Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced
preliminary, positive safety and efficacy results from the first
patient (<2 years of age) dosed in the Phase 1/2 CHORD trial
investigating otoferlin gene therapy (DB-OTO) in children with
profound genetic hearing loss due to mutations of the otoferlin
gene.
“The children who are being enrolled in CHORD are often born
with profound hearing loss due to mutations in a single gene,
otoferlin, which essentially turns off their auditory circuits,”
said Professor Manohar Bance, M.B., an ear surgeon and principal
trial investigator at Cambridge University Hospitals NHS Foundation
Trust in the United Kingdom. “Cochlear implants are the current
standard of care but are unable to replicate the full complexity
and range of sound. With these very preliminary DB-OTO results, we
now have encouraging evidence that this gene therapy may be able to
help turn these auditory circuits back on. We look forward to
following this child and others further to determine if DB-OTO gene
therapy can restore clinically meaningful hearing as they are
learning to interact with the world.”
In the trial, the child received an intracochlear injection of
DB-OTO in one ear. At planned follow-ups, the child experienced
improvements in auditory responses through week 6 compared to
baseline, per auditory brainstem response (ABR) and behavioral
(pure tone) audiometry. ABR, a clinically accepted physiologic
measure of hearing sensitivity, is often absent in those with
classic otoferlin-related hearing loss and was absent in both ears
of the child at baseline. There were no concerning safety signals
through week 6 following treatment.
Congenital hearing loss (hearing loss present at birth) is a
significant unmet medical need with no approved pharmacologic
treatment options that affects approximately 1.7 out of every 1,000
children born in the U.S. While hearing loss caused by mutations of
the otoferlin gene is ultra-rare, the majority of permanent,
congenital hearing loss cases diagnosed in developed countries are
sensorineural and result from a single gene defect, making them
suitable targets for gene therapy.
“These preliminary DB-OTO results provide early and encouraging
proof-of-concept for the treatment of otoferlin-related hearing
loss, as well as our pipeline of gene therapies to address more
common forms of genetic hearing loss and other therapeutic areas,”
said Christos Kyratsous, Ph.D., Senior Vice President of Research
and co-head of Genetic Medicines at Regeneron. “The ongoing CHORD
trial is our first clinical-stage auditory program, and we are
incredibly grateful to the investigators and the family of this
child for embarking on this breakthrough trial. We remain committed
to advancing this research and hope these results mean that
children with genetic hearing loss will eventually be able to
benefit from the revolutionary promise of gene therapies like
DB-OTO.”
DB-OTO was originally developed under a collaboration between
Regeneron and Decibel Therapeutics that was initially established
in 2017, with an extension announced in 2021. In September 2023,
Regeneron acquired Decibel Therapeutics, cementing this
long-standing collaboration. In addition to the DB-OTO development
program, other clinical efforts include AAV.103 for people with
GJB2-related hearing loss and AAV.104 for people with stereocilin
(STRC)-related hearing loss.
The potential use of DB-OTO for otoferlin-related hearing loss
is currently under clinical development, and its safety and
efficacy have not been evaluated by any regulatory authority.
About the CHORD TrialThe CHORD trial (NCT#
05788536) is a Phase 1/2 first-in-human, multicenter, open-label
trial to evaluate the safety, tolerability, and preliminary
efficacy of DB-OTO in pediatric patients with otoferlin
mutations.
Currently enrolling children across sites in the U.S., United
Kingdom and Spain (<18 years of age; staggered by age in the
U.S.), CHORD is being conducted in two parts. In the initial
dose-escalation cohort (Part A), patients will receive a single
intracochlear injection of DB-OTO in one ear, while in expansion
cohort (Part B), patients will receive single intracochlear
injections of DB-OTO in both ears at the selected dose from Part
A.
Additional information about the trial, including enrollment,
can be obtained by contacting clinicaltrials@decibeltx.com or
1-617-370-8701.
About DB-OTODB-OTO is an investigational
cell-selective, adeno-associated virus (AAV) gene therapy designed
to provide durable, physiological hearing to individuals with
profound, congenital hearing loss caused by mutations of the
otoferlin gene. The treatment aims to deliver a working copy of the
faulty otoferlin gene using a modified, non-pathogenic virus that
is delivered via an injection into the cochlea under general
anesthesia (similar to the procedure used for cochlear
implantation). In this gene therapy, the introduced otoferlin gene
is under the control of a proprietary cell-specific Myo15 promoter,
which is intended to restrict expression only to the cells that
normally express otoferlin.
DB-OTO received Orphan Drug and Rare Pediatric Disease
designations from the U.S. Food and Drug Administration in 2021. In
the European Union, Orphan Drug Designation was granted by the
European Medicines Agency in 2023.
About RegeneronRegeneron (NASDAQ: REGN) is a
leading biotechnology company that invents, develops and
commercializes life-transforming medicines for people with serious
diseases. Founded and led for 35 years by physician-scientists, our
unique ability to repeatedly and consistently translate science
into medicine has led to numerous FDA-approved treatments and
product candidates in development, almost all of which were
homegrown in our laboratories. Regeneron’s medicines and pipeline
are designed to help patients with eye diseases, allergic and
inflammatory diseases, cancer, cardiovascular and metabolic
diseases, hematologic conditions, infectious diseases and rare
diseases.
