Akcea Announces Completion of Landmark Study to Assess Disease Burden in People Living with FCS
Results published in 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."
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:
Head of Communications and Investor Relations
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
One Liberty Plaza - 165 Broadway
NY 10006 New York
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
New Samsung Galaxy Ultrasonic Fingerprint System World’s First to Achieve FIDO Biometric Certification20.2.2019 21:30:00 | Pressemelding
Samsung’s new “in-display” Ultrasonic Fingerprint system certified by FIDO Alliance as meeting globally recognized standards for biometric recognition performance and Presentation Attack Detection (PAD) MOUNTAIN VIEW, Calif., Feb. 20, 2019 (GLOBE NEWSWIRE) -- The FIDO Alliance announced today that the Samsung Galaxy S10 and S10+ smartphones are the first products to feature certification from the FIDO Alliance’s new Biometric Component Certification Program. This certification validates that the new in-display fingerprint recognition system meets industry standards for user verification and detecting presentation (or “spoof”) attacks. “As mobile devices increasingly become our primary means of storing sensitive information and conducting critical transactions, the industry leaders in FIDO Alliance have determined the market needs a standard-based means to assess the efficacy of biometric certification components -- which is why we recently launched our Biometric Component Certification
Infovista Expands 5G Network Testing and Planning Capabilities20.2.2019 15:00:00 | Pressemelding
ASHBURN, Va., Feb. 20, 2019 (GLOBE NEWSWIRE) -- Infovista, the leader in modern network performance, today announced new versions of its TEMS Investigation and Planet solutions. With this announcement, Infovista is the only vendor offering the complete 5G portfolio comprising full network planning and testing in a single, integrated platform. Infovista is now uniquely positioned to help mobile network operators roll out 5G NR networks by including the ability to provide integrated Geodata, 5G Planning and Network Testing capabilities. Tweet This: @Infovista expands 5G network testing and planning capabilities, offering complete 5G portfolio in a single, integrated platform: https://bit.ly/2Nm8Rup With the new release of TEMS Investigation, Infovista introduces support for 5G smartphones based on the Qualcomm X50 chipset. With initial support of Qualcomm based devices and plans for agnostic device support, customers will be able to choose between different test devices and scanners to s
Reminder: Constellation Brands to Present at Consumer Analyst Group of New York Conference, February 20, 201920.2.2019 14:30:00 | Pressemelding
VICTOR, N.Y., Feb. 20, 2019 (GLOBE NEWSWIRE) -- Constellation Brands, Inc. (NYSE: STZ and STZ.B), a leading beverage alcohol company, reiterates that Bill Newlands, president and chief operating officer, and David Klein, chief financial officer, will present at the Consumer Analyst Group of New York (CAGNY) conference on Wednesday, February 20, 2019, at the Boca Raton Resort & Club in Florida. The presentation is scheduled to begin at 3:00 p.m. EST and is expected to cover the company’s strategic business activities, including a discussion on portfolio strategy; financial and operational performance; and outlook for the future, including a discussion on the accounting methodology and potential impact on fourth quarter fiscal year 2019 non-cash equity in earnings from the company’s Canopy Growth investment. A live audio webcast of the presentation can be accessed on the company’s website at www.cbrands.com by following the instructions in the “Investors” section under “Events & Presenta
Fortuna provides construction update at its Lindero gold project in Argentina20.2.2019 11:00:00 | Pressemelding
VANCOUVER, British Columbia, Feb. 20, 2019 (GLOBE NEWSWIRE) -- Fortuna Silver Mines Inc. (NYSE: FSM) (TSX: FVI) is pleased to provide an update on construction activities at its 100 percent owned Lindero gold Project located in the Province of Salta, Argentina. Following the cessation of unusually heavy rainfall in Salta Province in recent weeks, which resulted in damage to the road leading to the Lindero Project site (see Fortuna news release dated February 7, 2019), management is pleased to report that the road has been repaired and on February 18th the mobilization of personnel and materials to the project site resumed. Mr. Jorge A. Ganoza, President, CEO and Director, commented, “Abnormal rains and electrical thunder storms not seen in a decade in the region are impacting our construction activities and schedule projections. A large amount of the float in the construction schedule has been consumed due to a slow start and ramp-up in excavations at the leach pad and crushing plant,
DoubleVerify Acquires Digital Video Technology Company, Zentrick20.2.2019 09:00:00 | Pressemelding
Solving 30% breakage in video ad delivery NEW YORK and GHENT, Belgium, Feb. 20, 2019 (GLOBE NEWSWIRE) -- DoubleVerify, the leading independent provider of marketing measurement software and analytics, today announced that it has acquired Zentrick, a digital video technology company that provides middleware solutions to drive the seamless execution and performance of online video advertising for the world’s largest brands, platforms and publishers. The Zentrick engineering teams will continue to operate from offices in Ghent, Belgium. The transaction was completed as an all cash, all stock offer on February 15, 2019. Video advertising spend soared to $28 Billion in 2018. However, the ecosystem necessary to support the growth in video ad delivery has become highly fragmented. Today over 30% of video ads that are sold are unable to be delivered by publishers due to various forms of technical ‘breakage’ in the ad delivery chain – an issue that’s particularly acute in the key areas of mobil
ADC Therapeutics Announces First Patient Dosed in Phase I Clinical Trial of ADCT-402 (loncastuximab tesirine) and Ibrutinib in Patients with Advanced Diffuse Large B-Cell Lymphoma or Mantle Cell Lymphoma20.2.2019 07:00:00 | Pressemelding
Combination trial to evaluate safety and anti-tumor activity of agents that target B-cell cancers LAUSANNE, Switzerland, Feb. 20, 2019 (GLOBE NEWSWIRE) -- ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), today announced that the first patient has been dosed in a Phase I clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumor activity of ADCT-402 (loncastuximab tesirine) in combination with Pharmacyclics LLC’s ibrutinib in patients with advanced diffuse large B-cell lymphoma (DLBCL) or mantle cell lymphoma (MCL). ADCT-402, an ADC designed to target and kill CD19-expressing malignant B-cells, is also being evaluated in an ongoing pivotal Phase II clinical trial in patients with relapsed or refractory (R/R) DLBCL and a Phase I clinical trial in combination with IMFINZI® (durvalumab) in patients with multiple types of R/R non-Hodgkin lymphoma. Ibrutinib, a sma