Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis
21 Octobre 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex vivo, announced
today that the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation to NTLA-2001 for the treatment of
transthyretin (ATTR) amyloidosis. This investigational therapy is
the first CRISPR therapy to be administered systemically to edit a
disease-causing gene inside the human body. NTLA-2001 has the
potential to be the first single-dose treatment for ATTR
amyloidosis as it may be able to halt and reverse the devastating
complications of this disease. ATTR amyloidosis is a rare condition
that can impact a number of organs and tissues within the body
through the accumulation of misfolded transthyretin (TTR) protein
deposits.
“Orphan drug designation underscores the FDA’s recognition of
NTLA-2001’s potential promise as a single-dose, novel therapy for
the treatment of ATTR amyloidosis,” said Intellia President and
Chief Executive Officer John Leonard, M.D. “At Intellia, we are
committed to advancing our modular genome editing platform to
develop potentially curative treatment options for life-threatening
diseases, and we look forward to working with the ATTR amyloidosis
community and the FDA to bring a much-needed treatment option to
patients.”
NTLA-2001 is currently being studied in a Phase 1 trial in
adults with hereditary ATTR amyloidosis with polyneuropathy
(ATTRv-PN). In June 2021, Intellia and its collaborator Regeneron
announced positive interim clinical results from the first two
cohorts of this study. These results, which were published in the
New England Journal of Medicine, represented the first-ever
clinical data supporting the safety and efficacy of in vivo CRISPR
genome editing in humans.
The FDA's Orphan Drug Designation program provides orphan status
to drugs defined as those intended for the treatment, diagnosis or
prevention of rare diseases that affect fewer than 200,000 people
in the United States. Orphan drug designation qualifies the sponsor
of the drug for certain development incentives, including tax
credits for qualified clinical testing, prescription drug user fee
exemptions and seven-year marketing exclusivity upon FDA approval.
The decision by the FDA follows a March 2021 decision by the
European Commission (EC) to also grant NTLA-2001 orphan drug
designation for the treatment of ATTR amyloidosis.
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 deposits 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 any 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.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first curative treatment for ATTR amyloidosis. NTLA-2001 is
the first investigational CRISPR therapy candidate to be
administered systemically, or intravenously, 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. Interim Phase 1
clinical data released in June 2021 confirm substantial,
dose-dependent reduction of TTR protein following a single dose of
NTLA-2001. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics using
CRISPR/Cas9 technology. To fully realize the transformative
potential of CRISPR/Cas9, 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 CRISPR/Cas9 to create new classes of genetic medicine. Learn
more at intelliatx.com. Follow us on
Twitter @intelliatweets.
Forward-Looking StatementsThis 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: being able to complete clinical studies for
NTLA-2001 for the treatment of transthyretin (“ATTR”) amyloidosis
pursuant to its clinical trial applications (“CTA”), including
submitting additional regulatory applications in other countries;
ability to demonstrate effectiveness of NTLA-2001 in treating or
reversing ATTR amyloidosis in patients; advancement and expansion
of its CRISPR/Cas9 technology to develop human therapeutic
products, as well as its ability to maintain and expand its related
intellectual property portfolio; expectations of the potential
impact of the coronavirus disease 2019 pandemic on strategy, future
operations and timing of its clinical trials or IND submissions;
ability to optimize the impact of its collaborations on its
development programs, including but not limited to its
collaborations with Regeneron, including its co-development
programs for ATTR amyloidosis; and statements regarding the timing
of regulatory filings regarding its development programs.
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; 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; 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.com Lina
LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Lisa QuTen Bridge
Communications+1-678-662-9166media@intelliatx.com
lqu@tenbridgecommunications.com
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