Recent FX-322 Study Readouts Support
Single-Dose Administration as Part of Future Trial Designs; Company
Planning Additional Single Injection Studies
Results from Exploratory FX-322 Phase 1b
Studies in Patients with Age-Related and Severe Hearing Loss
Anticipated in Q2 and Q3 Respectively
Company Ends Year with $220 Million in
Unrestricted Cash Providing Runway Into 2023
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 fourth quarter and year ended December
31, 2020.
“We recently obtained valuable data from two clinical studies in
patients with sensorineural hearing loss that have provided us with
important learnings that already are informing our future
development plans for FX-322, specifically around trial design and
using a single administration approach in our studies,” said David
L. Lucchino, Frequency’s President and CEO. “Combined with data
from our published Phase 1/2 results, we have now shown
statistically significant and clinically meaningful improvements in
speech intelligibility from two independent, single injection
studies that we believe provide encouraging support for FX-322 as a
potential novel treatment option as we drive forward in this new
area of regenerative medicine.”
Mr. Lucchino continued: “Upcoming readouts from our ongoing
studies will be further clarifying, and we will use these insights
as we plan for future placebo-controlled FX-322 clinical studies
using a single administration regimen. We have utilized a
multi-study development approach that enables us to continually
learn more about the clinical profile of FX-322, its potential
application in different potential populations, as well as how to
carry out trials most effectively in this emerging therapeutic
area. In Q2, we anticipate results of a FX-322 Phase 1b study in
patients with age-related hearing loss and in Q3 results of a
FX-322 Phase 1b study of patients with severe sensorineural hearing
loss, which will further inform our path forward. There is a
tremendous need to advance the standard of care for people with
sensorineural hearing loss, and we are grateful to the patient
community and all of those that have participated in our clinical
studies for their continued support as we work to advance a
medicine for hearing restoration.”
FX-322 Day-90 Study Detail and Ongoing Program
- FX-322 Phase 2a Study Day 90 Topline Results: The
Company recently shared interim data from its four-arm, Phase 2a
study of FX-322 conducted in 95 subjects aged 18-65 with mild to
moderately severe sensorineural hearing loss (SNHL). In the study,
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. Subjects
were evaluated for hearing improvement using Word Recognition (WR),
Words-In-Noise (WIN), pure tone audiometry and additional
exploratory measures. 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, 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. No treatment-related serious adverse events were
observed in the study.
- FX-322 - 111 Phase 1b: The Company also shared
preliminary data 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. These data were based on results from 32
of 33 subjects that completed the 90-day clinical assessment
period. The single dose had a favorable safety profile and was well
tolerated.
- FX-322 - 112 Phase 1b Study in Age-Related Hearing Loss:
In October 2020, Frequency commenced a Phase 1b safety study of
FX-322 (FX-322-112) for individuals with age-related hearing loss.
The study, which has completed enrollment, is 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 Phase 1b 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 are being evaluated. Safety, otologic and audiologic
assessments are also being conducted at day 30 and 90 following
administration of FX-322 or placebo. Frequency expects to obtain
topline results from this study in 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 (FX-322-113). Enrollment is continuing for the
study of up to 30 individuals. This study employs a similar design
and endpoints to the ongoing age-related Phase 1b study. Frequency
expects to obtain topline results from this study in Q3 2021.
Other FX-322 Program Highlights
- FX-322 Phase 1/2 Publication and Presentation: In
February 2021, the results from the completed FX-322 Phase 1/2
study were published in Otology & Neurotology, a leading
peer-reviewed journal focused on disorders of the ear. The data
demonstrate hearing improvements in adults with acquired SNHL, and
the first known linkage of pharmacokinetics and pharmacodynamics
for a potential hearing restoration therapy. The Phase 1/2 study
data were also presented at the leading international hearing
research conference, the Association for Research in Otolaryngology
(ARO) 44th Annual Midwinter Meeting on February 22, 2021.
- Key Opinion Leader Webcast on Potential for Restorative
Treatments for SNHL: In January 2021, Company management and
leading hearing loss researchers and clinicians held an investor
event to discuss current interventions for the treatment of
acquired sensorineural hearing loss (SNHL), unmet medical needs for
hearing loss patients and the potential of FX-322 to transform the
current standard of care.
- FX-322 Phase 1/2 Durability Data: In September 2020 at
the American Academy of Otolaryngology – Head and Neck Surgery
(AAO-HNS) Annual Meeting, data from a Phase 1/2 follow-up study
showed that some patients with chronic SNHL had maintained
significant improvements in key measures of hearing following a
single injection of FX-322. Susan King, M.D., FACS, the lead
investigator on both studies, outlined data which showed sustained
improvements 13 – 21 months after initial dosing in measures of
speech intelligibility (clarity) and in some patients, increased
audibility (volume) threshold values at the highest tested
frequency.
- Clinical Data from Exploratory Study Confirming Delivery of
FX-322 to the Cochlea: In May 2020, the Company announced
top-line data from an exploratory clinical study designed to show
whether drug levels of FX-322 in the cochlea can be directly
measured. In addition to confirming the viability of the approach,
study results showed measurable concentrations of FX-322 in every
sample analyzed and that anatomical factors did not prevent the
active agents of FX-322 from reaching the cochlea. Further, the
levels of FX-322 in the cochlea were predicted to reach the
therapeutically active range of the treatment.
