REG - Syncona Limited - Beacon presents data from AGTC-501 in XLRP

SynconaAnnouncement

04/12/2023 07:00

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RNS Number : 4659V  Syncona Limited  04 December 2023

Syncona Limited

 

Beacon presents data from AGTC-501 in XLRP

 

4 December 2023

 

 

Syncona Ltd, a leading life science investor focused on creating, building and
scaling a portfolio of global leaders in life science, notes that its
portfolio company, Beacon Therapeutics ("Beacon"), has presented unmasked
three-month data from the Phase II SKYLINE trial of its AAV viral vector-based
gene therapy AGTC-501 in X-Linked Retinitis Pigmentosa (XLRP), at the
FLORetina ICOOR 2023 Congress in Rome.

 

Highlights from the presentation include:

·    Encouraging efficacy profile with robust and statistically
significant improvement in retinal sensitivity, the primary endpoint for the
trial; 75% (6/8) response rate in the higher dose cohort

·   Data underlines the favourable safety profile of AGTC-501, with no
clinically significant safety events related to the therapy

 

Beacon expects to present 12-month data from the SKYLINE trial in H1 CY2024,
as well as commence the pivotal Phase II/III VISTA trial. 24-month data from
the SKYLINE trial is expected to be presented in H2 CY2024.

 

Chris Hollowood, Chief Executive Officer of Syncona Investment Management
Limited, said: "This data released by Beacon from the SKYLINE trial underlines
the potential of the company's lead AGTC-501 therapy in slowing the
progression of XLRP, a devastating disease where patients become legally blind
in their mid-40s. We are pleased to see the Beacon team continue to make
significant progress as they progress towards key milestones, including the
upcoming initiation of a pivotal trial. We also look forward to seeing further
longer-term efficacy read-outs from SKYLINE in 2024, which will be key to
demonstrating the durability profile of AGTC-501."

 

The presentation can be accessed on Beacon's investor website
at https://www.beacontx.com/press-releases/
(https://www.beacontx.com/press-releases/) and the full text of the press
release from the company is contained below.

 

 

 

 ENDS 

 

Copies of this press release and other corporate information can be found on
the company website at: www.synconaltd.com (http://www.synconaltd.com)

 

 

Enquiries

 

Syncona Ltd

 

Annabel Clark / Fergus Witt

Tel: +44 (0) 20 3981 7940

 

FTI Consulting

 

Ben Atwell / Natalie Garland-Collins / 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 and building companies to deliver transformational treatments to
patients in areas of high unmet need.

 

Our strategy is to create, build and scale companies around exceptional
science to create a diversified portfolio of 20-25 globally leading healthcare
businesses for the benefit of all our stakeholders. We focus on developing
treatments for patients by working in close partnership with world-class
academic founders and 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 Announces Unmasked Three-Month Data from Phase 2 SKYLINE Trial of
AGTC-501 for X-Linked Retinitis Pigmentosa

 

·      Encouraging efficacy profile with significant improvement in
retinal sensitivity in higher dose cohort

·      Favourable safety profile with no clinically significant safety
events related to the therapy

·      Further 12-month data from the Phase 2 SKYLINE trial to be
presented in H1 2024

·      Pivotal Phase 2/3 VISTA trial for AGTC-501 in XLRP expected to
initiate in H1 2024

 

London, UK, Cambridge, MA, 4 December 2023 - Beacon Therapeutics Holdings
Limited ('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene
therapy company with a purpose to save and restore the vision of patients with
blinding retinal diseases, announces that it presented unmasked three-month
data from its randomized, controlled, multicenter Phase 2 SKYLINE trial of
AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP) at the
FLORetina ICOOR 2023 Congress in Rome, held between 30 November - 3 December,
2023.

 

XLRP is an Orphan Disease affecting approximately 17,000 patients in the US
and EU5. It is predominantly caused by mutations in the retinitis pigmentosa
GTPase regulator (RPGR) gene. Unlike other approaches in the space, AGTC-501
expresses the full length RPGR protein, thereby addressing the entirety of
photoreceptor damage caused by XLRP, including both rod and cone loss.

The data demonstrated robust improvements in retinal sensitivity, the trial's
primary efficacy endpoint. Responses were seen in 6 out of 8 patients from the
high dose cohort (75% response rate), and in 1 out of 6 patients in the low
dose cohort (16.7% response rate), three months post-dosing. The Company
considers a responder to be a patient who has an improvement in retinal
sensitivity as assessed by microperimetry of at least 7 decibels (dB) in at
least 5 loci.  It is also notable that in the high dose cohort the mean
retinal sensitivity of the entire macula increased, with change from baseline
being a secondary endpoint in the trial.

 

AGTC-501 was also found to be generally well-tolerated in this trial with no
serious adverse events (SAEs) attributed to the study agent. The majority of
non-serious adverse events (AEs) were mild to moderate in severity, with no
difference found between the two dose levels.

 

Beacon expects to initiate the pivotal Phase 2/3 VISTA trial of AGTC-501 in H1
2024, as well as present 12-month data from the SKYLINE trial, with the
24-month interim analysis from the Phase 1/2 HORIZON trial already supporting
the durability profile of the therapy.

 

Presentation - Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis
Pigmentosa Phase 2 Randomized, Controlled, Multicenter Clinical Trial
(Skyline) 3-Month Results

 

Presenter - Andreas K. Lauer, MD, Thiele-Petti Endowed Chair and Professor
Casey Eye Institute - Oregon Health & Science University Portland, Oregon,
United States

 

 

The presentation took place on Saturday, 2 December 2023.

 

Dr. Nadia Waheed, Chief Medical Officer of Beacon Therapeutics, commented: "We
are pleased to present further data from AGTC-501, a therapy for patients
suffering from XLRP, a blinding condition for which there is currently no
approved treatment. This data underlines the strong efficacy and safety
profile of the therapy. We look forward to presenting further 12-month data
from the trial early next year, as well as commencing the Phase 2/3 VISTA
trial, bringing us closer to our ambition to change the lives of patients with
a devastating blinding condition."

 

Contacts:

 

Beacon Therapeutics: David Fellows, CEO

Email: info@beacontx.com

 

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 in-licensed from the University of
Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease.

 

Lead development candidate AGTC-501, is a gene therapy program in Phase 2
clinical trials for the treatment of XLRP, an inherited monogenic recessive
disorder that causes progressive vision loss 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 Therapeutics also has access to a target generation technology platform
that will identify, screen, and search secreted proteins in the ophthalmology
space.

 

The Company is supported by funds from Syncona and additional investors
including Oxford Science Enterprises.

 

Find out more about us online at: www.beacontx.com (http://www.beacontx.com) .

 

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