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    Ionis reports first quarter 2025 financial results

    4/30/25 7:00:00 AM ET
    $IONS
    Biotechnology: Pharmaceutical Preparations
    Health Care
    Get the next $IONS alert in real time by email

    - 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 momentum this year, including our second independent launch for donidalorsen in hereditary angioedema and Phase 3 results for olezarsen for severe hypertriglyceridemia in the third quarter. We also continue to advance our next wave of wholly owned neurology medicines, including ION582 for Angelman syndrome, which is on track to start Phase 3 development shortly. Our advancing pipeline of transformational medicines, together with strong commercial and financial execution, position Ionis to deliver increasing value for all stakeholders."

    First Quarter 2025 Summary Financial Results(1):

     

     

    Three months ended

    March 31,

     

    2025

     

     

    2024

     

     

     

    (amounts in millions)

    Total revenue

     

    $

    132

     

    $

    119

     

    Operating expenses

     

    $

    278

     

    $

    269

     

    Operating expenses on a non-GAAP basis

     

    $

    249

     

    $

    238

     

    Loss from operations

     

    $

    (146

    )

    $

    (150

    )

    Loss from operations on a non-GAAP basis

     

    $

    (117

    )

    $

    (119

    )

    (1)

    Reconciliation of GAAP to non-GAAP basis contained later in this release.

    Recent Financial Highlights

    • Revenue increased 10% in the first quarter of 2025 compared to the same period last year, driven by higher commercial revenue including new TRYNGOLZA product revenue following approval in late December and higher SPINRAZA and WAINUA royalty revenue
    • Operating expenses increased slightly in the first quarter of 2025 driven by commercialization efforts for TRYNGOLZA, donidalorsen and WAINUA
    • Increased 2025 financial guidance reflects recent successful licensing transactions:

     

    Full Year 2025 Guidance

     

    Previous

    Guidance

     

    New

    Guidance

    Revenue

     

    >$600 million

     

    $725-750 million

    Operating loss on a non-GAAP basis

     

    <$495 million

     

    <$375 million

    Cash, cash equivalents and short-term investments

     

    ~$1.7 billion

     

    ~$1.9 billion

    Recent Highlights - Marketed Medicines

    • TRYNGOLZATM (olezarsen), the first-ever treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to diet, generated net product sales of over $6 million in its first full quarter following approval in the U.S. on December 19, 2024
      • Commercialization rights in countries outside of the U.S., Canada and China licensed to Sobi
      • EU approval decision anticipated in H2:2025
    • WAINUATM (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved commercial and regulatory milestones:
      • Generated sales of $39 million resulting in royalty revenue of $9 million in the first quarter of 2025
      • Launch underway in numerous countries, including the EU following approval by the European Commission (EC); additional global submissions in progress to expand WAINUA access
    • SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $424 million resulting in royalty revenue of $48 million in the first quarter of 2025
      • Higher dose nusinersen under review for marketing approval in U.S. (PDUFA date of September 22, 2025) and EU

    Recent Highlights - Late-Stage Wholly Owned Pipeline

    • Olezarsen on track for topline Phase 3 data from supportive ESSENCE study in Q2:2025 and pivotal CORE and CORE2 studies in patients with severe hypertriglyceridemia (sHTG) in Q3:2025, positioning olezarsen to potentially treat this second, more prevalent patient population with high unmet need
      • Published Phase 3 study design and baseline characteristics for CORE, CORE2 and ESSENCE studies in the American Heart Journal, highlighting the potential to generate robust and meaningful data
    • Donidalorsen on track for launch this year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE), assuming approval:
      • Under review for marketing approval in U.S. (PDUFA date of August 21, 2025) and EU
      • Presented positive Phase 2 open label extension (OLE) study data demonstrating an overall sustained mean reduction in HAE attack rates of 96% in patients treated up to 196 weeks with every four weeks or every eight weeks dosing
      • Published positive patient-reported outcomes (PROs) from Phase 3 OASIS-HAE study in Allergy showing donidalorsen significantly improved quality of life (QoL) and other PROs compared to placebo
    • ION582 on track to initiate Phase 3 development in Angelman syndrome (AS) in Q2:2025

    Recent Highlights - Partnered Pipeline

    • Pelacarsen Phase 3 Lp(a) HORIZON study design and baseline characteristics published in the American Heart Journal highlighting the potential to generate robust and meaningful data
    • Licensed sapablursen global development and commercialization rights to Ono Pharmaceutical Co. generating $280 million in an upfront payment, with the potential to earn up to $660 million in additional payments plus royalties in the mid-teen percentage range on annual net sales
    • IONIS-MAPTRx (BIIB080) received FDA Fast Track designation for the treatment of Alzheimer's disease

