REG - Syncona Limited - Freeline reports Full Year 2021 Financial Results

SynconaAnnouncement

01/04/2022 07:15

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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/ (http://www.freeline.life/investors/) and the
full text of the announcement from Freeline is contained below.

  ENDS 

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 α-Gal A expression over two years in the first patient
and promising activity with near-normal α-Gal A levels in the second patient,
who was dosed in June 2021 and remained 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™, 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 patients 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 (http://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 31,                                             December 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 (mailto:arne.naeveke@freeline.life)

+1 617 312 2521

 

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