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1. 08 January 2021 Biogen Announces First Patient Treated in RESPOND Study Evaluating Benefit of SPINRAZA® (nusinersen) in Patients Treated With Zolgensma® (onasemnogene abeparvovec)

   • The global Phase 4 RESPOND study will evaluate the efficacy and safety of SPINRAZA in patients with a suboptimal clinical response to Zolgensma
     
   • Clinical and real-world experience have reported that some patients previously treated with Zolgensma have also been treated with SPINRAZA^1,2,3,4
   • Biogen remains committed to exploring the potential of SPINRAZA to optimize outcomes for patients with SMA

   CAMBRIDGE, Mass., Jan. 08, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced that the first patient has been treated in the global clinical study, RESPOND. The Phase 4 study will examine the clinical benefit and assess the safety of SPINRAZA^® (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical…

   • The global Phase 4 RESPOND study will evaluate the efficacy and safety of SPINRAZA in patients with a suboptimal clinical response to Zolgensma
     
     
   • Clinical and real-world experience have reported that some patients previously treated with Zolgensma have also been treated with SPINRAZA^1,2,3,4
   • Biogen remains committed to exploring the potential of SPINRAZA to optimize outcomes for patients with SMA

   CAMBRIDGE, Mass., Jan. 08, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ:BIIB) today announced that the first patient has been treated in the global clinical study, RESPOND. The Phase 4 study will examine the clinical benefit and assess the safety of SPINRAZA^® (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma^® (onasemnogene abeparvovec). RESPOND will be conducted at approximately 20 sites worldwide and aims to enroll up to 60 children with SMA.

   "SMA treatments have changed what is possible for children born with the disease but they have also raised new questions," said Dr. Nicole Gusset, President of SMA Europe and mother of a child with SMA. "We appreciate that the RESPOND study will collect data to help provide answers so individuals living with SMA can make informed treatment decisions."

   Children with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons that support sitting, walking and basic functions of life like breathing and swallowing. The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its mechanism of action, which leads to continuous production of SMN protein, may also benefit patients who have been insufficiently treated with gene therapy.

   "In clinical practice, there is a sense of urgency to address motor neuron loss in SMA from the earliest sign or even prior to symptoms, to prevent additional disease progression," said Julie Parsons, M.D., professor of Clinical Pediatrics and Neurology and Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders at Children's Hospital Colorado and the University of Colorado School of Medicine, and primary investigator of the RESPOND study. "In some patients treated with gene therapy, we have recognized that further motor neuron protection may be needed. Our hope is that results from RESPOND will demonstrate if SPINRAZA can optimize treatment for some of our youngest patients."

   RESPOND is a two-year, open-label study to evaluate the efficacy and safety of SPINRAZA in SMA patients previously treated with Zolgensma to further optimize treatment decisions. The primary endpoint is the total score on the Hammersmith Infant Neurological Examination Section 2. Secondary endpoints include safety, change from baseline on additional motor function measures, other clinical outcomes (e.g., swallowing) and caregiver burden. Neurofilament levels, an exploratory endpoint, will also be evaluated as a marker of biological disease activity.

   The study will enroll 60 children up to 3 years old who are determined by the investigator to have the potential for additional clinical improvement after receiving Zolgensma. It has been reported that, to date, 40 percent of children in the long-term study of Zolgensma have been subsequently treated with SPINRAZA.¹ Physicians will use criteria that may include one or more of the following: suboptimal motor function (e.g., a score lower than 50 on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders [CHOP INTEND]); the need for respiratory support; abnormal swallowing or feeding ability; or other factors deemed relevant by the investigator.

   The primary study group will include 40 infants aged 9 months or younger (at the time of first SPINRAZA dose) who have 2 copies of SMN2 (likely to develop SMA Type 1) and received Zolgensma at 6 months old or younger. A second study group will include 20 children within a broader age range (up to 3 years old at the time of first SPINRAZA dose). After a screening period, participants will receive the approved 12 mg dose of SPINRAZA: four loading doses, followed by maintenance doses every four months,⁵ over the two-year study period.

   More information on the study (NCT04488133) is available at clinicaltrials.gov.

