Opus Genetics Reports Positive Pediatric Data for OPGx-LCA5

Opus Genetics has announced encouraging 3-month results from the pediatric cohort of its ongoing Phase 1/2 clinical trial (OPGx-LCA5-1001), evaluating OPGx-LCA5, an investigational gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5). The therapy demonstrated functional visual improvements across several key measures, reinforcing its potential to provide durable benefit in both pediatric and adult patients affected by this ultra-rare inherited retinal disease.

Clinical Trial Overview and Pediatric Cohort Outcomes

The trial enrolled three pediatric participants aged 16–17 years, each with severe baseline visual impairment, who received a single subretinal injection of OPGx-LCA5. Across all three individuals, early visual improvements were observed in multiple functional assessments:

• Visual Acuity (VA): The pediatric cohort showed a group average improvement of 0.3 logMAR, which exceeded the visual gains observed in adult participants at the same time point.

• Full-Field Stimulus Testing (FST): All participants experienced greater than 1 log unit improvement in cone sensitivity to both red and blue light in the treated eyes beginning at 1 month, suggesting recovery in retinal sensitivity.

• Multi-Luminance Orientation and Mobility Test (MLoMT): Each participant identified more objects at 3 months compared to baseline, with two out of three showing greater improvement in the treated eye versus the control eye.

• Microperimetry: Although two participants were unable to perform microperimetry due to poor visual acuity and nystagmus, the third participant demonstrated early signs of improved fixation stability, indicating potential functional retinal recovery.

Durability and Safety Profile

In addition to the pediatric results, combined data from adult participants in the trial support sustained improvements in visual acuity through 18 months, both in mean change from baseline and interocular difference, highlighting the potential durability of the treatment.

To date, six participants (three adults, three pediatrics) have received OPGx-LCA5, and the treatment has been well-tolerated:

• No dose-limiting toxicities

• No ocular serious adverse events

• All ocular adverse events were mild and expected

• One pediatric participant had a pre-existing cataract that worsened following surgery, but it did not affect observed improvements in retinal sensitivity.

Expert and Leadership Commentary

George Magrath, MD, CEO of Opus Genetics, stated:

“These pediatric results are particularly exciting, as they provide evidence that OPGx-LCA5 can potentially restore cone-mediated vision in teenagers who had already experienced profound vision loss. These outcomes, alongside observed durable improvements in adults out to 18 months, give us confidence in the potential for OPGx-LCA5 to deliver meaningful and lasting benefit to patients. We expect to meet with the US FDA in the fourth quarter of this year to discuss these results and the next steps for our LCA5 program.”

Tomas S. Aleman, MD, Principal Investigator of the study and retina specialist at the Scheie Eye Institute, University of Pennsylvania, added:

“Seeing pediatric participants achieve measurable improvements in visual acuity, retinal sensitivity, and real-world navigation tasks within three months and adult participants maintaining those improvements is a remarkable step forward. This is important evidence supporting that gene augmentation therapy can potentially restore cone function in patients with LCA5.”