TIDMSYNC
RNS Number : 8412G
Syncona Limited
01 April 2022
Syncona Limited
Freeline reports Full Year 2021 Financial Results and Business
Highlights
01 April 2022
Syncona Ltd, a leading healthcare company focused on founding,
building and funding a portfolio of global leaders in life science,
notes that its portfolio company, Freeline Therapeutics Holdings
Plc (NASDAQ: FRLN) ("Freeline"), announced its financial results
for the full year 2021 and an update on recent business
highlights.
The announcement can be accessed on Freeline's website at:
www.freeline.life/investors/ and the full text of the announcement
from Freeline is contained below.
[S]
Enquiries
Syncona Ltd
Natalie Garland-Collins / Fergus Witt
Tel: +44 (0) 7714 916615
FTI Consulting
Ben Atwell / 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 a portfolio of global leaders
in life science 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 Reports Full Year 2021 Financial Results and Business
Highlights
Phase 1/2 programs on track in Hemophilia B and Gaucher disease
and accelerated in Fabry disease
Extended financial runway and streamlined organization support
Company
through key data readouts across all three clinical programs
Strengthened leadership team with appointment of Chief Medical
Officer
and recent addition of Chief Scientific Officer
LONDON, March 31, 2022 - Freeline Therapeutics Holdings plc
(Nasdaq: FRLN) (the "Company" or "Freeline") today reported
financial results for the full year 2021 and provided a business
update.
"2022 is shaping up to be a watershed year for Freeline,
building on the strong foundation we put in place in the second
half of last year," said Michael Parini, Chief Executive Officer of
Freeline. "Under new leadership, our streamlined organization has
executed with urgency and increased financial discipline on a
refocused set of clinical programs and corporate priorities. We
also have recently strengthened our balance sheet to enable us to
deliver meaningful clinical data readouts through 2022 and beyond
to demonstrate the value of our promising gene therapy candidates
as we advance on the path towards pivotal Phase 3 studies.
Additionally, we are working on a new R&D strategy to explore
the application of our science and platform technologies to new
disease areas, including extending these strengths to efforts
beyond rare monogenic disorders."
Key Clinical Program Updates
FLT180a in Hemophilia B - On track to fully enroll Phase 1/2
B-LIEVE dose confirmation study by the end of the third quarter of
2022, enabling initiation of pivotal Phase 3 study start-up
activities in H1 2023.
-- Potential for FLT180a to provide functional cure through
durable normalization of Factor IX activity (50-150%) demonstrated
in the long-term follow-up study of the Phase 1/2 dose-finding
trial, B-AMAZE. Data demonstrating sustained FIX expression up to
3.5 years and decline in annualized exogenous Factor IX consumption
and bleeding after gene therapy reported at the 63(rd) American
Society of Hematology (ASH) Annual Meeting in December 2021.
-- B-LIEVE study currently underway to confirm 7.7e11 vg/kg dose
and prophylactic immune management to be used in pivotal Phase 3
study. The first patient in the B-LIEVE study was dosed in March
2022, with completion of the first dose cohort and reporting of
initial data both expected in the first half of 2022.
FLT190 in Fabry Disease - Updated clinical development plan and
timelines for Phase 1/2 MARVEL-1 study accelerates dose escalation
to mid-year 2022.
-- Following a comprehensive review of the pre-clinical data and
the efficacy and safety data generated to date in the MARVEL-1
study with the study's independent Data Monitoring Committee, the
Company plans to proceed immediately to the second dose cohort
(1.5e12 vg/kg).
-- The Company expects to dose the first patient in the second
dose cohort in mid- 2022 and provide a program update in the second
half of 2022.
-- Reported data from the lowest dose cohort in MARVEL-1 trial,
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 was dosed in
June 2021 and remain ed off enzyme replacement therapy for more
than 26 weeks post-dosing , as of the last data cut-off date of
December 22, 2021 .
