REG - Syncona Limited - Spur presents new data

SynconaAnnouncement

Thu 11:30

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RNS Number : 8169I  Syncona Limited  15 May 2025

15 May 2025

 

Syncona Limited

 

Spur Therapeutics announces new data at ASGCT

 

Syncona Ltd, ("Syncona" or the "Company") a leading life science investor
focused on creating, building and scaling global leaders in life science,
notes that its portfolio company Spur Therapeutics ("Spur") announced new data
from its gene therapy programmes in Gaucher disease, GBA1 Parkinson's disease
and adrenomyeloneuropathy (AMN). These data were presented at the American
Society of Gene and Cell Therapy (ASGCT) 28(th) Annual Meeting in New Orleans,
US.

 

Key highlights from the presentations include:

 

·    Updated clinical data from the Phase I/II GALILEO-1 trial and a
longer-term follow-up study of FLT201 in Gaucher disease demonstrated that the
adeno-associated virus (AAV) gene therapy candidate continues to deliver
durable enzyme expression and sustained clinical benefit

·     Updated pre-clinical data for FLT201 demonstrated robust and
sustained enzyme expression, maintained out past 3.5 years

·  Pre-clinical data supported best-in-class potential for SPR301 for a
form of Parkinson's disease characterised by mutations in GBA1 (the same gene
implicated in Gaucher disease); data demonstrated that the AAV gene therapy
candidate enhanced enzyme expression in key brain regions

·    A safety update from the Phase I/II PROPEL clinical trial of SBT101
demonstrated that the gene therapy candidate for AMN continues to be generally
well-tolerated

 

Chris Hollowood, Chief Executive Officer of Syncona Investment Management
Limited and Chair of Spur Therapeutics, said: "The data presented by Spur at
ASGCT is very encouraging, for their lead programme in Gaucher disease but
also for the broader pipeline. Targeting the same genetic pathway, SPR301 is
demonstrating best-in-class potential in GBA1 Parkinson's disease, whilst
FLT201 is demonstrating durable clinical benefit in Gaucher disease. We look
forward to the company initiating their Phase III trial of FLT201."

 

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

 

 

 ENDS 

 

Enquiries

 

Syncona Ltd

 

Annabel Clark / Tim Stamper

Tel: +44 (0) 20 3981 7912

 

FTI Consulting

 

Ben Atwell / Natalie Garland-Collins

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 Presents Positive New Clinical and Preclinical Data on Its
Gene Therapy Programs at ASGCT 28(th) Annual Meeting

Oral presentation highlights data showing FLT201 results in durable enzyme
expression and sustained clinical benefit in Gaucher disease

 Preclinical data for SPR301 support best-in-class potential for GBA1
Parkinson's disease

SBT101 continues to be well tolerated in adrenomyeloneuropathy patients

LONDON, May 15, 2025 -  Spur Therapeutics
(https://www.globenewswire.com/Tracker?data=ObSzm6mvoqOBzwobIlULvI8pPnL03PqsKosFpUVKv2eGnEB8qnSEM3v_2lgFUfAztdFGTbkSjkmYA2KglkAFwrpqMwkyLN_EsuTL_2g7EM8=)
 today announced positive new data from its gene therapy programs in Gaucher
disease, GBA1 Parkinson's disease and adrenomyeloneuropathy (AMN). These data
are being presented in oral and poster presentations at the American Society
of Gene and Cell Therapy (ASGCT) 28(th )Annual Meeting.

"At Spur, we are advancing a new generation of gene therapies tailored to meet
the unique needs of each disease we target," said Pamela Foulds, M.D., Chief
Medical Officer at Spur Therapeutics. "Our lead program, FLT201, is designed
to produce a more stable version of the enzyme deficient in Gaucher disease,
with data showing strong safety and efficacy signals up to nearly two years
after a single dose. As we prepare to initiate a Phase 3 trial for FLT201,
these results strengthen our confidence in its potential to dramatically
reduce both the disease and treatment burden for people with Gaucher.
Alongside promising preclinical results from our Parkinson's program, which
uses the same engineered enzyme further optimized for the brain, these
findings highlight the power of our approach to provide gene therapies that
set new standards of care for people living with serious diseases."

FLT201 for Gaucher disease: Durable enzyme expression and clinical benefit

Today's oral presentation includes updated data from the Phase 1/2 GALILEO-1
trial of FLT201, an adeno-associated virus (AAV) gene therapy candidate for
Gaucher disease type 1, as well as the ongoing long-term follow-up study. Six
patients were treated in the trial with a single infusion of FLT201 at the low
dose of 4.5e11 vg/kg. The data are from four patients who were taken off prior
enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) after
dosing. All were taken off ERT or SRT within 12 weeks and remain off prior
therapies up to 21 months after dosing, as of the data cutoff. The
presentation also includes data on glucocerebrosidase (GCase) expression in
non-human primates (NHPs).

