Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that
the investigational gene therapy DB-OTO improved hearing to normal
levels in one child (dosed at 11 months of age) within 24 weeks,
and initial hearing improvements were observed in a second child
(dosed at 4 years of age) at a 6-week assessment. Both children
were born with profound genetic deafness due to variants of the
otoferlin gene, and the child dosed at 11 months of age is one of
the youngest in the world to receive a gene therapy for genetic
deafness. The results are from the ongoing Phase 1/2 CHORD trial,
which is currently enrolling infants and children and were detailed
during an oral presentation at the American Society of Gene and
Cell Therapy (ASGCT) annual conference.
“The opportunity of providing the full complexity and spectrum
of sound in children born with profound genetic deafness is a
phenomenon I did not expect to see in my lifetime,” said Lawrence
R. Lustig, M.D., Chair of Columbia University’s Department of
Otolaryngology - Head & Neck Surgery and a clinical trial
investigator. “These impressive results showcase the revolutionary
promise of DB-OTO as a potential treatment for otoferlin-related
deafness, and we are excited to see how this translates into an
individual’s development, especially since early intervention is
associated with better outcomes for speech development. With the
DB-OTO CHORD trial now enrolling participants in sites across the
U.S. and Europe, we’re part of the beginning of a new era of gene
therapy research that looks to create treatment options that
address the root cause of profound genetic deafness.”
In the trial, both children received a single intracochlear
injection of DB-OTO in one ear. The surgical procedure leverages
the same approach used for cochlear implants, which is amenable for
use in young infants. Hearing improvements were assessed by pure
tone audiometry (PTA) and auditory brainstem response (ABR). PTA is
considered by auditory experts to be the gold standard measurement
of hearing and is measured through behavioral confirmation of sound
(e.g., turning head towards sound) emitted at different intensity
levels (measured in decibels or dB). ABR corroborates these
behavioral responses, serving as an objective confirmation of
hearing function, by measuring electrical brainstem responses to
sound emitted at different dBs.
At baseline, both participants had no behavioral (PTA) or
electrophysiological (ABR) responses at maximum sound levels (≥100
dB). Following treatment with DB-OTO, both children showed auditory
responses at the first efficacy assessment of 4 weeks.
As presented at ASGCT, the first participant dosed in the trial
was 16 months of age at the 24-week assessment and showed:
- Improvement of hearing to normal levels across
key speech frequencies, with an average 84 dB improvement from
baseline and one frequency measure reaching 10 dB in hearing level
per PTA. Across all tested frequencies, an average 80 dB
improvement from baseline was observed.
- Positive ABR responses, with best frequency
reaching 45 dB.
The second participant dosed in the trial was 4 years of age at
the 6-week assessment and experienced consistent results to the
first participant at the same timepoint, including:
- Initial improvement of hearing with responses to loud
sounds, which was observed across key speech frequencies,
with an average 19 dB improvement from baseline and one frequency
measure reaching 80 dB in hearing level per PTA. Across all tested
frequencies, an average 16 dB improvement from baseline was
observed.
- Positive ABR responses, with best frequency
reaching 75 dB.
Both the surgical procedure (delivery and post-operation) and
DB-OTO were well tolerated, and there were no related adverse
events or serious adverse events following treatment.
DB-OTO received Orphan Drug, Rare Pediatric Disease and Fast
Track Designations from the U.S. Food and Drug Administration and
Orphan Drug Designation was granted by the European Medicines
Agency. The potential use of DB-OTO for otoferlin-related hearing
loss is currently under clinical investigation, and its safety and
efficacy have not been evaluated by any regulatory authority.
About Otoferlin-related Hearing LossCongenital
deafness (hearing loss present at birth) is a significant unmet
medical need that affects approximately 1.7 out of every 1,000
children born in the U.S. Although approximately half of these
cases have genetic causes, otoferlin-related hearing loss is
ultra-rare. This specific condition is caused by variants in the
otoferlin gene, which impairs the production of the OTOF protein
that is critical for the communication between the sensory cells of
the inner ear and the auditory nerve. While hearing aids and
cochlear implants can amplify sound to improve hearing for
individuals with a range of hearing loss, these devices do not
currently restore the full spectrum of sound.
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 infants, children and adolescents with
otoferlin variants.
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), participants will receive a single
intracochlear injection of DB-OTO in one ear, while in expansion
cohort (Part B), participants will receive simultaneous 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@regeneron.com.
About DB-OTO and the Regeneron Auditory
ProgramDB-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 variants of the otoferlin gene.
The treatment aims to deliver a working copy to replace 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 newly introduced otoferlin
gene is under the control of a proprietary cell-specific Myo15
promoter, which is intended to restrict expression only to inner
hair cells that normally express otoferlin.
The ongoing CHORD trial is Regeneron’s first clinical-stage
auditory program. In addition to DB-OTO, AAV.103 is also being
investigated for people with GJB2-related hearing loss.
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 by
physician-scientists, our unique ability to repeatedly and
consistently translate science into medicine has led to
numerous approved treatments and product candidates in development,
most of which were homegrown in our laboratories. Our medicines and
pipeline are designed to help patients with eye diseases, allergic
and inflammatory diseases, cancer, cardiovascular and metabolic
diseases, neurological diseases, hematologic conditions,
infectious diseases, and rare diseases.
Regeneron pushes the boundaries of scientific discovery
and accelerates drug development using our proprietary
technologies, such as VelociSuite®, which produces optimized fully
human antibodies and new classes of bispecific antibodies. We are
shaping the next frontier of medicine with data-powered insights
from the Regeneron Genetics Center® and pioneering genetic medicine
platforms, enabling us to identify innovative targets and
complementary approaches to potentially treat or cure diseases.
For more information, please visit www.Regeneron.com or follow
Regeneron on LinkedIn, Instagram, Facebook or X.
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
Products and Regeneron’s Product Candidates; ongoing regulatory
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), other litigation and other proceedings
and government investigations relating to the Company and/or its
operations (including the pending civil proceedings initiated or
joined by the U.S. Department of Justice and the U.S. Attorney's
Office for the District of Massachusetts), 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, 2023 and its Form 10-Q
for the quarterly period ended March 31, 2024. 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
information, future events, or otherwise.
Regeneron uses its media and investor relations website and
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Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website (https://investor.regeneron.com) and its
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Contacts: |
|
Media
Relations Tammy
Allen Tel: +1 914-306-2698tammy.allen@regeneron.com |
Investor
RelationsVesna TosicTel: +1
914-847-5443vesna.tosic@regeneron.com |
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