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1. 30 September 2020 Ionis treatment for Alexander disease receives orphan drug designation from U.S. FDA

   CARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating rare neurodegenerative disease that can result in death. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood. Alexander disease has been estimated to occur in about one in one million births. Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer…

   CARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating rare neurodegenerative disease that can result in death. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood. Alexander disease has been estimated to occur in about one in one million births. Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.

   

   Alexander disease is caused by a mutation in a protein resulting in overproduction of glial fibrillary acidic protein (GFAP) in the brain. ION373 is an Ionis-owned investigational antisense medicine designed to stop the mutated gene from producing excess GFAP.

   Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including waiver of certain administrative fees, grants and tax credits for clinical trials, and seven years of market exclusivity following drug approval. Earlier this year, the European Medicines Agency (EMA) granted orphan drug designation to ION373. EMA provides regulatory, financial and market incentives to develop therapies for life threatening or chronically debilitating conditions affecting not more than five in 10,000 people in the European Union (EU) and for which there is no satisfactory method of diagnosis, prevention or treatment.

   "Receiving FDA orphan drug status for ION373 reflects the urgent need for a novel medicine to treat Alexander disease. We look forward to working closely with regulators, clinical investigators, Alexander disease patients and their families to advance this important medicine and make it available to those who need it," said Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs.

   ION373 is one of several Ionis-owned investigational medicines designed to treat neurological diseases. Others include ION716 (Prion disease), ION283 (Lafora disease) and ION363 (amyotrophic lateral sclerosis or ALS).

   About Alexander disease

   Alexander disease (AxD) is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that protects a nerve fiber and supports signal conduction). Two major types of AxD have been defined. Type I onset typically occurs before 4 years of age and patients can experience head enlargement, seizures, limb stiffness, delayed or declining cognition, and lack of growth. Type II onset typically occurs after the age of 4 and symptoms can include difficulty speaking, swallowing, and making coordinated movements. AxD is most often fatal. There are treatments that can relieve symptoms, but there is no disease modifying therapy yet available to patients.

   About Ionis Pharmaceuticals, Inc.

   As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for all patients, children and adults with spinal muscular atrophy, as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.

   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, financial guidance and the therapeutic and commercial potential of ION373 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, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. 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, 2019, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. 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.

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   SOURCE Ionis Pharmaceuticals, Inc.

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2. 03 September 2020 Ionis to present at upcoming virtual investor conferences

   CARLSBAD, Calif., Sept. 3, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics, today announced that management will present a company overview at the following virtual investor conferences:

   

   • Wells Fargo 2020 Healthcare Conference on Thursday, September 10, 2020;
   • Morgan Stanley 18^th Annual Global Healthcare Conference on Monday, September 14, 2020;
   • BofA Global Healthcare Conference on Friday, September 18, 2020; and
   • Oppenheimer Fall Healthcare Life Sciences & MedTech Summit on Tuesday, September 22, 2020.

   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
   
   As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.

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

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   SOURCE Ionis Pharmaceuticals, Inc.

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3. 02 September 2020 New England Journal of Medicine publishes study results evaluating Ionis antisense therapy in treatment of patients with hereditary angioedema

   CARLSBAD, Calif., Sept. 2, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced the publication today of the results from a compassionate-use study evaluating IONIS-PKK_Rx and IONIS-PKK-L_Rx in patients living with severe bradykinin-mediated angioedema in The New England Journal of Medicine (NEJM). IONIS-PKK_Rx and IONIS-PKK-L_Rx are investigational antisense medicines 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 hereditary angioedema (HAE). In the study, researchers found that the drugs reduced plasma prekallikrein activity levels and showed evidence of clinical efficacy in reducing the number of breakthrough attacks…

   CARLSBAD, Calif., Sept. 2, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced the publication today of the results from a compassionate-use study evaluating IONIS-PKK_Rx and IONIS-PKK-L_Rx in patients living with severe bradykinin-mediated angioedema in The New England Journal of Medicine (NEJM). IONIS-PKK_Rx and IONIS-PKK-L_Rx are investigational antisense medicines 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 hereditary angioedema (HAE). In the study, researchers found that the drugs reduced plasma prekallikrein activity levels and showed evidence of clinical efficacy in reducing the number of breakthrough attacks per month in patients over the course of the treatment, including complete resolution in a patient with Type 1 HAE. To view the published study, please visit NEJM.org.

