Alexion and Dicerna Announce Collaboration to Discover and Develop RNAi Therapies for Complement-Mediated Diseases
Business Wire 24-Oct-2018 6:30 AM
- Collaboration provides opportunity to build on Alexion's more than
two decades of complement leadership with expansion into RNAi-based
therapies using Dicerna's GalXC™ technology -
- Agreement provides Alexion with exclusive worldwide licenses as
well as development and commercial rights for two preclinical GalXC RNAi
molecules, with option for two additional candidates -
- Dicerna to receive upfront payment of $22 million and equity
investment of $15 million, with potential for additional
milestone-dependent and royalty payments -
- Collaboration further strengthens Dicerna's position as a leading
developer of RNAi-based therapeutics -
Alexion
Pharmaceuticals, Inc. (NASDAQ:ALXN) and Dicerna
Pharmaceuticals, Inc. (NASDAQ:DRNA) today announced a collaboration
to discover and develop RNA interference (RNAi) therapies for
complement-mediated diseases. An RNAi-based approach to blocking the
production of complement pathway factors offers the potential to inhibit
the uncontrolled complement activation that leads to many diseases. The
agreement provides Alexion with exclusive worldwide licenses as well as
development and commercial rights for two of Dicerna's preclinical,
subcutaneously delivered GalXC™ RNAi molecules and an exclusive option
for other preclinical GalXC RNAi molecules for two additional targets
within the complement pathway.
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"With Soliris, Alexion has demonstrated the transformative impact of
complement inhibition on multiple serious and debilitating diseases,"
said John Orloff, M.D., Executive Vice President and Head of Research &
Development at Alexion. "This collaboration provides the opportunity to
continue building on our more than two decades of complement expertise
using Dicerna's proprietary GalXC RNAi technology platform, which
provides a potentially promising new way of inhibiting the uncontrolled
complement activation that we know plays a significant role in many
devastating diseases."
"Dicerna's proprietary GalXC technology is designed to silence the
expression of disease-driving genes in a way that is highly specific,
generally well tolerated, and allows for convenient, infrequent
subcutaneous administration. Having recently demonstrated clinical
proof-of-concept for DCR-PHXC, our lead program for the treatment of
primary hyperoxaluria, we are eager to expand and advance our pipeline
of innovative GalXC therapies, including both proprietary and partnered
programs," said Douglas M. Fambrough, Ph.D., President and Chief
Executive Officer of Dicerna. "Our collaboration with Alexion provides
access to the deep expertise and resources of an established leader in
complement-mediated diseases. We look forward to working with Alexion to
discover and develop promising new RNAi therapies."
Under the terms of the agreement, Alexion and Dicerna will collaborate
on the discovery and development of subcutaneously delivered GalXC RNAi
molecules directed to two complement pathway targets for the treatment
of complement-mediated diseases. In addition, Alexion will have the
right to exercise options, for additional payment, for two additional
GalXC RNAi molecules directed to complement pathway targets. Dicerna
will lead 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. Dicerna will receive an immediate
upfront payment of $22 million, with Alexion making a concurrent $15
million equity investment in Dicerna at a premium to market as of the
collaboration effective date. The collaboration also provides for
potential additional development and approval-related milestone payments
of up to $105 million per target, plus sales milestones and mid-single
to low-double digit royalties on future product sales.
About RNAi
RNA interference (RNAi) is a biologic process in which certain
double-stranded RNA molecules inhibit the expression of disease-causing
genes by destroying the messenger RNAs (mRNAs) of those genes. It
reflects a new approach in the development of specific and powerful
therapies. Rather than targeting and binding to proteins to inhibit
their activity, RNAi exerts its effects one step earlier in the gene
silencing process by targeting the mRNA, the instruction set that
directs the building of the protein. By attaching to this instruction
set, RNAi is believed to have the ability to attack any target,
including disease-causing genes that are beyond the reach of
conventional antibody and small-molecule modalities. Additionally,
RNAi-based therapeutic approaches hold the potential to offer more
convenience for patients via infrequent subcutaneous dosing and a long
duration of effect.
About Dicerna's GalXC™ RNAi Technology Platform
The proprietary RNAi technology platform called GalXC™, invented by
Dicerna, aims to advance the development of next-generation RNAi-based
therapies designed to silence disease-driving genes in the liver.
GalXC-based therapies are processed by the Dicer enzyme, which is the
natural initiation point for RNAi within the human cell. Using GalXC,
Dicerna scientists attach N-acetylgalactosamine sugars directly to the
extended region of the proprietary Dicer substrate short-interfering RNA
(DsiRNA) molecules, yielding multiple conjugate delivery configurations
that allow flexible and efficient conjugation to the targeting ligands
while stabilizing the RNAi duplex. Dicerna believes this stabilization
will enable subcutaneous delivery of RNAi therapies to hepatocytes in
the liver, where they are designed to specifically bind to receptors on
target cells, potentially leading to internalization and access to the
RNAi machinery within the cells. By using the Dicer enzyme as the entry
point into RNAi, 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 rare diseases,
viral infectious diseases, chronic liver diseases and cardiovascular
diseases.
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 the first and only
approved complement inhibitor to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome
(aHUS), and anti-acetylcholine receptor (AchR) antibody-positive
generalized myasthenia gravis (gMG). 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 two late-stage therapies, including a second
complement inhibitor and a copper-binding agent for Wilson disease.
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. Alexion has been named to the Forbes list of the
World's Most Innovative Companies seven years in a row and is
headquartered in Boston, Massachusetts' Innovation District. The company
also 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 Dicerna
Dicerna is a biopharmaceutical company focused on the discovery and
development of innovative, subcutaneously delivered RNAi-based
therapeutics for the treatment of diseases involving the liver,
including rare diseases, viral infectious diseases, chronic liver
diseases, and cardiovascular diseases. Dicerna is leveraging its
proprietary GalXC™ RNAi technology platform to build a broad pipeline in
these core therapeutic areas, focusing on target genes where connections
between target gene and diseases are well understood and documented.
Dicerna intends to discover, develop and commercialize novel
therapeutics either on its own or in collaboration with pharmaceutical
partners. For more information, please visit www.dicerna.com.
[DRNA]
Forward-Looking Statement
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 therapeutic
and commercial potential of GalXC™; (ii) research and development plans
related to GalXC; (iii) the potential of RNAi therapies for the
treatment of complement-mediated diseases; and (iv) the potential for
the collaboration between Alexion and Dicerna. 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. 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 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.
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