Ionis Pharmaceuticals Inc (IONS)

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%?

Yeah, right...You shorts sure can get ugly.

If you did a bit of DD you'd know that in the second half of the trial a new protocol was put in place that prevented the negative outcomes...period.

I bought more.

Now the EU has just granted marketing authorization for this life saving drug for children

https://seekingalpha.com/article/4073697-ionis-pharmaceuticals-falls-safety-risks-phase-3-clinical-trial