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RNS Number : 9258R
Syncona Limited
11 July 2022
Syncona Limited
Freeline presents data in haemophilia B at the ISTH Congress and
provides programme update
11 July 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"), has presented safety and initial efficacy data
from the first cohort of the Phase I/II dose-confirmation B-LIEVE
trial for FLT180a, the company's gene therapy candidate for people
with haemophilia B, at the International Society on Thrombosis and
Haemostasis (ISTH) Congress being held in London, 9-13 July
2022.
A summary of the data presented is as follows:
-- As of the data cut-off of 23 May 2022, a one-time FLT180a
dose of 7.7e11 vg/kg generated levels of factor IX (FIX) expression
in the normal range (93, 92 and 80 IU/dL) for the three patients in
cohort one, through days 77, 56 and 36, respectively. [1]
-- Patients stopped FIX prophylactic replacement therapy and did
not require FIX replacement or experience bleeding following
treatment with FLT180a.
-- The treatment and prophylactic immune management regimen were
well tolerated. No serious adverse events or infusion reactions
were observed. All adverse events were mild, and most were
transient.
-- Dosing of cohort two was completed in June 2022, with early
results showing a similar initial response to FLT180a.
Following the data cut-off, two patients in cohort one have
experienced a decrease in FIX expression together with a mild and
transient increase in liver enzymes. All patients continue to have
expression levels above baseline, with no patients experiencing a
bleed or requiring FIX supplementation.
Freeline continues to focus its resources on executing on its
strategic priorities to ensure that the company is well positioned
to deliver value. As a result, the company is evaluating strategic
options for FLT180a, which may include seeking a partner for the
programme to advance it through clinical development.
Chris Hollowood, Chief Investment Officer of Syncona Investment
Management Limited and Chair of Freeline said: "Freeline is
continuing to focus on delivering best-in-class or first-in-class
treatments to patients with debilitating systemic disorders. Whilst
we have continued to be encouraged by the data presented in
haemophilia B, Syncona is supportive of Freeline's decision to
review the appropriate path forward for the programme. Prioritising
the company's programmes in Gaucher and Fabry disease, which have
the potential to be first-in-class, positions the company to
deliver the best value for Freeline's shareholders, and impact for
patients with these devastating diseases. We continue to work
closely with the company as it executes on its strategic priorities
and advances these two lead programmes through the clinic."
The poster #PB0213 entitled: "Results from B-LIEVE, a Phase 1/2
Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients
with Hemophilia B" by Guy Young et al. will be made available on
the Investors section of Freeline's website. The full text of the
announcement from Freeline is 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 / Julia Bradshaw / 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.
Positive Initial Clinical Data from the B-LIEVE
Dose-Confirmation Trial for FLT180a in Hemophilia B Presented at
the International Society on Thrombosis and Haemostasis
Congress
FLT180a generated protective FIX levels with no bleeding or need
for FIX replacement
The treatment was well tolerated with a good safety profile
LONDON, July 10, 2022 - Freeline Therapeutics Holdings plc
(Nasdaq: FRLN) today announced the presentation of safety and
initial efficacy data from the first cohort of the Phase 1/2
dose-confirmation B--LIEVE trial for FLT180a, the company's
AAVS3-based gene therapy candidate for people with hemophilia B, at
the International Society on Thrombosis and Haemostasis (ISTH)
Congress being held in London, July 9-13, 2022.
As of the data cut-off of May 23, 2022, a one-time FLT180a dose
of 7.7e11 vg/kg generated a rapid increase of coagulation factor IX
(FIX), reaching levels in the normal range (93, 92 and 80 IU/dL)
for the three patients in cohort 1 through days 77, 56 and 36,
respectively. Patients stopped FIX prophylaxis and did not require
FIX replacement or experience bleeding following treatment with
FLT180a.
The treatment and the prophylactic immune management regimen
were well tolerated. No serious adverse events or infusion
reactions were observed, and there has been no evidence of FIX
inhibitors. All adverse events (AEs) were mild, and most were
transient. AEs related to immune management were consistent with
the known profiles of corticosteroids and tacrolimus.
Dosing of cohort two was completed in June, with early results
showing a similar initial response to FLT180a. Based on the data
from cohort one and consistent with the advice of the independent
Data Monitoring Committee of the B-LIEVE trial, patients in cohort
two received the same dose of FLT180a and prophylactic immune
management regimen that were used in the first cohort.
As the data continue to evolve since the data cut-off for cohort
one, two patients have experienced a decrease in FIX expression
together with a mild and transient increase in liver enzymes. All
patients continue to have expression levels above baseline, and no
patient has experienced a bleed or required FIX
supplementation.
"The initial data show that FLT180a provides rapid and
consistent elevations in FIX to normal levels, which can prevent
bleeding and the need for regular FIX replacement in people with
hemophilia B ," said Pamela Foulds, MD, Chief Medical Officer of
Freeline. "Emerging data suggest that while FIX expression was
maintained at protective levels, a further refined immune
management regimen may be required to avoid mild and transient
transaminitis and to sustain FIX levels in the normal range.
Potential adjustments in cohort two and forthcoming results from
that cohort will help us interpret this further."
