Otonomy Provides Corporate and Product Pipeline Update
January 09 2018 - 6:30AM
Otonomy, Inc. (NASDAQ:OTIC), a biopharmaceutical company
dedicated to the development of innovative therapeutics for
diseases and disorders of the ear, today provided an update on its
product pipeline and financial guidance.
- OTIVIDEX™ demonstrated
statistical significance for multiple efficacy endpoints in the
AVERTS-2 trial of patients with Ménière’s disease: As
previously announced, the AVERTS-2 Phase 3 trial achieved its
primary efficacy endpoint, count of definitive vertigo days (DVD)
by Poisson Regression analysis in Month 3, for all 174 patients
enrolled in the trial (p value = 0.029). This endpoint and a number
of additional efficacy endpoints were also statistically
significant for the 111 patients who were enrolled in the AVERTS-2
trial through Month 3 at the time of study termination. These
endpoints are shown below.
Analysis of Patients Enrolled through Month 3 (n =
111) |
p value |
Count of DVD by Poisson Regression Analysis |
0.014 |
Mean Vertigo Severity Score |
0.030 |
Change in Vertigo Frequency from Baseline |
0.030 |
Numbers of Days Sick at Home or Bedridden |
0.042 |
The clinically significant treatment benefit demonstrated by
OTIVIDEX versus placebo in AVERTS-2 was consistent with
expectations from the Phase 2b trial. Based on completion of data
review, the AVERTS-1 trial failed due to a significantly higher
placebo response and was not attributable to a difference in
patient demographics or baseline characteristics compared to
AVERTS-2. A review of the AVERTS trials including consultation with
outside experts suggests that the higher placebo response was
primarily due to increased expectation bias in the U.S. trial.
Otonomy has requested a Type C meeting with the U.S. Food and Drug
Administration (FDA) and expects to meet during the first quarter
of 2018 to review the AVERTS results and any remaining clinical
requirements for registration of OTIVIDEX in Ménière’s disease. The
company expects that any remaining clinical development required
for registration will be initiated in mid-2018.
- Advancing three distinct programs for hearing loss that
address different pathologies and broad patient
populations: Hearing loss is a large and growing unmet
need with estimates by the World Health Organization that more than
360 million people worldwide have disabling levels of loss. This
leads to social isolation, lower quality of life, and higher rates
of dementia and depression. Common causes include aging, noise,
exposure to ototoxic drugs, and genetics, with increased noise
exposure from use of recreational music devices accelerating the
onset of hearing loss. The pathologies of hearing loss typically
involve damage to hair cells and/or spiral ganglion neurons in the
inner ear. As briefly described below, Otonomy is advancing three
distinct hearing loss programs targeting different pathologies:
repair of synaptopathy for treatment of hidden hearing loss
(OTO-413), protection of hair cells from ototoxic drugs including
cisplatin chemotherapy (OTO-5XX), and hair cell regeneration for
treatment of severe hearing loss (OTO-6XX).
- OTO-413 is a sustained exposure formulation of BDNF
that has been advanced into IND-enabling activities for the
treatment of hidden hearing loss: OTO-413 is a proprietary
formulation of brain-derived neurotropic factor (BDNF) which is a
naturally occurring protein involved in neuron growth and repair.
Nonclinical studies by Otonomy and other research groups have
demonstrated that local administration of BDNF repairs ribbon
synapses damaged due to noise trauma or exposure to ototoxic
chemicals and restores hearing function. Otonomy has initiated
nonclinical testing and manufacturing for OTO-413 to support an
Investigational New Drug (IND) Application, with a Phase 1/2
clinical trial expected to begin in hearing loss patients in the
first half of 2019. The initial indication for OTO-413 will be
patients with hidden hearing loss, a synaptopathy-related hearing
loss characterized by speech-in-noise hearing difficulty. This
condition affects nearly 3% of the U.S. population1.
- OTO-5XX is an otoprotectant in development for the
prevention of cisplatin-induced hearing loss (CIHL): A
number of drug classes cause ototoxicity including platinum-based
chemotherapeutic agents and aminoglycosides. Otonomy established
feasibility of conducting clinical trials in patients undergoing
cisplatin chemotherapy through a small Phase 2 trial with OTIVIDEX
in pediatric patients. The company has identified a therapeutic
target that offers a higher level of otoprotection than steroids
based on nonclinical proof-of-concept studies, and is evaluating
molecules in this class. Selection of a candidate for clinical
development is expected to occur in the second half of 2018. The
initial indication for OTO-5XX will be the prevention of CIHL,
which is relevant for approximately 500,000 patients in the U.S.
who are treated each year with platinum-based chemotherapies.
