Beacon Therapeutics announces positive 3-month data from Phase 2 DAWN trial of AGTC-501 in patients with X-linked retinitis pigmentosa

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Beacon Therapeutics presented 3-month interim safety and efficacy data from the Phase 2 DAWN trial of laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP) at the FLORetina ICOOR 2024 meeting in Florence, Italy.

According to the company, the data show early promise in improving low luminance visual acuity (LLVA), a key measure of visual function in patients with this debilitating disease.¹

Key highlights from the presentation include:

• Laru-zova was well-tolerated by all participants, with no treatment-emergent adverse events (TEAEs) related to the study agent, including no ocular inflammatory events.
• Early data demonstrate improvements in LLVA, a functional visual outcome that is particularly relevant for patients with XLRP.
• The benefit-risk profile of laru-zova supports the ongoing clinical development of the therapy for patients with XLRP caused by RPGR mutations.

The DAWN trial is a non-randomized, open-label study investigating two doses of laru-zova in participants with XLRP who had previously received full-length AAV-vector-based gene therapy targeting the RPGR protein. The trial is designed to assess the efficacy, safety, and tolerability of laru-zova in the untreated eye of these patients.

XLRP is a severe, progressive inherited retinal disease that leads to blindness, typically by middle age, and currently has no approved treatments. It primarily affects young males, with an estimated prevalence of 1 in 25,000 males in the US, Europe, and Australia. XLRP is most commonly caused by mutations in the RPGR gene.¹

Laru-zova is a gene therapy that expresses the full-length RPGR protein, which may address both rod and cone photoreceptor loss caused by the disease. The therapy aims to halt or slow disease progression, potentially representing a best-in-class treatment for XLRP.

Lance Baldo, MD, CEO of Beacon Therapeutics, expressed optimism about the results.

“The strong safety profile of laru-zova, coupled with the early signs of improvement in low luminance visual acuity, underscores the potential of this therapy to significantly impact patients with XLRP,” he said in a statement. “These data not only support the ongoing VISTA trial but also reflect our commitment to bringing hope to the patients and families affected by this devastating disease."

Beacon Therapeutics is currently enrolling patients in its pivotal Phase 2/3 VISTA trial, a multi-center study designed to further evaluate the efficacy and safety of laru-zova in XLRP.

Two presentations were made at FLORetina-ICOOR 2024, They included:

• Subretinal Gene Therapy Laru-Zova (AGTC-501) for XLRP: Phase 2 DAWN Trial Preliminary Results
• RPGR Gene Therapy and Beacon's Clinical Trials: Beacon Therapeutics' Subretinal Gene Therapy Laru-Zova (AGTC-501) for XLRP

The presentations were delivered by Professor Paulo Eduardo Stanga, MD, founder and chief medical officer of The Retina Clinic London.

Laru-zova (AGTC-501) is Beacon Therapeutics' lead gene therapy candidate, being developed for the treatment of XLRP, a monogenic recessive disorder caused by mutations in the RPGR gene. Laru-zova is designed to express the full-length RPGR protein, which may address the full spectrum of photoreceptor damage caused by XLRP, including both rod and cone loss. The therapy is currently under investigation as a potential treatment for progressive vision loss in patients with XLRP.¹

Reference

1. Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP). Syncona. Published 2024. Accessed December 9, 2024. https://www.synconaltd.com/news-and-insights/news/beacon-therapeutics-announces-positive-3-month-data-from-phase-2-dawn-trial-of-laru-zova-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-xlrp/