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RNS Number : 2044U
Syncona Limited
01 April 2021
Syncona Limited
Freeline Reports Financial Results and Business Highlights
01 April 2021
Syncona Ltd, a leading healthcare company focused on founding,
building and funding a portfolio of global leaders in life science,
notes the announcement that its portfolio company, Freeline
Therapeutics Holdings plc (Nasdaq: FRLN), a clinical-stage, fully
integrated, next generation, systemic, liver directed, AAV-based
gene therapy company announced its financial results for the full
year 2020 and provided an update on recent business highlights.
The announcement can be accessed on Freeline's website at:
https://www.freeline.life/investors-media/newsroom/ and the full
text of the announcement from Freeline is contained below.
[S]
Enquiries
Syncona Ltd
Annabel Clay
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 dynamic 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 strategic 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 few treatment options, build
sustainable life science companies and deliver strong risk-adjusted
returns to shareholders.
Freeline Reports Full Year 2020 Financial Results and Business
Highlights
Plans to have three AAV gene therapy programs in the clinic by
year-end 2021
Data from FLT180a Phase 1/2 trial for Hemophilia B demonstrated
potential for durable normalization of Factor IX activity
Strong 2020 year-end cash position of $230 million funds key
upcoming clinical milestones
LONDON, March 31, 2021 - Freeline Therapeutics Holdings plc
(Nasdaq: FRLN) (the "Company" or "Freeline"), a clinical-stage
biotechnology company developing transformative gene therapies for
patients suffering from inherited systemic debilitating diseases ,
today reported financial results for the full year 2020 and
provided an update on recent business highlights.
"We are pleased with our current progress and aim to have three
Freeline programs in the clinic by the end of 2021," said Theresa
Heggie, Chief Executive Officer of Freeline. "We are looking
forward to initiation of the Phase 1/2 dose-confirmation study for
our lead program, FLT180a for the treatment of Hemophilia B, later
this year to confirm both the dose and the immune management
regimen for our planned pivotal Phase 3 study. Importantly, the
data from the Phase 1/2 dose-finding trial we completed last year
demonstrated the potential of FLT180a to provide a functional cure
through the durable normalization of Factor IX activity. We also
plan to progress dose escalation for the Phase 1/2 clinical study
of FLT190 for the treatment of Fabry disease during 2021 .
Additionally, we are on track to dose the first patient in our
FLT201 program for the treatment of Type 1 Gaucher disease by the
end of 2021.
"Each of our pipeline programs leverages the Freeline research
and manufacturing platform, allowing development of novel AAV gene
therapy candidates using optimized cassette design, rapid candidate
screening and cutting-edge analytics," continued Ms. Heggie. "We
believe that our in-house protein engineering capabilities as well
as our proprietary AAVS3 capsid are key differentiators for
Freeline, allowing us to deliver high protein expression at low
doses for enhanced efficacy and the potential to address diseases
that are beyond the reach of first-generation AAV vectors."
Key Pipeline and Operational Updates
Hemophilia B
-- Reported data for FLT180a for the treatment of Hemophilia B
from the Phase 1/2 dose-finding clinical trial with a minimum of
six-month post-treatment data for all 10 patients, supporting the
potential for FLT180a to provide a functional cure with full
normalization of Factor IX ("FIX") activity in the normal range of
50 to 150 percent. Long-term follow-up data has shown durable FIX
activity, with up to three-year data in the earliest cohort .
-- Announced updated clinical development plan for FLT180a to
address Chemistry, Manufacturing and Control ("CMC") feedback from
the U.S. Food & Drug Administration ("FDA") relating to the
characterization and comparability of the investigational drug
product used in the Phase 1/2 dose-finding clinical trial that was
produced at smaller scale, as compared to the current
investigational drug product that is produced at
commercial-scale.
-- Plan to initiate Phase 1/2 dose-confirmation trial for
FLT180a by the end of 2021 to confirm both the dose and the immune
management regimen for the planned pivotal Phase 3 clinical trial.
