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Syncona Limited
16 August 2021
Syncona Limited
Freeline Reports Executive Change and Second Quarter Financial
Results
16 August 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 that
Michael J. Parini has succeeded Theresa Heggie as Chief Executive
Officer ("CEO"). Freeline also reported financial results for the
second quarter of 2021 and provided updates on its lead program
FLT180a for Hemophilia B, FLT190 for Fabry disease, FLT201 for
Gaucher disease Type 1 and FLT210 for Hemophilia A. The key updates
are as follows:
-- On track for three programs in the clinic by year end; trial
site initiation for FLT180a for Hemophilia B and FLT201 for Gaucher
disease Type 1 expected by year end; program for FLT190 for Fabry
disease progressing
-- Completed first dose cohort for FLT190 Phase 1/2 dose-finding
trial and advancing to next dose level; data to be presented by
year end
-- Updated durability data from FLT180a Phase 1/2 dose-finding
study to be presented by year end
-- Defined strategic priorities to accelerate value creation;
near-term efficiencies extend cash runway into first quarter of
2023
-- Michael J. Parini, previously Freeline's President and Chief
Operating Officer, succeeds Theresa Heggie as CEO and following the
departure of Julie Krop as Chief Medical Officer, Alison Long, MD,
PhD, SVP, Head of Clinical Development, will serve in the role of
interim Chief Medical Officer while an external search for a new
CMO is underway
Chris Hollowood, Chief Investment Officer of Syncona Limited and
Chair of Freeline said: "I am pleased to announce that the Freeline
Board of Directors has appointed Michael J. Parini as CEO and
Executive Director of Freeline effective immediately. Michael has a
long track record of building strong executive leadership teams and
has had a significant impact on Freeline since he joined from
Vertex Pharmaceuticals. In a short period of time, he has mobilized
program execution across our pipeline, increased focus on our core
scientific and platform technology capabilities, and driven
efficiencies across the organization that have extended our cash
runway. In doing so, Michael has demonstrated his ability to unlock
value at Freeline and deliver on the promise of its therapies and
science to patients.
I wish to thank Theresa for leading Freeline through the
successful completion of its initial public offering last year and
the extraordinary challenges of the COVID-19 pandemic. We wish her
well in her future endeavors."
The announcement can be accessed on Freeline's website at:
https://www.freeline.life/investors/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 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 Appoints Michael J. Parini as Chief Executive Officer
and Reports Second Quarter 2021 Financial Results
On track for three programs in the clinic by year end; trial
site initiation for FLT180a for Hemophilia B and FLT201 for Gaucher
disease Type 1 expected by year end; program for FLT190 for Fabry
disease progressing
Completed first dose cohort for FLT190 Phase 1/2 dose-finding
trial and advancing to next dose level; data presented by year
end
Updated durability data from FLT180a Phase 1/2 dose-finding
trial presented by year end
Defined Company strategic priorities to accelerate value
creation; near-term efficiencies extend cash runway into first
quarter of 2023
LONDON, August 16, 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 that Michael J. Parini has
succeeded Theresa Heggie as Chief Executive Officer ("CEO"). The
Company also reported financial results for the second quarter of
2021 and provided updates on its lead program FLT180a for
Hemophilia B, FLT190 for Fabry disease, FLT201 for Gaucher disease
Type 1 and FLT210 for Hemophilia A.
"Today we are pleased to announce that Freeline's Board of
Directors has appointed Michael J. Parini as CEO and Executive
Director of the Company, effective immediately," said Dr. Chris
Hollowood, Chairman of Freeline. "Michael has a long track record
of building strong executive leadership teams and has had a
significant positive impact on Freeline since he joined the Company
from Vertex Pharmaceuticals. In a short period of time, he has
mobilized program execution across our pipeline, increased focus on
our core scientific and platform technology capabilities, and
driven efficiencies across the organization that have extended our
cash runway. In doing so, Michael has demonstrated his ability to
unlock value at Freeline and deliver on the promise of its
therapies and science to patients."
Dr. Hollowood continued, "I wish to thank Theresa for leading
the Company through the successful completion of its initial public
offering last year and the extraordinary challenges of the COVID-19
pandemic. We wish her well in her future endeavors."
"Freeline is at the forefront of gene therapy, leveraging a
platform innovation engine that holds the potential to deliver
functional cures to patients who suffer from debilitating
diseases," said Mr. Parini, CEO of Freeline. "I joined Freeline to
deliver on the differentiated promise of our pipeline and
technology, and am honored and excited to take the helm at this
critical time for the Company."
