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 |
|---|---|---|---|
| 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 |
| 1/2/2024 | $52.00 → $62.00 | Neutral → Buy | BofA Securities |
| 10/23/2023 | $33.00 → $52.00 | Underperform → Neutral | BofA Securities |
| 9/29/2023 | $63.00 | Strong Buy | Raymond James |
| 7/31/2023 | $36.00 → $60.00 | Neutral → Buy | Citigroup |
| 6/7/2023 | $58.00 | Overweight | Piper Sandler |
– Accelerating value creation with numerous commercial, regulatory and pipeline milestones – – Ionis outlines clear path to sustained positive cash flow – CARLSBAD, Calif., Jan. 13, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced highlights from the Company's 2024 achievements and previewed important milestones expected in 2025. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 10:30am PT; the presentation is available today on the Ionis website. "With last month's U.S. approval
Three blockbuster-potential assets in late-stage clinical trials and four pivotal readouts expected in 2025; anticipate four commercial assets by 2028 Study 1 of Essential3 program for ulixacaltamide in essential tremor (ET) on track for Q1 2025 interim analysis; NDA filing for ulixacaltamide expected in 2025 Enrollment in EMBOLD study of relutrigine cohort 2 is on track; targeting NDA filing in 2026 Vormatrigine ENERGY program advancing with read-outs of RADIANT in first half of 2025 and POWER1 by year-end 2025 UCB has exercised its option to license KCNT1 small molecule candidate for global development and commercialization Cash and investments ~ $470 million at the end of 2024 suppor
CARLSBAD, Calif., Jan. 8, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that Brett P. Monia, Ph.D., chief executive officer, will present a company overview at the 43rd Annual J.P. Morgan Healthcare Conference at 10:30 a.m. PT on Wednesday, January 15, 2025. A live webcast of the presentation can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events. A replay will be available on the Ionis website within 48 hours and will be archived for a limited time. About Ionis Pharmaceuticals, Inc.For three decade
TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvalsIonis to host webcast today at 6:45pm ET CARLSBAD, Calif., Dec. 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndr
BOSTON, Nov. 26, 2024 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that that it will present preclinical and clinical data from three of its epilepsy programs at the American Epilepsy Society (AES) Annual Meeting, being held from December 6 to 10, 2024 in Los Angeles, California. "At Praxis, we are on the brink of transformative change with a leading pipeline that includes relutrigine and vormatrigine, the most potent and most fun
CARLSBAD, Calif., Nov. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that management will participate in fireside chats at the following investor conferences: Guggenheim's Inaugural Healthcare Conference on Wednesday, November 13, 2024UBS Global Healthcare Conference on Wednesday, November 13, 2024Stifel 2024 Healthcare Conference on Monday, November 18, 2024Jefferies London Healthcare Conference on Wednesday, November 20, 2024Citi's 2024 Global Healthcare Conference on Tuesday, December 3, 2024Piper Sandler 36th Annual Healthcare Conference
Positive End of Phase 2 discussion with FDA, including alignment on Phase 3 designBayley-4 expressive communication selected as Phase 3 study primary endpointInitiation of ION582 Phase 3 study planned for H1 2025Ionis to share ION582 program update at the FAST Global Science Summit in NovemberCARLSBAD, Calif., Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS). AS typically presents in infancy and is characterized by profound intellectual disabi
WAINUATM U.S. launch progressing well; approved in UK; positive CHMP opinion Olezarsen FCS PDUFA December 19, 2024 Donidalorsen HAE PDUFA August 21, 2025; EU regulatory submission in process On track to achieve 2024 P&L financial guidance; increased 2024 cash guidance CARLSBAD, Calif., Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results for the third quarter of 2024. "Today, we stand on the cusp of a new era for Ionis, with our first co-commercialization launch proceeding well with WAINUA, our first planned
Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approvalDonidalorsen PDUFA date set for August 21, 2025Donidalorsen has the potential to be Ionis' second independent commercial launchCARLSBAD, Calif., Nov. 4, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. The FDA has set an action date of August 21, 2025 under the Prescription Dru
Webcast scheduled for Wednesday, November 6 at 11:30 a.m. Eastern Time CARLSBAD, Calif., Oct. 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Wednesday, November 6th at 11:30 a.m. Eastern Time to discuss its third quarter 2024 financial results. 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 futur
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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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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
BofA Securities upgraded Ionis Pharma from Neutral to Buy and set a new price target of $62.00 from $52.00 previously
BofA Securities upgraded Ionis Pharma from Underperform to Neutral and set a new price target of $52.00 from $33.00 previously
Raymond James initiated coverage of Ionis Pharma with a rating of Strong Buy and set a new price target of $63.00
Citigroup upgraded Ionis Pharma from Neutral to Buy and set a new price target of $60.00 from $36.00 previously
Piper Sandler resumed coverage of Ionis Pharma with a rating of Overweight and set a new price target of $58.00
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
Royalty Pharma to acquire an interest in SPINRAZA® and pelacarsen royalties – Ionis retains majority of royalties and all milestones from Novartis for pelacarsenRoyalty Pharma to pay Ionis $500 million upfront and up to $625 million in milestonesAgreement enables Ionis to achieve commercial readiness for multiple late-stage programs and advance its innovative pipeline of genetic medicines NEW YORK and CARLSBAD, Calif., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Royalty Pharma plc (NASDAQ:RPRX) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that Royalty Pharma has acquired an interest in Ionis' royalty in Biogen's SPINRAZA® (nusinersen) and Novartis' pelacarsen for up to $1.125 b
Royalty Pharma to acquire an interest in SPINRAZA® and pelacarsen royalties -- Ionis retains majority of royalties and all milestones from Novartis for pelacarsenRoyalty Pharma to pay Ionis $500 million upfront and up to $625 million in milestones Agreement enables Ionis to achieve commercial readiness for multiple late-stage programs and advance its innovative pipeline of genetic medicinesCARLSBAD, Calif. and NEW YORK, Jan. 9, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and Royalty Pharma plc (NASDAQ:RPRX) today announced that Royalty Pharma has acquired an interest in Ionis' royalty in Biogen's SPINRAZA® (nusinersen) and Novartis' pelacarsen for up to $1.125 billion, inc
Advanced key priorities: initiated manufacturing infrastructure project to support growth Increased 2022 cash and investments guidance CARLSBAD, Calif., Nov. 9, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results for the third quarter of 2022. Financial results are summarized below: Three months ended September 30, Nine months ended September 30, 2022 2021 2022 2021 (amounts in millions) Total revenue $160 $133 $435 $370 Operating expenses $219 $219 $637 $621 Operating expenses on a non-GAAP basis $195 $185 $562 $499