AKCA Akcea Therapeutics Inc.

11.02
-0.09  -1%
Previous Close 11.11
Open 11
52 Week Low 8
52 Week High 22.79
Market Cap $1,119,616,781
Shares 101,598,619
Float 100,140,466
Enterprise Value $781,881,256
Volume 73,443
Av. Daily Volume 302,405
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Upcoming Catalysts

Drug Stage Catalyst Date
Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
NDA Filing
NDA Filing
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AKCEA-TTR-LRx (NEURO-TTRansform)
hATTR Amyloidosis
Phase 3
Phase 3
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AKCEA-TTR-LRx - CARDIO-TTRansform
ATTR cardiomyopathy
Phase 3
Phase 3
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AKCEA-APO(a)-LRx
Hyperlipoproteinemia(a) and established cardiovascular disease
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
AKCEA-APOCIII-LRx
Hyperlipoproteinemia and established cardiovascular disease
Phase 2
Phase 2
Phase 3 trial to be initiated 2020.
AKCEA-ANGPTL3-LRx
Non-alcoholic fatty liver disease (NAFLD), Hypertriglyceridemia and Type 2 Diabetes Mellitus
Phase 2b
Phase 2b
Phase 2b data met primary endpoint - January 28, 2020.
Volanesorsen
Familial partial lipodystrophy (FPL)
Phase 3
Phase 3
Phase 3 data met primary endpoint - August 6, 2019.
Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Approved
Approved
FDA Approval announced October 5, 2018,

Latest News

  1. BOSTON, Aug. 13, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the German Association of Statutory Sick Funds has agreed upon new pricing and reimbursement of WAYLIVRA® (volanesorsen) as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. WAYLIVRA has been commercially available in Germany since August 15, 2019. As part of reimbursement and market access protocol in Germany, Akcea was required to renegotiate the WAYLIVRA launch price 12 months after first commercialization, which will…

    BOSTON, Aug. 13, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the German Association of Statutory Sick Funds has agreed upon new pricing and reimbursement of WAYLIVRA® (volanesorsen) as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. WAYLIVRA has been commercially available in Germany since August 15, 2019. As part of reimbursement and market access protocol in Germany, Akcea was required to renegotiate the WAYLIVRA launch price 12 months after first commercialization, which will go into effect on August 15, 2020.

    "We are pleased that renegotiations in Germany were swiftly completed, demonstrating that payers in the region continue to recognize the value that WAYLIVRA offers to FCS patients. While WAYLIVRA has been commercially available to FCS patients in the region for some time, achieving this new, 2-year mutually agreed upon pricing and reimbursement is a significant milestone for the global FCS community of patients and caregivers, who are currently underserved," said Michael Pollock, SVP, head of Europe at Akcea. "We continue to build on our momentum in Europe and are on track to launch WAYLIVRA in additional European countries with the goal of bringing this much needed therapy to all eligible patients living with FCS."

    "FCS patients in Germany as well as their families have been living with the significant burden of this disease for years, including physical, social, emotional and financial effects, and I am pleased to be able to offer appropriate patients a treatment option supported by extensive clinical data including results from the Phase 3 APPROACH study," said Professor Ioanna Gouni-Berthold from University of Cologne.

    FCS is a debilitating genetic disease caused by impaired function of the enzyme lipoprotein lipase (LPL), which results in elevated triglyceride levels and significant risk and disease burden, including unpredictable and potentially fatal acute pancreatitis as well as chronic complications due to permanent organ damage. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide. WAYLIVRA is designed to reduce the production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.

    About WAYLIVRA® (volanesorsen)

    With conditional marketing authorization from the European Commission (EC) as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate, WAYLIVRA is now the only therapy indicated for people with familial chylomicronemia syndrome (FCS). Akcea is working to confirm a path forward for WAYLIVRA in the U.S. and Canada.

    WAYLIVRA, a product of Ionis' proprietary antisense technology, is designed to reduce the production of apoC-III, a protein that regulates plasma triglycerides and may also affect other metabolic parameters.