   

   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. Without preventive treatment, attacks can be frequent and severe and, in some patients, life-threatening. The majority of HAE cases are caused by genetic mutations that lead to either a deficiency (Type 1 HAE) or dysfunction (Type 2 HAE) of C1 esterase inhibitor (C1-INH), which regulates multiple pathways, including the kallikrein-kinin and contact system. In the third, especially rare form of the disorder (Type 3 HAE or HAE-nC1-INH), which occurs predominantly in women, and in which the cause is often unknown, patients have a higher frequency of facial, pharyngeal and tongue swelling.

   "The results of this study are encouraging and support continued development of IONIS-PKK-L_Rx as a potential treatment in patients with severe hereditary angioedema for whom current therapies offer limited therapeutic benefit. The study also highlights the progress we continue to make advancing investigational medicines that are wholly owned by Ionis," said Richard S. Geary, Ph.D., Ionis' executive vice president of Development and a co-author on the paper published in NEJM.

   In the study, two patients – Patient 1 with Type 1 HAE and Patient 2 with Type 3 HAE – were first treated with IONIS-PKK_Rx for a period of 12 to 16 weeks, after which they received IONIS-PKK-L_Rx at a dose of 80 mg every three to four weeks for seven to eight months at the time of data analysis. During treatment with the ligand-conjugated IONIS-PKK-L_Rx and the unconjugated parent drug, IONIS-PKK_Rx, there was a clinically meaningful reduction in HAE attack rates in both patients. Plasma prekallikrein activity levels decreased substantially following treatment.

   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. IONIS-PKK-L_Rx is an investigational antisense medicine that is being developed because it has the potential to provide significant efficacy with the convenience of once per month low volume subcutaneous injections.

   Ionis' Forward-looking Statement

   This press release includes forward-looking statements regarding Ionis' business and the therapeutic potential of IONIS-PKK-L_Rx, IONIS-PKK_Rx 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, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. 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, 2019, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. 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.

   About Ionis Pharmaceuticals

   As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.

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

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   SOURCE Ionis Pharmaceuticals, Inc.

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4. 31 August 2020 Ionis Pharmaceuticals to acquire remaining stake in Akcea Therapeutics

   CARLSBAD, Calif. and BOSTON, Aug. 31, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and its affiliate Akcea Therapeutics, Inc. (NASDAQ:AKCA) today announced that they have entered into a definitive agreement under which Ionis will acquire all of the outstanding shares of Akcea common stock it does not already own, approximately 24%, for $18.15 per share in cash. This corresponds to a total transaction value of approximately $500 million on a fully diluted basis. The transaction has been approved by the Ionis and Akcea Boards of Directors, and by the independent Affiliate Transactions Committee of Akcea's Board of Directors. Akcea's Board of Directors has recommended to shareholders of Akcea that they tender their shares into…

   CARLSBAD, Calif. and BOSTON, Aug. 31, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) and its affiliate Akcea Therapeutics, Inc. (NASDAQ:AKCA) today announced that they have entered into a definitive agreement under which Ionis will acquire all of the outstanding shares of Akcea common stock it does not already own, approximately 24%, for $18.15 per share in cash. This corresponds to a total transaction value of approximately $500 million on a fully diluted basis. The transaction has been approved by the Ionis and Akcea Boards of Directors, and by the independent Affiliate Transactions Committee of Akcea's Board of Directors. Akcea's Board of Directors has recommended to shareholders of Akcea that they tender their shares into the tender offer.

   

   "This acquisition is another step forward in Ionis' evolution and creates a stronger, more efficient organization to the benefit of all stakeholders," said Brett Monia, Chief Executive Officer of Ionis. "We believe becoming one company – with one vision and one set of strategic priorities, led by one team – will deliver significant strategic value, enhancing the future success of our company, accelerating our next phase of growth and positioning us to most effectively deliver our medicines to patients. Following the completion of the transaction, Ionis will retain more value from Akcea's rich pipeline and commercial products, further strengthening our financial position and supporting continued investments in our future." 

   "Akcea values the skills, experience and expertise of our teams and I am proud of what we've accomplished in developing and delivering transformational medicines to patients," said Damien McDevitt, Chief Executive Officer of Akcea. "This acquisition by Ionis is a testament to the achievements of the Akcea team."

   "This transaction delivers immediate and certain value with a premium to Akcea shareholders," said Barbara Yanni, Chair of the Akcea Affiliate Transactions Committee. "Our close collaboration with Ionis has been key to enabling the launch of two commercial antisense medicines in just two years as well as advancing a late-stage pipeline and securing important partnerships."

