- Otsuka to leverage strong commercial infrastructure and rare
disease experience to reach European HAE patients
- Ionis plans to independently bring donidalorsen to U.S.
patients if approved
- Donidalorsen Phase 3 results expected in the first half of
2024
CARLSBAD, Calif., Dec. 18,
2023 /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 in Europe to
commercialize donidalorsen, an investigational prophylactic
treatment for hereditary angioedema (HAE). Ionis will maintain
responsibility for the non-clinical and clinical development of
donidalorsen, and Otsuka will be responsible for European
regulatory filings and commercialization.
Ionis plans to independently launch donidalorsen in the U.S. if
approved, as part of the company's strategy to deliver a steady
flow of wholly owned medicines to patients.
"We are excited to collaborate with Otsuka given their proven
results in bringing rare disease medicines to patients in
Europe," said Brett P. Monia, Ph.D., Ionis' chief executive
officer. "This agreement is aligned with our strategy to initially
focus our commercialization efforts on the U.S. market. We are
encouraged by the strong product profile of donidalorsen to date
and look forward to reporting pivotal topline Phase 3 donidalorsen
results in HAE in the first half of next year."
Otsuka brings expertise in delivering rare disease medicines to
patients, a robust commercial infrastructure, and deep knowledge of
regional and local regulations across European countries. As part
of the agreement, Ionis will receive a $65
million upfront payment and milestone payments based on
achievement of regulatory and sales targets. Ionis is also eligible
to earn tiered royalties ranging from 20 to 30 percent (based on
aggregate annual net sales).
Makoto Inoue, president and
representative director of Otsuka Pharmaceutical Co., Ltd.
commented, "Otsuka has developed drugs in Europe for rare diseases such as autosomal
dominant polycystic kidney disease (ADPKD). Through this
collaboration with Ionis, a leader in RNA-targeted therapy, if
regulatory approval is received, we look forward to bringing
donidalorsen to patients in Europe
to address the unmet medical needs of patients with HAE."
Ionis recently reported two-year results from the Phase 2
open-label extension (OLE) trial showing that donidalorsen
treatment resulted in a 96% overall sustained mean reduction from
baseline in HAE attack rates and was recently granted Orphan Drug
Designation in the U.S.
Injection site (IS) discoloration and IS reaction were the only
study drug-related treatment-emergent adverse events (TEAEs)
reported in more than one patient (n=2, 11.8% each). No serious
adverse events were reported in the OLE study, and no TEAEs led to
study discontinuation. The company plans to report Phase 3 results
with donidalorsen for prophylactic treatment of HAE in the first
half of 2024.
About Hereditary Angioedema
(HAE)
HAE is a rare and life-threatening genetic disease characterized
by unpredictable and frequently severe swelling of the skin,
gastrointestinal (GI) tract, upper respiratory system, face, and
throat, which can be life-threatening.1-5 HAE is
estimated to affect more than 20,000 patients in the U.S. and
Europe.6 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 the prekallikrein, or PKK, pathway. PKK
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 more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and
follow us on Twitter @ionispharma.
Ionis' Forward-looking
Statements
This press release includes forward-looking statements regarding
Ionis' business, and the therapeutic and commercial potential of
donidalorsen, Ionis' technologies, and other products in
development. 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 but not limited to those related to our
commercial products and the medicines in our pipeline, and
particularly 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. 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 Dec. 31, 2022, and most recent
Form 10-Q, which are on file with the SEC. Copies of these and
other documents are available at www.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
1. Manning ME. Dermatol Ther (Heidelb). 2021; 11:1829-1838.
2. Valerieva A, et al. Balkan Med J. 2021;8:89-103.
3. Santacroce R, et al. J Clin Med. 2021;10:2023.
4. Pines JM, et al. J Emerg Med. 2021;60:35-43.
5. Maurer M, et al. World Allergy Organ J. 2022;15:100627.
6. Weller K, et al. Allergy. 2016;71(8): 1203-1209.
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SOURCE Ionis Pharmaceuticals, Inc.