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 interim data released today include 15 hereditary
ATTR amyloidosis with polyneuropathy (ATTRv-PN) patients treated
across four single-ascending dose cohorts. Single doses of 0.1
mg/kg, 0.3 mg/kg, 0.7 mg/kg, and 1.0 mg/kg of NTLA-2001 were
administered via intravenous infusion and changes from baseline
values of serum transthyretin (TTR) protein were measured for
each patient. Treatment with NTLA-2001 led to dose-dependent
reductions in serum TTR and achieved maximal reductions by day 28,
with mean reductions of 52%, 87%, and 86% among the three patients
each in the 0.1 mg/kg, 0.3 mg/kg, and 0.7 mg/kg dose groups,
respectively, and 93% among the six patients in the 1.0 mg/kg dose
group.
Mean serum TTR reduction remained durable through the
observation period, with patient follow-up ranging from two to 12
months. Additionally, the serum TTR reduction observed was
consistent across all patients receiving doses at or greater than
0.3 mg/kg. At the 1.0 mg/kg dose level, all six patients achieved
greater than 80% reduction and four of six patients achieved
greater than 90% reduction by day 28. Further, the reduction in
serum TTR observed at day 28 was sustained through the last
measured timepoint for each of the six patients, which ranged from
two to six months.
“Today’s update reinforces Intellia’s progress in opening a new
era of medicine. These data suggest that treatment with a one-time,
systemically delivered CRISPR-based investigational therapy has the
potential to substantially reduce levels of a disease-causing
protein. Data from the ongoing, first-in-human study of NTLA-2001
demonstrated rapid, deep and durable reduction of serum TTR
protein. At 1.0 mg/kg, the highest dose level studied, patients
with polyneuropathy reached a 93% mean serum TTR reduction by day
28. We believe this deep and consistent reduction shows promise for
halting and even reversing disease progression in people with ATTR
amyloidosis,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “The NTLA-2001 proof-of-concept further
validates our CRISPR technology platform and also supports the
continued development of our genome editing approaches for a
variety of diseases. Based on the safety and activity data
generated to date, we believe we have increased the probability of
success for our broader pipeline. Intellia looks forward to
advancing our second in vivo clinical candidate, NTLA-2002, for the
treatment of hereditary angioedema and additional in vivo
candidates in 2022 in our pursuit of harnessing the full potential
of genomic medicines.”
NTLA-2001 is the first CRISPR/Cas9-based therapy candidate to be
administered systemically for precision editing of a gene in
humans. It is designed to inactivate the TTR gene in
liver cells to reduce the production of misfolded TTR protein,
which accumulates in tissues throughout the body and causes the
debilitating and often fatal complications of ATTR amyloidosis.
“Our Intellia collaboration continues to move the tremendous
promise of genetics-based medicines closer to reality. Today’s data
provide further insights from the first pioneering clinical trial
in which CRISPR-based technology has been used to precisely edit a
disease-causing gene in humans, and the durability results support
the notion that this approach could one day be deployed for
long-lasting benefit,” said George D. Yancopoulos, M.D.,
Ph.D., President and Chief Scientific Officer of Regeneron.
“Regeneron has always thrived at the intersection of biology and
technology. We continue to expand both our research efforts through
the Regeneron Genetics Center and our toolkit of cutting-edge
technologies for genetics-based therapeutics, many of which are
being developed in collaboration with Intellia. Genetics medicine
holds enormous potential to treat, and potentially even cure, many
life-threatening diseases, and we look forward to advancing this
next chapter of medicine.”
At all four dose levels, NTLA-2001 was generally well tolerated.
The majority of adverse events were mild in severity with 73% (n =
11) of patients reporting a maximal adverse event severity of Grade
1. The most frequent adverse events included headache,
infusion-related reactions, back pain, rash, and nausea. There were
no clinically significant liver findings observed. There was a
single 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. Per the study protocol, the 1.0 mg/kg
dose group was subsequently expanded from three to six patients to
further characterize safety at this dose level. No additional
patients in the 1.0 mg/kg dose group reported a Grade 2 or higher
related adverse event. In addition, there was a single unrelated
serious adverse event of COVID-19 pneumonia reported in the 0.7
mg/kg dose group.
The Phase 1 study, run by Intellia as the program’s development
and commercialization lead, is evaluating NTLA-2001 in patients
with ATTRv-PN and ATTR amyloidosis with cardiomyopathy (ATTR-CM).
Part 2 of the Phase 1 study will be a single-dose ATTRv-PN
expansion cohort expected to begin in the first quarter of 2022. A
fixed dose of 80 mg, which is expected to deliver a similar
exposure to the 1.0 mg/kg dose, was selected for evaluation in Part
2 pending regulatory feedback. The transition from weight-based
dosing to fixed dosing is based on the safety, tolerability,
pharmacokinetic and activity profile of NTLA-2001 observed in Part
1 of the polyneuropathy arm. Patients also continue to be dosed
with NTLA-2001 in Part 1 of the cardiomyopathy arm at the 0.7 mg/kg
dose level, with plans to dose escalate to 1.0 mg/kg.
Intellia expects to complete enrollment of the Phase 1 study for
both ATTRv-PN and ATTR-CM subjects in 2022 and present additional
data at a medical meeting later this year. Intellia and Regeneron
plan to move towards pivotal studies for both forms of ATTR
amyloidosis, with an initial focus on the cardiomyopathy
manifestations of the disease.
“In this Phase 1 study, NTLA-2001 was generally well tolerated
as a single-dose treatment for ATTR amyloidosis patients with
polyneuropathy, resulting in deep and durable reductions of serum
TTR. These observations are consistent with animal data indicating
potential life-long serum TTR suppression,” said Ed Gane, M.D.,
Professor of Medicine at the University of Auckland, New Zealand
and Chief Hepatologist, Transplant Physician and Deputy Director of
the New Zealand Liver Transplant Unit at Auckland City Hospital,
and study investigator. “Importantly, these early results suggest
NTLA-2001 has the potential to deliver profound benefits for
patients around the world.”
Intellia Therapeutics Investor Event and Webcast
InformationIntellia will host a live webcast today,
Monday, February 28, 2022, at 4:30 p.m. ET to review today’s 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 ability of NTLA-2001 to halt and reverse disease
progression in people with ATTR amyloidosis, the expected timing of
data releases, regulatory filings, and the initiation and
completion of clinical trials; 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; 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 StatementsThis 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. 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 ContactsInvestors:Ian
KarpSenior Vice President, Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiDirector, Investor
Relations+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: Alexandra
Bowie+1-202-213-1643alexandra.bowie@regeneron.com
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