Apr 30, 2019

REGENXBIO’s Gene Therapy for Wet AMD Performing Encouragingly in Human Study

Eye On the Cure Research News

Unlike current treatments requiring multiple injections, REGENXBIO’s gene therapy is administered as a one-time subretinal injection to the affected eye.

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Though there are FDA-approved treatments for wet age-related macular degeneration (AMD) such as Lucentis™ (ranibizumab) and Eylea® (aflibercept), these therapies, known as anti-VEGFs, need to be injected into the eye at a doctor’s office for the rest of the patient’s life. In some cases, the injections are given monthly. In other cases, the anti-VEGFs are administered on an as needed basis. Regardless, the never-ending injections are neither pleasant nor convenient for the patient.

REGENXBIO’s emerging gene therapy, RGX-314, is designed to greatly reduce the need for repetitive anti-VEGF injections to stave off the harmful, leaky blood vessels from wet AMD by providing sustained production of a protein similar to ranibizumab. RGX-314 is administered as a one-time subretinal injection to the affected eye.

Allen Ho, MD, a retinal physician and surgeon at Wills Eye Hospital, presented promising interim, six-month results from a Phase 1/2 clinical trial for RGX-314 during the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit on April 26. The summit, hosted by the Foundation Fighting Blindness and Casey Eye Institute, Oregon Health & Science University, preceded the 2019 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) taking place in Vancouver, Canada, April 28–May 2, 2019.

Interim RGX-314 trial results suggested a dose dependent response to the gene therapy. The clinical trial team observed that fewer rescue injections of an anti-VEGF (such as Lucentis or Eylea) were needed for higher doses of RGX-314.

Dr. Ho presented data for 18 patients in three dosing groups. The six trial participants who received the lowest dose of RGX-314 needed an average of 4.7 anti-VEGF rescue injections. The six who received the medium dose of RGX-314 needed an average of 3.8 rescue injections. The six patients who received the highest dose of RGX-314 only needed an average of 1.3 rescue injections.

Dr. Ho noted that 30 patients have now received the treatment in the Phase 1/2 trial and that REGENXBIO is planning a Phase 2b trial for RGX-314 in late 2019.