Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron
Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive
interim data from an ongoing Phase 1 clinical study of their lead
in vivo genome editing candidate, NTLA-2001, which is being
developed as a single-dose treatment for transthyretin (ATTR)
amyloidosis. The Phase 1 study, run by Intellia as the program’s
development and commercialization lead, is evaluating NTLA-2001 in
people living with hereditary transthyretin amyloidosis with
polyneuropathy (ATTRv-PN). NTLA-2001 is the first CRISPR/Cas9-based
therapy candidate to be administered systemically, via intravenous
infusion, for precision editing of a gene in a target tissue in
humans. NTLA-2001 is designed to inactivate the TTR gene
in liver cells to prevent the production of misfolded transthyretin
(TTR) protein, which accumulates in tissues throughout the body and
causes the debilitating and often fatal complications of ATTR
amyloidosis. The interim data were presented today at the 2021
Peripheral Nerve Society (PNS) Annual Meeting and published in The
New England Journal of Medicine
(nejm.org/doi/full/10.1056/NEJMoa2107454.)1.
“These are the first ever clinical data suggesting that we can
precisely edit target cells within the body to treat genetic
disease with a single intravenous infusion of CRISPR. The interim
results support our belief that NTLA-2001 has the potential to halt
and reverse the devastating complications of ATTR amyloidosis with
a single dose,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “Solving the challenge of targeted delivery of
CRISPR/Cas9 to the liver, as we have with NTLA-2001, also unlocks
the door to treating a wide array of other genetic diseases with
our modular platform, and we intend to move quickly to advance and
expand our pipeline. With these data, we believe we are truly
opening a new era of medicine.”
The interim data released today cover the first six ATTRv-PN
patients across two single-ascending dose cohorts of the Phase 1
study, which is currently being conducted in the United Kingdom and
New Zealand. Single doses of either 0.1 mg/kg or 0.3 mg/kg of
NTLA-2001 were administered systemically. Reductions in serum TTR
levels were measured from baseline to day 28. Treatment with
NTLA-2001 led to dose-dependent reductions in serum TTR, with mean
reductions of 52% among the three patients in the 0.1 mg/kg dose
group, and 87% among the three patients in the 0.3 mg/kg dose
group, including one patient with a 96% reduction. By contrast, the
standard of care for ATTRv-PN, which requires chronic treatment,
typically yields TTR reductions of approximately 80%.
“This is exciting early data both for people living with this
devastating disease and for the entire scientific community working
to maximize the potential of genetics-based medicines through
cutting-edge research and technologies,” said George D.
Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of
Regeneron, which first partnered with Intellia in 2016 to advance
CRISPR/Cas9 gene-editing technology for in
vivo therapeutic development. “Thanks to large-scale human
genetics research, there have been many new genetic targets
identified and confirmed to have an impact on human health.
Combining this knowledge with the precision and enhanced
convenience of a single CRISPR infusion unlocks new possibilities
in treating – and potentially even curing – life-threatening and
historically difficult-to-address diseases.”
At both dose levels, NTLA-2001 was generally well-tolerated by
the six patients included in the interim analysis, with no serious
adverse events and no liver findings by day 28. Given the safety
and tolerability profile so far, NTLA-2001 is continuing to be
evaluated in the dose-escalation portion of the study, to
determine if a higher dose could result in a deeper reduction in
disease-causing protein levels leading to the potential for more
meaningful clinical benefit. As of the date of this
release, Cohort 3, evaluating NTLA-2001 at the 1 mg/kg dose
level, is actively enrolling.
Following the identification of a recommended dose in the
dose-escalation portion of the study, Intellia expects to begin a
single-dose expansion cohort in Part 2 of the Phase 1 trial later
this year. After completion of the Phase 1 trial, the company plans
to move to pivotal studies for both polyneuropathy and
cardiomyopathy manifestations of ATTR amyloidosis.
“ATTR amyloidosis is a progressive and fatal disease that
usually requires chronic, lifelong treatment. These interim Phase 1
data support NTLA-2001 as the only one-time treatment either on the
market or in development,” said Julian Gillmore, M.D., Ph.D.,
Professor of Medicine, National Amyloidosis Centre, UCL
Division of Medicine, Royal Free Hospital, U.K., and the
Phase 1 study’s national coordinating investigator. “As the
first-ever systemically administered CRISPR therapy candidate,
NTLA-2001 shows strong potential to stop the production and
accumulation of the misfolded TTR protein by inactivating the TTR
gene at the root of the disease. This approach could deliver
life-changing, lifelong benefits to patients with all forms of ATTR
amyloidosis, who continue to experience debilitating symptoms and
complications of disease while on the standard of care. While
further investigation is needed, these results are highly
encouraging.”
