Data From Recent FX-322 Clinical Readouts,
Including New Data from Phase 1b Study in Presbycusis (Age-Related
Hearing Loss), Further Clarify Acquired Sensorineural Hearing Loss
Target Patient Populations and Future Study Design
Company Anticipates Initiation of New FX-322
Phase 2 Study in H2 2021
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 business updates and
financial results for the first quarter ended March 31, 2021.
“As we work to develop a novel medicine for the treatment of the
most common form of hearing loss, our initial focus has been to
investigate the broadest set of potential conditions and severities
through a comprehensive series of probing studies. With data from
all of our single administration studies, we have been able to
better define the groups of patients that we believe are responding
to FX-322, while also informing the optimal study design for new
placebo-controlled Phase 2 trials,” said David L. Lucchino,
Frequency’s Chief Executive Officer.
Most recently, Frequency completed a Phase 1b study (FX-322-112)
in subjects with presbycusis (age-related hearing loss). No
subjects enrolled in the 112 study had either noise-induced or
sudden sensorineural hearing loss (SSNHL), conditions where FX-322
associated hearing benefits were observed in prior studies. In the
112 study, the Company observed no significant treatment effect
with FX-322 administration compared to placebo. Results showed a
favorable safety and tolerability profile and, importantly, that
the inclusion of multiple baseline hearing tests prior to treatment
mitigated the impact of potential study bias (e.g. placebo
response) previously observed in Frequency’s Phase 2a study
(FX-322-202). This lead-in approach was also used in the Company’s
recently reported open-label study of subjects with mild-to-severe
SNHL (FX-322-111). The Company expects to utilize study lead-in
measures, and other approaches, to minimize potential bias in
future clinical studies.
“To date, Frequency has shown statistically significant hearing
benefits in multiple, independent FX-322 studies and these hearing
benefits have been observed in subjects diagnosed with sudden
sensorineural or noise-induced hearing loss. Based on the available
clinical data, we are able to continue to refine the targeted
patient populations we expect to include in our next set of
clinical studies, populations that include millions of
individuals,” Mr. Lucchino concluded.
Clinical Study Overview and Recent Corporate
Highlights
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.
In February, findings from Frequency’s Phase 1/2 study of FX-322
were published in the journal Otology & Neurotology, which
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
and the level of background noise in which words could be
identified in the WIN test.
These effects also were observed to be durable in four out of
the five responders in the study 13 – 21 months following initial
administration. In the study, there were no meaningful changes in
the WR and WIN scores of the placebo group and FX-322 was also
shown to be well tolerated. The publication also includes data
demonstrating consistent cochlear drug delivery in both preclinical
and human studies.
The Company’s clinical development strategy has been to build
off these data and subsequent findings regarding delivery and
durability, conducting exploratory studies in subjects with
different etiologies and severities of SNHL in order to best
understand the population that may be best treated by FX-322.
FX-322-112 Phase 1b Study in Presbycusis (Age-Related Hearing
Loss or ARHL): The recently completed study was a double-blind,
placebo-controlled, randomized, multicenter safety study of 30
individuals aged 66-85 with age-related hearing loss. The primary
objectives of the study were 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 hearing
measures, as well as safety, otologic and audiologic assessments
were also evaluated in the study. By design, the study recruited
subjects with no medical history of noise-induced or sudden
sensorineural hearing loss (SSNHL), etiologies where FX-322
associated hearing benefits were observed in prior studies. This
was done as the Company continues to separately evaluate subjects
with specific forms of hearing loss to better refine cohorts for
future studies. While the treatment effect was not significant
compared to placebo, results from the FX-322-112 study showed a
favorable safety and tolerability profile, no treatment-related
serious adverse events were reported and no meaningful improvements
in the placebo group were observed.
FX-322-111 Phase 1b Study (Open-Label Administration
Study): In March 2021, Frequency announced data from a
multi-center, randomized open-label study of subjects with mild to
severe SNHL (n=33) designed to evaluate the impact of injection
conditions on tolerability. Subjects 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,
thirty-four percent (34%) of the 32 subjects that completed the
study achieved a ten percent (10%) or greater absolute improvement
in WR scores in the treated ear, which was statistically
significant compared to the untreated ear. 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.
FX-322-202 Phase 2a Study in Mild to Moderately Severe
SNHL: In March 2021, the Company announced interim day-90
topline study results. No treatment-related serious adverse events
were observed in the study. While WR scores increased across all
groups, repeated weekly injections appeared to dampen the hearing
benefit observed compared to other single-injection studies and
there was no discernible hearing benefit of FX-322 over placebo.
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. The Company
expects to utilize lead-in hearing measures, and other study design
approaches, in future studies to minimize the risk of study bias.
The Company expects to report complete results of the study in late
Q2 2021.
FX-322-113 Phase 1b Study in Severe SNHL: In November
2020, Frequency commenced a Phase 1b study in patients aged 18-65
with severe SNHL. Enrollment remains ongoing. The primary
objectives of the study are to assess the local and systemic safety
of a single dose of FX-322 and evaluate hearing responses in a more
severe adult cohort. Study participants are randomized 4:1 to
receive either FX-322 or placebo in one ear. Validated measures of
hearing including WR, the Bamford-Kowal-Bench Speech-in-Noise
(BKB-SIN) test and pure tone audiometry are utilized in the study.
