REG - Syncona Limited - Beacon presents positive data

SynconaAnnouncement

07/05/2025 07:00

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RNS Number : 5357H  Syncona Limited  07 May 2025

07 May 2025

 

Syncona Limited

 

Beacon Therapeutics announced positive data from Phase II DAWN trial

 

Syncona Ltd, ("Syncona" or the "Company") a leading life science investor
focused on creating, building and scaling global leaders in life science,
notes that its portfolio company Beacon Therapeutics ("Beacon")  announced
six-month interim safety and efficacy results from its Phase II DAWN trial of
the company's lead programme, laru-zova (laruparetigene zovaparvovec), in
patients with X-linked retinitis pigmentosa (XLRP) at the Association for
Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting. The
publishing of this data is a capital access milestone 1  for Beacon.

 

Key highlights from the presentation include:

 

·      Patients in the trial showed improvements across several key
visual function measures, demonstrating laru-zova's potential to enhance
vision in patients with XLRP

o  Data demonstrated early improvements in low luminance visual acuity
(LLVA), an important measure of visual function, with greater two and three
line improvements in the study eyes compared to previously treated fellow eyes
in participants evaluated at month six or beyond.

o  Data also showed early and sustained improvements in mean sensitivity in
study eyes, as observed by microperimetry, indicating enhanced visual function
in participants evaluated at month six or beyond.

·      Laru-zova was generally well-tolerated by patients in the DAWN
trial at six months

o  Ocular treatment-emergent adverse events were generally non-serious and
mild or moderate in severity, with a majority related to surgical procedures
and steroids required by the protocol that have since resolved

o  There were no suspected unexpected serious adverse reactions, retinal
detachments or endophthalmitis (inflammation inside the eye) reported

Elisa Petris, Partner of Syncona Investment Management Limited and Board
Director of Beacon Therapeutics, said: "We are really pleased with the
progress at Beacon. It is fantastic to see continued visual improvements in
patients evaluated at the six-month time point in this data update. We are
optimistic as the company progresses enrolment for its Phase II/III pivotal
study and look forward to seeing data in this crucial programme. We see a
really differentiated opportunity with this therapy to transform the lives of
patients with a devastating retinal blinding condition."

 

Beacon's announcement is copied below and can be accessed at the company's
website at beacontx.com (https://www.beacontx.com/) .

 

 

 ENDS 

 

Enquiries

 

Syncona Ltd

 

Annabel Clark / Tim Stamper

Tel: +44 (0) 20 3981 7912

 

FTI Consulting

 

Ben Atwell / Natalie Garland-Collins

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, 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 close partnership with
world-class academic founders and 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 Capital Access Milestones

 

A capital access milestone is a de-risking event for a portfolio company that
is expected to enable access to capital, which underpins progression towards a
company's next milestone. It is less likely that a capital access milestone
(relative to a key value inflection point) will drive significant NAV growth
for Syncona, for example by increasing the possibility of a realisation event,
such as M&A.

 

 

Beacon Therapeutics Announces Positive Phase 2 Interim 6-Month Data from DAWN
Trial of Laru-zova in Patients with X-linked Retinitis Pigmentosa (XLRP) at
ARVO 2025

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

Laru-zova was generally well-tolerated by DAWN participants at 6 months

LONDON and CAMBRIDGE, Mass., May 6, 2025 - Beacon Therapeutics Holdings
Limited ('Beacon Therapeutics' or 'the Company'), a leading clinical-stage
biotechnology company with a mission to save and restore the vision of
patients with blinding retinal diseases, today announced 6-month interim
safety and efficacy results from the Phase 2 DAWN trial of the Company's lead
program, laru-zova (laruparetigene zovaparvovec), in patients with X-linked
retinitis pigmentosa (XLRP) at the Association for Research in Vision and
Ophthalmology (ARVO) 2025 Annual Meeting being held May 4-8, 2025 in Salt Lake
City. Laru-zova was generally well-tolerated by all DAWN participants
evaluated at 6 months or beyond and initial data showed promising improvements
in visual function across several key measures.

 

"Over the past five years we have built a compelling body of safety and
efficacy data on laru-zova across three different clinical studies," said
Lance Baldo, M.D., Chief Executive Officer of Beacon Therapeutics. "We are
pleased to be sharing the 6-month data update from the DAWN Phase 2 study that
continues to demonstrate laru-zova's potential to enhance vision in patients
with XLRP, including improvements in multiple measures of visual function. We
look forward to continuing the advancement of this exciting novel treatment
option for patients suffering from XLRP."

 

XLRP is an inherited retinal disease often caused by mutations to the RPGR
gene, affecting 1 in 25,000 males in the U.S., Europe and Australia. The
disease often leads to blindness by middle age, with no available treatment
options. 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 using a well-established vector with a
proprietary capsid designed for high transduction of photoreceptors, and a
codon-optimized gene to produce the full-length protein.

 

Key data highlights include:

·    Ocular treatment-emergent adverse events (TEAEs) were generally
non-serious and mild or moderate in severity, with a majority related to
surgical procedures and steroids required by the protocol that have since
resolved. There were no suspected unexpected serious adverse reactions,
retinal detachments or endophthalmitis reported.

·    Data demonstrated early improvements in low luminance visual acuity
(LLVA), an important measure of visual function, with a greater number of two
and three line improvements in the study eyes compared to previously treated
fellow eyes in participants evaluated at month 6 or beyond.

·    Data also showed early and sustained improvements in mean sensitivity
in study eyes, as observed by microperimetry, indicating enhanced visual
function in participants evaluated at month 6 or beyond.

 

DAWN is an 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 study aims to assess two dose levels of
laru-zova for efficacy, safety and tolerability in the untreated eye of
participants who previously received gene therapy for XLRP.

 

The Company continues to enroll patients for its pivotal Phase 2/3 VISTA trial
of laru-zova for patients with XLRP.

 

Presentation Title: Subretinal gene therapy laru-zova (AGTC-501) for X-linked
retinitis pigmentosa (XLRP): Phase 2 DAWN preliminary month 6+ results

 

Presenting Author: 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, and Chief of the Paul H.
Casey Ophthalmic Genetics Division at the Casey Eye Institute, Oregon Health
and Science University in Portland, Oregon

 

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 VISTA Studies

 

DAWN (NCT06275620 (https://clinicaltrials.gov/study/NCT06275620) ) is a 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.

 

VISTA (NCT04850118 (https://clinicaltrials.gov/study/NCT04850118) ) is a Phase
2/3, randomized, controlled, masked, multi-center pivotal study evaluating the
efficacy, safety and tolerability of laru-zova in two study groups compared to
an untreated control group. The study will evaluate the proportion of
participants with a 15 or more letter increase from baseline in LLVA and
additional measures of functional vision. The VISTA study is currently
enrolling.

 

About XLRP

X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease that
predominantly affects males, typically caused by mutations in the retinitis
pigmentosa GTPase regulator (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 vision of patients with blinding retinal diseases. The
Company has an established scientific foundation that includes a late-stage
clinical development candidate to treat XLRP and two preclinical programs
targeting dry age-related macular degeneration (AMD) and an inherited cone-rod
dystrophy (CRD).

 

Beacon Therapeutics' investors include Syncona Limited, Forbion, Oxford
Science Enterprises, TCGX and Advent Life Sciences, among others. Learn more
about Beacon Therapeutics at beacontx.com (https://www.beacontx.com/) .

 

 

 

 

 

 

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