FLT201 in Gaucher Disease - On track to dose patients in
first-in-human, Phase 1/2 GALILEO-1 study in H1 2022.
-- Phase 1/2 GALILEO-1 study initiated at the end of 2021, and
study design presented at the 18(th) annual WORLD Symposium(TM),
February 7-11, 2022.
-- The Company expects to dose two patients in the first dose
cohort in the first half of 2022 and two patients in the second
cohort in the second half of 2022, with initial data from such pa t
ients expected in the second half of the year.
-- GALILEO-1 is a first-in-human open-label, international,
multicenter clinical study evaluating the safety, tolerability, and
efficacy of a single intravenous infusion of FLT201 with a novel
prophylactic immune management regimen in adults with Gaucher
disease Type 1.
-- Adaptive dose-escalation study will identify a dose of FLT201
for further development in a pivotal Phase 3 clinical trial.
-- FLT201 consists of a rationally designed AAV capsid (AAVS3)
containing an expression cassette that encodes for a novel
glucocerebrosidase variant (GCasevar85) under the control of a
liver-specific promotor.
Key Corporate Updates
Freeline begins 2022 with new company leadership.
Freeline continues to strengthen its leadership team, building
on key appointments in 2021, which included Michael J. Parini as
Chief Executive Officer and member of the board, Pamela Foulds,
M.D. as Chief Medical Officer, and James Bircher as Chief Technical
Operations Officer. The Company recently announced in February 2022
the appointment of Henning R. Stennicke, PhD, as Chief Scientific
Officer to lead research and discovery.
2021 Financial Highlights
1. Cash Position: Cash and cash equivalents were $117.6 million
as of December 31, 2021, as compared to $229.9 million as of
year-end 2020. On March 15, 2022, Freeline closed a $26.1 million
registered direct offering with certain long-term shareholders. The
proceeds will support the advancement of Freeline's clinical-stage
programs and the extension of the Company's scientific capabilities
and platform technologies to additional disease areas. As a result
of this offering, Freeline expects that its current level of cash
and cash equivalents will enable the Company to fund its operating
expenses into the second half of 2023.
2. Research and Development (R&D) Expenses: R&D expenses
for the year ended December 31, 2021 were $95.4 million, as
compared to $76.1 million for the same period in 2020. The increase
of $19.3 million was driven by an increased investment in
activities related to the current and proposed clinical trials for
FLT201, as well as an increase in personnel expenses primarily
related to R&D and manufacturing personnel hired to support
increased clinical activity in 2021.
3. General and Administrative ("G&A") Expenses: G&A
expenses for the year ended December 31, 2021 were $44.5 million,
as compared to $26.3 million for the same period in 2020. The
increase of $18.3 million was driven primarily by an increase in
personnel expenses, primarily related to personnel hired in
corporate, legal, general and administrative functions to support
our growth initiatives and our new public company requirements, as
well as an increase in legal and professional fees related to
expenses associated with the Company's status as a public company,
including annual and periodic reporting, implementation of equity
compensation programs, and more extensive governance.
4. As of December 31, 2021, the Company had 35,854,591 ordinary shares outstanding.
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, Fabry disease, and Gaucher disease Type 1. Freeline is
headquartered in the UK and has operations in Germany and the
US.
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. All statements, other than historical
facts, including statements regarding the potential for the Company
to develop a scientific strategy where its differentiated
capabilities allow it to address unmet needs and create
transformative treatments for patients, the timing of initiation,
enrollment, continuation, completion and the outcome of clinical
trials and related preparatory work, and the Company's expectations
regarding its use of cash and cash runway, are forward-looking
statements. In some cases, you can identify such forward-looking
statements by terminology such as "anticipate," "intend,"
"believe," "estimate," "plan," "seek," "project" "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, enrollment,
continuation, 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; business interruptions
resulting from geopolitical actions, including global hostilities,
war and terrorism, global pandemics or natural disasters, including
earthquakes, typhoons, floods and fires; 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
.
