Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington's diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic form of ALS; ION859 for Parkinson's disease; ION464 for multiple system atrophy and Parkinson's disease; ION541 for amyotrophic lateral sclerosis; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases; IONIS-APOCIII-LRx for treatment resistant hypertension; Pelacarsen for Lp(a)cardiovascular disease risk reduction; Vupanorsen for sHTG/CVDRR; IONIS-FXI-LRx for clotting disorders; ION449 for cardiovascular diseases; and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease; IONIS-PKK-LRx for hereditary angioedema; Danvatirsen for cancer; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-HBVRx for hepatitis B virus; IONIS-AR-2.5Rx for prostate cancer; IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy; and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca; GlaxoSmithKline plc; Janssen Biotech, Inc.; Bayer AG; Novartis AG; Pfizer, Inc.; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.
IPO Year:
Exchange: NASDAQ
Website: ionispharma.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 10/8/2025 | $80.00 | Neutral → Overweight | Analyst |
| 9/26/2025 | $65.00 | Sell → Neutral | Goldman |
| 9/3/2025 | $70.00 | Market Perform → Outperform | BMO Capital Markets |
| 7/31/2025 | $62.00 | Equal-Weight → Overweight | Morgan Stanley |
| 7/1/2025 | $57.00 | Equal Weight → Overweight | Barclays |
| 4/7/2025 | $45.00 | Buy | H.C. Wainwright |
| 3/31/2025 | $39.00 | Neutral | Redburn Atlantic |
| 8/2/2024 | $67.00 → $60.00 | Outperform → Market Perform | BMO Capital Markets |
| 7/24/2024 | $53.00 → $62.00 | Market Perform → Outperform | Leerink Partners |
| 7/16/2024 | $29.00 → $75.00 | Buy | Jefferies |
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Analyst upgraded Ionis Pharma from Neutral to Overweight and set a new price target of $80.00
Goldman upgraded Ionis Pharma from Sell to Neutral and set a new price target of $65.00
BMO Capital Markets upgraded Ionis Pharma from Market Perform to Outperform and set a new price target of $70.00
Morgan Stanley upgraded Ionis Pharma from Equal-Weight to Overweight and set a new price target of $62.00
Barclays upgraded Ionis Pharma from Equal Weight to Overweight and set a new price target of $57.00
H.C. Wainwright initiated coverage of Ionis Pharma with a rating of Buy and set a new price target of $45.00
Redburn Atlantic initiated coverage of Ionis Pharma with a rating of Neutral and set a new price target of $39.00
BMO Capital Markets downgraded Ionis Pharma from Outperform to Market Perform and set a new price target of $60.00 from $67.00 previously
Leerink Partners upgraded Ionis Pharma from Market Perform to Outperform and set a new price target of $62.00 from $53.00 previously
Jefferies resumed coverage of Ionis Pharma with a rating of Buy and set a new price target of $75.00 from $29.00 previously
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Positive CHMP opinion is based on data from the DEVOTE study which evaluated the high dose regimen of nusinersen in treatment-naive participants and those transitioning from the currently approved 12mg dose regimen1Final European Commission decision expected in January 2026SMA is a rare, genetic, neuromuscular disease that affects individuals of all ages2 CAMBRIDGE, Mass., Nov. 17, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending by consensus the approval of the high dose regimen of nusinersen for the treatment of 5q spinal muscular a
CAMBRIDGE, Mass., Nov. 14, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) has completed the acquisition of Alcyone Therapeutics, a clinical-stage biotechnology company known for its innovative CNS therapy delivery solutions, such as the ThecaFlex DRx™ drug delivery system. ThecaFlex DRx™ is an investigational implantable device designed to provide an alternative to repeat lumbar punctures in chronic intrathecal administration of medicines, which could ease both patient experience and accessibility for a broader population of people living with neurologic disorders. ThecaFlex DRx™ has been in development since 2019, with the PIERRE and PIERRE-PK clinical studies for nusinersen curren
Recommendation based on breadth of clinical evidence demonstrating significant HAE attack rate reduction with DAWNZERA European Commission decision expected in Q1 2026 Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of DAWNZERA™ (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. The positive opinion is now referred to the European Commission (EC) for an approval decision. "We believe the positive opinion from t
- Refinancing transaction with proceeds to be utilized to repurchase or repay the 2026 Convertible Notes prior to or at maturity Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today the pricing of $700.0 million aggregate principal amount of 0.00% Convertible Senior Notes due 2030 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). Ionis also granted the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the date on which the notes are first issued, up to an additional $70.0 million aggregate principal
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that management will participate in fireside chats at the following investor conferences: Stifel 2025 Healthcare Conference on Thursday, November 13, 2025 Jefferies Global Healthcare Conference on Tuesday, November 18, 2025 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 For three decades, Ionis has invented medicines that bring better futures to people with serious d
- Refinancing transaction with proceeds to be utilized to repurchase or repay the 2026 Convertible Notes prior to or at maturity Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it intends to offer, subject to market conditions and other factors, $700.0 million aggregate principal amount of Convertible Senior Notes due 2030 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). Ionis also intends to grant the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the first date on which the notes are
