Gene therapy for GA, wet AMD appears safe in pooled analysis
The first aggregate safety data from 2 studies of JNJ-81201887 (JNJ-1887), a single intravitreal gene therapy injection that expresses soluble CD59 in patients with late-stage age-related macular degeneration (AMD), has shown that it was well tolerated with a generally favorable safety profile, and the inflammatory profile was manageable in patients with both geographic atrophy (GA) and wet AMD, according to a poster presented at ARVO 2023.
The 2 studies, Phase 1 trial 1001 and Phase 1 trial 1002, evaluated the safety of JNJ-1887 in patients with GA and wet AMD, respectively. In trial 1001, which was an open-label, single-center study that lasted for 24 months, 17 patients were sequentially enrolled into low, intermediate, or high dose groups without corticosteroid prophylaxis.
In trial 1002, which was an open-label, multicenter study that lasted for 12 months, 25 treatment-naïve patients with wet AMD received an anti-vascular endothelial growth factor (VEGF) injection followed by a single intravitreal injection of JNJ-1887 at doses of either 3.56×1011 vg/eye or 1.071×1012 vg/eye, with a protocol-defined oral corticosteroid prophylaxis regimen.
The patient characteristics at baseline and concomitant medications were consistent with each disease under study. JNJ-1887 was well tolerated across all cohorts in both studies, with no dose-limiting toxicities or serious or systemic adverse events related to study intervention. The inflammatory profile in both studies was manageable, with 5/17 patients experiencing mild ocular inflammation in trial 1001, which resolved in 4 patients following either topical steroids or observation, and 4/25 patients experiencing mild or moderate ocular inflammation in trial 1002, which resolved following a 7-day course of oral steroids and topical steroids of varied duration.
Lad EM, et al. Pooled safety analysis of a single intravitreal injection of JNJ-1887 (gene therapy, AAVCAGsCD59) in patients with age-related macular degeneration (AMD). Presented at: ARVO 2023.
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