   About SPINRAZA^® (nusinersen)
   
   SPINRAZA is approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is available in more than 50 countries. As a foundation of care in SMA, more than 11,000 individuals have been treated with SPINRAZA worldwide.⁶

   SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.⁵ It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.⁵

   SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 7 years, combined with unsurpassed real-world experience.⁷ The SPINRAZA clinical development program encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations,⁷ including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.

   Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics. Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country's product website.

   About Spinal Muscular Atrophy (SMA)
   
   SMA is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness.⁸ SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missing SMN1 gene, with a spectrum of disease severity.⁸ Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time.⁹ In the absence of treatment, children with the most severe form of SMA would not be expected to reach their second birthday.⁸

   SMA impacts approximately one in 11,000 live births,¹⁰ is a leading cause of genetic death among infants¹⁰ and causes a range of disability in teenagers and adults.⁹

   About Biogen
   
   At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimer's disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology and pain.

   We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

   Biogen Safe Harbor 
   
   This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits, safety and efficacy of SPINRAZA; the results of certain real-world data; the identification and treatment of SMA; our research and development program for the treatment of SMA; the potential benefits and results from early treatment of SMA; the enrollment of the RESPOND study; risks and uncertainties associated with drug development and commercialization; and the potential of our commercial business, including SPINRAZA. These statements may be identified by words such as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "plan," "possible," "potential," "will," "would" and other words and terms of similar meaning. You should not place undue reliance on these statements or the scientific data presented.

   These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation risks that we may not fully enroll the RESPOND study or it will take longer than expected; unexpected concerns that may arise from additional data, analysis or results obtained during the RESPOND study; the occurrence of adverse safety events; risks of unexpected costs or delays; the risks of other unexpected hurdles; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

   References:

   1. Zolgensma EU Summary of Product Characteristics (SmPC). Available at: https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf. Accessed: December 2020.
   2. Finkel R, et al. Presented at the Muscular Dystrophy Association (MDA) 2020 Clinical & Scientific Conference.
   3. Harada Y, et al. Presented at the Muscular Dystrophy Association (MDA) 2020 Clinical & Scientific Conference.
   4. Finkel R, et al. Presented at the World Muscle Society's (WMS) 2020 Virtual Congress.
   5. SPINRAZA U.S. Prescribing Information. Available at: https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: December 2020.
   6. Based on commercial patients, early access patients, and clinical trial participants through September 30, 2020. 
   7. Core Data Sheet, Version 9, January 2019. SPINRAZA. Biogen Inc, Cambridge, MA.
   8. National Institute of Neurological Disorders and Stroke, NIH. Spinal Muscular Atrophy Fact Sheet. Available at https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Spinal-Muscular-Atrophy-Fact-Sheet. Accessed: December 2020.
   9. Wadman RI, Wijngaarde CA, Stam M, et al. Muscle strength and motor function throughout life in a cross-sectional cohort of 180 patients with spinal muscular atrophy types 1c–4. Eur J Neurol. 2018;25(3):512-518.
   10. Cure SMA. About SMA. Available at https://www.curesma.org/about-sma/. Accessed: December 2020.

   MEDIA CONTACT: David Caouette + 1 617 679 4945

   INVESTOR CONTACT: Joe Mara +1 781 464 2442

   
   
   

   

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2. 07 January 2021 Ionis appoints Eugene Schneider, M.D., as executive vice president and chief clinical development officer

   CARLSBAD, Calif., Jan. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced the appointment of Eugene Schneider, M.D., as executive vice president and chief clinical development officer. In this role, Dr. Schneider will oversee clinical development of novel antisense medicines across all of Ionis' therapeutic franchises. He will report to Richard S. Geary, Ph.D., Ionis' executive vice president and chief development officer, and serve on the company's executive team.

   

   Since joining Ionis in 2013, Dr. Schneider has played a key role in more than 20 clinical trials conducted in the U.S. and globally. He is an accomplished biopharmaceutical industry veteran with two decades of experience leading global medical and scientific affairs and the development and commercialization of medicines to treat a range of disorders, including pediatric, rare and central nervous system diseases.

