Otonomy Reports Corporate and Product Pipeline Update
January 06 2022 - 7:51AM
Otonomy, Inc. (Nasdaq: OTIC), a biopharmaceutical company
dedicated to the development of innovative therapeutics for
neurotology, today provided an update on its product pipeline and
financial guidance. Consistent with previously stated timing, the
company expects to report results for the Phase 2a cohort of
OTO-413 in hearing loss patients early in the second quarter of
2022 and the Phase 2 trial of OTO-313 in tinnitus patients in
mid-2022. The company also plans to initiate a clinical safety
evaluation of higher unilateral as well as bilateral dosing of
OTO-313 and, as previously announced, has initiated clinical
evaluation of higher dosing of OTO-413. Results of these multiple
studies are expected to inform initiation of a full dose-ranging
Phase 2 trial for OTO-413 by the end of 2022 and start of the Phase
3 clinical program for OTO-313 in the first half of 2023.
The company finished 2021 with approximately $77 million in
cash, cash equivalents and short term investments and expects that
its current capital is sufficient to fund operations into the
second half of 2023.
“We are excited to begin 2022, a catalyst-rich year for Otonomy
because of our multiple clinical trial readouts for both OTO-313
and OTO-413, novel products that target large patient populations
with high disease burden and no approved drug treatment,” said
David A. Weber, Ph.D., president and CEO of Otonomy. “For OTO-313,
we expect to generate robust clinical data to inform the Phase 3
program by having efficacy readouts for Months 1 and 2 as well as
the extended follow-up period out to Month 4, and we will also have
safety data for both a higher unilateral OTO-313 dose and bilateral
dosing. Similarly, efficacy data from the OTO-413 Phase 2a cohort
plus ongoing dose escalation evaluation will provide a good basis
for initiating a full dose-ranging Phase 2 efficacy trial for
OTO-413 later this year.”
Product Pipeline Update
- OTO-313: Phase 2 trial in tinnitus is ongoing with
top-line results expected in mid-2022; initiating safety evaluation
of higher and bilateral dosing. Otonomy is conducting a
Phase 2 trial of OTO-313 that will enroll approximately 140
patients with persistent, unilateral tinnitus of at least moderate
severity. Patients are being randomized 1:1 to a single
intratympanic injection of OTO-313 (0.32 mg) or placebo. The
primary endpoint is the same as reported for the successful Phase
1/2 trial: a responder analysis based on the proportion of patients
who report a clinically meaningful improvement in the Tinnitus
Functional Index (TFI) from baseline to Months 1 and 2 following
treatment. To assess durability of the OTO-313 treatment effect,
the follow-up period has been extended out to four
months. Following the positive Phase 1/2 trial, Otonomy
conducted additional nonclinical testing and successfully completed
a Type C meeting with the U.S. Food and Drug Administration (FDA)
in December supporting initiation of a 1-month safety study for
bilateral and higher (0.64 mg) dosing of OTO-313. Additional
efficacy results from the Phase 2 extended observation period and
safety data from the bilateral and higher unilateral dose study are
anticipated in the second half of 2022. Altogether, these multiple
clinical data readouts for OTO-313 are expected to support an
End-of-Phase 2 meeting with the FDA and inform the design of the
Phase 3 clinical program planned to start in the first half of
2023.
- OTO-413: Phase 2a cohort in hearing loss is fully
enrolled with top-line results expected early in the second quarter
of 2022; clinical evaluation of higher dosing initiated.
Otonomy has completed enrollment in the Phase 2a cohort for the
highest OTO-413 dose (0.3 mg) evaluated in the initial Phase 1/2
trial cohorts. A total of 33 patients with hearing loss were
randomized 2:1 to receive a single intratympanic injection of
OTO-413 or placebo. Patients are being followed for three months
and assessed using multiple clinically-validated speech-in-noise
hearing tests. Otonomy has also initiated enrollment to evaluate
safety of at least one higher dose of OTO-413 (starting with 0.75
mg). Each dose cohort will enroll approximately 12 hearing loss
patients randomized 2:1 to OTO-413 or placebo and patients will be
assessed as in the Phase 2a. Based on results from the Phase 2a
cohort and higher-dose safety evaluation, Otonomy expects to
initiate a full dose-ranging Phase 2 efficacy trial by the end of
2022.
