Victoria Johnson | CGTLive | March 14, 2024
Patients with X-linked retinitis pigmentosa (XLRP) treated with AGTC-501 gene therapy experienced improvements in visual function including retinal sensitivity as assessed by macular Integrity Assessment (MAIA) microperimetry and full-field stimulus threshold (FST) with a favorable benefit-risk profile.
Updated, 12-month data from the phase 2 SKYLINE trial (NCT04850118) were presented by Mark Pennesi MD, PhD, Director, Ophthalmic Genetics Retina Foundation Dallas, and Texas Professor of Ophthalmology Professor of Molecular and Medical Genetics, Paul H. Casey Ophthalmic Genetics Division Casey Eye Institute, Oregon Health & Science University, at the 47th Annual Maula Society Meeting, held Feb 07 - 10, 2024, in Palm Springs, California.
“The benefit-risk profile is favorable and supports continued clinical development for the treatment of patients with XLRP caused by RPGR mutations,” Pennesi said during his presentation.
AGTC-501 delivers a full-length RPGR via an adeno-associated virus (AAV) vector delivered by subretinal injection.
The data are from 14 participants, 6 that received low-dose (7.5 E+10 vg/eye) and 8 that received high-dose (6.8 E+11 vg/eye) AGTC-501.
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REFERENCES
Pennesi M. Subretinal AGTC-501 Gene Therapy for XLRP: 12-Month Interim Safety & Efficacy Results of the Phase 2 SKYLINE Trial. Presented at:https://www.beacontx.com/news-and-events/subretinal-agtc-501-gene-therapy-for-xlrp-12-month-interim-safety-efficacy-results-of-the-phase-2-skyline-trial/
Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa. News release. Beacon Therapeutics. https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-12-month-data-from-phase-2-skyline-trial-of-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-302056840.html
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