Syncona’s portfolio company, Freeline, releases positive data on gene therapy for Gaucher disease

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Syncona Ltd (LON:SYNC), a leading healthcare company focused on creating, building and scaling global leaders in life science, has noted that its portfolio company, Freeline Therapeutics Holdings plc (Nasdaq:FRLN) 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.”

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