Interim FX-322 Phase 2a Results Show Four
Injection Schedule Had No Discernible Hearing Benefit
Separate FX-322 Phase 1b Study Confirms Hearing
Improvement from Single Injection
Conference call at 8:30am ET today
Frequency Therapeutics, Inc. (Nasdaq: FREQ), today announced
topline, day-90 data from its FX-322 Phase 2a study (FX-322-202).
The interim results show that four weekly injections in subjects
with mild to moderately severe sensorineural hearing loss (SNHL)
did not demonstrate improvements in hearing measures versus
placebo. No treatment-related serious adverse events were observed
in the study. Frequency also announced new data from a parallel
study demonstrating hearing improvement from a single injection of
FX-322. The Company plans to advance further development of FX-322
as a single dose regimen.
FX-322 is Frequency’s lead product candidate, designed to
regenerate auditory sensory hair cells in the cochlea and improve
hearing in patients with SNHL.
In the four-arm Phase 2a study, 95 subjects aged 18-65 were
evaluated for hearing improvement using Word Recognition (WR),
Words-In-Noise (WIN), pure tone audiometry and additional
exploratory measures. All subjects were administered a total of
four weekly intratympanic injections comprised of zero, one, two,
or four doses of FX-322 with the balance of injections comprised of
placebo doses.
While WR scores increased across all groups, repeated weekly
injections appeared to dampen the hearing benefit observed compared
to other single-injection studies. The Phase 2a interim results
also showed an unexpected apparent level of hearing benefit in the
placebo group that did not occur in previous trials and exceeded
well-established published standards, potentially suggesting bias
due to trial design. Given these challenges observed in the Phase
2a study design, there was no discernible hearing benefit of FX-322
over placebo.
“Our FX-322 interim Phase 2a clinical results were unexpected,
especially given our prior published Phase 1/2 data. We have
already gained important learnings from this study that will inform
our future development efforts. The results clearly demonstrated
that a treatment regimen using four weekly injections is
unfavorable and we will continue to move ahead with single-dose
administrations in future studies. We believe in the potential of
FX-322 given the demonstrated hearing signal and favorable safety
profile observed with a single dose and in the future, we may
evaluate re-treatment at longer intervals,” said David L. Lucchino,
Frequency’s President and Chief Executive Officer.
The Company also announced preliminary results today from a
recently completed open-label, single-dose study of FX-322
(FX-322-111) designed to evaluate the impact of injection
conditions on tolerability. In the multi-center, randomized study,
subjects with mild to severe SNHL (n=33) were injected in one ear
with FX-322, with the untreated ear as the control. Hearing
function was tested over the course of 90 days following
dosing.
At day 90 following dosing, thirty-four percent (34%) of
subjects achieved a ten percent (10%) or greater absolute
improvement in WR scores in the treated ear, which was clinically
meaningful and statistically significant compared to the untreated
ear (p <0.05). This included a subset of subjects that more than
doubled their WR scores. The single dose had a favorable safety
profile and was well tolerated. These latest results are supportive
of the data reported in the prior single dose Phase 1/2
(FX-322-201) study, recently published in the peer-reviewed journal
Otology & Neurotology.
“We now have two independent, single-dose studies showing a
hearing signal with FX-322 and with statistically significant
improvements in speech intelligibility. More single dose studies
are underway to further understand the potential benefit of FX-322
in additional patient populations. Frequency is well resourced, and
with the support of our collaborator Astellas Pharma, we will
continue to advance development of potential hearing restoration
therapeutics for the millions of individuals impacted by
sensorineural hearing loss,” added Mr. Lucchino. “We are very
thankful to the people who participated in our studies and to the
staff at all of the sites for their contributions and continued
engagement, especially during the pandemic.”
FX-322 Ongoing Development Activities
Clinical development activities are ongoing to evaluate FX-322’s
clinical profile in patients with age-related hearing loss (ARHL)
and severe SNHL, to identify additional patient populations that
may benefit from this potentially restorative therapeutic
approach.
This includes a double-blind, placebo-controlled Phase 1b study
(FX-322-112) of subjects with ARHL (ages 66-85), which is fully
enrolled with 30 subjects. The primary objectives of the Phase 1b
ARHL study are to assess the local and systemic safety of a single
dose of FX-322 and evaluate hearing responses in an older adult
cohort. Study participants were randomized 4:1 to receive either
FX-322 or placebo in one ear. Validated measures of hearing
including WR, WIN and pure tone audiometry were used. Safety,
otologic and audiologic assessments were conducted at days 30 and
90 following administration of FX-322 or placebo. Frequency expects
to share results from this study in Q2 2021.
Frequency is also conducting an FX-322 Phase 1b study of up to
30 subjects ages 18-65 with severe SNHL (FX-322-113). This study,
which has a similar study design as the ARHL study, is continuing
to enroll subjects and results are anticipated in Q3 2021.
The Company plans to report final results of the Phase 2a study
in late Q2 2021.
About Sensorineural Hearing Loss
Sensorineural hearing loss is the most common form of hearing
loss, typically resulting from damage to auditory sensory hair
cells in the inner ear. These cells convert sound waves to a signal
sent to the brain. Sensory hair cells may be lost due to chronic
noise exposure, aging, certain viral infections or exposure to
drugs that are toxic to the ear. This type of damage impacts
hundreds of millions of individuals in the U.S. and worldwide.
Webcast Details
Webcast details can be found on the Frequency Therapeutics’
investor relations site at
https://investors.frequencytx.com/events-and-presentations, or by
clicking here to enter the webcast
directly.
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 subjects with sensorineural hearing loss. The Company is
also 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 interpretation and implications of the results of the
interim day-90 Phase 2a data and the FX-322-111 data, including
advancing FX-322 as a single-dose regimen and re-treatment at
longer intervals, the impact of the trial design of the Phase 2a
study on clinical data, the timing of results of the Company’s
clinical studies, the treatment potential of 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, the Company’s capital
resources, the license and collaboration with Astellas Pharma Inc.,
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/20210323005208/en/
Investor Contact: Carlo Tanzi, Ph.D. Kendall Investor Relations
Tel: 617-914-0008 Email: ctanzi@kendallir.com
Media Contact: Suzanne Day Frequency Therapeutics Tel:
781-496-2211 Email: sday@frequencytx.com
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