Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
updated data from an ongoing Phase 1/2 clinical study of NTLA-2002
for the treatment of hereditary angioedema (HAE). The interim
analyses were shared today in a Distinguished Industry Abstract
oral presentation at the American College of Allergy, Asthma &
Immunology (ACAAI) 2022 Annual Scientific Meeting, being held
November 10 – 14 in Louisville, Kentucky.
The data presented are from 10 adult patients with HAE in the
Phase 1, dose-escalation portion of the study, with a data cut-off
date of September 28, 2022. Single doses of 25 mg (n=3), 50 mg
(n=4) and 75 mg (n=3) of NTLA-2002 were administered via
intravenous infusion, and changes from baseline values of plasma
kallikrein protein were measured for each patient.
Plasma Kallikrein Reduction Administration of
NTLA-2002 led to deep, dose-dependent reductions in plasma
kallikrein as described below, based on complete cohort biomarker
data availability. For the 25 mg and 75 mg cohorts, these deep
reductions in plasma kallikrein were sustained through the
observation period, which ranged from week 16 to week 32.
Cohort |
Mean plasma kallikrein reduction at latest
follow-up |
25 mg (n=3) |
64% (week 32) |
50 mg (n=4) |
81% (day 22) |
75 mg (n=3) |
92% (week 16) |
HAE Attack Rate Reduction HAE attack rates are
measured in the dose-escalation portion of the study, with the
first analysis occurring at the end of the pre-specified 16-week
primary observation period. To date, all patients in the 25 mg and
75 mg dose cohorts have reached the end of this initial observation
period in ongoing follow-up as described below. Patients in the 50
mg cohort have not completed the primary 16-week observation
period.
Cohort |
Baseline attack rate in screening period |
Mean HAE attack rate reduction - week 1 to 16 |
Mean HAE attack rate reduction - week 5 to 16 |
Duration of ongoing attack-free interval |
25 mg (n=3) |
1.1 to 7.2 attacks / month |
91% |
89% |
5.5 – 10.6 months |
75 mg (n=3) |
4.0 to 5.9 attacks / month |
78% |
89% |
2.3 – 4.2 months |
“We see early evidence that our one-time CRISPR-based
investigational therapy may offer patients suffering from
hereditary angioedema a functional cure for their disease,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“Based on the extended data across multiple dose cohorts, we are
strongly encouraged that all patients who received a single dose of
NTLA-2002 subsequently became attack-free. In the patients with the
longest follow-up to date, their attack-free interval has been
maintained 5 to 10 months from their last attack. Importantly, the
safety data from all 10 patients are highly encouraging, further
supporting NTLA-2002’s potential to change the future HAE treatment
paradigm. As the second clinical program from our in vivo pipeline
to demonstrate deep and consistent protein reduction following a
one-time administration, the latest interim data further reinforce
the enormous potential of our modular CRISPR genome editing
platform to treat a host of genetic diseases.”
At all three dose levels, NTLA-2002 was generally
well-tolerated, and the majority of adverse events were mild in
severity. The most frequent adverse events were infusion-related
reactions, which were mostly Grade 1 and resolved within one day.
There have been no dose-limiting toxicities, no serious adverse
events and no adverse events of Grade 3 or higher observed to date.
No clinically significant laboratory abnormalities were
observed.
Intellia expects to select up to two doses to further evaluate
NTLA-2002 in the Phase 2, placebo-controlled, dose-expansion
portion of the study, which is expected to begin in the first half
of 2023. Intellia anticipates expanding country and site
participation, including U.S. clinical sites, as part of the Phase
2 study.
Intellia Therapeutics Investor Event and Webcast
Information Intellia will host a live webcast, Monday,
November 14, 2022, at 8:00 a.m. ET, to review the interim results
from NTLA-2002. 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-2002 Clinical ProgramIntellia’s
multi-national Phase 1/2 study is evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in
adults with Type I or Type II hereditary angioedema (HAE). This
includes the measurement of plasma kallikrein protein levels and
activity as determined by HAE attack rate measures. The Phase 1
portion of the study is an open-label, single-ascending dose design
used to identify up to two dose levels of NTLA-2002 that will be
further evaluated in the randomized, placebo-controlled Phase 2
portion of the study. This Phase 1/2 study will identify the dose
of NTLA-2002 for use in future studies. Visit
clinicaltrials.gov (NCT05120830) for more details.
About NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002
is the first single-dose investigational treatment being explored
in clinical trials for the potential to continuously reduce
kallikrein activity and prevent attacks in people living with
hereditary angioedema (HAE). NTLA-2002 is a wholly owned
investigational CRISPR therapeutic candidate designed to inactivate
the kallikrein B1 (KLKB1) gene, which encodes for
prekallikrein, the kallikrein precursor protein. NTLA-2002 is
Intellia’s second investigational CRISPR therapeutic candidate
to be administered systemically, by intravenous infusion, to edit
disease-causing genes inside the human body with a single dose of
treatment. 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
together carry out the precision editing.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disorder
characterized by severe, recurring and unpredictable inflammatory
attacks in various organs and tissues of the body, which can be
painful, debilitating and life-threatening. It is estimated that
one in 50,000 people are affected by HAE, and current treatment
options often include life-long therapies, which may require
chronic intravenous (IV) or subcutaneous (SC) administration as
often as twice per week, or daily oral administration to ensure
constant pathway suppression for disease control. Despite chronic
administration, breakthrough attacks still occur. Kallikrein
inhibition is a clinically validated strategy for the preventive
treatment of HAE attacks.
About Intellia Therapeutics
Intellia 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.
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-2002 for the treatment of hereditary
angioedema (“HAE”); its ability to generate data to demonstrate
NTLA-2002 as a potential single-dose treatment for HAE, including
safety, kallikrein reduction and attack rate data; its belief that
NTLA-2002 may offer patients suffering from HAE a functional cure
for their disease; its expectation to begin the Phase 2
dose-expansion of the study in the first half of 2023, and its
expectation to expand country and site participation, including
U.S. clinical sites, as part of the Phase 2 study; its ability to
develop its modular CRISPR genome editing platform to treat a host
of genetic diseases; 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 and clinical studies, including those in its NTLA-2002
program, in any future studies, including human clinical trials
evaluating treatments for other genetic diseases; its ability to
develop other in vivo or ex vivo cell therapeutics of all types,
and NTLA-2002 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 1/2
study for NTLA-2002 for the treatment of HAE; 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-2002; the risk that any one or more of
Intellia’s product candidates, including NTLA-2002, will not be
successfully developed and commercialized; the risk that the
results of preclinical studies or clinical studies, including for
NTLA-2002, 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
to treat other genetic diseases. 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 filed with the U.S.
Securities and Exchange Commission (“SEC”), as well as discussions
of potential risks, uncertainties and other important factors in
Intellia’s other filings with the 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.com
Lina 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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