Enrollment Completed for Phase 2b Study of
FX-322 for the Treatment of Sensorineural Hearing Loss; Readout
Expected in Q1 2023
Company on Track for Q4 Clinical Trial Start of
its Second Hearing Restoration Program, FX-345
Multiple Sclerosis Remyelination Program
Candidate Advanced to IND-Enabling Studies, with Anticipated 2H
2023 IND Filing
Frequency Therapeutics, Inc. (Nasdaq: FREQ), a clinical-stage
regenerative medicine company focused on developing therapeutics to
activate a person’s innate potential to restore function, today
announced business updates and financial results for the third
quarter ended September 30, 2022.
“This past quarter has been one of momentum and execution. With
the completion of enrollment for the Phase 2b study of our lead
therapeutic candidate, FX-322, we are now preparing for a data
readout next quarter for what may be the first medicine to treat
hearing loss. The FX-322-208 study is designed to show a
statistically significant improvement in speech perception, and its
pre-specified endpoint would also demonstrate a clinically
meaningful outcome for patients. We believe the pending FX-322 data
have the potential to support a future marketing application and
the design of a subsequent Phase 3 study,” said David L. Lucchino,
Frequency’s chief executive officer.
Mr. Lucchino continued: “We continue to progress with our second
hearing restoration candidate, FX-345, and anticipate initiating a
Phase 1b trial in the coming weeks. With a readout planned for the
second half of next year, we look forward to exploring the impact
of greater cochlear drug exposure and whether that will enable us
to expand the SNHL populations we may treat. Finally, our
remyelination in multiple sclerosis program is also progressing in
IND-enabling studies and we remain on track to move this high
potential program toward an IND filing in the second half of 2023.
We expect our current cash position will enable us to reach our
next set of clinical milestones as we work to continue to advance
our pipeline and expand our research focus.”
The Company will review its FX-322 clinical program and other
pipeline activity on a webcast with management and key opinion
leaders on Dec. 13, 2022, at 8am ET.
Recent Pipeline Progress and Corporate
Highlights
FX-322-208 Phase 2b Study in Acquired Sensorineural Hearing
Loss (SNHL): In October, Frequency announced enrollment
completion for its FX-322-208 study. FX-322-208 is a randomized,
placebo-controlled, multi-center study designed to evaluate the
efficacy of a single administration of FX-322 on speech perception.
The study enrolled 142 participants, exceeding the original
enrollment target of 124. The FX-322-208 Phase 2b study enrolled
subjects with hearing loss associated with either noise-induced or
sudden SNHL, indications where the Company observed the greatest
concentration of speech perception improvements in prior studies.
The study’s pre-specified primary efficacy endpoint is the
improvement in a measure of speech perception, the ability to hear
more words correctly. The Company has aligned with the US Food and
Drug Administration (FDA) on this endpoint. FX-322-208 study
results are expected in Q1 2023.
The FX-322-208 study is powered at 80% (significance level of
0.05) to observe a statistically significant and clinically
meaningful improvement in speech perception at Day 90 following
dosing, with study responders defined as individuals exceeding the
upper 95% confidence interval in the speech perception test. The
Company has not publicly disclosed the specific test used for the
primary endpoint to maximize the rigor of the study and mitigate
potential bias.
During the study, subjects participate in a range of audiologic
exams, including pure-tone audiometry, word recognition in quiet,
word recognition in noise, the Tinnitus Functional Index (TFI), as
well as multiple patient-reported outcome measures. The study’s
rigorous design includes a lead-in phase with multiple baseline
measures and subjects with instability of baseline tests were
disqualified from participation in the study. More than 200
individuals have been dosed with a single injection of FX-322 in
prior or ongoing studies, and the drug candidate has continued to
exhibit a favorable safety profile with no drug-related serious
adverse events.
FX-345, a Second Program for SNHL: In the coming weeks,
the Company plans to initiate a Phase 1b study in subjects with
SNHL to assess safety, tolerability and preliminary impact on a
number of audiometric measures. The Company anticipates study
results in H2 2023. FX-345 is the Company’s second investigational
therapeutic candidate for SNHL. Pharmacokinetic measures and human
modeling data suggest that administration of FX-345 can result in
therapeutically active drug levels in a larger portion of the
cochlea, which could potentially address a broader set of patients
with SNHL.
Pre-clinical Program for Remyelination in Multiple Sclerosis
(MS): Frequency is advancing to IND-enabling studies its
program for remyelination in MS. The Company previously reported
that it had identified a new biological target relevant to
myelination and demonstrated that modulation of this target drives
robust oligodendrocyte differentiation and expression of myelin
proteins in vitro. The Company has identified multiple novel
chemical entities that induce robust remyelination in an in vivo
animal model and plans to file an IND for the program in H2
2023.
Sublease Agreement: On July 8, 2022, the Company entered
into a two-year agreement to sublease excess laboratory and office
space, significantly reducing expenses. The Company has sufficient
laboratory and other workspace for its teams and to support
upcoming milestones and future plans.
Third Quarter 2022 Financial
Results
Cash Position: Cash, cash equivalents and marketable
securities as of September 30, 2022, were $99.3 million (excluding
restricted cash). The Company is believed to be appropriately
resourced to advance its pipeline of potential first-in-class
treatments through key development milestones, including completion
of the Phase 2b study of FX-322, a Phase 1b study of FX-345 and a
Phase 1 study for the MS program.
Based on current plans and assumptions, the Company believes its
existing cash, cash equivalents and marketable securities will be
sufficient to fund its operations into 2024. This guidance does not
include potential future milestones which could be received from
Astellas Pharma Inc. (Astellas) for continued FX-322
development.
