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 |
|---|---|---|---|
| 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 |
| 6/14/2024 | $44.00 | Underperform → Mkt Perform | Bernstein |
| 4/10/2024 | $58.00 | Peer Perform → Outperform | Wolfe Research |
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- Pivotal Phase 3 REVEAL study enrollment on track to complete in 2026 - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 for the treatment of Angelman syndrome (AS), a rare neurological disease that often presents in infancy and is characterized by profound intellectual disability, impaired communication, motor impairment and debilitating seizures. "With no approved disease-modifying treatments available for people living with Angelman syndrome, receiving this Breakthrough Therapy designation for ION582 underscores both the severity of this disease and the significant unmet
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that it will host Ionis Innovation Day on Tuesday, October 7, 2025, in New York. The agenda will feature presentations from members of the Ionis leadership team as well as a key thought leader in the treatment of severe hypertriglyceridemia, familial chylomicronemia syndrome and lipid disorders, Robert D. Fishberg, M.D., cardiologist with Atlantic Medical Group in Springfield, NJ, and assistant clinical professor of medicine at the Sidney Kimmel Medical College, Thomas Jefferson University. The presentations will be webcast live from 8:30 a.m. to 12:30 p.m. Eastern time. Please click here to register for the event. A webcast re
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that management will participate in fireside chats at the following investor conferences: 2025 Wells Fargo Healthcare Conference on Thursday, September 4, 2025 H.C. Wainwright 27th Annual Global Investment Conference on Monday, September 8, 2025 Morgan Stanley 23rd Annual Global Healthcare Conference on Tuesday, September 9, 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 Pharm
– Up to 72% (p<0.0001) placebo-adjusted mean reduction in fasting triglycerides – – 85% (p=0.0002) reduction in acute pancreatitis events, the first and only time achieved for the treatment of sHTG – – Favorable safety and tolerability profile – – sNDA submission planned by end of year – – Ionis to host webcast today at 8:30 a.m. ET – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced positive topline results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG). In the studies, olezarsen demonstrated a highly statistically significant placebo-adjusted mean reduction in fasting triglycerides of up to 72% and a highly
- DAWNZERA demonstrated significant and sustained HAE attack rate reduction and long-term disease control - - Offers longest dosing option for HAE, with dosing every 4 or 8 weeks - - Compelling profile supported by recently published switch data - - Ionis' second independent launch in just nine months, with potential for two additional launches next year - - Ionis to host webcast today at 12:15pm ET - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is th
- 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
– Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA – – European Commission decision expected by Q4 2025 – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4 2
– Patients switched to donidalorsen without increased breakthrough attacks and experienced further reductions in mean attack rate from baseline – – 84% of patients surveyed preferred donidalorsen over their prior treatment – – Donidalorsen is currently under review with the U.S. FDA as a potential first-in-class prophylactic treatment for HAE; PDUFA date August 21 – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that positive results from the Phase 3 OASISplus prospective switch study of donidalorsen in patients with hereditary angioedema (HAE) were published in The Journal of Allergy and Clinical Immunology (JACI) In Practice. Results indicate that patients who switched to
Webcast scheduled for Wednesday, July 30 at 11:30 a.m. Eastern Time Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Wednesday, July 30th at 11:30 a.m. Eastern Time to discuss its second 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 six marketed medicines and a leading pipeline in neurology, cardiology, and
New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who transitioned to the investigational higher dose regimen of nusinersen from 12 mg SPINRAZA® (nusinersen) Final results from the landmark NURTURE study highlight the profound impact of early treatment with 12 mg SPINRAZA in clinically presymptomatic SMA with 92% of children achieving the ability to walk independently CAMBRIDGE, Mass., June 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). These latest findings from
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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
Bernstein upgraded Ionis Pharma from Underperform to Mkt Perform and set a new price target of $44.00
Wolfe Research upgraded Ionis Pharma from Peer Perform to Outperform and set a new price target of $58.00
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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
- 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
Eplontersen NDA submitted to FDA; tofersen under regulatory review for marketing approval in U.S. and EU Phase 3 data planned for eplontersen and olezarsen; robust late-stage pipeline expanding with two new Phase 3 programs Ionis provides full year 2023 financial guidance CARLSBAD, Calif., Feb. 22, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company"), a genetic medicines company, today reported financial results for the fourth quarter and full year ended December 31, 2022. Financial results are summarized below: Three months ended December 31, Year e