TARRYTOWN, N.Y. and
CAMBRIDGE, Mass., April 8, 2019 /PRNewswire/ --
Companies to also jointly advance select number of
preclinical disease programs with targets expressed in the liver
and treatments for C5 complement-mediated diseases
Regeneron to invest $800
million through upfront cash and equity investment in
Alnylam, with up to additional $200
million in potential near-term
milestones
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Alnylam
Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced a
collaboration to discover, develop and commercialize new RNA
interference (RNAi) therapeutics for a broad range of diseases by
addressing disease targets expressed in the eye and central nervous
system (CNS), in addition to a select number of targets expressed
in the liver. The collaboration will leverage both companies'
scientific and technological expertise and will build on Alnylam's
recent preclinical data showing potent and highly durable delivery
of RNAi therapeutics to achieve target gene silencing in the eye
and CNS. The collaboration will also benefit from Regeneron's
industry-leading VelociSuite® technologies and
capabilities from the Regeneron Genetics Center (RGC).
Under the terms of the alliance, Alnylam will work exclusively
with Regeneron to discover RNAi therapeutics for eye and CNS
diseases. Regeneron will lead development and commercialization for
all programs targeting eye diseases, with Alnylam entitled to
potential milestone and royalty payments. The companies will
jointly advance and alternate leadership on CNS programs, with the
lead party retaining global development and commercial
responsibility. For CNS programs, both companies will have the
option at candidate selection to participate equally in potential
future profits of programs led by the other party.
The collaboration also includes a select number of RNAi
therapeutic programs designed to target genes expressed in the
liver, which can influence a wide variety of diseases throughout
the body. These programs include a planned joint effort evaluating
anti-C5 antibody-siRNA combinations for C5 complement-mediated
diseases including evaluating the combination of Regeneron's
pozelimab (REGN3918), currently in Phase 1 development, with
Alnylam's cemdisiran, currently in Phase 2 development. Alnylam
will retain control of cemdisiran monotherapy development, and
Regeneron will lead combination development. The parties will
equally share investment and potential future profits on the
monotherapy program, and Alnylam will receive royalties on any
potential combination product sales. For all other alliance liver
programs, the parties will alternate leadership and participate
equally in potential profits. The companies will continue their
previously-announced collaboration to identify RNAi
therapeutics for the chronic liver disease nonalcoholic
steatohepatitis (NASH) based on novel RGC findings. Alnylam retains
broad global rights to all of its other unpartnered liver-directed
clinical and preclinical pipeline programs.
"At Regeneron we believe the best use of our resources is to
invest in potentially game-changing science that will yield
innovative medicines for patients with serious diseases. This
collaboration couples proven and emerging RNAi technology, which
holds important promise in many diseases, with Regeneron's
world-leading genetics research and target discovery engine," said
George D. Yancopoulos, M.D., Ph.D.,
President and Chief Scientific Officer of Regeneron. "This
collaboration enables us to reach targets inside the cell,
complementing our expertise in antibodies, which are ideal for
extracellular targets and those on the cell surface. Through
the RGC and our other research groups, we are already identifying
additional targets that may be well-suited for RNAi-based drug
development, particularly in the eye and CNS."
"This new industry-leading alliance is aimed at realizing what
we believe to be a significant opportunity for RNAi therapeutics as
potentially transformative medicines for ocular and CNS diseases.
We are thrilled to collaborate with Regeneron, a like-minded
science-based organization, to significantly accelerate our efforts
to bring RNAi therapeutics to patients," said John Maraganore, Ph.D., Chief Executive Officer
of Alnylam. "Importantly, the alliance structure enables Alnylam to
continue to build its industry-leading pipeline of RNAi
therapeutics while retaining significant product rights. In
addition, the near-term payments under this new agreement will
strengthen Alnylam's balance sheet with over $2 billion in pro forma cash upon closing
of the transaction, supporting our global efforts to develop and
commercialize multiple products as potentially breakthrough
medicines and advance our profile toward sustainable
profitability."
Regeneron has agreed to make a $400
million upfront payment to Alnylam and to purchase
$400 million of Alnylam equity at a
price per share of $90.00 (4.44
million common shares), based on the volume-weighted average price
over the last fifteen-trading-day period. Alnylam is eligible to
receive up to an additional $200
million in milestone payments upon achievement of certain
criteria during early clinical development for the eye and CNS
programs. The companies plan to advance programs directed to 30
targets and introduce many into clinical development during the
initial five-year discovery period, which includes an option to
extend. For each program, Regeneron will provide Alnylam with
$2.5 million in funding at program
initiation and an additional $2.5
million at lead candidate identification, translating to the
potential for approximately $30
million in annual discovery funding to Alnylam as the
alliance reaches steady state. The alliance and equity-related
agreements are subject to customary closing conditions and
clearances, including clearance under the Hart-Scott Rodino
Antitrust Improvements Act.
Alnylam, alongside multiple other leading life sciences
companies, is also a member of Regeneron's pre-competitive
consortium to sequence the DNA of 500,000 individuals in the UK
Biobank health resource and subsequently make the data publicly
available to the global research community.
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.
About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that
invents life-transforming medicines for people with serious
diseases. Founded and led for 30 years by physician-scientists, our
unique ability to repeatedly and consistently translate science
into medicine has led to seven FDA-approved treatments and numerous
product candidates in development, all of which were homegrown in
our laboratories. Our medicines and pipeline are designed to help
patients with eye disease, heart disease, allergic and inflammatory
diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our
proprietary VelociSuite® technologies,
such as VelocImmune® which produces
optimized fully-human antibodies, and ambitious research
initiatives such as the Regeneron Genetics Center, which is
conducting one of the largest genetics sequencing efforts in the
world.
