Syncona
Limited
Spur
provides update on FLT201 and planned Phase III clinical
trial
8 July 2024
Syncona Ltd, a leading life science
investor focused on creating, building and scaling global leaders
in life science, notes that its portfolio company Spur Therapeutics
("Spur") has completed enrolment in the Phase I/II GALILEO-1
clinical trial of FLT201, its adeno-associated virus (AAV) gene
therapy candidate for Gaucher disease. Based on the encouraging
data from this Phase I/II trial, Spur has selected a single
infusion of FLT201 at a low dose of 4.5e11 vg/kg for its Phase III
trial expected to start in CY2025.
Six patients have been dosed in the
GALILEO-1 trial and 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. One patient was
identified to have pre-existing neutralising antibodies to the AAV3
capsid used to deliver FLT201, below the protocol cut-off. This
patient has been excluded from the efficacy analysis. All six
patients are included in the safety analysis, with FLT201
continuing to demonstrate a favourable safety and tolerability
profile.
Efficacy data as of 30 June 2024
data cut-off:
· Lyso-Gb1, an established biomarker of clinical response in
Gaucher disease, was substantially reduced in the four patients
with persistently high lyso-Gb1 levels, despite years of treatment
with currently approved therapies.
o One
patient entered the trial with well-controlled lyso-Gb1, and these
levels have been maintained.
· Maintenance of normal levels, or improvement to normal levels,
of haemoglobin and platelets seen in five patients, which are
well-accepted regulatory endpoints for Gaucher disease.
· Improvement seen in bone marrow burden in five patients, which
shows FLT201 is reaching deeper tissues that are poorly addressed
by currently approved therapies.
· Clinically relevant improvement in patient-reported pain and
fatigue, leading to improved function, in the one patient who
entered the trial with debilitating chronic pain and
fatigue.
· Two
patients developed antibodies to GCase, the enzyme that is
deficient in people with Gaucher disease, but still experienced
benefits from treatment with FLT201.
o Improvements seen in lyso-Gb1 and bone marrow burden from
baseline, as well as maintenance of haemoglobin and platelets in
normal ranges.
o 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 Phase I/II GALILEO-1 trial in the second half of
2024.
Chris Hollowood, CEO of Syncona
Investment Management Limited and Chair of Spur, said:
"We continue to be highly encouraged by the data
coming from the FLT201 programme in Gaucher disease. The safety and
tolerability of the treatment, alongside these compelling efficacy
signals, suggest FLT201 has the potential to play an important role
in the treatment of patients with Gaucher disease. Spur is
expecting to report further data on FLT201 in the second half of
the year, and we look forward to working with the team as they move
towards initiating a Phase III trial in CY2025."
Spur's announcement is copied below
and can be accessed on the company's website at
https://spurtherapeutics.com/.
[ENDS]
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1000
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Spur Therapeutics Completes
Enrollment in Phase 1/2 GALILEO-1 Trial of FLT201 in Gaucher
Disease and Selects Dose for Planned Phase 3
Trial
Single infusion of FLT201 at
low dose of 4.5e11 vg/kg demonstrates compelling safety and
benefit
Expect to initiate Phase 3
registrational trial in 2025
LONDON, July 8, 2024 - 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.
o 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.
o 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 FLT201
FLT201 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 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.
Contact
Naomi
Aoki
naomi.aoki@spurtherapeutics.com
+ 1 617 283 4298