Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa

• AGTC-501 was generally safe and well-tolerated, and showed robust improvements in visual function at the 12-month analysis.
• Pivotal Phase 2/3 VISTA trial for AGTC-501 in XLRP expected to begin in H1 2024.

London, UK, Cambridge, MA, 08 February 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, yesterday 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 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. 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, M.D., Ph.D., 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

The presentation took place on Wednesday, February 7 at 6:25 p.m. PT.



About Beacon Therapeutics

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. The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another in-licensed from the University of Oxford targeting cone-rod dystrophy (CRD), an inherited retinal disease.
Lead development candidate AGTC-501 is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.
Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.
The Company is supported by funds from Syncona and additional investors including Oxford Science Enterprises.
Find out more about Beacon Therapeutics at