the PPS sinks 4%!!!!
WTH ?!?!
I wish we had an effective SEC and less parasites gaming the market!
Not gonna happen in this era of deregulation.
But i'm buying more just to spite them. This is a great company with an enormous pipeline.

https://seekingalpha.com/article/4086620-ionis-bullish-boost-phase-3-data

7:47 am ET
*Ionis Pharma Reports Earning of $50M Milestone Payment from Biogen Following EC Marketing Authorization for SPINRAZA
Benzinga
7:30 am ET
SPINRAZA(R) (Nusinersen) Approved in the European Union as First Treatment for Spinal Muscular Atrophy
BusinessWire

I don't understand why we are only up 6%?