IONS Ionis Pharmaceuticals Inc.

42.36
-0.33  -1%
Previous Close 42.69
Open 42.5
52 Week Low 41.42
52 Week High 64.37
Market Cap $5,966,923,258
Shares 140,862,211
Float 118,588,562
Enterprise Value $4,960,606,787
Volume 2,121,113
Av. Daily Volume 1,313,631
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Upcoming Catalysts

Drug Stage Catalyst Date
IONIS-AGT-LRx
Hypertension
Phase 2
Phase 2
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IONIS-GHR-LRx
Acromegaly
Phase 2
Phase 2
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IONIS-ENaC-2.5Rx
Cystic fibrosis
Phase 1/2
Phase 1/2
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IONIS-MAPTRx
Alzheimer's disease
Phase 1/2
Phase 1/2
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Vupanorsen (AKCEA-ANGPTL3-LRx) - (TRANSLATE-TIMI 70)
Cardiovascular (CV) risk reduction and hypertriglyceridemia
Phase 2b
Phase 2b
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IONIS-C9Rx
Amyotrophic lateral sclerosis (ALS)
Phase 1/2
Phase 1/2
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Tofersen
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3
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AKCEA-TTR-LRx - CARDIO-TTRansform
ATTR cardiomyopathy
Phase 3
Phase 3
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AKCEA-TTR-LRx (NEURO-TTRansform)
hATTR Amyloidosis
Phase 3
Phase 3
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AKCEA-APO(a)-LRx
Hyperlipoproteinemia(a) and established cardiovascular disease
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ION363
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3
Phase 3 trial initiation announced April 5, 2021.
IONIS-PKK-LRx
Hereditary Angioedema
Phase 2
Phase 2
Phase 2 data released March 29, 2021, Primary endpoint met.
Tominersen (IONIS-HTT Rx)
Huntington's Disease
Phase 3
Phase 3
Phase 3 trial to be discontinued following review of data by an Independent Data Monitoring Committee.
ION449 (AZD8233)
Dyslipidaemia
Phase 2b
Phase 2b
Phase 2b trial initiation announced November 30, 2020.
AKCEA-APOCIII-LRx (BALANCE)
Familial Chylomicronemia Syndrome (FCS)
Phase 3
Phase 3
Phase 3 trial initiation announced December 1, 2020.
Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
CRL
CRL
CRL issued August 27, 2018. To be refiled.
SPINRAZA (nusinersen) - high dose
Spinal muscular atrophy (SMA)
Phase 2/3
Phase 2/3
Phase 2/3 commencement of dosing announced April 2, 2020.
Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Approved
Approved
FDA Approval announced October 5, 2018,
Volanesorsen
Familial partial lipodystrophy (FPL)
Phase 3
Phase 3
Phase 3 data met primary endpoint - August 6, 2019.
SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Approved
Approved
Approved December 23, 2016.

Latest News

  1. CARLSBAD, Calif., April 8, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today the pricing of $550.0 million aggregate principal amount of 0% Convertible Senior Notes due 2026 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). The aggregate principal amount of the offering was increased from the previously announced offering size of $500.0 million. Ionis also granted the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the date on which the notes are first issued, up to an additional $82.5 million aggregate principal amount of notes…

    CARLSBAD, Calif., April 8, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today the pricing of $550.0 million aggregate principal amount of 0% Convertible Senior Notes due 2026 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). The aggregate principal amount of the offering was increased from the previously announced offering size of $500.0 million. Ionis also granted the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the date on which the notes are first issued, up to an additional $82.5 million aggregate principal amount of notes from Ionis. The sale of the notes is expected to close on April 12, 2021, subject to customary closing conditions.

    The notes will be general unsecured obligations of Ionis, will not bear regular interest and the principal amount of the notes will not accrete. The notes will mature on April 1, 2026, unless earlier converted or repurchased.