Mr. Parini continued, "I see three immediate strategic
priorities as we turn the page on a new chapter for the Company.
First and foremost is our commitment to advancing our pipeline with
urgency for patients. Our recent execution in the clinic is a
testament to this commitment, and we remain on track to deliver all
expected data and developmental milestones across our four clinical
and pre-clinical stage gene therapy programs before year end.
"Our second strategic priority is to drive increased focus,
financial discipline and operational efficiency in our business.
Based on a thorough review of our operations, we have identified
initial savings to extend our cash runway by nearly two quarters
into the first quarter of 2023, funding operations through multiple
value-generating milestones. We will continue this work to optimize
our business, operational and platform strategy.
"Finally, our third priority is to unlock the full value in our
proprietary scientific and platform technology. We are refreshing
our scientific strategy to leverage our unique combination of
manufacturing quality, capsid potency and protein engineering
capabilities, which we believe can drive lower and safer doses for
high therapeutic effect, into the next wave of pipeline programs.
We plan to focus our research and platform efforts where our
differentiated capabilities allow us to address unmet needs and
create transformative treatments for patients."
Mr. Parini added, "We believe gene therapy holds tremendous
promise. With our three strategic priorities in place, I look
forward to building a world-class team and organization to realize
the full potential of Freeline."
Key Pipeline and Operational Updates
Hemophilia B
-- On track to report up to four-year durability data from the
Company's Phase 1/2 dose-finding trial of FLT180a for the treatment
of Hemophilia B by year end.
-- Actively enrolling the ECLIPSE run-in study for the planned
six-month Phase 1/2 dose-confirmation trial of FLT180a.
-- On track to initiate clinical trial sites for the Phase 1/2
dose-confirmation trial by year end. The trial is designed to
confirm the dose and immune management regimen for the planned
Phase 3 pivotal trial of FLT180a. The Company is targeting full
enrollment of the Phase 1/2 trial during the first half of 2022 and
a six-month data readout by the end of 2022.
-- Pursuing Accelerated Approval with the US Food & 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.
Targeting initiation of the planned Phase 3 pivotal trial targeted
by mid-2023, with data readout and filing of a Biologics License
Application ("BLA") targeted by the end of 2024.
Fabry Disease
-- The first patient in the first and lowest-dose cohort of the
Phase 1/2 dose-finding trial assessing the safety and efficacy of
FLT190 for the treatment of Fabry disease in adult patients
continues to show sustained, elevated level of plasma GLA activity
over nearly two years post-dosing.
-- The second patient was dosed in June, completing the first
dose cohort. On track to dose additional patients at the next dose
cohort level and report data by year end.
-- Presented data at the American Society of Gene and Cell
Therapy ("ASGCT") Annual Meeting in May 2021 demonstrating GLA
uptake and metabolic cross-correction in Fabry disease relevant
cell lines.
Gaucher Disease (Type 1)
-- On track to initiate the Phase 1/2 dose-finding trial of
FLT201 for the treatment of Gaucher disease Type 1 by year end.
-- FDA Granted Orphan Drug Designation and the European
Medicines Agency issued a positive opinion on Orphan Drug
Designation for FLT201.
Hemophilia A
-- On track to complete preclinical IND/CTA enabling studies of
FLT210 for the treatment of Hemophilia A by year end.
Platform Technology
-- Presented four posters at the ASGCT Annual Meeting in May
2021 detailing advancements in the Company's high-throughput rAAV
manufacturing platform optimization and candidate selection,
development scale-up of suspension cell-based AAV manufacturing and
quantification assay strategy for AAV-based gene therapies, as well
as assay development to measure transduction efficiency.
Corporate
-- Announced leadership transformation with the appointment of
Michael J. Parini as CEO. Mr. Parini assumed the role of CEO and
Executive Director effective immediately, replacing former CEO
Theresa Heggie. Since March of 2021, Mr. Parini served as the
Company's President and Chief Operating Officer ("COO") and is an
experienced public company biotechnology executive with previous
leadership roles at both Vertex Pharmaceuticals, Inc. and Pfizer
Inc.
-- Announced that Alison Long, MD, PhD, SVP, Head of Clinical
Development, will serve in the role of interim Chief Medical
Officer ("CMO") while an external search for a new CMO is
conducted. Dr. Long has been instrumental in catalyzing the
momentum in the Company's clinical and pre-clinical programs. She
is an established leader with 30 years of combined clinical and
industry experience, including extensive gene therapy drug
development experience previously leading clinical departments and
programs at uniQure N.V. and, most recently, Spark Therapeutics,
Inc., where she was Head of Clinical Research &
Development.
