Phase I Trial Dose Expansion and Status of Expected Phase II Trial
REGENXBIO recently announced it has completed dosing (six subjects) in the Phase I clinical trial ofRGX-314 at the 1.6 x 10^11 GC/eye (Cohort 4) dose. A total of 24 subjects have now been dosed in the trial. REGENXBIO plans to initiate a Phase II clinical trial forRGX-314 in 2019. Final determination of the study design is under way. REGENXBIO expects to provide further information regarding the initial assessments of protein expression levels from Cohort 4, anticipated Phase II clinical trial design and overall plans for theRGX-314 clinical program in early 2019.
“We continue to be encouraged by theRGX-314 interim results and the potential of NAV® gene therapy as aone-time treatment for wet AMD, a leading cause of irreversible blindness and visual impairment in the world,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We extend our thanks to the patients and the investigators who have participated in this trial and contributed to the growing body of evidence to give hope to patients living with wet AMD for a long-lasting solution to their condition.”
Seven leading retinal surgery centers across the United States are participating in the Phase I clinical trial ofRGX-314. This multi-center, open-label, multiple-cohort, dose-escalation clinical trial is designed to assess the safety and tolerability ofRGX-314 as aone-time therapy for patients with previously treated wet AMD. For further details on the trial, enrollment criteria and eligibility, visit clinicaltrials.gov/ct2/show/NCT03066258.
About the Phase I Clinical Trial ofRGX-314
RGX-314 is currently being evaluated in a Phase I, multi-center, open-label, multiple-cohort,dose-escalation study in adult subjects with wet AMD in the United States. The study has enrolled 24 previously treated wet AMD subjects across four cohorts that are responsive to anti-VEGF therapy and are 50 years of age or older. The study is designed to evaluate four doses ofRGX-314 (3 x 10^9 GC/eye, 1 x 10^10 GC/eye, 6 x 10^10 GC/eye, and 1.6 x 10^11 GC/eye). The primary purpose of the clinical study is to evaluate the safety and tolerability ofRGX-314 at 24 weeks after a single dose administered by subretinal delivery. Primary endpoints include safety and tolerability and secondary endpoints include ocular examinations, visual acuity, imaging (including spectral domain optical coherence tomography(SD-OCT)) and the need for additional anti-VEGF therapy. Following completion of the primary study period, subjects will enter afollow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect.
AboutRGX-314
RGX-314 is being developed as aone-time subretinal treatment for wet AMD. It includes the NAV AAV8 vector encoding an antibody fragment which inhibits VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss. 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 ofRGX-314.
About Wet AMD
Wet AMD is characterized by loss of vision due to new leaky blood vessel formation in the retina. This results in fluid leakage that can manifest in physical changes in the structure of the retina and loss of vision. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan. There may be more than 2 million people 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 improve vision and retinal fluid in the majority of patients. These therapies, however, require repetitive and inconvenient intraocular injections, typically ranging from every four to eight weeks in frequency, to maintain efficacy. Patients often experience a decline in the initial vision gain from therapy with reduced frequency of treatment over time.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
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