Syncona
Limited
Beacon presents 12-month
clinical data from Phase II trial in XLRP
8 February 2024
Syncona Ltd, a leading healthcare
company focused on creating, building and scaling global leaders in
life science, notes that its portfolio company, Beacon Therapeutics
Holdings Limited ("Beacon") presented 12-month interim safety and
efficacy results from its Phase II SKYLINE trial of its lead asset,
AGTC-501, in patients with X-linked retinitis pigmentosa (XLRP) at
the 47th Annual Macula Society Meeting in Palm Springs,
California.
Highlights from the presentation
include:
· Encouraging efficacy shown by improvements in retinal
sensitivity, the primary endpoint for the trial, with a
63% response rate in the higher dose
cohort[1]
· Patients also showing robust improvements in visual function,
a secondary endpoint for the trial
· AGTC-501 was generally well tolerated with no clinically
significant safety events associated with treatment
· Pivotal Phase II/III VISTA trial for AGTC-501 in XLRP expected
to begin in H1 CY2024
Elisa Petris, Lead Partner at
Syncona Investment Management Limited, said: "This 12-month data presented by
Beacon from the SKYLINE trial shows a strong response from the
company's lead AGTC-501 therapy, which continues to display a
favourable efficacy and safety profile. It is pleasing to see
improvements in visual function amongst patients who have been
treated. This underlines AGTC-501's potential in XLRP, a disease
area where there is currently no approved treatment. Key to
understanding the longer term durability profile of the therapy
will be 24-month data from SKYLINE, which is expected in H2
CY2024."
The presentation can be accessed on
Beacon's website at https://www.beacontx.com/news-and-events/ and
the full text of the announcement from Beacon is contained
below.
[ENDS]
Enquiries
Syncona Ltd
Annabel Clark
Tel: +44 (0) 7714 916615
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, across
development stage and therapeutic areas, 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.
Copies of this press release and
other corporate information can be found on the company website at:
www.synconaltd.com Forward-looking statements - this announcement
contains certain forward-looking statements
with respect to the portfolio of investments of Syncona Limited.
These statements and forecasts involve risk and uncertainty because
they relate to events and depend upon circumstances that may or may
not occur in the future. There are a number of factors that could
cause actual results or developments to differ materially from
those expressed or implied by these forward-looking statements. In
particular, many companies in the Syncona Limited portfolio are
conducting scientific research and clinical trials where the
outcome is inherently uncertain and there is significant risk of
negative results or adverse events arising. In addition, many
companies in the Syncona Limited portfolio have yet to
commercialise a product and their ability to do so may be affected
by operational, commercial and other risks.
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 Announces
Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in
Patients with X-Linked Retinitis Pigmentosa
· AGTC-501 was generally safe and well-tolerated and showed
robust improvements in visual function at the 12-month
analysis.
· Pivotal Phase 2/3 VISTA trial for AGTC-501 in XLRP expected to
begin in H1 2024.
London, UK, Cambridge, MA, 08
February 2024 - Beacon Therapeutics Holdings Limited ('Beacon
Therapeutics' or 'the Company'), a leading ophthalmic gene therapy
company with a mission to save and restore the vision of patients
with blinding retinal diseases, yesterday announced the
presentation of 12-month interim safety and efficacy results of the
Phase 2 SKYLINE trial in patients with X-linked retinitis
pigmentosa (XLRP) at the 47th Annual Macula Society
Meeting in Palm Springs, California.
Twelve-month data from males with
XLRP demonstrated a response rate (defined as an improvement in
retinal sensitivity, as assessed by microperimetry, of at least 7
decibels (dB) in at least 5 loci) of 63% in study eyes treated with
a high dose (6.8 E+11 vg/eye) of AGTC-501. Response rates in study
eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were
similar to the untreated fellow eyes in the high dose cohort (0%
for both). In addition, patients in the high dose cohort also
demonstrated a robust improvement in visual function, including
mean retinal sensitivity. AGTC-501 was well tolerated and
there were no clinically significant safety events associated with
treatment, and any treatment-related adverse events were mostly
nonserious and mild to moderate in severity.
XLRP is an orphan disease
predominantly caused by mutations in the retinitis pigmentosa
GTPase regulator (RPGR) gene. AGTC-501 expresses the full length
RPGR protein, and it is therefore expected to rescue function of
both rod and cone photoreceptors, making it uniquely well-suited as
a potential treatment to improve the lives of patients with
XLRP.
Dr. Nadia Waheed, Chief Medical
Officer of Beacon Therapeutics, said, "These data, which
demonstrate a favorable safety profile and notable improvement in
visual function, are another positive step in the development of
AGTC-501 for XLRP, a blinding, orphan disease for which there is
currently no approved treatment. We look forward to announcing the
initiation of our Phase 2/3 VISTA trial in the first half of
2024."
Presentation -
Subretinal AGTC-501 Gene Therapy for XLRP: 12-Month Interim Safety
& Efficacy Results of the Phase 2 SKYLINE
Trial
Presenter - Mark Pennesi, M.D.,
Ph.D., FARVO, Director, Ophthalmic Genetics at the Retina
Foundation in Dallas, Texas; Professor of Ophthalmology and
Professor of Molecular and Medical Genetics, Paul H. Casey
Ophthalmic Genetics Division at the Casey Eye Institute, Oregon
Health and Science University
The presentation took place on
Wednesday, February 7 at 6:25 p.m. PT
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), 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 currently being investigated 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 Beacon
Therapeutics at beacontx.com.
###