    The European Commission's marketing authorization of WAYLIVRA is based on results from the Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension (OLE) study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with WAYLIVRA reduced triglycerides 77% (-94% when compared to placebo). All patients in the trial maintained a low-fat diet.

    WAYLIVRA is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. The most frequently observed adverse reactions (more than 10%) during treatment with WAYLIVRA were events associated with injection site reactions, immunogenicity and reduction in platelet levels. 

    The WAYLIVRA Early Access Program (EAP) has been initiated in Europe, the U.S. and Canada and is currently enrolling eligible patients. Click here for more information on the WAYLIVRA EAP. For more information on WAYLIVRA, please visit www.WAYLIVRA.eu.

    About FCS

    FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL or 10mmol/L) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL production or function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at www.lpldalliance.org and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here

    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 us 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 TEGSEDI®, WAYLIVRA® and Akcea's other 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-announces-new-pricing-and-reimbursement-of-waylivra-volanesorsen-in-germany-301111204.html

    SOURCE Akcea Therapeutics, Inc.

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  2. BOSTON, Aug. 4, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing medicines to treat patients with serious and rare diseases, today reported financial results for the second quarter ended June 30, 2020. The company reported a net loss for the three and six months ended June 30, 2020 on a GAAP basis of $50 million and $92 million, respectively. On a non-GAAP basis, the company reported a net loss for the three and six months ended June 30, 2020 of $33 million and $69 million, respectively. Akcea had $390 million of cash, cash equivalents and short-term investments as of June 30, 2020.

    BOSTON, Aug. 4, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing medicines to treat patients with serious and rare diseases, today reported financial results for the second quarter ended June 30, 2020. The company reported a net loss for the three and six months ended June 30, 2020 on a GAAP basis of $50 million and $92 million, respectively. On a non-GAAP basis, the company reported a net loss for the three and six months ended June 30, 2020 of $33 million and $69 million, respectively. Akcea had $390 million of cash, cash equivalents and short-term investments as of June 30, 2020.

    "We achieved important commercial milestones during the second quarter and continued to see consistent revenue growth with both TEGSEDI and WAYLIVRA," said Damien McDevitt, chief executive officer of Akcea. "We also continued to enhance and build upon the strength of our management team with the addition of several key leaders. We now have a full team in place and look forward to their many contributions as we move the company forward. Our focus remains on executing on our commercial launches, expanding our pipeline, and delivering our robust portfolio of medicines to patients living with serious and rare diseases."

    "In the second quarter, we achieved commercial product revenues of $16 million from TEGSEDI and WAYLIVRA, an increase of 66% from the prior year quarter, and an increase of 8% from the first quarter 2020," said Michael Price, chief financial officer of Akcea. "We remain well capitalized with $390 million in cash and short-term investments as of the end of June, putting us in a strong financial position to execute on our strategic priorities going forward."

    Upcoming Events

    • Launch of TEGSEDI in additional E.U. countries, and through PTC Therapeutics in Latin America
    • Launch of WAYLIVRA in additional E.U. countries
    • Refile marketing application for WAYLIVRA in the U.S. with the Food and Drug Administration, or FDA
    • Initiate a Phase 3 study of AKCEA-APOCIII-LRx for Familial Chylomicronemia Syndrome, or FCS, this year
    • Present the positive Phase 2 results of AKCEA-APOCIII-LRx and vupanorsen (AKCEA-ANGPTL3-LRx) at an upcoming medical congress in Q3

    Recent Organizational Developments

    • Strengthened and expanded the management team
      • Michael Price as EVP and Chief Financial Officer
      • Carla Poulson as SVP and Chief Human Resources Officer
      • Dr. William Andrews as Chief Medical Officer
      • Tracy Palmer Berns as Chief Compliance Officer
      • Dr. Kia Motesharei as SVP, Business Development and Corporate Strategy
      • Dr. Christophe Hotermans as SVP, Global Medical Affairs