    Ionis estimates that the net proceeds from the offering will be approximately $536.2 million (or approximately $616.8 million if the initial purchasers exercise their option to purchase additional notes in full), after deducting the initial purchasers' discounts and commissions and estimated offering expenses payable by Ionis. Ionis expects to use approximately $257.0 million of the net proceeds from the offering to repurchase approximately $247.9 million in aggregate principal amount of its 1% Convertible Senior Notes due 2021 (the "2021 notes") in privately negotiated transactions. In addition, Ionis expects to use approximately $40.8 million of the net proceeds from the offering to pay the cost of the convertible note hedge transactions described below (after such cost is partially offset by the proceeds to Ionis from the sale of the warrant transactions described below). Ionis expects to use the remaining net proceeds from the offering for general corporate purposes, including expansion of manufacturing, research and development, and commercial infrastructure to support its wholly owned pipeline. 

    Before January 1, 2026, holders will have the right to convert their notes only upon the satisfaction of specified conditions and during certain periods. On or after January 1, 2026 until the close of business on the second scheduled trading day immediately preceding the maturity date, holders may convert all or any portion of their notes at any time. Upon conversion, Ionis will pay or deliver, as the case may be, cash, shares of its common stock or a combination of cash and shares of its common stock, at its election. The conversion rate will initially be 17.2902 shares of Ionis' common stock per $1,000 principal amount of notes (equivalent to an initial conversion price of approximately $57.84 per share of Ionis' common stock). The initial conversion price represents a premium of approximately 32.5% over the last reported sale price of $43.65 per share of Ionis' common stock on April 7, 2021. The conversion rate will be subject to adjustment in some events but will not be adjusted for any accrued or unpaid special interest.

    Ionis may not redeem the notes prior to the maturity date, and no sinking fund is provided for the notes.

    If Ionis undergoes a "fundamental change" (as defined in the indenture for the notes), then, subject to certain conditions and limited exceptions, holders may require Ionis to repurchase for cash all or any portion of their notes at a fundamental change repurchase price equal to 100% of the principal amount of the notes to be repurchased, plus accrued and unpaid special interest, if any, to, but excluding, the fundamental change repurchase date. In addition, following certain corporate events that occur prior to the maturity date, Ionis will, in certain circumstances, increase the conversion rate for a holder who elects to convert its notes in connection with such a corporate event.

    In connection with the pricing of the notes, Ionis entered into convertible note hedge transactions with certain of the initial purchasers or their affiliates and other financial institutions (the "Option Counterparties"). Ionis also entered into warrant transactions with the Option Counterparties. The convertible note hedge transactions are generally expected to reduce potential dilution to Ionis' common stock upon any conversion of notes and/or offset any cash payments Ionis is required to make in excess of the principal amount of converted notes, as the case may be.  However, the warrant transactions would separately have a dilutive effect to the extent that the market price per share of Ionis' common stock exceeds the strike price of any warrants. The strike price for the warrant transactions will initially be equal to approximately $76.39 per share, which represents a 75.0% premium to the closing sale price of Ionis' common stock on April 7, 2021. If the initial purchasers exercise their option to purchase additional notes, Ionis expects to enter into additional convertible note hedge transactions and additional warrant transactions relating to the additional notes with the Option Counterparties.

    In connection with establishing their initial hedges of the convertible note hedge and warrant transactions, Ionis expects that the Option Counterparties or their respective affiliates will purchase shares of Ionis' common stock and/or enter into various derivative transactions with respect to Ionis' common stock concurrently with or shortly after the pricing of the notes. This activity could increase (or reduce the size of any decrease in) the market price of Ionis' common stock or the notes at that time.

    In addition, the Option Counterparties or their respective affiliates may modify their hedge positions by entering into or unwinding various derivatives with respect to Ionis' common stock and/or by purchasing or selling Ionis' common stock or other securities of Ionis in secondary market transactions following the pricing of the notes and prior to the maturity of the notes (and are likely to do so during any observation period related to a conversion of notes or in connection with any repurchase of notes by Ionis on any fundamental change repurchase date or otherwise). This activity could also cause or avoid an increase or a decrease in the market price of Ionis' common stock or the notes, which could affect the ability of noteholders to convert the notes and, to the extent the activity occurs during any observation period related to a conversion of notes, it could affect the amount and value of the consideration that noteholders will receive upon conversion of such notes.

