Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
positive interim results from an ongoing Phase 1/2 clinical study
of NTLA-2002, its second in vivo genome editing candidate.
NTLA-2002 is a systemically administered CRISPR candidate being
developed for hereditary angioedema (HAE) and is designed to knock
out the KLKB1 gene in liver cells, thereby reducing the production
of kallikrein protein. Uncontrolled activity of kallikrein is
responsible for the overproduction of bradykinin, which leads to
the recurring, debilitating and potentially fatal swelling attacks
that occur in people living with HAE. The interim data were shared
today in an oral presentation at the 2022 Bradykinin Symposium held
in Berlin, Germany.
The data presented are from the initial six adult patients with
HAE in the ongoing dose-escalation study with a data cut-off date
of July 27, 2022. Single doses of 25 mg (n=3) 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. Administration of NTLA-2002 led to dose-dependent
reductions in plasma kallikrein and achieved maximal reductions by
week eight, with mean reductions of 65% and 92% in the 25 mg and 75
mg dose cohorts, respectively. Furthermore, these reductions were
sustained through at least 16 weeks in the 25 mg cohort and eight
weeks in the 75 mg cohort for which complete cohort biomarker data
were available.
In addition to plasma kallikrein levels, HAE attack rates are
also being measured in the study, with the first analysis occurring
at the end of the pre-specified 16-week primary observation period.
To date, all three patients in the 25 mg dose cohort have reached
the end of this initial observation period. Patients in this group
had a baseline HAE attack rate ranging from 1.1 to 7.2 attacks per
month, as confirmed by the investigator. Treatment with a single
dose of 25 mg of NTLA-2002 resulted in a mean reduction in HAE
attacks of 91% throughout the 16-week observation period.
Additionally, two of the three patients have not had a single HAE
attack since treatment, and all three patients have been attack
free since week 10 (follow-up through weeks 24 - 32). Patients in
the 75 mg cohort have not completed the primary 16-week observation
period. Attack-rate data for this cohort will be presented at the
American College of Allergy, Asthma & Immunology (ACAAI) Annual
Scientific Meeting, November 10 – 14 in Louisville, Kentucky.
Prophylaxis medications are permitted in the Phase 1 part of the
study. Two of the three patients in the 25 mg cohort were actively
receiving prophylaxis therapy prior to administration of NTLA-2002.
For these two patients, the study protocol permitted investigators
to withdraw the patient’s prophylaxis therapy after completion of
the 16-week primary observation period. This treatment approach was
implemented for the two applicable patients in this cohort, and
neither patient has had an HAE attack since discontinuing their
prophylaxis therapy through the latest follow-up.
“These initial data represent a significant milestone for both
Intellia and people around the world suffering from genetic
diseases, such as HAE,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “We are strongly encouraged by the
greater than 90% reduction in HAE attacks observed in the 25 mg
dose cohort, as these interim results support our belief that a
single dose of NTLA-2002 has the potential to permanently prevent
the debilitating swelling attacks associated with HAE.
Additionally, today’s announcement continues to validate our genome
editing approach and the modular platform we have built. This is
now the second time in history clinical data have been generated
suggesting we can precisely edit target cells within the human body
to potentially treat genetic diseases with a single, systemic
administration of a CRISPR-based therapy. We plan to move as
quickly and judiciously as possible on behalf of people living with
HAE and a number of additional genetic diseases in the months and
years ahead.” At both 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,
including any significant elevation in liver enzymes.
“Many people living with HAE continue to experience breakthrough
attacks despite currently available treatments and often find the
burden of untreated attacks, frequent infusions or injections to be
tremendously disruptive to their lives,” said Hilary Longhurst,
M.D., Ph.D., Faculty of Medical and Health Sciences, University of
Auckland, New Zealand, and the trial’s principal investigator in
New Zealand. “These early data support NTLA-2002 as a potential
one-time treatment capable of producing profound reductions in HAE
attacks. While the clinical data are still emerging, I am highly
optimistic that NTLA-2002 could become a new treatment option for
the HAE community.”
Based on the interim data presented today, Intellia selected a
third dose of 50 mg to be evaluated in the ongoing dose-escalation
portion of the Phase 1/2 study. Dosing at this level has recently
completed, and Intellia expects to select up to two doses to
further evaluate 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 today,
Friday, September 16, 2022, at 8:00 a.m. ET, to provide a clinical
update from its in vivo portfolio, during which the company will
review the presented clinical data at the 2022 Bradykinin Symposium
alongside interim results from NTLA-2001. 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 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.
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 including
permanently preventing debilitating swelling attacks; its ability
to develop its modular platform and full-spectrum approach to
advance its complex genome editing capabilities, including to apply
its proprietary cell engineering 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,
including those in its NTLA-2002 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-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’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 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: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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