Revenue: The $80.0 million upfront payment from Astellas,
initially recorded as deferred revenue, was fully recognized as of
June 30, 2021. As such, no revenue was recorded for the three and
nine months ended September 30, 2022, compared to $0 and $14.1
million in the comparable periods of 2021.
Research and Development Expenses: Research and
development expenses were $11.7 million for the three months ended
September 30, 2022, as compared to $15.7 million for the comparable
period of 2021. Research and development expenses were $38.8
million for the nine months ended September 30, 2022, as compared
to $48.2 million for the comparable period of 2021. Excluding
stock-based compensation expense of $2.1 million for the three
months ended September 30, 2022, and $6.0 million for the nine
months ended September 30, 2022, research and development expenses
for the three and nine months ended September 30, 2022, were $9.6
million and $32.8 million, respectively.
General and Administrative Expenses: General and
administrative expenses were $8.6 million for the three months
ended September 30, 2022, as compared to $9.3 million for the
comparable period of 2021. General and administrative expenses were
$26.0 million for the nine months ended September 30, 2022, as
compared to $28.6 million for the comparable period of 2021.
Excluding stock-based compensation expense of $3.5 million for the
three months ended September 30, 2022, and $9.4 million for the
nine months ended September 30, 2022, general and administrative
expenses for the three and nine months ended September 30, 2022,
were $5.1 million and $16.6 million, respectively.
Net Loss: Net loss was $19.5 million for the three months
ended September 30, 2022, as compared to $25.2 million for the
comparable period of 2021. Net loss was $64.2 million for the nine
months ended September 30, 2022, as compared to $63.2 million for
the comparable period of 2021.
About Frequency Therapeutics
Frequency Therapeutics is leading a new category in regenerative
medicine that aims to restore human function – first in hearing
loss and then in multiple sclerosis – by developing therapeutics
that activate a person’s innate regenerative potential within the
body through the activation of progenitor cells. Frequency’s
hearing research focuses on cochlear restoration and auditory
repair, and its lead asset, FX-322, is a small-molecule combination
product candidate that is the first to show statistically
significant and clinically meaningful hearing improvements in
clinical trials for sensorineural hearing loss. Frequency is also
following early restorative signals in MS to develop medicines with
similar underlying regenerative science being brought to hearing
loss.
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, the
Massachusetts Institute of Technology, and the Scripps Research
Institute.
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 timing and design of the Phase 2b study (FX-322-208),
including the timing of results, the ability of design features to
reduce bias, and the indications of the enrolled subjects, the
commencement of any future FX-322 trials, the interpretation and
implications of the results and learnings of other FX-322 clinical
studies, the timing of future trials of FX-345, including the
timing of results, the timing of IND submission in the MS
remyelination program, the treatment potential of FX-322, FX-345,
and the MS remyelination program, estimates of the size of the
hearing loss population, the acceptance by the FDA of particular
endpoints in the Company’s trials, the sufficiency of the Company’s
laboratory and workspaces, the potential application of the
progenitor cell activation (PCA) platform to other diseases, and
the sufficiency of the Company’s capital resources.
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 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 and the
inability to identify additional potential product candidates; the
lengthy, expensive and uncertain process of clinical drug
development and regulatory approval; the Company’s 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; ability to seek and receive Breakthrough Therapy
designation for FX-322; the Company’s ability to enroll and retain
patients in clinical trials; 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 changing laws and
regulations, including healthcare and environmental, health, data
privacy and safety laws and regulations; failure to obtain,
maintain and enforce protection of patents and other intellectual
property rights covering product candidates; security breaches or
failure to protect private personal information; attracting and
retaining key personnel; and the Company’s 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 8, 2022 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 September
30,
Nine Months Ended September
30,
2022
2021
2022
2021
Revenue
$
—
$
—
$
—
$
14,068
Operating expenses:
Research and development
11,715
15,662
38,769
48,169
General and administrative
8,560
9,328
26,037
28,571
Total operating expenses
20,275
24,990
64,806
76,740
Loss from operations
(20,275
)
(24,990
)
(64,806
)
(62,672
)
Interest income
351
172
871
315
Interest expense
(263
)
(182
)
(649
)
(582
)
Realized gain (loss) on investments
1
(9
)
3
(23
)
Foreign exchange (loss) gain
(5
)
(4
)
(7
)
16
Other income (expense), net
621
(139
)
361
(227
)
Loss before income taxes
(19,570
)
(25,152
)
(64,227
)
(63,173
)
Income tax benefit (provision)
23
(3
)
9
(13
)
Net loss
$
(19,547
)
$
(25,155
)
$
(64,218
)
$
(63,186
)
Net loss per share attributable to common
stockholders-basic and diluted
$
(0.55
)
$
(0.73
)
$
(1.83
)
$
(1.84
)
Weighted-average shares of common stock
outstanding-basic and diluted
35,247,680
34,448,746
35,013,189
34,268,736
Frequency Therapeutics,
Inc.
Consolidated Balance Sheet
Data
(in thousands)
(unaudited)
September 30, 2022
December 31, 2021
Cash, cash equivalents and marketable
securities
99,347
142,426
Working capital
80,987
123,319
Total assets
136,013
185,358
Total liabilities
54,040
54,534
Accumulated deficit
(244,303
)
(180,085
)
Total stockholders' equity
81,973
130,824
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221108005739/en/
Investor Contact: Carlo Tanzi, Ph.D. Kendall Investor Relations
ctanzi@kendallir.com 617-914-0008
Media Contact: Frequency Therapeutics Email:
media@frequencytx.com
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