Regenxbio gene therapy delivers long-term treatment effect in wet AMD
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According to an ongoing phase 1/2a trial of RGX-314 gene therapy for wet age-related macular degeneration, six participants who have reached 2-year follow-up have experienced long-term, durable treatment effects and improvements in vision.
The trial drug, a potential one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal conditions treated with anti-VEGF, has been well-tolerated at all dose levels through five cohorts of participants, according to a press release from Regenxbio.
“I’m pleased to say the data continues to support what we’ve previously reported for this study. RGX-314 has been well tolerated in 42 patients and has demonstrated long-lasting, durable anti-VEGF effects in the eye over 2 years after a one-time administration in severe wet AMD patients in cohort three,” Ken Mills, president and CEO of Regenxbio, said during a conference call presentation.
The trial includes 42 patients with severe wet AMD who required frequent anti-VEGF injections. The participants were treated across five dose cohorts, with doses ranging from 3 × 109 GC per eye to 2.5 × 1011 GC per eye, and are being assessed each month for 24 months, with safety follow-up for 5 years after RGX-314 administration.
Researchers are evaluating the reduction in anti-VEGF intravitreal injections, change in best corrected visual acuity and central retinal thickness, and protein expression levels.
Six patients in cohort three experienced a mean improvement of 14 letters, stable retinal thickness with a mean 2 µm increase and stable intraocular RGX-314 protein expression over 2 years. Four patients who did not receive anti-VEGF injections after 9 months experienced a mean 14-letter improvement in visual acuity and a mean change of +9 µm in retinal thickness.
“This indicates the increase in BCVA was not driven by the patients who did receive anti-VEGF injections,” Steve Pakola, MD, chief medical officer of Regenxbio, said in the presentation.
In addition, 73% of patients in cohort five remain anti-VEGF injection-free over 9 months.
The company anticipates providing 1-year data from cohorts four and five later this year.