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RNS Number : 0196B
Syncona Limited
08 February 2022
Syncona Limited
Freeline presents data in Fabry disease
8 February 2022
Syncona Ltd, a leading healthcare company focused on founding,
building and funding global leaders in life science, notes that its
portfolio company, Freeline Therapeutics Holdings plc (Nasdaq:
FRLN) ("Freeline"), will present encouraging new data from its
ongoing Phase I/II MARVEL-1 dose-finding clinical trial of FLT190
in Fabry disease at the 18(th) Annual WORLDSymposium(TM) taking
place on February 7-11, 2022 in San Diego, California.
Highlights from the data:
-- FLT190 continues to be well tolerated with previously
observed mild myocarditis found not to be associated with enduring
clinical sequelae
-- Results from the first dose cohort continue to demonstrate
promising efficacy with a potential dose-dependent increase in
levels of the key enzyme (<ALPHA>-Gal A), which is absent or
markedly deficient in Fabry patients. Durable levels of
<ALPHA>-Gal A sustained up to two years following dosing in
Patient 1, with Patient 2 continuing to remain off enzyme
replacement therapy more than 26 weeks post-dosing [1]
Freeline will also separately present further detail on the
trial design of the Phase I/II GALILEO-1 study of FLT201 in Gaucher
disease.
Chris Hollowood, Chief Investment Officer of Syncona Investment
Management Limited and Chair of Freeline said: "We are pleased to
see this data from the Fabry study, which further demonstrates that
the therapy has an encouraging safety and efficacy profile.
Continued <ALPHA>-Gal A expression in the first patient
following dosing in 2019 suggests good durability in response to
the initial dose level, suggesting this could be a potentially
transformative treatment for patients with Fabry disease. We look
forward to seeing further data published later in the year in the
Fabry study."
The poster presentations will be available on the Investors
section of Freeline's website following presentation at the
WORLDSymposium(TM) conference. Freeline has issued a release to
introduce the presentations, which is also available on the company
website, with the full text contained below.
[S]
Copies of this press release and other corporate information can
be found on the company website at: www.synconaltd.com
Forward-looking statements - this announcement contains certain
forward-looking statements with respect to the portfolio of
investments of Syncona Limited. These statements and forecasts
involve risk and uncertainty because they relate to events and
depend upon circumstances that may or may not occur in the future.
There are a number of factors that could cause actual results or
developments to differ materially from those expressed or implied
by these forward-looking statements. In particular, many companies
in the Syncona Limited portfolio are conducting scientific research
and clinical trials where the outcome is inherently uncertain and
there is significant risk of negative results or adverse events
arising. In addition, many companies in the Syncona Limited
portfolio have yet to commercialise a product and their ability to
do so may be affected by operational, commercial and other
risks.
Enquiries
Syncona Ltd
Natalie Garland-Collins / Fergus Witt
Tel: +44 (0) 7714 916615
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 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 Presents on Its Fabry and Gaucher Disease AAV-Based
Gene Therapies at the 18(th) Annual WORLDSymposium(TM)
FLT190 well-tolerated with promising early efficacy in Fabry
disease including sustained <ALPHA>-Gal A expression up to
two years
GALILEO-1, a first-in-human, open-label, international,
multicenter Phase 1/2 clinical trial evaluating FLT201 in Gaucher
disease Type 1 initiated
LONDON, February 8, 2022 - Freeline Therapeutics Holdings plc
(Nasdaq: FRLN) (the "Company" or "Freeline"), a clinical-stage
biotechnology company developing transformative AAV-mediated gene
therapies for people with inherited systemic debilitating diseases,
will present updated data from the ongoing Phase 1/2 MARVEL-1
clinical trial evaluating FLT190 for the treatment of patients with
Fabry disease, and the clinical trial design for GALILEO-1, a Phase
1/2 safety and efficacy study of FLT201 in adult patients with
Gaucher disease Type 1 at the 18(th) Annual WORLDSymposium(TM)
taking place February 7 - 11, 2022 in San Diego, California.
"Freeline's proprietary AAVS3 capsid has the potential to
deliver transformative treatments for patients with challenging
diseases including Fabry disease and Gaucher disease," said Pam
Foulds, MD, Freeline's Chief Medical Officer. "It is highly
encouraging to observe early signals of durability and efficacy in
MARVEL-1 in Fabry disease, with sustained expression up to two
years as of our last data reading. Advancing the study will allow
further exploration of the potential of FLT190 to provide a
functional cure for Fabry disease, which may eliminate the need for
enzyme replacement therapy and improve patient outcomes. With
FLT201 in Gaucher disease Type 1 advancing to first-in-human
studies, I believe the Freeline approach, which leverages a novel
capsid and engineered, stable GCase variant, has the potential to
overcome the limitations of currently available treatments for
Gaucher by addressing difficult-to-reach tissues including bone
marrow and lung."
