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Syncona Limited
14 December 2021
Syncona Limited
Freeline reports 2022 corporate priorities and guidance
14 December 2021
Syncona Ltd, a leading healthcare company focused on founding,
building and funding global leaders in life science, notes that its
portfolio company, Freeline Therapeutics Holdings plc (Nasdaq:
FRLN) ("Freeline"), has announced its 2022 corporate priorities as
well as providing updated guidance across its programmes. The key
updates are as follows:
-- The company anticipates data across all programmes by mid-2022
o The Phase I/II B-LIEVE dose-confirmation trial of FLT180a in
haemophilia B launched in Q4 2021, one quarter ahead of its latest
guidance
o The Phase I/II dose-finding trial of FLT201 in Gaucher disease
Type 1 is on track for trial site initiation by year-end 2021 and
first patient dosed in Q1 2022
o The Phase I/II MARVEL-1 dose-finding trial of FLT190 in Fabry
disease is progressing in the clinic, with the next patient
expected to be dosed in H1 2022
-- Freeline has decided to discontinue further development of
its pre-clinical work for FLT210 in hemophilia A, as it continues
to prioritise its investments on the highest value activities
-- To streamline its operations, Freeline has proposed
implementing an approximate 25% reduction in the size of its
workforce
-- As a result of these changes, the company expects that its
cash runway will be extended by an additional quarter to Q2
2023
The announcement can be accessed via the Investors section of
the Freeline website. A copy of the announcement is also set out
below.
[S]
Copies of this press release and other corporate information can
be found on the company website at: www.synconaltd.com
Forward-looking statements - this announcement contains certain
forward-looking statements with respect to the portfolio of
investments of Syncona Limited. These statements and forecasts
involve risk and uncertainty because they relate to events and
depend upon circumstances that may or may not occur in the future.
There are a number of factors that could cause actual results or
developments to differ materially from those expressed or implied
by these forward-looking statements. In particular, many companies
in the Syncona Limited portfolio are conducting scientific research
and clinical trials where the outcome is inherently uncertain and
there is significant risk of negative results or adverse events
arising. In addition, many companies in the Syncona Limited
portfolio have yet to commercialise a product and their ability to
do so may be affected by operational, commercial and other
risks.
Enquiries
Syncona Ltd
Annabel Clay / Fergus Witt
Tel: +44 (0) 20 3981 7940
FTI Consulting
Ben Atwell / Natalie Garland-Collins / Tim Stamper
Tel: +44 (0) 20 3727 1000
About Syncona
Syncona's purpose is to invest to extend and enhance human life.
We do this by founding and building companies to deliver
transformational treatments to patients in areas of high unmet
need.
Our strategy is to found, build and fund companies around
exceptional science to create a diversified portfolio of 15-20
globally leading healthcare businesses for the benefit of all our
stakeholders. We focus on developing treatments for patients by
working in close partnership with world-class academic founders and
management teams. Our balance sheet underpins our strategy enabling
us to take a long-term view as we look to improve the lives of
patients with no or poor treatment options, build sustainable life
science companies and deliver strong risk-adjusted returns to
shareholders.
Freeline Announces 2022 Corporate Priorities and Guidance
Promising execution in the clinic with programs in hemophilia B,
Fabry disease and Gaucher disease; potential to deliver
transformative therapies driven by Freeline's differentiated
platform
Data anticipated across all programs by mid-2022
FLT180a B-LIEVE trial for hemophilia B launched in Q4 2021, one
quarter ahead of latest guidance
FLT201 Phase 1/2 trial for Gaucher disease Type 1 on track for
trial site initiation by year-end 2021; dosing in Q1 2022; FLT190
MARVEL-1 trial for Fabry disease progressing in the clinic;
next patient expected to be dosed in H1 2022
Company continues to prioritize investment s on the highest
value activities; hones corporate focus with deprioritization of
hemophilia A program and proposed 25% workforce reduction
LONDON, December 13, 2021 - Freeline Therapeutics Holdings plc
(Nasdaq: FRLN) (the "Company" or "Freeline"), a clinical-stage
biotechnology company developing transformative AAV-mediated gene
therapies for patients suffering from inherited systemic
debilitating diseases, today announced its 2022 corporate
priorities and provided updated guidance.
