REG - Syncona Limited - Freeline reports new data and 3rd Quarter Results
10/11/2021 08:05
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RNS Number : 8653R Syncona Limited 10 November 2021
Syncona Limited
Freeline reports new data from FLT190 and Third Quarter Financial Results
10 November 2021
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 ("Freeline"), a
clinical-stage biotechnology company developing transformative AAV-mediated
gene therapies for patients suffering from inherited systemic debilitating
diseases, has announced new data from its ongoing Phase I/II MARVEL-1
dose-finding clinical trial of FLT190 for the treatment of Fabry disease and
provided updates on its pipeline programs. Freeline also announced that Pamela
Foulds, MD has been appointed as Chief Medical Officer (CMO) and reported
financial results for the third quarter of 2021.
Highlights:
· Second patient dosed in Phase I/II trial for Fabry disease has
generated encouraging data, the patient remains off enzyme replacement therapy
more than 16 weeks post-dosing; first patient shows durable expression of the
key enzyme (α-Gal A), which is absent or markedly deficient in patients, over
two years with promising efficacy
· Third patient in the trial for Fabry's disease will be dosed at
the same dose level as the second patient with additional safety monitoring
after a case of mild and transient myocarditis was observed
· Freeline is evaluating whether additional studies will be required to
enable the business to progress Haemophilia A into the clinic and the
necessity of conducting these additional studies
Outlook: three clinical studies by the end of CY2021 and multiple data
read-outs in CY2022
· Freeline remains on track to report long-term durability data from
Phase I/II dose-finding trial for Haemophilia B by end of CY2021
· The Haemophilia B Phase I/II dose-confirmation study will now
start in Q1 CY2022, instead of by end of CY2021 as the business engages with
the regulatory authorities to update study protocols
· In the Phase I/II trial for Fabry's disease, the company expects
to continue patient dosing in Q1 CY2022 with further interim data outs from
the study expected in CY2022
· On track for trial site initiation for the Phase I/II study for
Gaucher Type 1 by end of CY2021 with data read-outs from the trial expected in
CY2022
Chris Hollowood, Chief Investment Officer of Syncona Investment Management
Limited and Chair of Freeline said: "We are pleased that Freeline has been
able to re-initiate clinical studies and are encouraged by the data generated
by the second patient dosed in the Phase I/II Fabry's study. We are fully
supportive of the safety protocols recommended by the data monitoring
committee and look forward to seeing further progress from this study.
By the end of the calendar year, the business will have three live clinical
studies with the expected site initiation of its Phase I/II programme for
Gaucher's disease and multiple data read-outs expected across these programmes
in 2022 with the potential to drive value for the business. We remain excited
by the potential of the therapies the company is developing to have a
meaningful impact on the lives of patients."
The announcement can be accessed on Freeline's website at:
https://www.freeline.life/investors-media/newsroom/
(https://www.freeline.life/investors-media/newsroom/) and the full text of the
announcement from Freeline is contained below.
ENDS
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 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 New Data from Fabry Disease Program, Pipeline and Company
Updates, Including Appointment of Pamela Foulds, MD as Chief Medical Officer,
and Third Quarter 2021 Financial Results
Lowest dose cohort of MARVEL-1 trial of FLT190 for Fabry disease demonstrates
durable α-Gal A expression over two years in the first patient and promising
efficacy with near normal α-Gal A levels in the second patient, who remains
off enzyme replacement therapy more than 16 weeks post-dosing
Third patient in MARVEL-1 to be dosed at 7.5e11 vg/kg dose level in first
quarter 2022 with additional safety monitoring after case of mild and
transient myocarditis observed
FLT201 Phase 1/2 trial for Gaucher disease Type 1 on track for trial site
initiation by year-end; FLT180a Phase 1/2 dose confirmation trial for
hemophilia B to be in clinic by first quarter 2022
LONDON, November 9, 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 new data from
its ongoing Phase 1/2 MARVEL-1 dose-finding clinical trial of FLT190 for the
treatment of Fabry disease and provided updates on its pipeline programs. The
Company also announced that Pamela Foulds, MD has been appointed as Chief
Medical Officer (CMO) and reported financial results for the third quarter of
2021.
"Today we are pleased to announce that enzyme expression data from the second
patient in our Phase 1/2 dose-finding trial of FLT190 are highly encouraging,
with expression of alpha-galactosidase A reaching near-normal levels and the
patient thus far remaining off enzyme replacement therapy since dosing," said
Michael Parini, Chief Executive Officer of Freeline. "These results were
achieved in our lowest dose cohort and already FLT190 appears to be having a
significant impact on α-Gal A activity and disease process in Fabry, which is
the underlying goal and promise of the FLT190 program. We also learned that
our proactive immune management regimen, which is being deployed across all of
our programs, has been effective as no elevations of liver enzymes have been
observed throughout the treatment period."
