Decibel Therapeutics Announces Approval of Clinical Trial Application by the U.K. Medicines and Healthcare Products Regulatory Agency for Lead Gene Therapy Candidate DB-OTO
January 24 2023 - 6:00AM
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 authorization of its Clinical Trial Application (CTA) by
the United Kingdom (U.K.) Medicines and Healthcare products
Regulatory Agency (MHRA) 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 designed to provide
durable hearing in individuals with profound, congenital hearing
loss caused by mutations of the otoferlin gene.
The U.K. CTA is part of an international regulatory strategy for
clinical development of DB-OTO, which also includes a regulatory
clearance in October 2022 for an Investigational New Drug (IND)
application in the United States and a CTA submitted to the Spanish
Agency of Medicines and Medical Devices.
“This CTA marks the second regulatory clearance for DB-OTO and
what we believe to be the first in Europe for any gene therapy
targeting otoferlin. This is an important milestone as we execute
our international clinical development strategy, which addresses
pediatric patients of diverse ages, including infants two years of
age and younger,” said Laurence Reid, Ph.D., Chief Executive
Officer at Decibel. “We believe DB-OTO has transformative potential
for individuals with otoferlin-related hearing loss, and we intend
to initiate the clinical trial in the first half of 2023. We expect
to share initial data from the first cohort of patients in the
first quarter of 2024.”
DB-OTO received Orphan Drug and Rare Pediatric Disease
designations from the U.S. Food and Drug Administration in 2021.
Currently, there are no approved pharmacologic treatment options
for individuals with otoferlin-related hearing loss.
The Phase 1/2 dose escalation clinical trial 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.
The Company intends to provide an update on the design of the
clinical trial in mid-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 Quarterly
Report on Form 10-Q for the quarterly period ended September 30,
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 Investor
Relations, Inc.julie.seidel@sternir.com212-362-1200
Media Contact:Chris RaileyTen Bridge
CommunicationsChris@tenbridgecommunications.com617-834-0936
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