AGTC and Otonomy Announce Strategic Collaboration to Develop and Commercialize Gene Therapy for Congenital Hearing Loss
October 01 2019 - 7:30AM
Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
biotechnology company conducting human clinical trials of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare diseases, and Otonomy, Inc. (NASDAQ: OTIC), a
biopharmaceutical company dedicated to the development of
innovative therapeutics for neurotology, today announced that they
have entered into a strategic collaboration to co-develop and
co-commercialize an AAV-based gene therapy to restore hearing in
patients with sensorineural hearing loss caused by a mutation in
the gap junction protein beta 2 gene (GJB2)— the most common cause
of congenital hearing loss.
Mutations in GJB2 account for approximately 30%
of all genetic hearing loss cases. Patients with this mutation can
have severe-to-profound deafness in both ears that is identified in
screening tests routinely performed in newborns. Under the
collaboration agreement, the parties will equally share the program
costs and proceeds, and can include additional genetic hearing loss
targets in the future.
“There is a tremendous unmet need for effective
drug therapies to treat sensorineural hearing loss and this is
especially true in children born with severe or progressive hearing
loss due to genetic mutations,” said Lawrence R. Lustig, M.D.,
Chair, Department of Otolaryngology – Head & Neck Surgery,
Columbia University College of Physicians and Surgeons. “Gene
therapy holds great promise to restore functional and lasting
hearing in these cases, with early intervention providing
significant benefits in the development of language skills,
socialization and overall quality of life for patients and their
families.”
“We are excited to initiate this collaboration
which combines our deep capabilities, technology and expertise in
gene therapy development and manufacturing with Otonomy’s extensive
experience in otic drug delivery and existing infrastructure for
preclinical and clinical development in neurotology,” said Sue
Washer, president and CEO of AGTC. “In addition to the
positive data that we recently released supporting our XLRP and
achromatopsia clinical trials, advancing this program in
neurotology further demonstrates the versatility of our gene
therapy platform and its applicability to treat a broad range of
indications.”
“As leaders in neurotology, we are pleased to
add our expertise to that of the highly experienced AGTC team,
leveraging what each of us does best and sharing the cost and
effort to bring a novel therapeutic to patients with profound
need,” said David A. Weber, Ph.D., president and CEO of Otonomy.
“The addition of this gene therapy program further extends our
broad, innovative pipeline across the largest market opportunities
in neurotology including acquired and genetic forms of hearing
loss, tinnitus, and balance disorders such as Ménière’s
disease.”
About Genetic Hearing
LossHearing loss is the most common inherited sensory
disorder. In developed countries such as the United States, genetic
mutations are responsible for the vast majority of hearing loss in
young children, which is estimated to affect 1 out of 500 children
before speech develops. Because congenital hearing loss is one of
the most prevalent chronic conditions in children, neonatal
screening is routinely performed. This is typically followed by
genetic testing in those cases where a deficit has been detected.
Mutations have been identified in over 100 genes including the gap
junction protein beta 2 gene (GBJ2), which is the most frequent
cause of severe-to-profound congenital hearing loss. The GJB2 gene
encodes connexin-26 which is expressed in cochlear support cells,
forming gap junctions that control potassium homeostasis which is
critical for the survival and function of hair cells and normal
hearing. Mutations in GJB2 impair gap junctions and cochlear
homeostasis leading to hair cell dysfunction and hearing loss. The
goal of GJB2 gene therapy is to restore functional gap junctions
and preserve hair cells to improve hearing.
About AGTCAGTC is a
clinical-stage biotechnology company that uses a proprietary gene
therapy platform to develop transformational genetic therapies for
patients suffering from rare and debilitating diseases. Its initial
focus is in the field of ophthalmology, where it has active
clinical trials in X-linked retinitis pigmentosa (XLRP) and
achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its
clinical trials, AGTC has preclinical programs in optogenetics,
adrenoleukodystrophy (ALD), which is a disease of the central
nervous system (CNS) and other ophthalmology and otology
indications. The optogenetics program is being developed in
collaboration with Bionic Sight. AGTC has a significant
intellectual property portfolio and extensive expertise in the
design of gene therapy products including capsids, promoters and
expression cassettes, as well as expertise in the formulation,
manufacture and physical delivery of gene therapy products. For
additional information please visit www.agtc.com
About OtonomyOtonomy 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 including Ménière’s
disease, 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 AGTC and/or Otonomy. Forward-looking statements in this
press release include, but are not limited to, the potential
benefits of and activity under the collaboration agreement
between AGTC and Otonomy, including but not limited to
development and commercialization activity, and cost and proceeds
sharing, the potential benefits of the therapy, the strategy and
development activities of AGTC and Otonomy, and statements by the
president and CEO of AGTC and the president and CEO of
Otonomy. AGTC’s and 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: the risk of the occurrence of any event, change
or other circumstance that could give rise to the termination of
the collaboration agreement between AGTC and Otonomy; uncertainties
inherent in the clinical drug development process; competition in
the biopharmaceutical industry; expectations regarding either
company’s expertise and capabilities; AGTC’s and
Otonomy's ability to protect its intellectual property;
expectations regarding potential therapy benefits, market size and
opportunity; the ability to obtain regulatory approval for such
therapies; AGTC’s and Otonomy's ability to manage operating
expenses; implementation of their respective business
models and strategic plans for their business, products and
technology; and other risks. Information regarding the foregoing
and additional risks may be found in the section entitled “Risk
Factors” in AGTC’s Annual Report on Form 10-K for the fiscal
year ended June 30, 2019, filed with the Securities and Exchange
Commission (the “SEC”) and in Otonomy's Quarterly Report
on Form 10-Q filed with the SEC on August 1, 2019,
and AGTC’s 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 AGTC and Otonomy as
of the date hereof. AGTC and Otonomy each disclaim any
obligation to update any forward-looking statements, except as
required by law.
AGTC Contacts:IR/PR Contacts: David Carey (IR)
or Tom Vickery (PR)Lazar Partners Ltd.T: (212) 867-1768 or (646)
871-8485dcarey@lazarpartners.com or tvickery@lazarpartners.com
Otonomy Contacts:Media Inquiries Spectrum
Leticia Diaz Vice President 202.587.2517
ldiaz@spectrumscience.com
Investor Inquiries Westwicke Partners Robert H. Uhl Managing
Director 858.356.5932 robert.uhl@westwicke.com
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