2020 and Recent Business Activity:
- Expanded Leadership Team: The Company recently has made
several important leadership hires, building out its finance,
manufacturing and pre-commercial functions:
- Peter P. Pfreundschuh was appointed to the role of Chief
Financial Officer in December 2020 to oversee the Company’s
financial strategy and activities related to accounting, investor
relations, business development and business operations. He joined
Frequency from UroGen Pharma Ltd., where he served as Chief
Financial Officer, Chief Compliance Officer and Corporate
Secretary.
- Quentin McCubbin, Ph.D. was appointed as Chief Manufacturing
Officer in January 2021. Dr. McCubbin is overseeing Company
technical operations, leading drug product formulation, chemistry
manufacturing and controls (CMC) and supply chain, to support the
development of Frequency’s pipeline of product candidates. He
joined Frequency from Cerevel Therapeutics, a clinical-stage
biotechnology company where he served as Head of Technical
Operations.
- Kevin Franck, Ph.D. was appointed to the role Senior Vice
President of Strategic Marketing and New Product Planning in
February 2021. Dr. Franck will lead pre-commercial strategy and
launch planning for Frequency’s clinical pipeline. A licensed
audiologist, Dr. Franck joined Frequency from Massachusetts Eye and
Ear, where he served as Director of Audiology and was on the
Harvard Medical School faculty in the Department of
Otolaryngology-Head and Neck Surgery.
- Progress on Remyelination Program for Multiple
Sclerosis: The Company continues to advance preclinical
programs to support the advancement of a therapeutic candidate for
remyelination in multiple sclerosis into the clinic.
Fourth Quarter 2020 Financial Results
Cash Position: Cash, cash equivalents and short-term
investments on Dec. 31, 2020 were $220.3 million. Based on current
plans and assumptions, the Company expects its existing cash and
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
Pharma for continued FX-322 development.
Revenue: Revenue was $10.0 million and $37.0 million for
the three- and twelve-month periods ended Dec. 31, 2020,
respectively. The Company had revenue of $4.7 million and $28.9
million in each of the comparable periods of 2019.
Research & Development Expenses: Research and
development expenses were $11.8 million and $37.4 million for the
three- and twelve-month periods ended Dec. 31, 2020, respectively,
as compared to $6.2 million and $18.8 million for the comparable
periods of 2019. The increases are due to increased costs related
to the Company’s lead product candidate, FX-322, including external
development costs related to the Company’s ongoing Phase 2a
clinical trial, as well as increased personnel-related costs due to
additional headcount to support the growth of Frequency’s research
and development organization.
General and Administrative Expenses: General and
administrative expenses were $8.4 million and $27.1 million for the
three- and twelve-month periods ended Dec. 31, 2020, respectively,
as compared to $5.0 million and $14.8 million for the comparable
periods of 2019. The increases are 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 insurance, consulting and professional fees.
Net Loss: Net loss was $10.2 million and $26.5 million
for the three- and twelve-month periods ended Dec. 31, 2020,
respectively, as compared to $5.5 million and $18.7 million for the
comparable periods of 2019. The increase in net loss in 2020
reflects the increase in research and development costs associated
with the growth of Frequency’s research and development
organization and the increase in general and administrative
expenses required to support the growth of the Company and the cost
associated with operating 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 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 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-K filed with the Securities
and Exchange Commission (SEC) on March 29, 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)
Three Months Ended December
31,
Year Ended December
31,
2020
2019
2020
2019
Revenue
$
9,950
$
4,709
$
36,984
$
28,947
Operating expenses:
Royalty
—
—
—
16,000
Research and development
11,828
6,196
37,415
18,784
General and administrative
8,399
5,001
27,119
14,838
Total operating expenses
20,227
11,197
64,534
49,622
Loss from operations
(10,277
)
(6,488
)
(27,550
)
(20,675
)
Interest income
32
942
994
1,784
Realized gain on investments
19
50
84
138
Foreign exchange gain (loss)
(31
)
3
(4
)
7
Loss before income taxes
(10,257
)
(5,493
)
(26,476
)
(18,746
)
Income taxes
25
-
(35
)
-
Net loss
(10,232
)
(5,493
)
(26,511
)
(18,746
)
Cumulative Series C convertible preferred
stock
dividends
-
(40
)
-
(1,054
)
Net loss attributable to common
stockholders
$
(10,232
)
$
(5,533
)
$
(26,511
)
$
(19,800
)
Net loss per share attributable to common
stockholders - basic and diluted
$
(0.30
)
$
(0.19
)
$
(0.82
)
$
(2.29
)
Weighted-average shares of common stock
outstanding - basic and diluted
33,807,943
28,409,518
32,253,227
8,649,245
Frequency Therapeutics, Inc.
Consolidated Balance Sheet Data (in thousands)
December 31, 2020
December 31, 2019
Cash, cash equivalents and short-term
investments
$
220,341
$
217,355
Working capital
198,430
168,575
Total assets
264,722
223,218
Total liabilities
72,231
55,860
Accumulated deficit
(95,399
)
(68,888
)
Total stockholders’ equity
192,491
167,358
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210329005687/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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