Beacon Therapeutics Holdings Limited, a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, 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 47th Annual 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.
Dr. Nadia Waheed, chief medical officer of Beacon Therapeutics, said, “These data, which demonstrate a favourable 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. We look forward to announcing the initiation of our phase 2/3 VISTA trial in the first half of 2024.”
Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.
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