The latest data from the ongoing PERCEIVE study seems to be consistent with the findings of clinical trials of voretigene neparvovec (VN), the first approved ocular gene therapy for patients with RPE65-mediated inherited retinal dystrophy and sufficient viable retinal cells.
“Based on this year 2 interim analysis, the PERCEIVE study demonstrates the safety and effectiveness of VN, which is consistent with those observed in the pivotal VN clinical trials,” reported Prof M. Dominik Fischer MD, FEBO, Oxford Eye Hospital, UK.
Biallelic RPE65 mutation-associated IRDs result in progressive vision loss, ultimately leading to complete blindness, explained Prof Fischer. Voretigene neparvovec is a recombinant adeno-associated virus type 2 vector containing human RPE65 complementary DNA that expresses the RPE65 protein. The therapy is currently being tested in an ongoing, prospective, longitudinal, multicentre study that began in December 2019 with a 5-year enrolment period and is planned for completion by December 2029.
At the 2-year cut-off point, 106 patients were enrolled, of whom 103 had received VN. At least 1 ocular adverse event (AE) was reported in 35 patients (34.0%). The most common ocular AE was chorioretinal atrophy, while other reported ocular AEs included 4 cases of foveal degeneration, vitritis (4), eye inflammation (3), retinal tears (2), and increased intraocular pressure (5).
Prof Fischer noted that visual function has not been affected thus far in patients in which chorioretinal atrophy was reported as an AE following VN administration.
“As the study progresses, such AEs and the long-term safety profile of VN will be better characterized. Furthermore, PERCEIVE will provide real-world evidence on the long-term durability of vision improvements observed with VN therapy,” he concluded.
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