REGENXBIO Announces Additional Positive Interim Phase I/IIa and Long-Term Follow-Up Data of RGX-314 for the Treatment of Wet AMD
- RGX-314 using subretinal delivery continues to be generally well-tolerated at all dose levels
- Positive interim update from Cohorts 4 and 5 at 1.5 years after RGX-314 administration
- Durable treatment effect observed with stable visual acuity, decreased retinal thickness, and reductions in anti-VEGF injection burden
- Long-term, durable treatment effect over three years demonstrated in Cohort 3
- Mean improvement in vision and stable retinal thickness
- 50% of patients (3/6) remain anti-VEGF injection-free over three years; 67% of patients (4/6) are anti-VEGF injection-free from nine months to three years
- ATMOSPHERE™, the first of two planned pivotal trials for RGX-314, is active and enrolling
"The continued durability of treatment effect up to three years after RGX-314 administration highlights the potential of RGX-314 as a one-time treatment option for patients with wet AMD. The results from the Phase I/IIa trial of RGX-314 using subretinal delivery have informed the key design elements of our pivotal program, in which we plan to conduct two randomized, well-controlled clinical trials, enrolling approximately 700 patients total," said
"I am excited about this data out to three years, which demonstrates that one-time treatment with RGX-314 has the potential to result in long-term stability to improvement of visual acuity outcomes and retinal anatomy, while alleviating treatment burden," said
Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD Using Subretinal Delivery
In the Phase I/IIa trial of RGX-314, 42 patients with severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3x109 GC/eye to 2.5x1011 GC/eye.
Summary of Data for Cohorts 4 and 5
Today's update includes data from Cohorts 4 and 5 as of
Patients in Cohorts 4 and 5 at 1.5 years after administration of RGX-314 demonstrated stable visual acuity with a mean Best Corrected Visual Acuity (BCVA) change of +1 letters and -1 letters from baseline, respectively, as well as decreased central retinal thickness (CRT), with a mean change of -46 µm and -93 µm, respectively.
There was a meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the mean annualized injection rate during the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.4 injections over 1.5 years following administration of RGX-314, a 58.3% reduction in anti-VEGF treatment burden. Patients in Cohort 5 received a mean of 1.7 injections over 1.5 years following administration of RGX-314, a reduction in anti-VEGF treatment burden of 81.2%.
In Cohort 4, four out of 12 (33%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of +2 letters at 1.5 years. Eight out of 11 (73%) patients have received no anti-VEGF injections after six months following RGX-314 administration and demonstrated a mean BCVA change from baseline of -2 letters at 1.5 years.
Summary of Long-Term Follow-Up (LTFU) Study Data
Following the Phase I/IIa trial, patients are encouraged to enroll in a LTFU study to assess safety and efficacy up to five years after RGX-314 administration. Patients in the LTFU study have scheduled visits every six months for the first year and then annual visits until the end of the study. Patient management is per physician discretion. Data collected during the scheduled study visits include safety, BCVA, and CRT. In addition, chart reviews are conducted at each scheduled study visit to collect the number of retina specialist visits and anti-VEGF injections each patient has received since the prior scheduled study visit.
All six patients from Cohort 3 of the Phase I/IIa trial enrolled in the LTFU study, and long-term treatment effect was demonstrated over three years. These patients demonstrated a mean BCVA improvement of +12 letters from baseline at three years. Retinal anatomy as measured by machine-read CRT remained stable at three years compared to the two-year timepoint.
Patients also demonstrated long-term reductions in anti-VEGF treatment burden over three years with a mean annualized rate of 2.4 anti-VEGF injections after administration of RGX-314, which is a reduction of 66.7% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Three out of six (50%) patients received no anti-VEGF injections over three years following one-time administration of RGX-314. Four out of six (67%) patients have received no anti-VEGF injections from nine months to three years after RGX-314 administration. The four patients who did not receive anti-VEGF injections after nine months demonstrated a mean BCVA improvement from baseline of +11 letters at three years.
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
RGX-314 is being developed 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 the Phase I/IIa Clinical Trial of RGX-314 and Long-Term Follow-Up Study
RGX-314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose-escalation study in adult patients with wet AMD in
ATMOSPHERE is a multi-center, randomized, active-controlled trial to evaluate the efficacy and safety of a single-administration of RGX-314 versus standard of care in patients with wet AMD. The trial is designed to enroll 300 patients at a 1:1:1 ratio across two RGX-314 dose arms (6.4x1010 genome copies (GC)/eye and 1.3x1011 GC/eye delivered subretinally) and an active control arm of monthly intravitreal injections of ranibizumab (0.5 mg/eye). The primary endpoint of the trial is non-inferiority to ranibizumab based on change from baseline in Best Corrected Visual Acuity (BCVA) at 54 weeks. Secondary endpoints of the trial include safety and tolerability, change in central retinal thickness (CRT) and need for supplemental anti-VEGF injections. Patient selection criteria will include patients with wet AMD who are responsive to anti-VEGF treatment and will be independent of preexisting neutralizing antibody status. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314. The trial will be conducted at approximately 60 clinical sites based in
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