Spur Therapeutics announced preclinical data for FLT201, its gene therapy candidate for Gaucher disease, published in Molecular Therapy. The data demonstrates FLT201’s potential to surpass the current standard of care, enzyme replacement therapy (ERT), by delivering a more stable version of the deficient enzyme. This publication supports FLT201’s progression into clinical development.
This advancement is crucial for Gaucher disease patients who often experience persistent symptoms like pain, fatigue, and bone disease despite lifelong ERT infusions. A single-dose gene therapy offering superior and durable efficacy could significantly improve patients’ quality of life and reduce the substantial treatment burden associated with bi-weekly infusions. The potential for FLT201 to effectively target tissues like bone and lung, which are difficult for ERT to reach, further underscores its importance.
Preclinical studies revealed that the engineered enzyme in FLT201, GCase85, exhibited significantly greater stability and tissue uptake compared to both ERT and wild-type gene therapy. This resulted in superior reduction of disease-causing substrates in affected tissues. Furthermore, a single FLT201 infusion in non-human primates demonstrated sustained enzyme activity for 3.5 years without adverse effects. These promising preclinical results are supported by positive safety and efficacy data from the recently completed Phase 1/2 GALILEO-1 trial in adults with Gaucher disease.
Spur Therapeutics is now preparing for a Phase 3 trial of FLT201. The positive preclinical and early clinical data suggest that FLT201 could represent a significant advancement in Gaucher disease treatment, potentially offering a one-time, long-lasting solution for patients and transforming the standard of care. Success in Phase 3 could pave the way for regulatory approval and market entry, offering a new hope for individuals living with this chronic condition.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
