GlobeNewswire

Akcea Therapeutics Recognizes Rare Disease Day by Spotlighting Several Historic "Firsts" for People Living with Familial Chylomicronemia Syndrome

Dela

Upcoming global FCS Summit will enable advocates to connect and build a stronger FCS community

CAMBRIDGE, Mass., Feb. 28, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced the company's support for Rare Disease Day 2018, including many first-time efforts to connect and support the global community for individuals and families affected by familial chylomicronemia syndrome (FCS).

"As with many individuals who are diagnosed with a rare disease, people living with FCS and their caregivers often express feelings of isolation. Most have never met another person with FCS," said Paula Soteropoulos, president and chief executive officer of Akcea. "We are proud to support efforts to help more people living with FCS build the connections they need for information, support and advocacy. There has been a lot of progress over the past several years, but there is still much more work ahead. With a stronger and more connected FCS community, we can raise more awareness of the burden of this disease, help patients receive a diagnosis faster, and work to improve the lives of more patients in need around the world."

Established in 2008 by EURORDIS, Rare Disease Day takes place on the last day of February each year, with the goal to raise awareness about rare diseases and their impact on patients and their caregivers.

Among the "firsts" for the FCS community in 2017 or planned for 2018:

  • Akcea will sponsor the FCS Global Connection Summit, a first-ever global advocacy meeting taking place in March 2018 in The Netherlands. The program will support efforts to connect, share learnings and build a stronger FCS advocacy community.
     
  • "Connection and Hope," a new video sponsored by Akcea featuring an FCS patient named Aaron and his community of caregivers, will be distributed to global audiences. Aaron's story helps to demonstrate the value of connecting with others with similar journeys when diagnosed with a rare disease. To view this video, click here.
     
  • Akcea sponsored a meeting in Canada for the FCS community that included a demonstration of customized meal preparation in collaboration with The Culinary Institute.
     
  • A new outline of nutritional resources specifically designed for people living with FCS, developed by dieticians and other experts and supported by Akcea, is now available at on FCSFocus.com.
     
  • A series of webinars led by registered dieticians, nutritionists and psychologists with information about healthy eating for people living with FCS was launched.
     
  • Results of the first study of the impact of FCS on patient quality of life - known as the IN-FOCUS Study - were presented at the National Organization for Rare Diseases (NORD) Rare Diseases and Orphan Products Breakthrough Summit in Washington, D.C.

ABOUT FCS
FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available. Additional information on FCS is available at www.fcsfocus.com, through the FCS Foundation at http://www.livingwithfcs.org and the LPLD Alliance at www.lpldalliance.org. For a full list of organizations supporting the FCS community worldwide, please click here.

ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. Akcea is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, all with the potential to treat multiple diseases. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. The most advanced drug in its pipeline, volanesorsen, is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally with a focus on lipid specialists as the primary call point. Akcea is located in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.

FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of volanesorsen and other products in development. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. 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. 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 its annual report on Form 10-K for the year ended December 31, 2017, which is on file with the SEC.

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(TM) is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics(TM) is a trademark of Ionis Pharmaceuticals, Inc.

Investor and Media Contact
Kathleen Gallagher
Head of Communications, Akcea Therapeutics
E: kgallagher@akceatx.com 
T: +1 617 207 8509




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Akcea Therapeutics, Inc. via Globenewswire

Om

GlobeNewswire



Följ GlobeNewswire

Abonnera på våra pressmeddelanden.

Senaste pressmeddelandena från GlobeNewswire

Arcoma AB: Arcoma presenterar på Redeye Life Science Day den 21:a november16.11.2018 12:53Pressmeddelande

16 November Den 21 november presenterar Jesper Söderqvist, VD på Arcoma, den senaste utvecklingen i Arcoma på Haymarket by Scandic i Stockholm. Presentationen "Arcoma, ett tillväxtbolag i en växande marknad" hålls klockan 13:00 och kan följas i livesändning på redeye.se/live/lsday-2018. Presentationen kommer även i efterhand att finnas tillgänglig på www.redeye.se/live/ls-day-2018 samt på Bolagets sida i Redeye Universe (redeye.se/company/Arcoma) Om Arcoma Arcoma, med lång erfarenhet av branschen, är en ledande leverantör av integrerade digitala röntgensystem med hög kvalitet och avancerad teknik. Arcomas produkter erbjuder den senaste digitala bildtekniken kombinerat med tekniskt avancerade rörliga positioneringssystem, vilket tillsammans med ergonomisk skandinavisk design, erbjuder kunden kompletta, konfigurerbara och funktionella digitala röntgensystem. Bolagets produkter säljs via återförsäljare samt via OEM-kunder och det finns idag över 3 500 av Arcomas röntgensystem installerade

Iconovo: kundprojekt blir framflyttat tre till sex månader8.11.2018 15:45Pressmeddelande

