Serum TTR reductions were sustained at all doses tested with
follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six
months in the 0.7 and 1.0 mg/kg cohorts
Pharmacokinetic modeling and simulation indicated that an 80
mg fixed dose provides similar exposure to the 1.0 mg/kg dose,
where treatment with NTLA-2001 resulted in 93% mean and 98% maximum
serum TTR reduction by day 28
Intellia to host investor event to discuss updated data from
Phase 1 study of NTLA-2001, the first-ever systemically
administered in vivo CRISPR investigational therapy today,
Friday, June 24, at 8:00 a.m. ET
CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 24,
2022 /PRNewswire/ -- Intellia Therapeutics, Inc.
(NASDAQ: NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:
REGN) today announced additional positive interim data from
an ongoing Phase 1 study of their lead investigational in vivo
genome editing candidate, NTLA-2001, which is being developed as a
single-dose treatment for transthyretin (ATTR) amyloidosis. The
data were presented in an oral presentation at the European
Association for the Study of the Liver (EASL) International Liver
Congress™ 2022, taking place June 22
– 26 in London.
The presentation today included extended follow-up data from 15
patients with hereditary ATTR amyloidosis with polyneuropathy
(ATTRv-PN) treated across four single-ascending dose cohorts in
Part 1 of the study. Results demonstrated sustained durability of
serum transthyretin (TTR) reduction through the last measured
timepoint in the ongoing observation. These data support
NTLA-2001's continued development as a potential one-time treatment
to permanently inactivate the TTR gene and reduce the
disease-causing protein. At the highest dose evaluated, treatment
with NTLA-2001 at 1.0 mg/kg resulted in a 93% mean and 98% maximum
serum TTR reduction by day 28 across the six patients treated. With
longer-term follow-up data now available, these deep reductions
continue to be sustained through six months, with an observed mean
reduction of 93%. Additionally, three patients in the 1.0 mg/kg
cohort have reached nine months in the follow-up period with no
evidence of a loss in TTR reduction after a single dose. In the 0.7
mg/kg dose cohort, the 86% mean serum TTR reduction observed at day
28 also remained durable through six months. Further, in the 0.1
and 0.3 mg/kg cohorts, patients have now reached 12 months of
follow-up, and a durable response to treatment continues to be
observed. Notably, patients in the 0.3 mg/kg cohort sustained an
89% mean serum TTR reduction at 12 months.
At all four dose levels, NTLA-2001 was generally well
tolerated through the follow-up period (median follow-up of 10
months). The majority of adverse events were mild in
severity with 73% (n = 11) of patients reporting a maximal adverse
event severity of Grade 1. There was a single possibly related
serious adverse event of vomiting (Grade 3) reported in a patient
with concomitant medical history of gastroparesis in the 1.0 mg/kg
dose group. The most frequent adverse events included headache,
infusion-related reactions, back pain, rash and nausea. All
infusion-related reactions were considered mild, resolving without
clinical sequelae.
The safety and activity profile of NTLA-2001 observed in Part 1
indicates that NTLA-2001 has a favorable therapeutic window. These
data combined with pharmacokinetic modeling and simulation data
support the utilization of a fixed dose of 80 mg in Part 2, which
is anticipated to yield similar exposures to the 1.0 mg/kg dose.
Dosing is ongoing in Part 2, the single-dose expansion cohort of
the polyneuropathy arm.
"Based on the interim data shared today, we believe NTLA-2001's
potential to be a transformational treatment for patients with ATTR
amyloidosis is becoming clearer. The safety, depth of serum TTR
reduction and durability profile demonstrated thus far highlights
its potential for halting and reversing the disease after a single
dose," said Intellia President and Chief Executive Officer
John Leonard, M.D. "These data
further underscore the power of genomic medicines and bolster the
probability of success across our broader in vivo genome
editing platform. We look forward to progressing the clinical
development of the first-ever systemically administered in
vivo CRISPR investigational therapy."