"We are also delighted to further build our leadership team with the addition
of Dr. Pamela Foulds, whom we welcome to Freeline as our new Chief Medical
Officer," said Mr. Parini. "Given our continued focus on delivering our
clinical-stage assets and identifying new disease opportunities to tackle with
our potent capsid and strong protein engineering capabilities, Pam's expertise
will be invaluable as we evaluate our programs and determine how to best
advance them and unlock the value in our pipeline."
"We will be dosing a third patient in the first dose cohort of MARVEL-1, which
will allow us to gather additional information on a potential risk of mild and
transient myocarditis in this patient population with underlying and
contributory cardiac disease. This step is consistent with the recommendation
of our independent Data Monitoring Committee, with whom we have worked to
review the data."
"The mild and transient myocarditis was an unexpected observation in the
MARVEL-1 study given the very strong safety record Freeline has established in
hemophilia, where myocarditis was not observed in the 10 patients treated in
the B-AMAZE study," said Dr. Atul B. Mehta, formerly Clinical Director of the
Lysosomal Storage Disorders Unit at the Royal Free London NHS Foundation Trust
and Emeritus Professor of Haematology at University College London. "It's
likely that Fabry patients are predisposed to developing this by virtue of the
pre-existing cardiomyopathy they often have as a result of the disease.
Appropriate measures for even closer observation and monitoring have been
introduced to ensure the safety of future trial participants."
Mr. Parini continued, "As Freeline and others continue to explore the
potential of gene therapy, we are working with regulatory authorities to
update study protocols for FLT180a for hemophilia B and FLT201 for Gaucher
disease Type 1 to include additional data collection. We believe this
proactive step is the right approach for patients. As a result of these
updates, we expect to initiate the trial of FLT180a in the first quarter of
2022 instead of by the end of this year and continue to anticipate interim
data readouts in 2022 from both trials. We also are working with regulatory
authorities to augment data monitoring for FLT190 and expect to continue
patient dosing in the first quarter of 2022 following these updates."
Key Pipeline and Operational Updates
Fabry Disease
· The Company reported an interim program update on Patients One and
Two and progress in its Phase 1/2 MARVEL-1 dose-finding trial of FLT190. The
data cutoff date was October 6, 2021.
· Patient Two:
o Patient Two was dosed at the lowest dose cohort of 7.5e11 vg/kg in June
2021 and experienced a sustained and durable response with an increase in
expression of plasma α-galactosidase A (α-Gal A), the missing enzyme in
Fabry disease, to near normal levels, from 0.0 nmol/hr/mL at baseline to an
average of 3.9 nmol/hr/mL from weeks 6 to 16 post-dosing. Thus far, the
patient remains off enzyme replacement therapy (ERT).
o With respect to safety, the treatment was well-tolerated with no
dose-limiting toxicities or serious adverse events (SAEs). As of the cutoff
date, there were no adverse events higher than Grade 1 (mild). Patient Two has
a history of cardiac disease and on routine weekly monitoring, an incidental
finding of changes in cardiac markers, troponin-T and electrocardiogram (ECG),
was observed, although the patient was asymptomatic. After evaluation, these
findings were determined to be consistent with mild and transient myocarditis.
Patient 2's troponin-T has since reverted to baseline, and the ECG remained
stable as of the cutoff date. Ventricular functioning in the heart has
remained normal throughout.
o Patient Two has experienced no elevations in liver enzymes under our
current immune management regimen, which has evolved in response to patient
data in the hemophilia B and Fabry programs.
o The total vector genome (vg) dose Patient Two received was approximately
40% higher than Patient One due to differences in their weights. Patient Two
saw a 400% increase in enzyme levels compared with Patient One.
· Patient One:
o Long-term follow-up from Patient One shows durable response with plasma
α-Gal A activity levels remaining elevated at two years post-dose and
generally steady at an average of three times baseline. The patient had a
subtherapeutic response with plasma α-Gal A at 0.8-1.3 nmol/hr/mL and
restarted ERT at week six.
o Patient One has experienced no enduring clinical sequelae of the transient
mild myocarditis episode previously reported in 2019. Ventricular functioning
in the heart remained normal throughout with cardiac magnetic resonance
imaging (MRI) showing no evidence of scarring on follow-up at one- and
two-years post-dosing.