The Company is targeting full enrollment of the Phase 1/2
dose-confirmation trial during the first half of 2022 and a
six-month data readout by the end of 2022.
-- Plan to report up to four-year durability data for FLT180a
from the Phase 1/2 dose-finding clinical trial by the end of
2021.
Fabry Disease
-- Reported preliminary data from Phase 1/2 dose-finding
clinical trial evaluating the safety and efficacy of FLT190 for the
treatment of Fabry disease, demonstrating that a single
administration of a low dose of FLT190 was generally well-tolerated
and led to a three to four-fold increase over baseline in plasma
<ALPHA>-galactosidase A ("<ALPHA>GLA") activity. This
increase was achieved by week four, and the patient continues to
show stable elevated GLA expression at 12 months
post-treatment.
-- Plan to progress dose escalation for the FLT190 Phase 1/2 clinical trial during 2021.
Gaucher Disease
-- Filed a CTA for FLT201 for the treatment of Type 1 Gaucher
disease with the UK Medicine and Healthcare products Regulatory
Agency ("MHRA").
-- Presented preclinical data at the 17th annual WORLD Symposium(TM) detailing the invention and characterization of <BETA>-glucocerebrosidase variant 85 (" GCase(var85) "), which was developed in-house and is produced by the Company's proprietary FLT201 gene therapy construct.
-- Presented preclinical data at the 17th annual WORLD
Symposium(TM) demonstrating the potential of FLT201 to deliver
sustained levels of GCase(var85) to tissues not sufficiently
addressed by velaglucerase alfa, a standard-of-care enzyme
replacement therapy ("ERT") for Type 1 Gaucher disease. FLT201 data
demonstrate increased plasma stability, a 20-fold longer half-life,
enhanced tissue uptake and substrate reduction, illustrating the
potential for FLT201 to offer significant improvements in clinical
outcomes over existing standard of care.
-- Currently plan to commence clinical development of FLT201 by
targeting initiation of first-in-human dose-finding trials by the
end of 2021.
H emophilia A
-- Presented an analysis of the CHESS II Study at the 14th
Annual Congress of the European Association for Haemophilia and
Allied Disorders (EAHAD), highlighting the significant burden of
joint pain and bleeding in factor-treated patients with Hemophilia
A and B. Data suggest that despite treatment, patients face
significant unmet clinical needs including continued bleeds and
poor underlying joint health.
-- Plan to complete preclinical proof-of-concept studies of the
safety, efficacy and durability of FLT210 to support an IND filing
in 2022.
Platform Technology
-- Presented data at the 12th Annual Protein and Antibody
Engineering Summit (PEGS) demonstrating how the Company's
proprietary, leading-edge analytical technologies characterize its
AAV product by determining the proportion of capsids containing
full-length vector genomes versus shorter vector fragments.
-- Plan to further develop programs that leverage the Company's
proprietary protein engineering and manufacturing capabilities.
Corporate
Freeline continues to strengthen its leadership team, building
on key appointments last year, which include Theresa Heggie as
Chief Executive Officer and member of the Board, Julie Krop, M.D.
as Chief Medical Officer and Mark Baldry as Chief Commercial
Officer, with the recent appointment of Michael J. Parini as
President and Chief Operating Officer. The Company also recently
strengthened the Board with the appointment of Colin A. Love, who
brings extensive experience in the manufacturing of complex
biotechnology products.
The Company also announced today that Jan Thirkettle, Ph.D.,
Chief Development Officer, has decided to leave Freeline to pursue
a new opportunity. Dr. Thirkettle will continue in his current role
until June 30, 2021, to ensure a smooth transition of his
responsibilities. The Company has begun an external search for Dr.
Thirkettle's replacement.
In 2020, Freeline closed an extended Series C financing with an
additional $79 million of new capital, augmenting the initial 2019
Series C funding of $40 million. The Company also completed a
successful Nasdaq IPO with net proceeds of $161.8 million.
