REGENXBIO Presents Additional Positive Interim Data from Trials of RGX-314 in Wet AMD and Diabetic Retinopathy Using Suprachoroidal Delivery at AAO 2021
- Suprachoroidal delivery of RGX-314 in Phase II AAVIATE® trial for the treatment of wet AMD continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events
- Positive initial data from Cohort 2 in AAVIATE trial at six months after one-time treatment of RGX-314
- Treatment effect observed with stable visual acuity and retinal thickness
- Demonstrated meaningful reduction (>70%) in anti-VEGF treatment burden; 40% of patients in Cohort 2 were anti-VEGF injection-free
- Additional data from Cohort 1 in Phase II ALTITUDE™ trial for the treatment of DR demonstrated stable visual acuity at three months after one-time treatment of RGX-314
"We are pleased to share this initial data from Cohort 2 of the AAVIATE trial which provides encouraging evidence of the emerging clinical profile of RGX-314 for the treatment of wet AMD using suprachoroidal delivery. In the data reported today, RGX-314 was observed to be well tolerated in Cohort 2, with stable visual acuity and retinal thickness as well as a meaningful reduction in anti-VEGF treatment burden at six months," said
"These initial results from patients in Cohort 2 of the AAVIATE trial at six months after suprachoroidal administration of RGX-314 reinforce the potential impact that RGX-314 could have on the overall clinical management of patients with wet AMD," said
Study Design and Safety Update in Phase II AAVIATE Trial of RGX-314 for the Treatment of Wet AMD Using Suprachoroidal Delivery
AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector®. Twenty patients in Cohort 1 were randomized to receive RGX-314 at a dose level of 2.5x1011 genomic copies per eye (GC/eye) versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Twenty patients in Cohort 2 were randomized to receive RGX-314 at a dose level of 5x1011 GC/eye through two injections versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Cohort 3 is evaluating RGX-314 at the same dose level as Cohort 2 in 20 patients who are neutralizing antibody (NAb) positive. Enrollment is ongoing in two additional cohorts (Cohorts 4 and 5) to evaluate RGX-314 at a third dose level of 1x1012 GC/eye. Cohort 4 will enroll 15 patients who will be dosed with RGX-314 and Cohort 5 will evaluate the same dose level of RGX-314 in 20 patients who are NAb positive. Patients do not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.
Summary of Data for Cohort 2 in Phase II AAVIATE Trial of RGX-314 for the Treatment of Wet AMD at Six Months
In Cohort 2, patients dosed with RGX-314 demonstrated stable Best Corrected Visual Acuity (BCVA) and central retinal thickness (CRT) at 6 months. Fifteen patients in Cohort 2 dosed with RGX-314 had a mean BCVA change of -0.1 letters (95% Confidence Interval: -3.8, 3.6) when measured from Day 1 (at Screening) and +0.2 letters (-2.7, 3.1) when measured from Week 1 (prior to Randomization). These patients also demonstrated stable central retinal thickness (CRT), with a mean change of -33 µm (-71, 5) at six months from Day 1. Ten control patients receiving monthly injections of ranibizumab in Cohorts 1 and 2 had a mean BCVA change at six months of +4.0 letters (-0.5, 8.5) when measured from Day 1 and +1.3 letters (-2.2, 4.8) when measured from Week 1. Patients receiving monthly injections of ranibizumab had a mean change of CRT of -12 µm (-33, 8) at six months from Day 1.
There was a meaningful reduction in anti-vascular endothelial growth factor (anti-VEGF) treatment burden in patients following administration of RGX-314 compared to the mean annualized injection rate during the 12 months prior to administration. Patients in Cohort 2 received a mean of 1.3 injections over six months following administration of RGX-314, which represents a 71.8% reduction in anti-VEGF treatment burden. Six out of 15 patients (40%) in Cohort 2 received no anti-VEGF injections over six months following RGX-314 administration. In these patients, visual acuity and CRT was observed to be stable from Day 1 over six months, with a mean change of BCVA of +1.0 letters (-3.8, 5.8), and a mean change of CRT of +8 µm (-9.2, 24.2).
Summary of Visual Acuity Data for Cohort 1 in Phase II ALTITUDE Trial of RGX-314 for the Treatment of Diabetic Retinopathy at Three Months
Data presented today are available on the "Presentations and Publications" section of the
RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
About Wet AMD
Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in
About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide. DR affects approximately eight million people in
About REGENXBIO Inc.
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things,
1 One patient in Cohort 1 discontinued the study after Week 12 as a result of death, which was assessed to be unrelated to RGX-314. At the time of the death, the subject was free of anti-VEGF injections.
SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of
Investor Relations and Corporate Communications
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-presents-additional-positive-interim-data-from-trials-of-rgx-314-in-wet-amd-and-diabetic-retinopathy-using-suprachoroidal-delivery-at-aao-2021-301423126.html