Spur Therapeutics Announces First Patient Dosed in GALILEO-3 Pivotal Clinical Trial of FLT201 for the Treatment of Gaucher Disease Type 1
Completion of enrollment anticipated within 12 months
GALILEO-3 is intended to support the registration of FLT201 in the adult GD1 population
LONDON, July 07, 2026 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced that the first patient has been dosed in GALILEO-3, its Phase 3 pivotal clinical trial evaluating avigbagene parvec (FLT201), an investigational gene therapy for Gaucher disease type 1 (GD1). The trial will assess the safety and efficacy of FLT201 in adults currently on stable standard-of-care therapies. Spur has aligned with the U.S. Food and Drug Administration (FDA) on a single-arm trial design intended to support regulatory approval.
“Dosing the first patient in our Phase 3 trial is an important step forward in our efforts to redefine what is possible for individuals living with Gaucher disease,” said Michael Parini, Chief Executive Officer. “Building on the favorable safety and efficacy, including maintenance or improvement in hemoglobin, platelet count, bone disease and organ volume seen in our GALILEO-1 and -2 trials, we are now focused on accelerating this potential best-in-class, one-time treatment toward registration, and the eager response from the community to participate in this trial reflects the real potential for FLT201 to address persistent unmet needs.”
GALILEO-3 is an open-label, non-randomized, global, multicenter, Phase 3 study that will enroll approximately 45 adults with GD1 who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years. Participants will receive a single intravenous infusion of FLT201 (4.5 x 1011 vg/kg) alongside a prophylactic immunosuppressive regimen. The primary endpoint is the proportion of participants maintaining stable hemoglobin concentration at Week 52. Secondary endpoints include safety and tolerability, along with stability of platelet counts, spleen and liver volumes, biomarker changes, bone marrow burden and mineral density changes, and patient-reported quality-of-life outcomes. Spur expects to complete enrollment in the study within 12 months. The study was designed based on learnings from Spur’s GALILEO-1 and GALILEO-2 trials, which demonstrated continuous GCase85 expression, rapid reductions in toxic biomarkers, and no dose-limiting toxicities. More information on the trial design and sites can be found at www.clinicaltrials.gov/study/NCT07223944.
“The Gaucher community is eager for further innovation that can move the standard of living care beyond chronic ERT or SRT, which not only require lifelong infusions, but also leave patients with some features of Gaucher disease that are not fully addressed by currently available treatments,” said Dr. Priya Kishnani, Professor of Pediatrics at Duke University and an investigator in GALILEO-3. “The GALILEO-3 trial will help further evaluate the safety and efficacy of FLT201 and its potential to improve outcomes for the patients we treat.”
Gaucher disease is a rare genetic disorder caused by mutations in the GBA1 gene, leading to toxic cellular accumulation and resulting in severe symptoms such as organ enlargement, bone disease, anemia, and debilitating fatigue. While ERT and SRT are the current standards of care, significant unmet medical needs remain. FLT201 is designed to overcome the limitations of these chronic treatments. Utilizing Spur’s proprietary, liver-tropic capsid and an engineered beta-glucocerebrosidase (GCase85), FLT201 delivers continuous, sustained enzyme exposure beyond what is achievable with ERT. This extended stability allows for a low vector genome dose, which is key to a favorable safety profile. FLT201 has both Orphan Drug and RMAT (regenerative medicine advanced therapy) designations from the FDA.
About Spur Therapeutics
Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and has a preclinical gene therapy candidate for Parkinson’s disease. Expanding our impact, and advancing the practice of genetic medicine.
Toward life-changing therapies, and brighter futures. Toward More™
For more information, visit www.spurtherapeutics.com or connect with Spur on LinkedIn.
Contact
media@spurtherapeutics.com
Legal Disclaimer:
EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