· An independent Data Monitoring Committee (DMC) conducted a
comprehensive review of safety and efficacy data from the two patients dosed
in the lowest dose cohort in the MARVEL-1 trial. The DMC has recommended that
Freeline proceed with dosing a third patient in the first cohort at the same
7.5e11 vg/kg dose level with additional cardiac monitoring, which will be
followed by an additional DMC meeting.
· The Company is engaging with regulatory authorities to update the
study protocols and trial design for FLT190 and expects to continue patient
dosing in the first quarter of 2022 following these updates. Freeline
continues to anticipate interim data readouts from the MARVEL-1 trial in 2022,
sharing further efficacy and safety data.
Hemophilia B
· On track to report long-term durability data from the Company's
Phase 1/2 dose-finding trial of FLT180a for the treatment of hemophilia B by
year-end.
· The Company is engaging with regulatory authorities to update the
study protocol for the Phase 1/2 B-LIEVE dose-confirmation trial of FLT180a.
The protocol changes and subsequent review shifts guidance by approximately
one quarter for the FLT180a program, with study start expected in the first
quarter of 2022 instead of by year-end 2021. We are currently evaluating the
timing of our Phase 3 pivotal trial and filing of a Biologics License
Application and will provide more concrete guidance next year. The Company
continues to anticipate interim data readouts in 2022.
· Pursuing Accelerated Approval with the U.S. Food and Drug
Administration (FDA) using the surrogate endpoint of factor IX (FIX) activity
levels combined with demonstration of a positive correlation between 26-week
FIX activity levels and 52-week annualized bleeding rate (ABR), assuming
robust data at 26 weeks.
· Enrollment in the ECLIPSE run-in study for the Phase 1/2 B-LIEVE
dose-confirmation trial of FLT180a is proceeding more quickly than expected.
As a result, we believe we have identified a sufficient number of patients to
fully enroll the B-LIEVE trial.
Gaucher Disease Type 1
· Announced Orphan Drug Designations for FLT201 for the treatment of
Gaucher disease Type 1 from the FDA and European Commission (EC).
· The Company is engaging with regulatory authorities to update the
study protocol for the Phase 1/2 dose-finding trial of FLT201 and remains on
track for trial site initiation by year end 2021. We continue to expect trial
start and interim data readouts on safety and efficacy in 2022.
Hemophilia A
· The Company expects to complete ongoing preclinical studies of FLT210
for the treatment of hemophilia A by year end as previously anticipated. Based
on data generated in these studies to date, the Company is evaluating whether
additional studies will enhance an IND filing and the necessity of conducting
these additional studies.
Platform Technology
· The Company presented two preclinical posters at the 2021 European
Society for Gene and Cell Therapy Virtual Congress detailing analysis methods
for liver transduction and expression in non-human primate liver after AAVS3
delivery and the Company's second generation two-plasmid packaging system for
AAV vectors to improve quality and yield.
Corporate
· Today the Company announced the expansion and strengthening of its
executive leadership team with the appointment of Pamela Foulds, MD as Chief
Medical Officer. Dr. Foulds is a US-trained physician, with over 20 years of
experience as a leader in the life sciences sector and extensive practice in
the rare disease field. She is an experienced CMO, having served in that
capacity at both Auregen BioTherapeutics SA and Aegerion Pharmaceuticals Inc.
Prior to that, she spent eight years at Biogen Inc., working in both
hemophilia and neurology. Earlier in her career, Dr. Foulds oversaw medical
affairs for the lysosomal storage disease programs at Genzyme. Dr. Foulds will
report to Chief Executive Officer Michael Parini and serve on the executive
leadership team. She will be based in the US leading the Company's global
clinical, medical and regulatory activities. Dr. Foulds holds a Bachelor of
Science degree from Georgetown University and a medical degree from Georgetown
University Medical School. She was trained in Neurology at Stanford University
Hospital.
Q3 2021 Financial Highlights
· Cash Position: Cash and cash equivalents were $136.4 million as of
September 30, 2021, as compared to $230.0 million as of December 31, 2020.
Based on the Company's revised operating plan, Freeline expects that its
current level of cash and cash equivalents will enable the Company to fund its
operating expenses into the first quarter of 2023. The Company continues to
review its operations to focus resources on efforts that are expected to
return the highest value to its shareholders.
