Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapies leveraging CRISPR-based technologies, today presented
additional interim results from its ongoing Phase 1 study of
NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing
therapy in development as a single-dose treatment for transthyretin
(ATTR) amyloidosis. Results were presented in an oral presentation
at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in
Madrid, Spain.
“With 65 patients reported from the Phase 1 study, this update
represents the largest clinical dataset for an in vivo CRISPR-based
investigational therapy. These positive interim results add to the
growing body of data that demonstrates deep and durable reductions
of serum TTR after a single dose of NTLA-2001. The consistent and
profound levels of reduction in all patients bolster our confidence
that NTLA-2001 could potentially reset the standard of care for
ATTR amyloidosis — both for treating the disease and how response
is evaluated,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “We have also observed early signals of clinical
activity in the initial cohorts and look forward to presenting the
first clinical data beyond serum TTR levels once we have longer
follow-up across all cohorts."
The Phase 1 trial is a two-part study evaluating NTLA-2001 in
patients with either ATTR amyloidosis with cardiomyopathy (ATTR-CM)
or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The
data presented today, with a cutoff date of May 11, 2023, are from
the initial 65 out of 72 patients dosed in the Phase 1 study, which
has now completed enrollment. The results from the final seven
patients dosed, who were enrolled after the data cutoff, will be
reported at a future date.
In the newly reported dose-expansion portion, administration of
NTLA-2001 at the 55 mg and 80 mg dose led to deep serum TTR
reductions consistent with the results previously reported from
patients in the dose-escalation portion who received the
corresponding weight-based dose, 0.7 mg/kg and 1.0 mg/kg,
respectively.
Across all patients who received a dose of 0.3 mg/kg or higher
(n=62), the median serum TTR reduction was 91% and the median
absolute residual serum TTR concentration was 17 µg/mL at day 28.
The persistently low levels of TTR concentration are expected to
reduce the rate of ongoing amyloid formation and hold the
possibility for amyloid clearance to reverse the symptoms of the
disease. If clinically validated, the use of absolute residual TTR
concentration levels could become a new benchmark for evaluating
ATTR amyloidosis.
The reduction of serum TTR compared to baseline was sustained
through the latest follow-up. With 29 patients now reaching at
least 12 months of follow-up, all patients continued to show a
long-lasting response with no evidence of loss in activity over
time.
NTLA-2001 was generally well tolerated across all patients and
at all dose levels tested. The most commonly reported adverse
events were infusion-related reactions, which occurred in 38% of
patients. The majority of adverse events, including
infusion-related reactions, were Grade 1 or 2 in severity,
transient and resolved spontaneously. Other adverse events that
were reported in greater than 10% of patients included headache,
diarrhea and back pain, and were all Grade 1 or 2. All patients
received a full dose of NTLA-2001 and remain on study. No
dose-limiting toxicities were observed. Based on the safety and
activity of NTLA-2001, the 55mg dose has now been selected to be
evaluated in the upcoming pivotal Phase 3 study.
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 in people living with ATTR
amyloidosis. As previously announced, Intellia recently received
IND clearance from the FDA to begin a Phase 3 trial of NTLA-2001
for ATTR-CM and expects to initiate the global pivotal trial by the
end of this year. Additionally, the Company is actively preparing
for a Phase 3 trial for ATTRv-PN.
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 and clinical
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). The Phase 1 trial is now closed for enrollment.
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 X (formerly known as Twitter) @intelliatx.
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: the safety, efficacy and advancement of its clinical
program for NTLA-2001 for the treatment of transthyretin (“ATTR”)
amyloidosis pursuant to its clinical trial applications (“CTA”) and
investigational new drug (“IND”) submissions, including the
expected timing of data releases, regulatory filings, and the
initiation and completion of clinical trials such as its ability to
initiate a global pivotal Phase 3 trial by year-end; its ability to
generate data to demonstrate NTLA-2001 as a potential single-dose
treatment for ATTR amyloidosis to permanently inactivate the TTR
gene and reduce the disease-causing protein in people living with
ATTR amyloidosis; its beliefs concerning observed early signals of
clinical activity in the initial cohorts; its belief that NTLA-2001
may reduce the rate of ongoing amyloid formation, reverse the
symptoms of ATTR amyloidosis, including possible amyloid clearance,
and reset the standard of care for ATTR amyloidosis; its belief
that the use of absolute residual TTR concentration levels could
become a new benchmark for evaluating ATTR amyloidosis, if
clinically validated; 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; 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 and
clinical studies, including those in its NTLA-2001 program, in any
future studies, including human clinical trials; 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 initiation and conduct of a global pivotal Phase 3
study for NTLA-2001 for the treatment of ATTR-CM and that the
results of such Phase 3 study may not be positive; 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 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 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 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. 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:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communicationsian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.commcrenson@tenbridgecommunications.com
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