IONS Ionis Pharmaceuticals Inc.

60.79
+0.59  (+1%)
Previous Close 60.2
Open 61
52 Week Low 39.32
52 Week High 73.09
Market Cap $8,469,326,447
Shares 139,321,047
Float 117,047,398
Enterprise Value $6,801,825,029
Volume 615,336
Av. Daily Volume 856,311
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Upcoming Catalysts

Drug Stage Catalyst Date
Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
NDA Filing
NDA Filing
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IONIS-ENaC-2.5Rx
Healthy volunteers
Phase 1
Phase 1
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IONIS-PKK-LRx
Hereditary Angioedema
Phase 2
Phase 2
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IONIS-GHR-LRx
Acromegaly
Phase 2
Phase 2
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IONIS-FXIRx
End-stage renal disease
Phase 2b
Phase 2b
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Durvalumab Plus Danvatirsen
Refractory head and neck cancer
Phase 1/2
Phase 1/2
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IONIS-SOD1Rx / BIIB067
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3
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Tominersen (IONIS-HTT Rx)
Huntington's Disease
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
AKCEA-APOCIII-LRx
Hyperlipoproteinemia and established cardiovascular disease
Phase 2
Phase 2
Phase 2 data met primary and secondary endpoints - January 22, 2020. Phase 3 trial to be initiated 2020.
SPINRAZA (nusinersen) - high dose
Spinal muscular atrophy (SMA)
Phase 2/3
Phase 2/3
Phase 2/3 commencement of dosing announced April 2, 2020.
SPINRAZA - DEVOTE
Spinal muscular atrophy (SMA)
Phase 2/3
Phase 2/3
Phase 2/3 initiation announced September 18, 2019.
AKCEA-APO(a)-LRx
Hyperlipoproteinemia(a) and established cardiovascular disease
Phase 3
Phase 3
Phase 3 trial has been initiated - noted February 25, 2020.
AKCEA-TTR-LRx
hATTR Amyloidosis
Phase 3
Phase 3
Phase 3 trial initiation announced November 25, 2019.
AKCEA-TTR-LRx - CARDIO-TTRansform
ATTR cardiomyopathy
Phase 3
Phase 3
Phase 3 trial initiation announced January 10, 2020.
IONIS-MAPTRx
Alzheimer's disease
Phase 1/2
Phase 1/2
Phase 1/2 trial initiation announced October 13, 2017.
Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Approved
Approved
FDA Approval announced October 5, 2018,
IONIS-FB-LRx
Geographic Atrophy (GA)
Phase 2
Phase 2
Phase 2 trial to be initiated early 2019.
Inotersen (IONIS-TTRRx)
CARDIO-TTR - Cardiomyopathy form of TTR amyloidosis.
Phase 2
Phase 2
Partner GSK announced May 2016 decision not to be proceed with Phase 3 outcome trial
IONIS-GCGRRx
Type 2 diabetes
Phase 2
Phase 2
Phase 2 data released January 4, 2016.
IONIS-APO(a)Rx
High lipoprotein(a), or Lp(a)
Phase 2
Phase 2
Phase 2 data released 3Q 2015
SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Approved
Approved
Approved December 23, 2016.
IONIS-PTP1BRx
Type 2 diabetes
Phase 2
Phase 2
Phase 2 released Feb 2015. Achieved statistically significant results but data released was as of 36 weeks rather than 27 weeks for the primary endpoint
IONIS-ANGPTL3-LRx
Cardiovascular disease
Phase 1/2
Phase 1/2
Phase 1/2 interim data released November 2016.

Latest News

  1. BOSTON, June 29, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that Tracy Palmer Berns has joined the company as chief compliance officer. In this role, Ms. Berns will be responsible for the company's ongoing compliance program focusing on policy development and training. She will also collaborate with other members of the executive team to provide strategic counsel related to development of business goals and compliance matters related to Akcea's commercial products and investigational therapies.

    "With Tracy's extensive compliance and legal expertise, she will help inform and continually evolve our focus and commitment to ethical business practices. During this exciting time at Akcea, when we are building new levels of momentum with our commercialization strategies and development programs, Tracy is an important addition to the leadership team," said Alex Howarth, chief operating officer at Akcea.

