REG - Syncona Limited - Beacon positive interim data for Phase 2 studies

SynconaAnnouncement

04/09/2025 13:15

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RNS Number : 0497Y  Syncona Limited  04 September 2025

4 September 2025

 

Syncona Limited ("Syncona")

 

Beacon announces positive interim data for Phase 2 studies in XLRP

 

Syncona Ltd, a leading life science investor, today notes that its portfolio
company Beacon Therapeutics ("Beacon") announced new results from two Phase 2
trials, SKYLINE and DAWN, evaluating the company's lead programme, laru-zova,
in patients with X-linked retinitis pigmentosa (XLRP).

 

The data demonstrated that laru-zova was generally well-tolerated by SKYLINE
participants through month 36 and DAWN participants at 9 months or beyond and
showed sustained improvements across several key measures of visual function,
including low luminance visual acuity and microperimetry. The results were
presented at the EURETINA 2025 Conference being held 4-7 September 2025, in
Paris, France.

 

Beacon is also evaluating low luminance visual acuity as the primary endpoint
in its ongoing pivotal VISTA trial of laru-zova for the treatment of XLRP.
Beacon recently completed enrolment of this trial and expects to report
topline data in the second half of calendar year 2026, which the Syncona
Investment Management Limited team view as a potential key value inflection
point for the company.

 

Chris Hollowood, Chief Executive Officer of Syncona Investment Management
Limited, said: "We are pleased to see further data from Beacon's DAWN and
SKYLINE clinical trials, which underline the potential of its gene therapy to
have a profound impact on patients living with a devastating blinding
condition. We look forward to continuing to work closely with the company as
it progresses through its pivotal study."

 

Enquiries

 

Syncona Ltd

 

Annabel Clark

Tel: +44 (0) 20 3981 7940

 

FTI Consulting

 

Ben Atwell / Natalie Garland-Collins / Tim Stamper

Tel: +44 (0) 20 3727 1000

 

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 Interim 9+ Month Results from DAWN
Trial and 36-Month Phase 2 SKYLINE Trial Data for Laru-zova in Patients with
X-linked Retinitis Pigmentosa (XLRP) at EURETINA 2025

 

Data showed sustained improvements across several key measures of visual
function, including low luminance visual acuity and microperimetry

 

Laru-zova was generally well-tolerated by SKYLINE participants through month
36 and DAWN participants at 9 months or beyond

 

LONDON and CAMBRIDGE, Mass., Sept. 4, 2025 - Beacon Therapeutics Holdings
Limited ('Beacon Therapeutics' or 'the Company'), a leading clinical-stage
biotechnology company with a mission to save and restore vision in people with
rare and prevalent ocular diseases, today announced new results from two Phase
2 trials, SKYLINE and DAWN, evaluating the Company's lead program, laru-zova,
in patients with XLRP. The data demonstrated that laru-zova was generally
well-tolerated by SKYLINE participants through month 36 and DAWN participants
at 9 months or beyond and showed sustained improvements across several key
measures of visual function, including low luminance visual acuity and
microperimetry. The results were presented at the EURETINA 2025 Conference
being held September 4-7, 2025, in Paris, France.

 

"We are pleased to be sharing key data from our DAWN and SKYLINE trials,
building on one of the most significant bodies of evidence for a gene therapy
in ocular diseases," said Daniel Chung, D.O., M.A., Chief Medical Officer of
Beacon Therapeutics. "These new data updates reinforce our belief in the
potential for laru-zova to be a meaningful treatment option for people living
with XLRP. We look forward to continuing to advance laru-zova through clinical
development, while engaging with regulators and the patient community."

 

XLRP is an inherited retinal disease that often leads to blindness, with no
available treatment options. It is typically caused by mutations to the
retinitis pigmentosa GTPase regulator (RPGR) gene, affecting approximately 1
in 25,000 males in the U.S., Europe and Australia. Laru-zova is a potential
best-in-class gene therapy designed to restore the natural function of both
rods and cones in XLRP by delivering a functional copy of the RPGR(ORF15) gene
designed to produce the full-length protein.

 

Key DAWN data highlights include:

·      Data continued to show early improvements in low luminance visual
acuity (LLVA) and early and sustained improvements in mean sensitivity in
study eyes, as observed by microperimetry, representing enhanced visual
function in participants evaluated at month 9 or beyond.

·      Laru-zova continued to be well-tolerated by all participants
evaluated at month 9 or beyond.

 

Key SKYLINE data highlights include:

·      Participants who received the high dose of laru-zova showed
durable improvements in retinal sensitivity through month 36, as observed by
microperimetry.