"Our strategy is to advance gene therapy programs that have the
potential to deliver best-in-class or first-in-class treatments,"
said Michael Parini, Chief Executive Officer of Freeline. "While we
continue to believe FLT180a has the potential to deliver a
best-in-class gene therapy for people with hemophilia B, the
availability of other treatment options and the need to prioritize
our valuable resources dictate that we evaluate strategic options
for FLT180a. These include, but are not limited to, seeking a
partner that would enable the continuation of FLT180a through Phase
3 development."
"With two other promising programs in Fabry disease and Gaucher
disease in the clinic, each with the potential to be first-in-class
gene therapy treatments for these debilitating diseases, we will
continue to focus our attention and resources on those programs
that offer the highest value for patients and Freeline's
shareholders, " said Parini.
The poster #PB0213 entitled "Results from B-LIEVE, a Phase 1/2
Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients
with Hemophilia B" by Guy Young et al. will be presented from
6:30pm-7:30pm BST today and will be made available on the Investors
section of Freeline's website.
About the B-LIEVE Dose-Confirmation Trial
B-LIEVE is a Phase 1/2 dose confirmation trial of FLT180a using
a short course of prophylactic immune management with the goal of
normalizing FIX levels in patients with severe and moderately
severe hemophilia B . The starting dose of 7.7e11 vg/kg was
selected based on the results of the Phase 1/2 B-AMAZE dose-finding
trial and modeling. The goal of the B-LIEVE trial is to finalize a
dose for a pivotal Phase 3 trial that enables predictable and
sustained factor IX (FIX) expression at protective levels.
About FLT180a for People with Hemophilia B
Freeline's FLT180a candidate uses a potent AAVS3 capsid
rationally designed for effective targeting and transduction of
liver cells and containing an expression cassette encoding a gain
of function Padua variant of human factor IX (FIX). FLT180a has
been studied in B-AMAZE, a Phase 1/2 dose-finding trial with the
goal of normalizing FIX activity in patients with moderately severe
and severe hemophilia B. 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 to finalize a
dose for a Phase 3 pivotal trial is in progress.
About Hemophilia B
Hemophilia B is a rare, debilitating , hereditary bleeding
disorder caused by a defect in the gene encoding coagulation factor
IX (FIX). Hemophilia B is linked to the X chromosome and mainly
affects boys and men; however, women who carry an affected copy of
the coagulation factor gene may also experience symptoms.
Hemophilia B is classified as mild, moderate or severe, depending
on the level of FIX in the blood, and is diagnosed through blood
tests. The 2020 Annual Global Survey by the World Federation of
Hemophilia estimates that there are approximately 15,000 patients
with hemophilia B in the United States, Europe and Japan. A
meta-analysis using national registries in Australia, Canada,
France, Italy, New Zealand and the UK estimated a prevalence in
males of 3.8 in 100,000 or approximately 1 in 30,000. ([i])
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 may
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 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 U.S.
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 opinions, expectations, beliefs, plans,
objectives, assumptions or projections of Freeline Therapeutics
Holdings plc (the "Company") regarding future events or future
results, in contrast with statements that reflect historical facts.
Examples include, among other topics, statements regarding the
timing, progress and results of the Company's Phase 1/2 B-LIEVE
dose confirmation clinical trial of FLT180a and data readouts from
that trial, whether a further refined immune management regimen may
be required, the potential of FLT180a to deliver a best-in-class
gene therapy for people with hemophilia B, whether the Company's
evaluation of strategic options for FLT180a will result in any
particular course of action and the potential of the Company's
programs in Fabry disease and Gaucher disease to be first-in-class
gene therapy treatments for such diseases and offer the highest
value for patients and the Company's shareholders. In some cases,
you can identify such forward-looking statements by terminology
such as "anticipate," "intend," "believe," "estimate," "plan,"
"seek," "project" or "expect," "may," "will," "would," "could" or
"should," the negative of these terms or similar expressions.
Forward-looking statements are based on management's current
beliefs and assumptions and on information currently available to
the Company, and you should not place undue reliance on such
statements. Forward-looking statements are subject to many risks
and uncertainties, including the Company's recurring losses from
operations; the uncertainties inherent in research and development
of the Company's product candidates, including statements regarding
the timing of initiation, completion and the outcome of clinical
studies or trials and related preparatory work and regulatory
review, regulatory submission dates, regulatory approval dates
and/or launch dates, as well as risks associated with preclinical
and clinical data, including the possibility of unfavorable new
preclinical, clinical or safety data and further analyses of
existing preclinical, clinical or safety data; the Company's
ability to design and implement successful clinical trials for its
product candidates; whether the Company's cash resources will be
sufficient to fund the Company's foreseeable and unforeseeable
operating expenses and capital expenditure requirements for the
Company's expected timeline; the potential for a pandemic, epidemic
or outbreak of infectious diseases in the United States, United
Kingdom or European Union, 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, 2021 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
.
Media Contact:
Arne Naeveke, PhD
Vice President, Head of Corporate Communications
arne.naeveke@freeline.life
+1 617 312 2521
IR Contact:
investor@freeline.life
References
i Iorio A et al. Annals of Internal Medicine
2019;171(8):540-7
[1] FIX activity in the normal range is defined as between 50 to
150 IU/dL
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