Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today presented
additional 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 in collaboration with Regeneron
Pharmaceuticals. Results were presented in a Late-Breaking Science
oral presentation at the American Heart Association (AHA)
Scientific Sessions 2022, held November 5 – 7 in Chicago, Illinois.
The interim data from the dose-escalation
portion of the Phase 1 study include 12 adult patients with ATTR
amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart
Association (NYHA) Class I – III heart failure. The data presented
were as of a data cutoff date of August 25, 2022. Single doses of
0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via a single
intravenous infusion, and the change from baseline in serum
transthyretin (TTR) protein concentration was measured for each
patient.
Administration of NTLA-2001 led to deep and durable 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
dose level (n=9) was 93% (91%, 97%).
These deep reductions in serum TTR were
sustained through the observation period, with patient follow-up
ranging from four to six months. These data highlight 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.
“This presentation at the AHA Scientific
Sessions marks the first time Intellia has had the opportunity to
share interim data from our landmark clinical trial of NTLA-2001
for the treatment of ATTR amyloidosis with the cardiology
community,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “We believe a single dose of NTLA-2001 has the
potential to halt and potentially reverse this life-threatening
disease. With the depth and consistency of TTR protein reduction
seen with NTLA-2001 thus far, we envision a future where physicians
and patients can make treatment decisions based on a therapy’s
ability to achieve specific threshold protein levels. We look
forward to progressing this first-ever systemically administered in
vivo CRISPR investigational therapy toward completion of the Phase
1 study and subsequent pivotal studies in collaboration with our
partners at Regeneron."
At both dose levels, NTLA-2001 was generally
well tolerated. As previously reported, two of 12 patients reported
transient infusion reactions, which were the only observed
treatment-related adverse events. One patient in the 0.7 mg/kg dose
NYHA Class III cohort experienced a Grade 3 infusion-related
reaction, which resolved without clinical sequalae. 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 laboratory abnormalities 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). Dosing at 55 mg, the fixed dose
corresponding to 0.7 mg/kg, is ongoing in Part 2, the
dose-expansion portion of the study. Intellia remains on track to
complete, by the end of this year, planned enrollment of both arms
of the Phase 1 study that will inform the dose selection for
subsequent pivotal studies.
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.
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 transthyretin 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 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 and clinical 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.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.comLina LiSenior
Director, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
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