    Recent Commercial Achievements

    • Achieved total product sales of $16 million, maintaining consistent quarter over quarter sales growth
    • Finalized pricing and reimbursement in Spain, Portugal, Italy and Austria for TEGSEDI
    • Secured listing for TEGSEDI in several Canadian provinces, clearing the path to reimbursement following the signing of the Letter of Intent with the pan-Canadian Pharmaceutical Alliance, or pCPA
    • Added first patient on commercial therapy in Greece for WAYLIVRA
    • Successfully concluded pricing and reimbursement negotiations in Germany for WAYLIVRA 
    • Marketing Authorization Application submitted to ANVISA in Brazil through PTC Therapeutics for WAYLIVRA

    Recent Pipeline Achievements

    • AKCEA-APO(a)-LRx granted Fast Track Designation for the treatment of Lp(a)-driven cardiovascular disease; trial readout is currently expected in 2024
    • Results from the NEURO-TTR Phase 3 Open-Label Extension (OLE) study in Patients with Polyneuropathy Driven by Hereditary Transthyretin Amyloidosis were published in the European Journal of Neurology. Find more info at www.doi.org

    Financial Results

    All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

    Revenue

    Akcea's total revenue for the three and six months ended June 30, 2020 was $22 million and $38 million, respectively, which was comprised of product revenue and research and development collaboration revenue. Revenue from sales of TEGSEDI and WAYLIVRA during the three and six months ended June 30, 2020 was $16 million and $32 million, respectively, compared to $10 million and $17 million for the same periods in 2019. Akcea did not recognize licensing revenue in 2020, whereas licensing revenue totaled $6 million from PTC Therapeutics for the three and six months ended June 30, 2019. Akcea's research and development collaboration revenue for the three and six months ended June 30, 2020 was $6 million and $7 million, respectively, compared to $11 million and $168 million for the same periods in 2019. The decrease in research and development collaboration revenue for the six months ended June 30, 2020 compared to the same period in 2019 was primarily related to the $150 million license fee the company earned in the first quarter of 2019 as a result of Novartis' exercise of its option to license AKCEA-APO(a)-LRx.

    Expenses

    Akcea's operating expenses, net of the reimbursement due from Ionis through the companies' profit/loss share arrangement, for the three and six months ended June 30, 2020 on a GAAP basis were $73 million and $135 million, respectively, and on a non-GAAP basis were $57 million and $111 million, respectively. These amounts compare to GAAP operating expenses of $65 million and $203 million and non-GAAP operating expenses of $51 million and $170 million for the same periods in 2019. The decrease in operating expenses for the six months ended June 30, 2020 compared to the same period in the prior year was primarily due to the one-time $75 million sublicense fee paid to Ionis as a result of Novartis' license of AKCEA-APO(a)-LRx in 2019 and lower development costs as a result of studies completed in 2019.

    Net Loss

    Akcea reported a net loss of $50 million and $92 million on a GAAP basis for the three and six months ended June 30, 2020, respectively, compared to a net loss of $37 million and $10 million for the same periods in 2019. Akcea reported a non-GAAP net loss of $33 million and $69 million for the three and six months ended June 30, 2020, respectively. This compared to a non-GAAP net loss of $23 million and non-GAAP net income of $23 million for the same periods in 2019. The change from non-GAAP net income to non-GAAP net loss was due to the decrease in research and development collaboration revenue described above.

    For the three and six months ended June 30, 2020, basic and diluted net loss per share of common stock owned by Ionis were $0.49 and $0.91, respectively. For the three and six months ended June 30, 2020, basic and diluted net loss per share of common stock owned by others was $0.49 and $0.91, respectively. For the three and six months ended June 30, 2019, basic and diluted net loss per share of common stock owned by Ionis was $0.40 and $0.06, respectively.  For the three and six months ended June 30, 2019, basic and diluted net loss per share of common stock owned by others was $0.40 and $0.26, respectively.

    Balance Sheet

    As of June 30, 2020, Akcea had cash, cash equivalents and short-term investments of $390 million compared to $464 million at December 31, 2019. With its strong balance sheet, the Company believes it is well positioned to continue to execute on its strategy and potentially broaden its pipeline.

