FLT-201 / Spur Therapeutics - LARVOL DELTA

Durability of FLT201: an Investigational Gene Therapy for Gaucher Disease Type 1 Encoding an Engineered Variant of the GCase Enzyme (ASGCT 2025) - "The clinical relevance of this data is discussed. Disease Focus of Abstract:Storage/Lysosomal Disorders"

Gene therapy • Gaucher Disease • Gene Therapies • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Type 1 Gaucher Disease • GBA1

GALILEO-1: A Gene Therapy Study in Patients With Gaucher Disease Type 1 (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Spur Therapeutics | Trial completion date: Jan 2025 ➔ Jul 2025

Trial completion date • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease Type 1. (PubMed, Mol Ther) - "Preclinical data indicates that FLT201 could offer a durable treatment for Gaucher disease Type 1, addressing unmet needs related to substrate accumulation in tissues poorly treated by current enzyme replacement therapy. The improved stability of the engineered GCase85 variant is predicted to be crucial for FLT201's therapeutic effectiveness."

Journal • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease • GBA1

Spur Therapeutics Announces Oral and Poster Presentations Highlighting Data from Clinical and Preclinical Programs at Upcoming ASGCT Annual Meeting (GlobeNewswire) - "Spur Therapeutics...announced that it will share new data from its gene therapy programs in Gaucher disease, GBA1 Parkinson’s disease and adrenomyeloneuropathy in oral and poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting....The oral presentation will showcase clinical and preclinical durability data on FLT201, Spur’s adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, which is expected to begin a Phase 3 trial this year."

Clinical data • New P3 trial • Preclinical • Gaucher Disease • Parkinson's Disease

Spur Therapeutics Announces Positive Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Gene Therapy Candidate for Gaucher Disease, at WORLDSymposium (GlobeNewswire) - P1 | N=18 | GALILEO-1 (NCT05324943) | Sponsor: Spur Therapeutics | "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; 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."

P1/2 data • Gaucher Disease

Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease (GlobeNewswire) - "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...Spur expects to dose the first patient in a Phase 3 trial of FLT201 in the second half of 2025."

FDA event • New P3 trial • Type 1 Gaucher Disease

Spur Therapeutics Announces Platform and Poster Presentations Highlighting New Clinical Data for FLT201 in Gaucher Disease at Upcoming WORLDSymposium (GlobeNewswire) - "Spur Therapeutics today announced that it will share updated clinical data from its Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1, in platform and poster presentations at the 21st Annual WORLDSymposiumTM being held February 3-7, 2025, in San Diego."

P1/2 data • Gaucher Disease

Results from GALILEO-1, a first-in-human clinical trial of FLT201 gene therapy in patients with Gaucher disease Type 1 (ESGCT 2024) - No abstract available

Clinical • Gene therapy • P1 data • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) (clinicaltrials.gov) - P1/2 | N=75 | Recruiting | Sponsor: Freeline Therapeutics

New P1/2 trial • Viral vector • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

GALILEO-1: A Gene Therapy Study in Patients With Gaucher Disease Type 1 (clinicaltrials.gov) - P1 | N=18 | Active, not recruiting | Sponsor: Freeline Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Results from GALILEO‐1, a first‐in‐human clinical trial of FLT201 gene therapy in patients with Gaucher disease Type 1 (ESGCT 2023) - "Tests at screening showed GCase activity (plasma) of 0.07 micromol/L/h, GCase activity (dried blood spot) of 0.3 micromol/L/h, and Lyso-Gb1 (dried blood spot) of 102.85 ng/mL. Study data from participants enrolled into the first dose cohort, including patient demographics, safety and GCase levels, will be presented."

Clinical • Gene therapy • P1 data • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Results from GALILEO-1, A First-in-Human Clinical Trial of FLT01, an AAV-Gene Therapy, in Adults with Gaucher Disease Type 1 (ASGCT 2024) - "FLT201 is the furthest advanced investigational AAV-gene therapy for Gaucher disease Type 1 currently under development. Emerging data from the participants dosed in the GALILEO-1 study so far demonstrates an acceptable safety profile of FLT201, with robust, durable expression of GCase alongside evidence of lyso-Gb1 clearance. A description of FLT201, a potentially transformative therapy for people with Gaucher disease type 1, and an overview of the GALILEO-1 study including data from all participants dosed will be presented."

