Results Published in Leading Peer-Reviewed
Otolaryngology Journal, Otology & Neurotology
Statistically Significant Improvements Observed
in Word Recognition Tests in Quiet and in Noise; Suggests Potential
to Improve Speech Intelligibility, a Major Unmet Need for
Individuals with Hearing Loss
Company Expects Interim FX-322 Phase 2a Study
Data Later this Quarter
Frequency Therapeutics, Inc. (Nasdaq: FREQ), a
clinical-stage biotechnology company focused on harnessing the
body’s innate biology to repair or reverse damage caused by a broad
range of degenerative diseases, today announced the publication of
its FX-322 Phase 1/2 study results in Otology & Neurotology, a
leading peer-reviewed journal focused on disorders of the ear. The
data show hearing improvements in adults with acquired
sensorineural hearing loss (SNHL) and the first-known linkage of
pharmacokinetics and pharmacodynamics for a potential hearing
restoration therapy.
Findings from the Phase 1/2 study of FX-322, the company’s lead
product candidate to treat SNHL, showed statistically significant
increases in word recognition (WR) and words-in-noise (WIN) scores.
Individuals with stable SNHL that received a single dose of FX-322
showed improvements in the number of words recognized in quiet from
baseline to day 90 in the WR test (p=0.029) and the level of
background noise in which words could be identified in the WIN test
(p=0.012). There were no meaningful changes in the WR and WIN
scores of the placebo group. FX-322 was also shown to be well
tolerated. The publication includes data demonstrating consistent
cochlear drug delivery in both preclinical and human studies.
FX-322 is currently being evaluated in a larger Phase 2a study,
with results anticipated later this quarter.
The study data will be presented today at the leading
international hearing research conference, the Association for
Research in Otolaryngology 44th Annual MidWinter Meeting.
“Healthy hearing is not just about the volume of sound,
but about the broader ability to communicate. The most frequent
complaint from patients with hearing loss is their inability to
understand speech, which is due to a lack of clarity and loss of
intelligibility where patients can’t recognize words and follow
conversations, particularly in background noise,” said Christina
Runge, Ph.D., a professor in the Department of Otolaryngology and
Communication Sciences at the Medical College of Wisconsin,
Director of the Koss Cochlear Implant Program, a member of the
Company’s clinical advisory board and an author on the publication.
“These Phase 1/2 data are the first to show a potential therapeutic
solution to address intelligibility, a key unmet need for those
with SNHL.”
SNHL accounts for more than 90 percent of all hearing loss and
results from damage to auditory sensory hair cells due to chronic
noise exposure, aging, certain viral infections or from toxic
medications. SNHL impacts more than 40 million people in the U.S.
and hundreds of millions of people globally.
“Having a treatment that regenerates sensory hair cells would
have a profound impact on the field, providing physicians with a
meaningful intervention for restoring hearing loss,” said Ravi N.
Samy, M.D., FACS, Professor of Otolaryngology & Neurosurgery
and Chief of the Division of Otology/Neurotology at the University
of Cincinnati / Cincinnati Children’s. “These exciting early
clinical data begin to show the potential of a localized approach
to repairing the cochlea, the ability to deliver a treatment to an
area of the body that historically has been impossible to access
and restore, and above all, clinically meaningful hearing
improvements in patients.”
Hearing loss typically begins in higher frequencies and often
impacts the ability to hear and distinguish between certain
consonants, making it difficult to understand words, especially in
noise. Hearing aids amplify sound but do not improve hearing
clarity in higher frequencies or repair the underlying damage to
the inner ear. FX-322, an injectable treatment candidate made up of
two small molecules, is designed to activate progenitor cells
(pre-programmed stem cells) to regenerate auditory hair cells in
the cochlea and restore hearing function.
“These Phase 1/2 study data build upon the favorable word
recognition performances that we previously reported, while
bolstering the trends we saw in words-in-noise scores,” said Will
McLean, Ph.D., Frequency’s Vice President of Biology and
Regenerative Medicine, a Company co-founder and the lead author on
the paper. “Increases in speech intelligibility and improvements
when listening in noisy environments can have a significant impact
on quality of life, and we are excited about the potential of our
approach for patients and to advance the field of hearing
restoration.”
FX-322 Development Program
FX-322 is Frequency’s lead product candidate for the treatment
of acquired SNHL. The Phase 1/2 study was a randomized,
double-blind, placebo-controlled, single-dose, multi-center study
to assess the safety of FX-322 in 23 patients with stable SNHL.
Patients received an intratympanic injection of FX-322 or placebo
in one ear during an office-based procedure. In the study, 15
patients received a single dose of FX-322 in one ear while eight
patients received placebo.
Of the six patients treated with FX-322 who had baseline word
recognition in quiet scores of below 90 percent, four showed
clinically meaningful improvements. Also, when assessed at the
group level, patients treated with FX-322 outperformed the placebo
group in word recognition in a quiet background when averaged
across all time points.
The FX-322 Phase 2a study is a randomized, double-blind,
placebo-controlled, single- and repeat-dose study in which the
Company enrolled 95 patients aged 18 to 65 with mild to moderately
severe SNHL. The study uses validated measures of hearing function
including WR, WIN and pure tone audiometry.