   "Dr. Schneider's proven track record overseeing successful studies, assessing risk-benefit profiles of investigational medicines and providing consistent clinical due diligence in safety reporting will strengthen Ionis' industry leadership as we expand our portfolio of wholly owned and partnered medicines and build our commercial organization to maximize the value of our rich pipeline," said Dr. Geary.

   "I am excited to join Ionis' talented executive team and look forward to continuing my work with them and all Ionis employees who are deeply committed to delivering potentially transformative medicines to patients who depend on us," said Dr. Schneider.   

   Before joining Ionis, Dr. Schneider was senior medical director, clinical research and development at Synageva BioPharma, where he developed a novel enzyme replacement therapy for Lysosomal Acid Lipase Deficiency from its first-in-man study to registration. Prior to Synageva, he was senior medical director, medical and scientific affairs at Biovail Technologies Ltd., where he was primarily responsible for the development and commercialization of a diverse portfolio of medicines designed to address patient needs related to central nervous system disorders.

   Dr. Schneider received his medical degree from the University of Medicine and Dentistry of New Jersey and completed his training in pediatric and adolescent medicine at Robert Wood Johnson University Hospital in New Brunswick, New Jersey.

   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.

    

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   SOURCE Ionis Pharmaceuticals, Inc.

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3. 17 December 2020 Stanley T. Crooke, Ph.D., M.D, to retire from Ionis to focus on his scientific interests and n-Lorem Foundation

   CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit organization he founded, the n-Lorem Foundation. Dr. Crooke will continue to serve as a scientific advisor to Ionis including providing advice regarding Ionis' research and development programs and guiding the core antisense research group he founded at Ionis.

   

   Dr. Crooke founded Ionis more than 30 years ago and is credited with being the pioneer of RNA-targeted therapeutics. Under his leadership, Ionis developed its novel antisense technology and created one of the largest, most advanced pipelines in the industry. He has personally led the development of more than 20 marketed drugs throughout his career and supported the creation and growth of several companies based on Ionis' antisense technology. In addition, Dr. Crooke is responsible for creating one of the most innovative and scientifically driven cultures that has an unwavering passion for discovering and developing future-creating therapies and a steadfast commitment to saying yes to the patients who are depending on them.

   "The brilliance of Stan's vision isn't just represented by the founding and success of Ionis, but it is also reflected in his creation of an entirely new chemical class of medicines, antisense oligonucleotides," said Joseph Loscalzo, M.D., Ph.D., head, Department of Medicine and Physician-in-Chief, Brigham & Woman's Hospital, Harvard Medical School and Ionis board member. "His scientific contributions will no doubt continue to provide extraordinary benefit to the healthcare industry and the many patients who once were told there isn't any hope or treatment for their disease. With Stan solely focusing on his scientific interests and n-Lorem, we can only expect his impact to continue."

   Dr. Crooke has received numerous honors and awards for his pioneering work in RNA-targeted therapeutics including the prestigious Massry Prize, the Oligonucleotide Therapeutics Society Lifetime Achievement Award, the American Chemical Society's E.B. Hershberg Award for Important Discoveries in Medicinally Active Substances, the Prix Galien Best Biotechnology Award for Spinraza, the Scrip Lifetime Achievement Award, and BIO's Helix Award for the most important innovation. In addition, Ionis' board of directors named the main headquarters building in honor of Dr. Crooke in 2019 to recognize his extraordinary role in creating Ionis. Dr. Crooke has published nearly 600 scientific publications, edited more than 20 books, and is a named inventor on numerous patents.

   "Thanks to Stan's vision and perseverance, thousands of patients are benefiting from the transformative and life-changing antisense medicines discovered and developed by Ionis," said Brett P. Monia, Ph.D., Ionis chief executive officer. "We are pleased that Stan will continue to provide advice and counsel to Ionis and applaud his efforts to use the technology that was created at Ionis to bring hope and therapies to the patients who will benefit greatly from the n-Lorem foundation."

   n-Lorem Foundation, founded in 2019 by Dr. Crooke and initially funded by him, his wife Dr. Rosanne Crooke, Ionis and Biogen, provides free individualized antisense treatments to patients living with ultra-rare diseases (1 to 30 patients worldwide) for life. In less than a year of its founding, the Foundation has garnered support from global partners such as the Korean Institute of Toxicology, many donors, has received more than 40 requests for treatment and has 10 patients being treated or about to be treated with an investigational antisense medicine that was designed for each one's specific genetic mutation that's causing their disease.  

