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
- Demonstrated early improvements in low luminance visual acuity (LLVA)
- Early and sustained improvements in mean sensitivity observed via microperimetry
- Treatment was generally well-tolerated with mostly mild to moderate adverse events
- No serious complications like retinal detachments or endophthalmitis reported
- Company continues enrollment in pivotal Phase 2/3 VISTA trial
- Majority of adverse events were related to surgical procedures and required steroids
- Limited data set (only 6-month interim results)
- Currently no approved treatment options for XLRP
Insights
Laru-zova shows promising improvements in visual function measures for X-linked Retinitis Pigmentosa patients with acceptable safety, advancing hope for the first effective treatment.
The interim 6-month data from the Phase 2 DAWN trial represents encouraging progress for patients with X-linked Retinitis Pigmentosa. XLRP is a devastating inherited retinal disease affecting approximately 1 in 25,000 males, with no approved treatments currently available. The improvements observed in low luminance visual acuity are particularly significant, as diminished vision in dim lighting is often among the earliest and most impactful functional losses for these patients.
The safety profile appears manageable, with adverse events primarily related to the surgical procedure and protocol-required steroids rather than the therapeutic agent itself. Importantly, the absence of serious complications like retinal detachments or endophthalmitis – which can occur with subretinal injections – is reassuring at this stage.
What distinguishes laru-zova is its mechanism of delivering a functional copy of the RPGRORF15 gene using a proprietary capsid designed specifically for high photoreceptor transduction. This addresses the fundamental genetic defect causing XLRP. The DAWN trial's unique design, studying patients previously treated with other gene therapies, provides valuable insights into sequential therapeutic approaches.
While these interim results merit optimism, longer follow-up will be essential to determine durability of effect and meaningful quality-of-life improvements. The concurrent advancement of the pivotal VISTA trial will provide more definitive evidence regarding laru-zova's potential to become the first approved therapy for this blinding condition.
Positive Phase 2 data strengthens laru-zova's profile in treating XLRP, an untapped market with no approved therapies, supporting continued clinical advancement.
The positive interim data from Beacon Therapeutics' Phase 2 DAWN trial strengthens the clinical profile of laru-zova, potentially enhancing the company's position in the ophthalmologic gene therapy landscape. The demonstrated improvements across multiple visual function measures address a disease with no currently approved treatments.
Rare disease therapeutics typically command premium pricing structures, which could make XLRP commercially viable despite its limited patient population of 1 in 25,000 males. The efficacy shown in previously treated patients suggests laru-zova could potentially serve both treatment-naïve patients and those who have received prior gene therapies, expanding its potential utility.
The company's dual-track approach with both the DAWN Phase 2 and pivotal VISTA Phase 2/3 trials demonstrates a comprehensive clinical development strategy. The specific capsid engineering highlighted in the release shows Beacon's focus on optimizing delivery to photoreceptors, a critical factor for gene therapy success in retinal diseases.
While these 6-month interim results are promising, significant development milestones remain before potential commercialization. Gene therapy development involves complex manufacturing, regulatory, and clinical challenges. The data represents a meaningful risk-reduction event in laru-zova's development journey, supporting continued advancement of this program targeting a serious unmet medical need with potential best-in-class properties.
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 06, 2025 (GLOBE NEWSWIRE) -- 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 RPGRORF15 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
Media & Investors:
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 RPGRORF15 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) 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) 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.
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