    First Quarter 2025 Financial Results

    "We are increasing our 2025 financial guidance including raising revenue guidance by more than 20 percent due to our strong first quarter results and recent successful licensing transactions. We are also substantially improving our operating loss and cash guidance and now expect to end the year with approximately $1.9 billion in cash. Our strong financial position and commitment to drive operating leverage position Ionis to advance our strategic priorities and successfully navigate the dynamic macroeconomic environment," said Elizabeth L. Hougen, chief financial officer, Ionis. "Our first quarter results reflected encouraging early performance in the TRYNGOLZA launch, adding product revenue to our P&L for the first time. Moving forward, the three additional independent launches anticipated over the next couple of years position Ionis to deliver substantial and growing product revenue. This product revenue coupled with anticipated increasing royalty revenue from multiple partner launches and disciplined investment, position Ionis to achieve sustained positive cash flow."

    Revenue

    Ionis' revenue was comprised of the following:

    Three months ended

    March 31,

     

     

    2025

     

    2024

    Revenue

     

    (amounts in millions)

    Commercial revenue:

     

     

     

     

    Product sales, net:

     

     

     

     

    TRYNGOLZA sales, net

     

    $

    6

     

    $

    -

    Total product sales, net

     

     

    6

     

     

    -

    Royalty revenue:

     

     

     

     

    SPINRAZA royalties

     

     

    48

     

     

    38

    WAINUA royalties

     

     

    9

     

     

    1

    Other royalties

     

     

    7

     

     

    10

    Total royalty revenue

     

     

    64

     

     

    49

    Other commercial revenue:

     

     

     

     

    TEGSEDI and WAYLIVRA revenue, net

     

     

    6

     

     

    9

    Other revenue

     

     

    -

     

     

    1

    Total other commercial revenue

     

     

    6

     

     

    10

    Total commercial revenue

     

     

    76

     

     

    59

    Research and development revenue:

     

     

     

     

    Collaborative agreement revenue

     

     

    46

     

     

    49

    WAINUA joint development revenue

     

     

    10

     

     

    11

    Total research and development revenue

     

     

    56

     

     

    60

    Total revenue

     

    $

    132

     

    $

    119

    Commercial revenue for the first quarter of 2025 increased 28% compared to the same period in 2024, driven in part by revenue from U.S. product sales from the launch of TRYNGOLZA. Higher royalty revenues from SPINRAZA, WAINUA and QALSODY also contributed to the year over year increase.

    The remainder of the Company's revenue came from programs under its R&D collaborations, reflecting the value that Ionis' pipeline and technology continues to generate.

    Operating Expenses

    SG&A expenses increased in the first quarter of 2025 compared to the same period in 2024 primarily due to the launches of WAINUA and TRYNGOLZA, and advancing launch preparation activities for donidalorsen. This increase was partially offset by a decrease in R&D expenses as several late-stage studies ended. Overall, this led to a slight increase in total operating expenses.

    Balance Sheet

    As of March 31, 2025, Ionis' cash, cash equivalents and short-term investments were $2.1 billion, compared to $2.3 billion at December 31, 2024. Ionis' working capital decreased over the same period primarily due to the Company's lower cash and short-term investments balance. Ionis generated $280 million from the global license of sapablursen in the second quarter.

    Webcast

    Management will host a conference call and webcast to discuss Ionis' first quarter 2025 results at 11:30 a.m. Eastern time on Wednesday, April 30, 2025. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company's first quarter 2025 earnings slides click here.

    Ionis' Marketed Medicines

    INDICATION for TRYNGOLZA™ (olezarsen)

    TRYNGOLZA™ (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

    IMPORTANT SAFETY INFORMATION

    CONTRAINDICATIONS

    TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.

    WARNINGS AND PRECAUTIONS

    Hypersensitivity Reactions

    Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.

    ADVERSE REACTIONS

    The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia.

    Please see full Prescribing Information for TRYNGOLZA.

    INDICATION for WAINUA™ (eplontersen)

    WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

    IMPORTANT SAFETY INFORMATION for WAINUA™ (eplontersen)

    WARNINGS AND PRECAUTIONS

    Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

    ADVERSE REACTIONS

    Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

    Please see link to U.S. Full Prescribing Information for WAINUA.

