Opus Genetics announced the first pediatric patient was dosed in its ongoing phase 1/2 clinical trial evaluating OPGx-LCA5, the company's investigational gene therapy for the treatment of Leber congenital amaurosis (LCA). In a press release, Opus said it will share initial data from the pediatric cohort of the phase 1/2  clinical trial by Q3 2025.

Additionally, the company commented on the ongoing trial of OPGx-LCA5 in an adult cohort, stating the drug achieved early clinical proof of concept, demonstrating meaningful visual improvement starting as early as 1 month following treatment in the first 3 adult patients, as was evident in the 6-month data first released in October 2024. Opus said it plans to share the new 12-month data on these 3 adult LCA5 patients at a major medical conference during the second quarter of 2025. An FDA Type D meeting is scheduled in March to discuss the pivotal trial design and endpoints for OPGx-LCA5.

The phase 1/2 clinical trial was designed to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene, according to the press release. It is an open-label trial. Efficacy endpoints include measurement of functional vision using: 1) the Multi-Luminance orientation and Mobility Test (MLoMT); 2) Full-Field Stimulus Testing (FST), which measures the retina's sensitivity to light; and 3) microperimetry, which measures point-wise sensitivity to light. For more information, visit clinicaltrials.gov (NCT05616793).