- 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
- Collaboration further strengthens Dicerna’s position as a leading developer of RNAi-based therapeutics -
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“With Soliris, Alexion has demonstrated the transformative impact of
complement inhibition on multiple serious and debilitating diseases,”
said
“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
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
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
[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
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Source:
Alexion
Media
Megan Goulart, 857-338-8634
Senior
Director, Corporate Communications
or
Investors
Susan
Altschuller, Ph.D., 857-338-8788
Vice President, Investor Relations
or
Dicerna
Pharmaceuticals
Investors
Rx Communications Group
Paula
Schwartz, 917-322-2216
pschwartz@rxir.com
or
Media
SmithSolve
Alex
Van Rees, 973-442-1555 ext. 111
alex.vanrees@smithsolve.com