FLT201 aims to alleviate symptoms of Gaucher disease by delivering a stable version of the GCase enzyme using Spur's proprietary AAVS3 capsid, potentially halting disease progression.

Chris Hollowood, CEO of Syncona, expressed optimism about FLT201's potential to become a leading gene therapy for Gaucher disease, especially for patients transitioning from existing therapies.

The upcoming Phase 3 trial will involve transitioning patients off prior enzyme replacement or substrate reduction therapies before administering a single low-dose infusion of FLT201.

Gaucher disease affects around 18,000 individuals across the US, UK, and major European nations, resulting from a deficiency in the glucocerebrosidase enzyme.

Data from the Phase I/II GALILEO-1 trial indicated that patients maintained normal hemoglobin levels for over a year following a single infusion of FLT201.

Spur Therapeutics recently received positive feedback from the FDA regarding its Phase 3 trial for FLT201, a gene therapy targeting Gaucher disease type 1.

The primary endpoint for this trial will focus on the maintenance or improvement of hemoglobin levels after one year, a critical regulatory measure for Gaucher disease.

In addition to hemoglobin levels, key secondary endpoints will assess improvements in platelet counts, liver and spleen volumes, and overall patient-reported outcomes.

Safety analysis from the trial revealed no severe adverse events related to FLT201, although one patient with neutralizing antibodies was excluded from efficacy analysis.

Spur is preparing to select over 45 clinical sites across multiple regions, including the US, Canada, UK, Europe, Israel, and Latin America for the Phase 3 study.

The FDA has shown alignment on the potential for accelerated approval based on reductions in glucosylsphingosine (lyso-Gb1) levels, which are key indicators of clinical response.

The Phase 3 trial is expected to commence in the second half of 2025, with projections for completion by the end of 2027, marking a significant milestone for Syncona.