    Conference Call

    At 4:30 p.m. Eastern Time today, August 4, 2020, Akcea will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 888-317-6003, passcode 9496130 or access the webcast at www.akceatx.com. A webcast replay will be available for a limited time at the same address.

    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, 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 TEGSEDI®, WAYLIVRA®, and Akcea's other 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.

     

    AKCEA THERAPEUTICS, INC.

    SELECTED FINANCIAL INFORMATION

    Condensed Consolidated Statements of Operations

    (In Thousands, Except Per Share Data)







    Three Months Ended





    Six Months Ended





    June 30,





    June 30,





    2020





    2019





    2020





    2019





    (unaudited)





    (unaudited)

    Revenue:































    Commercial revenue































    Product revenue, net



    $

    16,364





    $

    9,865





    $

    31,523





    $

    16,619

    Licensing revenue











    6,036













    6,036

    Total commercial revenue





    16,364







    15,901







    31,523







    22,655

    Research and development and license revenue under

       collaborative agreements





    6,013







    10,722







    6,928







    167,784

    Total revenue





    22,377







    26,623







    38,451







    190,439

    Expenses:































    Cost of sales and license





    5,046







    5,783







    9,829







    8,227

    Research and development





    29,731







    20,271







    47,086







    119,890

    Selling, general and administrative





    42,139







    50,740







    88,385







    95,342

    Net loss share from commercial activities under arrangement

       with Ionis Pharmaceuticals, Inc.





    (3,448)







    (11,465)







    (10,499)







    (20,521)

    Total expenses





    73,468







    65,329







    134,801







    202,938

    Loss from operations





    (51,091)







    (38,706)







    (96,350)







    (12,499)

    Other income (expense):































    Investment income





    1,055







    1,571







    2,826







    2,795

    Other expense





    (146)







    (28)







    (276)







    (140)

    Loss before income tax expense





    (50,182)







    (37,163)







    (93,800)







    (9,844)

    Income tax benefit (expense)





    605







    (160)







    1,373







    (292)

    Net loss



    $

    (49,577)





    $

    (37,323)





    $

    (92,427)





    $

    (10,136)

    Net loss per share of common stock owned by Ionis, basic and

       diluted



    $

    (0.49)





    $

    (0.40)





    $

    (0.91)





    $

    (0.06)

    Weighted-average shares of common stock outstanding owned by

       Ionis, basic and diluted





    77,095







    70,221







    77,095







    69,406

    Net loss per share of common stock owned by others, basic and

       diluted



    $

    (0.49)





    $

    (0.40)





    $

    (0.91)





    $

    (0.26)

    Weighted-average shares of common stock outstanding owned by

       others, basic and diluted





    24,382







    22,574







    24,196







    22,351

     

    AKCEA THERAPEUTICS, INC.

    Reconciliation of GAAP to Non-GAAP Basis:

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

    (In Thousands)







    Three Months Ended





    Six Months Ended





    June 30,





    June 30,





    2020





    2019





    2020





    2019





    (unaudited)





    (unaudited)

    As reported operating expenses according to GAAP



    $

    73,468





    $

    65,329





    $

    134,801





    $

    202,938

    Excluding compensation expense related to equity awards





    16,121







    14,363







    23,402







    32,923

    Non-GAAP operating expenses



    $

    57,347





    $

    50,966





    $

    111,399





    $

    170,015

    As reported loss from operations according to GAAP



    $

    (51,091)





    $

    (38,706)





    $

    (96,350)





    $

    (12,499)

    Excluding compensation expense related to equity awards





    16,121







    14,363







    23,402







    32,923

    Non-GAAP (loss) income from operations



    $

    (34,970)





    $

    (24,343)





    $

    (72,948)





    $

    20,424

    As reported net loss according to GAAP



    $

    (49,577)





    $

    (37,323)





    $

    (92,427)





    $

    (10,136)

    Excluding compensation expense related to equity awards





    16,121







    14,363







    23,402







    32,923

    Income tax effect related to compensation expense related to equity awards





    478



















    Non-GAAP net (loss) income



    $

    (32,978)





    $

    (22,960)





    $

    (69,025)





    $

    22,787

     

    Reconciliation of GAAP to non-GAAP Basis

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

    AKCEA THERAPEUTICS, INC.

