REGENXBIO Announces IND Active for Phase I Trial

REGENXBIO Inc. recently announced the Investigational New Drug application (IND) is active for the planned multi-center, open-label, multiple-cohort, dose-escalation Phase I clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD).

“The goal of the RGX-314 program is to develop a single-dose treatment for wet AMD that prevents future disease recurrence while reducing or eliminating the need for regular injections that are the current standard of care in wet AMD,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We are on track to meet our next program objectives for RGX-314, beginning with trial enrollment by mid-2017 and an interim trial update by the end of the year, and we look forward to working with leading US researchers and retina surgeons on this novel clinical program.”

RGX-314 is being developed under a multi-institutional collaboration with world-renowned gene therapy and ophthalmology experts James M. Wilson, MD, PhD, Jean Bennett, MD, PhD, and Albert Maguire, MD, from the University of Pennsylvania’s Gene Therapy Program and Center for Advanced Retinal and Ocular Therapeutics (Penn), respectively, and Peter Campochiaro, MD, at the Johns Hopkins Wilmer Eye Institute (Johns Hopkins).

“In animal studies, treatment with RGX-314 gene therapy led to rapid and sustained anti-VEGF protein detected in the eyes of treated animals. Preclinical studies have shown anti-VEGF mRNA and protein distributed widely throughout the retina. This high protein expression observed using RGX-314’s NAV AAV8 vector may make this approach suitable for an ocular therapeutic in wet AMD,” said Dr. Maguire. “Six leading retinal surgery centers across the US, including Penn and Johns Hopkins, are expected to participate in the Phase I trial of RGX-314.”

RGX 314 will be evaluated in a Phase I, multi-center, open-label, multiple-cohort, dose escalation study in adult subjects with wet AMD in the US. The study is expected to include approximately 18 previously treated wet AMD subjects that are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy and are 50 years of age or older. The study is designed to evaluate three doses of RGX-314 (3 × 10^9 genome copies (GC)/eye, 1 × 10^10 GC/eye, and 6 × 10^10 GC/eye). Primary endpoints include adverse events, certain laboratory measures (including immunological parameters) and ocular examinations and imaging (including BCVA and SD OCT). The primary purpose of the clinical study is to evaluate the safety and tolerability of RGX-314 at 24 weeks after a single dose of RGX-314 administered by subretinal delivery. Following completion of the primary study period, it is expected that subjects will enter the follow-up period and will continue to be assessed until week 106 to assess long-term safety and durability of effect.

Wet AMD is characterized by loss of vision due to excess blood vessel formation between two layers of cells in the retina, which results in fluid leakage that can result in physical changes in the structure of the retina and changes in vision. Wet AMD is a leading cause of total and partial vision loss in the US, Europe, and Japan, and there may be over 2 million individuals living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to halt or significantly impede the loss of vision in the majority of patients with wet AMD. All of these therapies, however, require repetitive and inconvenient intraocular injections, typically ranging from every 4 to 8 weeks in frequency, to maintain efficacy. Patients often experience vision loss with reduced frequency of treatment.

RGX-314 is being developed as a novel, one-time subretinal treatment for wet AMD that includes the NAV AAV8 vector encoding a gene for a monoclonal antibody fragment. The expressed protein is designed to neutralize VEGF activity, modifying the pathway for formation of new leaky blood vessels and retinal fluid accumulation. In preclinical animal models with conditions similar to macular degeneration, significant and dose-dependent reduction of blood vessel growth and prevention of disease progression was observed after a single subretinal dose of RGX-314.

REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. REGENXBIO’s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO’s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third-party NAV Technology Platform Licensees. REGENXBIO and its licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. For more information, visit www.regenexbio.com.