Opus Genetics Doses First Pediatric Patient in Phase 1/2 Trial for OPGx-LCA5 Gene Therapy
Opus Genetics announced the first pediatric patient has been dosed in its ongoing phase 1/2 clinical trial evaluating OPGx-LCA5, its investigational gene therapy for Leber congenital amaurosis (LCA). Initial data from the pediatric cohort is expected to be shared by Q3 2025.
According to Opus, the ongoing trial has demonstrated early clinical proof of concept, showing meaningful visual improvement starting as early as 1 month post-treatment in the first three adult patients. The 6-month data, first released in October 2024 and reviewed at a company-sponsored KOL event in December 2024, confirmed these improvements. Opus now plans to present the 12-month data on these adult LCA5 patients at a major medical conference in the second quarter of 2025. Additionally, an FDA Type D meeting is scheduled for March to discuss the pivotal trial design and registrational endpoints.
“We are proud of the progress of our ongoing OPGx-LCA5 clinical trial and pleased to reach this critical next step as we expand the trial to pediatric patients. Early intervention in pediatric LCA5 patients is particularly important, as it offers the best chance to preserve or restore visual function before the disease progresses,” George Magrath, MD, Chief Executive Officer of Opus Genetics, said in a company news release. “We are encouraged by the new 12-month results that confirm the durability of the positive response observed at 6 months. This data will be shared in our scheduled meeting with the FDA in March to discuss a proposed phase 3 trial design. We are hopeful that this may offer a potentially life-changing therapeutic option for individuals living with LCA and will continue to work closely with the medical and patient communities to advance this important program. We’re grateful to our partners at the University of Pennsylvania for their dedication to our program.”
Phase 1/2 Trial Design
This clinical trial evaluates 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. It is an open-label, phase 1/2 trial assessing OPGx-LCA5. Efficacy endpoints include:
Measurement of functional vision using the Multi-Luminance Orientation and Mobility Test (MLoMT)
Full-Field Stimulus Testing (FST) to measure retinal sensitivity to light
Microperimetry to assess point-wise sensitivity to light
For more information, visit clinicaltrials.gov (NCT05616793).