FLT-201

May 27, 2025 -- Spur Therapeutics today announced the publication of preclinical proof-of-concept data for FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease in Molecular Therapy, the flagship journal of the American Society of Gene & Cell Therapy. These data supported the advancement of FLT201 into clinical development and highlight its potential to improve upon the current standard of care. “FLT201 was designed to address the shortcomings of existing therapies for Gaucher disease,” said Pamela Foulds, M.D., Chief Medical Officer at Spur. “Many people with Gaucher disease continue to experience symptoms, including pain, fatigue and bone disease, despite lifelong, biweekly infusions of enzyme replacement therapy, or ERT. By delivering a more stable, rationally engineered version of the enzyme deficient in people with Gaucher disease, FLT201 has the potential provide better outcomes for patients with a single dose, dramatically reducing the burden of disease and the burden of treatment.” The publication, titled “FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease Type 1,” highlights preclinical data showing that FLT201 outperformed both ERT and gene therapy delivering wildtype glucocerebrosidase (GCase). Enhanced stability: GCase85, the rationally engineered version of GCase developed by Spur, demonstrated 6-fold longer half-life in serum and more than a 20-fold longer half-life at lysosomal pH than wildtype GCase in in vitro studies. Greater uptake in tissues: In a mouse model of Gaucher disease, this enhanced stability led to substantially higher GCase activity compared to ERT and wildtype GCase gene therapy. Notably, enzyme activity was observed in difficult-to-reach tissues, including bone and lung, which are poorly addressed by ERT. Superior substrate reduction: FLT201 also resulted in greater reductions of disease-causing substrates in affected tissues in this mouse model compared to ERT and wildtype GCase gene therapy. Durable expression. A single infusion of FLT201 resulted in robust increase in plasma GCase and sustained plasma GCase activity in non-human primates (NHPs), with expression maintained out to 3.5 years and no treatment-related adverse effects observed. Data from the recently completed Phase 1/2 GALILEO-1 trial have shown a compelling safety and efficacy profile in adults with Gaucher disease following administration of a single low dose of FLT201. Spur is now preparing to advance FLT201 into a Phase 3 trial. 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, a potential first-in-class gene therapy candidate for adrenomyeloneuropathy and a preclinical gene therapy candidate for Parkinson’s disease, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of dementia and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine. The content above comes from the network. if any infringement, please contact us to modify.

Oral and poster presentations highlight FLT201’s potential to set a new standard of care LONDON, UK I February 04, 2025 I Spur Therapeutics today announced data from its Phase 1/2 GALILEO-1 trial of FLT201, an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1, showing rapid and sustained improvements in glucosylsphingosine (lyso-Gb1), one of the best predictors of clinical response in Gaucher disease, as well as improvement or maintenance of blood counts, organ volume and bone marrow burden in patients treated with a single infusion of FLT201. FLT201 continues to demonstrate a favorable safety and tolerability profile in all patients treated in the study. These data are being showcased this week in oral and poster presentations at the 21 st Annual WORLD Symposium . “Gaucher disease is a debilitating chronic disorder, and despite treatment with currently approved therapies, many patients continue to have serious symptoms,” said Pamela Foulds, M.D., Spur’s Chief Medical Officer. “Data from our Phase 1/2 study being presented at the WORLD Symposium show that a single infusion of FLT201 led to improvements in persistent symptoms and disease involvement in patients who have been on approved therapies for years. These improvements, together with the favorable safety profile and durability of responses, highlight FLT201’s potential to provide better efficacy and dramatically reduce the treatment burden for people with Gaucher disease.” “FLT201 is the result of our unwavering focus on developing gene therapies that change people’s lives,” said Michael Parini, Spur’s Chief Executive Officer. “We purposefully designed FLT201 to overcome the shortcomings of currently approved therapies, engineering a more stable version of the GCase enzyme deficient in people with Gaucher disease and packaging it in a capsid that is highly efficient at transducing cells to produce the enzyme. We are seeing that work translate into benefits beyond what current therapies provide at a low dose that we believe could offer an important safety advantage over other gene therapies in development. We are moving quickly to initiate a Phase 3 study of FLT201.” Compelling Safety and Efficacy Data for FLT201 Building on previously reported data, the oral and poster presentations at the WORLD Symposium demonstrate the durable benefits and longer-term safety profile of FLT201. In addition to updated safety and tolerability data, the presentations include recent assessments of biomarker and efficacy endpoints from the GALILEO-1 study, a first-in-human, international, multicenter dose-finding study in adults with Gaucher disease type 1, and the GALILEO-2 long-term follow up study. Six patients were treated in GALILEO-1 with a single infusion of FLT201 at a low dose of 4.5e11 vg/kg. Patients have been followed for between nine and 17 months after dosing and have all rolled over into GALILEO-2. All six patients are included in the safety analysis; one patient with detectable pre-existing neutralizing antibodies to the AAVS3 capsid was excluded from the efficacy analysis. Prior to the trial, patients had been on a stable dose of either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for between four and 24 years. All patients taken off ERT or SRT remain off those therapies. The data as of December 6, 2024 cut-off date demonstrated: As announced yesterday, Spur has gained alignment with the U.S. Food and Drug Administration on the design of a single-arm Phase 3 study to support potential accelerated approval of FLT201 based on reductions in lyso-Gb1 and full approval based on improvement or maintenance of hemoglobin levels. Secondary endpoints will include platelet counts and organ volume, with exploratory endpoints including bone health assessments and patient-reported outcomes. Spur expects to dose the first patient in the Phase 3 study in the second half of 2025. About FLT201 FLT201 is an adeno-associated virus (AAV) gene therapy candidate in clinical development as a potential one-time treatment for Gaucher disease. FLT201 leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the enzyme deficient in people with Gaucher disease, with the goal of stopping disease progression, reducing or eliminating symptoms, and allowing patients to come off current lifelong treatments. Data from the completed Phase 1/2 GALILEO-1 clinical trial of FLT201 have shown improvements across a number of key biomarkers and clinical assessments, including substantial reductions in the toxic buildup of substrate that results from the enzyme deficiency, as well as a favorable safety and efficacy profile. A Phase 3 trial for FLT201 is expected to start in the second half of 2025. About Gaucher Disease Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells, which then accumulate in tissues and organs throughout the body, causing inflammation and dysfunction. Despite treatment with currently approved therapies, many people with Gaucher disease continue to experience debilitating symptoms, including enlarged organs, fatigue, bone pain and reduced lung function. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, France, Germany, Spain, Italy and Israel. 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 a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular 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 and X . 1 For updated scores for each patient, please refer to the overview deck available on the News & Data section of www.spurtherapeutics.com . SOURCE: Spur Therapeutics