23 October 2024
Syncona
Limited
Spur presents positive data
update from Phase I/II trial of FLT201 in Gaucher
disease
Syncona Ltd, ("Syncona" or the
"Company") a leading life science investor focused on creating, building and scaling global leaders in life
science, today notes that its portfolio
company, Spur Therapeutics ("Spur") presented positive new data from its
Phase I/II GALILEO-1 study of FLT201, its novel gene therapy
candidate, in Gaucher disease at the European Society of Gene and
Cell Therapy (ESGCT) 31st Annual Congress in Rome,
Italy.
Six patients were treated with a
single infusion of FLT201 at a dose of 4.5e11 vg/kg and have been
followed for between seven and 15 months after dosing. The efficacy
results include five of the six patients, with one patient excluded
after being found to have detectable pre-existing neutralising
antibodies to the AAVS3 capsid used to deliver FLT201. All six
patients are included in the safety analysis, with FLT201
continuing to demonstrate a favourable safety and tolerability
profile. The publishing of this data is a key value inflection
point for Spur, reinforcing the potential of FLT201 as a therapy
for Gaucher disease patients, whilst underlining the strength of
Spur's technology.
Data as of 27 September 2024
demonstrated:
· A
favourable safety and tolerability profile, with no infusion
reactions, dose limiting toxicities or severe adverse
events.
· Durable reductions, ranging from 42% to 96%, in lyso-Gb1
levels, an established biomarker of clinical response in Gaucher
disease, in patients with persistently high lyso-Gb1 levels,
despite years on prior treatment with enzyme replacement therapy
(ERT) or substrate reduction therapy (SRT).
o Low
lyso-Gb1 levels maintained for more than a year to date after
withdrawal of prior therapy in one patient who entered the trial
with well-controlled lyso-Gb1 levels.
· Substantial improvements in bone marrow burden seen in all
five patients, suggesting FLT201 is penetrating deeper tissues
which are poorly addressed by existing treatments.
· Improvement or maintenance of spleen and liver
volume.
· Maintenance of haemoglobin levels, an established endpoint for
Gaucher disease clinical trials, observed up to a year post
withdrawal of treatment with ERT or SRT.
o Improvement or maintenance of platelet counts also seen post
withdrawal of treatment with ERT or SRT.
Chris Hollowood, Chief Executive Officer of Syncona Investment
Management Limited and Chair of Spur
Therapeutics, said: "The data
presented at ESGCT further demonstrates FLT201's potential to
transform the lives of patients with Gaucher disease. The maturing
data that we are seeing from the GALILEO-1
trial underlines the durability profile of FLT201 and reinforces
the long-lasting potential of this therapy beyond current standard
of care for the thousands of patients with the disease.
Furthermore, the data de-risks Spur's technology and supports the
advancement of the company's pre-clinical pipeline into more
prevalent disorders, including Parkinson's disease. The delivery of
this data at ESGCT is a key value inflection point for Spur, and we
continue to support the company as it prepares to initiate 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]
Enquiries
Syncona Ltd
Natalie Garland-Collins / Fergus
Witt
Tel: +44 (0) 20 3981 7912
FTI Consulting
Ben Atwell / Tim Stamper
Tel: +44 (0) 20 3727 1000
About Syncona
Syncona's purpose is to invest to
extend and enhance human life. We do this by creating, building and
scaling companies to deliver transformational treatments to
patients in areas of high unmet need.
We aim to build and maintain a
diversified portfolio of 20-25 globally leading life science
businesses, across development stage, modality and therapeutic
area, for the benefit of all our stakeholders. We focus on
developing treatments that deliver patient impact by working in
close partnership with world-class academic founders and
experienced management teams. Our balance sheet underpins our
strategy, enabling us to take a long-term view as we look to
improve the lives of patients with no or poor treatment options,
build sustainable life science companies and deliver strong
risk-adjusted returns to shareholders.
Syncona Limited seeks to achieve returns over the long term.
Investors should seek to ensure they understand the risks and
opportunities of an investment in Syncona Limited, including the
information in our published documentation, before
investing.