   SIGNIFICANT FINANCIAL BENEFITS

   This transaction also further strengthens Ionis' financial position:

   • Ionis will realize more financial upside from Akcea's rich pipeline and commercial products.
   • Ionis will gain full access to Akcea's significant cash on hand of approximately $390 million as of June 30, 2020 and future cash flows to further invest in the Company's future and further support Ionis' capital allocation strategy.
   • Ionis will achieve meaningful cost synergies.

   TERMS OF THE AGREEMENT

   Under the terms of the agreement, Ionis will commence a tender offer for all outstanding shares of Akcea common stock not already owned by Ionis at a price of $18.15 per share in cash. The closing of the tender offer will be subject to a majority of Akcea's shares not already owned by Ionis, its affiliates or their respective directors and executive officers being tendered in the tender offer. 

   Promptly following the completion of the tender offer, Ionis will acquire all remaining shares of Akcea common stock at the same price of $18.15 per share in cash through a second-step merger. Ionis and Akcea expect to complete the transaction in the fourth quarter of 2020, subject to other customary closing conditions.

   Ionis expects to fund the acquisition through existing cash resources. The transaction is not subject to any financing condition.

   ADVISORS

   Goldman Sachs & Co. LLC and Stifel, Nicolaus & Company, Incorporated are serving as financial advisors to Ionis, and Skadden, Arps, Slate, Meagher & Flom LLP is serving as legal counsel to Ionis. Cowen is serving as financial advisor to the Affiliate Transactions Committee of Akcea's Board of Directors, and Ropes & Gray LLP is serving as legal counsel to the Affiliate Transactions Committee of Akcea's Board of Directors.

   ABOUT IONIS PHARMACEUTICALS, INC.

   As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.

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

   ABOUT AKCEA THERAPEUTICS, INC.

   Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing medicines to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen), as well as advancing a mature pipeline of novel medicines, including AKCEA-APO(a)-L_Rx, vupanorsen (AKCEA-ANGPTL3-L_Rx), AKCEA-APOCIII-L_Rx, and AKCEA-TTR-L_Rx, with the potential to treat multiple diseases. All six medicines were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. TEGSEDI is approved in the U.S., E.U., Canada and Brazil, and WAYLIVRA is approved in the E.U. Akcea is headquartered in Boston, Massachusetts, and is building the infrastructure to commercialize its medicines globally. Additional information about Akcea is available at www.akceatx.com and you can follow the Company on twitter at @akceatx.

   Notice to Investors and Security Holders               

   The tender offer referred to in this communication has not yet commenced. The description contained in this communication is neither an offer to purchase nor a solicitation of an offer to sell any securities, nor is it a substitute for the tender offer materials that Ionis will file with the Securities and Exchange Commission (the "SEC"). The solicitation and offer to buy shares of Akcea common stock will only be made pursuant to an offer to purchase and related tender offer materials. At the time the tender offer is commenced, Ionis will file a tender offer statement on Schedule TO and thereafter Akcea will file a solicitation/recommendation statement on Schedule 14D-9 with the SEC with respect to the Offer. THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT ON SCHEDULE 14D-9 WILL CONTAIN IMPORTANT INFORMATION. AKCEA SHAREHOLDERS ARE URGED TO READ THESE DOCUMENTS CAREFULLY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION THAT HOLDERS SHOULD CONSIDER BEFORE MAKING ANY DECISION REGARDING TENDERING THEIR SHARES. The offer to purchase, the related letter of transmittal and the solicitation/recommendation statement will be made available free of charge at the SEC's website at www.sec.gov. Additional copies may be obtained for free by contacting Ionis or Akcea, as applicable. Copies of the documents filed with the SEC by Ionis will be available free of charge on Ionis' internet website at https://ir.ionispharma.com/financial-information/sec-filings or by contacting Ionis' investor relations contact at (760) 603-2681. Copies of the documents filed with the SEC by Akcea will be available free of charge on Akcea's internet website at https://ir.akceatx.com/ or by contacting Akcea's investor relations contact at (617) 841-9535.

   In addition to the offer to purchase, the related letter of transmittal and certain other tender offer documents to be filed by Ionis, as well as the solicitation/recommendation statement to be filed by Akcea, Ionis and Akcea will also file quarterly and current reports with the SEC. Ionis' and Akcea's filings with the SEC are available to the public from commercial document-retrieval services and at the website maintained by the SEC at http://www.sec.gov.

