Laru-zova shows safety, low luminance visual acuity gains in X-linked retinitis pigmentosa

Laru-zova demonstrated a positive safety profile and early efficacy in improving low luminance visual acuity in patients with X-linked retinitis pigmentosa, according to a presentation at the FLORetina-ICOOR meeting.

“These data show promising early improvements in low luminance visual acuity (LLVA) and may suggest modification of the underlying disease pathology,” lead author Paolo E. Stanga, MD, of The Retina Clinic London, told Healio.

In the nonrandomized, open-label, multicenter phase 2 DAWN trial, seven previously treated participants with X-linked retinitis pigmentosa caused by mutations in the RPGR gene received two doses of high-dose laru-zova (AGTC-501, Beacon Therapeutics) (6.8 E+11 vg/eye) in the fellow eye, and three participants received two doses of low-dose laru-zova (3.7 E+11 vg/eye) in the fellow eye. The primary endpoint was the number and proportion of ocular or non-ocular treatment-emergent adverse events or serious adverse events ranked as grade 3 or higher. Secondary endpoints included changes from baseline at 12 months in LLVA, best corrected visual acuity, mean microperimetry, full-field sensitivity threshold, mobility and Michigan Retinal Degeneration Questionnaire.

According to the presentation, laru-zova was well tolerated overall. Ocular treatment-emergent adverse events were mostly non-serious at 3 months and were reported as mild or moderate, with none related to the study agent. There were no reported cases of retinal detachments or endophthalmitis and no study agent-related ocular inflammatory adverse events.

In addition, the study demonstrated improvements at 3 months in mean LLVA as shown by ETDRS letters.

The data suggest that a higher low luminance deficit at baseline may be a predictor of LLVA response at 3 months, according to the presentation.

“If patients are able to achieve two- to three-line improvement, that would be clinically meaningful and offer hope to patients affected by this devastating eye disease with no current treatment options,” Stanga told Healio.

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Sources/Disclosures

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Source:

Stanga PE, et al. Subretinal gene therapy laru-zova (AGTC-501) for X-linked retinitis pigmentosa (XLRP) phase 2
multicenter study (DAWN): Preliminary results. Presented at: FLORetina-ICOOR; Dec. 5-8, 2024; Florence, Italy.

Disclosures:
Stanga reports having financial disclosures for AbbVie, Annexon, Apellis, Astellas, Beacon Therapeutics, Breye Therapeutics, EyeBio, Genentech, Gyroscope, Horizon, Intalight, Iveric Bio, Janssen, Keeler, LKC, LumiThera, Nanoscope, Norlase, Novartis, Oculis, Opthea, Optos, Quantel, Regenxbio, Roche, Topcon and Zeiss.

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