Fortsatt arbete med ett av Iconovos pågående kundprojekt kommer att flyttas fram tre till sex månader. Projektet avser utveckling av produkt anpassad för kund som ska svara för produktion och marknadslansering. Det aktuella arbetet var planerat att ske under fjärde kvartalet men beräknas ske under nästa år, och detta påverkar Iconovos omsättning under 2018 negativt med cirka 2 miljoner kronor. Orsaken är att kunden har beslutat att genomföra en mindre klinisk studie för att verifiera produkten innan nästa steg tas vilken omfattar investeringar i betydande mångmiljonbelopp i produktionskapacitet, baserat på Iconovos utveckling. Studien görs för kundens egna räkning och är inte villkorad av tillsynsmyndigheter. "Detta påverkar vårt resultat på kort sikt, men det faktum att kunden har beslutat att göra en betydande investering i faktisk produktion betyder att vi med stor säkerhet kan se fram emot licensintäkter under en lång tidsrymd framöver i samband med att produkten lanseras på markna

Stillfront Group AB: Inbjudan till Stillfront Groups kvartalsrapport för tredje kvartalet 20187.11.2018 14:34Pressmeddelande

PRESSMEDDELANDE 7 november 2018 Inbjudan till Stillfront Groups kvartalsrapport för tredje kvartalet 2018 Stillfront Groups kvartalsrapport för det tredje kvartalet 2018 kommer att publiceras klockan 07.00 den 22 november 2018. En webbsänd telefonkonferens kommer att hållas klockan 10.00 samma dag där Jörgen Larsson, VD och Sten Wranne, CFO, kommenterar resultatet. Presentationen och rapporten kommer efter presentationen att finnas tillgänglig på www.stillfront.com Webbsändningen nås på adressen: https://tv.streamfabriken.com/stillfront-q3-2018 För att delta via telefon, vänligen ring: SE: +46 8 566 426 63 UK: +44 20 300 898 01 US: +1 8 557 532 235 FÖR YTTERLIGARE INFORMATION, VÄNLIGEN KONTAKTA: Sofia Wretman, IR Phone: +46 708 11 64 30 sofia@stillfront.com OM STILLFRONT Stillfront är en oberoende utvecklare, förläggare och distributör av digitala spel - med visionen att bli en ledande indie-spelutvecklare och förläggare. Stillfront är verksamt genom tio näst intill självständiga dotte

Stillfront Group AB: Invitation to Stillfront Group Q3 presentation7.11.2018 14:34Pressmeddelande

Invitation to Stillfront Group Q3 presentation Stillfront Group interim report for the third quarter 2018 will be published at 07.00 CET on 22 November 2018. A presentation of the report will be held the same day at 10.00 CET via telephone conference or audiocast where Jörgen Larsson, CEO and CFO Sten Wranne are to comment on the report. The presentation can be viewed live at https://tv.streamfabriken.com/stillfront-q3-2018 To participate via phone please call: SE: +46 8 566 426 63 UK: +44 20 300 898 01 US: +1 8 557 532 235 FOR FURTHER INFORMATION, PLEASE CONTACT: Sofia Wretman, IR Phone: +46 708 11 64 30 sofia@stillfront.com ABOUT STILLFRONT Stillfront is a group of independent creators, publishers and distributors of digital games - with a vision to become the leading group of indie game creators and publishers. Stillfront operates through ten near-autonomous subsidiaries: Bytro Labs in Germany, Coldwood Interactive in Sweden, Power Challenge in the UK and Sweden, Dorado Online Games

Modus Therapeutics announces FDA Acceptance of Sevuparin IND for the treatment of sickle cell disease7.11.2018 08:30Pressmeddelande

STOCKHOLM, November 7, 2018. Karolinska Development's portfolio company Modus Therapeutics announces today that the U.S Food & Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application to initiate a Phase I clinical trial with subcutaneous sevuparin for the treatment of sickle cell disease (SCD) FDA has decided to accept Modus Therapeutics' IND application of sevuparin for the treatment of SCD. Sevuparin is an innovative, proprietary modified polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. It has the potential to restore blood flow and prevent further microvascular obstructions in children and adults with SCD. At present, sevuparin is being evaluated as an intravenous infusion in a Phase II study in SCD patients with ongoing painful crises (also called vaso occlusive crises, VOC). In order to broaden the clinical scope and utility of sevuparin in the treatment of SCD, t

Modus Therapeutics announces FDA Acceptance of Sevuparin IND for the treatment of sickle cell disease7.11.2018 08:30Pressmeddelande

STOCKHOLM, November 7, 2018. Karolinska Development's portfolio company Modus Therapeutics announces today that the U.S Food & Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application to initiate a Phase I clinical trial with subcutaneous sevuparin for the treatment of sickle cell disease (SCD) FDA has decided to accept Modus Therapeutics' IND application of sevuparin for the treatment of SCD. Sevuparin is an innovative, proprietary modified polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. It has the potential to restore blood flow and prevent further microvascular obstructions in children and adults with SCD. At present, sevuparin is being evaluated as an intravenous infusion in a Phase II study in SCD patients with ongoing painful crises (also called vaso occlusive crises, VOC). In order to broaden the clinical scope and utility of sevuparin in the treatment of SCD, t

I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.

Besök vårt pressrum