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 |
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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 |
Leerink Partners analyst Mani Foroohar upgrades Ionis Pharmaceuticals (NASDAQ:IONS) from Market Perform to Outperform and raises the price target from $53 to $62.
TD Cowen analyst Yaron Werber maintains Ionis Pharmaceuticals (NASDAQ:IONS) with a Buy and raises the price target from $54 to $59.
B of A Securities analyst Jason Gerberry maintains Ionis Pharmaceuticals (NASDAQ:IONS) with a Buy and raises the price target from $67 to $68.
Needham analyst Joseph Stringer reiterates Ionis Pharmaceuticals (NASDAQ:IONS) with a Buy and maintains $60 price target.
Ionis Pharmaceuticals Inc (NASDAQ:IONS) released results Monday from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS). AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide. The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor function measures. Also Read: Ionis Pharmaceuticals Reveals Late-Stage Donidalorsen Data, Analyst Says Results Show Competitive Hereditary Angioedema Profile. Overall, 97% of peop
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced positive results from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS) demonstrating consistent and encouraging clinical improvement on measures assessing all functional domains including communication, cognition and motor function. Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms as measured by the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C). ION582 showed favorable safety and tolerability at all dose levels in the study. Detailed results will be presented in a compa
Providing a diverse range of perspectives from bullish to bearish, 6 analysts have published ratings on Ionis Pharmaceuticals (NASDAQ:IONS) in the last three months. The following table provides a quick overview of their recent ratings, highlighting the changing sentiments over the past 30 days and comparing them to the preceding months. Bullish Somewhat Bullish Indifferent Somewhat Bearish Bearish Total Ratings 4 1 1 0 0 Last 30D 2 0 0 0 0 1M Ago 0 0 1 0 0 2M Ago 1 0 0 0 0 3M Ago 1 1 0 0 0 The 12-month price targets, analyzed by analysts, offer insights with an average target of $66.17, a high estimate of $82.00, and a low estimate of $53.00. Marking an increase of 14.09
B of A Securities analyst Jason Gerberry maintains Ionis Pharmaceuticals (NASDAQ:IONS) with a Buy and raises the price target from $64 to $67.
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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
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
BOSTON, Sept. 04, 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 International League Against Epilepsy (ILAE) 15th European Epilepsy Congress (EEC), being held from September 7 to 11, 2024 in Rome, Italy. "We are pleased to highlight the continued progress across our portfolio of epilepsy assets, building on the strong evidence we have already ge
Positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA; Biogen plans to submit for regulatory approval of this investigational dose regimenHigher dose nusinersen regimen showed statistically significant improvement compared to a prespecified matched sham control groupAmong key measures of clinical efficacy, higher dose regimen showed positive trends compared to the approved dosing regimen CAMBRIDGE, Mass., Sept. 04, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a higher dose re
CARLSBAD, Calif., Aug. 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that management will participate in fireside chats at the following investor conferences: 2024 Wells Fargo Healthcare Conference on Wednesday, September 4, 2024Morgan Stanley 22nd Annual Global Healthcare Conference on Thursday, September 5, 2024A 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. A replay will be available on the Ionis website within 48
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
Alcyone's ThecaFlex is an implantable medical device under investigation for routine subcutaneous administration of therapeutics to the cerebrospinal fluid (CSF).The first stage of the pivotal IDE PIERRE trial has been completed.10 spinal muscular atrophy (SMA) patients have undergone ThecaFlex implantation and have received nusinersen through the device.No device-related adverse events, including infections, were observed in the 30 days after implantation.FDA has agreed to an additional 80-SMA patient, 30-center, enrollment after review of the clinical data by Data Monitoring Safety Committee of first 10 SMA patients implanted.LOWELL, Mass., July 31, 2024 /PRNewswire/ -- Alcyone Therapeutic
ION582 showed robust and consistent benefit in communication, cognition and motor function in a broad patient population evaluated with a comprehensive set of assessment tools that collect input from parents and clinicians97% of patients in the medium and high dose groups saw improvement in overall Angelman syndrome symptoms as measured by the SAS-CGI-C Improvements on the Bayley-4 in cognition, communication and motor function exceeded those observed in natural history studiesIonis plans to initiate Phase 3 development in H1 2025Ionis to host webcast on Monday, July 22 at 8:00am ETCARLSBAD, Calif., July 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced posit
Webcast scheduled for Thursday, August 1 at 11:30 a.m. Eastern Time CARLSBAD, Calif., July 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it will host a live webcast on Thursday, August 1st at 11:30 a.m. Eastern Time to discuss its second 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 futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardi
Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition CARLSBAD, Calif., July 18, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine in development for the treatment of children and adults with a rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD). The primary endpoint is percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT). Topline data are anticipated in the second ha
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
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