    Before Akcea, Ms. Berns served as vice president, chief compliance officer and legal affairs at AMAG Pharmaceuticals, Inc. where she was responsible for the company's compliance program and therapeutic product legal team and provided legal advice on regulatory, development, pharmacovigilance and quality matters. From 2005-2017 she held various roles at Medtronic (formerly Covidien), ultimately serving as vice president, ethics and compliance in the Minimally Invasive Therapies Group, a $30 billion division of the company.

    She has also served as regulatory manager of the EP division at C.R. Bard, regulatory affairs manager and staff attorney at ABIOMED, senior consultant at EXPERTech and associate general counsel at Thermo Electron Corporation. Ms. Berns received her law degree from Columbia University School of Law and her bachelor's degree from Knox College in Illinois.

    "I am pleased to join Akcea as the company continues to make progress in building its commercialization and product portfolio while expanding its commitment to serving rare disease populations who are in desperate need of treatment options," said Ms. Berns. "I look forward to joining with the leadership team in their important goal to bring promising therapies to address significant areas of unmet need in global health."

    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)-LRx, vupanorsen (AKCEA-ANGPTL3-LRx), AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, 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. 

    FORWARD-LOOKING STATEMENT

    This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of Akcea's medicines 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 medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Akcea's 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 forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. 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 programs are described in additional detail in Akcea's 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 the 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.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/akcea-appoints-tracy-palmer-berns-as-chief-compliance-officer-301084801.html

    SOURCE Akcea Therapeutics, Inc.

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  2. BOSTON, June 17, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat patients with serious and rare diseases, today announced that Damien McDevitt, chief executive officer, and Kyle Jenne, chief commercial officer, will participate in a fireside chat at the BMO 2020 Prescriptions for Success Healthcare Conference, which is being held virtually, on Tuesday, June 23, 2020 at 3:30 p.m. ET.

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

    A live webcast of the presentation will be available on the "Media & Investors" section of the Akcea website. A replay will be available within 48 hours and will be archived for a limited time.

    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)-LRx, vupanorsen (AKCEA-ANGPTL3-LRx), AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, 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. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. 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. 

    FORWARD-LOOKING STATEMENT

    This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of Akcea's medicines 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 medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Akcea's 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 forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. 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 programs are described in additional detail in Akcea's 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 the 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.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/akcea-therapeutics-to-present-at-the-bmo-2020-prescriptions-for-success-healthcare-conference-301078216.html

    SOURCE Akcea Therapeutics, Inc.

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  3. Antisense Therapeutics ((ASX:ANP, OTC:ATHJY), an Australian biopharmaceutical company developing and commercializing antisense pharmaceuticals for unmet need in rare diseases announces a virtual poster presentation on the Muscular Dystrophy Association (MDA) Virtual 2020 Conference website.

    For 70 years MDA has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. As the largest source of funding for neuromuscular disease research outside of the US federal government, MDA has committed more than $1 billion since its inception to accelerate the discovery of therapies and cures.

    The poster presentation includes the recently reported data from the Phase II clinical trial…

    Antisense Therapeutics ((ASX:ANP, OTC:ATHJY), an Australian biopharmaceutical company developing and commercializing antisense pharmaceuticals for unmet need in rare diseases announces a virtual poster presentation on the Muscular Dystrophy Association (MDA) Virtual 2020 Conference website.

    For 70 years MDA has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. As the largest source of funding for neuromuscular disease research outside of the US federal government, MDA has committed more than $1 billion since its inception to accelerate the discovery of therapies and cures.

    The poster presentation includes the recently reported data from the Phase II clinical trial of the Company's immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) where ATL1102 met the primary endpoint of the study with confirmation of the drug's safety and tolerability. ATL1102 also demonstrated strong effects on secondary endpoints including activity on the targeted CD49d immune cells consistent with the drug's proposed mechanism of action and outcomes on disease progression parameters that exceeded the Company's expectations with improvement or stabilisation across different measures of muscle function and strength. The positive effects on disease progression were further supported by MRI data that suggested a stabilisation in the percentage of fat in muscles and preservation of functional muscle mass.