    Condensed Consolidated Balance Sheets

    (In Thousands)







    June 30,





    December 31,







    2020





    2019







    (unaudited)



    Assets:

















    Cash and cash equivalents



    $

    135,744





    $

    306,866



    Short-term investments





    254,144







    156,806



    Accounts receivable





    14,049







    10,496



    Receivable from Ionis Pharmaceuticals, Inc.











    3,231



    Inventories





    13,756







    8,817



    Other current assets





    10,480







    10,689



    Property, plant and equipment, net





    5,895







    5,261



    Operating lease right-of-use assets





    10,646







    11,094



    Intangible assets, net





    80,127







    83,051



    Deposits and other assets





    3,096







    2,939



    Total assets



    $

    527,937





    $

    599,250



    Liabilities and stockholders' equity:

















    Accounts payable



    $

    4,253





    $

    10,216



    Payable to Ionis Pharmaceuticals, Inc.





    5,320









    Accrued compensation





    8,313







    12,793



    Accrued liabilities





    12,316







    14,191



    Deferred revenue





    263







    2,165



    Other current liabilities





    4,716







    2,633



    Long-term portion of lease liabilities





    13,545







    14,248



    Other long-term liabilities





    183









    Stockholders' equity





    479,028







    543,004



    Total liabilities and stockholders' equity



    $

    527,937





    $

    599,250



     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/akcea-reports-financial-results-and-highlights-for-second-quarter-2020-301105998.html

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  3. BOSTON, July 22, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the Main Association of Austrian Social Security Institutions (Dachverband der Österreichischen Sozialversicherungsträger, or DV) has granted approval for the national reimbursement of TEGSEDI® (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.

    "We are pleased that after an in-depth assessment of TEGSEDI, the Main Association of Austrian Social Security Institutions sees the significant unmet medical need in hATTR amyloidosis and recognizes the demonstrated benefit of TEGSEDI for people living with this progressing and potentially fatal disease in Austria," said Michael Pollock, SVP, head of Europe at Akcea. "This is a significant milestone for our team and we are excited to bring this much-needed treatment to eligible patients in Austria."

    hATTR amyloidosis is an under-recognized, debilitating and progressive disease that is caused by the buildup of transthyretin (TTR) proteins that misfold due to inherited genetic mutations. It is characterized by the deposition of amyloid fibrils throughout the body including in nervous tissue and can have a devastating impact on patients' quality of life.

    TEGSEDI is a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by silencing the defective gene in these patients and reducing production of the abnormal TTR protein. It is the first antisense oligonucleotide medicine available for patients in Austria with hATTR amyloidosis with polyneuropathy and also the first treatment available that can be self-administered in the comfort of their own home. 

    "Many patients in Austria have been living with the progressive and devastating effects of this disease for years with limited treatment options, and I am pleased to now be able to offer them an additional option that has been extensively studied," said Professor Wolfgang Löscher, Department of Neurology, University Hospital Innsbruck. "Research also suggests that earlier access to treatment for hATTR amyloidosis may help slow the progression of the disease, making this approval of reimbursement even more significant for the hATTR amyloidosis community in Austria."

    The positive reimbursement recommendation for TEGSEDI issued by the Main Association of Austrian Social Security Institutions was based on results from the Phase 3 NEURO-TTR study of the therapy in patients with hATTR amyloidosis with symptoms of polyneuropathy. Data from the study showed that patients treated with TEGSEDI experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression. 

    In 2018, TEGESDI was the first RNA-targeted therapeutic approved by the European Commission for adult patients with hATTR amyloidosis. Since then, TEGSEDI has continued to receive regulatory and reimbursement approvals globally, most recently gaining reimbursement approvals in Italy and Portugal. 

    ABOUT TEGSEDI® (INOTERSEN)

    TEGSEDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. TEGSEDI, discovered and developed by Ionis Pharmaceuticals, is the world's first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein. TEGSEDI also received marketing authorization in the European Union and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.