    For more information about SPINRAZA and QALSODY, visit https://www.spinraza.com/ and https://www.qalsody.com/, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

    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 select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.

    Ionis' Forward-looking Statement

    This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

    In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries.

    Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. TRYNGOLZATM is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies.

     

    IONIS PHARMACEUTICALS, INC.

    SELECTED FINANCIAL INFORMATION

    Condensed Consolidated Statements of Operations

    (In Millions, Except Per Share Data)

     

     

     

     

    Three months ended

    March 31,

     

     

     

    2025

     

     

     

    2024

     

     

     

    (unaudited)

    Revenue:

     

     

     

     

    Commercial revenue:

     

     

     

     

    Product sales, net

     

    $

    6

     

     

    $

    -

     

    Royalty revenue

     

     

    64

     

     

     

    49

     

    Other commercial revenue

     

     

    6

     

     

     

    10

     

    Total commercial revenue

     

     

    76

     

     

     

    59

     

    Research and development revenue:

     

     

     

     

    Collaborative agreement revenue

     

     

    46

     

     

     

    49

     

    WAINUA joint development revenue

     

     

    10

     

     

     

    11

     

    Total research and development revenue

     

     

    56

     

     

     

    60

     

    Total revenue

     

     

    132

     

     

     

    119

     

    Expenses:

     

     

     

     

    Cost of sales

     

     

    1

     

     

     

    2

     

    Research, development and patent

     

     

    201

     

     

     

    214

     

    Selling, general and administrative

     

     

    76

     

     

     

    53

     

    Total operating expenses

     

     

    278

     

     

     

    269

     

    Loss from operations

     

     

    (146

    )

     

     

    (150

    )

     

     

     

     

     

    Other income (expense):

     

     

     

     

    Interest expense related to the sale of future royalties

     

     

    (19

    )

     

     

    (18

    )

    Other income, net

     

     

    18

     

     

     

    25

     

    Loss before income tax expense

     

     

    (147

    )

     

     

    (143

    )

     

     

     

     

     

    Income tax expense

     

     

    -

     

     

     

    -

     

     

     

     

     

     

    Net loss

     

    $

    (147

    )

     

    $

    (143

    )

     

     

     

     

     

    Basic and diluted net loss per share

     

    $

    (0.93

    )

     

    $

    (0.98

    )

    Shares used in computing basic and diluted net loss per share

     

     

    159

     

     

     

    146

     

     

    IONIS PHARMACEUTICALS, INC.

    Reconciliation of GAAP to Non-GAAP Basis:

    Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss

    (In Millions)

     

     

     

     

     

    Three months ended

    March 31,

     

     

     

    2025

     

     

     

    2024

     

     

     

    (unaudited)

    As reported research, development and patent expenses according to GAAP

     

    $

    201

     

     

    $

    214

     

    Excluding compensation expense related to equity awards

     

     

    (20

    )

     

     

    (22

    )

    Non-GAAP research, development and patent expenses

     

    $

    181

     

     

    $

    192

     

     

     

     

     

     

    As reported selling, general and administrative expenses according to GAAP

     

    $

    76

     

     

    $

    53

     

    Excluding compensation expense related to equity awards

     

     

    (9

    )

     

     

    (9

    )

    Non-GAAP selling, general and administrative expenses

     

    $

    67

     

     

    $

    44

     

     

     

     

     

     

    As reported operating expenses according to GAAP

     

    $

    278

     

     

    $

    269

     

    Excluding compensation expense related to equity awards

     

     

    (29

    )

     

     

    (31

    )

    Non-GAAP operating expenses

     

    $

    249

     

     

    $

    238

     

     

     

     

     

     

    As reported loss from operations according to GAAP

     

    $

    (146

    )

     

    $

    (150

    )

    Excluding compensation expense related to equity awards

     

     

    (29

    )

     

     

    (31

    )

    Non-GAAP loss from operations

     

    $

    (117

    )

     

    $

    (119

    )

     

     

     

     

     

    As reported net loss according to GAAP

     

    $

    (147

    )

     

    $

    (143

    )

    Excluding compensation expense related to equity awards and related tax effects

     

     

    (29

    )

     

     

    (31

    )

    Non-GAAP net loss

     

    $

    (118

    )

     

    $

    (112

    )

    Reconciliation of GAAP to Non-GAAP Basis

    As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' non-GAAP results is consistent with how Ionis' management internally evaluates the performance of its operations.