    Further, in connection with any repurchases of the 2021 notes, Ionis expects that holders of the 2021 notes who agree to have their 2021 notes repurchased and who have hedged their equity price risk with respect to such notes (the "hedged holders") will unwind all or part of their hedge positions by buying Ionis' common stock and/or entering into or unwinding various derivative transactions with respect to Ionis' common stock. The amount of Ionis' common stock to be purchased by the hedged holders may be substantial in relation to the historic average daily trading volume of Ionis' common stock. This activity by the hedged holders could increase (or reduce the size of any decrease in) the market price of Ionis' common stock, including concurrently with the pricing of the notes, resulting in a higher effective conversion price of the notes. Ionis cannot predict the magnitude of such market activity or the overall effect it may have had on the price of the notes in the offering or Ionis' common stock.

    The notes, the warrants and any shares of common stock issuable upon conversion of the notes or exercise of the warrants have not been and will not be registered under the Securities Act, any state securities laws or the securities laws of any other jurisdiction, and unless so registered, may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

    This press release is neither an offer to sell nor a solicitation of an offer to buy any of these securities nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to the registration or qualification thereof under the securities laws of any such state or jurisdiction.

    About Ionis Pharmaceuticals

    For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

    Ionis' Forward-looking Statement

    This press release includes forward-looking statements regarding the offering and the convertible note hedge and warrant transactions, including statements regarding the anticipated completion and timing of the offering and the convertible note hedge and warrant transactions and Ionis' expected use of proceeds from the offering. Any statement describing Ionis' expectations, 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, without limitation, changes in market conditions, whether Ionis will be able to satisfy closing conditions related to the offering, whether and on what terms Ionis may repurchase any of the 2021 notes and unanticipated uses of capital. 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. 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, 2020, which is 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" refers to Ionis Pharmaceuticals and its subsidiaries.

    Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-prices-private-placement-of-convertible-senior-notes-301264710.html

    SOURCE Ionis Pharmaceuticals, Inc.

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  2. CARLSBAD, Calif., April 6, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it intends to offer, subject to market conditions and other factors, $500.0 million aggregate principal amount of Convertible Senior Notes due 2026 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). Ionis also intends to grant the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the first date on which the notes are issued, up to an additional $75.0 million principal amount of notes.

    CARLSBAD, Calif., April 6, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that it intends to offer, subject to market conditions and other factors, $500.0 million aggregate principal amount of Convertible Senior Notes due 2026 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). Ionis also intends to grant the initial purchasers of the notes an option to purchase, within the 13-day period beginning on, and including, the first date on which the notes are issued, up to an additional $75.0 million principal amount of notes.

    The notes will be general unsecured obligations of Ionis and will accrue interest payable semiannually in arrears. Upon conversion, Ionis will pay or deliver, as the case may be, cash, shares of its common stock or a combination of cash and shares of its common stock, at its election. The interest rate, initial conversion rate and other terms of the notes will be determined at the time of pricing of the offering.

    Ionis expects to use a portion of the net proceeds from the offering to repurchase for cash certain of its 1% Convertible Senior Notes due 2021 (the "2021 notes") in privately negotiated transactions. In addition, Ionis expects to use a portion of the net proceeds from the offering to pay the cost of the convertible note hedge transactions described below (after such cost is partially offset by the proceeds to Ionis from the sale of the warrant transactions described below). Ionis expects to use the remaining net proceeds from the offering for general corporate purposes, including expansion of manufacturing, research and development, and commercial infrastructure to support its wholly owned pipeline.

    In connection with the pricing of the notes, Ionis expects to enter into convertible note hedge transactions with one or more of the initial purchasers or their affiliates and/or other financial institutions (the "Option Counterparties"). The convertible note hedge transactions are generally expected to reduce potential dilution to Ionis common stock upon any conversion of notes and/or offset any cash payments Ionis is required to make in excess of the principal amount of converted notes, as the case may be. Ionis also expects to enter into privately negotiated warrant transactions with the Option Counterparties. The warrant transactions would separately have a dilutive effect to the extent that the market price per share of Ionis common stock exceeds the strike price of the warrants. If the initial purchasers exercise their option to purchase additional notes, Ionis expects to enter into additional convertible note hedge and additional warrant transactions relating to the additional notes with the Option Counterparties.