Safety, otologic and audiologic assessments are being conducted at
days 30 and 90 following administration of FX-322 or placebo.
Frequency expects to obtain topline results from this study in the
second half of 2021.
Expanded Clinical Development Team: In April 2021, the
Company announced the expansion of its clinical development team
with the addition of Jeffery T. Lichtenhan, Ph.D., a leading expert
in hearing diagnostics and measurement. He joins Frequency from the
Washington University School of Medicine in St. Louis, where he was
Assistant Professor of Otolaryngology and Audiology and
Communication Sciences. In February 2021, Frequency previously
announced the appointment of Kevin Franck, Ph.D., who joined the
Company as Senior Vice President of Strategic Marketing and New
Product Planning from Massachusetts Eye and Ear, where he served as
Director of Audiology and was on the Harvard Medical School Faculty
of the Department of Otolaryngology-Head and Neck Surgery.
Multiple Sclerosis Preclinical Program: Frequency
continues to advance preclinical research efforts designed to
repair neurological damage in patients with multiple sclerosis.
Research efforts remain underway to confirm the optimal candidate
for a future clinical program.
First Quarter 2021 Financial Results
Cash Position: Cash, cash equivalents and short-term
investments on March 31, 2021 were $194.9 million, as compared to
$220.3 million on December 31, 2020. Excluding cash payments of
approximately $2.0 million made during the first quarter ended
March 31, 2021 related to fixed assets and the buildout of the
Company’s new Lexington facility, cash, cash equivalents, and
short-term investments at March 31, 2021 would have been $196.9
million. Based on current plans and assumptions, the Company
expects its existing cash, cash equivalents and short-term
investments will be sufficient to fund its operations into 2023.
This guidance does not include potential future milestones which
could be received from Astellas for continued FX-322
development.
Revenue: Revenue was $4.7 million for the first quarter
of 2021. The Company had revenue of $7.3 million in the comparable
period of 2020.
Research & Development Expenses: Research and
development expenses were $15.1 million for the first quarter of
2021, as compared to $6.7 million for the first quarter of 2020.
The increase was due to increased costs related to the Company’s
lead product candidate, FX-322, including external development
costs related to the Company’s ongoing trials for FX-322, as well
as increased personnel-related costs due to additional headcount to
support the growth of Frequency’s research and development
organization. Excluding stock-based compensation expense of $1.5
million, research and development expenses for the three months
ended March 31, 2021 were $13.6 million.
General and Administrative Expenses: General and
administrative expenses were $9.7 million for the first quarter of
2021, as compared to $6.2 million for the first quarter of 2020.
The increase was primarily due to an increase in personnel-related
costs, including stock-based compensation, for additional headcount
required to support the growth of the Company as well as costs
associated with being a public company, primarily comprised of
professional fees. Excluding stock-based compensation expense of
$3.1 million, general and administrative expenses for the three
months ended March 31, 2021 were $6.6 million.
Net Loss: Net loss was $20.4 million for the first
quarter of 2021, as compared to $4.9 million for the first quarter
of 2020. The increase in net loss reflects the increase in research
and development costs associated with the growth of Frequency’s
research and development organization as well as the increase in
general and administrative expenses required to support the growth
of Frequency as a public company.
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 Lexington, 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,
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, the FX-322-112 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, including timing of
initiation of additional planned studies in FX-322, 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 timing of the Company’s remyelination program, the
sufficiency of 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 May 13, 2021 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.
Frequency Therapeutics,
Inc.
Consolidated Statements of
Operations
(in thousands, except share
and per share amounts)
(unaudited)
Three Months Ended March
31,
2021
2020
Revenue
$
4,651
$
7,264
Operating expenses:
Research and development
15,106
6,670
General and administrative
9,744
6,249
Total operating expenses
24,850
12,919
Loss from operations
(20,199
)
(5,655
)
Interest income
25
710
Interest (expense)
(218
)
—
Realized (loss) gain on investments
(4
)
69
Foreign exchange gain
21
1
Loss before income taxes
$
(20,375
)
$
(4,875
)
Income taxes
—
(38
)
Net loss
$
(20,375
)
$
(4,913
)
Net loss per share attributable to common
stockholders-basic and diluted
$
(0.60
)
$
(0.16
)
Weighted-average shares of common stock
outstanding-basic and diluted
34,115,682
30,868,220
Frequency Therapeutics,
Inc.
Consolidated Balance Sheet
Data
(in thousands)
(unaudited)
March 31, 2021
December 31, 2020
Cash, cash equivalents and short-term
investments
$
194,909
$
220,341
Working capital
181,159
198,430
Total assets
241,511
264,722
Total liabilities
64,104
72,231
Accumulated deficit
(115,774
)
(95,399
)
Total stockholders’ equity
177,407
192,491
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210513005190/en/
Investor Contact: Carlo Tanzi, Ph.D. Kendall Investor Relations
ctanzi@kendallir.com 617-914-0008
Media Contact: Suzanne Day Frequency Therapeutics Tel:
781-496-2211 Email: sday@frequencytx.com
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