     

    IONIS PHARMACEUTICALS, INC.

    Condensed Consolidated Balance Sheets

    (In Millions)

     

     

     

     

     

     

    March 31,

    2025

    December 31,

    2024

     

     

    (unaudited)

     

    Assets:

     

     

     

    Cash, cash equivalents and short-term investments

     

    $

    2,145

    $

    2,298

    Contracts receivable

     

     

    40

     

    92

    Other current assets

     

     

    234

     

    230

    Property, plant and equipment, net

     

     

    103

     

    94

    Right-of-use assets

     

     

    159

     

    162

    Other assets

     

     

    132

     

    127

    Total assets

     

    $

    2,813

    $

    3,003

     

     

     

     

    Liabilities and stockholders' equity:

     

     

     

    Current portion of deferred contract revenue

     

    $

    81

    $

    79

    Other current liabilities

     

     

    169

     

    229

    1.75% convertible senior notes, net

     

     

    566

     

    565

    0% convertible senior notes, net

     

     

    629

     

    629

    Liability related to sale of future royalties, net

     

     

    536

     

    542

    Long-term lease liabilities

     

     

    159

     

    162

    Long-term obligations, less current portion

     

     

    56

     

    52

    Long-term deferred contract revenue

     

     

    141

     

    157

    Total stockholders' equity

     

     

    476

     

    588

    Total liabilities and stockholders' equity

     

    $

    2,813

    $

    3,003

     

    Key 2025 and 2026 Value Driving Events(1)

    New Product Launches

    Program

    Indication

    2025

    2026

    Donidalorsen (U.S.)

    HAE

    •

     

    TRYNGOLZA (U.S.)

    FCS

    Achieved

     

    WAINZUA (EU)

    ATTRv-PN

    Achieved

     

    Olezarsen (U.S.)

    sHTG

     

    •

    Zilganersen (U.S.)

    Alexander disease

     

    •

    Regulatory Actions

    Program

    Indication

    Regulatory Action

    2025

    2026

    Donidalorsen

    HAE

    U.S. approval decision

    •

     

    EU approval decision

     

    •

    TRYNGOLZA

    FCS

    EU approval decision

    •

     

    Olezarsen

    sHTG

    U.S. submission

    •

     

    U.S. approval decision

     

    •

    Zilganersen

    Alexander disease

    U.S. submission

     

    •

    U.S. approval decision

     

    •

    Nusinersen

    (higher dose)

    SMA

    U.S. and EU submissions

    Achieved

     

    U.S. approval decision

    •

     

    WAINZUA

    ATTRv-PN

    EU approval decision

    Achieved

     

    Pelacarsen

    Lp(a)- CVD

    U.S. submission

     

    •

    Bepirovirsen

    HBV

    Regulatory submission(s)

     

    •

    Regulatory decision(s)

     

    •

    Key Phase 3 Clinical Events

    Program

    Indication

    Event

    2025

    2026

    Olezarsen

    sHTG

    CORE, CORE2 and Essence data

    •

     

    Zilganersen

    Alexander disease

    Phase 3 data

    •

     

    ION582

    Angelman syndrome

    Phase 3 study start

    •

     

    Phase 3 enrollment completion

     

    •

    Pelacarsen

    Lp(a)-CVD

    Lp(a) HORIZON data

     

    •

    Bepirovirsen

    HBV

    B-Well data

     

    •

    Eplontersen

    ATTR-CM

    CARDIO-TTRansform data

     

    •

    Sefaxersen

    IgAN

    IMAGINATION data

     

    •

    Ulefnersen

    FUS-ALS

    FUSION data

     

    •

    (1)

    Timing expectations based on current assumptions and subject to change.

    •

    Indicates that the milestone is anticipated in the respective year.

     

    View source version on businesswire.com: https://www.businesswire.com/news/home/20250430859857/en/

    Ionis Investor Contact:

    D. Wade Walke, Ph.D.

    [email protected]

    760-603-2331

    Ionis Media Contact:

    Hayley Soffer

    [email protected]

    760-603-4679

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    • Ionis Pharmaceuticals Inc. filed SEC Form 8-K: Leadership Update, Financial Statements and Exhibits

      8-K - IONIS PHARMACEUTICALS INC (0000874015) (Filer)

      6/12/25 9:00:44 AM ET
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      8-K - IONIS PHARMACEUTICALS INC (0000874015) (Filer)

      6/10/25 6:02:16 AM ET
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    • Ionis Pharmaceuticals Inc. filed SEC Form 8-K: Other Events, Financial Statements and Exhibits

