REGENXBIO Announces Additional Positive Interim Data from Trials of RGX-314 for the Treatment of Wet AMD
- Company announced positive interim data from and the expansion of Phase II AAVIATE® trial of RGX-314 for the treatment of wet AMD using suprachoroidal delivery
- RGX-314 continues to be well tolerated in 85 patients from Cohorts 1-5 with no drug-related serious adverse events
- Meaningful reduction in treatment burden at six months across all dose levels, 85% reduction in treatment burden observed at third dose level
- 67% of patients in Cohort 4 were injection-free
- No meaningful differences in outcomes at six months for patients who are NAb positive
- Phase II trial expanded to include new cohort at third dose level with short-course prophylactic ocular steroids following RGX-314 administration
- Positive interim data presented at the AAO Annual Meeting from the Phase I/IIa Long-term Follow-up study of RGX-314 for the treatment of wet AMD using subretinal delivery
- RGX-314 continues to be well-tolerated and demonstrates long-term, durable treatment effect up to four years
- Two pivotal trials, ATMOSPHERE® and ASCENT™, are active and enrolling patients
- Pivotal trials are expected to support BLA submission in 2024
- New interim data from Phase II ALTITUDE® trial of RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery expected at the
Retina Society55th Annual Scientific Meeting in November
- Conference call
Monday, October 3at 8:30 a.m. ET
"Today's announcements come nearly a year into our collaboration with AbbVie to advance RGX-314 in wet AMD and other retinal diseases. These new subretinal and suprachoroidal data highlight the potential impact of RGX-314 for the millions of patients facing vision loss from wet AMD," said
Data Summary and Safety Update for the Phase II AAVIATE Trial of RGX-314 using Suprachoroidal Delivery
The Phase II AAVIATE trial of RGX-314 for the treatment of wet AMD using in-office suprachoroidal delivery continues to show positive interim results. AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation trial that is evaluating the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314. The primary endpoint of the trial is mean change in vision in patients dosed with RGX-314, as measured by best corrected visual acuity (BCVA) at Week 40 from baseline, compared to patients receiving monthly injections of ranibizumab. Other endpoints include mean change in central retinal thickness (CRT) and number of anti-vascular endothelial growth factor (anti-VEGF) intravitreal injections received following administration of RGX-314.
Patients treated in the RGX-314 arms and the ranibizumab control arm both continue to demonstrate stable BCVA and CRT at 6 months. In addition, a meaningful reduction in anti-VEGF treatment burden following administration of RGX-314 compared to mean annualized injection rate during the 12 months prior to administration was observed and ranged from -63.8% to -84.7% across all cohorts. The highest reduction in treatment burden was observed in the third dose level, with patients receiving a mean of 1.3 injections over six months following administration of RGX-314, which represents an 84.7% reduction in anti-VEGF treatment burden. Ten out of 15 patients (67%) in the third dose level received no anti-VEGF injections over six months following RGX-314 administration. In these patients, visual acuity and CRT was observed to be stable over six months.
Additionally, the interim data from the second dose level (Cohorts 2 and 3) suggests there is no meaningful difference in safety and vision outcomes for patients who are neutralizing antibody (NAb) positive.
Phase II AAVIATE Trial Expansion to Cohort 6
"We are pleased to share updated data from the AAVIATE trial, including new 6-month data from Cohorts 1-4 which provides continued evidence of the emerging clinical profile of RGX-314 for the treatment of wet AMD using suprachoroidal delivery," said
Data Summary from Phase I/IIa Long-term Follow-up Study of RGX-314 using Subretinal Delivery
The Phase I/IIa study evaluated RGX-314 in 42 patients with wet AMD using subretinal delivery and was designed as an open label, dose escalation study evaluating five doses of RGX-314 over two years. Dose dependent increases in treatment effect were observed, and doses similar to those used in Cohort 3 and Cohort 4 of the Phase I/IIa trial were advanced into the ongoing pivotal trials, ATMOSPHERE and ASCENT. After the Phase I/IIa study's completion, patients were encouraged to enroll into a long-term follow-up study for up to five years after RGX-314 administration. Data presented at AAO highlights the results of the 16 (out of 18) patients from Cohorts 3 and 4 who enrolled in the LTFU study, with data now out to 4 years and 3 years, respectively. Data from this study demonstrating that RGX-314 continues to be well-tolerated with long-term, durable treatment effect up to four years is expected to support the anticipated BLA filing for RGX-314 in 2024.
Patients treated with RGX-314 continue to demonstrate a long-term, durable treatment effect in Cohort 3 up to 4 years and Cohort 4 up to three years. Stable to improved visual acuity was observed, with a mean BCVA of +12 letters from baseline at four years for Cohort 3 patients and -5 letters from baseline at three years for Cohort 4 patients following RGX-314 administration.
Patients also demonstrated meaningful long-term reductions in anti-VEGF treatment burden following administration of RGX-314. Patients in Cohort 3 received a mean annualized rate of 2.4 injections through 4 years following administration of RGX-314 (versus 6.8 injections in the 12 months prior to treatment), representing a 67.0% reduction in mean annualized injection rate. Patients in Cohort 4 received a mean annualized rate of 4.4 injections through 3 years following administration of RGX-314 (versus 10.2 injections in the 12 months prior to treatment), representing a 58.4% reduction in mean annualized injection rate.
"These positive interim data from the long-term follow-up and AAVIATE trials continue to reinforce the potential clinical benefit of a one-time administration of RGX-314 in the overall management of patients with neovascular AMD," said
These study findings are available under the Presentations & Publications page in the Media section of
In connection with this announcement,
About the AAVIATE® Trial
The multi-center, open-label, randomized, active-controlled, dose-escalation Phase II AAVIATE trial is evaluating the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 in patients with wet AMD using the Clearside 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) through one injection 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 NAb positive. Cohort 4 is evaluating RGX-314 in 15 patients at a dose level of 1x1012 GC/eye and Cohort 5 is evaluating the same dose level of RGX-314 in 20 patients who are NAb positive. Cohort 6 is evaluating patients at the same dose level as Cohorts 4 and 5 and includes a short course of prophylactic steroids following administration of RGX-314.
RGX-314, being developed in collaboration with AbbVie, 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 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,
SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.
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