– Up to 72% placebo-adjusted mean reduction in fasting triglyceride levels at six months, with reductions sustained through 12 months – – 86% of olezarsen-treated patients achieved triglyceride levels less than 500 mg/dL, below the risk threshold for acute pancreatitis – – First and only investigational treatment for sHTG to significantly reduce acute pancreatitis events – – Data simultaneously published in The New England Journal of Medicine – – Ionis to host webcast today at 3:00 p.m. ET – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced positive results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). The studie
DAWNZERA showed durable efficacy, demonstrating 94% overall mean HAE attack rate reduction at one year in OASISplus open-label extension study Patients in OASISplus switch study achieved 68% improvement in mean HAE attack rate compared to prior prophylactic therapy one year after switching to DAWNZERA Long-term efficacy and safety maintained up to four years in Phase 2 open-label extension Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced new long-term data for DAWNZERA™ (donidalorsen), the first and only RNA-targeted prophylactic medicine for hereditary angioedema (HAE), to be presented at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Me
- TRYNGOLZA® generated $32 million in net product sales in the third quarter 2025 - - DAWNZERA™ (donidalorsen) launch off to encouraging start - - Olezarsen significantly reduced triglycerides and acute pancreatitis events in severe hypertriglyceridemia (sHTG) in landmark Phase 3 studies; sNDA submission on track by year-end – - Positive pivotal zilganersen results in Alexander disease position Ionis for first independent neurology launch in 2026 - - Increasing 2025 financial guidance driven by continued strength across the business – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results and provided key updates for the third quarter ended Sept
– Ionis to host webcast on Saturday, November 8 at 3:00 p.m. ET to discuss results – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that detailed data from the landmark pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG) will be presented during a late-breaking session, titled "Groundbreaking Trials in Cardiometabolic Therapeutics", at the American Heart Association (AHA) Scientific Sessions in New Orleans. Ionis previously reported positive topline results from the Phase 3 CORE and CORE2 studies, in which olezarsen demonstrated a highly statistically significant reduction in fasting triglycerides and acute pancreatitis events
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Live Leadership Updates
Chris Schade appointed as Board ChairpersonLynne Parshall, Robert Plenge, M.D., Ph.D., and Nancy Simonian, M.D., appointed to Board of DirectorsCAMBRIDGE, Mass., July 17, 2024 /PRNewswire/ -- Alltrna, a Flagship Pioneering company unlocking transfer RNA (tRNA) biology and pioneering tRNA therapeutics to regulate the protein universe and resolve disease, today announced the appointment of Chris Schade, Growth Partner at Flagship Pioneering, as Chairperson of the Board, succeeding Noubar Afeyan, Ph.D., Co-Founder of Alltrna and Founder and CEO of Flagship Pioneering. In addition, Alltrna also announced the appointments to the company's Board of Directors of Lynne Parshall, founding Chief Opera
Contineum Therapeutics, Inc. (NASDAQ:CTNM), a clinical stage biopharmaceutical company focused on discovering and developing novel, oral small molecule therapies that target biological pathways associated with specific clinical impairments for the treatment of neuroscience, inflammation and immunology (NI&I) indications, today announced the appointment of Sarah Boyce, President and Chief Executive Officer of Avidity Biosciences, Inc. (NASDAQ:RNA), as an independent member of its Board of Directors. Ms. Boyce brings over 25 years of global commercial and clinical development expertise in the life sciences industry to Contineum's board. Ms. Boyce has extensive expertise in global pharmaceut
CARLSBAD, Calif., Dec. 14, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the appointment of Michael Yang, an experienced biopharmaceutical executive, to the Ionis Board of Directors. Mr. Yang's appointment expands the total number of Ionis Board members to 10. Mr. Yang has more than 20 years of broad senior level leadership experience in biotech, pharmaceutical and medical device companies, where he launched new platforms, expanded global revenues and diversified product lines. He most recently served as President, Chief Executive Officer and B
SAN DIEGO, March 08, 2021 (GLOBE NEWSWIRE) -- TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and utilizing a product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., announced today the appointment of Lisa Johnson-Pratt, M.D., Senior Vice President, New Product Planning at Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), to its Board of Directors. “We are very pleased to welcome Lisa to TRACON’s Board of Directors,” said Dr. Charles Theuer, President and Chief Executive Officer of TRACON. “She is a physi
CARLSBAD, Calif., Jan. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the appointment of Eugene Schneider, M.D., as executive vice president and chief clinical development officer. In this role, Dr. Schneider will oversee clinical development of novel antisense medicines across all of Ionis' therapeutic franchises. He will report to Richard S. Geary, Ph.D., Ionis' executive vice president and chief development officer, and serve on the company's executive team. Since joining Ionis in 2013, Dr. Schneider has played a key role in more than 20 clinical trials conducted in the U.S. and globally. He is an accomplished biopharmaceutical industry veteran with