"We're pleased to share updated data that enhance the safety and
durability profile of NTLA-2001, increasing our confidence in its
potential as a one-time, systemically delivered and long-lasting
CRISPR-based therapy," said George D.
Yancopoulos, Ph.D., M.D., President and Chief Scientific
Officer of Regeneron. "Single-dose in vivo gene editing
could one day help patients with a variety of hard-to-treat genetic
diseases, making it one of the most exciting medical breakthroughs
on the horizon today."
The Phase 1 study, run by Intellia as the program's development
and commercialization lead as part of a multi-target
collaboration with Regeneron, is evaluating NTLA-2001 in
patients with either ATTRv-PN or ATTR amyloidosis with
cardiomyopathy (ATTR-CM). The cardiomyopathy arm, evaluating
NTLA-2001 across patients classified with New York Heart
Association (NYHA) Class I – III heart failure, is ongoing. The
companies plan to present the first interim data from the
cardiomyopathy arm in the second half of 2022. Enrollment across
both ATTRv-PN and ATTR-CM patient populations is expected to
complete in 2022.
Intellia Therapeutics Investor Event and Webcast
Information
Intellia will host a live webcast today, Friday, June 24, 2022, at 8:00 a.m. ET, 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-2001
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001
could potentially be the first single-dose 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. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron. The 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) or transthyretin amyloidosis with
cardiomyopathy (ATTR-CM). Visit clinicaltrials.gov
(NCT04601051) for more details.
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 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.
About Intellia Therapeutics
Intellia Therapeutics, a
leading clinical-stage genome editing company, is developing novel,
potentially curative therapeutics leveraging CRISPR-based
technologies. To fully realize the transformative potential of
CRISPR-based technologies, 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 using engineered human
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 genome editing to create new classes of genetic medicine. Learn
more at intelliatx.com. Follow us on
Twitter @intelliatx.
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, 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 or licensees (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 or licensees (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 or
licensees (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, licensees, 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-COV® (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, 2021 and its Form 10-Q for the
quarterly period ended March 31,
2022. 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
(https://newsroom.regeneron.com/) and its Twitter
feed (https://twitter.com/regeneron).
Intellia Forward-Looking Statements
This 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 the safety, efficacy and
advancement of our clinical program for NTLA-2001 for the treatment
of ATTR amyloidosis, including the potential for NTLA-2001
to be a transformative treatment for people with ATTR
amyloidosis; the expected timing of data releases,
regulatory filings, and the initiation and completion of clinical
trials, including completion of enrollment across both
ATTRv-PN and ATTR-CM patient populations in 2022; our
ability to successfully secure additional clinical studies
authorizations, such as investigational new drug applications
("IND") and clinical trial applications ("CTA"); our belief that
NTLA-2001 can be approved as a single-dose therapy; our plans to
present data at upcoming scientific conferences, including
the presentation of interim data from the cardiomyopathy arm
in the second half of 2022; the advancement, expansion,
acceleration and success 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 for NTLA-2002 for
the treatment of hereditary angioedema; our 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 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 Contacts:
|
|
|
|
Media
Relations
Ella
Campbell
+1-914-847-7017
ella.campbell@regeneron.com
|
Investor
Relations Vesna
Tosic
+1
914-847-5443
Vesna.Tosic@regeneron.com
|
|
|
Intellia Contacts:
|
|
|
|
Media
Relations
Matt
Crenson
Ten Bridge
Communications
+1-917-640-7930
media@intelliatx.com
mcrenson@tenbridgecommunications.com
|
Investor
Relations
Ian
Karp
Senior Vice President,
Investor Relations and Corporate Communications
+1-857-449-4175
ian.karp@intelliatx.com
|
|
Lina Li Director, Investor Relations
and Corporate Communications
+1-857-706-1612
lina.li@intelliatx.com
|
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SOURCE Regeneron Pharmaceuticals, Inc.