Collaboration seeks to develop novel treatments
for chronic liver diseases, including nonalcoholic steatohepatitis
(NASH)
Boehringer Ingelheim and Dicerna Pharmaceuticals (NASDAQ:DRNA),
a leading developer of investigational RNA interference (RNAi)
therapeutics, today announced that Boehringer Ingelheim has
exercised an option to receive exclusive rights to a second hepatic
disease target emerging from its research collaboration and license
agreement with Dicerna. The collaboration, established in October
2017, aims to discover and develop novel GalXC™ RNAi therapeutics
for the treatment of chronic liver diseases, with an initial focus
on nonalcoholic steatohepatitis (NASH), a devastating disease for
which there is no approved treatment.
The option is the second target under the two companies’
research collaboration and license agreement. Under the terms of
the agreement, Boehringer Ingelheim will be responsible for future
clinical development and commercialization of the therapeutic
target. Dicerna is eligible to receive development and commercial
milestone payments, and royalties on worldwide net sales.
“It is extremely gratifying for Boehringer Ingelheim to exercise
its option for a second therapeutic candidate from our productive
research collaboration,” said Douglas M. Fambrough, President and
Chief Executive Officer of Dicerna. “The option exercise
underscores the potential of Dicerna’s GalXC technology platform in
facilitating the discovery and development of novel RNAi therapies
for nonalcoholic steatohepatitis and other chronic liver diseases.
We look forward to continued progress and the potential advancement
of clinical candidates from our partnership.”
Dicerna and Boehringer Ingelheim selected the target based on
its ability to be drugged using Dicerna’s proprietary GalXC
technology platform. The GalXC platform uses RNAi to inhibit the
expression of disease-causing genes by destroying the messenger
RNAs (mRNAs) of those genes. The approach has the potential to
treat diseases by silencing previously inaccessible drug
targets.
“Boehringer Ingelheim is pleased with the progress of our
initial collaboration with Dicerna and based on early findings and
our strong relationship, we are expanding our research efforts,”
said Clive R. Wood, Ph.D., Corporate Senior Vice President
Discovery Research at Boehringer Ingelheim. “Today’s announcement
exemplifies our commitment to discovering new therapies for NASH
and other chronic liver diseases and advances our research efforts
in this priority area.”
NASH is caused by the buildup of fat in the liver, potentially
leading to liver fibrosis and cirrhosis. It has an especially high
prevalence among obese and diabetic patients and is an area of high
unmet medical need. NASH is expected to soon become the most common
cause of advanced liver disorders, and it often necessitates liver
transplantation.
Dicerna is building a portfolio of research and development
programs to advance the treatment of diseases involving the liver,
including chronic liver diseases, viral infectious diseases,
cardiovascular diseases and rare diseases. The company aims to
leverage its proprietary GalXC technology to develop innovative
RNAi therapeutics to positively transform the lives of individuals
living with these diseases.
About Dicerna's GalXC™ RNAi Technology Platform
Dicerna’s proprietary 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.
GalXC-based therapies are processed by the Dicer enzyme, which is
the natural initiation point for RNAi within the human cell. Using
GalXC, Dicerna attaches 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, chronic liver diseases,
cardiovascular diseases, and viral infectious diseases.
About Dicerna Pharmaceuticals, Inc.
Dicerna Pharmaceuticals, Inc., 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, chronic
liver diseases, cardiovascular diseases, and viral infectious
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. Dicerna has strategic collaborations with
Boehringer Ingelheim, Eli Lilly and Company, and Alexion
Pharmaceuticals. For more information, please visit
www.dicerna.com.
About Boehringer Ingelheim
Innovative medicines for people and animals have for more than
130 years been what the research-driven pharmaceutical company
Boehringer Ingelheim stands for. Boehringer Ingelheim is one of the
pharmaceutical industry’s top 20 companies and to this day remains
family-owned. Day by day, some 50,000 employees create value
through innovation for the three business areas, human
pharmaceuticals, animal health and biopharmaceutical contract
manufacturing. In 2016, Boehringer Ingelheim achieved net sales of
around 15.9 billion euros. With more than three billion euros,
R&D expenditure corresponds to 19.6 per cent of net sales.
Social responsibility comes naturally to Boehringer Ingelheim.
That is why the company is involved in social projects such as the
“Making More Health” initiative. Boehringer Ingelheim also actively
promotes workforce diversity and benefits from its employees’
different experiences and skills. Furthermore, the focus is on
environmental protection and sustainability in everything the
company does. More information about Boehringer Ingelheim can be
found on www.boehringer-ingelheim.com or in our annual report:
http://annualreport.boehringer-ingelheim.com.
Cautionary Note on 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
Dicerna makes regarding: (i) the therapeutic and commercial
potential of GalXC™; (ii) research and development plans related to
GalXC; and (iii) the potential of our technology and drug
candidates in our research and development pipeline. 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 pre-clinical research
collaboration. Applicable risks and uncertainties include those
relating to Dicerna's preclinical research and other risks
identified under the heading "Risk Factors" included in Dicerna's
most recent Form 10-Q filing and in other future filings with the
SEC. The forward-looking statements contained in this press release
reflect Dicerna's current views with respect to future events, and
Dicerna does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
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version on businesswire.com: https://www.businesswire.com/news/home/20190104005105/en/
Contacts for Boehringer Ingelheim:Dr. Reinhard MalinHead
of Communications Innovation UnitBoehringer Ingelheim Corporate
Center GmbH Media + PRreinhard.malin@boehringer-ingelheim.comP: +49
6132 77-90815: M: + 49 151 150 20
690www.boehringer-ingelheim.com
Linda RuckelAssociate Director, Media and Corporate
ReputationBoehringer Ingelheim Pharmaceuticals, Inc.Ridgefield,
Connecticutlinda.ruckel@boehringer-ingelheim.comP: 203-791-6672 ::
C: 917-692-0848www.boehringer-ingelheim.com
Contacts for Dicerna Pharmaceuticals:Paula
SchwartzManaging DirectorRx Communications
Grouppschwartz@rxir.comP: 917-322-2216www.rxir.com
Alex Van ReesSenior Account DirectorSmithSolve
Communicationsalex.vanrees@smithsolve.comP: 973-442-1555 ext.
111www.smithsolve.com
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