Regeneron is accelerating and improving the traditional drug
development process through its
proprietary VelociSuite® technologies, such
as VelocImmune®, which uses unique genetically humanized mice
to produce optimized fully human antibodies and bispecific
antibodies, and through ambitious research initiatives such as the
Regeneron Genetics Center®, which is conducting one of the largest
genetics sequencing efforts in the world.
For additional information about Regeneron, please
visit www.regeneron.com or follow Regeneron
on LinkedIn.
Forward-Looking Statements and Use of Digital
MediaThis press release includes forward-looking
statements that involve risks and uncertainties relating to future
events and the future performance of Regeneron
Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and
actual events or results may differ materially from these
forward-looking statements. Words such as “anticipate,” “expect,”
“intend,” “plan,” “believe,” “seek,” “estimate,” variations of such
words, and similar expressions are intended to identify such
forward-looking statements, although not all forward-looking
statements contain these identifying words. These statements
concern, and these risks and uncertainties include, among others,
the nature, timing, and possible success and therapeutic
applications of products marketed or otherwise commercialized by
Regeneron and/or its collaborators or licensees (collectively,
“Regeneron’s Products”) and product candidates being developed by
Regeneron and/or its collaborators or licensees (collectively,
“Regeneron’s Product Candidates”) and research and clinical
programs now underway or planned, including without limitation the
investigational gene therapy DB-OTO discussed in this press release
as well as Regeneron’s other genetic medicine programs for hearing
loss and other therapeutic areas referenced in this press release;
the likelihood, timing, and scope of possible regulatory approval
and commercial launch of Regeneron’s Product Candidates and new
indications for Regeneron’s Products, such as DB-OTO in children
with genetic hearing loss due to mutations of the otoferlin gene;
the extent to which the results from the research and development
programs conducted by Regeneron and/or its collaborators or
licensees (including the preliminary data discussed in this press
release) may be further replicated and/or lead to advancement of
product candidates to clinical trials, therapeutic applications, or
regulatory approval; uncertainty of the utilization, market
acceptance, and commercial success of Regeneron’s Products and
Regeneron’s Product Candidates and the impact of studies (whether
conducted by Regeneron or others and whether mandated or
voluntary), including the studies discussed or referenced in this
press release, on any of the foregoing or any potential regulatory
approval of Regeneron’s Products and Regeneron’s Product Candidates
(such as DB-OTO); the ability of Regeneron’s collaborators,
licensees, suppliers, or other third parties (as applicable) to
perform manufacturing, filling, finishing, packaging, labeling,
distribution, and other steps related to Regeneron’s Products and
Regeneron’s Product Candidates; the ability of Regeneron to manage
supply chains for multiple products and product candidates; safety
issues resulting from the administration of Regeneron’s Products
and Regeneron’s Product Candidates (such as DB-OTO) in patients,
including serious complications or side effects in connection with
the use of Regeneron’s Products and Regeneron’s Product Candidates
in clinical trials; determinations by regulatory and administrative
governmental authorities which may delay or restrict Regeneron’s
ability to continue to develop or commercialize Regeneron’s
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obligations and oversight impacting Regeneron’s Products, research
and clinical programs, and business, including those relating to
patient privacy; the availability and extent of reimbursement of
Regeneron’s Products from third-party payers, including private
payer healthcare and insurance programs, health maintenance
organizations, pharmacy benefit management companies, and
government programs such as Medicare and Medicaid; coverage and
reimbursement determinations by such payers and new policies and
procedures adopted by such payers; competing drugs and product
candidates that may be superior to, or more cost effective than,
Regeneron’s Products and Regeneron’s Product Candidates;
unanticipated expenses; the costs of developing, producing, and
selling products; the ability of Regeneron to meet any of its
financial projections or guidance and changes to the assumptions
underlying those projections or guidance; the potential for any
license, collaboration, or supply agreement, including Regeneron’s
agreements with Sanofi and Bayer (or their respective affiliated
companies, as applicable) to be cancelled or terminated; the impact
of public health outbreaks, epidemics, or pandemics (such as the
COVID-19 pandemic) on Regeneron's business; and risks associated
with intellectual property of other parties and pending or future
litigation relating thereto (including without limitation the
patent litigation and other related proceedings relating to
EYLEA® (aflibercept) Injection and
REGEN-COV® (casirivimab and imdevimab)), other litigation and
other proceedings and government investigations relating to the
Company and/or its operations, the ultimate outcome of any such
proceedings and investigations, and the impact any of the foregoing
may have on Regeneron’s business, prospects, operating results, and
financial condition. A more complete description of these and other
material risks can be found in Regeneron’s filings with
the U.S. Securities and Exchange Commission, including its
Form 10-K for the year ended December 31, 2022 and its
Form 10-Q for the quarterly period ended June 30, 2023. Any
forward-looking statements are made based on management’s current
beliefs and judgment, and the reader is cautioned not to rely on
any forward-looking statements made by Regeneron. Regeneron does
not undertake any obligation to update (publicly or otherwise) any
forward-looking statement, including without limitation any
financial projection or guidance, whether as a result of new
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information about Regeneron is routinely posted and is accessible
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Contacts: |
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Media
Relations |
Investor
Relations |
Tammy Allen |
Vesna Tosic |
Tel: +1 914-306-2698 |
Tel: +1 914-847-5443 |
tammy.allen@regeneron.com |
vesna.tosic@regeneron.com |
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