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 |
|---|---|---|---|
| 7/16/2026 | Outperform → Peer Perform | Wolfe Research | |
| 5/19/2026 | $115.00 | Buy | Citigroup |
| 4/21/2026 | $110.00 | Buy | Canaccord Genuity |
| 4/10/2026 | $104.00 | Outperform | Raymond James |
| 1/28/2026 | $95.00 | Overweight | Barclays |
| 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 |
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Live FDA approvals issued by the Food and Drug Administration and FDA breaking news
Submission status for IONIS PHARMS INC's drug TRYNGOLZA (AUTOINJECTOR) (SUPPL-4) with active ingredient OLEZARSEN SODIUM has changed to 'Approval' on 06/24/2026. Application Category: NDA, Application Number: 218614, Application Classification: Efficacy
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Wolfe Research downgraded Ionis Pharma from Outperform to Peer Perform
Citigroup initiated coverage of Ionis Pharma with a rating of Buy and set a new price target of $115.00
Canaccord Genuity initiated coverage of Ionis Pharma with a rating of Buy and set a new price target of $110.00
Raymond James resumed coverage of Ionis Pharma with a rating of Outperform and set a new price target of $104.00
Barclays resumed coverage of Ionis Pharma with a rating of Overweight and set a new price target of $95.00
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
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Webcast scheduled for Wednesday, July 29 at 8:30 a.m. Eastern TimeIonis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Wednesday, July 29 at 8:30 a.m. Eastern Time to discuss its second 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
- Clinical outcomes: Diranersen demonstrated efficacy across all studied doses at 18 months, with consistent results across multiple prespecified clinical endpoints; the 60 mg dose showed the strongest response with slowing of clinical decline on the cognitive endpoints—42% on ADAS-Cog13 and 50% on MMSE—alongside a 26% slowing on CDR-SB - - Biomarker response: Diranersen is the first tau-directed therapy to demonstrate robust reductions in both CSF total tau, with mean reductions of 50–65%, and brain tau pathology, as measured by PET, across all studied doses in a Phase 2 study - - Dose response: CDR-SB results favored diranersen versus placebo across all studied doses; higher doses wer
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an investigational RNA-targeted medicine for people living with Dravet syndrome, a rare, severe and lifelong neurological disorder. Dravet syndrome typically begins in infancy and is associated with prolonged seizures, developmental delays, cognitive impairments and an increased risk of sudden death. "The first participant to receive ION337 in the ASCEND study marks an important step toward advancing a potential disease modifying therapy for people living with Dravet syndrome," said Holly Kordasiewicz, Ph.D., executive vice president, chief developme
– Study missed primary endpoint of composite outcome of cardiovascular (CV) mortality and recurrent CV events; in a pre-specified subgroup analysis, a nominally significant result was observed with eplontersen monotherapy – – Favorable safety profile consistent with previous results – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and partner AstraZeneca today announced that the CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet the primary efficacy endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent CV clinical events up to Week 140 compared with placebo. In this contemporary pati
- Topline data from REVEAL anticipated in 2H 2027 - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that it has completed enrollment in the pivotal cohort (Cohort 1) of the global Phase 3 REVEAL study evaluating obudanersen (ION582), an investigational RNA-targeted medicine for people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder associated with significant impairments in communication, physical function and cognition. The pivotal cohort enrolled 136 participants aged 2 to < 18 years old with a confirmed clinical diagnosis of AS with genetic confirmation of either a UBE3A deletion or UBE3A mutation. The adult cohort (Cohort 2) comprised of pa
- 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. Recorda
– 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 autoinjecto
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the appointment of Ludwig Hantson, Ph.D., an accomplished global biopharmaceutical leader, to the Ionis Board of Directors, effective immediately."We are pleased to welcome Dr. Hantson to the board," said Joseph Loscalzo, M.D., Ph.D., chairman of the Ionis Board of Directors. "Ludwig's biopharma executive leadership experience will be especially meaningful as Ionis prepares for a steady cadence of new commercial launches, including two additional independent launches this year, while continuing to scale its operations as a fully integrated biotechnology company."Dr. Hantson has more than 30 years of leadership experience at biopharmac
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that new data analyses from the landmark Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG) will be presented during four oral presentations at upcoming medical conferences. Data will be presented at the 2026 Scientific Sessions of the American Diabetes Association® (ADA) in New Orleans (June 5-8), the National Lipid Association (NLA) Scientific Sessions in Chicago (June 11-14) and ENDO 2026, the Endocrine Society's annual meeting in Chicago (June 13-16). "People living with sHTG are at risk of acute pancreatitis, a debilitatingly painful complication that can potentially be life-threateni
– 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
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Live Leadership Updates
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the appointment of Ludwig Hantson, Ph.D., an accomplished global biopharmaceutical leader, to the Ionis Board of Directors, effective immediately."We are pleased to welcome Dr. Hantson to the board," said Joseph Loscalzo, M.D., Ph.D., chairman of the Ionis Board of Directors. "Ludwig's biopharma executive leadership experience will be especially meaningful as Ionis prepares for a steady cadence of new commercial launches, including two additional independent launches this year, while continuing to scale its operations as a fully integrated biotechnology company."Dr. Hantson has more than 30 years of leadership experience at biopharmac
– Lynne Parshall and Joseph Wender to retire at end of term in June 2026 and Peter Reikes to join Ionis Board – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced upcoming transitions on its Board of Directors. B. Lynne Parshall, Esq., and Joseph Wender will be retiring at the end of their term and Peter N. Reikes will be rejoining as a director. The changes will be effective June 4, 2026. Ms. Parshall has served as a director since September 2000. Previously, she served as Ionis' chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December 2012. Ms. Parshall joined Ionis in 1991 and served in various executive ro
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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- TRYNGOLZA® (olezarsen) showed increasing demand in FCS driven by strong launch execution – - Olezarsen sNDA accepted by the FDA for Priority Review; sHTG launch preparations on track – – Increasing annual TRYNGOLZA peak net sales guidance to >$3B for sHTG – - Zilganersen NDA for Alexander disease accepted for Priority Review, paving way for Ionis' first independent launch from leading neurology pipeline – - Strong first-quarter performance and outlook for the year supports improved 2026 financial guidance - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results and provided key updates for the first quarter ended March 31, 2026. "Ionis' stro
- TRYNGOLZA® generated $108 million in net product sales in 2025, the first year of launch – - Olezarsen sHTG launch preparations on track, sNDA submitted - - Exceeded 2025 financial guidance, 2026 guidance reflects commitment to independently deliver a steady cadence of innovative medicines to patients - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results and provided key updates for the fourth quarter and year ended December 31, 2025. "2025 was a defining year for Ionis, marked by the successful execution of our first two independent launches and multiple positive data readouts across our pipeline, positioning Ionis for continued success in 2
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