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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– 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
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the company was named as a top employer to work for by Science magazine in its annual Top Employer survey of the biopharmaceutical and related industries. In its first year of eligibility for the award, Ionis was ranked second based on industry-leading innovation, strong alignment of company and employee values, employee loyalty and a culture of respect. "Our people are our greatest strength at Ionis. We're thrilled to be featured as the #2 top employer by Science in our first year considered for the list – an accomplishment that reflects both the caliber of our science and the unique spirit of the Ionis culture," said Brett P. Mon
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that Brett P. Monia, Ph.D., Ionis' chief executive officer, was awarded the 2025 Lifetime Achievement Award from the Oligonucleotide Therapeutics Society (OTS), a nonprofit organization that fosters academia and industry-based research and development of oligonucleotide therapeutics. The OTS Lifetime Achievement Award recognizes individuals who have demonstrated a lasting commitment to the field of oligonucleotide therapeutics through education, research and therapeutic application and serve as outstanding examples to new and seasoned members of the field. A founding member of the OTS, Dr. Monia's contributions have focused on the
Webcast scheduled for Wednesday, October 29 at 11:30 a.m. Eastern Time Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Wednesday, October 29th at 11:30 a.m. Eastern Time to discuss its third quarter 2025 financial results and 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
– Two independent launches underway in less than nine months, with two more on track for 2026 – – Growing pipeline of wholly owned medicines in neurology and cardiometabolic disease and leading technology for next-generation medicines – – Clear path to sustained positive cash flow – – Ionis to webcast Innovation Day at 8:30 a.m. ET – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today is hosting its 2025 Innovation Day in New York City. Ionis leaders will highlight the company's recent and near-term independent launches, growing pipeline of wholly owned medicines, innovative technology and clear path to sustained positive cash flow. Additionally, a leading physician will discuss the cur
CAMBRIDGE, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the Company's supplemental New Drug Application (sNDA) for the high dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA). The FDA letter requested an update to the technical information be included in the Chemistry Manufacturing and Controls (CMC) module of the sNDA. The letter did not cite any deficiencies in the clinical data of the high dose regimen. The FDA provided options for resolution, and Biogen is planning to resubmit the application promptly based upon readily availabl
– First and only investigational medicine to demonstrate a clinically meaningful and disease-modifying impact on this rare, often fatal neurological condition – – NDA submission planned in Q1 2026 – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test (10MWT) compared to control at week
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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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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
Live finance-specific insights
- 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,
QALSODY approved for SOD1-ALS; MAA under review in EU Reported positive Phase 3 eplontersen ATTRv-PN data; December 22, 2023 PDUFA date On track to achieve 2023 financial guidance CARLSBAD, Calif., May 3, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), today reported financial results for the first quarter of 2023. Financial results are summarized below: Three months ended March 31, 2023 2022 (amounts in millions) Total revenue $131 $142 Operating expenses $245 $199 Operating expenses on a non-GAAP basis $218 $173 Loss from operations ($114) ($5