    The approval is based on data from the NEURO-TTR study that was a Phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant improvement compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with TEGSEDI experienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.

    The approval is also based on data from the NEURO-TTR Open Label Extension (OLE) that is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of TEGSEDI.

    For TEGSEDI's full prescribing information, please visit www.TEGSEDI.com.

    IMPORTANT SAFETY INFORMATION

    BOXED WARNING: THROMBOCYTOPENIA AND GLOMERULONEPHRITIS

    Thrombocytopenia

    • TEGSEDI causes reductions in platelet count that may result in sudden and unpredictable thrombocytopenia, which can be life-threatening. One clinical trial patient died from intracranial hemorrhage
    • TEGSEDI is contraindicated in patients with a platelet count below 100 x 109 /L
    • Prior to starting TEGSEDI, obtain a platelet count. During treatment, monitor platelet counts weekly if values are 75 x 109 /L or greater, and more frequently if values are less than 75 x 109 /L
    • If a patient develops signs or symptoms of thrombocytopenia, obtain a platelet count as soon as possible. The patient should not receive additional TEGSEDI unless a platelet count is determined to be interpretable and acceptable by a medical professional
    • Following discontinuation of treatment for any reason, continue to monitor platelet count for 8 weeks, or longer if platelet counts are less than normal, to verify that platelet counts remain above 75 x 109 /L

    Glomerulonephritis

    • TEGSEDI can cause glomerulonephritis that may require immunosuppressive treatment and may result in dialysis-dependent renal failure. One clinical trial patient who developed glomerulonephritis and did not receive immunosuppressive treatment remained dialysis dependent. In clinical trials, cases of glomerulonephritis were accompanied by nephrotic syndrome, which can have manifestations of edema, hypercoagulability with venous or arterial thrombosis, and increased susceptibility to infection
    • TEGSEDI should generally not be initiated in patients with urinary protein to creatinine ratio (UPCR) of 1000 mg/g or higher
    • Prior to starting TEGSEDI, measure the serum creatinine, estimated glomerular filtration rate (eGFR), urine protein to creatinine ratio (UPCR), and perform a urinalysis. During treatment, monitor serum creatinine, eGFR urinalysis, and UPCR every 2 weeks. TEGSEDI should not be given to patients who develop a UPCR of 1000 mg/g or higher or eGFR below 45 mL/minute/1.73 m2, pending further evaluation of the cause
    • If a dose is held, once eGFR increases to ≥45 mL/minute/1.73 m2, UPCR decreases to below 1000 mg/g, or the underlying cause of the decline in renal function is corrected, weekly dosing may be reinitiated. In patients with UPCR of 2000 mg/g or higher, perform further evaluation for acute glomerulonephritis, as clinically indicated. If acute glomerulonephritis is confirmed, TEGSEDI should be permanently discontinued

    TEGSEDI REMS Program

    • Because of the risks of serious bleeding caused by severe thrombocytopenia and because of glomerulonephritis, both of which require frequent monitoring, TEGSEDI is available only through a restricted distribution program under a Risk Evaluation and Mitigation Strategy (REMS) called the TEGSEDI REMS Program

    CONTRAINDICATIONS

    TEGSEDI is contraindicated in patients with

    • Platelet count below 100 x 109 /L
    • History of acute glomerulonephritis caused by TEGSEDI
    • History of a hypersensitivity reaction to TEGSEDI

    WARNINGS AND PRECAUTIONS

    Thrombocytopenia

    TEGSEDI causes reductions in platelet count that may result in sudden and unpredictable thrombocytopenia that can be life-threatening. In Study 1, platelet counts below 100 x 109 /L occurred in 25% of TEGSEDI-treated patients compared with 2% of patients on placebo. Platelet counts below 75 x 109 /L occurred in 14% of TEGSEDI-treated patients compared with no patients on placebo. One patient in a clinical trial experienced a fatal intracranial hemorrhage. Do not initiate TEGSEDI in patients with a platelet count below 100 x 109 /L. Follow recommended monitoring and treatment recommendations for platelet count.

