Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval

On track to dose first patient in Phase 3 trial in second half of 2025

/EIN News/ -- LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced positive feedback from its end-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), supporting its planned Phase 3 trial for FLT201, an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1. The feedback included alignment on the potential to seek accelerated approval based on reductions in glucosylsphingosine (lyso-Gb1) under the Accelerated Approval Program. Lyso-Gb1 is one of the best predictors of clinical response in Gaucher disease, and FLT201 showed rapid and sustained reductions in lyso-Gb1 in its Phase 1/2 GALILEO-1 trial.

Full approval would be based on a primary endpoint of maintenance or improvement in hemoglobin at one year in the Phase 3 study. Hemoglobin is a well-established regulatory endpoint in Gaucher disease, and data from GALILEO-1 has shown maintenance of normal hemoglobin levels in patients treated with a single infusion of FLT201 beyond a year after withdrawal of their prior treatments. Spur expects to dose the first patient in a Phase 3 trial of FLT201 in the second half of 2025.

“We are very pleased with the outcome of our end-of-Phase 2 discussion with the FDA, providing us with a clear path forward,” said Michael Parini, Spur’s Chief Executive Officer. “We believe that FLT201 has potential to be a first- and best-in-class gene therapy that sets a new standard of care for Gaucher disease. We are moving expeditiously to get the Phase 3 trial underway with the goal of providing people with Gaucher a potentially transformative one-time therapy that eliminates or alleviates ongoing symptoms while dramatically reducing the treatment burden.”

The data from GALILEO-1 support the advancement of FLT201 into a Phase 3 trial and formed the basis of the EOP2 feedback from the FDA. Based on alignment with the FDA, Spur expects the Phase 3 trial will be a single-arm study in adults with Gaucher disease type 1, similar in design to GALILEO-1. Prior to entering the trial, patients will have been on a stable dose of either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years and then taken off those therapies at a set timepoint after receiving a single infusion of FLT201 at a low dose of 4.5e11 vg/kg. The FDA is aligned with the selection of this dose, which showed a compelling safety and efficacy profile in GALILEO-1.

Patients will serve as their own comparators and will be evaluated based on improvements or maintenance of key endpoints relative to their baseline assessments. FDA provided positive feedback on the potential for accelerated approval based on improvements in lyso-Gb1 levels six months after dosing and full approval based on improvement or maintenance of hemoglobin levels a year after dosing. Other key secondary and exploratory endpoints will include platelet counts, liver and spleen volume, assessments of bone health, and patient-reported outcomes on pain, fatigue, and physical and mental well-being. Spur expects to enroll approximately 40-45 patients in the Phase 3 study.

Spur is actively preparing for the Phase 3 study and expects to complete site selection this month, with more than 45 sites across the United States, Canada, the United Kingdom, Europe, Israel and Latin America.

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.

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