·      There was a greater response rate in the high-dose study eyes
compared to the low-dose group or untreated fellow eye.

·      Laru-zova continued to be well-tolerated by participants in both
low- and high-dose groups through month 36.

 

Beacon is also evaluating LLVA as the primary endpoint in its ongoing pivotal
VISTA trial of laru-zova for the treatment of XLRP. Beacon recently completed
enrollment
(https://www.beacontx.com/news-and-events/beacon-therapeutics-completes-enrollment-in-registrational-phase-2-3-vista-trial-of-laru-zova-for-patients-with-xlrp/)
of this trial and expects to report topline data in the second half of 2026.

 

Presentation details:

 

Presentation title: Subretinal gene therapy laru-zova for X-linked retinitis
pigmentosa (XLRP): Phase 2 DAWN trial, preliminary month 9+ results

Date: Thursday, September 4, 2025

Presenter: Rajiv Anand, M.D., FRCS, FRCOphth, Texas Retina Associates and
Retina Foundation of the Southwest

 

Presentation title: Subretinal laru-zova gene therapy for XLRP: 36-month
results of the randomized, controlled, multicenter Phase 2 SKYLINE trial

Date: Thursday, September 4, 2025

Presenter: Paul Yang, M.D., Ph.D. Chief, Paul H. Casey Ophthalmic Genetics
Division, Casey Eye Institute, OHSU

 

Contact:
info@beacontx.com (mailto:info@beacontx.com)

 

Media & Investors:
beacon@icrhealthcare.com (mailto:beacon@icrhealthcare.com)

 

About laru-zova

Laru-zova (laruparetigene zovaparvovec) is a potential best-in-class gene
therapy currently being investigated for the treatment of patients with
X-linked retinitis pigmentosa (XLRP). Laru-zova has the potential to restore
the natural function of both rods and cones in XLRP by delivering a functional
copy of the RPGR(ORF15) gene designed to produce the full-length protein.
Laru-zova has Regenerative Medicine Advanced Therapy (RMAT) and Fast Track
designations from the U.S. Food and Drug Administration (FDA), Priority
Medicines (PRIME) designation from the European Medicines Agency (EMA),
Innovative Licensing and Access Pathway (ILAP) from the UK's Medicines and
Healthcare products Regulatory Agency (MHRA), as well as Orphan Drug
Designation (ODD) from the FDA and EMA.

 

Laru-zova is investigational and has not been approved by FDA for use.

 

About the DAWN and SKYLINE Studies

DAWN (NCT06275620) is an ongoing, fully enrolled, Phase 2, open-label study of
laru-zova in the fellow eye of male participants with XLRP who have previously
been treated with an AAV vector-based gene therapy delivering the full-length
RPGR protein. The objective of DAWN is to assess two different dose levels of
laru-zova for efficacy, safety and tolerability in the target population. DAWN
is also evaluating the changes in visual function and functional vision, and
is the first trial in the laru-zova clinical development program that is
collecting and evaluating low luminance visual acuity (LLVA) data.

 

SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study
evaluating the safety, efficacy and tolerability of laru-zova in 14 male
patients with XLRP caused by mutations in the RPGR gene. The study's primary
endpoint is the proportion of response by microperimetry between the study and
fellow eye at Month 12. NCT06333249.

 

About XLRP

X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease that
predominantly affects males, typically caused by mutations in the RPGR gene.
The mutations, which affect approximately 1 in 25,000 males in the U.S.,
Europe and Australia, result in progressive photoreceptor loss over time and
visual dysfunction beginning in childhood, eventually leading to blindness and
impacting quality of life with no approved treatments.

 

About Beacon Therapeutics

Beacon Therapeutics is a clinical-stage biotechnology company with a mission
to save and restore the sight and improve the lives of people living with rare
and prevalent ocular diseases to help them to live life to the fullest. The
Company is harnessing the transformative power of gene therapy where they can
deliver the most meaningful outcomes for severe ocular diseases. Beacon's
pipeline currently targets high-impact blinding retinal diseases such as
X-linked retinitis pigmentosa (XLRP) and geographic atrophy.

 

Beacon Therapeutics' investors include Forbion, Syncona Limited, Oxford
Science Enterprises, TCGX and Advent Life Sciences, among others. Learn more
about Beacon Therapeutics at beacontx.com (https://www.beacontx.com/) and
follow on LinkedIn (https://www.linkedin.com/company/beacon-therapeutics/) for
more updates.

 

 

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