      8-K - IONIS PHARMACEUTICALS INC (0000874015) (Filer)

      5/19/25 7:18:21 AM ET
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    • Director Hayden Michael R bought $477,900 worth of shares (15,000 units at $31.86), increasing direct ownership by 43% to 50,219 units (SEC Form 4)

      4 - IONIS PHARMACEUTICALS INC (0000874015) (Issuer)

      5/2/25 4:38:26 PM ET
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    • Director Hayden Michael R bought $181,079 worth of shares (5,000 units at $36.22), increasing direct ownership by 17% to 35,219 units (SEC Form 4)

      4 - IONIS PHARMACEUTICALS INC (0000874015) (Issuer)

      12/26/24 4:57:52 PM ET
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    • Ionis Pharma upgraded by Barclays with a new price target

      Barclays upgraded Ionis Pharma from Equal Weight to Overweight and set a new price target of $57.00

      7/1/25 8:12:05 AM ET
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    • H.C. Wainwright initiated coverage on Ionis Pharma with a new price target

      H.C. Wainwright initiated coverage of Ionis Pharma with a rating of Buy and set a new price target of $45.00

      4/7/25 8:43:57 AM ET
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    • Redburn Atlantic initiated coverage on Ionis Pharma with a new price target

      Redburn Atlantic initiated coverage of Ionis Pharma with a rating of Neutral and set a new price target of $39.00

      3/31/25 8:13:04 AM ET
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    • New Data for Nusinersen Underscore Biogen's Commitment to Advancing Clinical Research to Improve Outcomes in SMA

      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

      6/27/25 10:00:23 AM ET
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    • Ionis announces Biogen to advance salanersen into SMA registrational studies based on positive interim Phase 1 results

      – Investigational salanersen (ION306/BIIB115) developed using novel Ionis antisense chemistry with the potential to achieve high efficacy and once-yearly dosing – – 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 salanersen – – Biogen is engaging with regulators to advance salanersen to registrational stage studies – Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that its partner, Biogen, shared positive topline results from the Phase 1 study of salanersen (ION306/BIIB115), an investigational antisense oligo

      6/25/25 7:31:00 AM ET
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    • Biogen to Advance Investigational Spinal Muscular Atrophy Asset to Registrational Studies Based on Positive Interim Phase 1 Results

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      6/25/25 7:30:37 AM ET
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    • SEC Form 4 filed by Director Yang Michael J.

      4 - IONIS PHARMACEUTICALS INC (0000874015) (Issuer)

      7/2/25 4:53:41 PM ET
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    • SEC Form 4 filed by Director Parshall B Lynne

      4 - IONIS PHARMACEUTICALS INC (0000874015) (Issuer)

      7/2/25 4:53:31 PM ET
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    • SEC Form 4 filed by Director Wender Joseph H

      4 - IONIS PHARMACEUTICALS INC (0000874015) (Issuer)

      7/2/25 4:53:35 PM ET
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    • Alltrna Announces Updates to Its Board of Directors

      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

      7/17/24 8:00:00 AM ET
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    • Contineum Therapeutics Announces Appointment of Sarah Boyce to Board of Directors

      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

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    • Ionis announces the appointment of Michael Yang to Board of Directors

      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

      12/14/23 4:05:00 PM ET
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    • Amendment: SEC Form SC 13G/A filed by Ionis Pharmaceuticals Inc.

      SC 13G/A - IONIS PHARMACEUTICALS INC (0000874015) (Subject)

      11/14/24 8:34:57 PM ET
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    • Amendment: SEC Form SC 13G/A filed by Ionis Pharmaceuticals Inc.

      SC 13G/A - IONIS PHARMACEUTICALS INC (0000874015) (Subject)

      11/14/24 1:28:29 PM ET
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    • SEC Form SC 13G filed by Ionis Pharmaceuticals Inc.

      SC 13G - IONIS PHARMACEUTICALS INC (0000874015) (Subject)

      11/8/24 10:52:38 AM ET
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    • Biogen to Advance Investigational Spinal Muscular Atrophy Asset to Registrational Studies Based on Positive Interim Phase 1 Results

      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

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    • Ionis reports first quarter 2025 financial results

      - 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

      4/30/25 7:00:00 AM ET
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    • Praxis Precision Medicines Provides Corporate Update and Reports Second Quarter 2024 Financial Results

      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

      8/13/24 7:00:00 AM ET
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