Consolidated Statement of Operations Data
(in thousands of U.S. dollars, except per share data)
For the Twelve Months Ended December
31,
------------------------------------------------------------------------------------------------------------------
2021 2020 2019
------------------------------- ---------------------------------- -----------------------------------
OPERATING EXPENSES:
$ $ $
Research and development 95,431 76,149 47,043
General and administrative 44,567 26,300 16,601
Restructuring expense 2,381 - -
---------------------------------- -----------------------------------
Total operating
expenses 142,379 102,449 63,644
------------------------------- ---------------------------------- -----------------------------------
LOSS FROM OPERATIONS: (142,379) (102,449) (63,644)
OTHER INCOME (EXPENSE)
NET:
Other income (expense),
net (165) (9,288) (793)
Interest income, net 404 275 74
Benefit from R&D tax credit 2,091 15,269 10,595
------------------------------- ---------------------------------- -----------------------------------
Total other income
(expense),
net 2,330 6,256 9,876
------------------------------- ---------------------------------- -----------------------------------
Loss before income taxes (140,049) (96,193) (53,768)
Income tax expense (342) (129) (141)
------------------------------- ---------------------------------- -----------------------------------
Net loss (140,391) (96,322) (53,909)
=============================== ================================== ===================================
Net loss per share attributable
to ordinary $ $ $
shareholders-basic and
diluted (3.93) (6.81) (8.49)
=============================== ================================== ===================================
Weighted average ordinary
shares outstanding-basic
and diluted 35,704,368 14,152,843 6,347,818
=============================== ================================== ===================================
Consolidated Balance Sheet Data
(in thousands of U.S. dollars)
December
December 31, 31,
----------------------------- -----------------------------
2021 2020
----------------------------- -----------------------------
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 117,662 $ 229,974
Account receivable - 97
Prepaid expenses and other current
assets 10,630 28,105
----------------------------- -----------------------------
Total current assets 128,292 258,176
Property and equipment, net 9,906 8,608
Intangible assets, net 8 23
Other non-current assets 2,919 1,805
Total assets $ 141,125 $ 268,612
============================= =============================
LIABILITIES, PREFERRED SHARES AND
SHAREHOLDERS' EQUITY
CURRENT LIABILITIES:
Accounts payable $ 5,187 $ 8,093
Accrued expenses and other current
liabilities 15,497 10,719
Total current liabilities 20,684 18,812
----------------------------- -----------------------------
Total liabilities 20,684 18,812
============================= =============================
Commitments and contingencies
SHAREHOLDERS' EQUITY:
Deferred shares 137 155
Additional paid-in capital 467,213 456,293
Accumulated other comprehensive loss 9,472 9,342
Accumulated deficit (356,381) (215,990)
----------------------------- -----------------------------
Total shareholders' equity 120,441 249,800
TOTAL LIABILITIES AND SHAREHOLDERS'
EQUITY $ 141,125 $ 268,612
============================= =============================
IR Contact:
investor@freeline.life
Media Contact:
Arne Naeveke, PhD
Vice President, Head of Corporate Communications
arne.naeveke@freeline.life
+1 617 312 2521
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
RNS may use your IP address to confirm compliance with the terms
and conditions, to analyse how you engage with the information
contained in this communication, and to share such analysis on an
anonymised basis with others as part of our commercial services.
For further information about how RNS and the London Stock Exchange
use the personal data you provide us, please see our Privacy
Policy.
END
PFUQFLFXLXLBBBL
(END) Dow Jones Newswires
April 01, 2022 02:01 ET (06:01 GMT)
Syncona (LSE:SYNC)
Gráfica de Acción Histórica
De Mar 2024 a Abr 2024
Syncona (LSE:SYNC)
Gráfica de Acción Histórica
De Abr 2023 a Abr 2024