Spur Therapeutics Showcases
Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its
Novel Gene Therapy Candidate for Gaucher Disease, in Oral
Presentation at ESGCT 31st Annual
Congress
Data highlight potential of
FLT201 to set new standard of care for Gaucher
disease
Dramatic and durable
reductions observed in lyso-Gb1, an established biomarker of
clinical response, in patients with persistently high levels
despite years of prior treatment on currently approved
therapies
Substantial improvements in
bone marrow burden demonstrate FLT201 penetrating deeper tissues
that currently approved therapies poorly address
Favorable safety and
tolerability continue to be seen in all patients
dosed
On track to initiate Phase 3
trial of FLT201 in 2025
LONDON, October 23, 2024 - Spur
Therapeutics today announced
positive new data from its ongoing Phase 1/2 GALILEO-1 trial of
FLT201, an adeno-associated virus (AAV) gene therapy candidate for
Gaucher disease type 1,
showing durable reductions in glucosylsphingosine (lyso-Gb1), one
of the best predictors of clinical response in Gaucher
disease, and bone marrow burden, as
well as improvements or maintenance of blood counts and organ volume 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 in an oral
presentation at the European Society of Gene and Cell Therapy
(ESGCT) 31st Annual Congress taking place in
Rome, Italy.
"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. "Consistent with
what is seen in real-world practice, patients entered our trial
with different unmet needs. All of them had bone disease, which
current therapies poorly address. Most had high levels of harmful
substrate accumulation. Some also had an enlarged spleen, low
platelets, pain or fatigue. What is exciting is that after a single
infusion of FLT201, patients improved in areas their disease was
poorly controlled and remained well-controlled in areas their
disease was controlled. These data underscore FLT201's potential to
set a new standard of care for Gaucher disease type 1."
Positive New Clinical Data for FLT201
Today's oral presentation at ESGCT
during the session titled "AAV Vectors as Tools in Gene Therapy of
Rare Diseases - Recent Development to Improve Efficacy and Safety"
will include updated data on safety and tolerability, as well as
biomarker and efficacy endpoints from the ongoing Phase 1/2
GALILEO-1 study, a first-in-human, international, multicenter
dose-finding study in adults with Gaucher disease Type 1. Dosing
has been completed in the trial. A total of six patients were
treated with a single infusion of FLT201 at a dose of 4.5e11 vg/kg
and have been followed for between seven
and 15 months after
dosing. All six patients are included in the safety 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.
The data as
of September 27, 2024 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,
ranging from 42% to 96%, in lyso-Gb1 levels in patients who entered
the trial with persistently high levels despite years of treatment
with enzyme replacement therapy (ERT) or substrate reduction
therapy (SRT). Low lyso-Gb1 levels maintained for more than a year
as of the cut-off date 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 and one of the best predictors
of clinical response in Gaucher disease.
·
Substantial improvements in bone marrow burden
(BMB) seen in all five patients, including those who entered the
trial with severe bone involvement. Notably, BMB is correlated with
increased fractures, necrosis, bone pain and joint
replacements.
·
Improvement or maintenance of spleen and liver
volume.
·
Maintenance of hemoglobin levels, an established
endpoint for Gaucher disease clinical trials, was observed beyond a
year post withdrawal of treatment with ERT or SRT. Improvement or
maintenance of platelet counts was also seen post withdrawal of
treatment with ERT or SRT.
"FLT201 exemplifies our focus on
developing the next generation of gene therapies, optimizing each
component of our gene therapy candidates with the aim of changing
the course of disease and changing people's lives," said Michael
Parini, Spur's Chief Executive Officer. "Our more stable GCase85
enzyme, combined with our potent and proprietary capsid, is leading
to improvements in bone, pain and fatigue, all of which are key
areas of ongoing need in Gaucher disease. These data highlight
FLT201's potential to provide better outcomes than current standard
of care while dramatically reducing the treatment burden for
patients, and we are actively engaging with regulators to advance
FLT201 into a Phase 3 trial."
Spur expects to report additional
data from the Phase 1/2 GALILEO-1 trial in the first half of 2025
and initiate a Phase 3 trial for FLT201 in adults with Gaucher
disease type 1 in 2025.
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.
Investor Contact
Naomi Aoki
naomi.aoki@spurtherapeutics.com
+ 1 617 283 4298
Media Contact
Carolyn Noyes
carolyn.noyes@spurtherapeutics.com
+ 1 617 780 2182