NEW YORK – Spur Therapeutics on Monday said it is engaging with regulators as it prepares for a registrational Phase III clinical trial of its lead gene therapy candidate for Gaucher disease, a study that the company expects to initiate next year.
London-based Spur, formerly known as Freeline Therapeutics, has selected the dosage for the single infusion of FLT201 that patients will receive in the Phase III clinical trial based on safety and efficacy data from its Phase I/II GALILEO-1 study. Also on Monday, Spur said it has completed enrollment in that Phase I/II clinical trial.
FLT201 is an adeno-associated virus vector-based gene therapy that the company is developing for Gaucher disease, a rare and inherited condition caused by a mutation in the GBA1 gene that leads to a deficiency in the enzyme GCase, which is needed to metabolize certain lipids. FLT201 delivers an engineered longer-acting version of this enzyme called GCase85.
Six patients with Gaucher disease type 1 have been dosed in the first-in-human, dose-finding GALILEO-1 study, and all of them have at least 16 weeks of follow-up data.
As of June 30, patients in the GALILEO-1 study reported improvements in pain and fatigue symptoms, and investigators have observed reductions in glucosylsphingosine, a biomarker associated with Gaucher disease severity. The gene therapy has had a favorable safety and tolerability profile, according to the company.
Spur expects to report additional data from the Phase I/II clinical trial in the second half of 2024.
Spur launched in June as a combination of two gene therapy companies owned by British investment trust Syncona: Freeline and SwanBio Therapeutics. Freeline, which originally developed FLT201, acquired SwanBio as part of the rebrand under the Spur name. Freeline's management team continues to lead the rebranded company.