   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.

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   SOURCE Ionis Pharmaceuticals, Inc.

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4. 08 December 2020 Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

   CARLSBAD, Calif., Dec. 8, 2020 /PRNewswire/ -- At its Investor Day yesterday, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) highlighted the company's significant achievements in 2020 and outlined its strategy to realize the substantial opportunity of its pipeline.

   

   Ionis has been preparing and prioritizing its growing wholly owned pipeline for commercialization in line with its commercial strategy. The company's commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care.  

   Delivering on these three priorities will have meaningful impact to patients, their families, and healthcare providers all while reducing the burden on healthcare systems and driving value for all Ionis' stakeholders including patients and shareholders.

   Ionis also has plans to expand opportunistically to new products in additional treatment areas such as hematology, endocrinology and pulmonology.

   Ionis projects having the opportunity to launch six or more new products through 2026, with each being ready for launch in a close window, ranging from 18 to 24 months between each new product launch. The expectation is that the implementation of this strategy will drive double-digit revenue growth and substantial earnings growth.

   Brett P. Monia, Ph.D., chief executive officer at Ionis, said, "In 2020, we pursued an aggressive agenda focused on building our commercial plans and capabilities, progressing the Ionis-owned pipeline, advancing our technology and growing our leadership position in RNA-targeted therapeutics. We are pleased to say that we delivered against all these objectives. We invested in building our commercial plans and capabilities and began implementation. These actions were accelerated through the acquisition of our commercial affiliate Akcea. We have also progressed and substantially expanded the Ionis-owned pipeline. In addition, we have six Phase 3 trials underway, initiated 13 Phase 2 trials, achieved multiple, positive clinical proof-of-concept readouts, and advanced new delivery platforms."

   Dr. Monia continued, "For years we have been recognized for our excellence in research, early drug development and scientific innovation. We will now add to this by building a strong and efficient commercial organization of equal excellence. All of which will provide substantial benefit to patients and shareholders for years to come."

   Highlights of Investor Day

   • Ionis estimates the total market opportunity for the indications targeted by its pipeline is well in excess of $15 billion, with a significant portion from its wholly owned medicines.

   • Ionis' cardiovascular franchise includes many potential first-in-class and/or best-in-class medicines targeting a full spectrum of cardiovascular disease risk factors. The company is positioned to potentially launch multiple Ionis-owned cardiovascular medicines through 2026, including:
     
      
   • AKCEA-APOCIII-L_Rx: One product with the potential for addressing multiple indications targeting elevated triglycerides and the opportunity to set a new standard of care for triglyceride management
   • 91% of patients achieved normal serum triglycerides levels with favorable safety and tolerability in Phase 2
   • As announced on Dec. 1, 2020, a Phase 3 study in patients with familial chylomicronemia syndrome (FCS) is now underway
   • Evaluating additional indications with plans to initiate an additional Phase 3 study in 2021
   • AKCEA-TTR-L_Rx: Opportunity to significantly expand ATTR franchise
   • Robust target reductions of more than 90% and favorable safety and tolerability demonstrated in Phase 1
   • Flexibility of at-home monthly self-administration
   • Two Phase 3 studies underway – CARDIO-TTRransform for patients with hereditary or wild type TTR cardiomyopathy and NEURO-TTRansform for patients with hereditary TTR polyneuropathy. 
   • IONIS-AGT-L_Rx: Large unmet need in patients with treatment-resistant hypertension (RHTN)
   • Two Phase 2 clinical studies: Patients with mild HTN and patients with uncontrolled HTN who are on two (65%) or three (35%) antihypertensive medications 
   • Positive Phase 2 study in patients in uncontrolled HTN: patients achieved mean reductions of 12 mmHg and 6 mmHg in systolic and diastolic blood pressure from their own baseline, respectively, after eight weeks of once-weekly 80 mg IONIS-AGT-L_Rx
   • IONIS-AGT-L_Rx has demonstrated a favorable safety and tolerability profile in clinical trials to date
   • More detailed results to be presented at an upcoming medical conference

