Spur Therapeutics Completes Enrollment in Phase 1/2 GALILEO-1 Trial of FLT201 in Gaucher Disease and Selects Dose for Planned Phase 3 Trial
July 08 2024 - 7:30AM
Spur Therapeutics, formerly Freeline Therapeutics, today announced
it has completed enrollment in the Phase 1/2 GALILEO-1 clinical
trial of FLT201, its adeno-associated virus (AAV) gene therapy
candidate for Gaucher disease. Based on compelling safety and
efficacy data from the GALILEO-1 trial, Spur has selected a single
infusion of FLT201 at a low dose of 4.5e11 vg/kg for further
development in a Phase 3 trial expected to begin next year.
“We are very pleased with the progress of FLT201,” said Michael
Parini, Chief Executive Officer of Spur Therapeutics. “The data
from the GALILEO-1 trial strengthen our conviction in FLT201’s
potential to set a new standard of care for Gaucher disease and
give us confidence to move forward at the current dose, which has
shown strong signals of efficacy combined with a favorable safety
and tolerability profile. We are preparing to start the Phase 3
trial next year and are committed to bringing forward what we
believe is a first- and best-in-class gene therapy to redefine the
treatment landscape for Gaucher disease.”
Six patients have been dosed in GALILEO-1, a first-in-human,
international, multicenter dose-finding study in adults with
Gaucher disease Type 1. All patients were treated with a single
infusion of FLT201 at a dose of 4.5e11 vg/kg. Two of the six
patients have completed the full nine months of follow-up. The
others have been followed for between 16 and 38 weeks after dosing.
All six patients are included in the safety analysis. Five of the
six patients are included in the efficacy analysis; one patient
with detectable pre-existing neutralizing antibodies (NAbs) to the
AAVS3 capsid below the protocol cut-off has been excluded from the
efficacy analysis.
Data as of the June 30, 2024 data cut-off demonstrate:
- Favorable safety and tolerability.
- Dramatic reductions in glucosylsphingosine (lyso-Gb1) – a
Gaucher-specific biomarker that is one of the best predictors of
disease severity and clinical response – in patients with
persistently high levels despite years of treatment with currently
approved therapies (4/4).
- Maintenance of lyso-Gb1 levels in a patient who entered the
trial with well-controlled lyso-Gb1 (1/1).
- Maintenance of normal levels or improvement to normal levels of
hemoglobin and platelets, which are well-accepted regulatory
endpoints for Gaucher disease (5/5).
- Improvement in bone marrow burden (5/5), which shows FLT201 is
reaching deeper tissues that currently approved therapies poorly
address.
- Clinically relevant improvement in patient-reported pain and
fatigue, leading to improved function, in the one patient who
entered trial with debilitating chronic pain and fatigue.
- Patients experienced these benefits regardless of antibodies to
glucocerebrosidase (GCase) (n=2), the enzyme that is deficient in
people with Gaucher disease, consistent with clinical experience
with enzyme replacement therapy and preclinical data for FLT201.
- Improvements in lyso-Gb1 and bone marrow burden from baseline,
as well as maintenance of hemoglobin and platelets in normal
ranges, continue to be observed after antibodies were
detected.
- In the patient with longest exposure (greater than three months
beyond detection of antibodies), the antibodies appear to have been
transient and improvements in clinical parameters continue to be
observed.
Spur expects to report additional data from the GALILEO-1 trial
in the second half of 2024.
FLT201 has been granted Regenerative Medicine Advanced Therapy
(RMAT) designation by the US Food and Drug Administration, Priority
Medicines (PRIME) designation by the European Medicines Agency and
the Innovative Licensing and Access Pathway (ILAP) by the U.K.
Medicines and Healthcare products Regulatory Agency. Designed to
expedite the drug development process for investigational therapies
intended to treat, modify, reverse or cure a serious or
life-threatening disease, the designations provide opportunities
for enhanced interactions with regulators and expedited review
processes. Spur is actively engaging with regulators as it prepares
for the planned Phase 3 trial of FLT201.
About FLT201FLT201 is an adeno-associated virus
(AAV) gene therapy candidate that is currently being investigated
in the Phase 1/2 GALILEO-1 clinical trial in adults with 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. Preclinical and clinical data for FLT201 have
shown robust and durable expression and a substantial reduction in
the toxic buildup of substrate that results from the enzyme
deficiency. For more information about the GALILEO-1 trial, please
visit clinicaltrials.gov (NCT05324943).
About Gaucher DiseaseGaucher 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 TherapeuticsSpur 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.
ContactNaomi
Aokinaomi.aoki@spurtherapeutics.com+ 1 617 283 4298