Otonomy and AGTC Present Preclinical Proof-of-Concept Results for OTO-825 Gene Therapy at ASGCT Annual Meeting
May 14 2021 - 7:30AM
Otonomy, Inc. (Nasdaq: OTIC), a biopharmaceutical company
dedicated to the development of innovative therapeutics for
neurotology, today announced preclinical proof-of-concept results
for OTO-825 presented at the American Society of Gene & Cell
Therapy (ASGCT) Annual Meeting. OTO-825 is an AAV-mediated gene
therapy targeting the gap junction beta-2 (GJB2) gene developed
under the company’s collaboration with Applied Genetic Technologies
Corporation (Nasdaq: AGTC). These results demonstrate that a single
administration of OTO-825 rescues hearing loss and cochlear damage
in two preclinical models that represent a range of hearing loss
severity caused by GJB2 deficiency.
"The treatment of GJB2 deficiency is an important unmet medical
need since it is the most common genetic mutation causing
congenital hearing loss with many patients experiencing significant
impairment beginning at birth,” said Alan C. Foster, Ph.D., chief
scientific officer of Otonomy. “These proof-of-concept results in
two independent preclinical models are an important milestone for
the program because they validate the therapeutic potential of
OTO-825 across a range of hearing loss levels observed in patients
and support its advancement into IND enabling activities, which are
currently underway.”
The joint oral presentation by Otonomy and AGTC demonstrates the
following:
- OTO-825 induces expression of Connexin26, the protein product
of the GJB2 gene, in target cochlear cells including support cells
of the organ of Corti and spiral limbus, and fibrocytes of the
spiral ligament in both rodents and non-human primates.
- Connexin26 expressed by OTO-825 forms functional gap junctions,
which are required for proper functioning of cochlear hair
cells.
- Two conditional knockout models developed in mice mimic the
human setting for GJB2 deficiency by displaying hearing loss and
damage to the integrity of cochlear tissue.
- A single intracochlear administration of OTO-825 rescues
expression of Connexin26 in both models.
- OTO-825 induces significant improvement in hearing across
multiple frequencies and normalizes cochlear morphology in both
models.
Mutations in the GJB2 gene account for approximately 30% of
congenital hearing loss cases. Patients with GJB2 mutation can have
severe-to-profound deafness in both ears that is identified in
screening tests routinely performed in newborns.
About Otonomy
Otonomy is a biopharmaceutical company dedicated to the
development of innovative therapeutics for neurotology. The company
pioneered the application of drug delivery technology to the ear in
order to develop products that achieve sustained drug exposure from
a single local administration. This approach is covered by a broad
patent estate and is being utilized to develop a pipeline of
products addressing important unmet medical needs with a focus on
hearing loss and tinnitus. For additional information please visit
www.otonomy.com.
Cautionary Note Regarding Forward Looking
Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements generally relate to future events
or the future financial or operating performance of Otonomy.
Forward-looking statements in this press release include, but are
not limited to, statements related to plans and expectations
regarding OTO-825; and statements by Otonomy’s chief scientific
officer.
Otonomy’s expectations regarding these matters may not
materialize, and actual results in future periods are subject to
risks and uncertainties. Actual results may differ materially from
those indicated by these forward-looking statements as a result of
these risks and uncertainties, including but not limited to: delays
and disruption resulting from the COVID-19 pandemic;
Otonomy’s ability to obtain additional financing; the uncertainties
inherent in the clinical drug development process, including,
without limitation, Otonomy’s ability to adequately demonstrate the
safety and efficacy of its product candidates and the nonclinical
and clinical results for its product candidates, which may not
support further development; the risks of the occurrence of any
event, change or other circumstance that could impact the
performance under or give rise to the termination of Otonomy’s
collaboration, co-promotion or license agreements, including its
collaboration agreement with AGTC, or that could impact Otonomy’s
ability to repay or comply with the terms of the loan provided by
Oxford Finance LLC; side effects or adverse events associated with
Otonomy’s product candidates; competition in the biopharmaceutical
industry; Otonomy’s dependence on third parties to conduct
nonclinical studies and clinical trials, and for the manufacture of
its product candidates; Otonomy’s ability to protect its
intellectual property in the United States and throughout the world
and to ensure compliance with various laws and regulations in
countries in which it conducts clinical trials; expectations
regarding potential therapy benefits, market size, opportunity and
growth; Otonomy’s ability to manage operating expenses;
implementation of Otonomy’s business model and strategic plans for
its business, products and technology; general economic and market
conditions; and other risks. Information regarding the
foregoing and additional risks may be found in the section entitled
"Risk Factors" in Otonomy’s Quarterly Report on Form 10-Q filed
with the Securities and Exchange Commission (SEC) on May 11, 2021,
and Otonomy’s future reports to be filed with the SEC. The
forward-looking statements in this press release are based on
information available to Otonomy as of the date hereof. Otonomy
disclaims any obligation to update any forward-looking statements,
except as required by law.
Contacts:
Media InquiriesSpectrum ScienceChloé-Anne RamseyVice
President404.865.3601cramsey@spectrumscience.com
Investor InquiriesWestwicke ICRRobert H. UhlManaging
Director858.356.5932robert.uhl@westwicke.com
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