− 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 –
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 RNAiRNAi (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 PharmaceuticalsAlnylam (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 StatementsVarious 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/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Historical Stock Chart
From Aug 2024 to Sep 2024
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Historical Stock Chart
From Sep 2023 to Sep 2024