Beacon Therapeutics reported that laruparetigene zovaparvovec (laru-zova), its investigational gene therapy for X-linked retinitis pigmentosa (XLRP), met the primary endpoint of the pivotal VISTA trial, with significant improvements in low-luminance visual acuity (LLVA) at 12 months.

The randomized, controlled phase 2/3 trial enrolled 85 male participants aged 12 years to 48 years with XLRP caused by mutations in the RPGR gene. At month 12, at least a 15-letter improvement in LLVA was achieved by 31.0% of participants receiving the high dose (P=.0019) and 24.1% receiving the low dose (P=.0106), compared with no participants in the untreated control group.

Microperimetry results also favored treatment. The least-squares mean difference in macular sensitivity compared with untreated controls was 1.201 dB for the high-dose group (P=.0614) and 1.312 dB for the low-dose group (P=.0405).

Beacon reported predominantly mild to moderate ocular treatment-emergent adverse events. Two ocular serious adverse events occurred in the low-dose group and were attributed to the surgical procedure.

The company plans to begin rolling submission of a biologics license application to the US Food and Drug Administration later this year. Additional VISTA data are scheduled for presentation at Retina Subspecialty Day during the American Academy of Ophthalmology annual meeting in October. RP