LONDON - Syncona Ltd, a prominent life science investor, has announced through its portfolio company Spur Therapeutics that it has observed positive outcomes from the Phase I/II GALILEO-1 study of FLT201, a novel gene therapy candidate for Gaucher disease. The findings were presented at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) held in Rome, Italy.
The trial involved six patients who received a one-time infusion of FLT201 at a dose of 4.5e11 vg/kg. They have been monitored for a period ranging from seven to 15 months post-dosing. Efficacy results are based on five patients, with one excluded due to pre-existing neutralising antibodies against the AAVS3 capsid that delivers FLT201. Safety analyses included all participants, with the treatment showing a favorable safety and tolerability profile.
Data from the study, as of September 27, 2024, indicate no infusion reactions, dose limiting toxicities, or severe adverse events. The therapy has led to durable reductions in lyso-Gb1 levels, a key biomarker of clinical response in Gaucher disease, by 42% to 96%. These reductions were observed even in patients with high lyso-Gb1 levels despite previous enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).
Improvements in bone marrow burden were noted in all five patients, suggesting that FLT201 reaches deeper tissues not effectively targeted by existing treatments. Additionally, the therapy has been associated with the maintenance or improvement of spleen and liver volumes, hemoglobin levels, and platelet counts post withdrawal of ERT or SRT.
Chris Hollowood, CEO of Syncona Investment Management Limited and Chair of Spur Therapeutics, commented on the significance of the data, highlighting FLT201's potential to significantly impact the treatment landscape for Gaucher disease. He stated the data supports the durability of FLT201 and its potential to offer long-term benefits over current treatments. The positive trial outcomes also bolster the credibility of Spur's technology and aid in the progression of its pre-clinical pipeline into more common disorders like Parkinson's disease.
Syncona is preparing to support Spur Therapeutics as it moves towards initiating a Phase III trial in the calendar year 2025. This announcement is based on a press release statement from Syncona Ltd.
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