Opus Genetics reports 1-month pediatric results in gene therapy for inherited retinal disease

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Opus Genetics announced the 1-month open-label phase 1/2 results from the first pediatric patients treated with its investigational gene therapy candidate, OPGx-LCA5, for Leber congenital amaurosis (LCA) 5-related inherited retinal disease (IRD). IRD is a rare and severe genetic condition that causes early-onset vision loss.¹

The new data builds upon previously reported 6- and 12-month positive results from adult patients treated in the same study.

The company stated the importance of the results:

• LCA5-related IRD has no approved treatments.
• Gene therapy offers the potential to restore vision early, before irreversible retinal damage occurs.
• This is part of a growing effort to bring gene therapy into pediatric care, where early intervention may have a profound impact on healthspan and quality of life.

The company described that their early data are “meaningful: the 16-year-old participant showed clinically significant improvement in visual function after just 1 month, reporting brighter vision and new independence in mobility and reading, without any drug-related adverse events.”

The patient received one subretinal injection of OPGx-LCA5 and clinically meaningful improvement in vision was observed at the 1-month time point. No drug-related adverse events were reported.

A second pediatric patient was recently dosed, and the pediatric cohort is expected to complete enrollment in the second quarter of 2025, according to the press release. The initial data from all 3 patients is anticipated in the third quarter of 2025.

These results build on 6- and 12-month data in adults, where sustained improvements in visual function have been observed. A potential registrational trial could begin as early as the first quarter of 2026, following a productive Type D meeting with the US Food and Drug Administration.

“The preliminary results observed in our pediatric patient are encouraging and are consistent with the improvements we previously observed in adults," Tomas Aleman, MD, Scheie Eye Institute Perelman, the study principal investigator, said.

"[The patient] noticed that objects were significantly brighter and was able to distinguish letters and navigate with an independence she had never had before, after only 1-month following treatment. We’re excited to continue enrolling patients and studying improvement over longer periods of time in this important study.”

George Magrath, MD, chief executive officer of Opus Genetics, added, “We believe these findings provided further evidence supporting the potential of OPGx-LCA5 to restore meaningful vision in patients affected by LCA5. The meeting with the FDA regarding the design of our program may lead to the start of a trial in 2026. Early intervention may be particularly beneficial in pediatric patients, given the progressive nature of this disease.”

The 1-year results are going to be presented at the annual meeting of the Association for Research in Vision and Ophthalmology on May 4, 2025.

Next steps for OPGx-LCA5

An FDA Type D meeting discussed the potential regulatory path for OPGx LCA5, including the design of a potential registrational study. Opus proposed a single arm, adaptive pivotal study, to enroll as few as 19 patients, with a primary endpoint utilizing the multi-luminance orientation and mobility test (MLoMT), which is a functional vision and patient mobility test. MLoMT is a virtual-reality version of the multi-luminance mobility test, which provided evidence to support a prior FDA approval.

The company also received constructive feedback on its proposed statistical analysis plan as well as chemistry, manufacturing, and controls (CMC). The FDA requested additional information on these topics, and Opus plans to submit further materials and continue discussions with the FDA.

Opus anticipates the pivotal trial could initiate in Q1 2026, according to the press release.

Reference:

1. Opus Genetics, Inc. Opus Genetics, Inc. Published April 8, 2025. Accessed April 8, 2025. https://ir.opusgtx.com/press-releases/detail/483/opus-genetics-announces-one-month-clinical-data-from-pediatric-patient-in-phase-12-trial-of-opgx-lca5-gene-therapy-in-inherited-retinal-diseases