Intellia intends to present additional data from the study at a
medical or scientific meeting later this year.
1 Gillmore JD, Gane E, Taubel J, et al. CRISPR /Cas9 In Vivo
Gene Editing for Transthyretin Amyloidosis. N Engl J Med 2021.
nejm.org/doi/full/10.1056/NEJMoa2107454
Intellia Therapeutics Investor Event and Webcast
InformationIntellia will host a live webcast on Monday,
June 28, 2021 at 8:00 a.m. E.T. to review the presented data. To
join the webcast, please visit this link, or the Events and
Presentations page of the Investors & Media section of the
company’s website at www.intelliatx.com. A replay of the webcast
will be available on Intellia’s website for at least 30 days
following the call.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first curative treatment for ATTR amyloidosis. NTLA-2001 is
the first investigational CRISPR therapy candidate to be
administered systemically, or through a vein, to edit genes inside
the human body. Intellia’s proprietary non-viral platform deploys
lipid nanoparticles to deliver to the liver a two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 enzyme, which
carries out the precision editing. Robust preclinical data, showing
deep and long-lasting transthyretin (TTR) reduction following in
vivo inactivation of the target gene, supports NTLA-2001’s
potential as a single-administration therapeutic. Interim Phase 1
clinical data released in June 2021 confirm substantial,
dose-dependent reduction of TTR protein following a single dose of
NTLA-2001. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron.
About the NTLA-2001 Clinical ProgramThe
global Phase 1 trial is an open-label, multi-center, two-part study
of NTLA-2001 in adults with hereditary transthyretin amyloidosis
with polyneuropathy (ATTRv-PN). The trial’s primary objectives are
to assess the safety, tolerability, pharmacokinetics and
pharmacodynamics of NTLA-2001. Patients receive a single dose of
NTLA-2001 via intravenous administration. The study will enroll up
to 38 participants (ages 18-80 years) and consists of a
single-ascending dose phase followed by a dose-expansion phase,
which is expected to begin later in 2021.
Visit clinicaltrials.gov (NCT04601051) or
eudract.ema.europa.eu/ (2020-002034-32) for more details.
Enrollment is ongoing at clinical trial sites in the U.K. and
New Zealand, and Intellia is submitting additional regulatory
applications to support a planned global study expansion. After
completion of the Phase 1 trial, the company plans to move to
pivotal studies for both polyneuropathy and cardiomyopathy
manifestations of ATTR amyloidosis.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare,
progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis
occurs when a person is born with mutations in
the TTR gene, which causes the liver to produce
structurally abnormal transthyretin (TTR) protein with a propensity
to misfold. These damaged proteins build up as amyloid in the body,
causing serious complications in multiple tissues, including the
heart, nerves and digestive system. ATTRv amyloidosis predominantly
manifests as polyneuropathy (ATTRv-PN), which can lead to nerve
damage, or cardiomyopathy (ATTRv-CM), which can lead to heart
failure. Some individuals without the genetic mutation produce
non-mutated, or wild-type TTR proteins that become unstable over
time, misfolding and aggregating in disease-causing amyloid
deposits. This condition, called wild-type ATTR (ATTRwt)
amyloidosis, primarily affects the heart. There are an estimated
50,000 people worldwide living with ATTRv amyloidosis and between
200,000 and 500,000 people with ATTRwt amyloidosis.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics using
CRISPR/Cas9 technology. To fully realize the transformative
potential of CRISPR/Cas9, Intellia is pursuing two primary
approaches. The company’s in vivo programs use intravenously
administered CRISPR as the therapy, in which proprietary delivery
technology enables highly precise editing of disease-causing genes
directly within specific target tissues. Intellia’s ex vivo
programs use CRISPR to create the therapy by removing,
re-engineering and re-infusing the patient’s own cells to treat
cancer and autoimmune diseases. Intellia’s deep scientific,
technical and clinical development experience, along with its
robust intellectual property portfolio, have enabled the company to
take a leadership role in harnessing the full potential of
CRISPR/Cas9 to create new classes of genetic medicine. Learn more
at intelliatx.com. Follow us on Twitter @intelliatweets.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that
invents life-transforming medicines for people with serious
diseases. Founded and led for over 30 years by
physician-scientists, our unique ability to repeatedly and
consistently translate science into medicine has led to nine
FDA-approved treatments and numerous product candidates in
development, almost 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, pain, hematologic
conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary VelociSuite®
technologies, such as VelocImmune®, which uses unique genetically
humanized mice 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.
