ABBV-RGX-314 : REGENXBIO reports positive Phase 2 data on diabetic retinopathy gene therapy candidate

REGENXBIO Inc. has disclosed new findings from its Phase II ALTITUDE trial, focusing on ABBV-RGX-314 as a gene therapy for diabetic retinopathy (DR) treatment. This announcement comes ahead of a detailed presentation set for the American Academy of Ophthalmology meeting by Dr. Mark Barakat of Retinal Consultants of Arizona.

ALTITUDE Trial and Diabetic Retinopathy:

The ALTITUDE trial, a comprehensive study evaluating the safety and efficacy of ABBV-RGX-314, employs suprachoroidal delivery using the SCS Microinjector. Patients with non-proliferative diabetic retinopathy (NPDR) and mild proliferative diabetic retinopathy (PDR) received varied doses of the treatment, showing the gene therapy’s potential to address the unmet need for lasting DR treatments.

Trial Results and Safety Profile:

As reported, ABBV-RGX-314 was well tolerated at dose levels 1 and 2, with seven serious adverse events none of which were drug-related. The one-year data highlighted no significant adverse events like chorioretinitis, vasculitis, or hypotony, emphasizing the treatment’s safety.

Impact on Disease Progression:

The data indicated that dose level 2 of ABBV-RGX-314 has a notable impact on preventing disease progression and reducing vision-threatening events by 89% in NPDR patients, marking a considerable advance in the management of this condition.

“We are pleased that ABBV-RGX-314 at dose level 2 continues to be well tolerated and demonstrate clinically meaningful improvements for patients with non-proliferative DR,” commented Dr. Steve Pakola, Chief Medical Officer of REGENXBIO.

Dr. Barakat also noted, “DR is a leading cause of vision loss in working-age adults and is a global public health concern… There is an unmet need for sustainable treatment options that prevent the progression of early diabetic retinopathy to proliferative diabetic retinopathy and development of vision-threatening complications over the long term.”

Looking Forward:

With these promising results, REGENXBIO Inc., in collaboration with AbbVie, continues to investigate ABBV-RGX-314, which utilizes the NAV AAV8 vector to inhibit VEGF, for the treatment of wet age-related macular degeneration, DR, and other chronic retinal diseases. The medical community eagerly awaits additional findings from the ongoing ALTITUDE trial, with hopes of establishing a new standard in DR treatment.