– Option Exercise Triggers $20 Million Payment
to Dicerna –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Dicerna™
Pharmaceuticals, Inc. (NASDAQ:DRNA) today announced that Alexion
has exercised its option for exclusive rights to two additional
targets within the complement pathway for the discovery and
development of GalXC™ RNAi molecules. This expands the companies’
existing research collaboration and license agreement to now
encompass four targets within the complement pathway. In connection
with the option exercise, Alexion will pay Dicerna a total of $20
million, or $10 million in option exercise fees per additional new
target.
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“Our collaboration with Dicerna to discover and develop GalXC
RNAi therapies, which have the potential to inhibit uncontrolled
activation of the complement system in new ways, represents an
exciting opportunity for Alexion to expand its leadership in
developing new medicines for complement-mediated diseases,” said
John Orloff, M.D., Executive Vice President and Head of Research
& Development at Alexion. “We are encouraged by the
collaboration’s progress and the strong relationship our teams have
developed over the past year, and we are pleased to expand our
efforts to include additional targets in the complement
pathway.”
“With more than 20 years of experience, Alexion is a clear
leader in the field of complement biology, and we are very pleased
by our collaboration’s success to date in identifying potential new
GalXC RNAi-based therapeutic approaches to treating
complement-related diseases. Alexion’s decision to exercise its
option, along with our other new collaborations in different
therapeutic areas, underscores the growing recognition of the
significant potential for RNAi therapies as a new modality in the
treatment of a broad range of diseases,” said Douglas M. Fambrough,
Ph.D., President and Chief Executive Officer of Dicerna. “We are
very proud to be at the forefront of RNAi innovation and look
forward to continued advancement of our core development portfolio
and progress across our collaborative programs in 2020.”
The collaboration between Alexion and Dicerna, which was
initiated in October 2018, is focused on the discovery and
development of subcutaneously delivered GalXC RNAi molecules
directed to a total of four complement pathway targets for the
treatment of complement-mediated diseases. Dicerna is leading the
joint discovery and research efforts through the preclinical stage,
and Alexion will lead development efforts beginning with Phase 1
studies. The agreement provides Alexion with exclusive worldwide
licenses and commercial rights to the GalXC RNAi molecules
developed in the collaboration in exchange for development- and
approval-related milestones, sales milestones and mid-single to
low-double-digit royalties on future product sales.
About the Complement System
The complement system is a part of the immune system that
enhances, or complements, the ability of antibodies to clear small
organisms or damaged cells from an organism, promote inflammation
and attack the pathogen’s cell membrane. While the complement
system is essential to maintaining health, it can also become
over-activated, contributing to the development of numerous
devastating diseases.
About Dicerna's GalXC™ RNAi Technology Platform
Dicerna’s proprietary RNA interference (RNAi) technology
platform, called GalXC™, aims to advance the development of
next-generation RNAi-based therapies designed to silence
disease-driving genes in the liver and other tissues.
Liver-targeted GalXC-based compounds enable subcutaneous delivery
of RNAi therapies that are designed to bind specifically to
receptors on liver cells, leading to internalization and access to
the RNAi machinery within the cells. The GalXC approach seeks to
optimize the activity of the RNAi pathway so that it operates in
the most specific and potent fashion. Compounds produced via GalXC
are intended to be broadly applicable across multiple therapeutic
areas, including both liver and non-liver indications.
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases through the
discovery, development and commercialization of life-changing
therapies. As the global leader in complement biology and
inhibition for more than 20 years, Alexion has developed and
commercializes two approved complement inhibitors to treat patients
with paroxysmal nocturnal hemoglobinuria (PNH) and atypical
hemolytic uremic syndrome (aHUS), as well as the first and only
approved complement inhibitor to treat anti-acetylcholine receptor
(AChR) antibody-positive generalized myasthenia gravis (gMG) and
neuromyelitis optica spectrum disorder (NMOSD). Alexion also 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). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. 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, metabolic disorders and cardiology. Headquartered in
Boston, Massachusetts, Alexion has offices around the globe and
serves patients in more than 50 countries. This press release and
further information about Alexion can be found at:
www.alexion.com.
[ALXN-G]
About DicernaTM Pharmaceuticals, Inc.
Dicerna™ Pharmaceuticals, Inc., is a biopharmaceutical company
using ribonucleic acid interference (RNAi) to develop medicines
that silence genes that cause disease. The company is applying its
proprietary GalXC™ technology to develop potent, selective and safe
RNAi therapies for treatment of rare diseases, chronic liver
diseases, cardiovascular diseases, neurodegenerative diseases, pain
and viral infectious disease. Dicerna aims to treat disease by
addressing the underlying causes of illness with capabilities that
extend beyond the liver to address a broad range of diseases,
focusing on target genes where connections between gene and disease
are well understood and documented. Dicerna intends to discover,
develop and commercialize novel therapies either on its own or in
collaboration with pharmaceutical partners. Dicerna has strategic
collaborations with Novo Nordisk A/S*, Roche*, Eli Lilly and
Company, Alexion Pharmaceuticals, Inc. and Boehringer Ingelheim
International GmbH. For more information, please visit
www.dicerna.com.
[DRNA]
Forward-Looking Statements
This press release includes forward-looking statements. Such
forward-looking statements are subject to risks and uncertainties
that could cause actual results to differ materially from those
expressed or implied in such statements. Examples of
forward-looking statements include, among others, statements we
make regarding: (i) the receipt of option exercise fees; (ii) the
therapeutic and commercial potential of GalXC™, including its
potential to inhibit uncontrolled activation of the complement
system in new ways; (iii) research and development plans of Alexion
related to GalXC; (iv) the potential of RNAi therapies for the
treatment of complement-mediated diseases and a broad range of
diseases; & (v) the potential for the collaboration between
Alexion and Dicerna and this collaboration representing an exciting
opportunity for Alexion to expand its leadership in developing new
medicines for complement-mediated diseases. The process by which an
early-stage platform such as GalXC could potentially lead to an
approved product is long and subject to highly significant risks,
particularly with respect to a preclinical research collaboration
and the anticipated benefits of the platform may not be realized.
In addition, the four targets of the collaboration may not result
in any product and this may be after significant investments by the
parties to the collaboration. Applicable risks and uncertainties
include those relating to preclinical research and other risks
identified under the heading "Risk Factors" included in Alexion’s
and Dicerna’s most recent Form 10-Q filings and in other future
filings with the SEC. The forward-looking statements contained in
this press release reflect Alexion’s and Dicerna's current views
with respect to future events, and neither Alexion nor Dicerna
undertakes and specifically disclaims any obligation to update any
forward-looking statements, except as required by law.
* Novo Nordisk A/S and Roche transactions are subject to
clearance under the Hart-Scott-Rodino Antitrust Improvements Act of
1976 and other customary closing conditions.
Dicerna™ and GalXC™ are trademarks of Dicerna Pharmaceuticals,
Inc.
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version on businesswire.com: https://www.businesswire.com/news/home/20191216005150/en/
Alexion Media Megan Goulart, 857-338-8634 Senior
Director, Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice President,
Investor Relations
Dicerna Media Dicerna Pharmaceuticals, Inc. Amy Trevvett,
617-612-6253 atrevvett@dicerna.com or SmithSolve Alex Van Rees,
973-442-1555 ext. 111 alex.vanrees@smithsolve.com
Investors Stern Investor Relations, Inc. Lauren Stival,
212-362-1200 Lauren.stival@sternir.com
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