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Diabetic Macular Edema

Positive results from Phase 2 trial of ABBV-RGX-314 for diabetic retinopathy presented at AAO 2023

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Positive results from the ongoing Phase 2 ALTITUDE trial of ABBV-RGX-314, a potential one-time gene therapy for the treatment of diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME), were reported at AAO 2023.

The trial showed promising outcomes in terms of safety and efficacy, including stability and improvement in DR severity scores and a significant reduction in the long-term risk of vision-threatening events.

The ALTITUDE trial is an open-label, randomized, controlled study evaluating the efficacy, safety, and tolerability of suprachoroidal delivery of ABBV-RGX-314 using the SCS Microinjector. The trial includes patients with moderately severe or severe non-proliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy (PDR).

As of September 25, 2023, ABBV-RGX-314 was well tolerated at both dose levels tested. Seven serious adverse events were reported, none of which were considered related to the drug. Common ocular treatment-emergent adverse events included conjunctival hemorrhage and conjunctival hyperemia. Three patients experienced mild intraocular inflammation, which resolved with topical corticosteroids. Six patients had mild to moderate episcleritis, also resolved with topical corticosteroids.

No cases of chorioretinitis, vasculitis, occlusion, or hypotony were reported. Best Corrected Visual Acuity remained stable throughout the one-year period.

In NPDR patients treated with ABBV-RGX-314 at dose level 2, there was a notable prevention of disease progression as measured by the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (DRSS). Furthermore, dose level 2 demonstrated an 89% reduction in the risk of developing vision-threatening events in these patients.

Patients at dose levels 1 and 2 showed meaningful improvements in disease severity and a reduction in vision-threatening events. Specifically, in dose level 2, patients with baseline NPDR demonstrated:

-100% stable to improved disease severity

-70.8% achieved ≥1 step improvement compared to 25.0% in the control group

-0% worsened ≥2 steps compared to 37.5% in the control group

-4.2% developed vision-threatening events compared to 37.5% in the control group

Read the full press release here.