Alexion Receives Three New U.S. Patents for Soliris ® (eculizumab), Extending
Patent Protection Into 2027
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the United States Patent and Trademark Office (USPTO) has
issued U.S. Patents No. 9,732,149; 9,718,880; and 9,725,504, which are directed to the composition of matter of eculizumab
(Soliris®), pharmaceutical formulations of eculizumab, and methods of treating paroxysmal nocturnal hemoglobinuria (PNH)
with eculizumab, respectively, and which will expire in 2027.
“Alexion is the leader in complement biology and has invested decades of ground-breaking research and development in Soliris. We
continue to strengthen our patent portfolio around this unique complement inhibitor,” said Ludwig Hantson, Chief Executive Officer
of Alexion. “These new patents work in concert with other patents and regulatory exclusivities to protect Soliris in all
indications.”
The new composition of matter patent is directed to the full-length amino acid sequence of eculizumab and covers molecules that
contain the same sequence. The new formulation patent is directed to pharmaceutical compositions that contain eculizumab,
independent of their intended use. The new method of use patent is directed to treating PNH with eculizumab, and supplements other
patents that are directed to treating atypical hemolytic uremic syndrome (aHUS) and other complement-mediated diseases with
eculizumab.
Alexion is pursuing corresponding patent applications in other regions and countries, including Europe and Japan. In addition,
Alexion is pursuing patent applications for pending additional indications of Soliris, such as for the treatment of refractory
generalized myasthenia gravis (gMG).
About Soliris ® (eculizumab)
Soliris® is a first-in-class complement inhibitor that works by inhibiting the terminal part of the complement
cascade, a part of the immune system that, when activated in an uncontrolled manner, plays a role in serious ultra-rare disorders
like paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
Soliris is approved in the U.S., EU, Japan and other countries as the first and only treatment for patients with PNH and aHUS.
In addition, Alexion has submitted applications in the U.S., EU and Japan for Soliris as a potential treatment of refractory gMG in
patients who are anti-acetylcholine receptor (AChR) antibody-positive. In June 2017, the Committee for Medicinal Products for Human
Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for this new indication. Soliris is not indicated for
the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). Alexion and Soliris have received
some of the pharmaceutical industry's highest honors for the medical innovation in complement inhibition: the Prix Galien USA
(2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
For more information on Soliris, please see full prescribing information for Soliris, including BOXED WARNING regarding risk of
serious meningococcal infection, available at www.soliris.net.
Important Soliris Safety Information
The U.S. prescribing information for Soliris includes the following warnings and precautions: Life-threatening and fatal
meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly
life-threatening or fatal if not recognized and treated early. Comply with the most current Centers for Disease Control (CDC)’s
Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement
deficiencies. Immunize patients with meningococcal vaccines at least two weeks prior to administering the first dose of Soliris,
unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early
signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the
program. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS
(1-888-765-4747) or at www.solirisrems.com .
Patients may have increased susceptibility to infections, especially with encapsulated bacteria. Aspergillus infections have
occurred in immunocompromised and neutropenic patients. Children treated with Soliris may be at increased risk of developing
serious infections due to Streptococcus pneumoniae and Haemophilus influenza type b (Hib). Soliris treatment of
patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during
Soliris treatment has not been established. Administration of Soliris may result in infusion reactions, including anaphylaxis or
other hypersensitivity reactions.
In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were
headache, nasopharyngitis, back pain and nausea. In patients with aHUS, the most frequently reported adverse events observed with
Soliris treatment in clinical studies were headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting,
nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia.
About Alexion
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with
devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and commercializes the
first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical
hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion has two highly innovative enzyme
replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal
acid lipase deficiency (LAL-D). As the leader in complement biology for over 20 years, Alexion focuses its research efforts on
novel molecules and targets in the complement cascade, and its development efforts on the core therapeutic areas of hematology,
nephrology, neurology, and metabolic disorders. This press release and further information about Alexion can be found at: www.alexion.com.
[ALXN-G]
Forward-Looking Statement
This news release contains forward-looking statements, including statements related to Soliris intellectual property, and the
strength and scope of Soliris intellectual property protection. Forward-looking statements are subject to factors that may cause
Alexion's results and plans to differ from those expected, including for example, the risks and uncertainties of drug development,
decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the
marketing of eculizumab, delays, interruptions or failures in the manufacture and supply of our products and our product
candidates, failure to satisfactorily address matters raised by the FDA and other regulatory agencies, the possibility that results
of clinical trials are not predictive of safety and efficacy results of our products in broader patient populations, the
possibility that clinical trials of our product candidates could be delayed, the adequacy of our pharmacovigilance and drug safety
reporting processes, the risk that third party payers (including governmental agencies) will not reimburse or continue to reimburse
for the use of our products at acceptable rates or at all, the outcome of challenges and opposition proceedings to our intellectual
property, assertion or potential assertion by third parties that the manufacture, use or sale of our products infringes their
intellectual property, risks regarding government investigations, including investigations of Alexion by the SEC and DOJ, the risk
that anticipated regulatory filings are delayed, the risk that estimates regarding the number of patients with the diseases our
products treat are inaccurate, and a variety of other risks set forth from time to time in Alexion's filings with the U.S.
Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for
the period ended June 30, 2017 and in our other filings with the U.S. Securities and Exchange Commission. Alexion does not intend
to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty
arises under law.
Alexion Pharmaceuticals, Inc.
Media
Arne Naeveke, PhD, 475-230-3774
Executive Director, Product Communications
or
Investors
Elena Ridloff, CFA, 475-230-3601
Vice President, Investor Relations
or
Catherine Hu, 475-230-3599
Director, Investor Relations
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