2020 Financial Highlights
Freeline plans to begin reporting financial results on a
quarterly basis in 2021.
Research and development (R&D) expenses for the year ended
December 31, 2020 were $76.1 million, as compared to $47.0 million
for the same period in 2019. The increase of $29.1 million was
driven by an increased investment in activities related to the
current and proposed clinical trials for FLT180a and FLT190, and
overall research and development, which includes earlier pipeline
programs, discovery and further development of the Freeline
platform.
General and administrative (G&A) expenses for the year ended
December 31, 2020 were $26.3 million, as compared to $16.6 million
for the same period in 2019. The increase of $9.7 million is driven
primarily by legal and professional fees related to financing
activities, as well as an increase in headcount and related
personnel costs.
Cash and cash equivalents were $230.0 million as of December 31,
2020, as compared to $73.7 million as of year-end 2019. Based on
the Company's current operating plan, Freeline expects that its
current level of cash and cash equivalents will enable the Company
to fund its operating expenses into the third quarter of 2022.
As of December 31, 2020, the Company had 35,854,945 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 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 and Hemophilia A.
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. 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, timing, progress and results of the
Company's preclinical studies and clinical trials, including the
initiation of the Phase 1/2 dose confirmation trial for FLT180a and
data readouts from that trial, progress with respect to the
dose-escalation for the Phase 1/2 clinical study of FLT190,
commencement of clinical trials in our FLT201 program, and
completion of proof-of-concept studies of FLT201; statements that
the Company's updated clinical development plan for FLT180a will
allow it to address CMC feedback from the FDA in parallel with the
Phase 1/2 dose confirmation trial; business plans and prospects;
capital allocation objectives; 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; 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. We 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 our Annual
Report on Form 20-F for the fiscal year ended December 31, 2020 and
in our 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. You may get 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 Year Ended December 31,
-------------------------------------
2020 2019 2018
----------- ---------- ----------
OPERATING EXPENSES:
Research and development(1) $ 76,149 $ 47,043 $ 34,191
General and administrative(1) 26,300 16,601 6,790
Total operating expenses 102,449 63,644 40,981
LOSS FROM OPERATIONS: (102,449) (63,644) (40,981)
OTHER INCOME, NET:
Other expense, net (9,288) (793) (390)
Interest income, net 275 74 (151)
Benefit from R&D tax credit 15,269 10,595 8,266
---------- --------- ---------
Total other income, net 6,256 9,876 7,725
---------- --------- ---------
Loss before income taxes (96,193) (53,768) (33,256)
Income tax expense (129) (141) (27)
---------- --------- ---------
NET LOSS $ (96,322) $ (53,909) $ (33,283)
Net loss per share attributable to ordinary
shareholders-basic
and diluted $ (6.81) $ (8.49) $ (7.20)
Weighted average ordinary shares outstanding - basic
and diluted 14,152,843 6,347,818 4,621,495
________________
(1) Includes share-based compensation expenses as follows:
For the Year Ended December 31,
-----------------------------------------
2020 2019 2018
-------------- -------------- ---------
Research and development $ 2,510 $ 373 $ 45
General and administrative 3,694 1,086 69
Total share-based compensation 6,204 1,459 114
Consolidated Balance Sheet Data
(in thousands of U.S. dollars)
December December December
31, 31, 31,
------------- ------------- --------------
2020 2019 2018
------------- ------------- --------------
Cash and cash equivalents $229,974 $73,702 $16,051
Working capital(1) 239,364 76,612 16,926
Net Assets 249,800 84,956 22,971
Total assets 268,612 97,390 32,134
Additional paid-in capital 456,293 207,622 91,574
Accumulated deficit (215,990) (119,668) (65,759)
Total shareholders' equity 249,800 84,956 22,971
________________
(1) Working capital is defined as total current assets
minus total current liabilities.
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