REG - Syncona Limited - Spur announced positive data from Phase I/II trial

SynconaAnnouncement

04/02/2025 13:15

For best results when printing this announcement, please click on link below:
https://newsfile.refinitiv.com/getnewsfile/v1/story?guid=urn:newsml:reuters.com:20250204:nRSD8574Va&default-theme=true

RNS Number : 8574V  Syncona Limited  04 February 2025

4 February 2025

 

Syncona Limited

 

Spur announced positive data from Phase I/II trial of FLT201 in Gaucher
disease

 

Syncona Ltd, ("Syncona"), a leading life science investor focused on creating,
building and scaling a portfolio of global leaders in life science, today
notes that its portfolio company, Spur Therapeutics
("Spur") announced positive new data from its Phase I/II GALILEO-1 study of
FLT201, its novel gene therapy candidate, in Gaucher disease at the 21(st)
Annual WORLDSymposium in San Diego, CA, USA.

Six patients have been treated in GALILEO-1 with a single infusion of FLT201
at a low dose of 4.5e11 vg/kg and have been followed for between nine and 17
months after dosing. All six patients are included in the safety analysis; one
patient with detectable pre-existing neutralising antibodies to the AAVS3
capsid was excluded from the efficacy analysis. Prior to the trial all of the
patients had been treated with existing therapies enzyme replacement therapy
(ERT) or substrate reduction therapy (SRT).

These data follow yesterday's announcement that Spur had received positive
feedback from its end-of-Phase II meeting with the US Food and Drug
Administration (FDA), with alignment on the potential to seek accelerated
approval 1  based on reductions in glucosylsphingosine (lyso-Gb1), an
established biomarker of clinical response in Gaucher disease. Full approval
would be based on a primary endpoint of maintenance or improvement in
haemoglobin at one year in the Phase III study. The completion of the pivotal
stage of the Phase III trial in CY2027 is a key value inflection point for
Syncona 2 .

Data as of 6 December 2024 demonstrated:

·    Continued favourable safety and tolerability profile, with no
infusion reactions, dose limiting toxicities or treatment-related severe
adverse events

·      Durable reductions in lyso-Gb1, ranging from 33% to 96% in
patients who entered the trial with high levels

o  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 haemoglobin levels (Phase III primary
endpoint) beyond a year after withdrawal of ERT or SRT

·     Sustained improvements or maintenance in platelet counts and
spleen and liver volume after withdrawal of ERT or SRT

·   Improvements in bone marrow burden (BMB) in four of the five patients,
indicating the clearance of substrate and reappearance of healthy marrow, and
maintenance of BMB in the fifth patient. Previously reported BMB scores have
been updated to correct a reporting error by an outside clinical research
organisation(( 3 ))

 

Chris Hollowood, Chief Executive Officer of Syncona Investment Management
Limited and Chair of Spur Therapeutics, said: "These data further reinforce
our belief that FLT201 has the potential to be a first- and best-in-class gene
therapy for Gaucher disease. Through a single infusion, FLT201 has shown
improvements to symptoms that had been persistent in patients receiving
approved therapies for years and in some cases decades. We look forward to the
initiation of Spur's Phase III FLT201 trial later this year, following the
company's recent successful end-of-Phase II meeting with the US FDA."

Spur's announcement is copied below and can be accessed on the company's
website at https://spurtherapeutics.com/ (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.

 

Forward-looking statements - this announcement contains certain
forward-looking statements with respect to the portfolio of investments of
Syncona Limited. These statements and forecasts involve risk and uncertainty
because they relate to events and depend upon circumstances that may or may
not occur in the future. There are a number of factors that could cause actual
results or developments to differ materially from those expressed or implied
by these forward-looking statements. In particular, many companies in the
Syncona Limited portfolio are conducting scientific research and clinical
trials where the outcome is inherently uncertain and there is significant risk
of negative results or adverse events arising. In addition, many companies in
the Syncona Limited portfolio have yet to commercialise a product and their
ability to do so may be affected by operational, commercial and other risks.

 

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.

 

Notes

About Key Value Inflection Points

A key value inflection point is a material de-risking event for a portfolio
company that has the potential to drive significant NAV growth for Syncona,
for example by increasing the possibility of a realisation event, such as
M&A. These milestones can also enable companies to access significant
capital including through financings and IPOs, which may take place at
valuation uplifts and underpin progression to a subsequent key value
inflection point which has the potential to drive greater value. M&A or
capital access is unlikely to occur immediately following a key value
inflection point.

 

 

 

 

Spur Therapeutics Announces Positive Data from Phase 1/2 GALILEO-1 Trial of
FLT201, Its Gene Therapy Candidate for Gaucher Disease, at WORLDSymposium™

 
 

Oral and poster presentations highlight FLT201's potential to set a new
standard of care

 

LONDON, February 4, 2025 - Spur Therapeutics
(http://www.spurtherapeutics.com/) today announced data from its Phase 1/2
GALILEO-1 trial of FLT201, an adeno-associated virus (AAV) gene therapy
candidate for Gaucher disease type 1, showing rapid and sustained improvements
in glucosylsphingosine (lyso-Gb1), one of the best predictors of clinical
response in Gaucher disease, as well as improvement or maintenance of blood
counts, organ volume and bone marrow burden 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 this week in oral and poster presentations at the 21(st)
Annual WORLDSymposium.