- OTO-6XX induces hair cell regeneration and is being
developed for the treatment of severe hearing loss:
Considerable interest and attention has been focused by otology
researchers over the past several decades for ways to regenerate
hair cells as an approach to treating severe hearing loss, which is
estimated to affect 6.6 million people in the U.S.2 This effort has
included extensive research with non-mammalian species that are
capable of hair cell regeneration in order to identify pathways for
therapeutic intervention. Targeting one of these pathways, Otonomy
has demonstrated regeneration of hair cells in a nonclinical
proof-of-concept model using a class of small molecules. Selection
of a candidate for clinical development is expected to occur in the
second half of 2018.
- Developing OTO-313, an improved formulation of
gacyclidine for the treatment of tinnitus: Gacyclidine is
a potent and selective N-Methyl-D-Aspartate (NMDA) receptor
antagonist, a molecular class with potential for treating tinnitus
based on both nonclinical and clinical studies. A Phase 1 clinical
safety trial has been successfully completed using OTO-311, a
poloxamer-based formulation of gacyclidine, with no safety concerns
observed. Otonomy has shifted development to OTO-313, an
alternative formulation of gacyclidine that has improved properties
compared to OTO-311. The company expects to initiate a Phase 1/2
clinical trial for OTO-313 in tinnitus patients in the first half
of 2019.
- OTIPRIO® divestiture
discussions in process with multiple parties: As announced
in November 2017, Otonomy has discontinued commercial support of
OTIPRIO (ciprofloxacin otic suspension) and is pursuing a sale of
the asset. FDA review of the Supplemental New Drug Application
(sNDA) for acute otitis externa is ongoing with a Prescription Drug
User Fee Act (PDUFA) action date of March 2, 2018. OTIPRIO
continues to be available for purchase by customers with net sales
for the fourth quarter of 2017 totaling $0.3 million, comparable to
net sales recorded for the prior quarter.
- Financial guidance: Recent actions taken to
discontinue OTIPRIO commercial support and eliminate other
non-essential positions have reduced ongoing operating costs
significantly. Otonomy expects GAAP operating expenses for 2017 to
total in the range of $95-$100 million with non-GAAP expenses
totaling $73-$78 million. The company expects GAAP operating
expenses for 2018 to total in the range of $52-$57 million with
non-GAAP expenses to total $40-$45 million. Otonomy's cash balance
including cash, cash equivalents, and short-term investments
totaled $120 million at the end of 2017. This cash balance is
expected to fund the completion of clinical development required
for U.S. registration of OTIVIDEX in Ménière’s disease, and support
advancement of the other programs.
"We are excited to begin 2018 given our broad product pipeline
targeting important unmet medical needs and large patient
populations in otology, and our strong balance sheet to support its
development," said David A. Weber, Ph.D., president and CEO of
Otonomy. "Results of the successful AVERTS-2 trial clearly
demonstrate that OTIVIDEX provides a significant clinical benefit
for patients with Ménière’s disease, and our advancement of
multiple programs for hearing loss and continued development of
gacyclidine for tinnitus together illustrate our commitment to
developing innovative treatments for this emerging field."
1Tremblay et al., Ear Hear (2015)2Goman and Lin, Am J Public
Health (2016)
About OtonomyOtonomy is a biopharmaceutical
company dedicated to the development of innovative therapeutics for
diseases and disorders of the ear. 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 Otonomy.
Forward-looking statements in this press release include, but are
not limited to, plans to meet with the FDA regarding the clinical
development requirements for OTIVIDEX™ and the timing of any such
meeting, timing of any remaining OTIVIDEX clinical work required,
timing of IND filing and Phase 1/2 clinical trial for OTO-413,
timing of Phase 1/2 clinical trial for OTO-313, financial guidance
for 2018, timing of candidate selection for OTO-5XX and OTO-6XX
programs, ability to fund completion of OTIVIDEX clinical
development, and the ability of Otonomy to complete a divestiture
of OTIPRIO®, and statements by Otonomy’s president and CEO.
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: Otonomy's
limited operating history and its expectation that it will incur
significant losses for the foreseeable future; Otonomy's ability to
obtain additional financing; Otonomy's dependence on the regulatory
success and advancement of its product candidates; 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, the
nonclinical and clinical results for its product candidates, which
may not support further development, and challenges related to
patient enrollment in clinical trials; Otonomy's ability to obtain
regulatory approval for its product candidates; 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; Otonomy's dependence on third parties for the manufacture
of its product candidates; Otonomy's dependence on a small number
of suppliers for raw materials; Otonomy's ability to protect its
intellectual property related to its product candidates in the
United States and throughout the world; expectations regarding
potential 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; 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 (the "SEC") on November 8, 2017,
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 InquiriesCanale CommunicationsHeidi Chokeir, Ph.D.Senior
Vice President619.849.5377heidi@canalecomm.com
Investor InquiriesWestwicke PartnersRobert H. UhlManaging
Director858.356.5932robert.uhl@westwicke.com
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