Enrollment Underway for FX-322-208 Phase 2b
Study in Subjects with Sudden and Noise-Induced Sensorineural
Hearing Loss; Aligns with FDA on Speech Perception as Primary
Endpoint for FX-322 Studies
Expands Pipeline with New Pre-Clinical Program
for Hearing Restoration that May Provide Broader Cochlear Exposure
and the Potential to Address Expanded SNHL Patient Populations
Introduces Remyelination Program in Multiple
Sclerosis (MS), Sharing In Vivo Data Showing Substantially Greater
Activity than Biologic or Small Molecule Comparators
Frequency Therapeutics, Inc. (Nasdaq: FREQ), a clinical-stage
regenerative medicine company focused on developing therapeutics to
activate a person’s innate regenerative potential to restore
function, today announced business updates and financial results
for the third quarter ended September 30, 2021.
“Frequency has made significant operational progress throughout
this year, highlighted by the initiation of a Phase 2b study
(FX-322-208) of FX-322, our lead program for sensorineural hearing
loss (SNHL). We have designed this study following careful analysis
of multiple completed FX-322 exploratory clinical studies, and
through this work, we’ve increased our understanding of the
specific patient etiologies and severities where hearing benefits
were observed. We have also reached alignment with the U.S. Food
and Drug Administration (FDA) on speech perception as the primary
efficacy endpoint that we believe helps de-risk future development
activities,” said David L. Lucchino, Frequency’s Chief Executive
Officer.
“At our recent R&D event, we detailed results from our
completed FX-322 clinical studies, showing all of the data
demonstrating a clear signal of clinical benefit in a significant
segment of individuals with SNHL. We also showed how the breadth of
our clinical efforts informed the inclusion criteria of our ongoing
FX-322-208 Phase 2b study. Furthermore, we were excited to
introduce two new research programs, the first a pre-clinical
development candidate to treat SNHL that can reach deeper into the
cochlea and which may provide the opportunity to treat different
patient populations. The second is a novel candidate for
remyelination in MS that has shown significant remyelinating effect
in vivo and has the potential to provide a highly differentiated
offering compared to currently approved MS treatments.”
FX-322 Clinical Study Overview and Recent Corporate
Highlights
FX-322-208 Phase 2b Study in Acquired SNHL: In October,
Frequency announced that the first subject was dosed in a new Phase
2b study of acquired SNHL. FX-322-208 is a randomized,
placebo-controlled, multi-center study designed to evaluate the
impact of a single administration of FX-322 on speech perception in
approximately 124 subjects with SNHL.
The study’s primary endpoint is speech perception, a measure of
sound clarity and understanding speech. FX-322-208 will include
subjects with hearing loss associated with either noise-induced or
sudden SNHL. The FDA, in a recent Type-C meeting with the Company,
agreed that speech perception is an acceptable primary efficacy
endpoint. A variety of other listening tests, including multiple
measures of speech perception and pure tone thresholds, will also
be assessed. Based on learnings from prior studies, extensive study
design elements have been included in FX-322-208 to mitigate
potential bias and help ensure the consistency of baseline hearing
assessments.
Since 2018, Frequency has run exploratory learning studies with
the aim of determining the optimal patient populations where FX-322
may restore lost hearing. Given the diversity of etiologies and
severities and the heterogeneity of hearing loss, this
comprehensive approach was utilized to narrow in on the target
population for further development. Data from these studies has
enabled Frequency to design the FX-322-208 study inclusive of the
hearing loss etiologies and severities where speech perception
improvements were observed in prior FX-322 clinical studies.
To date, 169 individuals have been dosed with FX-322 across
previous studies and no drug-related serious adverse events have
been reported. The Company is also planning in Q4 2021 to report
data from its FX-322-113 Phase 1b study in subjects with severe
SNHL.
Introduction of FX-345, a Second Sensory Hair Cell
Regeneration Program for SNHL: In November, Frequency announced
a new, highly potent, investigational therapeutic candidate for
SNHL called FX-345. 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 would enable Frequency to explore the potential for
a hearing restoration therapeutic that could address a broad set of
hearing loss severities and etiologies. FX-345 is a combination of
two small molecules, including a more potent GSK3 inhibitor. An
investigational new drug (IND) application for FX-345 is
anticipated in Q2 2022.
Pre-clinical Program for Remyelination in Multiple
Sclerosis: In November, Frequency also announced a discovery
effort to remyelinate neurons in the central nervous system to
treat MS. The Company has 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.
Frequency developed multiple novel chemical entities that induce
robust remyelination in vivo, including FREQ-162, which is its most
advanced compound to date. A single dose of FREQ-162 induces robust
increases in oligodendrocyte differentiation throughout the brain
and significant remyelination in extensively demyelinated, aged
mice. The Company is progressing these assets in dose range finding
studies and IND-enabling toxicology studies in order to advance the
program into clinical trials. FREQ-162 is one of several compounds
Frequency has discovered that show potent remyelination activity.
