Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with XLRP
Beacon Therapeutics announced the presentation of 12-month interim safety and efficacy results for the phase 2 SKYLINE trial in patients with X-linked retinitis pigmentosa (XLRP) at the Macula Society Meeting in Palm Springs, California.
Twelve-month data from males with XLRP demonstrated a response rate (defined as an improvement in retinal sensitivity, as assessed by microperimetry, of at least 7 decibels (dB) in at least 5 loci) of 63% in study eyes treated with a high dose (6.8 E+11 vg/eye) of AGTC-501. Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both). In addition, patients in the high dose cohort also demonstrated a robust improvement in visual function, including mean retinal sensitivity. AGTC-501 was well-tolerated and there were no clinically significant safety events associated with treatment, and any treatment-related adverse events were mostly nonserious and mild to moderate in severity.
XLRP is an orphan disease predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, and it is therefore expected to rescue function of both rod and cone photoreceptors, making it uniquely well-suited as a potential treatment to improve the lives of patients with XLRP.
“These data, which demonstrate a favorable safety profile and notable improvement in visual function, are another positive step in the development of AGTC-501 for XLRP, a blinding, orphan disease for which there is currently no approved treatment," Nadia Waheed, MD, MPH, Chief Medical Officer of Beacon Therapeutics, said in a company news release. "We look forward to announcing the initiation of our phase 2/3 VISTA trial in the first half of 2024.”
Presentation – Subretinal AGTC-501 Gene Therapy for XLRP: 12-Month Interim Safety & Efficacy Results of the Phase 2 SKYLINE Trial
Presenter – Mark Pennesi, MD, PhD, FARVO, Director, Ophthalmic Genetics at the Retina Foundation in Dallas, Texas; Professor of Ophthalmology and Professor of Molecular and Medical Genetics, Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute, Oregon Health and Science University