"Today we are announcing a tightening of our focus to deliver on
the promise of Freeline to advance therapies that have the highest
potential to transform the lives of patients suffering from
debilitating diseases. Leveraging our highly potent capsid and
unique platform capabilities, we believe we can deliver
best-in-class or first-in-class treatments, positioning Freeline
for long-term success as a leader in gene therapy," said Michael
Parini, Chief Executive Officer of Freeline. "2021 was a
transformational year at Freeline as we made progress across our
three strategic priorities - execution, prioritization and
innovation - to lay the foundation for success in 2022 and
beyond."
"Starting with execution, we are on track to initiate our Phase
1/2 dose-finding study of FLT201 in Gaucher disease by year-end, in
line with prior guidance, and look forward to dosing the first
patient in the first quarter of 2022. Our compelling preclinical
data suggest that the potency of our AAVS3 capsid combined with our
proprietary protein engineering positions FLT201 as a potentially
transformative therapy for Gaucher disease Type 1, a serious,
life-long disease with significant unmet medical need. FLT201 will
be our third program in the clinic and is poised to be the first
AAV gene therapy program for Gaucher disease Type 1 to enter the
clinic."
"We continue to make strong progress in hemophilia B, initiating
our Phase 1/2 dose-confirmation B-LIEVE study one quarter ahead of
latest guidance. We are rapidly advancing FLT180a for hemophilia B,
having already identified a sufficient number of patients to fully
enroll the trial via the ECLIPSE run-in study. We look forward to
dosing patients in the first quarter of 2022. The most recent data
we released at the American Society of Hematology (ASH) Annual
Meeting strengthens our belief that FLT180a has the potential to be
a best-in-class gene therapy treatment for hemophilia B."
"We also will advance FLT190 for Fabry disease throughout 2022,
with additional patients to be dosed and data expected through the
year. Based on the encouraging data from the first two patients
dosed in our ongoing MARVEL-1 trial, we continue to believe that
FLT190 has the potential to deliver high levels of
<ALPHA>-Gal A expression to keep Fabry patients off enzyme
replacement therapy at relatively low doses compared to other
treatments. We expect to dose the third patient in our ongoing
clinical trial in the first half of 2022."
Mr. Parini continued, "Successful execution of these programs
requires focused and strategic allocation of resources. To drive
continued advancement of FLT180a, FLT190 and FLT201, all of which
have the potential to be first or best-in-class, Freeline has
undertaken a detailed strategic review of our programs and
operations. Following this review, the Company has decided to halt
further development of our preclinical work for FLT210 in
hemophilia A, given the additional work needed. Freeline will seek
to explore potential third-party partnership opportunities to
progress this important work, which will allow us to focus our time
and resources on the programs most likely to return transformative
benefits for patients.
" Today we also announced that, in an effort to streamline our
operations, the Company is implementing an approximate 25%
reduction in the size of our workforce. This change follows our
program optimization and will enable us to better focus our time
and resources on the highest value activities. We are confident
these steps will create a more efficient and sustainable company ,
positioning Freeline for long-term success across its clinical
programs and platform technology. I would like to express my
sincere gratitude to our departing colleagues who are impacted by
this decision. Their dedication and contributions have been
invaluable to advancing our mission, and we will honor their work
through continued focus on delivering our three core programs to
the patients waiting for functional cures for their diseases."
"We are excited about our programs and what's to come in 2022.
These decisions to focus our efforts will result in a reduction in
operating expenses that extends our cash runway by an additional
quarter to the second quarter of 2023 and provides a solid
financial foundation to support the future success of our programs
and Company. We are ready, with increased focus and commitment, to
deliver the next generation of innovations for patients ," said Mr.
Parini.
Recent Updates, Key 2022 Priorities and Program Guidance
Gaucher Disease Type 1
-- Phase 1/2 dose-finding trial of FLT201 on track to be initiated in Q4 2021.
