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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/ 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
Enquiries
Syncona Ltd
Annabel Clark / Fergus Witt
Tel: +44 (0) 20 3981 7940
FTI Consulting
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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.
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