Gene therapy improves diabetic retinopathy severity at 1 year in phase 2 trial
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SAN FRANCISCO — A one-time in-office injection of a gene therapy helped improve disease severity in patients with diabetic retinopathy.
At Retina Subspecialty Day at the American Academy of Ophthalmology meeting, Mark R. Barakat, MD, said diabetic retinopathy is often left untreated due to a high treatment burden.
“The goal of a one-time in-office injection of gene therapy is to stabilize the disease severity, perhaps improve it and reduce the number of eyes that proceed to vision-threatening complications,” he said.
In the phase 2 ALTITUDE trial, patients with moderately severe nonproliferative diabetic retinopathy (NPDR), severe NPDR or mild proliferative diabetic retinopathy without diabetic macular edema received ABBV-RGX-314 (Regenxbio). Patients in cohort 1 received ABBV-RGX-314 at a dose of 2.5 × 1011 genomic copies per eye (dose level 1) (15 patients), while patients in cohorts 2 and 3 received ABBV-RGX-314 at a dose of 5 × 1011 genomic copies per eye (dose level 2) (35 patients). Cohorts 1 and 2 also included five observational controls.
Barakat said ABBV-RGX-314 was well tolerated at 1 year with seven cases of severe adverse events, but none were considered drug related.
In the observational controls, Barakat said most patients remained stable or experienced a worsening of Diabetic Retinopathy Severity Scale (DRSS). In patients who received dose level 1 of ABBV-RGX-314, about 26% experienced improvement in DRSS. In patients who received dose level 2, about 54% experienced improvement.
The improvement was more pronounced in patients with NPDR only. In dose level 1, about 66% of patients experienced improvement in DRSS, and in dose level 2, about 70% experienced improvement.
“Dose level 2, with baseline NPDR, 100% of patients showed stable to improved disease severity,” Barakat said. “Only 4.2% developed [vision-threatening events] vs. 37% in control, which represents an 89% reduction in these events.”
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Barakat MR. Suprachoroidal delivery of investigational ABBV-RGX-314 for diabetic retinopathy: The phase 2 ALTITUDE study dose levels 1 and 2: One-year results. Presented at: American Academy of Ophthalmology meeting; Nov. 3-6, 2023; San Francisco.
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