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 21st Annual WORLDSymposium.
“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 WORLDSymposium 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 WORLDSymposium 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:
- Favorable safety and tolerability, with no infusion reactions or dose limiting toxicities. All treatment-related adverse events were mild to moderate in nature.
- Durable reductions in lyso-Gb1, ranging from 33% to 96%, in patients who entered the trial with high levels; stable lyso-Gb1 levels for more than a year after the withdrawal of prior therapy in the one patient who entered the trial with well-controlled levels. Lyso-Gb1 levels in the blood are highly correlated with substrate levels in disease-affected tissues as well as with treatment response.
- Maintenance of normal hemoglobin levels beyond a year after withdrawal of ERT or SRT.
- Sustained improvements or maintenance in platelet counts and spleen and liver volume after withdrawal ERT or SRT.
- Improvements in bone marrow burden (BMB) in four of the five patients, indicating the clearance of substrate and reappearance of healthy marrow, and maintenance of BMB in the fifth patient. Previously reported BMB scores have been updated to correct a reporting error by an outside clinical research organization. 1
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
Post Views: 86
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 21st Annual WORLDSymposium.
“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 WORLDSymposium 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 WORLDSymposium 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:
- Favorable safety and tolerability, with no infusion reactions or dose limiting toxicities. All treatment-related adverse events were mild to moderate in nature.
- Durable reductions in lyso-Gb1, ranging from 33% to 96%, in patients who entered the trial with high levels; stable lyso-Gb1 levels for more than a year after the withdrawal of prior therapy in the one patient who entered the trial with well-controlled levels. Lyso-Gb1 levels in the blood are highly correlated with substrate levels in disease-affected tissues as well as with treatment response.
- Maintenance of normal hemoglobin levels beyond a year after withdrawal of ERT or SRT.
- Sustained improvements or maintenance in platelet counts and spleen and liver volume after withdrawal ERT or SRT.
- Improvements in bone marrow burden (BMB) in four of the five patients, indicating the clearance of substrate and reappearance of healthy marrow, and maintenance of BMB in the fifth patient. Previously reported BMB scores have been updated to correct a reporting error by an outside clinical research organization. 1
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
Post Views: 86
