FLT201 utilizes a proprietary AAVS3 capsid to deliver a more effective version of the GCase enzyme, aiming to alleviate symptoms and reduce the treatment burden for patients.

In the Phase 1/2 GALILEO-1 trial, FLT201 demonstrated rapid and sustained reductions in lyso-Gb1 levels, a key biomarker for Gaucher disease, along with improvements in blood counts and organ volumes.

The FDA has indicated alignment on seeking accelerated approval based on reductions in lyso-Gb1 levels, which are key predictors of clinical response.

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

Gaucher disease, which affects approximately 18,000 individuals across the US, UK, and major European nations, is caused by a deficiency in the glucocerebrosidase enzyme due to mutations in the GBA1 gene.

Despite existing treatments, many patients continue to experience persistent symptoms, underscoring the urgent need for more effective therapies.

Patients in the trial experienced durable reductions in lyso-Gb1 levels ranging from 33% to 96%, with some maintaining stable levels for over a year after discontinuing prior therapies.

Moreover, normal hemoglobin levels were maintained beyond a year after stopping enzyme replacement or substrate reduction therapies, with sustained improvements in platelet counts and organ volumes.

The upcoming Phase 3 trial will be a single-arm study involving approximately 40-45 adult patients who will serve as their own comparators.

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

The Phase 3 trial is expected to begin in the second half of 2025, with the first patient to be dosed then.

Spur Therapeutics is committed to advancing gene therapies for chronic conditions and is also exploring treatments for other diseases, including Parkinson's and cardiovascular diseases.