    In connection with establishing their initial hedges of the convertible note hedge and warrant transactions, Ionis expects that the Option Counterparties or their respective affiliates will purchase shares of Ionis common stock and/or enter into various derivative transactions with respect to Ionis' common stock concurrently with or shortly after the pricing of the notes. This activity could increase (or reduce the size of any decrease in) the market price of Ionis common stock or the notes at that time.

    In addition, the Option Counterparties or their respective affiliates may modify their hedge positions by entering into or unwinding various derivatives with respect to Ionis common stock and/or by purchasing or selling Ionis' common stock or other securities of Ionis in secondary market transactions following the pricing of the notes and prior to the maturity of the notes (and are likely to do so during any observation period related to a conversion of notes or in connection with any repurchase of notes by Ionis on any fundamental change repurchase date or otherwise). This activity could also cause or avoid an increase or a decrease in the market price of Ionis' common stock or the notes, which could affect the ability of noteholders to convert the notes and, to the extent the activity occurs during any observation period related to a conversion of notes, it could affect the amount and value of the consideration that noteholders will receive upon conversion of such notes.

    Further, in connection with any repurchases of the 2021 notes, Ionis expects that holders of the 2021 notes who agree to have their 2021 notes repurchased and who have hedged their equity price risk with respect to such notes (the "hedged holders") will unwind all or part of their hedge positions by buying Ionis' common stock and/or entering into or unwinding various derivative transactions with respect to Ionis' common stock. The amount of Ionis' common stock to be purchased by the hedged holders may be substantial in relation to the historic average daily trading volume of Ionis' common stock. This activity by the hedged holders could increase (or reduce the size of any decrease in) the market price of Ionis' common stock, including concurrently with the pricing of the notes, resulting in a higher effective conversion price of the notes. Ionis cannot predict the magnitude of such market activity or the overall effect it will have on the price of the notes offered hereby or Ionis' common stock.

    The notes, the warrants and any shares of common stock issuable upon conversion of the notes or exercise of the warrants have not been and will not be registered under the Securities Act, any state securities laws or the securities laws of any other jurisdiction, and unless so registered, may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

    This press release is neither an offer to sell nor a solicitation of an offer to buy any of these securities nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to the registration or qualification thereof under the securities laws of any such state or jurisdiction.

    About Ionis Pharmaceuticals

    For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

    Ionis' Forward-looking Statement

    This press release includes forward-looking statements regarding the proposed offering and the convertible note hedge and warrant transactions, including statements regarding the anticipated terms of the proposed offering and the convertible note hedge and warrant transactions and Ionis' expected use of proceeds from the proposed offering. Any statement describing Ionis' expectations, 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, without limitation, changes in market conditions, Ionis' ability to complete the proposed offering on the expected terms, or at all, whether Ionis will be able to satisfy closing conditions related to the proposed offering, whether and on what terms Ionis may repurchase any of the 2021 notes and unanticipated uses of capital. 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. 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, 2020, which is on file with the SEC. Copies of this and other documents are available from the Company.

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

    Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-announces-proposed-private-placement-of-convertible-notes-301263499.html

    SOURCE Ionis Pharmaceuticals, Inc.

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  3. CARLSBAD, Calif., April 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene (FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred to as FUS-ALS, which is the most common cause of juvenile-onset ALS. There is substantial evidence that mutations in the FUS gene are responsible for a toxic gain of function that can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS. ION363 is an investigational antisense medicine targeting the FUS RNA to reduce the production of the FUS protein. Antisense-mediated reduction of mutant…

    CARLSBAD, Calif., April 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene (FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred to as FUS-ALS, which is the most common cause of juvenile-onset ALS. There is substantial evidence that mutations in the FUS gene are responsible for a toxic gain of function that can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS. ION363 is an investigational antisense medicine targeting the FUS RNA to reduce the production of the FUS protein. Antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model prevents motor neuron loss. By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients.