   FORWARD-LOOKING STATEMENTS

   Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. assume no obligation to update forward-looking statements contained in this communication as a result of new information or future events or developments except as required by law. This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc., the therapeutic and commercial potential of TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) and the proposed acquisition of Akcea that are subject to risks and uncertainties that could cause actual results to differ materially from those expressly or implied by such statements. Any statement describing Akcea's or Ionis' goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of TEGSEDI and WAYLIVRA or other of Akcea's or Ionis' drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Risks and uncertainties also include, among other things, risks related to the satisfaction or waiver of the conditions to closing the proposed acquisition in the anticipated timeframe or at all, including uncertainties as to how many of Akcea's stockholders will tender their Shares in the tender offer and the possibility that the acquisition does not close; disruption from the transaction making it more difficult to maintain business and operational relationships; risks that anticipated synergies will not be realized or may be delayed; and the magnitude of transaction costs. Akcea's and 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 Akcea's and Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea and Ionis. In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's and Ionis' programs are described in additional detail in Akcea's and Ionis' quarterly reports on Form 10-Q and annual reports on Form 10-K, which are on file with the SEC. Copies of these and other documents are available from each company.

   In this press release, unless the context requires otherwise, "Ionis," "Akcea," "Company,"  "we," "our," and "us" refers to Ionis Pharmaceuticals or Akcea Therapeutics, as applicable.

   Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics^®, TEGSEDI^® and WAYLIVRA^® are trademarks of Akcea Therapeutics, Inc.

    

   

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   SOURCE Ionis Pharmaceuticals, Inc.; Akcea Therapeutics, Inc.

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5. 29 August 2020 Positive Phase 2 Clinical Data of Vupanorsen (AKCEA-ANGPTL3-L(Rx)) Presented at ESC Congress 2020

   BOSTON and CARLSBAD, Calif., Aug. 29, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), presented data from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-L_Rx) in an online Late Breaking Clinical Trial Session at the ESC Congress 2020, the annual meeting for the European Society of Cardiology.

   

   Vupanorsen is an investigational antisense therapy being developed to treat patients with certain cardiovascular diseases. In the Phase 2 study, vupanorsen met the primary endpoint of significant reductions in triglyceride (TG) levels and multiple secondary endpoints compared to placebo, with a favorable safety and tolerability…

   BOSTON and CARLSBAD, Calif., Aug. 29, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), presented data from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-L_Rx) in an online Late Breaking Clinical Trial Session at the ESC Congress 2020, the annual meeting for the European Society of Cardiology.

   

   Vupanorsen is an investigational antisense therapy being developed to treat patients with certain cardiovascular diseases. In the Phase 2 study, vupanorsen met the primary endpoint of significant reductions in triglyceride (TG) levels and multiple secondary endpoints compared to placebo, with a favorable safety and tolerability profile.

   "We are very encouraged by the demonstrated efficacy, safety and tolerability profile of vupanorsen," said Damien McDevitt, Ph.D., chief executive officer at Akcea. "There are millions of patients worldwide living with dyslipidemia that puts them at risk of cardiovascular events. By reducing ANGPTL3, vupanorsen has the potential to reduce the risk of cardiovascular events caused by dyslipidemia in many of these patients, thereby addressing a major area of continued unmet medical need. We look forward to working with Pfizer as they continue to advance this important clinical development program."

   Vupanorsen was developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform to reduce the production of angiopoietin-like 3 (ANGPTL3) protein from the liver, a key regulator of triglyceride and cholesterol metabolism.

   The goal of the randomized, double-blind, placebo-controlled, dose-ranging Phase 2 study was to assess the safety and efficacy of vupanorsen. A total of 105 patients with hypertriglyceridemia (fasting plasma TG levels >150 mg/dL), type 2 diabetes and non-alcoholic fatty liver disease (NAFLD) were randomized to three dosing cohorts in a 3:1 ratio (vupanorsen: placebo) within each cohort and treated for six months. The dosing cohorts explored different doses and dose regimens vs placebo, with patients receiving either 40 mg or 80 mg every four weeks or 20 mg every week. Participants received either vupanorsen or placebo via subcutaneous injection. Results from the Phase 2 study show:

   • Statistically significant dose-dependent reductions in fasting TGs at all dose levels, with the highest mean reduction of 53% at the dose of 80 mg every four weeks (44% mean reduction compared to placebo, P<0.0001)
   • Statistically significant dose-dependent reductions compared to placebo in ANGPTL3 (62%), very low-density lipoprotein (VLDL) cholesterol (38%), total cholesterol (19%), and non-high-density lipoprotein (non-HDL) cholesterol (18%) (numbers indicate mean reductions achieved with the 80 mg every four week dose)
   • No effect on glycemic parameters and no decrease in hepatic steatosis
   • No significant reductions in low-density lipoprotein cholesterol (LDL-C) levels compared to placebo in this patient population, which did not have high baseline LDL-C levels
   • A favorable tolerability and safety profile. The most common treatment-emergent adverse events were injection site reactions, which were mostly mild.