Intellia Forward-Looking StatementsThis press
release contains “forward-looking statements” of Intellia
Therapeutics, Inc. (“Intellia”, “we” or “our”) 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: ability to enroll and dose the necessary subjects in
the clinical studies for NTLA-2001 for the treatment of
transthyretin amyloidosis (“ATTR”), provide timing on data readouts
from the clinical studies, and successfully secure additional
clinical studies authorizations, such as investigational new drug
applications (“IND”) and clinical trial applications (“CTA”), in
other countries; ability to evaluate NTLA-2001 in a broader ATTR
population; expectation that clinical results will support
NTLA-2001’s safety and activity profile; belief that NTLA-2001 can
be approved as a single-dose therapy or that it can halt and
reverse ATTR progression; plans to present data at upcoming
scientific conferences; advancement, expansion and acceleration of
our CRISPR/Cas9 technology and in vivo pipeline to develop
breakthrough genome editing treatments for people living with
severe diseases; ability to demonstrate our platform’s modularity
and replicate or apply results achieved in preclinical studies,
including those in our ATTR program, in any future studies,
including human clinical trials; ability to optimize the impact of
our collaborations on our development programs, including but not
limited to our collaboration with Regeneron Pharmaceuticals, Inc.
(“Regeneron”); statements regarding the timing of regulatory
filings and clinical trial execution, including dosing of patients,
regarding our development programs; and potential commercial
opportunities, including value and market, for our product
candidates.
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 our ability to protect and maintain our intellectual
property position; risks related to our relationship with third
parties, including our licensors and licensees; risks related to
the ability of our licensors to protect and maintain their
intellectual property position; uncertainties related to regulatory
agencies’ evaluation of regulatory filings and other information
related to our product candidates; uncertainties related to the
authorization, initiation and conduct of studies and other
development requirements for our product candidates; the risk that
any one or more of our product candidates, including those that are
co-developed, 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 our
collaborations with Regeneron or our 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 and quarterly report on Form 10-Q, as
well as discussions of potential risks, uncertainties, and other
important factors in Intellia’s other filings with the Securities
and Exchange Commission (“SEC”). 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 Forward Looking Statements
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, and 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 or otherwise commercialized 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 product
candidates being developed by Regeneron and/or its collaborators
(collectively, “Regeneron’s Product Candidates”) and research and
clinical programs now underway or planned, such as NTLA-2001 (a
product candidate being developed for transthyretin (ATTR)
amyloidosis under a multi-target discovery, development, and
commercialization collaboration between Regeneron and Intellia
Therapeutics, Inc.); the extent to which the results from the
research and development programs conducted by Regeneron and/or its
collaborators (including the Phase 1 clinical study evaluating
NTLA-2001 discussed in this press release) may be replicated in
other studies and/or lead to advancement of product candidates to
clinical trials, therapeutic applications, or regulatory approval;
the potential of the CRISPR/Cas9 gene-editing technology discussed
in this press release for in vivo therapeutic development;
uncertainty of the utilization, market acceptance, and commercial
success of Regeneron's Products and Regeneron’s Product Candidates
and the impact of studies (whether conducted by Regeneron or others
and whether mandated or voluntary), including the Phase 1 clinical
study evaluating NTLA-2001 discussed in this press release, on any
of the foregoing or any potential regulatory approval of
Regeneron's Products and Regeneron’s Product Candidates (such as
NTLA-2001); the likelihood, timing, and scope of possible
regulatory approval and commercial launch of Regeneron's Product
Candidates and new indications for Regeneron's Products; 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 Regeneron’s Product Candidates;
the ability of Regeneron and/or its collaborators to manufacture
and manage supply chains for multiple products and product
candidates; safety issues resulting from the administration of
Regeneron's Products and Regeneron’s Product Candidates in
patients, including serious complications or side effects in
connection with the use of Regeneron's Products and Regeneron’s
Product Candidates (such as NTLA-2001) 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
Regeneron’s Product Candidates; ongoing regulatory obligations and
oversight impacting Regeneron's Products, research and clinical
programs, and business, including those relating to patient
privacy; 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, or more cost effective than,
Regeneron's Products and Regeneron’s 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; the potential for any
license, collaboration, or supply 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; 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, Dupixent® (dupilumab),
Praluent® (alirocumab), and REGEN-COVTM (casirivimab and
imdevimab)), 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,
2020 and its Form 10-Q for the quarterly period ended March 31,
2021. 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 or otherwise)
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 ContactsMedia:Matt
CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
Investors:Lina LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Regeneron
ContactsMedia:Alexandra
Bowie+1-202-213-1643alexandra.bowie@regeneron.com
Investors:Vesna
Tosic+1-914-847-5443Vesna.tosic@regeneron.com
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