-- The Company expects to dose two patients in the first dose
cohort in the first half of 2022, with initial data expected in Q3
2022. Data from additional patients expected prior to year-end
2022.
Hemophilia B
-- Reported long-term follow-up data from its Phase 1/2 B-AMAZE
dose-finding trial of FLT180a for the treatment of hemophilia B at
the 63(rd) American Society of Hematology (ASH) Annual Meeting.
Results suggest that a dose of 7.7e11 vg/kg, coupled with a short
course of prophylactic immune management, has the potential to
achieve durable factor IX (FIX) activity in the normal range
(50-150%).
-- Initiated the B-LIEVE study, a Phase 1/2 dose-confirmation
study of FLT180a manufactured at commercial scale and using a
starting dose of 7.7e11 vg/kg and a short course of prophylactic
immune management, in Q4 2021.
-- The Company expects to complete dosing of the first dose
cohort in the B-LIEVE study and report data from the first dose
cohort in the first half of 2022.
Fabry Disease
-- Reported data from the lowest dose cohort in MARVEL-1 trial
of FLT190 for Fabry disease, demonstrating durable
<ALPHA>-Gal A expression over two years in the first patient
and promising activity with near normal <ALPHA>-Gal A levels
in the second patient, who remains off enzyme replacement therapy
more than 16 weeks post-dosing.
-- The Company expects to dose the third patient at the at
7.5e11 vg/kg dose level in H1 2022, with updated data from the
first two patients dosed, and initial data from the third, in the
first half of 2022. Escalation to next dose level is expected in H2
2022, along with updated data.
Corporate
The company intends to reduce expenses and extend its existing
cash runway through a prioritization of its programs and reduction
in workforce:
-- The Company expects to incur charges relating to its proposed
workforce reduction between $ 2.1 and $2.6 million in the fourth
quarter of 2021, which is expected to result in $1.3 to $1.6
million of cash expenditures in the first quarter of 2022 and $0.8
to $1.0 million of cash expenditures in the second quarter of
2022.
-- The Company estimates the proposed reduction in force will
decrease aggregate cash expenditures by between $4.7 and $5.1
million in 2022 and between $7.0 and $7.6 million in 2023.
-- As a result of these initiatives, the Company expects its
current level of cash and cash equivalents will enable the Company
to fund its operating expenses into the second quarter of 2023.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company developing
transformative adeno-associated virus (AAV) vector-mediated
systemic gene therapies. The Company is dedicated to improving
patient lives through innovative, one-time treatments that provide
functional cures for inherited systemic debilitating diseases.
Freeline uses its proprietary, rationally designed AAV vector,
along with novel promoters and transgenes, to deliver a functional
copy of a therapeutic gene into human liver cells, thereby
expressing a persistent functional level of the missing or
dysfunctional protein into the patient's bloodstream. The Company's
integrated gene therapy platform includes in-house capabilities in
research, clinical development, manufacturing and
commercialization. The Company has clinical programs in hemophilia
B and Fabry disease, as well as preclinical programs in Gaucher
disease Type 1. Freeline is headquartered in the UK and has
operations in Germany and the US.
About Hemophilia
Hemophilia is a genetic bleeding disorder caused by a deficiency
in clotting factor protein that impairs blood clot formation. In
hemophilia B, there is a deficiency of the clotting factor IX
(nine) protein. Hemophilia B is an X-linked disease that mainly
affects boys and men; however, women who carry an affected copy of
the clotting factor gene may also experience symptoms. Hemophilia B
affects about one in every 30,000 males. Hemophilia is classified
as mild, moderate or severe, depending on the level of clotting
factor VIII or IX in the blood and is diagnosed through blood
tests.
About FLT180a for Hemophilia B
The Freeline hemophilia B program, FLT180a, uses a potent,
rationally designed capsid (AAVS3) containing an expression
cassette encoding a gain of function Padua variant of human factor
IX (FIX). FLT180a was studied in B-AMAZE, a Phase 1/2 dose-finding
trial in patients with severe and moderately severe hemophilia B
with the goal of normalizing FIX activity. Patients treated in
B-AMAZE are being followed in a long-term follow-up study. A Phase
1/2 dose-confirmation trial of FLT180a called B-LIEVE has been
initiated.
