Akcea Therapeutics Recognizes Rare Disease Day by Spotlighting Several Historic "Firsts" for People Living with Familial Chylomicronemia Syndrome
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.
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.
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
Head of Communications, Akcea Therapeutics
T: +1 617 207 8509
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
Abonnera på våra pressmeddelanden. Endast mejladress behövs och den används bara här. Du kan avanmäla dig när som helst.
Senaste pressmeddelandena från GlobeNewswire
Karolinska Development delays publication of its Annual Report for 2018 and changes date for the Annual General Meeting26.4.2019 08:00:00 CEST | Pressmeddelande
STOCKHOLM, SWEDEN - 26 April 2019. Karolinska Development AB (Nasdaq Stockholm: KDEV) announces today that The Board of Directors has decided to delay the publication of its Annual Report for 2018. Karolinska Development has changed the date for the Annual General Meeting to June 26, 2019. The date for publication of the Annual Report for 2018 has been changed from April 26 to April 30, 2019. Karolinska Development has changed the date for the Annual General Meeting from June 4 to June 26, 2019. Notice to the Annual General Meeting will be published in a separate press release. For more information, please contact: Viktor Drvota, CEO, Karolinska Development AB Phone: +46 73 982 52 02, e-mail: firstname.lastname@example.org Fredrik Järrsten, CFO, Karolinska Development AB Phone: +46 70 496 46 28, e-mail: email@example.com TO THE EDITORS About Karolinska Development AB Karolinska Development AB (Nasdaq Stockholm: KDEV) is a Nordic life sciences investmen
Karolinska Development senarelägger publicering av årsredovisningen för 2018 och meddelar nytt datum för årsstämma26.4.2019 08:00:00 CEST | Pressmeddelande
STOCKHOLM, SVERIGE - 26 april 2019. Karolinska Development AB (Nasdaq Stockholm: KDEV) meddelar idag att styrelsen har beslutat att senarelägga publiceringen av årsredovisningen för 2018. Karolinska Development har ändrat datum för årsstämman till den 26 juni 2019. Publiceringen av årsredovisningen för 2018 flyttas från den 26 april till den 30 april 2019. Karolinska Development har ändrat datum för årsstämman från den 4 juni till den 26 juni 2019. Kallelse till årsstämman kommer att publiceras senare i ett separat pressmeddelande. För ytterligare information, vänligen kontakta: Viktor Drvota, vd, Karolinska Development AB Tel: +46 73 982 52 02, e-mail: firstname.lastname@example.org Fredrik Järrsten, finanschef, Karolinska Development AB Tel: +46 70 496 46 28, e-mail: email@example.com TILL REDAKTÖRERNA Om Karolinska Development AB Karolinska Development AB (Nasdaq Stockholm: KDEV) är ett nordiskt investmentbolag inom life science. Bolaget fokuserar p
Karolinska Developments portföljbolag Aprea Therapeutics har fått särläkemedelsstatus och snabbspårstatus från FDA för APR-24616.4.2019 12:46:00 CEST | Pressmeddelande
STOCKHOLM, SVERIGE - 16 april 2019. Karolinska Development AB meddelar idag att portföljbolaget Aprea Therapeutics har av FDA fått särläkemedelsstatus för APR-246 för behandling av patienter med TP53-muterad myelodysplastiskt syndrom (MDS). Dessutom ger FDA bolaget snabbspårstatus (Fast Track Designation), för APR-246 för behandling av MDS. Amerikanska läkemedelsverket, FDA, ger särläkemedelsstatus till läkemedelskandidater för att snabba på utvärderingen och utvecklingen av säkra och effektiva behandlingar av ovanliga sjukdomar. Särläkemedelsstatus ger företag både regulatoriska och kommersiella incitament genom att läkemedlet får marknadsexklusivitet på den amerikanska marknaden i sju år efter marknadsgodkännande samtidigt som företaget får stöd från FDA när det gäller designen av kliniska prövningar, skatteförmåner för kostnader kopplade till kliniska prövningar och avgiftsbefrielse från FDA. FDA:s snabbspår underlättar utvecklingen av läkemedel som är avsedda för att behandla allva
Karolinska Development's portfolio company Aprea Therapeutics has received FDA Orphan Drug Designation and Fast Track Designation for APR-24616.4.2019 12:46:00 CEST | Pressmeddelande
STOCKHOLM, April 16, 2019. Karolinska Development's portfolio company Aprea Therapeutics has from FDA received an Orphan Drug Designation for APR-246 for the treatment of patients with Myelodysplastic Syndromes (MDS) having a TP53 mutation. In addition, FDA has also granted Fast Track Designation to APR-246 for treatment of MDS. Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, tax credits related to clinical trial expenses, and an exemption from FDA user fees. The FDA's Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to addre
Bergman & Beving AB: Bergman & Beving förvärvar KGC10.4.2019 14:00:00 CEST | Pressmeddelande
Pressmeddelande Bergman & Beving förvärvar KGC Bergman & Beving har idag tecknat avtal om förvärv av samtliga aktier i KGC Verktyg & Maskiner AB. KGC, med säte i Älvsjö, har i mer än 60 år utvecklat och levererat kvalitetsverktyg och tillbehör för murning och plattsättning i det egna varumärket KGC. Bolaget omsätter cirka 80 MSEK per år, och har 24 anställda. "KGC är ett ledande varumärke med högt anseende hos murare och plattsättare och har en mycket stark ställning på den svenska marknaden", säger Pontus Boman, VD och koncernchef. Tillträde beräknas ske den 1 maj 2019 och förvärvet bedöms ha en marginellt positiv påverkan på Bergman & Bevings resultat per aktie under innevarande räkenskapsår. Stockholm den 10 april 2019 Bergman & Beving AB (publ) För ytterligare information kontakta: Pontus Boman, VD & Koncernchef, telefon 010-454 77 00 Peter Schön, CFO, telefon 070-339 89 99 Denna information lämnades, genom ovanstående kontaktpersoners försorg, för offentliggörande den 10 april 201
Bergman & Beving AB: Bergman & Beving acquires KGC10.4.2019 14:00:00 CEST | Pressmeddelande
Press release Bergman & Beving acquires KGC Bergman & Beving has today signed an agreement to acquire all shares in KGC Verktyg & Maskiner AB. KGC, based in Älvsjö, has for more than 60 years developed and delivered quality tools and accessories for bricklayers and tilers in its own brand KGC. The business has a turnover of approximately SEK 80 million per year, and has 24 employees. "KGC is a leading brand with high reputation among bricklayers and tilers and has a very strong position in the Swedish market," says Pontus Boman, President and CEO. The closing is taking effect on 1 May 2019 and the acquisition is expected to have a marginal positive impact on Bergman & Beving's earnings per share during the current fiscal year. Stockholm, 10 April 2019 Bergman & Beving AB (publ) For further information, please contact: Pontus Boman, President & CEO, Tel: +46 10 454 77 00 Peter Schön, CFO, Tel: +46 70 339 89 99 The information was submitted for publication, through the agency of the cont
I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.Besök vårt pressrum