Gene Therapy ABBV-RGX-314 Reduces Treatment Burden by Nearly 100% in Wet AMD Patients, Demonstrating Durable Efficacy

Oct 29, 2024 07:20 CST Updated 07:20

ReGenXBio

Gene Therapy Developer

AbbVie

Innovative Drug Developer

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October 29, 2024

eMedClub News

Recently, REGENXBIO announced positive data from a Phase 2 study of the investigational gene therapy ABBV-RGX-314, jointly developed with AbbVie, in patients with bilateral wet age-related macular degeneration (AMD).Data shows that nine months after receiving ABBV-RGX-314 treatment, the patients' treatment burden was reduced by 97%.Detailed data were presented at this year's American Academy of Ophthalmology (AAO) meeting. According to the company's website,ABBV-RGX-314Expected to become the first gene therapy for the treatment of chronic retinal diseases.

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This study aims to evaluate individuals who have previously undergone unilateral treatmentABBV-RGX-314Among the treated patients, a single dose was administered to the other eye.Safety and Efficacy of Subretinal Delivery of ABBV-RGX-314. Treatment of the second eye in patients was performed one year or more after the first eye received ABBV-RGX-314 treatment.

The nine-month data from this bilateral eye study included nine patients who had received subretinal delivery of ABBV-RGX-314, participated in the Phase 1/2a or bridging study, and opted for treatment in the second eye. Prior to receiving ABBV-RGX-314, these patients had a high treatment burden in the other eye, with an average of nine anti-vascular endothelial growth factor (VEGF) treatment injections in the year before entering the study, including some anti-VEGF injections aimed at providing longer-lasting effects.

Nine months later, the key findings of the Phase 2 bilateral eye study include:

The burden of patients receiving anti-VEGF treatment annually is reduced by 97%.
100% of patients required zero or one supplemental injection.
78% of patients do not need injections at all.

In addition,The patient maintained stable best-corrected visual acuity (BCVA) and central retinal thickness (CRT) at nine months.ABBV-RGX-314 in Both EyesAqueous HumorSimilar levels of protein were produced in both. As of September 11, 2024, ABBV-RGX-314 was well-tolerated in the other treated eye, with no drug-related serious adverse events reported.

▲Development Plan of ABBV-RGX-314 (Image Source: Reference [2])

ABBV-RGX-314 uses an adeno-associated virus 8 (AAV8) vector carrying a transgene expressing an anti-VEGF antibody fragment.The protein expressed in the body inhibits blood vessel formation and retinal fluid accumulation by blocking VEGF signaling. Beyond treating wet AMD, both companies are also exploring the potential of ABBV-RGX-314 for treating diabetic retinopathy and other chronic retinal diseases.

References:

[1]https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-presents-positive-data-phase-ii-study-subretinal-abbv

[2] https://ir.regenxbio.com/static-files/528aa5e1-591f-4cd8-b728-80727c8c9f36

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Disclaimer: This article aims to convey industry development information and explore the frontier progress of biomedicine. The content of the article represents the author's viewpoint and does not represent the position of Yi Mai Ke, nor does it constitute any value judgment, investment advice, or medical guidance. If necessary, please consult a professional for investment or visit a regular hospital for medical advice.

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