Tinnitus and quality-of-life measures will be evaluated using
the Tinnitus Functional Index and the Hearing Handicap Inventory
for Adults, respectively, and the study also includes testing of
extended high frequencies (up to 16 kHz). The Phase 2a study has
four dose cohorts, and hearing function will be regularly tested
over the course of seven months following the first dosing.
The Company is conducting Phase 1b clinical studies to evaluate
the potential of FX-322 in additional patient groups, including
patients with age-related hearing loss and with severe SNHL. The
Company is also conducting an open-label safety study looking at
the administration conditions for FX-322.
A link to the publication, Improved Speech Intelligibility in
Subjects with Stable Sensorineural Hearing Loss Following
Intratympanic Dosing of FX-322 in a Phase 1b Study (W.J. McLean,
et. al.) can be found here. The Editor-in-Chief of Otology and
Neurotology is Lawrence Lustig, M.D., Chair, Department of
Otolaryngology - Head & Neck Surgery at Columbia
University.
About Frequency Therapeutics
Frequency Therapeutics is a leader in the development of
medicines designed to activate progenitor cells within the body to
treat degenerative diseases. The Company’s progenitor cell
activation (PCA) approach stimulates progenitor cells to create
functional tissue with the aim of developing disease modifying
therapies. The Company’s lead product candidate, FX-322, is
designed to regenerate auditory hair cells to restore hearing
function. FX-322 is being evaluated in multiple ongoing clinical
studies in patients with sensorineural hearing loss. The Company
also is evaluating additional diseases where its PCA approach could
create functional tissue, including in a pre-clinical program in
multiple sclerosis.
Headquartered in Woburn, Mass., Frequency has an ex-U.S. license
and collaboration agreement with Astellas Pharma Inc. for FX-322,
as well as additional collaboration and licensing agreements with
academic and nonprofit research organizations including
Massachusetts Eye and Ear, Mass General Brigham, the Massachusetts
Institute of Technology, The Scripps Research Institute and
Cambridge Enterprises Limited. For more information, visit
www.frequencytx.com and follow Frequency on Twitter
@Frequencytx.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including without limitation statements
regarding the Phase 2a clinical trial, the timing of top-line data
from the Phase 2a clinical trial, the timing and results of
top-line data from the Phase 1b studies in age-related hearing loss
and severe SNHL, the implications of the results of the Phase 1/2
data, the timing and results of the open-label safety study
evaluating the administration conditions for FX-322, the ability of
our technology platform to provide patient benefit, estimates of
the size of the hearing loss population and population at risk for
hearing loss, the Company’s ability to advance its hearing program
and further diversify its portfolio and the potential application
of the PCA platform to other diseases.
These forward-looking statements are based on management’s
current expectations. These statements are neither promises nor
guarantees, but involve known and unknown risks, uncertainties and
other important factors that may cause actual results, performance
or achievements to be materially different from any future results,
performance or achievements expressed or implied by the
forward-looking statements, including, but not limited to, the
following: the impact of COVID-19 on the Company’s ongoing and
planned clinical trials, research and development and manufacturing
activities, the relocation of the Company’s offices and laboratory
facilities, the Company’s business and financial markets; the
Company has incurred and will continue to incur significant losses
and is not and may never be profitable; the Company’s need for
additional funding to complete development and commercialization of
any product candidate; the Company’s dependence on the development
of FX-322; the unproven approach of the PCA platform; the lengthy,
expensive and uncertain process of clinical drug development and
regulatory approval; limited experience successfully obtaining
marketing approval for and commercializing product candidates; the
results of earlier clinical trials not being indicative of the
results from later clinical trials; differences between preliminary
or interim data and final data; adverse events or undesirable side
effects; disruptions at the FDA and other regulatory agencies;
failure to identify additional product candidates; new or changed
legislation; failure to maintain Fast Track designation for FX-322
and such designation failing to result in faster development or
regulatory review or approval; costly and damaging litigation,
including related to product liability or intellectual property or
brought by stockholders; dependence on Astellas Pharma Inc. for the
development and commercialization of FX-322 outside of the United
States; misconduct by employees or independent contractors;
reliance on third parties, including to conduct clinical trials and
manufacture product candidates; compliance with laws and
regulations, including healthcare and environmental, health, and
safety laws and regulations; failure to obtain, maintain and
enforce protection of patents and other intellectual property;
security breaches or failure to protect private personal
information; attracting and retaining key personnel; and ability to
manage growth.
These and other important factors discussed under the caption
“Risk factors” in the Company’s Form 10-Q filed with the Securities
and Exchange Commission (SEC) on November 16, 2020 and its other
reports filed with the SEC could cause actual results to differ
materially from those indicated by the forward-looking statements
made in this press release. Any such forward-looking statements
represent management’s estimates as of the date of this press
release. While the Company may elect to update such forward-looking
statements at some point in the future, it disclaims any obligation
to do so, even if subsequent events cause its views to change.
These forward-looking statements should not be relied upon as
representing the Company’s views as of any date subsequent to the
date of this press release.
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version on businesswire.com: https://www.businesswire.com/news/home/20210222005818/en/
Investor Contact: Carlo Tanzi, Ph.D. Kendall Investor Relations
Tel: 617-914-0008 ctanzi@kendallir.com
Media Contact: Suzanne Day Frequency Therapeutics Tel:
781-496-2211 Email: sday@frequencytx.com
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