· R&D Expenses: Research and development ("R&D") expenses for
the nine months ended September 30, 2021 were $70.8 million, as compared to
$55.3 million for the same period in 2020. The increase of $15.5 million was
driven by increased investment in activities related to the current and
proposed clinical trials for FLT201, facilities and overall R&D, which
includes earlier pipeline programs and further development of Freeline's
platform technology.
· G&A Expenses: General and administrative ("G&A") expenses for
the nine months ended September 30, 2021 were $37.2 million, as compared to
$14.9 million for the same period in 2020. The increase of $22.3 million was
driven primarily by 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,
more extensive governance requirements, and increased audit fees and expenses
related to US GAAP requirements, as well as an increase in non-cash
share-based compensation expense, primarily due to equity grants to employees
related to the completion of the Series C financing and the IPO.
· As of September 30, 2021, the Company had 35,814,255 ordinary shares
issued and 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 and
Fabry disease, as well as preclinical programs in Gaucher disease Type 1 and
hemophilia A. 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 A, there is a
deficiency of the clotting factor VIII (eight) protein and in hemophilia B,
there is a deficiency of the clotting factor IX (nine) protein. Hemophilia A
and B are X-linked diseases that mainly affect boys and men; however, women
who carry an affected copy of the clotting factor gene may also experience
symptoms. Hemophilia A is the most common type of hemophilia affecting about
one in every 5,000 males, while 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 in patients with
moderate and severe hemophilia. 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 is planned.
About Fabry Disease
Fabry disease is a genetic lysosomal disease that leads to a deficiency in
α-galactosidase A (α-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 α-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
α-Gal A that we believe 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 α-Gal A cDNA under the control of a liver-specific
promoter. The Company's current MARVEL-1 Phase 1/2 dose-finding trial
evaluates the 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 (GCase(var85)) under the control of a liver-specific promoter. The
GCase(var85) 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, timing, progress and results
of the Company's preclinical studies and clinical trials, including dosing of
a third patient in the Phase 1/2 MARVEL-1 dose-finding clinical trial of
FLT190, commencement of the Phase 1/2 B-LIEVE dose-confirmation clinical trial
of FLT180a and trial site initiation in the Phase 1/2 dose-finding clinical
trial of FLT201 and data readouts from those trials, whether we have
identified a sufficient number of patients to fully enroll the B-LIEVE trial
and completion of pre-clinical studies of FLT210; 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. 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 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
(http://www.sec.gov) .
Contact
David S. Arrington
Vice President Investor Relations & Corporate Communications
Freeline Therapeutics
david.arrington@freeline.life
+1 (646) 668 6947
Freeline Therapeutics Holdings plc
Unaudited Condensed Consolidated Statements of Operations Data
(in thousands of U.S. dollars, except per share data)
For the Nine Months Ended September 30,
2021 2020
OPERATING EXPENSES:
Research and development $ 70,827 $ 55,267
General and administrative 37,219 14,923
Total operating expenses 108,046 70,190
LOSS FROM OPERATIONS: (108,046) (70,190)
OTHER INCOME (EXPENSE) NET:
Other income (expense), net 493 748
Interest income, net 350 144
Benefit from R&D tax credit 1,541 11,127
Total other income (expense), net 2,384 12,019
Loss before income taxes (105,662) (58,171)
Income tax expense (29) (74)
Net loss (105,691) (58,245)
Net loss per share attributable to ordinary $ (2.96) $ (8.39)
shareholders-basic and diluted
Weighted average ordinary shares outstanding-basic 35,686,751 6,940,608
and diluted
Freeline Therapeutics Holdings plc
Unaudited Condensed Consolidated Balance Sheet Data
(in thousands of U.S. dollars, except per share data)
September 30, December 31,
2021 2020
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 136,377 $ 229,974
Account receivable - 97
Prepaid expenses and other current assets 22,161 28,105
Total current assets 158,538 258,176
Property and equipment, net 10,141 8,608
Intangible assets, net 10 23
Other non-current assets 2,077 1,805
Total assets $ 170,766 $ 268,612
LIABILITIES AND SHAREHOLDERS' EQUITY
CURRENT LIABILITIES:
Accounts payable $ 5,665 $ 8,093
Accrued expenses and other current liabilities 12,303 10,719
Total current liabilities 17,968 18,812
Total liabilities 17,968 18,812
Commitments and contingencies
SHAREHOLDERS' EQUITY:
Deferred shares 137 155
Additional paid-in capital 465,285 456,293
Accumulated other comprehensive loss 9,057 9,342
Accumulated deficit (321,681) (215,990)
Total shareholders' equity 152,798 249,800
TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 170,766 $ 268,612
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