The Phase 3 trial is anticipated to commence in the latter half of 2025, with the first patient expected to be dosed during that period.

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

This upcoming trial will be a single-arm study involving approximately 40 to 45 adult patients who will act as their own comparators.

The FDA has expressed alignment with Spur on pursuing accelerated approval based on reductions in glucosylsphingosine (lyso-Gb1) levels, which are key indicators of clinical response in Gaucher disease.

Spur Therapeutics is in the process of selecting over 45 clinical sites across the US, Canada, UK, Europe, Israel, and Latin America for the Phase 3 study.

Key secondary endpoints will assess improvements in platelet counts, liver and spleen volumes, as well as bone health and overall patient-reported well-being.

Gaucher disease currently affects around 18,000 individuals in various countries, including the US, UK, and several major European nations.

The primary endpoint for the trial's full approval will focus on the maintenance or improvement of hemoglobin levels after one year, a crucial regulatory measure for Gaucher disease.

Before receiving a single low-dose infusion of FLT201, patients will transition off their prior enzyme replacement or substrate reduction therapies.

FLT201 employs a proprietary AAVS3 capsid to deliver a longer-acting version of the enzyme that patients with Gaucher disease lack, aiming to alleviate symptoms and lessen treatment burdens.

Previous results from the Phase 1/2 GALILEO-1 trial showed that FLT201 led to rapid and sustained reductions in lyso-Gb1, supporting its progression to Phase 3.

In addition to FLT201, Spur Therapeutics is focused on developing gene therapies for chronic conditions and is exploring treatments for other diseases such as Parkinson's and cardiovascular diseases.