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CAMBRIDGE, Mass., Nov. 14, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) has completed the acquisition of Alcyone Therapeutics, a clinical-stage biotechnology company known for its innovative CNS therapy delivery solutions, such as the ThecaFlex DRx™ drug delivery system. ThecaFlex DRx™ is an investigational implantable device designed to provide an alternative to repeat lumbar punctures in chronic intrathecal administration of medicines, which could ease both patient experience and accessibility for a broader population of people living with neurologic disorders. ThecaFlex DRx™ has been in development since 2019, with the PIERRE and PIERRE-PK clinical studies for nusinersen curren
- TRYNGOLZA® generated $32 million in net product sales in the third quarter 2025 - - DAWNZERA™ (donidalorsen) launch off to encouraging start - - Olezarsen significantly reduced triglycerides and acute pancreatitis events in severe hypertriglyceridemia (sHTG) in landmark Phase 3 studies; sNDA submission on track by year-end – - Positive pivotal zilganersen results in Alexander disease position Ionis for first independent neurology launch in 2026 - - Increasing 2025 financial guidance driven by continued strength across the business – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results and provided key updates for the third quarter ended Sept
- TRYNGOLZATM delivers $19 million in net product sales in the second quarter 2025 - - Donidalorsen approval in hereditary angioedema (HAE) anticipated next month; Ionis' second independent launch - - Phase 3 data from the pivotal CORE and CORE2 studies in severe hypertriglyceridemia (sHTG) expected in September 2025 - - Increasing 2025 financial guidance based on strong performance and improved outlook - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results and provided key updates for the second quarter ended June 30, 2025. "During the second quarter, we continued to build momentum across our business," said Brett P. Monia, Ph.D., chief execu
Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular atrophy (SMA)Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersenBased on these encouraging Phase 1 data, Biogen is engaging with regulators to advance salanersen to registrational stage studies, building on extensive experience in SMA CAMBRIDGE, Mass., June 25, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) announced topline results from the Phase 1 study of
- Encouraging start to first independent launch with TRYNGOLZATM - - On track for second independent launch with donidalorsen PDUFA August 21, 2025 - - Increasing 2025 financial guidance by more than 20% - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results for the first quarter ended March 31, 2025. "With an encouraging start to the TRYNGOLZA launch for familial chylomicronemia syndrome, the first of four independent launches expected over the next two years, Ionis' new chapter as a fully integrated, commercial-stage biotechnology company is well underway," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "We look forward to continued m
Up to four programs in registrational phase by 2025 Planned interim analysis for ulixacaltamide Essential3 Study 1 to be conducted in Q4 2024 Topline results for Phase 2 EMBOLD study of relutrigine (PRAX-562) in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) expected in Q3 2024 PRAX-628 Phase 2/3 POWER1 study to initiate in Q4 2024 Initiation of additional Phase 2 study (RADIANT) for PRAX-628 in focal and generalized epilepsy in 2H 2024, with topline results expected in 1H 2025 Cash and investments of $434 million as of June 30, 2024, maintains runway into 2027 BOSTON, Aug. 13, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a
WAINUATM U.S. launch progressing well; approved in Canada; EU approval decision expected this year Olezarsen PDUFA December 19, 2024 for FCS Positive Phase 3 donidalorsen data for HAE; preparing U.S. and EU regulatory submissions On track to achieve 2024 financial guidance CARLSBAD, Calif., Aug. 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), today reported financial results for the second quarter of 2024. "Over the first half of this year, we continued to deliver on our goal to bring a steady cadence of medicines to people with serious dise
WAINUATM launch on track; EU and Canada approval decisions expected this year Olezarsen NDA submitted to FDA for FCS; preparing EU regulatory submission Positive Phase 3 donidalorsen data for HAE; preparing regulatory submissions On track to achieve 2024 financial guidance CARLSBAD, Calif., May 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), today reported financial results for the first quarter ended March 31, 2024. "Ionis is off to a great start in 2024, as we continue to execute on our vision to bring better futures to people with serious
WAINUA™ approved with launch underway; on track for EU and Canada approval decisions this year Positive Phase 3 olezarsen and donidalorsen data, preparing regulatory submissions for FCS and HAE, respectively Olezarsen granted Breakthough Therapy designation by the FDA for FCS Ionis provides full year 2024 financial guidance CARLSBAD, Calif., Feb. 21, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), today reported financial results for the fourth quarter and full year ended December 31, 2023. "This past year included many remarkable achievements a
Reported Phase 3 data showing eplontersen continued to show improvement in ATTRv-PN through 85 weeks; December 22, 2023 PDUFA date Completed enrollment in donidalorsen OASIS-HAE study and eplontersen CARDIO-TTRansform ATTR-CM study keeping Phase 3 data readouts on track On track to achieve 2023 financial guidance CARLSBAD, Calif., Aug. 9, 2023 /PRNewswire/ -- – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), today reported financial results for the second quarter of 2023. Financial results are summarized below: Three months ended June 30, Six months ended June 30,