6 December 2024
Syncona
Limited
Beacon announces positive
three-month data from Phase II DAWN trial of laru-zova (AGTC-501)
in XLRP
Syncona Ltd, ("Syncona") 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") announced the presentation of three-month
interim safety and efficacy data from the Phase II DAWN trial of
its lead asset, laru-zova (AGTC-501), in patients with X-linked
retinitis pigmentosa (XLRP), at the FLORetina-ICOOR Meeting 2024 in
Florence, Italy. The publishing of this data is a key value
inflection point for Beacon[1].
The DAWN study treats patients with
XLRP who have previously been treated with an AAV gene therapy in
their other eye. The purpose of DAWN is to assess two different
doses of laru-zova for efficacy, safety, and tolerability in the
untreated eye of participants who previously received gene therapy
for XLRP.
Key highlights from the announcement
include:
· The
three-month data demonstrate that laru-zova has been well-tolerated
by all participants
· The data also show
promising early improvements in low luminance visual acuity (LLVA),
which is a critical measure of visual function and a marker of
early disease progression
· The data
support the ongoing development of laru-zova in XLRP, a severe,
aggressive, inherited retinal disease that impacts boys and young
men and often leads to blindness by middle age, with no treatment
options available
Beacon Therapeutics is currently
enrolling for its Phase II/III pivotal VISTA trial of laru-zova in
XLRP, with a data readout expected in CY2026, a key value
inflection point for the company.
Chris Hollowood, Chief Executive Officer of Syncona Investment
Management Limited and Board Director of Beacon Therapeutics,
said: "Building on the recent
24-month data from the Phase II SKYLINE trial, these positive
results from DAWN further demonstrate the potential of Beacon's
laru-zova programme. The early visual improvements observed
reinforce the compelling efficacy data shown to date and offer
further hope to XLRP patients. We believe that laru-zova could be a
life-changing treatment for these patients and look forward to
further data, including from the Phase II/III VISTA trial, which
will be used to support regulatory approval."
Beacon's announcement is copied
below and can be accessed at the company's website
at beacontx.com.
[ENDS]
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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
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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.
About Key Value Inflection Points
A key value inflection point is a
material de-risking event for a portfolio company that has the
potential to drive significant NAV growth for Syncona, for example
by increasing the possibility of a realisation event, such as
M&A. These milestones can also enable companies to access
significant capital including through financings and IPOs, which
may take place at valuation uplifts and underpin progression to a
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drive greater value. M&A or capital access is unlikely to occur
immediately following a key value inflection point.
Beacon Therapeutics Announces
Positive 3-Month Data from Phase 2 DAWN Trial of
laru-zova (AGTC-501) in
Patients with X-Linked Retinitis Pigmentosa
(XLRP)
· To
date, laru-zova has been well-tolerated by all participants in the
Phase 2 DAWN study.
· Data
show promising early improvements in low luminance visual acuity
(LLVA), a critical measure of visual function.
· Data
build confidence in laru-zova as a potential treatment for patients
with XLRP.
· Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is
currently enrolling.
London, UK, Cambridge, MA, 6 December
2024 - 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, today announced the presentation of 3-month
interim safety and efficacy results of the Phase 2 DAWN trial in
patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence,
Italy.
Key
presentation highlights:
· The
three-month data show that laru-zova has been well-tolerated by all
participants.
· No
study agent-related treatment emergent adverse events (TEAEs) were
reported, including no ocular inflammatory adverse
events.
· Data
also show promising early improvements in LLVA, a critical measure
of visual function.
· The
benefit-risk profile supports on-going clinical development for the
treatment of patients with XLRP caused by RPGR
mutations.
DAWN is a non-randomized, open-label
study of laru-zova in participants with XLRP who have previously
been treated with a full-length AAV-vector based gene therapy
targeting the RPGR protein. The purpose of DAWN is to assess two
different doses of laru-zova for efficacy, safety and tolerability
in the untreated eye of participants who
previously received gene therapy for XLRP.
XLRP is a severe, aggressive,
inherited retinal disease that often leads to blindness by middle
age, with no treatment options available. XLRP primarily affects
young males with an estimated prevalence of 1 in 25,000 males in
US, Europe and Australia having XLRP with RPGR mutations. Laru-zova
expresses the full length RPGR protein and is therefore expected to
address the entirety of photoreceptor damage caused by XLRP,
including both rod and cone loss, representing a potential
best-in-class treatment for progressive vision loss in patients
with XLRP.
Lance Baldo, MD, chief executive
officer of Beacon Therapeutics, stated, "We are encouraged by the
early results from the Phase 2 DAWN study. The strong safety
profile observed to date is complemented by promising early
improvements in low luminance visual acuity - a meaningful and
functional measure of vision in patients with XLRP. These data not
only support the ongoing pivotal VISTA study, but also strengthen
our commitment to this opportunity to bring hope to patients and
families affected by this devastating disease."
Beacon Therapeutics is also enrolling
patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it
develops this potential treatment for patients with
XLRP.
Presentations -
•
Subretinal Gene Therapy laru-zova (AGTC-501) for
X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study
(DAWN): Preliminary Results
•
RPGR gene therapy and the Beacon clinical trials:
Beacon Therapeutics Subretinal Gene Therapy laru-zova (AGTC-501)
for X-Linked Retinitis Pigmentosa (XLRP)
Presenting Author - Professor
Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina
Clinic London
Contact:
info@beacontx.com
Media:
beacontherapeutics@edelman.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) and two
preclinical programs, one targeting dry age-related macular
degeneration (AMD) and another targeting an inherited 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 laru-zova
(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. Laru-zova
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.
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