   • Ionis' neurology franchise has the potential to establish the standard of care for millions of patients and generate substantial value as it advances its first-in-class medicines to the market. The neurological disease market is a nascent market poised for substantial growth. Ionis believes it can be the catalyst for this growth as it is positioned to launch multiple Ionis-owned medicines through 2026, including:
     
        
   • ION363: First medicine in development to specifically target FUS-ALS, a rare, rapidly progressing form of ALS
   • Pivotal study on track for initiation in 2021
   • Potential for a rapid path to the market
   • ION716: Potential to be first approved treatment for prion diseases
   • Designed to reduce production of prion protein, root cause of prion disease
   • Pursuing pre-symptomatic (genetic carriers) and symptomatic (genetic and sporadic) indications
   • Pivotal study planned for 2021, design should provide a rapid path to market

   Akcea Integration Update

   The recently completed acquisition of Akcea has created a stronger, more efficient company, further bolstering Ionis' financial strength. The integration of Akcea is ahead of schedule, delivering cost synergies and efficiencies. It was also announced that Akcea is going to commercialize TEGSEDI and WAYLIVRA in Europe through a distribution agreement with Swedish Orphan Biovitrum AB ("Sobi"), an international biopharmaceutical company that focuses on rare diseases. Under the terms of this agreement, Akcea retains the marketing authorization ("MAH") for both medicines in Europe. Additionally, Akcea will continue to maintain limited European operations including regulatory, manufacturing, and the management of relationships with key opinion leaders. Akcea will continue to lead the TEGSEDI and WAYLIVRA global commercial strategy. The agreement provides Ionis the flexibility to reinvest resources to support its other commercial plans.

   Oral Delivery Development Update

   Ionis and AstraZeneca are committed to bringing the best possible PCSK9 antisense treatments to patients and have been collaborating on both the subcutaneous and oral formulations. The subcutaneous formulation of ION449 has a potential best-in-class profile and is advancing rapidly toward Phase 3 development.

   Preclinical and early clinical data give Ionis and AstraZeneca confidence that they can achieve effective oral delivery of ION449 and other ASOs.  Based on ongoing research and experience to date, both companies believe they can improve upon the current oral formulation. Therefore, Ionis and AstraZeneca have decided to terminate the Phase 1 PCSK9 oral study. Ionis and AstraZeneca will continue to broadly work together to further optimize the oral delivery of ASOs, including ION449.

   Ionis is expanding its oral delivery to include medicines from its pipeline and has increased its internal investment in oral delivery research. The company plans on initiating one or more programs from its pipeline within the next year or two. Candidates for consideration include IONIS-TTR-L_Rx, IONIS-PKK-L_Rx, ION994 (AGT), and ION547. Success would further enhance the commercial value of Ionis-owned programs.

   Webcast

   Additional details are available in the replay of the webcast. It is available here for a limited time.

   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.

   Forward-looking Statements

   This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of SPINRAZA® (nusinersen), TEGSEDI® (inotersen), WAYLIVRA® (volanesorsen) and Ionis' technologies and products in development, including the business of Akcea Therapeutics, Inc., Ionis' wholly owned subsidiary. 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, 2019 and our most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of this and other documents are available at www.ionispharma.com. In this presentation, 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. Akcea Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA® is a registered trademark of Biogen.

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   SOURCE Ionis Pharmaceuticals, Inc.

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5. 02 December 2020 Ionis to participate in virtual fireside chat at BMO 2020 Growth and ESG conference

   CARLSBAD, Calif., Dec. 2, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), the leader in antisense therapeutics, today announced that management will participate in a virtual fireside chat at the BMO 2020 Growth and ESG Conference at 3:00 p.m. E.T. on Tuesday, December 8, 2020.

   

   Interested parties may access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time at the same address.

   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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