Decibel Therapeutics Announces Approval of Clinical Trial Application by the Spanish Agency of Medicines and Medical Devices (AEMPS) to Initiate Clinical Development of Lead Gene Therapy Candidate DB-OTO
May 12 2023 - 7:00AM
Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology
company dedicated to discovering and developing transformative
treatments to restore and improve hearing and balance, today
announced approval of its Clinical Trial Application (CTA) by the
Spanish Agency of Medicines and Medical Devices (AEMPS) to initiate
a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its
lead gene therapy product candidate. DB-OTO is being developed in
collaboration with Regeneron Pharmaceuticals, Inc. and is a
cell-selective, adeno-associated virus (AAV) gene therapy product
candidate designed to provide durable, physiological hearing in
individuals with profound, congenital hearing loss caused by
mutations of the otoferlin gene.
The CTA in Spain is part of an international regulatory strategy
for clinical development of DB-OTO, which also includes a
regulatory clearance for an Investigational New Drug (IND)
application in the United States in October 2022 and a CTA approval
by the U.K. Medicines and Healthcare Products Regulatory Agency
(MHRA) in January 2023. Decibel expects that clinical trial
participants in the U.K. and Spain will be infants two years of age
and younger. The Company anticipates reporting the initial safety
and tolerability data and preliminary efficacy data, as measured by
ABR, from the first patients in the Phase 1/2 clinical trial in the
first quarter of 2024.
“Spain is an important country within our DB-OTO clinical
development strategy, and our trial will leverage the
collaborations and natural history work that we have pursued there
over the past several years,” said Laurence Reid, Ph.D., Chief
Executive Officer at Decibel. “Our team believes that DB-OTO could
be a transformative treatment for individuals with
otoferlin-related hearing loss, and this approval broadens the
opportunity to evaluate its potential in infants two years of age
and younger.”
The Phase 1/2 dose escalation clinical trial, known as CHORD™,
is designed to evaluate the safety, tolerability and efficacy of
DB-OTO in pediatric patients with congenital hearing loss due to an
otoferlin deficiency. In addition to safety and tolerability
endpoints, the auditory brainstem response (ABR) -- an objective,
clinically accepted, physiologic measure of hearing sensitivity --
will be used as an efficacy endpoint in the clinical trial. The
ABR, which was used to characterize dose-response of DB-OTO after
intra-cochlear delivery in translational studies in animal models,
provides an opportunity to rapidly assess hearing functionality and
sensitivity.
DB-OTO received Orphan Drug and Rare Pediatric Disease
designations from the U.S. Food and Drug Administration in 2021 and
was granted European Orphan Drug status by the European Medicines
Agency (EMA) Committee on Orphan Medicinal Products (COMP) in March
2023.
About Decibel TherapeuticsDecibel Therapeutics
is a clinical-stage biotechnology company dedicated to discovering
and developing transformative treatments to restore and improve
hearing and balance, one of the largest areas of unmet need in
medicine. Decibel has built a proprietary platform that integrates
single-cell genomics and bioinformatic analyses, precision gene
therapy technologies and expertise in inner ear biology. Decibel is
leveraging its platform to advance gene therapies designed to
selectively replace genes for the treatment of congenital,
monogenic hearing loss and to regenerate inner ear hair cells for
the treatment of acquired hearing and balance disorders. Decibel’s
pipeline, including its lead gene therapy product candidate,
DB-OTO, to treat congenital, monogenic hearing loss, is designed to
deliver on our vision of creating a world of connection for people
with hearing and balance disorders. For more information about
Decibel Therapeutics, please visit www.decibeltx.com or follow us
on Twitter.
Forward-Looking Statements
This press release contains forward-looking statements that
involve substantial risks and uncertainties. All statements, other
than statements of historical facts, contained in this press
release, including statements regarding Decibel’s strategy, future
operations, prospects, plans, objectives of management, the
therapeutic potential for Decibel’s product candidates and
preclinical programs, the potential benefits of cell-selective
expression, the design of the Phase 1/2 clinical trial of DB-OTO
and the expected timelines for initiating a Phase 1/2 clinical
trial of DB-OTO and announcing data from the trial constitute
forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. The words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,”
“may,” “might,” “objective,” “ongoing,” “plan,” “predict,”
“project,” “potential,” “should,” or “would,” or the negative of
these terms, or other comparable terminology are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Decibel
may not actually achieve the plans, intentions or expectations
disclosed in these forward-looking statements, and you should not
place undue reliance on these forward-looking statements. Actual
results or events could differ materially from the plans,
intentions and expectations disclosed in these forward-looking
statements as a result of various important factors, including:
uncertainties inherent in the identification and development of
product candidates, including the timing of and Decibel’s ability
to obtain approval to initiate clinical development of its program
candidates, whether results from preclinical studies will be
predictive of the results of later preclinical studies and clinical
trials, whether Decibel’s cash resources are sufficient to fund its
foreseeable and unforeseeable operating expenses and capital
expenditure requirements, uncertainties related to the impact of
the COVID-19 pandemic on Decibel’s business and operations, as well
as the risks and uncertainties identified in Decibel’s filings with
the Securities and Exchange Commission (SEC), including those risks
detailed under the caption “Risk Factors” in Decibel’s Annual
Report on Form 10-K for the fiscal year ended December 31, 2022 and
in other filings Decibel may make with the SEC. In addition, the
forward-looking statements included in this press release represent
Decibel’s views as of the date of this press release. Decibel
anticipates that subsequent events and developments will cause its
views to change. However, while Decibel may elect to update these
forward-looking statements at some point in the future, it
specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing Decibel’s views as of any date subsequent to the date
of this press release.
Investor Contact:Julie SeidelStern IR,
Inc.212-362-1200Julie.seidel@sternir.com
Media Contact:Chris RaileyTen Bridge
Communications617-834-0936chris@tenbridgecommunications.com
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