Intellia Therapeutics, Inc. (NASDAQ:NTLA) today announced
that a late-breaking abstract featuring interim Phase 1 clinical
data from NTLA-2001, the Company’s lead CRISPR/Cas9 in vivo therapy
in development as a single-dose, systemically administered
treatment for transthyretin (ATTR) amyloidosis, has been selected
for an oral presentation at the 2021 Peripheral Nerve Society (PNS)
Annual Meeting, taking place this month.
The presentation will include interim data from the ongoing
dose-escalation portion of Intellia’s Phase 1 trial evaluating
NTLA-2001 in people living with hereditary ATTR amyloidosis with
polyneuropathy (hATTR-PN). Following safety assessment and dose
optimization, Intellia intends to further evaluate NTLA-2001 in a
broader population of people with ATTR amyloidosis, including those
with cardiomyopathy.
“These findings represent Intellia’s first clinical data readout
and an important step forward in our commitment to develop
breakthrough genome editing treatments for people living with
severe diseases, such as transthyretin (ATTR) amyloidosis,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“By knocking out the disease-causing gene, NTLA-2001 is designed to
halt progression and potentially reverse the disease with a single
dose, offering the potential of meaningful improvement over the
standard of care, which requires chronic, lifelong administration.
These interim data will provide a view of NTLA-2001’s safety and
activity profile as the dose-escalation portion of our study
progresses, as well as insight into the promise of our modular
platform to develop other systemically delivered in vivo
CRISPR/Cas9 therapies across a range of diseases with unmet
need.”
Presentation DetailsTitle: “In
vivo CRISPR/Cas9 Editing of the TTR Gene by NTLA-2001 in Patients
with Transthyretin Amyloidosis”Session: Platform
Session II Date and Time: Saturday, June 26, 2021
from 11:15-11:30 a.m. E.T.Presenter: Dr. Julian
Gillmore, M.D, Ph.D., FRCP, FRCPath, Professor of Medicine,
National Amyloidosis Centre, UCL Division of
Medicine, Royal Free Hospital, U.K., the trial’s national
coordinating investigator
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 the NTLA-2001 Clinical
ProgramIntellia’s global Phase 1 trial is an open-label,
multi-center, two-part study of NTLA-2001 in adults with hereditary
transthyretin amyloidosis with polyneuropathy (hATTR-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 in Part 1 and, following
the identification of an optimal dose, an expansion cohort in Part
2. Visit clinicaltrials.gov (NCT04601051) for more
details.
Enrollment is ongoing at global clinical trial sites, as
Intellia is submitting additional regulatory applications in other
countries as part of its development strategy. After completion of
the Phase 1 trial, the company is planning to rapidly move to
pivotal studies for both polyneuropathy and cardiomyopathy
patients.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first curative treatment for ATTR. NTLA-2001 is the first
experimental CRISPR therapy candidate to be administered
systemically, or through a vein, to edit genes inside the human
body. Intellia’s proprietary non-viral platform utilizes lipid
nanoparticles designed to deliver to the liver a simple,
two-part genome editing system: guide RNA specific
to the disease-causing gene and messenger RNA that encodes the
Cas9 protein. Our robust preclinical data showing deep and
long-lasting transthyretin (TTR) reduction following knockout of
the target gene in vivo support NTLA-2001’s potential as
a single-administration therapeutic. NTLA-2001 is part of a
co-development/co-promotion agreement between Intellia, the lead
development and commercialization party, and Regeneron
Pharmaceuticals, Inc.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR, is a rare, progressive and
fatal disease. Hereditary ATTR (hATTR) amyloidosis occurs when a
person is born with DNA mutations in the TTR gene, which
causes the liver to produce transthyretin (TTR) protein in a
misfolded form and build up in the body. hATTR amyloidosis can
predominantly manifest as polyneuropathy (hATTR-PN), which can lead
to nerve damage, and cardiomyopathy (hATTR-CM), which involves
heart muscle disease that can lead to heart failure. In addition,
non-mutated, or wild-type TTR protein, can also accumulate in the
body, leading to wild-type ATTR (wtATTR) amyloidosis. There are an
estimated 50,000 hATTR patients worldwide and between 200,000 and
500,000 people with wtATTR.
About Intellia TherapeuticsIntellia
Therapeutics is a leading clinical-stage genome editing company,
focused on the development of proprietary, potentially curative
therapeutics using the CRISPR/Cas9 system. Intellia believes the
CRISPR/Cas9 technology has the potential to transform medicine by
both producing therapeutics that permanently edit and/or correct
disease-associated genes in the human body with a single
administration, and creating enhanced engineered cells that can
treat oncological and immunological diseases. Intellia’s
combination of deep scientific, technical and clinical development
experience, along with its leading intellectual property portfolio,
puts it in a unique position to unlock broad therapeutic
applications of the CRISPR/Cas9 technology and create new classes
of therapeutic products. Learn more about Intellia and CRISPR/Cas9
at intelliatx.com. Follow us on Twitter @intelliatweets.
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
(“ATTR”) amyloidosis, 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 and expansion of our
CRISPR/Cas9 technology 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.
Intellia Contacts:
Investors:Lina LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:
Julie Ferguson (Interim) Head of External Affairs &
Communications+1-312-385-0098julie.ferguson@intelliatx.commedia@intelliatx.com
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