Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing potentially curative therapies
leveraging CRISPR-based technologies, today announced that interim
clinical data from the Phase 1 study of NTLA-2001, an
investigational therapy in development for the treatment of
transthyretin (ATTR) amyloidosis, will be shared in an oral
presentation at the European Association for the Study of the
Liver’s International Liver Congress™ 2022, taking place June 22 –
26 in London.
“We are pleased to be presenting longer-term, follow-up data
from the first-in-human study of NTLA-2001, which is designed to
potentially halt progression and reverse ATTR amyloidosis,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“We look forward to sharing interim data we believe demonstrates
the potential of our CRISPR-based investigational therapy to be a
single-dose treatment that provides a deep and durable
response.”
The presentation will include new data from the ongoing Phase 1
study evaluating NTLA-2001 in people living with hereditary ATTR
amyloidosis with polyneuropathy (ATTRv-PN). These include
additional results from all four dose-escalation cohorts in Part 1,
highlighting the durability of response after a single dose of
NTLA-2001. Data supporting the fixed dose selection for Part 2, the
single-dose expansion cohort of the polyneuropathy arm, will also
be presented.
The presentation will build upon interim data presented in
February, which showed significant reductions in serum TTR levels
from baseline in a dose-dependent manner in 15 patients with
ATTRv-PN. At the highest dose tested, 1.0 mg/kg, the mean and
maximum serum TTR reductions were 93% and 98%, respectively, by day
28 across the six patients treated. The Company also previously
announced it is now evaluating a fixed dose of 80 mg in Part 2 of
the Phase 1 study, which is expected to deliver a similar exposure
to the 1.0 mg/kg dose in the ATTRv-PN target population, and that
the first patient has been dosed in the expansion cohort in the
polyneuropathy arm. Intellia also continues to dose patients in the
cardiomyopathy arm of the Phase 1 study, which is evaluating
NTLA-2001 in dose-escalation cohorts of people with ATTR
amyloidosis with cardiomyopathy (ATTR-CM).
Presentation Details
Title: In vivo CRISPR/Cas9 editing of the TTR
gene with NTLA-2001 in patients with transthyretin amyloidosis –
dose selection considerations
Session: Rare liver diseases (including
paediatric and genetic)
Date and Time: Friday, June 24, 2022 from
10:00-11:30 a.m. BST
Presenter: Dr. Edward J. Gane, MBChB, MD,
FRACP, MNZM, Professor of Medicine at the University of Auckland,
New Zealand and Chief Hepatologist, Transplant Physician and Deputy
Director of the New Zealand Liver Transplant Unit at Auckland City
Hospital, the trial’s national investigator in New Zealand
Intellia Therapeutics Investor Event and Webcast
Information
Intellia will host a live webcast on Friday, June 24, 2022 at
8:00 a.m. ET 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 Transthyretin (ATTR)
AmyloidosisTransthyretin 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 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 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
concerning: its ability to successfully extend its leadership
position and harness the full potential of genomic medicines to
bolster its genome editing capabilities and pipeline; the safety,
efficacy, success and advancement of its clinical programs for
NTLA-2001 for the treatment of transthyretin amyloidosis pursuant
to its clinical trial applications (“CTA”), including the expected
timing of data releases, regulatory filings, and the initiation,
enrollment, and completion of clinical trials; the expansion of its
CRISPR/Cas9 technology and related technologies to advance
additional development candidates and timing expectations of
advancing such development candidates; its ability to maintain and
expand its related intellectual property portfolio; expectations of
the potential impact of the coronavirus disease pandemic, including
the impact of any variants, on strategy, future operations and
timing of its clinical trials; its ability to optimize the impact
of its collaborations on its development programs, including, but
not limited to, its collaboration with Regeneron Pharmaceuticals,
Inc.
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 Intellia’s ability to protect and maintain its
intellectual property position; risks related to Intellia’s
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to the authorization, initiation and conduct of studies and
other development requirements for its product candidates,
including uncertainties related to regulatory approvals to conduct
clinical trials; the risk that any one or more of Intellia’s
product candidates 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; the risk that clinical study
results will not be positive; and the risk that Intellia’s
collaborations with Regeneron or its other 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, 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
LiDirector, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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