Opus Genetics reports pediatric data from OPGx-LCA5 Phase 1/2 trial

Opus Genetics (IRD) announced three-month data from the pediatric cohort of its ongoing Phase 1/2 clinical trial evaluating OPGx-LCA5, an investigational gene augmentation therapy for Leber congenital amaurosis type 5. Three pediatric participants aged 16-17 with severe baseline vision impairment received a single subretinal injection of OPGx-LCA5. All three participants had improvements across multiple measures of visual function. Visual Acuity: For the pediatric cohort, early data showed a group average of a 0.3 logMAR improvement which is greater than was observed in the adult cohort. Participant 01-05 had a baseline visual acuity of 2.2 logMAR with an improvement of 0.5 logMAR reported at one month. Participant 01-06 had a baseline visual acuity of 0.96 logMAR with an improvement of 0.2 logMAR reported at three months. They reported perceiving a clear difference in brightness between their treated and untreated eyes. Participant 01-07 had a baseline visual acuity of 2.3 logMAR with an improvement of 0.7 logMAR reported at one month, which was maintained through three months. Full-Field Stimulus Testing: All three participants showed improvements in the treated eyes from one month. Participants showed greater than one log unit improvement in cone sensitivity to both red and blue light. These changes provide evidence of recovery in retinal sensitivity. Multi-Luminance Orientation and Mobility Test: All participants identified more objects through three-months compared to baseline. Two out of the three participants had greater improvement in the treated eye compared to the control eye. Microperimetry: Two of the three pediatric participants could not conduct a microperimetry test due to their poor visual acuity and nystagmus at screening. However, microperimetry data was obtained on one participant, for whom early signs of improved fixation stability were observed, consistent with functional retinal recovery. In addition, combined adult data support that improvements in visual acuity were sustained through 18 months, both in terms of mean change from baseline and mean interocular difference, underscoring the potential durability of the treatment response. OPGx-LCA5 has been well-tolerated in all six participants treated to date. No ocular serious adverse events or dose-limiting toxicities have been observed. All ocular adverse events were mild in severity and were anticipated. No events were related to the study drug. One pediatric participant had a pre-existing cataract that worsened at three months, which was attributed to the surgical procedure itself and did not obscure improvements in retinal sensitivity.