GlobeNewswire

Akcea Announces Completion of Landmark Study to Assess Disease Burden in People Living with FCS

Del

Results published in Expert Review of Cardiovascular Therapy

CAMBRIDGE, Mass., June 13, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced completion of a new study to assess the burden of people living with Familial Chylomicronemia Syndrome (FCS) and its impact on their daily lives. Results from the study known as Re-FOCUS were published on June 11, 2018 in the peer-reviewed journal Expert Review of Cardiovascular Therapy .

Twenty-two people with FCS participated in the Re-FOCUS survey. They were asked to assess the number and severity of a range of symptoms and daily life measures affected by FCS. The web-based global study was conducted among people with FCS treated with volanesorsen, Akcea's investigational therapy currently under regulatory review in the US, EU and Canada.

"FCS is an ultra-rare disease that has not been widely studied. This landmark study represents an important step in our efforts to identify and quantify the many ways that FCS can affect the physical, functional and emotional health of people living with FCS," said Michael W. Stevenson, R.Ph., Ph.D., global vice president, medical affairs at Akcea. "The Re-FOCUS study provides helpful, new information as we work to design and validate a tool that can accurately evaluate changes in disease burden with therapeutic intervention." 

The Re-FOCUS study represents another milestone in Akcea's ongoing efforts to understand the impact of FCS. In October 2017, Akcea announced results of the IN-FOCUS study, a survey of 166 people with FCS in 10 countries. The data provided new insight on the disease burden, including cognitive, emotional, and physical impairments that can affect daily life as well as the risk of recurrent, potentially fatal, acute pancreatitis.

"For people living with FCS, it impacts every part of daily life, often making it difficult to work, care for other family members or even join in life events with family and friends. Studies like IN-FOCUS and Re-FOCUS can enable patients and caregivers around the world to better understand and communicate about the devastating effects of this rare disease," said Lindsey Sutton, co-president of the FCS Foundation.

"We are working to increase understanding of FCS, through our clinical programs, scientific communications, and by bringing forward the voices of those who live with this disease," said Molly Harper, vice president, and global franchise head, cardiometabolics. "We would like to thank the FCS Foundation as well as all of the patients, caregivers and clinicians who participated in or supported this groundbreaking work, which will help build awareness and improve quality of care for patients with FCS and potentially other rare diseases."

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) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL 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. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. There is no effective therapy for FCS currently available. Additional information on FCS is available at www.fcsfocus.com, and 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, INC.
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDITM (inotersen), WAYLIVRATM (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. TEGSEDI is under regulatory review in the U.S., EU and Canada for the treatment of people with hereditary transthyretin amyloidosis (hATTR). WAYLIVRA 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 people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered 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 WAYLIVRATM (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), TEGSEDITM and WAYLIVRATM are trademarks of Akcea Therapeutics, Inc.

Akcea Media and Investor Contact:
Kathleen Gallagher                                                                         
Head of Communications and Investor Relations 
(617)-207-8509
kgallagher@akceatx.com




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

GlobeNewswire
GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York

https://globenewswire.com

GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

Følg saker fra GlobeNewswire

Registrer deg med din epostadresse under for å få de nyeste sakene fra GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra GlobeNewswire

Next Green Wave Launches Bold New Line of THC Products with Grammy Nominated King Louie18.6.2019 11:29:00 CESTPressemelding

VANCOUVER, British Columbia, June 18, 2019 (GLOBE NEWSWIRE) -- Next Green Wave Holdings Inc. (CSE: NGW) (OTCQB: NXGWF) ("Next Green Wave", “NGW” or the "Company") is celebrating its next brand roll-out with influential Chicago hip-hop star King Louie. Through an exclusive partnership with SD Cannabis, King Louie will be launching premium flower THC products and concentrates throughout his new ‘Life of Louie’ album world-tour and appearances at selected dispensaries throughout Southern California.* Next Green Wave CEO, Leigh Hughes commented, “We’re thrilled to be collaborating on this product launch with King Louie, he is a trailblazer in the hip-hop industry and influential ambassador of cannabis culture in America. Together, we have the opportunity to shape the norms and perceptions surrounding cannabis culture and will offer consumers an elevated experience through the quality and delivery of our extracted oils and exclusive flower products.” “It’s a lifestyle for us, so it’s gonna

Bombardier to supply 30 additional FLEXITY Trams to Brussels Transportation Company18.6.2019 10:41:00 CESTPressemelding

