REG - Syncona Limited - Beacon presents clinical data update

SynconaAnnouncement

19/09/2024 15:00

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RNS Number : 8736E  Syncona Limited  19 September 2024

19 September 2024

Syncona Limited

 

Beacon presents 36-month clinical data from Phase I/II trial in XLRP

 

 

 

Syncona Ltd, (the "Company"), a leading life science investor focused on
creating, building and scaling global leaders in life science, today notes
that its portfolio company, Beacon Therapeutics ("Beacon") presented 36-month
interim safety and efficacy results from its Phase I/II HORIZON trial of its
lead asset, AGTC-501, in patients with X-linked retinitis pigmentosa (XLRP).
Data was presented on 19 September 2024 at the 24(th) EURETINA conference in
Barcelona, Spain.

 

Key highlights from the presentation include:

·      With safety as the primary endpoint of this trial, AGTC-501
continues to be well-tolerated with no clinically significant safety events
related to the therapy reported amongst the 29 patients enrolled

·      Data also demonstrated that a difference in visual function
between the treated and untreated eyes was still observed at month 36, which
is the trial's secondary endpoint

·      Benefit-risk profile of AGTC-501 supports ongoing clinical
development for the treatment of patients with XLRP caused by RPGR mutations.

 

Beacon will publish 24-month data from the Phase II SKYLINE trial in XLRP
later in H2 CY2024, a potential key value inflection point for the company.

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

Chris Hollowood, Chief Executive Officer of Syncona Investment Management
Limited, said: "This data presented by Beacon highlights the strong safety
profile of this therapy, as well as promising efficacy signals and a continued
durability profile out to 36 months post-dosing, which is particularly
pleasing for an exploratory Phase I/II trial. We now look forward to the
upcoming 24-month data read-out from the Phase II SKYLINE trial which has the
benefit of an optimised dose and greater surgical repeatability. The Beacon
team continue to execute well, with both the Phase II DAWN and Phase II/III
registrational VISTA trials currently recruiting patients following the
introduction of an improved manufacturing process."

 

 

 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.

 

Beacon Therapeutics Presents 36-Month Interim Results from Phase I/2 HORIZON
Trial of AGTC-501 in Patients with XLRP

 

London, UK and Cambridge, MA, September 19, 2024 - Beacon Therapeutics
Holdings Limited ('Beacon' or 'the Company'), a leading ophthalmic gene
therapy company with a purpose to save and restore the vision of patients with
blinding retinal diseases, today presented 36-month interim results from its
Phase I/2 HORIZON trial of its lead asset, AGTC-501, in patients with X-linked
retinitis pigmentosa (XLRP) at the 24th EURETINA Congress held in Barcelona,
Spain.

 

Key presentation highlights included:

·      AGTC-501 was reported to be generally safe and well-tolerated in
the 29 patients enrolled with no clinically significant safety events related
to the therapy.

·      Data also demonstrated that a difference in visual function
between the treated and untreated eyes was still observed at month 36.

·      The benefit-risk profile of AGTC-501 supports ongoing clinical
development for the treatment of patients with XLRP caused by RPGR mutations.

 

Lance Baldo, MD, Chief Executive Officer of Beacon, stated, "This emerging
longer-term data is another clinical validation of the safety of AGTC-501 for
the treatment of XLRP. We look forward to achieving several upcoming clinical
milestones, including 24-month data from the Phase 2 SKYLINE trial in XLRP
later this year, and continued enrollment into our open-label Phase 2 DAWN
trial and Phase 2/3 VISTA trial."

 

HORIZON is a Phase 1/2, open-label, dose-escalation study of patients with
XLRP treated with subretinal AGTC-501, which has completed enrollment of 29
male participants and all participants are in long-term follow-up.

Presentation - Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis
Pigmentosa (XLRP) Phase 1/2 Multicenter Study (HORIZON): 36-Month Interim
Results

 

Presenter - Paul Yang, MD, PhD, Chief, Paul H. Casey Ophthalmic Genetics
Division, Casey Eye Institute, OHSU

 

The presentation took place on Thursday, September 19(th) at 15:30 CEST.

 

About Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to
save and restore the vision of patients with a range of prevalent and rare
retinal diseases that result in blindness.

 

The Company has an established scientific foundation that combines a
late-stage development candidate to treat X-linked retinitis pigmentosa
(XLRP), as well as two preclinical programs, one targeting dry age-related
macular degeneration (AMD) and another targeting cone-rod dystrophy (CRD).
Beacon Therapeutics also has access to a target generation technology platform
that will identify, screen, and search secreted proteins in the ophthalmology
space.

 

Lead development candidate AGTC-501, is a gene therapy program currently being
investigated for the treatment of XLRP, an inherited monogenic recessive
disorder that causes progressive vision loss, primarily in boys and young men.
XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase
regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein,
thereby addressing the full complement of photoreceptor damage caused by XLRP,
including both rod and cone loss.

 

Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science
Enterprises, TCGX, Advent Life Sciences and additional investors.

 

Find out more about Beacon Therapeutics at beacontx.com.

 

Contact:

info@beacontx.com (mailto:info@beacontx.com)

 

Media:

beacontherapeutics@edelman.com (mailto:beacontherapeutics@edelman.com)

 

###

 

 

 

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