About Fabry Disease
Fabry disease is a genetic lysosomal disease that leads to a
deficiency in <ALPHA>-galactosidase A (<ALPHA>-Gal A),
which is a key enzyme needed to break down a fatty substance called
globotriaosylceramide (Gb3) and lyso-Gb3. Without the enzyme, this
fatty substance builds up throughout the body, affecting tissues
and organs including skin, kidneys, heart and the nervous system.
Fabry disease occurs in all ethnic groups and is estimated to
affect one in every 40,000 people. Freeline is currently focused on
classic Fabry disease where patients have little to no functional
<ALPHA>-Gal A enzyme. The current standard of care is
lifelong intravenous infusions of enzyme replacement therapy (ERT)
or pharmacological chaperone therapy (PCT). Certain treatments can
carry a significant burden on the patient. The aim of Freeline's
investigational gene therapy program is to deliver a one-time
treatment of a long-lasting gene therapy that will provide a
sustained, therapeutically relevant level of <ALPHA>-Gal A
that the Company believes would eliminate the need for ERT or
PCT.
About FLT190 for Fabry Disease
FLT190 is an investigational AAV gene therapy in development as
a potential treatment for patients with Fabry disease. FLT190
consists of a potent, rationally designed capsid (AAVS3) containing
an expression cassette with a codon-optimized human
<ALPHA>-Gal A cDNA under the control of a liver-specific
promoter. The Company's current MARVEL-1 Phase 1/2 dose-finding
trial is evaluating the potential safety and efficacy of FLT190 in
Fabry patients, who often have pre-existing cardiac manifestations
due to underlying substrate accumulation and disease progression in
the heart. The treatment is administered by intravenous infusion
that lasts approximately one hour and does not require the patient
to undergo stem cell harvest or conditioning with chemotherapy.
About Gaucher Disease
Gaucher disease is a genetic disorder in which a fatty substance
called glucosylceramide accumulates in macrophages in certain
organs due to the lack of functional glucocerebrosidase (GCase).
The disorder is hereditary and presents in various subtypes.
Freeline is currently focused on Gaucher disease Type 1, the most
common type, which impacts the health of many organs of the body
including the spleen, liver, blood system and bones. The current
standard of care is intravenous infusion of ERT every two weeks,
which is a significant treatment burden on the patient.
About FLT201 for Gaucher Disease
FLT201 is an investigational gene therapy for the treatment of
Gaucher disease Type 1. It consists of a potent, rationally
designed AAV capsid (AAVS3) containing an expression cassette that
encodes for a novel glucocerebrosidase variant (GCasevar85) under
the control of a liver-specific promoter. The GCasevar85 contains
two novel amino acid substitutions to the wild-type human GCase,
which results in a 20-fold increase in GCase half-life at lysosomal
pH conditions, but similar catalytic parameters to those of
wild-type GCase and ERT. The Company's high-transducing AAVS3
capsid advances its goal to address unmet needs for those affected
by Gaucher disease by potentially enabling sustained, endogenous
production of GCase following a one-time intravenous infusion. The
aim of Freeline's investigational gene therapy program is to
deliver a one-time treatment of a long-lasting gene therapy that
will provide a sustained, therapeutically relevant level of
endogenous GCase, thus eliminating the need for ERT.