Additional FLEXITY trams to boost Brussels Transport Company’s fleet capacity and meet demand for growing passenger numbers BERLIN, June 18, 2019 (GLOBE NEWSWIRE) -- Note to editors: To view the photo associated with this press release, please visit the following link: https://www.globenewswire.com/NewsRoom/AttachmentNg/94e847eb-40b7-42c3-a196-d7783859b9c9 Mobility technology leader Bombardier Transportation has received an order to supply 30 additional BOMBARDIER FLEXITY trams to the Brussels Intercommunal Transportation Company (STIB) in Belgium. This second call off order is part of a framework contract for up to 175 trams with an initial call off of 60 trams signed in April 2018. The order is valued at approximately 67 million euro ($75 million US). First deliveries of the 30 additional five-car, 32-meter trams are planned from December 2020. Thomas Ryckalts, President of the STIB Board, said, “After a first order for 60 new trams, the STIB board took a decision in favor of our pas

Carpenter Technology Advances Aerospace Industry’s Shift to Electric18.6.2019 08:00:00 CESTPressemelding

LE BOURGET, France, June 18, 2019 (GLOBE NEWSWIRE) -- Carpenter Technology (NYSE: CRS) today announced its involvement with multiple OEMs to progress their electric aircraft aspirations. Carpenter Technology will provide advanced soft magnetic expertise and ultra-efficient electrical components to be used in R&D and demonstrator motors and engines, leading to construction of lighter-weight, longer-lasting electric propulsion systems. Alongside its partners, Carpenter Technology’s goal is to enable development of the highest power density motors available. “As the industry leader in electric propulsion material solutions, we are well positioned to support the development of electric systems to power the aircraft of tomorrow,” says Marshall Akins, Carpenter Technology’s Vice President, Aerospace Markets. “Our long history and comprehensive knowledge of how to develop and process these type of material solutions and then translate them into finished components so they function successfull

WillScot Announces Appointment of General Counsel17.6.2019 22:29:00 CESTPressemelding

BALTIMORE, June 17, 2019 (GLOBE NEWSWIRE) -- WillScot Corporation (“WillScot”) (Nasdaq: WSC) today announced the appointment of Mr. Hezron Timothy Lopez to serve as the Company’s Vice President, General Counsel & Corporate Secretary, effective June 17, 2019. Mr. Lopez joins WillScot after having served from 2012 to 2018 as Senior Vice President, General Counsel and Corporate Secretary of Herman Miller, Inc., a Nasdaq-listed manufacturer of home and office furniture. From 2008 to 2012, Mr. Lopez served as Associate General Counsel and Head of Merger & Acquisition, Commercial and International for A. O. Smith Corporation, a manufacturer of water heating equipment and water treatment products. About WillScot Corporation Headquartered in Baltimore, Maryland, WillScot (Nasdaq: WSC) is the public holding company for the Williams Scotsman family of companies and is the specialty rental services market leader providing innovative modular space and portable storage solutions across North Americ

Cineworld Group and Cinionic Strike 1,000+ Unit Deal to Roll Out Acclaimed Barco Laser Projectors to Theaters Worldwide17.6.2019 18:05:00 CESTPressemelding

Cineworld embarks on global renewal with Cinionic, powered by award-winning projection portfolio and enhanced services KORTRIJK, Belgium, June 17, 2019 (GLOBE NEWSWIRE) -- Cinionic, the Barco, CGS, and ALPD cinema joint venture providing a new visual standard with enhanced services and technology solutions, announces a significant expansion in its relationship with one of the world’s largest theater chains, Cineworld Group plc (LON:CINE), to continue delivering an elevated movie-going experience for audiences worldwide. Cinionic will illuminate Cineworld Group operated theaters in the U.S., EU and UK with enhanced services and award-winning portfolio of Barco laser projectors, including the newly launched Barco Series 4. Over the next 18 months, Cineworld will upgrade and deploy over 1,000 projectors worldwide with Barco Laser solutions to deliver a new visual standard in cinema, supported by Barco Alchemy media servers and maintained for the next decade through Cinionic’s enhanced ser

Rock Tech Engages KBM Resources Group for LiDAR Survey at Georgia Lake17.6.2019 14:15:00 CESTPressemelding

VANCOUVER, British Columbia, June 17, 2019 (GLOBE NEWSWIRE) -- Rock Tech Lithium Inc. (the "Company" or “Rock Tech”) (TSX-V: RCK; Frankfurt: RJIB) is pleased to announce that it has engaged KBM Resources Group (“KBM”) to complete a LiDAR and high-resolution imagery survey at its 100%-owned Georgia Lake lithium project in Ontario, Canada. The aerial acquisition of LiDAR and digital photography will cover the Nama Creek main zone and adjacent areas of the Georgia Lake lithium project. The LiDAR survey, with a relatively high resolution of 10 laser points per square metre and an absolute accuracy of 10 cm vertical and 20 cm horizontal, will produce LAS files with classified ground points including a bare earth model and georeferenced orthoimagery. “The ideal time to fly this survey is after the snow melt and before leaf-out,” commented Martin Stephan, Rock Tech’s Chief Executive Officer. “These conditions, combined with a favourable regional weather forecast, shall make for a successful L