Michael Parini, Chief Executive Officer of Freeline, said, "We
are proud of our steady execution in the clinic and believe this
continued progress showcases the Freeline team's dedication to
advancing new therapies for patients with great unmet need. Both
the MARVEL-1 and GALILEO-1 studies leverage the strengths of the
proprietary Freeline platform, with a highly potent AAVS3 capsid,
robust CMC, and protein engineering expertise. We will continue to
push forward cutting-edge science, and lead the way in safety, to
deliver new medicines to patients with the potential to change the
standard-of-care."
Highlights from the presentation titled, "Safety and efficacy of
FLT190 for the treatment of patients with Fabry disease: Results
from the MARVEL-1 Phase 1/2 clinical trial," presented by Derralynn
A. Hughes, include:
-- FLT190 has been well-tolerated ; a novel prophylactic immune
management regimen may have prevented vector-related transaminitis
in Patient 2
-- Results from the first dose cohort (7.5 x 10(11) vg/kg)
demonstrate promising efficacy with a potential dose-dependent
increase in plasma <ALPHA>-Gal A levels, durable
<ALPHA>-Gal A levels sustained for up to two years in Patient
1, and with Patient 2 continuing to remain off ERT more than 26
weeks post-dosing as of the data cut-off date of December 22,
2021
-- Mild, transient myocarditis was observed in both patients
approximately 6-8 weeks post-dose of FLT190 which was not
associated with enduring clinical sequelae on cardiac MRI and
suggests that cardiac monitoring may be valuable in gene therapy
trials for conditions like Fabry disease with known underlying
cardiac manifestations
Highlights from the presentation titled "Design of GALILEO-1, a
Phase 1/2 safety and efficacy study of FLT201 in adult patients
with Gaucher disease Type 1," presented by Derralynn A. Hughes
include:
-- FLT201 consists of a 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 promotor. FLT201 produces robust and sustained
secretion of GCase in preclinical models, as well as increased
stability at lysosomal and physiological pH in human serum
-- GCase(var85) contains two novel amino acid substitutions that
result in a 6-fold increase in half-life in human serum, and
20-fold increase in half-life at lysosomal pH conditions
-- GALILEO-1 is a first-in-human, open-label, international,
multicenter Phase 1/2 clinical trial evaluating a single
intravenous infusion of FLT201 with a novel prophylactic immune
management regimen to prevent vector-related transaminitis
-- Study objectives include evaluating the safety and
tolerability of FLT201, investigating the relationship of FLT201
dose to production of endogenous GCase and determining the
potential to improve clinical phenotype by reduction in the GCase
substrate
-- The adaptive dose-escalation study will identify a dose of
FLT201 for further development in a Phase 3 clinical trial, and
will include two parts: Part 1, in patients previously treated with
enzyme replacement therapy (ERT) or substrate reduction therapy
(SRT), and Part 2, previously untreated patients
The poster presentations will be available on the Investors
section of Freeline's website following presentation at the
WORLDSymposium(TM) conference.
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.
About Fabry Disease
Fabry disease is a genetic lysosomal disease that leads to a
deficiency in <ALPHA>-galactosidase A (<ALPHA>-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.
Freeline is currently focused on classic Fabry disease where
patients have little to no functional <ALPHA>-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 <ALPHA>-Gal A that the Company believes 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
<ALPHA>-Gal A cDNA under the control of a liver-specific
promoter. The Company's current MARVEL-1 Phase 1/2 dose-finding
trial is evaluating the potential 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 or oral substrate
reduction therapy (SRT). 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 GCase that the Company believes
would eliminate the need for ERT or SRT.
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.
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,
statements regarding the dosing, timing, progress and interim and
final results of the Company's Phase 1/2 MARVEL-1 dose-finding
clinical trial of FLT190 and Phase 1/2 GALILEO-1 dose-finding
clinical trial of FLT201 and statements regarding the potential of
the Company's product candidates to deliver transformative
treatments or change the standard-of-care for patients with
challenging diseases, including FLT190's potential to provide a
functional cure for Fabry disease, eliminate the need for enzyme
replacement therapy and improve patient outcomes, FLT201's
potential to overcome the limitations of currently available
treatments for Gaucher disease by addressing difficult-to-reach
tissues including bone marrow and lung. 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. 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
.
IR Contact:
investor@freeline.life
Media Contact:
media@freeline.life
[1] Data cut off date of December 22, 2021
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