    Symptoms of thrombocytopenia can include unusual or prolonged bleeding (eg, petechiae, easy bruising, hematoma, subconjunctival bleeding, gingival bleeding, epistaxis, hemoptysis, irregular or heavier than normal menstrual bleeding, hematemesis, hematuria, hematochezia, melena), neck stiffness, or atypical severe headache. Patients and caregivers should be instructed to be vigilant for symptoms of thrombocytopenia and seek immediate medical help if they have concerns.

     Glomerulonephritis and Renal Toxicity

    TEGSEDI can cause glomerulonephritis that may result in dialysis-dependent renal failure. In Study 1, glomerulonephritis occurred in 3 (3%) TEGSEDI-treated patients compared with no patients on placebo. One patient did not receive immunosuppressive treatment and remained dialysis-dependent. If glomerulonephritis is suspected, pursue prompt diagnosis and initiate immunosuppressive treatment as soon as possible. Follow recommended monitoring and treatment recommendations for renal parameters. TEGSEDI should generally not be initiated in patients with a UPCR of 1000 mg/g or greater. If acute glomerulonephritis is confirmed, TEGSEDI should be permanently discontinued.

    TEGSEDI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the TEGSEDI REMS Program because of risks of serious bleeding caused by severe thrombocytopenia and because of glomerulonephritis.

    Stroke and Cervicocephalic Arterial Dissection

    TEGSEDI may cause stroke and cervicocephalic arterial dissection. In clinical studies, 1 of 161 (0.6%) TEGSEDI-treated patients experienced carotid artery dissection and stroke. Educate patients on the symptoms of stroke and central nervous system arterial dissection. Instruct patients to seek help as soon as possible if symptoms of stroke or arterial dissection occur.

    Inflammatory and Immune Effects

    Inflammatory and immune changes are an effect of some antisense oligonucleotide drugs, including TEGSEDI. In clinical studies, serious inflammatory and immune adverse reactions occurred in TEGSEDI treated patients, including immune thrombocytopenia and glomerulonephritis, as well as a single case of antineutrophil cytoplasmic autoantibody (ANCA)–positive systemic vasculitis.

    Liver Injury

    In clinical studies, 8% of TEGSEDI-treated patients had an increased alanine aminotransferase (ALT) at least 3 times the upper limit of normal (ULN) compared with 3% of patients on placebo; 3% of TEGSEDI treated patients had an ALT at least 8 times the ULN compared with no patients on placebo. Monitor ALT, aspartate aminotransferase, and total bilirubin at baseline and every 4 months during treatment with TEGSEDI. If a patient develops clinical signs or symptoms suggestive of hepatic dysfunction, promptly measure serum transaminases and total bilirubin and interrupt or discontinue treatment with TEGSEDI, as appropriate.

    Liver Transplant Rejection

    In a clinical study, cases of liver transplant rejection were reported 2-4 months after starting TEGSEDI in patients whose liver allografts had previously been clinically stable (for over 10 years) prior to starting TEGSEDI. In these cases, the patients clinically improved and transaminase levels normalized after glucocorticoid administration and cessation of TEGSEDI.

    In patients with a history of liver transplant, monitor ALT, AST, and total bilirubin monthly. Discontinue TEGSEDI in patients who develop signs of liver transplant rejection.

    Hypersensitivity Reactions/Antibody Formation

    TEGSEDI can cause hypersensitivity reactions. In clinical studies, 6 of 161 (4%) TEGSEDI-treated patients stopped treatment because of a hypersensitivity reaction. These reactions generally occurred within 2 hours of administration of TEGSEDI. Antibodies to TEGSEDI were present when the reactions occurred. If a hypersensitivity reaction occurs, discontinue administration of TEGSEDI and initiate appropriate therapy. Do not use in patients who have a history of hypersensitivity reactions to TEGSEDI.