    ALS is a rare, rapidly progressing and fatal neurodegenerative disorder that affects approximately 55,000 people globally.i FUS-ALS is the third most common genetic cause of ALS. People with ALS experience muscle weakness, loss of movement, and difficulty breathing and swallowing, resulting in a severely declining quality of life and eventually death.

    "There is an urgent need for novel treatments for all forms of ALS, a devastating disease that affects far too many patients and their families. Advancement of ION363 to a pivotal trial is the latest example of the power of Ionis' antisense technology to potentially target the root causes of neurological diseases," said C. Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs. "Driven by our experience in developing medicines for motor neuron diseases such as ALS and spinal muscular atrophy and our intimate connection to the ALS patient community, Ionis made the decision to advance ION363 to the clinic and, ultimately, to the market because we believe we are uniquely positioned to make it available to patients living with FUS-ALS."

    The trial will be led by Neil Shneider, M.D., Ph.D., director of Columbia University's Eleanor and Lou Gehrig ALS Center. Ionis began collaborating with Dr. Shneider when the company learned of his efforts to develop a treatment for Jaci Hermstad, an Iowa woman living with FUS-ALS. Ionis shared its research and expertise with Dr. Shneider, resulting in an experimental treatment designed specifically for Jaci. Inspired by Jaci's spirit and courage, Ionis made the decision to invest in clinical studies so that many more patients can gain access to ION363, also known as "jacifusen." Since Jaci, several FUS-ALS patients have received treatment with ION363 under Dr. Shneider's investigator-initiated study through the U.S. Food and Drug Administration's expanded access pathway, sometimes called "compassionate use."

    "FUS-ALS is an atypically aggressive form of the disease, involving the youngest of ALS patients. Building on our expanded access program, a controlled clinical trial is the best way to demonstrate the efficacy of ION363 and to make this therapeutic available to all patients who could potentially benefit from it," said Dr. Shneider.

    The Phase 3 trial of ION363 is a global, multi-center study in up to 64 patients. Part one of the trial will consist of patients randomized to receive a multi-dose regimen of ION363 or placebo for 29 weeks, followed by part two, which will be an open-label period in which all patients in the trial will receive ION363 for 73 weeks.

    Learn more about the Phase 3 trial of ION363 at: https://clinicaltrials.gov/ct2/show/NCT04768972?term=ion363&draw=2&rank=1

    Ionis' other leading investigational medicines to treat ALS are tofersen (BIIB067), IONIS-C9Rx (BIIB078) and ION541 (BIIB105).

    About Ionis' Neurology Franchise

    The Ionis neurology franchise addresses all major brain regions and central nervous system types and currently has three Phase 3 studies ongoing with eight medicines in clinical development, five of which are wholly owned. Ionis is leading the way in treating the root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's and Parkinson's as well as rare diseases like amyotrophic lateral sclerosis (ALS) and Alexander disease. Ionis' marketed neurological disease medicines include SPINRAZA®, the global foundation of care for spinal muscular atrophy (SMA), commercialized by Biogen, and TEGSEDI®, the first and only self-administered, subcutaneous treatment for the polyneuropathy of hereditary ATTR amyloidosis in adults.

    About Ionis Pharmaceuticals

    For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

    To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.

    Ionis' Forward-looking Statement

    This press release includes forward-looking statements regarding Ionis' business, Ionis' technologies, tofersen, IONIS-C9Rx, ION541, ION363 and other products in development. 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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. 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, 2020, which is on file with the SEC. Copies of this and other documents are available from the Company.

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

    Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

    Tofersen, IONIS-C9Rx and ION541 are partnered with Biogen.

    i France, Germany, Italy, Japan, Spain, the United Kingdom and the United States.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-initiates-phase-3-trial-of-novel-antisense-medicine-to-treat-leading-cause-of-juvenile-onset-als-301261713.html

    SOURCE Ionis Pharmaceuticals, Inc.

