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
InformationIntellia 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-2001Based 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 TherapeuticsIntellia
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
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 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 Forward-Looking Statements and Use of Digital
MediaThis 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 Contacts
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiDirector, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Matt Crenson Ten Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
Regeneron Contacts
Investors: Vesna
Tosic+1-914-847-5443vesna.tosic@regeneron.com
Media: Ella Campbell
+1-914-847-7017ella.campbell@regeneron.com
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