Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program.
North Carolina-based Opus is developing the gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5) patients. The candidate uses an AAV8 vector and the same promoter technology as Roche’s Luxturna to deliver a functional LCA5 gene. Opus’ goal is to address the photoreceptor impairment that causes vision loss in patients with LCA5, a condition that it says affects around 200 people in the U.S.
Having reported data on three adults with LCA5 last year, Opus began enrolling adolescents with the rare disease to kick off its expansion into the pediatric population. The first three patients were aged 16 to 17 and had severe vision impairment at baseline.
As of the data cutoff, Opus reported an average of a 0.3 logMAR improvement across the cohort. Each 0.1 logMAR shift indicates a one line change on an eye test chart. Two patients had improvements of 0.2 and 0.7 logMAR three months after treatment, with the smallest improvement seen in the patient with the best vision at baseline. The third patient had an improvement of 0.5 logMAR after one month.
Opus compared the data favorably to the results seen in the first three adults to receive the therapy. The improvement in adults increased in the early part of the study, climbing (PDF) to a 0.48 logMAR change by the sixth month of the trial. Opus said the combined adult data support the durability of the vision improvements through 18 months.
Shares were up more than 3% premarket on the data drop Tuesday morning.
The top-line pediatric safety data shared by Opus largely support the encouraging signs the company reported in adults. No events were related to the study drug. One pediatric participant had a preexisting cataract that worsened at three months. Opus said the worsening was attributed to the surgical procedure used to deliver the gene therapy and didn’t obscure improvements in retinal sensitivity.
Opus plans to meet with the FDA in the fourth quarter to discuss the next steps for the program. Before sharing the pediatric data, the company said an accelerated clinical development pathway to a filing for FDA approval could be acceptable if the efficacy seen in children is similar to the results in adults.