CAMBRIDGE, Mass. and CARLSBAD, Calif.,
Inotersen is under regulatory review in the U.S. and EU with approvals planned for mid-2018 for the treatment of hereditary transthyretin amyloidosis, or hATTR. hATTR is a systemic, progressive and fatal disease. Akcea and Ionis are also developing AKCEA-TTR-LRx for hereditary and wild-type forms of ATTR and plan to commence clinical studies for AKCEA-TTR-LR in 2018.
In addition, following the close of the transaction, today, Sarah Boyce joined Akcea as president and a member of the Akcea board of directors reporting to Paula Soteropoulos, Akcea’s chief executive officer. Ms. Boyce was formerly the chief business officer at Ionis.
The transaction was subject to certain closing conditions, including a non-waivable condition that the stock purchase agreement, the license agreement and related agreements and the transaction be approved by the affirmative vote of holders representing a majority of the issued and outstanding shares of common stock other than Ionis and its affiliates, which excluded a vote of Akcea’s directors and officers. This affirmative vote was obtained at a special meeting of Akcea stockholders on
Under the agreement, Akcea paid Ionis an upfront licensing fee of $150 million through the issuance of 8,000,000 shares of common stock priced at
Inotersen is an antisense drug designed to reduce the production of transthyretin, or TTR protein, to treat TTR amyloidosis (ATTR), a systemic, progressive and fatal disease.
Inotersen is currently under regulatory review for marketing authorization in the U.S. and EU.
ABOUT HEREDITARY TRANSTHYRETIN AMYLOIDOSIS (hATTR)
hATTR is a progressive, systemic, and fatal genetic disease caused by the inappropriate formation and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects on multiple aspects of a patient’s life. Patients with hATTR often present with a mixed phenotype and experience overlapping symptoms of polyneuropathy and cardiomyopathy.
Ultimately, hATTR results in death within three to fifteen years of symptom onset. Therapeutic options for the treatment of patients with hATTR are limited and there are currently no disease-modifying drugs approved for hATTR. There are an estimated 50,000 patients with hATTR worldwide.
ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over 40 drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Inotersen and volanesorsen are two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. Inotersen is under regulatory review for marketing approval in the U.S. and EU for the treatment of patients with hereditary ATTR amyloidosis. Volanesorsen is under regulatory review for marketing approval in the U.S., EU and Canada for the treatment of patients with familial chylomicronemia syndrome, or FCS. Volanesorsen is also in a Phase 3 study in patients with familial partial lipodystrophy, or FPL. Akcea, an affiliate of Ionis focused on developing and commercializing drugs to treat patients with serious and rare diseases, will commercialize inotersen and volanesorsen, if approved. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
Akcea Therapeutics, an affiliate of
IONIS’ AND AKCEA’S FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the recently announced transaction between Ionis and Akcea, Ionis’ and Akcea’s business and the therapeutic and commercial potential of inotersen, AKCEA-TTR-LRx and other products in development. Any statement describing Ionis’ or Akcea’s goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of inotersen, volanesorsen or other of Ionis’ or Akcea’s 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. Ionis’ and 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 Ionis’ and 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 Ionis and Akcea. 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 annual reports on Form 10-K for the year ended
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