Syncona Limited Freeline announces further data (2116A)
19 Enero 2024 - 1:00AM
UK Regulatory
TIDMSYNC
RNS Number : 2116A
Syncona Limited
19 January 2024
Syncona Limited
Freeline announces further data in Gaucher programme
19 January 2024
Syncona Ltd, a leading healthcare company focused on creating,
building and scaling global leaders in life science, notes that its
portfolio company, Freeline Therapeutics Holdings plc (Nasdaq:
FRLN) ("Freeline") has announced further safety, tolerability and
enzyme activity data from the ongoing Phase I/II GALILEO-1 trial
evaluating FLT201, its novel gene therapy candidate, in Gaucher
disease, a debilitating genetic disorder caused by a deficiency of
the GCase enzyme.
Highlights include:
-- Freeline has now dosed five patients in the Phase I/II
GALILEO-1 trial to date; four patients had evaluable data and a
summary of the data is covered below.
-- FLT201 continued to be well tolerated with no serious adverse
events observed and all treatment-related adverse events were
mild.
-- FLT201 continued to induce robust increases in plasma GCase
activity demonstrating strong expression from the liver.
-- The levels of plasma GCase for the first three patients dosed
in the trial peaked between 9- and 11-weeks post-dosing:
o The levels of GCase in the first two patients reduced from
these peaks as the patients finished the prophylactic immune
management regimen in line with what has been seen in other gene
therapies.
-- Normalisation of GCase activity in leukocytes was observed
within four weeks of dosing; a key parameter for patients with
Gaucher disease. Patient 1 levels at the most recent timepoint are
below the normal range but remain substantially above baseline. All
other patients remain above the normal range.
-- Alaine transaminase (ALT) is an enzyme that is found in the
liver and can be used as a marker of liver toxicity or damage.
Controlling the level of ALTs in patients that receive
liver-directed gene therapy is believed to be key to ensuring
long-term stable expression of the therapy.
-- ALTs remain in the normal range for all patients that entered
the study in the normal range:
o One patient who entered the study with mildly elevated ALTs
has remained within 2.5 times the upper limit of normal.
-- Lyso-Gb1 is a well-established biomarker of clinical response
in Gaucher disease and reductions in lyso-Gb1 correlate with
positive clinical outcomes for Gaucher patients:
o Substantial reductions in lyso-Gb1 levels have been seen in
two patients who entered the trial with elevated levels despite
receiving enzyme replacement therapy or substrate reduction
therapy.
-- Syncona expects further data from the trial to be published in H2 CY2024.
This data has been published within the Scheme Circular relating
to the proposed acquisition of Freeline by Syncona, prepared in
accordance with Section 13E-3 of the Exchange Act. This document
has been filed with the US Securities and Exchange Commission and
is available on Freeline's website .
The acquisition is subject to approval or acceptance by the
required number of Freeline's minority shareholders and is expected
to close in the first quarter of 2024, with a General Meeting in
which shareholders will vote on the proposed acquisition scheduled
for 12 February 2024.
Chris Hollowood, CEO of Syncona Investment Management Limited,
said: "The data released to date from Freeline continues to show
the transformative potential of the FLT201 therapy to target the
root cause of Gaucher disease and provide a one-time, targeted
treatment. We look forward to dosing more patients and generating
longer term data from those already dosed patients that will inform
the path to a pivotal study. We continue to support the Freeline
management team as they focus on execution and believe that taking
the company private under our ownership provides the best route for
developing this therapy and bringing it to patients."
[ENDS]
Enquiries
Syncona Ltd
Annabel Clark
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 creating and building companies to deliver
transformational treatments to patients in areas of high unmet
need.
Our strategy is to create, build and scale companies around
exceptional science to create a diversified portfolio of 20-25
globally leading healthcare businesses, across development stage
and therapeutic areas, 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.
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.
Syncona Limited seeks to achieve returns over the long term.
Investors should seek to ensure they understand the risks and
opportunities of an investment in Syncona Limited, including the
information in our published documentation, before investing.
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