   "Antisense-mediated reduction of ANGPTL3 has the potential to address significant unmet needs in patients with cardiovascular diseases," said Daniel Gaudet, M.D., professor of medicine, Department of Medicine, University of Montreal. "Results from the Phase 2 study bring essential insights about this investigational therapy that may help guide disease management strategies and clinical research moving forward."

   "Clinical data from the Phase 2 study show an impressive and meaningful reduction in triglyceride levels and ANGPTL3, and add to the growing body of evidence supporting our LICA antisense technology  for large indications, such as cardiovascular disease," said Brett P. Monia, Ph.D., chief executive officer at Ionis. "We believe vupanorsen has the potential to bring much-needed benefit to this patient population."

   In November 2019, Akcea and Ionis announced the closing of a worldwide exclusive licensing agreement with Pfizer Inc. for vupanorsen. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with this Phase 2 study. Pfizer plans to further evaluate vupanorsen in a Phase 2b study among statin-treated patients with elevated non-HDL-C and high triglyceride levels, in order to identify the optimal dose to maximize target engagement and lipid lowering. The Phase 2b study is expected to initiate in 2H 2020.

   ABOUT VUPANORSEN (AKCEA-ANGPTL3-L_Rx)
   
   AKCEA-ANGPTL3-L_Rx (vupanorsen) is an investigational antisense therapy being developed to treat patients with certain cardiovascular diseases. This antisense medicine is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. AKCEA-ANGPTL3-L_Rx was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform. The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of low-density lipoprotein cholesterol (LDL-C) and triglycerides, and a decreased risk of diabetes and cardiovascular disease.¹ In a previous Phase 1 study, subjects treated with AKCEA-ANGPTL3-L_Rx achieved robust, dose-dependent reductions in ANGPTL3, triglycerides, LDL-C, non-HDL-C and total cholesterol with a positive safety and tolerability profile.² AKCEA-ANGPTL3-L_Rx was discovered by Ionis and has been co-developed by Akcea and Ionis.

   ABOUT AKCEA THERAPEUTICS
   
   Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing medicines to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI^® (inotersen) and WAYLIVRA^® (volanesorsen), as well as advancing a mature pipeline of novel medicines, including AKCEA-APO(a)-L_Rx, vupanorsen (AKCEA-ANGPTL3-L_Rx), AKCEA-APOCIII-L_Rx, and AKCEA-TTR-L_Rx, with the potential to treat multiple diseases. All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. TEGSEDI is approved in the U.S., E.U., Canada and Brazil, and WAYLIVRA is approved in the E.U. Akcea is headquartered in Boston and is building the infrastructure to commercialize its medicines globally. Additional information about Akcea is available at www.akceatx.com and you can follow us on Twitter at @akceatx. 

   ABOUT IONIS PHARMACEUTICALS, INC.
   
   As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases. To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.

   AKCEA AND IONIS FORWARD-LOOKING STATEMENT
   
   This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. and the therapeutic and commercial potential of AKCEA-ANGPTL3-L_Rx. Any statement describing Akcea's or Ionis' goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of AKCEA-ANGPTL3-L_Rx or other of Akcea's or Ionis' drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea's and 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 Akcea's and Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea and Ionis.  In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's and Ionis' programs are described in additional detail in Akcea's and Ionis' quarterly reports on Form 10-Q and annual reports on Form 10-K, which are on file with the SEC. Copies of these and other documents are available from each company.

   In this press release, unless the context requires otherwise, "Ionis," "Akcea," "Company," "Companies," "we," "our," and "us" refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.

   Ionis Pharmaceuticals^® is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics^®, TEGSEDI^® and WAYLIVRA^® are trademarks of Akcea Therapeutics, Inc.

   References

   1. JAMA Cardiol. 2018 Oct 1;3(10):957-966.
   2. N Engl J Med. 2017 Jul 20;377(3):222-232.

   

    

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   SOURCE Akcea Therapeutics, Inc.; Ionis Pharmaceuticals, Inc.

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