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 24th 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.
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 19th 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
Media:
beacontherapeutics@edelman.com
###