Regenxbio reports promising data on gene therapy for bilateral wet AMD
ABBV-RGX-314 shows promise as a one-time gene therapy for bilateral wet AMD, significantly reducing the need for frequent anti-VEGF injections while maintaining visual outcomes and demonstrating a favorable safety profile, according to data presented at AAO 2024.
The Phase 2 sub-study evaluated safety and efficacy by administering a single subretinal dose of ABBV-RGX-314 to the second eye of patients previously treated in the first eye. The patients had a high treatment burden before enrollment, receiving an average of 9 anti-VEGF injections in the previous year. The therapy showed promising durability, mirroring results from previous long-term trials lasting up to 4 years.
Key Findings:
- 97% reduction in annualized anti-VEGF injection burden.
- 100% of patients required either zero or one supplemental injection.
- 78% of patients were entirely injection-free at nine months.
- Patients maintained stable best-corrected visual acuity (BCVA) and central retinal thickness (CRT).
As of September 11, 2024, ABBV-RGX-314 was well tolerated with no serious drug-related adverse events. There were no reported cases of intraocular inflammation or other severe complications. Common side effects, such as mild retinal pigmentary changes and post-operative conjunctival hemorrhage, resolved within days to weeks without requiring special intervention.
“The majority of our patients with wet AMD eventually have bilateral disease and face a substantial treatment burden with frequent lifelong injections in both eyes. This leads to suboptimal real-world vision outcomes with current standard of care,” said presenting author Arshad Khanani, MD, MA, FASRS, in a press release. “The fellow eye dosing data with ABBV-RGX-314 is a milestone for the field of gene therapy for common retinal diseases, as this is the first time we have performed bilateral treatment for wet AMD patients. These results, combined with the durable treatment effect up to four years shown in long-term follow-up, highlight the potential of ABBV-RGX-314 as a one-time effective treatment option for patients with wet AMD.”
Reference
Khanani AM, et al. Subretinal Delivery of Investigational ABBV-RGX-314 as a Gene Therapy for nAMD: First Time Results of a Fellow Eye Bilateral Dosing Study. Poster presented at: American Academy of Ophthalmology Annual Meeting; October 2024; Chicago.
