Alexion Pharmaceuticals and Moderna Therapeutics Announce Exclusive Strategic Agreement to Develop Messenger RNA Therapeutics™

  Alexion Pharmaceuticals and Moderna Therapeutics Announce Exclusive
  Strategic Agreement to Develop Messenger RNA Therapeutics™ for Rare Diseases

Business Wire

CHESHIRE, Conn. & CAMBRIDGE, Mass. -- January 13, 2014

Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) and Moderna Therapeutics today
announced an exclusive strategic agreement for the discovery and development
of messenger RNA Therapeutics™ to treat rare diseases. Alexion is a global
leader in the development and commercialization of breakthrough therapies for
patients with severe and life-threatening rare diseases. Moderna is a pioneer
in developing messenger RNA (mRNA) Therapeutics™, a highly innovative
treatment modality to enable the in vivo production of therapeutic proteins.

Messenger RNA Therapeutics™ are designed to directly utilize the body’s
natural processes to enable the in vivo production of both intracellular
proteins, which remain within the cells, and secreted proteins, which are
released into the bloodstream and act to restore function elsewhere in the
body. As a result, the mRNA Therapeutics™ platform has the potential to speed
the development and manufacture of treatments for many rare diseases that are
currently untreatable with existing technologies.

Under the agreement, Alexion will make an upfront payment to Moderna of $100
million to purchase 10 product options to develop and commercialize treatments
for rare diseases with Moderna’s mRNA Therapeutics™ platform. Alexion will
lead the discovery, development and commercialization of the treatments
produced through this broad, long-term strategic agreement, while Moderna will
retain responsibility for the design and manufacture of the messenger RNA
against selected targets. Following option exercise by Alexion, Moderna will
be entitled to drug development and commercial milestone payments, as well as
high single to double digit royalties on commercial sales. In addition,
Alexion has made a $25 million preferred equity investment into Moderna today.

“This broad and long-term strategic agreement represents a significant
expansion of Alexion’s capabilities for drug discovery, and provides us with
the strong potential to further our mission of developing novel and
breakthrough therapies for patients with severe and life-threatening rare
diseases,” said Leonard Bell, M.D., Chief Executive Officer of Alexion
Pharmaceuticals. “We consider Moderna’s mRNA Therapeutics to be an especially
exciting drug discovery platform, and we look forward to combining Moderna’s
deep expertise in mRNA with Alexion’s significant global resources and
expertise in drug discovery, clinical studies, regulatory affairs and
commercialization for the benefit of patients worldwide.”

“Moderna’s strategy is to aggressively seek to commercialize its mRNA platform
for patients in need,” said Stéphane Bancel, President and founding CEO of
Moderna. “Given the broad potential of this new drug modality, we wanted to
forge a few long-term strategic relationships with best-in-class companies. As
we were looking for a global leader with a proven track record in the
discovery, development and global commercialization of treatments for patients
with rare diseases, entering into a strategic agreement with Alexion became an
obvious choice for us. We were very impressed with the Alexion team’s passion
to treat patients with severe and life-threatening diseases, its biology
expertise, as well as its clinical and commercial capabilities. We are very
pleased and honored to work with such a dedicated company to bring new
medicines to patients.”

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and rare disorders through the innovation,
development and commercialization of life-transforming therapeutic products.
Alexion is the global leader in complement inhibition and has developed and
markets Soliris^® (eculizumab) as a treatment for patients with PNH and aHUS,
two debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is currently approved in nearly 50
countries for the treatment of PNH, and in the United States, European Union,
Japan and other countries for the treatment of aHUS. Alexion is evaluating
other potential indications for Soliris in additional severe and ultra-rare
disorders beyond PNH and aHUS, and is developing other highly innovative
biotechnology product candidates across multiple therapeutic areas. This press
release and further information about Alexion Pharmaceuticals, Inc. can be
found at: www.alexionpharma.com.

About Moderna Therapeutics

Moderna is pioneering messenger RNA Therapeutics™, an entirely new in vivo
drug modality that produces human proteins or antibodies inside patient cells,
which are in turn secreted or active intracellularly. This breakthrough
platform addresses currently undruggable targets and offers a superior
alternative to existing drug modalities for a wide range of disease
conditions. Moderna has developed a broad intellectual property estate,
including more than 250 patent applications covering novel nucleotide
chemistries and drug compositions. The company plans to develop and
commercialize its innovative mRNA drugs through a combination of strategic
relationships as well as new formed ventures. Founded in late 2010 by Flagship
VentureLabs, Cambridge-based Moderna is privately held and currently has
strategic agreements with AstraZeneca and Alexion Pharmaceuticals.
www.modernatx.com

[ALXN-G]

Safe Harbor Statement

This news release contains forward-looking statements, including statements
related to anticipated target selection, drug discovery, clinical development,
regulatory and commercial milestones resulting from the agreement between
Alexion and Moderna. Forward-looking statements are subject to factors that
may cause Alexion's results and plans to differ from those expected, including
for example, outcomes of pre-clinical and clinical studies, success in
manufacturing target products, decisions of regulatory authorities regarding
marketing approvals, and a variety of other risks set forth from time to time
in Alexion's filings with the 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 September 30, 2013, and in Alexion's other filings
with the 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.

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Contact:

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
or
Media:
Alexion Pharmaceuticals, Inc.
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
or
Investors:
Rx Communications
Rhonda Chiger, 917-322-2569
or
Moderna Therapeutics
Maria Favorito, 617-761-6720
Maria.Favorito@fkhealth.com
 
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