- OTO-825: preclinical studies validate
therapeutic potential of GJB2 gene therapy for
congenital hearing loss with Investigational New Drug (IND) filing
expected in first half of 2023. OTO-825 is an AAV-based
gene therapy to restore hearing in patients with a mutation in the
gap junction beta-2 (GJB2) gene, the most common cause of
congenital hearing loss. Preclinical proof-of-concept results for
OTO-825 demonstrate that a single administration of OTO-825 rescues
hearing loss and cochlear damage in two preclinical models
representing a range of hearing loss severity caused by GJB2
deficiency. A Pre-IND meeting has been completed with the FDA and
IND-enabling activities are ongoing.
- OTO-510: preclinical development ongoing for novel and
proprietary otoprotection molecule. Cisplatin is a potent
chemotherapeutic agent that is widely used to treat a variety of
cancers in adults and children, however, it is commonly associated
with severe adverse effects including cisplatin-induced hearing
loss (CIHL). Otonomy has identified a novel series of molecules
with improved otoprotection in preclinical CIHL studies compared to
other agents in development. The goal of the OTO-510 program is
otoprotection without tumor protection.
- OTO-6XX: preclinical development ongoing for severe
hearing loss program. Otonomy is evaluating therapeutic
approaches to repair or regenerate cochlear hair cells that are
damaged due to noise, aging or exposure to ototoxic chemicals. This
mechanism is expected to be complementary to repair of cochlear
synapses, which is addressed by OTO-413.
Anticipated Upcoming Milestones
- Early second quarter 2022, announce top-line results for
OTO-413 Phase 2a cohort.
- In mid-2022, announce top-line results for OTO-313 Phase 2
clinical trial.
- In second half of 2022, announce top-line results for OTO-313
Phase 2 extended observation period and safety results for
bilateral and higher dosing.
- In second half of 2022, announce top-line results for OTO-413
higher dose evaluation.
- By end of 2022, initiate Phase 2 dose-ranging efficacy trial
for OTO-413 in hearing loss.
- In first half of 2023, initiate Phase 3 clinical program for
OTO-313 in tinnitus.
- In first half of 2023, file IND for OTO-825 in hearing loss
associated with GJB2 gene mutation.
Financial Updates and Guidance
- Cash Position: Cash, cash equivalents, and
short-term investments totaled approximately $77 million as of
December 31, 2021, compared to $86.3 million as of December 31,
2020.
- Operating Expenses: Otonomy expects that
non-GAAP operating expenses for 2022 will be in the range of
$42-$44 million, and that GAAP operating expenses will be in the
range of $52-$54 million.
- Cash Runway: Otonomy expects that its current
cash, cash equivalents, and short-term investments will be
sufficient to fund company operations into the second half of
2023.
Non-GAAP Operating Expenses
In this press release, Otonomy’s operating expenses are provided
in accordance with generally accepted accounting principles (GAAP)
in the United States and also on a non-GAAP basis. Non-GAAP
operating expenses exclude stock-based compensation. Non-GAAP
operating expenses are provided as a complement to operating
expenses provided in accordance with GAAP because management
believes non-GAAP operating expenses help indicate underlying
trends in the company’s business, are important in comparing
current results with prior period results and provide additional
information regarding the company’s financial position. Management
also uses non-GAAP operating expenses to establish budgets and
operational goals that are communicated internally and externally
and to manage the company’s business and to evaluate its
performance.
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 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 the design and conduct of,
activity, enrollment plans and patient populations for, and timing
of initiation and results for current and planned clinical trials;
Otonomy’s development plans and timelines for its product
candidates and programs; the potential benefits and advantages of
Otonomy’s product candidates and programs; expectations regarding
preclinical programs, including the potential benefits, development
activities and plans to file an IND; expectations regarding
Otonomy’s ability to advance its pipeline and regarding upcoming
catalysts; Otonomy’s anticipated upcoming milestones; expectations
regarding operating expenses for 2022 and cash runway; 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: delays and disruption
resulting from the COVID-19 pandemic and governmental
responses to the pandemic, including current and future impacts to
Otonomy’s operations, the initiation and progression of, and
enrollment in, its planned and current clinical trials, and patient
conduct and compliance; Otonomy’s ability to accurately forecast
financial results; Otonomy’s 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, conduct and compliance in clinical trials; the
integrity of patient-reported outcomes in its current and future
clinical trials; 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 any promotional, collaboration or
license agreements, 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; Otonomy’s ability to obtain regulatory approval
and successfully commercialize its product candidates, if approved;
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 November 10, 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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