GlobeNewswire

Akcea and Ionis Receive Positive EU CHMP Opinion for WAYLIVRATM (volanesorsen)

Del

First and only therapy approved for FCS, a serious and rare disease with no approved treatment options

BOSTON, Mass. and CARLSBAD, Calif., March 01, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization for WAYLIVRA as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) who are at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. Click here to read the EMA’s press release.

The positive opinion will now be referred to the European Commission (EC), which grants marketing authorization for medicines in the European Union for the adoption of a decision on an EU-wide marketing authorization. As part of the conditional marketing authorization, Akcea and Ionis will conduct a non-interventional post-authorization safety study (PASS) based on a Registry.

“This positive CHMP opinion is an important step forward as we work to bring WAYLIVRA to people living with FCS who currently have no treatment options. Once approved, WAYLIVRA will be the first and only therapy for people living with the devastating challenges of FCS. We are now anticipating approval in Europe in the coming months. We will build on the strong infrastructure we have in place for TEGSEDI® (inotersen) in Europe as we prepare for the launch of WAYLIVRA,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “This is a testament to the European Regulatory Authorities’ commitment to facilitating access to innovative medicines for patients in need.”

WAYLIVRA’s Marketing Authorization Application (MAA) is based on results from the Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension study and 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 by 77 percent after 3 months of treatment.  The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels. In addition to the OLE study, there are also ongoing global Early Access Programs for WAYLIVRA.

“The FCS community is encouraged by the positive CHMP opinion and we remain very hopeful that people living with FCS will soon have an approved treatment available in the EU,” said Jill Prawer, founder and chair, LPLD Alliance “Without a treatment, people living with FCS have debilitating daily symptoms as well as the constant fear of pancreatitis. This is a positive step forward for people living with FCS who currently have no treatment options. Patients around the world need and deserve a treatment for FCS.”

“WAYLIVRA will be the third medicine for which we will have received approval in the last two years. Our antisense technology platform continues to deliver many important scientific and medical advances that should support continuing growth,” said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals.  “We look forward to continuing to work with the community of patients and the dedicated clinicians and researchers around the world to make this treatment available to patients who need it as quickly as possible.”

ABOUT WAYLIVRA AND FCS
WAYLIVRA, a product of Ionis’ proprietary antisense technology, is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters.

WAYLIVRA is under regulatory review in the E.U. for the treatment of people with familial chylomicronemia syndrome (FCS). Akcea is working to confirm a path forward for WAYLIVRA in the US and in Canada.

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, including chronic pancreatitis and pancreatogenic diabetes. 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, 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

WAYLIVRA is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in mid-2019.

ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI® (inotersen) and advancing a mature pipeline of novel drugs, including WAYLIVRA™ (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 approved in the U.S., E.U. and Canada. WAYLIVRA is under regulatory review 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 Boston, Massachusetts. Additional information about Akcea is available at www.akceatx.com and you can follow us on twitter at @akceatx.

ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.  
 
To learn more about Ionis follow us on twitter @ionispharma or visit http://ir.ionispharma.com/

AKCEA’S AND IONIS’ FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. and the therapeutic and commercial potential of WAYLIVRATM (volanesorsen). Any statement describing Akcea’s or Ionis’ goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of WAYLIVRA or other of Akcea’s or Ionis’ drugs 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 drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea’s and Ionis’ 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 and Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea and Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis’ and Akcea’s programs are described in additional detail in Ionis’ and 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 each 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 WAYLIVRATM are trademarks of Akcea Therapeutics, Inc.

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

Akcea Media Contact:
Bill Berry
Berry & Company
T: 212 253-8881
bberry@berrypr.com

Lynn Granito
Berry & Company
T: 212 253-8881
lgranito@berrypr.com

Ionis Investor Contact:
D. Wade Walke, Ph.D.
Vice President, Investor Relations
760-603-2741
wwalke@ionisph.com

Ionis Media Contact:
Roslyn Patterson
Vice President, Corporate Communications
760-603-2681
rpatterson@ionisph.com

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

Real World Cloud Orchestration Facilitates Easy Migration to All-IP18.4.2019 22:05:00 CESTPressemelding

For All-IP service providers in Malta and the Netherlands, Patton Cloud edge orchestration has lowered costs with fast, simple activation and management of enterprise IP-Telephony services SmartNode VoIP… More than Just Talk! BERN, Switzerland, April 18, 2019 (GLOBE NEWSWIRE) -- Patton—a U.S. network-equipment manufacturer and multi-national provider of enabling solutions for virtualized network function virtualization (NFV) SD-WAN, All-IP, and unified communications (UC) services—has saved the day for Dutch and Maltese national IP-communications providers with its Patton Cloud network edge orchestration service. VIDEO: Cloud Powered Services Netherlands ISDN Services are going away in the Netherlands. Yet scores of business subscribers can't simply jump into an All-IP network overnight. Investments in ISDN-based systems, fused with established business-processes, preclude such reckless dismantling of on-premise legacy systems. Business customers need flexibility to manage migration to

Acreage Holdings Announces Signing of Arrangement Agreement18.4.2019 17:02:00 CESTPressemelding