For additional information about the company, please visit
www.regeneron.com or follow @Regeneron on Twitter.
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 of RNAi therapeutics
to achieve target gene silencing in the CNS and eye, Regeneron's
participation in the development and commercialization of RNAi
therapeutics directed to CNS, eye and a select number of liver
targets, as well as the planned joint effort evaluating an anti-C5
antibody-siRNA combination as well as a monotherapy approach, the
parties plans to advance 30 targets and file multiple
Investigational New Drug Applications during the discovery period,
Alnylam's expectations regarding funding for each program under the
collaboration at various stages of development, its expectations
regarding the receipt of upfront cash and an equity investment, as
well as potential development, regulatory and sales milestones and
royalties from Regeneron, its expectations regarding available cash
for its operations through multiple product launches, and
expectations regarding 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; Regeneron's ability to
successfully advance and develop programs targeting eye diseases,
resulting in the potential payment of milestones and royalties to
Alnylam; the parties ability to successfully develop and
commercialize CNS programs; 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
Annual Report on Form 10-K 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.
Regeneron Forward-Looking Statements and Use of Digital
Media
This press release includes forward-looking
statements that involve risks and uncertainties relating to future
events and the future performance of Regeneron Pharmaceuticals,
Inc. ("Regeneron" or the "Company"), and actual events or results
may differ materially from these forward-looking
statements. Words such as "anticipate," "expect," "intend,"
"plan," "believe," "seek," "estimate," variations of such words,
and similar expressions are intended to identify such
forward-looking statements, although not all forward-looking
statements contain these identifying words. These statements
concern, and these risks and uncertainties include, among others,
the nature, timing, and possible success and therapeutic
applications of Regeneron's or its collaborators' products, product
candidates, and research and clinical programs now underway or
planned, such as the RNA interference programs discussed in this
press release (including programs evaluating anti-C5 antibody-siRNA
combinations for C5 complement-mediated diseases and RNAi
therapeutics for the chronic liver disease nonalcoholic
steatohepatitis); the extent to which the results from the research
and development programs conducted by Regeneron or its
collaborators (including based on the collaboration discussed in
this press release) may be replicated in other studies and lead to
therapeutic applications; the potential for any license or
collaboration agreement, including Regeneron's agreements with
Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their
respective affiliated companies, as applicable), as well as
Regeneron's collaborations with Alnylam Pharmaceuticals, Inc.
discussed in this news release, to be cancelled or terminated
without any product success; the likelihood, timing, and scope of
possible regulatory approval and commercial launch of Regeneron's
late-stage product candidates and new indications for marketed
products; unforeseen safety issues resulting from the
administration of products and product candidates in patients,
including serious complications or side effects in connection with
the use of Regeneron's or its collaborators' product candidates in
clinical trials; ongoing regulatory obligations and oversight
impacting Regeneron's marketed products, research and clinical
programs, and business, including those relating to patient
privacy; determinations by regulatory and administrative
governmental authorities which may delay or restrict Regeneron's or
its collaborators' ability to continue to develop or commercialize
products and product candidates; competing drugs and product
candidates that may be superior to Regeneron's or its
collaborators' products and product candidates; uncertainty of
market acceptance and commercial success of Regeneron's or its
collaborators' products and product candidates and the impact of
studies (whether conducted by Regeneron or others and whether
mandated or voluntary) on the commercial success of Regeneron's or
its collaborators' products and product candidates; the ability of
Regeneron to manufacture and manage supply chains for multiple
products and product candidates; the ability of Regeneron's
collaborators, suppliers, or other third parties (as applicable) to
perform manufacturing, filling, finishing, packaging, labeling,
distribution, and other steps related to Regeneron's products and
product candidates; the availability and extent of reimbursement of
the Company's products from third-party payers, including private
payer healthcare and insurance programs, health maintenance
organizations, pharmacy benefit management companies, and
government programs such as Medicare and Medicaid; coverage and
reimbursement determinations by such payers and new policies and
procedures adopted by such payers; unanticipated expenses; the
costs of developing, producing, and selling products; the ability
of Regeneron to meet any of its financial projections or guidance
and changes to the assumptions underlying those projections or
guidance; and risks associated with intellectual property of other
parties and pending or future litigation relating thereto,
including without limitation the patent litigation and other
related proceedings relating to
EYLEA® (aflibercept) Injection, and
Dupixent® (dupilumab) Injection, and
Praluent® (alirocumab) Injection, the ultimate
outcome of any such proceedings, and the impact any of the
foregoing may have on Regeneron's business, prospects, operating
results, and financial condition. A more complete description of
these and other material risks can be found in Regeneron's filings
with the U.S. Securities and Exchange Commission, including its
Form 10-K for the year ended December
31, 2018. Any forward-looking statements are made based
on management's current beliefs and judgment, and the reader is
cautioned not to rely on any forward-looking statements made by
Regeneron. Regeneron does not undertake any obligation to update
publicly any forward-looking statement, including without
limitation any financial projection or guidance, whether as a
result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and
social media outlets to publish important information about the
Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website
(http://newsroom.regeneron.com) and its Twitter feed
(http://twitter.com/regeneron).
Regeneron Investor Relations
Mark Hudson
Tel: +1 (914)
847-3482
Mark.hudson@regeneron.com
Regeneron Media Relations
Hala Mirza
Tel: +1 (917)
929-1734
Hala.mirza@regeneron.com
Alnylam Investor Relations
Josh Brodsky
Tel: +1 (617) 551-8276
Alnylam Investor & Media Relations
Christine Regan Lindenboom
Tel: +1 (617)
682-4340
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SOURCE Regeneron Pharmaceuticals, Inc.