TARRYTOWN, N.Y. and CAMBRIDGE,
Mass., June 1, 2020
/PRNewswire/ --
- Regeneron and Intellia to co-develop potential hemophilia A
and B treatments using their jointly-owned targeted transgene
insertion capabilities
- Regeneron gains rights to develop products for
additional in vivo targets and new rights for ex vivo
product development
- Intellia receives $100 million
through upfront cash and equity investment
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and
Intellia Therapeutics, Inc. (NASDAQ: NTLA) announced an
expansion of their existing collaboration to provide Regeneron with
rights to develop products for additional in vivo
CRISPR/Cas9-based therapeutic targets and for the companies to
jointly develop potential products for the treatment of hemophilia
A and B. Regeneron also receives non-exclusive rights to
independently develop and commercialize ex vivo gene edited
products. Intellia will receive an upfront payment of $70 million, and Regeneron will make an
additional equity investment in Intellia of $30 million at $32.42 per share.
Regeneron and Intellia have worked together to make significant
advances with Intellia's CRISPR/Cas9 platform to enable the
targeted insertion of therapeutic proteins and antibodies. This
collaboration expansion allows the companies to leverage more fully
their jointly-developed targeted transgene insertion capabilities
and potentially accelerate efforts to discover and develop new
therapeutics, including products for hemophilia A and B. In
preclinical studies, the companies demonstrated the first
CRISPR/Cas9-mediated targeted transgene insertion in the liver of
non-human primates, which generated normal or higher levels of
circulating human Factor IX. Factor IX is a blood-clotting protein
that is missing or defective in hemophilia B patients. These
results suggest that transgene insertion may provide a functional
Factor 9 gene, which encodes for this important
protein.
"The Regeneron team works hard to push the boundaries of science
and technology, and we believe the precise in vivo gene
insertion capabilities jointly developed with Intellia could be a
promising therapeutic platform with significant potential in many
diseases, including those that have been historically difficult to
treat," said George D. Yancopoulos,
M.D., Ph.D., Co-Founder, President and Chief Scientific Officer,
Regeneron. "We're pleased to expand our work with Intellia, a
like-minded group of scientists focused on maximizing the potential
of CRISPR/Cas9 in order to help as many patients as possible."
"We're excited to work with Regeneron on what could
potentially be a cure for hemophilia A and B in this expansion
of our successful collaboration that builds on our
leading insertion capabilities," said Intellia's Chief Executive
Officer and President, John M.
Leonard, M.D. "We believe that our CRISPR/Cas9-based
technology addresses the limitations of current replacement and
gene therapy approaches, and
importantly, may provide a durable, potentially
life-long solution to these genetic diseases."
Under the amended agreement, the term of the companies' existing
collaboration is extended until April
2024, with Regeneron having an option to renew for an
additional two years. Regeneron will have rights to discover and
develop CRISPR/Cas9-based therapeutic products for an additional
five in vivo liver targets, for a total of up to 15 targets.
As currently set forth in the existing collaboration, the parties
will jointly research these in vivo targets, and thereafter
certain targets may be developed by Regeneron or Intellia, or
co-developed under certain conditions. Per the terms of the
original agreement, Regeneron will pay potential royalties and
milestone payments for the in vivo products it independently
develops. In addition, Regeneron will receive a royalty-bearing,
non-exclusive license to certain Intellia intellectual property to
develop and commercialize up to 10 ex vivo CRISPR/Cas9
products in defined cell types.
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
diseases, allergic and inflammatory diseases, cancer,
cardiovascular and metabolic diseases, infectious diseases, pain
and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our
proprietary VelociSuite® technologies,
including VelocImmune® which uses a
unique genetically-humanized mouse to produce optimized fully-human
antibodies and bispecific antibodies, and through 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.
About Intellia Therapeutics
Intellia
Therapeutics is a leading genome editing company focused on
developing proprietary, curative therapeutics using the CRISPR/Cas9
system. Intellia believes the CRISPR/Cas9 technology has the
potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients' diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more
about Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and follow us on Twitter
@intelliatweets.