Remyelination is believed to slow or even reverse progression of
disability in MS patients.
November 9 R&D Event: Frequency hosted an investor
event on November 9, where management and key opinion leaders
reviewed detailed FX-322 clinical study results and discussed the
design of the ongoing FX-322-208 Phase 2b study. Management also
presented research findings for FX-345 and its remyelination
program. An archived replay of the webcast is available on the
Investors & Media section of the Frequency Therapeutics
website.
Third Quarter 2021 Financial Results
Cash Position: Cash, cash equivalents and marketable
securities as of September 30, 2021 were $160.5 million (excluding
restricted cash), as compared to $220.3 million as of December 31,
2020. Based on current plans and assumptions, the Company expects
its existing cash, cash equivalents and marketable securities will
be sufficient to fund its operations into late 2023. This guidance
does not include potential future milestones which could be
received from Astellas for continued FX-322 development.
Revenue: Revenue was $14.1 million for the nine months
ending September 30, 2021 compared to $27.0 million in the
comparable period of 2020. The Company had no revenue in the three
months ending September 30, 2021 compared to $11.2 million in the
comparable period of 2020.
Research & Development Expenses: Research and
development expenses were $15.7 million and $48.2 million for the
three and nine month periods ending September 30, 2021,
respectively, as compared to $10.2 million and $25.6 million for
the comparable periods 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 $2.8 million and $7.4 million
for the three and nine months ending September 30, 2021, research
and development expenses for the three and nine months ending
September 30, 2021 were $12.9 million and $40.8 million,
respectively.
General and Administrative Expenses: General and
administrative expenses were $9.3 million and $28.6 million for the
three and nine months ending September 30, 2021, respectively, as
compared to $6.5 million and $18.7 million for the comparable
periods 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 and $9.2 million for the three and nine
months ending September 30, 2021, general and administrative
expenses for the three and nine months ending September 30, 2021
were $6.2 million and $19.4 million, respectively.
Net Loss: Net loss was $25.2 million and $63.2 million
for the three and nine months ending September 30, 2021, as
compared to $5.3 million and $16.3 million for the comparable
periods 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 leading a new category in regenerative
medicine that aims to restore 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 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 the same
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, 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 design of the new Phase 2b trial of FX-322, including
the type of SNHL that the enrolled patients will have and the
ability of design features to reduce bias, the interpretation and
implications of the results and learnings of other FX-322 clinical
studies, the acceptance by the FDA of particular endpoints in the
Company’s trials, the treatment potential of FX-322, FX-345, and
the novel approach for remyelination in multiple sclerosis, the
timing and progress of the FX-345 and remyelination programs, the
ability of our technology platform to provide patient benefit, the
ability to continue to develop our Progenitor Cell Activation (PCA)
platform and identify additional product candidates, the potential
application of the PCA platform to other diseases, the license and
collaboration with Astellas Pharma Inc., 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 15, 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 September
30,
Nine Months Ended September
30,
2021
2020
2021
2020
Revenue
$
—
$
11,247
$
14,068
$
27,034
Operating expenses:
Research and development
15,662
10,153
48,169
25,587
General and administrative
9,328
6,512
28,571
18,720
Total operating expenses
24,990
16,665
76,740
44,307
Loss from operations
(24,990
)
(5,418
)
(62,672
)
(17,273
)
Interest income
172
74
315
962
Interest (expense)
(182
)
—
(582
)
—
Realized (loss) gain on investments
(9
)
—
(23
)
65
Foreign exchange (loss) gain
(4
)
18
16
27
Other (expense), net
(139
)
—
(227
)
—
Loss before income taxes
(25,152
)
(5,326
)
(63,173
)
(16,219
)
Income taxes
(3
)
(15
)
(13
)
(60
)
Net loss
$
(25,155
)
$
(5,341
)
$
(63,186
)
$
(16,279
)
Net loss per share attributable to common
stockholders-basic and diluted
$
(0.73
)
$
(0.16
)
$
(1.84
)
$
(0.51
)
Weighted-average shares of common stock
outstanding-basic and diluted
34,448,746
33,073,889
34,268,736
31,729,702
Frequency Therapeutics,
Inc.
Consolidated Balance Sheet
Data
(in thousands)
(unaudited)
September 30, 2021
December 31, 2020
Cash, cash equivalents and marketable
securities
$
160,471
$
220,341
Working capital
139,424
198,430
Total assets
202,185
264,722
Total liabilities
55,069
72,231
Accumulated deficit
(158,585
)
(95,399
)
Total stockholders' equity
147,116
192,491
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211115005072/en/
Investor: Carlo Tanzi, Ph.D. Kendall Investor Relations
ctanzi@kendallir.com 617-914-0008 Media: Suzanne Day Frequency
Therapeutics sday@frequencytx.com 781-496-2211
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