Alnylam and Sanofi Conclude Research and Option Phase of 2014 RNAi Therapeutics Collaboration
Apr 08, 2019
− Alnylam to Advance Selected Investigational Asset in an Undisclosed
Rare Genetic Disease Through the End of IND-Enabling Studies –
− Material Collaboration Terms for Patisiran, Vutrisiran, and
Fitusiran, as Previously Announced, Remain Unchanged –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 8, 2019--
Alnylam
Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics
company, announced today that Alnylam and Sanofi have agreed to conclude
the research and option phase of the companies’ 2014 RNAi therapeutics
alliance in rare genetic diseases. The material collaboration terms for
patisiran, vutrisiran, and fitusiran, as previously announced, will
continue unchanged.
“Our landmark 2014 rare disease alliance with Sanofi resulted in the
advancement of three Phase 3 programs – patisiran, vutrisiran, and
fitusiran – and the global launch of ONPATTRO, the world’s first RNAi
therapeutic. We’re pleased to now conclude the research and product
option phase of the collaboration, allowing Alnylam to focus on future
continued growth of its rare disease pipeline,” said Yvonne Greenstreet,
MBChB, MBA, Chief Operating Officer of Alnylam. “We couldn’t be more
pleased with the success of this alliance over the last five years,
advancing RNAi therapeutics to patients afflicted with rare diseases
around the world, and we look forward to our continued collaboration
with Sanofi on our alliance programs.”
As part of the agreement, Alnylam will advance a selected
investigational asset in an undisclosed rare genetic disease through the
end of IND-enabling studies. Sanofi will be responsible for any
potential further development or commercialization of this asset. If
this product is approved, Alnylam will be eligible to receive tiered
double-digit royalties on its global net sales. In addition, Alnylam and
Sanofi have agreed to amend certain terms of the companies’ equity
agreement, with Sanofi obtaining a release of its lock-up of Alnylam
stock holdings, subject to certain trading restrictions, amongst other
provisions.
About RNAi
RNAi (RNA interference) is a natural cellular
process of gene silencing that represents one of the most promising and
rapidly advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough that
happens once every decade or so,” and was recognized with the award of
the 2006 Nobel Prize for Physiology or Medicine. By harnessing the
natural biological process of RNAi occurring in our cells, a new class
of medicines, known as RNAi therapeutics, is now a reality. Small
interfering RNA (siRNA), the molecules that mediate RNAi and comprise
Alnylam's RNAi therapeutic platform, function upstream of today’s
medicines by potently silencing messenger RNA (mRNA) – the genetic
precursors – that encode for disease-causing proteins, thus preventing
them from being made. This is a revolutionary approach with the
potential to transform the care of patients with genetic and other
diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq:ALNY) is
leading the translation of RNA interference (RNAi) into a whole new
class of innovative medicines with the potential to transform the lives
of people afflicted with rare genetic, cardio-metabolic, hepatic
infectious, and central nervous system (CNS)/ocular diseases. Based on
Nobel Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
severe and debilitating diseases. Founded in 2002, Alnylam is delivering
on a bold vision to turn scientific possibility into reality, with a
robust discovery platform. Alnylam’s first U.S. FDA-approved RNAi
therapeutic is ONPATTRO® (patisiran) lipid complex injection
available in the U.S. for the treatment of the polyneuropathy of
hereditary transthyretin-mediated (hATTR) amyloidosis in adults. In the
EU, ONPATTRO is approved for the treatment of hATTR amyloidosis in
adults with stage 1 or stage 2 polyneuropathy. Alnylam has a deep
pipeline of investigational medicines, including five product candidates
that are in late-stage development. Looking forward, Alnylam will
continue to execute on its "Alnylam 2020" strategy of building a
multi-product, commercial-stage biopharmaceutical company with a
sustainable pipeline of RNAi-based medicines to address the needs of
patients who have limited or inadequate treatment options. Alnylam
employs over 1,000 people worldwide and is headquartered in Cambridge,
MA. For more information about our people, science and pipeline, please
visit www.alnylam.com
and engage with us on Twitter at @Alnylam
or on LinkedIn.
Alnylam Forward-Looking Statements
Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's views with respect
to the potential for RNAi therapeutics, including patisiran, vutrisiran,
and fitusiran, its expectations regarding its alliance with Sanofi and
the potential for the receipt of future royalties on partnered programs,
the release of Sanofi from the lock-up restrictions on its stock
holdings in Alnylam, and its "Alnylam 2020" guidance for the advancement
and commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995. Actual results and
future plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation,
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches, successfully demonstrate the efficacy and safety of
its product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to occur in
other subjects or in additional studies or otherwise support further
development of product candidates for a specified indication or at all,
actions or advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials or
result in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting intellectual
property, Alnylam's ability to enforce its intellectual property rights
against third parties and defend its patent portfolio against challenges
from third parties, obtaining and maintaining regulatory approval,
pricing and reimbursement for products, progress in establishing a
commercial and ex-United States infrastructure, successfully launching,
marketing and selling its approved products globally, Alnylam’s ability
to successfully expand the indication for ONPATTRO in the future,
competition from others using technology similar to Alnylam's and others
developing products for similar uses, Alnylam's ability to manage its
growth and operating expenses, obtain additional funding to support its
business activities, and establish and maintain strategic business
alliances and new business initiatives, Alnylam's dependence on third
parties for development, manufacture and distribution of products, the
outcome of litigation, the risk of government investigations, and
unexpected expenditures, as well as those risks more fully discussed in
the "Risk Factors" filed with Alnylam's most recent Quarterly Report on
Form 10-Q filed with the Securities and Exchange Commission (SEC) and in
other filings that Alnylam makes with the SEC. In addition, any
forward-looking statements represent Alnylam's views only as of today
and should not be relied upon as representing its views as of any
subsequent date. Alnylam explicitly disclaims any obligation, except to
the extent required by law, to update any forward-looking statements.
With the exception of ONPATTRO (patisiran), none of Alnylam’s
investigational therapeutics have been approved by the U.S. Food and
Drug Administration, European Medicines Agency, or any other regulatory
authority and no conclusions can or should be drawn regarding the safety
or effectiveness of such investigational therapeutics.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190407005038/en/
Source: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom
(Investors
and Media)
617-682-4340
Josh Brodsky
(Investors)
617-551-8276
