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RNS Number : 3587V
Syncona Limited
13 December 2021
Syncona Limited
Freeline presents long-term follow-up data in haemophilia B
and
initiates dose-confirmation trial
13 December 2021
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"), presented data from its long-term follow-up
study of its Phase I/II B-AMAZE dose-finding trial of FLT180a for
the treatment of haemophilia B during the 63(rd) American Society
of Hematology (ASH) Annual Meeting & Exposition, being held
between December 11-14, 2021.
The results presented demonstrated sustained factor IX (FIX)
expression up to 3.5 years, and supported selection of a dose of
7.7e11 vg/kg and an immune management regimen that has the
potential to deliver FIX activity in the normal range (50-150%) for
patients with haemophilia B. Additionally, the company announced
initiation of the first trial site in the Phase I/II B-LIEVE
dose-confirmation trial of FLT180a, one quarter ahead of its latest
guidance. Freeline expects the B-LIEVE study will finalise a dose
for the planned pivotal Phase III trial and anticipates an interim
data readout from B-LIEVE in mid-2022.
Chris Hollowood, Chief Investment Officer of Syncona Investment
Management Limited and Chair of Freeline said: "We are pleased to
see this long-term data in severe haemophilia B patients, showing
sustained expression of FIX for up to three and half years after
dosing. The ability of this gene therapy product candidate to
deliver durable FIX activity in the normal range, using relatively
low doses, suggests its potential to provide a functional cure for
patients with this disease. It is encouraging that Freeline has
initiated its dose-confirmation study earlier than previously
guided, and we look forward to seeing an update in 2022."
The announcement and poster presentation are available on the
Investors section of the Freeline website. A copy of the
announcement is also set out 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
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 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 Long-Term Follow-Up Data from Phase 1/2
B--AMAZE Trial in Hemophilia B at the 2021 ASH Annual Meeting and
Announces
Early Initiation of Phase 1/2 B-LIEVE Dose-Confirmation
Trial
B-AMAZE long-term follow up data demonstrate sustained FIX
expression up to 3.5 years; potential for expression levels in the
normal range with relatively low vector doses
B-LIEVE trial to confirm dose and immune management for planned
Phase 3 pivotal trial launched one quarter ahead of latest
guidance
LONDON, December 13, 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 presents long-term follow-up data from
its Phase 1/2 B-AMAZE dose-finding trial of FLT180a for the
treatment of hemophilia B at the 63(rd) American Society of
Hematology (ASH) Annual Meeting and Exposition. The event is being
held from December 11 - 14, 2021 at the Georgia World Congress
Center. Freeline also announced the first trial site in the Phase
1/2 B-LIEVE dose-confirmation trial of FLT180a has been initiated,
one quarter ahead of its latest guidance. The Company anticipates
an interim data readout from the B-LIEVE trial in the middle of
2022.
"The data presented today at ASH demonstrate that FLT180a gene
therapy has the potential to deliver a durable, functional cure for
hemophilia B," said Michael Parini, Chief Executive Officer of
Freeline. " Patients in the first cohort experienced sustained
expression of Factor IX out to 3.5 years following treatment with
FLT180a. The data from our B-AMAZE study also informed our B-LIEVE
dose-confirmation study, enabling us to identify a dose and
prophylactic immune management regimen that we believe can get and
keep hemophilia B patients in the normal range of Factor IX
expression. These results provide an important proof-of-concept for
the Freeline platform, demonstrating the proprietary, rationally
designed AAVS3 capsid has the potential to normalize FIX activity
in patients with relatively low dosing due to its high
potency."
Mr. Parini continued, "We continue to execute across our
programs, initiating the B-LIEVE study, our Phase 1/2 dose
confirmation trial, ahead of schedule. We are excited to get this
trial started and completed to enable a Phase 3 pivotal study .
Enrollment in the ECLIPSE run-in study for the B-LIEVE trial has
proceeded more quickly than expected and, as a result, we believe
we have identified a sufficient number of patients to fully enroll
the B-LIEVE trial. We look forward to continued progress with
FLT180a as we work toward improving the lives of patients with
hemophilia B."
ASH Presentation Highlights
Key Findings
-- Results suggest that a dose of 7.7e11 vg/kg, coupled with a
short course of prophylactic immune management, has the potential
to achieve durable FIX activity in the normal range (50-150%). and
thereby prevent spontaneous bleeds and normalize hemostasis in the
event of traumatic bleeds.
-- Median follow up of 27.2 months (range 19.1-42.4) post-dosing across 10 patients.
-- Dose-dependent increase in FIX activity observed and FIX
expression sustained in nine of 10 patients.
-- Annualized exogenous FIX consumption and bleeding declined
after gene therapy. Across the nine of 10 patients with sustained
FIX expression after FLT180a treatment, only one reported traumatic
bleed was treated with FIX replacement; it occurred in a patient
who had an endogenous FIX level of 57% at the time.
-- The data cutoff date was September 20, 2021.
Safety
-- FLT180a was generally well tolerated with a favorable safety profile.
-- No infusion or allergic reactions and no evidence of FIX inhibitors were reported.
-- Transient transaminitis with or without an associated decline
in FIX was the most common FLT180a-related adverse event (AE).
B-LIEVE Trial Site Initiation
Freeline has begun to apply these findings to advance the
B-LIEVE trial, a Phase 1/2 dose-confirmation trial of FLT180a
manufactured at commercial scale and using a starting dose of
7.7e11 vg/kg and a short course of prophylactic immune management.
The first trial site was initiated on December 6, 2021.
The starting dose of FLT180a in B-LIEVE was selected based on
the results of B-AMAZE and multiple modeling approaches. Freeline
expects the B-LIEVE trial will finalize a dose for the planned
Phase 3 pivotal trial that is intended to consistently enable FIX
expression in the normal range for patients with hemophilia B.
Freeline is currently evaluating the timing of its Phase 3
pivotal trial and filing of a Biologics License Application and
will provide more concrete guidance next year.
ASH Poster Presentation Details
Title: Factor IX Expression within the Normal Range Prevents
Spontaneous Bleeds Requiring Treatment Following FLT180a Gene
Therapy in Patients with Severe Hemophilia B: Long-term Follow-up
Study of the B-AMAZE Program
Presenter: Pratima Chowdary, MD, MRCP, FRCPath, Katharine
Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital and
University College London
Date & Time: December 13, 2021, 6:00 pm - 8:00 pm ET
Publication #: 3967
Session Name: 801. Gene Therapies: Poster III
The poster presentation is available online on the Investors
section of the Freeline website.
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 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. 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 has been
initiated.
About Hemophilia
Hemophilia is a genetic bleeding disorder caused by a deficiency
in clotting factor protein that impairs blood clot formation. In
hemophilia B, there is a deficiency of the clotting factor IX
(nine) protein. Hemophilia B is an X-linked disease that mainly
affects boys and men; however, women who carry an affected copy of
the clotting factor gene may also experience symptoms. 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.
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 initiation, timing, progress and results
of the Company's clinical trials, including data readouts from the
Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a,
whether the Company has identified a sufficient number of patients
to fully enroll the B-LIEVE trial; and whether a dose of 7.7e11
vg/kg will be successful in preserving FIX activity levels in the
normal range. 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
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