REG - Syncona Limited - Beacon presents 12-month clinical data

SynconaAnnouncement

08/02/2024 12:15

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RNS Number : 4067C  Syncona Limited  08 February 2024

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  (#_ftn1)

·      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/
(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 47(th) 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
(https://www.xcdsystem.com/maculasociety/program/hDy5ljm/index.cfm?pgid=378)

 

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 (mailto:info@beacontx.com)

 

Media:

beacontherapeutics@edelman.com (mailto: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.

 

###

 

 

 

 1  (#_ftnref1) At the 3-month data cut off the response rate in the higher
dose cohort was 75%

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