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 impact of
SARS-CoV-2 (the virus that has caused the COVID-19 pandemic) on
Regeneron's business and its employees, collaborators, suppliers,
and other third parties on which Regeneron relies, Regeneron's and
its collaborators' ability to continue to conduct research and
clinical programs, Regeneron's ability to manage its supply chain,
net product sales of products marketed by Regeneron and/or its
collaborators (collectively, "Regeneron's Products"), and the
global economy; the nature, timing, and possible success and
therapeutic applications of Regeneron's Products and Regeneron's
product candidates and research and clinical programs now underway
or planned, such as the programs discussed in this press release to
develop products for in vivo CRISPR/Cas9-based therapeutic
targets with Intellia Therapeutics, Inc. (including for the
treatment of hemophilia A and B); the extent to which the results
from the research and development programs conducted by Regeneron
and/or its collaborators (including based on the collaboration
discussed in this press release) may lead to advancement of product
candidates to clinical trials or 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 collaboration with Intellia
Therapeutics, Inc. discussed in this press release, to be cancelled
or terminated without any further product success; the likelihood,
timing, and scope of possible regulatory approval and commercial
launch of Regeneron's product candidates and new indications for
Regeneron's Products; unforeseen safety issues resulting from the
administration of Regeneron's Products and product candidates in
patients, including serious complications or side effects in
connection with the use of Regeneron's Products and product
candidates in clinical trials; determinations by regulatory and
administrative governmental authorities which may delay or restrict
Regeneron's ability to continue to develop or commercialize
Regeneron's Products and product candidates; ongoing regulatory
obligations and oversight impacting Regeneron's Products, research
and clinical programs, and business, including those relating to
patient privacy; uncertainty of market acceptance and commercial
success of Regeneron's 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 Products and product candidates; the availability and
extent of reimbursement of Regeneron'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; competing drugs and
product candidates that may be superior to Regeneron's 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;
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
Dupixent® (dupilumab) and Praluent®
(alirocumab)), other litigation and other proceedings and
government investigations relating to the Company and/or its
operations, the ultimate outcome of any such proceedings and
investigations, 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, 2019 and its Form 10-Q
for the quarterly period ended March
31, 2020. 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).
Intellia's Forward-Looking Statements
This
press release contains "forward-looking statements"
of Intellia Therapeutics, Inc. ("Intellia" or the
"Company") within the meaning of the Private Securities Litigation
Reform Act of 1995. These forward-looking statements include, but
are not limited to, express or implied statements regarding
Intellia's beliefs and expectations regarding its: planned
submission of an investigational new drug ("IND") application or
similar clinical trial application for NTLA-2001 for the treatment
of transthyretin amyloidosis ("ATTR") in mid-2020 and its planned
dosing of first patients in the second half of 2020; plans to
submit an IND application or similar clinical trial
application for NTLA-5001, its first T cell receptor
("TCR")-directed engineered cell therapy development candidate for
its acute myeloid leukemia ("AML") program in the first half of
2021; plans to submit an IND or similar clinical trial application
for its hereditary angioedema ("HAE") program in the second half of
2021; plans to advance and complete preclinical studies, including
non-human primate studies for its ATTR program and HAE programs,
and other animal studies supporting other in vivo and ex vivo
programs, including its AML program; development of a proprietary
LNP/AAV hybrid delivery system, as well as its modular platform to
advance its complex genome editing capabilities, such as gene
insertion; further development of its proprietary cell engineering
process for multiple sequential editing; presentation of additional
data at upcoming scientific conferences, and other preclinical data
in 2020; advancement and expansion of its CRISPR/Cas9 technology to
develop human therapeutic products, as well as its ability to
maintain and expand its related intellectual property portfolio;
ability to demonstrate its platform's modularity and replicate or
apply results achieved in preclinical studies, including those in
its ATTR, AML, and HAE programs, in any future studies, including
human clinical trials; ability to develop other in vivo or ex vivo
cell therapeutics of all types, and those targeting WT1 in AML in
particular, using CRISPR/Cas9 technology; ability to optimize the
impact of its collaborations on its development programs, including
but not limited to its collaborations with Novartis or Regeneron
Pharmaceuticals, Inc., Regeneron's ability to enter into a
co-development and co-promotion agreement for the HAE program,
and the potential timing and receipt of future
milestones and royalties based on Intellia's collaboration with
Regeneron; and statements regarding the timing of regulatory
filings and clinical trial execution, including dosing of
patients in its development programs.
Any forward-looking statements in this press release are
based on management's current expectations and beliefs of future
events, and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
These risks and uncertainties include, but are not limited to:
risks related to Intellia's ability to protect and maintain its
intellectual property position; risks related to Intellia's
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to regulatory agencies' evaluation of regulatory filings
and other information related to its product
candidates; uncertainties related to the initiation and
conduct of studies and other development requirements for its
product candidates; the risk that any one or more of Intellia's
product candidates will not be successfully developed and
commercialized; the risk that the results of preclinical studies or
clinical studies will not be predictive of future results in
connection with future studies; and the risk that Intellia's
collaborations with Novartis or Regeneron or its other ex vivo
collaborations will not continue or will not be successful. For a
discussion of these and other risks and uncertainties, and other
important factors, any of which could cause Intellia's actual
results to differ from those contained in the forward-looking
statements, see the section entitled "Risk Factors" in Intellia's
most recent annual report on Form 10-K as well as discussions of
potential risks, uncertainties, and other important factors in
Intellia's other filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Intellia undertakes no duty to update this
information unless required by law.
Regeneron IR Contact:
Mark
Hudson
914.847.3482
Mark.hudson@regeneron.com
Regeneron Media Contact:
Alexandra Bowie
914.847.3407
Alexandra.bowie@regeneron.com
Intellia Media Contact:
Jennifer Mound Smoter
224.804.4462
Jenn.smoter@intelliatx.com
Intellia IR Contact:
Lina
Li
857.706.1162
lina.li@intelliatx.com
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SOURCE Regeneron Pharmaceuticals, Inc.