Clinical • Gene therapy • Late-breaking abstract • P1 data • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease • FLT1

GALILEO-1: A Gene Therapy Study in Patients With Gaucher Disease Type 1 (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Freeline Therapeutics | Phase classification: P1/2 ➔ P1

Gene therapy • Phase classification • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

In silico immunogenicity assessment of FLT201: an investigational gene therapy for Gaucher disease Type 1 encoding an engineered variant of the GCase enzyme (ESGCT 2022) - No abstract available

Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

GALILEO-1: A Gene Therapy Study in Patients With Gaucher Disease Type 1 (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Freeline Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

GALILEO-1: A Gene Therapy Study in Patients With Gaucher Disease Type 1 (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: Freeline Therapeutics

New P1/2 trial • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

[VIRTUAL] FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1 (ASGCT 2021) - "GCasevar85 catalytic parameters (KM) are comparable to wild-type GCase and ERTs used to treat GD1.The potency and functionality of GCasevar85 when secreted by the liver following treatment with AAV8 pseudotyped FLT201 [AAV8-FLT201] were evaluated in GCase-deficient mice (Gba9v/null) and compared to liver-secreted wild-type GCase following treatment with AAV8 pseudotyped FLT200 [AAV8-FLT200] and velaglucerase alfa (an ERT used to treat GD). Based on the data collected in Gba9v/null mice and clinical data for our AAVS3 platform, the use of FLT201 compared to FLT200 should allow reduction of the predicted efficacious clinical dose by at least 10-fold.Pre-clinical data show that AAV8 pseudotyped FLT201 genome is well tolerated and results in a significant increase in potency in GCase deficient mice, including hard-to-reach tissues such as bone marrow and lung. Therefore, FLT201 has the potential to address areas of current unmet medical need in GD1 patients."

Gaucher Disease • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease

Form 424B4 Freeline Therapeutics (Streetinsider.com) - Preclinical results from the 13-week GLP-compliant toxicology studies showed that administration of FLT190 was effective in producing significantly increased GLA activity...In July 2019, we began recruiting patients for our MARVEL-1 clinical trial, which is a Phase 1/2 dose-finding clinical trial to evaluate the safety and efficacy of FLT190 for the treatment of Fabry disease, with our first patient dosed in August 2019....But continue to recruit patients for our ECLIPSE study, through which we screen patients for eligibility and possible participation in our Phase 1/2 dose-finding clinical trial....We are developing FLT201 for the treatment of type 1 Gaucher disease...We are the only company to date that has announced a program for the development of an AAV gene therapy for the treatment of type 1 Gaucher disease. We plan to file the IND and initiate the Phase 1/2 clinical trial for this program in 2021."

New P1/2 trial • Preclinical • Trial status • Fabry Disease • Gaucher Disease • Genetic Disorders

IPO Launch: Freeline Therapeutics Proposes $125 Million IPO (The Street) - "According to an amended registration statement, Freeline Therapeutics (FRLN) intends to raise $125 million from the selling of ADSs that represent underlying common stock at an IPO. The company is developing a pipeline of candidates for the treatment of hemophilia, Gaucher disease, and Fabry disease....FRLN seeks an above-average size of IPO transaction to advance its hemophilia, Gaucher disease and Fabry disease care pipeline."

Financing • Fabry Disease • Gaucher Disease • Genetic Disorders • Hemophilia

Freeline Therapeutics and Checkmate Pharma Make Opening IPO Moves (Xconomy) - "The biotech IPO queue has added two more companies. Freeline Therapeutics and Checkmate Pharmaceuticals have each filed the regulatory paperwork to offer shares to the public....Freeline says in its prospectus that it expects to report additional data from the FLT180a study in the second half of this year. The company plans to advance the gene therapy to a pivotal study in 2021. The other programs in its pipeline include FLT190, a gene therapy for the rare enzyme deficiency Fabry disease that is in Phase 1/2 testing. Two other programs, FLT201 for Gaucher disease, another rare disorder caused by an enzyme deficiency, and FLT210 for hemophilia A, are in preclinical development."

Financing • Fabry Disease • Gaucher Disease • Genetic Disorders • Hemophilia • Type 1 Gaucher Disease