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

SynconaAnnouncement

23/10/2024 07:15

For best results when printing this announcement, please click on link below:
https://newsfile.refinitiv.com/getnewsfile/v1/story?guid=urn:newsml:reuters.com:20241023:nRSW2079Ja&default-theme=true

RNS Number : 2079J  Syncona Limited  23 October 2024

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) 31(st) 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/ (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 31(st) 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
(http://www.spurtherapeutics.com/) 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) 31(st) 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
(file:///C%3A/Users/Sean.OLeary/AppData/Local/Microsoft/Windows/INetCache/Content.Outlook/NZL6KHF4/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
(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

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  PFUFFFVLIELFFIS