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  4. CARLSBAD, Calif., March 29, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced today that data from a Phase 2 clinical study of IONIS-PKK-LRx met its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with hereditary angioedema (HAE) compared to placebo. The study demonstrated a mean reduction of 90% in the number of monthly HAE attacks in weeks one to 17 of the study (p <0.001) and a mean reduction of 97% in the number of monthly HAE attacks in weeks five to 17 (p=0.003). In weeks five to 17, 92% of patients treated with IONIS-PKK-LRx were attack-free compared to 0% in the placebo group (p <0.001).

    IONIS-PKK-LRx was developed using Ionis' advanced ligand-conjugated antisense (LICA) technology. IONIS-PKK-LRx is one of Ionis' wholly owned medicines to treat rare diseases.

    Hereditary angioedema is a rare autosomal dominant disease that results in recurrent, painful attacks of swelling affecting the arms, legs, face, intestinal track and airway. IONIS-PKK-LRx is an investigational antisense medicine designed to reduce the production of prekallikrein, or PKK, which plays a key role in the activation of inflammatory mediators associated with acute attacks of HAE. Without preventive treatment, HAE attacks can be frequent and severe and, in some patients, life-threatening. Physicians have long prescribed prophylactic treatment approaches, including C1-INH replacement therapies and more recently inhibitors of plasma kallikrein, to prevent and reduce the severity of HAE attacks. Despite these available therapies, patients with HAE may experience breakthrough attacks.

    "These topline Phase 2 study results support a profile for IONIS-PKK-LRx as a potential best-in-class prophylactic treatment for patients with HAE, with excellent efficacy, safety and tolerability along with the convenience of once per month low volume subcutaneous injections," said Kenneth Newman, M.D., M.B.A., Ionis' vice president of clinical development and leader of the pulmonology and immunology franchise. "These results highlight the potential benefits and advantages of IONIS-PKK-LRx for the treatment of hereditary angioedema and more broadly underscore the power of Ionis' antisense technology to target the root causes of rare diseases like HAE."

    In the Phase 2 clinical study, 20 adults with Type 1 or Type 2 HAE were randomized and received either IONIS-PKK-LRx 80mg (n=14) or placebo (n=6) subcutaneously once monthly for 17 weeks. The primary endpoint was the reduction of monthly HAE attacks compared to placebo. Secondary endpoints included the reduction of monthly attacks  in weeks five to 17, reduction in the number of moderate or severe attacks in weeks one to 17, the number of moderate or severe attacks in weeks five to 17 and the number of attacks requiring acute therapy in weeks five to 17. The majority of adverse events during the study were mild with a frequency that was similar between groups. The most common treatment-emergent adverse events (TEAEs) were headache and nausea, which were seen more frequently in the placebo arm compared to the active treatment arm. Ionis expects to present a full analysis of its Phase 2 study of IONIS-PKK-LRx at a medical conference later this year.

    Ionis will host a series of webinars on rare diseases for which the company is developing potential treatments. The first webinar, on hereditary angioedema, is scheduled for Monday, April 5. Visit www.ionispharma.com for more information and to register for the webinar.

    About Ionis Pharmaceuticals

    For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

    To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.

    Ionis' Forward-looking Statement

    This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of IONIS-PKK-LRx and Ionis' technologies and products in development. 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 related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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. 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, 2020, which is on file with the SEC. Copies of this and other documents are available from the Company.

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

    Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-reports-positive-topline-phase-2-study-results-of-its-novel-antisense-treatment-for-hereditary-angioedema-301257232.html

    SOURCE Ionis Pharmaceuticals, Inc.

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  5. CARLSBAD, Calif., March 26, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in RNA-targeted therapeutics, today announced that management will participate in virtual fireside chats at the following investor conferences:

    • 3rd Annual Stifel CNS Day on Thursday, April 1, 2021
    • 20th Annual Needham Healthcare Conference on Monday, April 12, 2021

    The above listed dates are subject to change. Details on presentation times or changes to presentation dates can be found on the Company's website. Please check www.ionispharma.com for the latest information.

    A live webcast of the presentations will be available on the Investors & Media section of the Ionis website. The replays will be available within 48 hours and will be archived for a limited time.

    About Ionis Pharmaceuticals

    For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.

    To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-to-present-at-upcoming-investor-conferences-301256446.html

    SOURCE Ionis Pharmaceuticals, Inc.

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