    The Poster can be viewed on our website at the following link: Poster Presentation – Muscular Dystrophy Virtual Conference 2020

    Mark Diamond, Chief Executive Officer of Antisense Therapeutics said: "Given the challenging times we are living in, MDA had to reimagine their annual conference and so have recently announced the launch of their 2020 MDA Virtual Conference Hub. Accordingly, we are very pleased that MDA have accepted our Phase II trial data for presentation. The Company is giving the clinical development of ATL1102 our highest priority. As we progress toward late stage clinical development, we intend to further increase international awareness of ATL1102's therapeutic potential in boys suffering this terrible disease. The support of MDA and similarly oriented advocacy groups will greatly enhance attainment of that objective."

    About Antisense Therapeutics is an Australian publicly-listed biotechnology company, developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases. The products are in-licensed from Ionis Pharmaceuticals Inc. (NASDAQ:IONS), an established leader in antisense drug development. The Company is developing ATL1102, an antisense inhibitor of the CD49d receptor, for Duchenne Muscular Dystrophy (DMD) patients and recently reported highly promising Phase II trial results. ATL1102 has also successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients with relapsing-remitting multiple sclerosis (RRMS). The Company has a second drug, ATL1103 designed to block GHr production that successfully reduced blood IGF-I levels in Phase II clinical trials in patients with the growth disorder acromegaly.

    About MDA For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation's top medical institutions. Our Resource Centre serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events, programming, and advocacy to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted at mda.org/COVID19. For more information, visit mda.org.

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    • After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation
    • Patients continued to maintain and make progressive gains in motor function compared to the natural history of the disease, with 96 percent now able to walk with assistance
    • The NURTURE study was recently extended to evaluate the longer-term efficacy and safety of SPINRAZA up to 8 years of age

    CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) --  Biogen Inc. (NASDAQ:BIIB) today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA) that is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants…

    • After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation
    • Patients continued to maintain and make progressive gains in motor function compared to the natural history of the disease, with 96 percent now able to walk with assistance
    • The NURTURE study was recently extended to evaluate the longer-term efficacy and safety of SPINRAZA up to 8 years of age

    CAMBRIDGE, Mass., June 10, 2020 (GLOBE NEWSWIRE) --  Biogen Inc. (NASDAQ:BIIB) today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA) that is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease. These results are being presented at the virtual Cure SMA Research & Clinical Care Meeting taking place June 10-12, 2020.

    The new data include nearly a year of additional follow-up for NURTURE study participants. As of February 2020, all patients treated (n=25; median age of 3.8 years old) were alive and remained free of permanent ventilation. In the absence of treatment, the majority of children with SMA Type 1 would, on average, not reach their second birthday. Additionally, all children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.

    "The impact of early and sustained SPINRAZA treatment on these infants and their families is remarkable. I've had the privilege to watch them grow into active young children, many of whom have experienced progress in motor function consistent with children their age who do not have SMA," said Kathryn Swoboda, M.D., the Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital. "The new results from NURTURE continue to bolster the substantial benefit of both prompt diagnosis and early and longer-term treatment with SPINRAZA."

    NURTURE is an ongoing, Phase 2, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA before 6 weeks old. The study has been extended by an additional three years, enabling Biogen to evaluate the longer-term efficacy and safety of SPINRAZA through 8 years of age and further understand the impact of early treatment. More information on the NURTURE study (NCT02386553) is available on clinicaltrials.gov.

    Additional results from the updated interim analysis as of February 2020 show:

    • All study participants who were previously able to walk with assistance (92 percent) and walk independently (88 percent) maintained that ability over the 11 months since the last data cut.1
    • Over the 11 months of follow-up, one child gained the ability to walk with assistance (increasing to 96 percent of all study participants) and also reached the maximum score on the Children's Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) scale, increasing the total number of study participants who achieved the maximum score to 21 of 25 (84 percent).
    • Patients with two copies of SMN2 were able to score and advance on the Hammersmith Functional Motor Scale Expanded scale (HFMSE), which is atypical to the natural history of the disease.
    • SPINRAZA was well-tolerated, with no new safety concerns identified over the extended follow-up period. No children have discontinued the study due to adverse events associated with treatment.

    About SPINRAZA® (nusinersen)2-4

    SPINRAZA is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is approved in more than 50 countries. As of March 31, 2020, more than 10,000 individuals have been treated with SPINRAZA. It is the only SMA treatment to combine unsurpassed real-world experience with a robust level of clinical evidence across a broad spectrum of patient populations.

    SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA, and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.

    SPINRAZA, a foundation of care in SMA, is an antisense oligonucleotide (ASO), developed using Ionis Pharmaceuticals' proprietary technology that is designed to target a root cause of SMA by increasing the amount of full-length survival motor neuron (SMN) protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.

    SPINRAZA currently maintains a robust clinical data set in SMA based on data from approximately 300 patients across a broad range of SMA populations demonstrating a favorable benefit:risk profile. SPINRAZA was evaluated in two randomized, double-blind, sham-controlled studies of infantile and later-onset SMA (ENDEAR and CHERISH, respectively) and supported by open-label studies that include pre-symptomatic infants (NURTURE), individuals with later-onset SMA (CS2/CS12) and an extension study of individuals who previously participated in the clinical development program (SHINE). The most common adverse events observed were respiratory infection, fever, constipation, headache, vomiting and back pain. Hypersensitivity, meningitis and hydrocephalus have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.

    Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.

    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. To learn more, please visit www.biogen.com and 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; clinical development programs, clinical trials and data readouts and presentations; and the potential benefits and results from early treatment of SMA. 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 the occurrence of adverse safety events; risks of unexpected costs, delays or 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; failure to obtain regulatory approvals in other jurisdictions; 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. De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842‐856. doi:10.1016/j.nmd.2019.09.007.
    2. Based on Commercial Patients, Early Access Patients, and Clinical Trial Participants as of March 31, 2020.
    3. Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.
    4. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.
    MEDIA CONTACT:INVESTOR CONTACT:
    David CaouetteJoe Mara
    + 1 617 679 4945+1 781 464 2442
       

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  4. BOSTON, June 9, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that Kia Motesharei, Ph.D., has joined the company as Senior Vice President of Business Development & Corporate Strategy. In this role Dr. Motesharei will drive business development activities at Akcea as well as continue to enhance the corporate strategy in conjunction with the Akcea leadership team.

    "As Akcea continues to build momentum including expanding our pipeline, advancing our global commercialization strategies and further strengthening our leadership team, we are pleased to have Kia join as the latest addition to our team," said Alex Howarth, chief operating officer at Akcea. "His depth of transactional expertise including in the rare disease sector will be invaluable and help us optimize and execute our corporate strategy moving forward."

    Dr. Motesharei has more than 20 years of business and corporate development and strategy experience with multinational companies. He has a successful track record of more than 100 transactions which include research and strategic alliances, product and technology licensing, distribution, divestitures, and merger and acquisition agreements with major pharmaceutical and biotechnology companies in the U.S., Europe, Japan, China, Latin America and the Middle East. Dr. Motesharei joins Akcea following a six-year tenure at Merck KGaA, where he most recently served as Vice President and Global Head of Licensing and Business Development, Neurology & Immunology. In his roles at Merck KGaA, he led and managed a transaction team executing more than 60 agreements with pharmaceutical, biotechnology and academic institutions. Prior to Merck KGaA, he was Vice President of Business Development & Alliance Management at Dyax Corp., a biopharmaceutical company specializing in rare disease, which was acquired by Shire and subsequently acquired by Takeda. Before that, Dr. Motesharei served as Chief Business Officer at the French biotechnology company Genfit and Vice President of Corporate Development at ActivX.

    He received his B.A. in chemistry from The Colorado College and his Ph.D. in organic chemistry from the University of California, Los Angeles. He completed his postdoctoral training at The Scripps Research Institute as a National Institutes of Health (NIH) Fellow.

    "Leveraging my experience within the rare and orphan disease area, I look forward to working with the executive leadership and the rest of the dedicated team at Akcea to bring life-changing therapies to patients suffering from genetic and rare diseases, who often lack treatment options," said Dr. Motesharei.

    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)-LRx, vupanorsen (AKCEA-ANGPTL3-LRx), AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, 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. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. 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. 

    FORWARD-LOOKING STATEMENT

    This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of Akcea's medicines 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 medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Akcea's 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 forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. 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 programs are described in additional detail in Akcea's 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 the 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.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/akcea-appoints-new-senior-vice-president-business-development-and-corporate-strategy-301072319.html

    SOURCE Akcea Therapeutics, Inc.

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