REG - Syncona Limited - Freeline announces further data

SynconaAnnouncement

19/01/2024 07:00

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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
(https://freeline.life/investors/sec-filings/) .

 

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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