"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. "Data from
our Phase 1/2 study being presented at the WORLDSymposium show that a single
infusion of FLT201 led to improvements in persistent symptoms and disease
involvement in patients who have been on approved therapies for years. These
improvements, together with the favorable safety profile and durability of
responses, highlight FLT201's potential to provide better efficacy and
dramatically reduce the treatment burden for people with Gaucher disease."

"FLT201 is the result of our unwavering focus on developing gene therapies
that change people's lives," said Michael Parini, Spur's Chief Executive
Officer. "We purposefully designed FLT201 to overcome the shortcomings of
currently approved therapies, engineering a more stable version of the GCase
enzyme deficient in people with Gaucher disease and packaging it in a capsid
that is highly efficient at transducing cells to produce the enzyme. We are
seeing that work translate into benefits beyond what current therapies provide
at a low dose that we believe could offer an important safety advantage over
other gene therapies in development. We are moving quickly to initiate a Phase
3 study of FLT201."

Compelling Safety and Efficacy Data for FLT201

Building on previously reported data, the oral and poster presentations at the
WORLDSymposium demonstrate the durable benefits and longer-term safety profile
of FLT201. In addition to updated safety and tolerability data, the
presentations include recent assessments of biomarker and efficacy endpoints
from the GALILEO-1 study, a first-in-human, international, multicenter
dose-finding study in adults with Gaucher disease type 1, and the GALILEO-2
long-term follow up study.

Six patients were treated in GALILEO-1 with a single infusion of FLT201 at a
low dose of 4.5e11 vg/kg. Patients have been followed for between nine and 17
months after dosing and have all rolled over into GALILEO-2. All six patients
are included in the safety analysis; one patient with detectable pre-existing
neutralizing antibodies to the AAVS3 capsid was excluded from the efficacy
analysis. Prior to the trial, patients had been on a stable dose of either
enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for
between four and 24 years. All patients taken off ERT or SRT remain off those
therapies.

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. Lyso-Gb1 levels in the blood are highly
correlated with substrate levels in disease-affected tissues as well as with
treatment response.

·    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.

·    Improvements in bone marrow burden (BMB) in four of the five
patients, indicating the clearance of substrate and reappearance of healthy
marrow, and maintenance of BMB in the fifth patient. Previously reported BMB
scores have been updated to correct a reporting error by an outside clinical
research organization.(( 4 ))

As announced yesterday, Spur has gained alignment with the U.S. Food and Drug
Administration on the design of a single-arm Phase 3 study to support
potential accelerated approval of FLT201 based on reductions in lyso-Gb1 and
full approval based on improvement or maintenance of hemoglobin levels.
Secondary endpoints will include platelet counts and organ volume, with
exploratory endpoints including bone health assessments and patient-reported
outcomes. Spur expects to dose the first patient in the Phase 3 study in the
second half of 2025.

 

About FLT201

FLT201 is an adeno-associated virus (AAV) gene therapy candidate in clinical
development as a potential one-time treatment for 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. Data from the completed Phase 1/2 GALILEO-1 clinical trial of
FLT201 have shown improvements across a number of key biomarkers and clinical
assessments, including substantial reductions in the toxic buildup of
substrate that results from the enzyme deficiency, as well as a favorable
safety and efficacy profile. A Phase 3 trial for FLT201 is expected to start
in the second half of 2025.

 

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
(http://www.spurtherapeutics.com) or connect with Spur on LinkedIn
(https://www.linkedin.com/company/spurtherapeutics/) and X
(https://x.com/SpurTherapeutx) .

Investor Contact

Naomi Aoki

naomi.aoki@spurtherapeutics.com (mailto:naomi.aoki@spurtherapeutics.com)
 

+ 1 617 283 4298

Media Contact

Carolyn Noyes

carolyn.noyes@spurtherapeutics.com (mailto:carolyn.noyes@spurtherapeutics.com)

+ 1 617 780 2182

 

 

 1  The FDA's Accelerated Approval Program allows for earlier approval of
drugs that treat serious conditions, and fill an unmet medical need based on a
surrogate endpoint. The use of a surrogate endpoint can considerably shorten
the time required prior to receiving FDA approval.

 2  The definition of a key value inflection point can be found in the notes
section.

 3  For updated scores for each patient, please refer to the overview deck
available on the News & Data section of www.spurtherapeutics.com
(http://www.spurtherapeutics.com) .

 4  For updated scores for each patient, please refer to the overview deck
available on the News & Data section of www.spurtherapeutics.com
(http://www.spurtherapeutics.com) .

This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact
rns@lseg.com (mailto:rns@lseg.com)
 or visit
www.rns.com (http://www.rns.com/)
.

RNS may use your IP address to confirm compliance with the terms and conditions, to analyse how you engage with the information contained in this communication, and to share such analysis on an anonymised basis with others as part of our commercial services. For further information about how RNS and the London Stock Exchange use the personal data you provide us, please see our
Privacy Policy (https://www.lseg.com/privacy-and-cookie-policy)
.   END  PFUBZLFBELLFBBQ