Q2 2021 Financial Highlights
Cash Position: Cash and cash equivalents were $164.7 million as
of June 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.
R&D Expenses: Research and development ("R&D") expenses
for the six months ended June 30, 2021 were $48.1 million, as
compared to $29.4 million for the same period in 2020. The increase
of $18.7 million was driven by increased investment in activities
related to the current and proposed clinical trials for FLT201 and
FLT210 and overall research and development, which includes earlier
pipeline programs and further development of the Freeline
platform.
G&A Expenses: General and administrative ("G&A")
expenses for the six months ended June 30, 2021 were $24.6 million,
as compared to $11.7 million for the same period in 2020. The
increase of $12.9 million was driven primarily by an increase in
personnel expenses, primarily due to an increase in headcount in
corporate, legal, general and administrative functions to support
the Company's growth initiatives and public company 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 June 30, 2021, the Company had 35,802,840 ordinary shares
outstanding.
About Michael J. Parini
Michael Parini joined Freeline in March of 2021 to serve as the
Company's President and Chief Operating Officer. Before coming to
Freeline, Mr. Parini was the Executive Vice President and Chief
Administrative, Legal and Business Development Officer at Vertex
Pharmaceuticals, Inc. from March 2020 until February 2021, among
other roles he held in his over five years with the company. Prior
to Vertex, Michael Parini spent over a decade at Pfizer Inc.
serving in multiple leadership roles within the company's global
legal team, including Senior Vice President and Associate General
Counsel.
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 Type 1 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, including its review
of strategy and operations; 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
and full enrollment 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 dose-finding clinical trial
of FLT190 and data readouts from that trial, commencement of the
Phase 1/2 dose-finding clinical trial of FLT201, and completion of
pre-clinical IND/CTA-enabling studies of FLT210; 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 .
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 Six Months Ended
June 30,
------------------------------------------------------------------
2021 2020
-------------------------------- --------------------------------
OPERATING EXPENSES:
Research and development $ 48,132 $ 29,445
General and administrative 24,615 11,663
Total operating expenses 72,747 41,108
-------------------------------- --------------------------------
LOSS FROM OPERATIONS: (72,747) (41,108)
OTHER INCOME (EXPENSE) NET:
Other income (expense), net (3,367) 862
Interest income, net 265 88
Benefit from R&D tax credit 1,050 6,894
-------------------------------- --------------------------------
Total other income (expense), net (2,052) 7,844
-------------------------------- --------------------------------
Loss before income taxes (74,799) (33,264)
Income tax expense (16) -
-------------------------------- --------------------------------
Net loss (74,815) (33,264)
================================ ================================
Net loss per share attributable
to ordinary
shareholders-basic and diluted $ (2.10) $ (3.51)
================================ ================================
Weighted average ordinary shares
outstanding-basic
and diluted 35,668,423 9,472,577
================================ ================================
Freeline Therapeutics Holdings plc
Unaudited Condensed Consolidated Balance Sheet Data
(in thousands of U.S. dollars, except per share data)
June 30 December 31,
----------- -----------------------------
2021 2020
----------- -----------------------------
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 164,682 $ 229,974
Account receivable - 97
Prepaid expenses and other
current assets 23,622 28,105
----------- -----------------------------
Total current assets 188,304 258,176
Property and equipment,
net 10,050 8,608
Intangible assets, net 11 23
Other non-current assets 2,689 1,805
Total assets $ 201,054 $ 268,612
=========== =============================
LIABILITIES, PREFERRED SHARES
AND SHAREHOLDERS' EQUITY
CURRENT LIABILITIES:
Accounts payable $ 4,550 $ 8,093
Accrued expenses and other
current liabilities 10,873 10,719
Total current liabilities 15,423 18,812
----------- -----------------------------
Total liabilities 15,423 18,812
=========== =============================
Commitments and contingencies
SHAREHOLDERS' EQUITY:
Deferred shares 137 155
Additional paid-in capital 462,104 456,293
Accumulated other comprehensive
loss 14,195 9,342
Accumulated deficit (290,805) (215,990)
----------- -----------------------------
Total shareholders' equity 185,631 249,800
TOTAL LIABILITIES AND SHAREHOLDERS'
EQUITY $ 201,054 $ 268,612
=========== =============================
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