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The following is a summary of “Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy Secondary to Age-Related Macular Degeneration,” published in the December 2024 issue of Ophthalmology by Heier et al. 

Researchers conducted a retrospective study to assess the safety and tolerability of a single intravitreal injection of JNJ-81201887 (JNJ-1887) in individuals with geographic atrophy (GA) secondary to advanced dry age-related macular degeneration (AMD).  

They included adult patients (≥50 years old) with GA secondary to AMD in the treated eye, having Snellen best-corrected visual acuity of 20/200 or worse (20/80 or worse after the first 3 participants), GA lesion size between 5 and 20 mm² (2–8 disc area), and best-corrected visual acuity of 20/800 or better in the fellow, untreated eye and 17 patients were sequentially enrolled into 3 cohorts: low-dose (3.56 × 10¹⁰ viral genome/eye; n = 3), intermediate-dose (1.07 × 10¹¹ viral genome/eye; n = 3), and high-dose (3.56 × 10¹¹ viral genome/eye; n = 11), without steroid prophylaxis, were assessed for safety and tolerability over 24 months.  

The results showed that baseline characteristics aligned with the disease, and all participants had foveal center–involved GA, JNJ-1887 was well-tolerated across cohorts, with no dose-limiting adverse events (AEs). No serious or systemic AEs related to the intervention in the study occurred, 5 of 17 participants (29%) experienced 5 mild ocular inflammation events, all of which resolved, with 4 of 5 patients improving after topical steroids or observation, 1 unresolved vitritis event, managed with observation, occurred in a participant with an unrelated fatal AE. No cases of endophthalmitis or new-onset choroidal neovascularization were reported, and GA lesion growth rate was similar across cohorts over 24 months. In the high-dose, treated eyes showed a continued decline in GA lesion growth, with a reduction in mean square root lesion growth from 0.211 mm (months 0–6) to 0.056 mm (months 18–24).  

Investigators concluded that all the 3 doses of JNJ-1887 demonstrated a manageable safety profile over 24 months, warranting further investigation for treating GA. 

Source: sciencedirect.com/science/article/pii/S0161642024003683