X-linked retinitis pigmentosa (XLRP) is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects an estimated 4 per 100,000 males with RPGR mutations in US, Europe and Australia1.
Beacon Therapeutics, a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, is investigating laru-zova (AGTC-501) for the treatment of patients with XLRP. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, making it uniquely well-suited to improve the lives of patients with XLRP as a potential treatment.
Beacon recently announced positive 24-month data from the Phase 2 SKYLINE Trial of laru-zova in patients with XLRP at the American Academy of Ophthalmology Annual Meeting in October.
- 24-month analysis indicated laru-zova was generally well-tolerated in the trial, with no clinically significant safety events associated with treatment
- Data demonstrates the durable profile of laru-zova, with the higher dose cohort showing a 57% response rate in the 24-month analysis of retinal sensitivity, the primary endpoint for the trial
Benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by retinitis pigmentosa GTPase regulator (RPGR) gene mutations
Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics, shared, “Our Phase 2 SKYLINE 24- month data reinforces laru-zova’s favorable safety profile and robust improvements in mean retinal sensitivity. We will continue to assess the long-term safety and durability of laru-zova but are encouraged by the results we’ve seen in the SKYLINE trial to date.” .
Beacon Therapeutics is enrolling its pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials for patients as the company progresses toward the development of a treatment for XLRP, announcing treatment of the first patient in the VISTA study in June, 2024.
Professor Paulo Stanga will be presenting preliminary results from the Phase 2 DAWN fellow eye study at FLORetina ICOOR in December.