Forward-Looking Statements
This press release contains statements that constitute "forward
looking statements" as that term is defined in the United States
Private Securities Litigation Reform Act of 1995, including
statements that express the Company's opinions, expectations,
beliefs, plans, objectives, assumptions or projections regarding
future events or future results, in contrast with statements that
reflect historical facts. Examples include, among other topics,
discussion of the Company's strategies, anticipated operating and
financial performance and financial condition; the Company's
expectations regarding its use of cash and cash runway; statements
regarding the initiation, dosing, timing, progress and interim and
final results of the Company's clinical trials, including the Phase
1/2 B-LIEVE dose-confirmation clinical trial of FLT180a, the Phase
1/2 dose-finding clinical trial of FLT201 and the Phase 1/2
MARVEL-1 dose-finding clinical trial of FLT190, whether the Company
has identified a sufficient number of patients to fully enroll the
B-LIEVE trial; statements regarding the expected timing of
regulatory filings; and manufacturing, research, pipeline, and
clinical trial plans, including anticipated clinical development
milestones for the Company's product candidates. In some cases, you
can identify such forward-looking statements by terminology such as
"anticipate," "intend," "believe," "estimate," "plan," "seek,"
"project" or "expect," "may," "will," "would," "could" or "should,"
the negative of these terms or similar expressions. Forward-looking
statements are based on management's current beliefs and
assumptions and on information currently available to the Company,
and you should not place undue reliance on such statements.
Forward-looking statements are subject to many risks and
uncertainties, including the Company's recurring losses from
operations; the uncertainties inherent in research and development
of the Company's product candidates, including statements regarding
the timing of initiation, completion and the outcome of clinical
studies or trials and related preparatory work
and regulatory review, regulatory submission dates, regulatory
approval dates and/or launch dates, as well as risks associated
with preclinical and clinical data, including the possibility of
unfavorable new preclinical, clinical or safety data and further
analyses of existing preclinical, clinical or safety data; the
Company's ability to design and implement successful clinical
trials for its product candidates; the recent departures of a
number of executive officers of the Company, and the Company's
ability to fill open positions, implement an orderly transition
process and retain key talent; whether the Company's cash resources
will be sufficient to fund the Company's foreseeable and
unforeseeable operating expenses and capital expenditure
requirements for the Company's expected timeline; the potential for
a pandemic, epidemic or outbreak of infectious diseases in the US,
UK or EU, including the COVID-19 pandemic, to disrupt and delay the
Company's clinical trial pipeline; the Company's failure to
demonstrate the safety and efficacy of its product candidates; the
fact that results obtained in earlier stage clinical testing may
not be indicative of results in future clinical trials; the
Company's ability to enroll patients in clinical trials for its
product candidates; the possibility that one or more of the
Company's product candidates may cause serious adverse, undesirable
or unacceptable side effects or have other properties that could
delay or prevent their regulatory approval or limit their
commercial potential; the Company's ability to obtain and maintain
regulatory approval of its product candidates; the Company's
limited manufacturing experience, which could result in delays in
the development, regulatory approval or commercialization of its
product candidates; and the Company's ability to identify or
discover additional product candidates, or failure to capitalize on
programs or product candidates. Such risks and uncertainties may
cause the statements to be inaccurate and readers are cautioned not
to place undue reliance on such statements. The Company cannot
guarantee that any forward-looking statement will be realized.
Should known or unknown risks or uncertainties materialize or
should underlying assumptions prove inaccurate, actual results
could vary materially from past results and those anticipated,
estimated or projected. Investors are cautioned not to put undue
reliance on forward-looking statements. A further list and
description of risks, uncertainties and other matters can be found
in the Company's Annual Report on Form 20-F for the fiscal year
ended December 31, 2020 and in subsequent reports on Form 6-K, in
each case including in the sections thereof captioned "Cautionary
Statement Regarding Forward-Looking Statements" and "Item 3.D. Risk
factors." Many of these risks are outside of the Company's control
and could cause its actual results to differ materially from those
it thought would occur. The forward-looking statements included in
this press release are made only as of the date hereof. The Company
does not undertake, and specifically declines, any obligation to
update any such statements or to publicly announce the results of
any revisions to any such statements to reflect future events or
developments, except as required by law. For further information,
please reference the Company's reports and documents filed with the
U.S. Securities and Exchange Commission (the "SEC"). You may review
these documents by visiting EDGAR on the SEC website at www.sec.gov
.
Contact
David S. Arrington
Vice President Investor Relations & Corporate
Communications
Freeline Therapeutics
david.arrington@freeline.life
+1 (646) 668 6947
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END
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