- Otsuka will be responsible for commercialization efforts for
donidalorsen across both Asia
Pacific and Europe
- Ionis plans to independently bring donidalorsen to U.S.
patients, if approved
CARLSBAD, Calif., June 18,
2024 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) today announced that it has entered into a
license agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka)
under which Otsuka obtains exclusive rights across the
Asia-Pacific region for
donidalorsen, an investigational RNA-targeted prophylactic medicine
for hereditary angioedema (HAE). Ionis will maintain primary
responsibility for the development of donidalorsen, while Otsuka
will be responsible for territory-specific development, regulatory
filings and commercialization in the Asia-Pacific region and Europe.
Ionis plans to file a New Drug Application with the U.S. Food
and Drug Administration (FDA) this year, and will independently
launch donidalorsen in the U.S., if approved. Ionis and Otsuka
previously announced a licensing agreement for donidalorsen in
Europe, and Otsuka is also
preparing to submit a Marketing Authorization Application to the
European Medicines Agency this year.
"Ionis and Otsuka share a steadfast commitment to bring
donidalorsen to as many people living with HAE as possible," said
Brett P. Monia, Ph.D., chief
executive officer of Ionis. "We look forward to working alongside
Otsuka to advance regulatory discussions across Europe and the Asia-Pacific region based on our positive
Phase 3 results for donidalorsen, which were presented late last
month. In the U.S., we've built a robust commercial infrastructure
in preparation for our near-term anticipated launches for olezarsen
in familial chylomicronemia syndrome and donidalorsen in HAE."
Under the terms of the Asia-Pacific agreement, Ionis will receive a
$20 million upfront payment and
milestone payments based on achievement of regulatory and sales
targets. Ionis is also eligible to earn tiered royalties, with
similar economic terms to the previous agreement for
Europe. Originating in Japan,
Otsuka brings deep knowledge of regional and local regulations
across the Asia-Pacific region, as
well as global expertise in delivering rare disease medicines to
patients and a robust commercial infrastructure.
Ionis recently reported positive results from the
Phase 3 OASIS-HAE and OASISplus studies, presented at the 2024
European Academy of Allergy and Clinical Immunology (EAACI) Annual
Congress in Valencia, Spain and
published in The New England Journal of Medicine.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially life-threatening genetic condition
that involves recurrent attacks of severe swelling (angioedema) in
various parts of the body, including the hands, feet, genitals,
stomach, face and/or throat. HAE is estimated to affect more than
20,000 patients in the U.S. and Europe. In the U.S., doctors frequently use
prophylactic treatment approaches to prevent and reduce the
severity of HAE attacks in patients.
About Donidalorsen
Donidalorsen is an investigational LIgand-Conjugated
Antisense (LICA) medicine designed to target prekallikrein
(PKK), which plays an important role in activating inflammatory
mediators associated with acute attacks of hereditary angioedema
(HAE). By reducing the production of PKK, donidalorsen could
be an effective prophylactic approach to preventing HAE
attacks.
About Ionis Pharmaceuticals, Inc.
For three
decades, Ionis has invented medicines that bring better futures to
people with serious diseases. Ionis currently has five marketed
medicines and a leading pipeline in neurology, cardiology, and
other areas of high patient need. As the pioneer in RNA-targeted
medicines, Ionis continues to drive innovation in RNA therapies in
addition to advancing new approaches in gene editing. A deep
understanding of disease biology and industry-leading technology
propels our work, coupled with a passion and urgency to deliver
life-changing advances for patients.
To learn more about Ionis, visit Ionis.com and follow us on
X (Twitter) and LinkedIn.
Ionis Forward-looking
Statements
This press release includes forward-looking
statements regarding Ionis' business, financial guidance and the
therapeutic and commercial potential of our commercial medicines,
donidalorsen, additional medicines in development and technologies.
Any statement describing Ionis' 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 including
those inherent in the process of discovering, developing and
commercializing medicines that are safe and effective for use as
human therapeutics, and in the endeavor of building a business
around such medicines. 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
Ionis' forward-looking statements reflect the good faith judgment
of its management, these statements are based only on facts and
factors currently known by Ionis. Except as required by law, we
undertake no obligation to update any forward-looking statements
for any reason. As a result, you are cautioned not to rely on these
forward-looking statements. These and other risks concerning Ionis'
programs are described in additional detail in Ionis' annual report
on Form 10-K for the year ended December 31,
2023, and most recent Form 10-Q, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our" and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of
Ionis Pharmaceuticals, Inc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
ir@ionis.com
760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com
760-603-4679
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SOURCE Ionis Pharmaceuticals, Inc.