FRANKFURT, Germany, April 18, 2019 (GLOBE NEWSWIRE) -- Acreage Holdings, Inc. (“Acreage”) (CSE:ACRG.U) (OTC:ACRGF) (FSE:0VZ) and Canopy Growth Corporation (“Canopy Growth”) (TSX:WEED) (NYSE:CGC) (together, the “Companies”) entered into a definitive arrangement agreement (the "Agreement") that grants Canopy Growth the right to acquire 100 percent of the shares of Acreage (the “Right”), with a requirement to do so at such time as cannabis production and sale becomes federally legal in the ‎United States of America (the "Transaction"), subject to obtaining the requisite prior approval of the shareholders of each of Acreage and Canopy Growth, respectively (the “Shareholder Approval”), as well as the approval of the Supreme Court of British Columbia, Canada (the “Court Approval”). ‎ Following the Shareholder Approval and the Court Approval, under the terms of the Agreement, and upon exercise of the Right, the total consideration payable pursuant to the ‎Transaction is valued at approximatel

WillScot to Participate at the Credit Suisse Extreme Services Conference18.4.2019 16:19:00 CESTPressemelding

BALTIMORE, April 18, 2019 (GLOBE NEWSWIRE) -- WillScot Corporation (“WillScot”) (NASDAQ: WSC), the specialty rental services market leader providing innovative modular space and portable storage solutions across North America, today announced that Brad Soultz, President and Chief Executive Officer, and Tim Boswell, Chief Financial Officer, will host a day of one-on-one and group meetings with investors at the Credit Suisse Extreme Services Conference to be held at Credit Suisse’s New York City office, on Tuesday, May 7, 2019. About WillScot Corporation Headquartered in Baltimore, Maryland, WillScot is the public holding company for the Williams Scotsman family of companies. WillScot trades on the Nasdaq stock exchange under the ticker symbol "WSC," and is the specialty rental services market leader providing innovative modular space and portable storage solutions across North America. WillScot is the modular space supplier of choice for the construction, education, health care, governm

TrueCommerce Datalliance Releases First Platform Update of 201918.4.2019 16:00:00 CESTPressemelding

Supply chain software company expands self-service options, more control for customers’ data CINCINNATI, April 18, 2019 (GLOBE NEWSWIRE) -- TrueCommerce, a global provider of trading partner connectivity, integration and unified commerce solutions, has announced today that TrueCommerce Datalliance, its division focusing on vendor managed inventory (VMI) and collaborative replenishment (CR) software, has released its first of three software updates for 2019. In addition to general reliability and stability fixes, the update provides customers with more control over updating specific fields and who has access to that information, as well as adding store-driven demand. Doug Bethea, Vice President of Consumer Goods Solutions said, “Every update is multifaceted. We implement necessary security changes and adjust code to optimize performance for our customers. We also bring our own ideas as value-adds. Additionally, we are always working with customers to implement advanced features as it is

Constellation Brands Enters Into Agreement with Canopy Growth Corporation to Modify Warrants and Other Rights18.4.2019 15:33:00 CESTPressemelding

VICTOR, N.Y., April 18, 2019 (GLOBE NEWSWIRE) -- Constellation Brands, Inc. (NYSE: STZ and STZ.B), a leading beverage alcohol company, today announced that it plans to enter into an agreement with Canopy Growth Corporation (“Canopy”) (TSX: WEED, NYSE: CGC), a leading diversified cannabis company, to modify certain warrants and other rights. These changes are the result of Canopy’s intentions to acquire Acreage Holdings, Inc. (“Acreage”) upon U.S. Federal cannabis legalization. Earlier today, Canopy announced (see Canopy press release “Canopy Growth Announces Option to Acquire Leading U.S. Multi-state Cannabis Operator, Acreage Holdings”) it has entered into an agreement with Acreage, a U.S. multi-state cannabis operator, where Canopy plans to acquire the shares of Acreage upon U.S. Federal cannabis legalization (the “Triggering Event”), subject to certain conditions. This transaction, as well as proposed modifications to certain Constellation warrants, are subject to approval by Canopy

Resverlogix’ BETonMACE Phase 3 Trial Successfully Reaches its Targeted 250 MACE Events18.4.2019 14:00:00 CESTPressemelding

World’s First Phase 3 BET Bromodomain Epigenetics Trial Reaches Events Target CALGARY, Alberta, April 18, 2019 (GLOBE NEWSWIRE) -- Resverlogix Corp. (“Resverlogix” or the "Company") (TSX:RVX) today announces that BETonMACE, the Company’s event-based, phase 3 registration trial has successfully reached 250 projected major adverse cardiac events (MACE), strictly defined as cardiovascular death, non-fatal myocardial infarction and stroke, moving the trial towards completion. Successful data from this trial would enable Resverlogix to proceed towards the regulatory approval and commercialization of its lead drug, apabetalone. “Resverlogix staff and stakeholders have worked diligently for the past 18 years to attain this extremely exciting goal,” said Donald McCaffrey, President and CEO of Resverlogix. “The trial’s extensive breakthrough clinical data will soon be available to Resverlogix. Successfully meeting the trial’s endpoints, the substantial data set and otherwise new findings would