15 October 2024
Syncona
Limited
Beacon announces positive
24-month data from Phase II trial of AGTC-501 in
XLRP
Syncona Ltd, ("Syncona" or 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") announced
the presentation of 24-month interim safety and
efficacy data from the Phase II SKYLINE trial of its lead asset,
AGTC-501, in patients with X-linked
retinitis pigmentosa (XLRP), at the
American Academy of Ophthalmology's Annual Meeting in Chicago,
Illinois. The publishing of this data is a
potential key value inflection point for Beacon.
Key highlights from the presentation
include:
· 24-month analysis indicated AGTC-501 was generally well
tolerated with no clinically significant safety events associated
with treatment
· Data
demonstrates the durable efficacy profile of AGTC-501 with the
higher dose cohort showing a 57% response rate in the 24-month
analysis of retinal sensitivity, the primary endpoint for the
trial[1]
· Benefit-risk profile supports on-going clinical development
for AGTC-501 for the treatment of patients with XLRP, a severe,
aggressive, inherited retinal disease that often leads to blindness
by middle age with no treatment options available
· Beacon
continues to enrol patients in its pivotal Phase II/III VISTA trial
and its Phase II DAWN trial for AGTC-501 in XLRP
Chris Hollowood, Chief Executive Officer of Syncona Investment
Management Limited and Board Director of Beacon
Therapeutics, said: "This data
supports our thesis that AGTC-501 can be a potentially
life-changing treatment for patients suffering from XLRP. The
continued efficacy of the therapy at 24-months post-dosing
underlines the potential of a one-time gene therapy for this
condition, with the data also showing a strong safety profile. The
delivery of this milestone is a potential key value inflection
point for Beacon, and we look forward to seeing upcoming data from
the Phase II DAWN trial and further progress in the Phase II/III
VISTA trial, with data from both of these trials along with SKYLINE
supporting the company's regulatory strategies in the US and
EU."
Beacon's announcement is copied
below and can be accessed at the company's website
at beacontx.com.
[ENDS]
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Beacon Therapeutics Announces
Positive 24-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in
Patients with X-Linked Retinitis Pigmentosa
· 24-month analysis indicated AGTC-501 was generally safe and
well-tolerated, with no clinically significant safety events
associated with treatment
· Data
demonstrates the durable profile of AGTC-501, with the higher dose
cohort showing a 57% response rate in the 24-month analysis of
retinal sensitivity, the primary endpoint for the trial
· Benefit-risk profile supports on-going clinical development
for the treatment of patients with X-Linked Retinitis Pigmentosa
(XLRP) caused by retinitis pigmentosa GTPase regulator (RPGR) gene
mutations
· Pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials for
AGTC-501 in XLRP are currently enrolling
London, UK, Cambridge, MA, 15 October
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 24-month
interim safety and efficacy results of the Phase 2 SKYLINE trial in
patients with XLRP at the American Academy of Ophthalmology's
Annual Meeting in Chicago, Illinois.
The 24-month data showed a response
rate of 57% (4/7) in study eyes treated with a high dose (6.8 E+11
vg/eye) of AGTC-501, defined as a patient who has an improvement in
retinal sensitivity as assessed by microperimetry of at least 7
decibels (dB) in at least 5 loci. Patients in the high-dose cohort
also showed a robust improvement in mean retinal sensitivity. Study
eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 showed a
response rate of 25% (1/4). There were no clinically significant
safety events associated with AGTC-501 treatment in this trial, and
any treatment-emergent adverse events were mostly non-serious and
mild to moderate in severity.
XLRP is a severe, aggressive,
inherited retinal disease that often leads to blindness by middle
age, with no treatment options available. XLRP primarily affects an
estimated 3.4 - 4.4 per 100,000 young males with RPGR mutations in
US, Europe and Australia. AGTC-501 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, making it uniquely well-suited to improve the lives of
patients with XLRP as a potential treatment.
Lance Baldo, MD, Chief Executive
Officer of Beacon Therapeutics, stated, "Our Phase 2 SKYLINE
24-month data reinforces AGTC-501's favorable safety profile and
robust improvements in mean retinal sensitivity. We will continue
to assess the long-term safety and durability of AGTC-501 but are
encouraged by the results we've seen in the SKYLINE trial to
date."
Beacon Therapeutics is also enrolling
its pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials as
the Company progresses toward the development of a treatment for
XLRP.
Poster - Subretinal AGTC-501
Gene Therapy for XLRP: 24-Month Interim Results of the Phase 2
SKYLINE Trial.
Presenting Author - Robert
Sisk, MD, FACS, FASRS, Associate Professor of Ophthalmology at the
University of Cincinnati; Vitreoretinal Surgeon and partner at
Cincinnati Eye Institute; Director of Pediatric Retinal Surgery and
Director of Ophthalmic Genetics at Cincinnati Children's
Hospital.
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 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 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.
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