The clinical data as of March 28, 2025, and preclinical data showed:

·    Robust reductions in glucosylsphingosine (lyso-Gb1) sustained up to
15 months after stopping prior therapy; stable lyso-Gb1 levels in the one
patient who entered the trial with well-controlled levels 14 months after
withdrawal of prior therapy. Lyso-Gb1 is one of the best predictors of
treatment response in Gaucher disease.

·    Maintenance of normal hemoglobin levels and stable or improved
platelet counts up to 18 months after stopping prior therapy.

·    Robust and sustained plasma GCase activity in NHPs, maintained out
past 3.5 years.

·    Anti-GCase antibodies were transient in humans and NHPs, with no
impact on clinical benefit in the one patient who developed antibodies after
successfully discontinuing prior therapy.(( 1 ))

SPR301 for GBA1 Parkinson's: Enhanced enzyme expression in key brain regions

Spur also presented preclinical data on SPR301, an AAV gene therapy candidate
for a form of Parkinson's disease characterized by mutations in GBA1, the same
gene implicated in Gaucher disease. SPR301 leverages GCase85, the same more
stable, rationally engineered enzyme used in FLT201, further optimized for
expression in the brain.

Key findings include:

·    Superior GCase exposure in Parkinson's-affected regions of the brain,
including the substantia nigra, in GCase-deficient mice compared to wildtype
GCase gene therapy, while minimizing harmful microglia activation.

·    Broader GCase distribution than wildtype gene therapy in
GCase-deficient mice, with dose-dependent substrate reduction.

·    Superior substrate reduction compared to wildtype gene therapy,
providing a therapeutic window that potentially allows for greater efficacy at
much lower doses with a favorable safety profile.

·    Greater reduction of α-synuclein, a hallmark of Parkinson's disease,
in neuronal cells in vitro compared to wildtype gene therapy.

SBT101 in AMN: Continues to be generally well-tolerated

Spur also presented a safety update from the Phase 1/2 PROPEL clinical trial
of SBT101, a gene therapy candidate for AMN. Eight patients were treated in
PROPEL across low- and high-dose cohorts and have been followed for four to 24
months. Both doses of SBT101 have been generally well tolerated, with most
treatment-emergent adverse events being non-serious. One patient died of
disease-related complications unrelated to SBT101, and another has progressed
to cerebral ALD, a more severe form of the disease.

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, a
potential first-in-class gene therapy candidate for adrenomyeloneuropathy and
a preclinical gene therapy candidate for Parkinson's disease, as well as a
research strategy to move gene therapy into more prevalent diseases, including
forms of 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
(https://www.globenewswire.com/Tracker?data=D1Vq3687Uz8NDXGoj6gKoHgFIl1LBAyPDYQbyZNjDo-3JuRlwvLzjtKT7V22Q8VlR3sjuLksqHwwY3tax2HF7T2aGuPsM9Ci6x6I9orsRxWQSVExtzBF5xyQhAAlBQwu3ZpV_RJYdz1SKLgKfKOE3akQlLaVp6tqpD7HjFWW9zf2i8-BRdjc7kLDxXzygaT6uPQtTXjw7gWdu5hRylfL0FByT_OuT32JA8V9lYcd7lmsP0Hw25DtoW8bFrsqXkqeHUirPTT3EDwj9PQ8E5jRHw==)
 or connect with Spur on LinkedIn
(https://www.globenewswire.com/Tracker?data=7IFafJI1Wq4E5Io-2Lws2JzQEgJlriOnM7NvUvyQbtlC5vk01nMlGnxYYdiETs49RZXdLKAdAda5tAjfu5cW9EIj4-uWTH80PZGGon08tJLhQGD4dLqOLsIOIt2DcQK2Etxi4PybP2kJx0pCUw5HwbYT5-q_ZxWmR8hC44kTCBlElK2go2OKHqhf-NhrFO6coIkUtxa2x1yWdELOxO30Bpkabx6haizMGGKYpj7uFZlJ2L8fWc3ksfuB3npttIpFa-z-erUtKQWMNhrgzVmIbQ==)
.

Investor Contact

Naomi Aoki
naomi.aoki@spurtherapeutics.com
(https://www.globenewswire.com/Tracker?data=cGrJMeJdD-F_J6fSSk2hbuE3dCxox5c0XXmHEhzON9o7T-Bcu-NNxl064u2Pk7FOWNuUPAdjXiu05rujxSMq-P6i6V2ynBzWIp1R1bM9bMDwYmF-3McDROxSNt2PjSxc)

+ 1 617 283 4298

Media Contact

Carolyn Noyes
carolyn.noyes@spurtherapeutics.com
(https://www.globenewswire.com/Tracker?data=nmruR68Z7QZP8S5_flV2hOiPl6L-aj_Rd8iNRtLYZtHjaM-LCANG_5Tg8Zz57aZKTuKwnLlJWtlKISNEfgoC1mQ6Xe2K0SCvCPxZOYJVVa_vih2BTcMI9UDl8C9GjRt66C2o5rYRFlarlcoIhvjmvQ==)

+ 1 617 780 2182

 

 

 

 1  Transient anti-GCase antibodies were also observed in a second participant
with low transduction efficiency who remains on SRT.

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