Interim data from the cardiomyopathy arm of the Phase 1 study
of NTLA-2001 showed deep and sustained mean serum transthyretin
(TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses,
respectively, at day 28
NTLA-2001 was generally well-tolerated at both dose
levels
Intellia to discuss data at investor event today,
Friday, September 16, at 8:00 a.m. ET
CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Sept. 16,
2022 /PRNewswire/ -- Intellia Therapeutics, Inc.
(NASDAQ: NTLA) and Regeneron Pharmaceuticals, Inc.
(NASDAQ:REGN) today announced positive interim results from
an ongoing Phase 1 clinical trial of NTLA-2001, an investigational
in vivo CRISPR/Cas9 genome editing therapy in development as
a single-dose treatment for transthyretin (ATTR) amyloidosis. The
interim data include 12 adult patients with ATTR amyloidosis with
cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA)
Class I – III heart failure. Single doses of 0.7 mg/kg and 1.0
mg/kg of NTLA-2001 were administered via intravenous infusion, and
the change from baseline in serum transthyretin (TTR) protein
concentration was measured for each patient.
Administration of NTLA-2001 led to rapid and deep reductions in
serum TTR by day 28 as follows:
Cohort
|
Mean (min, max) % serum
TTR reduction by day 28
|
0.7 mg/kg, NYHA Class
I/II (n=3)*
|
92% (91%,
95%)
|
0.7 mg/kg, NYHA Class
III (n=6)*
|
94% (91%,
97%)
|
1.0 mg/kg, NYHA Class
I/II (n=3)
|
92% (90%,
95%)
|
|
*Mean (min, max) %
serum TTR reduction by day 28 for 0.7 mg/kg cohort (n=9) was 93%
(91%, 97%).
|
|
These profound reductions in serum TTR were sustained throughout
the observation period, with patient follow-up ranging from two to
six months as of the data cut-off date of July 1, 2022. These data support NTLA-2001's
potential as a one-time treatment to permanently inactivate the
TTR gene and reduce the disease-causing protein in people
with ATTR-CM.
"ATTR amyloidosis is a multifaceted disease in need of
additional treatment options. These new interim results demonstrate
that NTLA-2001 can profoundly reduce serum TTR levels in patients
whose condition results in cardiomyopathy," said Intellia President
and Chief Executive Officer John
Leonard, M.D. "Together with the previously reported data
from the polyneuropathy arm of this landmark study, these results
strongly suggest that NTLA-2001 could serve as a single-dose
treatment regardless of disease manifestation. At these deep and
consistent levels of protein reduction, we believe NTLA-2001 has
the potential to halt and even reverse the underlying cause of ATTR
amyloidosis. Given the similarly robust TTR reductions observed at
the two doses tested, we have selected a fixed dose comparable to
the 0.7 mg/kg level for evaluation across both arms in the ongoing
dose-expansion portion of the study. We look forward to completing
the Phase 1 study as we advance closer to a potential pivotal
trial, which we expect will include patients in the
U.S."
"We're encouraged to see profound and sustained serum TTR
reductions in people with cardiomyopathy manifestations of this
rare and fatal disease, further bolstering the prospects for a
one-time, in vivo treatment for multiple ATTR patient
groups," said George D. Yancopoulos,
M.D., Ph.D., President and Chief Scientific Officer of Regeneron.
"Intellia and Regeneron are working together diligently to advance
this potentially groundbreaking application of CRISPR technology,
which could one day be used for many different genetic
diseases."
At both dose levels, NTLA-2001 was generally well tolerated. Two
of 12 patients reported transient infusion reactions, which was the
only observed treatment-related adverse event. One patient in the
0.7 mg/kg dose NYHA Class III cohort experienced a Grade 3
infusion-related reaction which resolved without clinical
consequence. Per the study protocol, this group was subsequently
expanded from three to six patients to further characterize safety
at this dose level. No additional patients in the 0.7 mg/kg dose
NYHA Class III cohort reported a treatment-related adverse event.
No clinically significant liver findings were observed at either
dose level.
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
ATTR-CM or hereditary ATTR amyloidosis with polyneuropathy
(ATTRv-PN). A protocol amendment has been submitted to
evaluate a fixed dose corresponding to 0.7 mg/kg in the
dose-expansion portion, with enrollment across both
arms expected to be completed by the end of 2022, subject
to regulatory feedback.
NTLA-2002 Interim Clinical
Results
In a separate press release issued earlier today, Intellia
announced positive interim clinical data from an ongoing Phase 1/2
clinical study of NTLA-2002, its second in vivo genome
editing candidate, for the treatment of hereditary angioedema
(HAE). Please visit this link, or the Press Releases section of the
company's website at www.intelliatx.com.
Intellia Therapeutics Investor
Event and Webcast Information
Intellia will host a live webcast today, Friday, September 16, 2022, at 8:00 a.m. ET. 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 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.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that
invents life-transforming medicines for people with serious
diseases. Founded and led for nearly 35 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
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 ability to conduct and complete
clinical studies for NTLA-2001 for the treatment of transtherytin
amyloidosis (ATTR); its ability to generate data to demonstrate
NTLA-2001 as a potential single-dose treatment for ATTR; the
belief that NTLA-2001 can halt and potentially even reverse the
underlying cause of ATTR; its ability to develop its modular
platform and full-spectrum approach to advance its complex genome
editing capabilities, including to apply its proprietary
CRISPR/Cas9 technology platform to additional product candidates;
the advancement and expansion of its CRISPR/Cas9 technology to
develop human therapeutic products; its ability to maintain and
expand its related intellectual property portfolio, and avoid or
acquire rights to valid intellectual property of third parties; its
ability to demonstrate its platform's modularity and replicate or
apply results achieved in preclinical studies, including those in
its NTLA-2001 program, in any future studies, including human
clinical trials; its ability to develop other in vivo or
ex vivo cell therapeutics of all types, and NTLA-2001 in
particular, using CRISPR/Cas9 technology; and the timing of
regulatory filings and clinical trial execution, including
enrollment and dosing of patients.
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 the successful enrollment of patients in the Phase
1 study for NTLA-2001 for the treatment of ATTRv-PN or
ATTR-CM; risks related to Intellia's ability to
protect and maintain its intellectual property position; risks
related to the authorization, initiation and conduct of studies and
other development requirements, including manufacturing, for
its in vivo and ex vivo product candidates, including
NTLA-2001; the risk that any one or more of Intellia's product
candidates, including NTLA-2001, will not be successfully developed
and commercialized; the risk that the results of preclinical
studies or clinical studies, including for NTLA-2001, will not be
predictive of future results in connection with future studies; and
the risk that Intellia's will not be able to demonstrate its
platform's modularity and replicate or apply results achieved in
preclinical studies to develop additional product candidates,
including to apply its proprietary CRISPR/Cas9 technology platform
successfully to additional product candidates. 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 of 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 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 genome
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 studies discussed or referenced 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 (such as NTLA-2001) 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 June 30, 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 Contacts:
Investors:
Ian Karp
Senior Vice President, Investor Relations and Corporate
Communications
+1-857-449-4175
ian.karp@intelliatx.com
Lina Li
Senior Director, Investor Relations and Corporate
Communications
+1-857-706-1612
lina.li@intelliatx.com
Media:
Rebecca Spalding
Ten Bridge Communications
+1-646-509-3831
media@intelliatx.com
rebecca@tenbridgecommunications.com
Regeneron Contacts:
Investors:
Vesna
Tosic
+1-914-847-5443
vesna.tosic@regeneron.com
Media:
Alexandra
Bowie
+1-914-847-3407
alexandra.bowie@regeneron.com
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