    Uninterpretable Platelet Counts: Reaction Between Antiplatelet Antibodies and Ethylenediaminetetraacetic acid (EDTA)

    In Study 1, 23% of TEGSEDI-treated patients had at least 1 uninterpretable platelet count caused by platelet clumping compared with 13% of patients on placebo. If there is suspicion of EDTA-mediated platelet clumping, perform a repeat platelet count using a different anticoagulant (eg, sodium citrate, heparin) in the blood collection tube. Recheck the platelet count as soon as possible if a platelet measurement is uninterpretable. Hold TEGSEDI dosing until an acceptable platelet count is confirmed with an interpretable blood sample.

    Reduced Serum Vitamin A Levels and Recommended Supplementation

    TEGSEDI treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance of vitamin A is advised for patients taking TEGSEDI. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (eg, night blindness).

    ADVERSE REACTIONS

    The most common adverse reactions that occurred in at least 20% of TEGSEDI-treated patients and more frequently than in those on placebo were injection site reactions, nausea, headache, fatigue, thrombocytopenia, and fever. Serious adverse reactions were more frequent in TEGSEDI-treated patients (32%) than in patients on placebo (21%).

    DRUG INTERACTIONS

    Because of the risk of thrombocytopenia, caution should be used when using antiplatelet drugs (including nonprescription products that affect platelets) or anticoagulants concomitantly with TEGSEDI. Because of the risk of glomerulonephritis and renal toxicity, caution should be used when using nephrotoxic drugs and other drugs that may impair renal function concomitantly with TEGSEDI.

    Please see full Prescribing Information, including boxed WARNING, at TEGSEDIhcp.com.

    ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS

    Hereditary ATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract. The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. Hereditary ATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis. Additional information on hereditary ATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com or by visiting www.hATTRGuide.com.

    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 us 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.

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  4. BOSTON, July 21, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., announced today that it will host a live webcast on Tuesday, August 4 at 4:30 p.m. Eastern Time to discuss its second quarter 2020 financial results and report on pipeline and business progress.

    Interested parties may listen to the call by dialing 1-888-317-6003, passcode 9496130, or access the webcast at https://www.webcaster4.com/Webcast/Page/2327/35686. A webcast replay will be available for a limited time at the same address.

    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, 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. 

     

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  5. BOSTON, July 20, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that Christophe Hotermans, M.D., Ph.D., has joined the company as senior vice president of global medical affairs where he will lead and evolve the execution of medical strategies, activities and operations worldwide in collaboration with executive management.

    "We are pleased to welcome Christophe to our leadership team and believe that his clinical experience and his experience leading the medical affairs function, including KOL engagement, medical communications and launch readiness, will be invaluable to the team," said Dr. William Andrews, chief medical officer at Akcea. "His expertise in developing global medical strategies in specialty care and rare diseases will be a great asset and will provide strategic expertise as we continue to successfully execute on our two commercial launches across the world."

    Dr. Hotermans' career has spanned more than 20 years, including more than 12 years in the biotechnology industry, where he held various roles in drug safety, International, U.S. and Global medical affairs, and the launch of five products across multiple therapeutic areas in specialty care and rare diseases. Prior to joining Akcea, Dr. Hotermans' served at Biogen for more than a decade in various roles, most recently as vice president and head of U.S. medical, where he was responsible for the U.S. medical strategy and execution across all therapeutic areas, including multiple sclerosis, Alzheimer's disease, movement disorders and amyotrophic lateral sclerosis. Prior to Biogen, he was chief of the neurology clinic at the University of Liège in Belgium.

    Dr. Hotermans received his medical degree and Ph.D. in medical sciences from The University of Liège, Belgium. He is an active member of The European Neurological Society, American Academy of Neurology and Medical Affairs Professional Society (MAPS) and has also authored multiple peer-reviewed journal publications.

    "It is an exciting time to join Akcea as the company makes important advances of multiple promising pipeline products and progress with the rollout of TEGSEDI and WAYLIVRA globally," said Dr. Hotermans. "I look forward to working with executive leadership, the medical affairs teams and engaging with doctors to bring transformational medicines to patients in need who are living with serious and rare diseases."

    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.

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