Syncona Ltd (LON:SYNC), a leading life science investor focused on creating, building and scaling a portfolio of global leaders in life science, notes that its portfolio company, Beacon Therapeutics, has presented unmasked three-month data from the Phase II SKYLINE trial of its AAV viral vector-based gene therapy AGTC-501 in X-Linked Retinitis Pigmentosa (XLRP), at the FLORetina ICOOR 2023 Congress in Rome.
Highlights from the presentation include:
· Encouraging efficacy profile with robust and statistically significant improvement in retinal sensitivity, the primary endpoint for the trial; 75% (6/8) response rate in the higher dose cohort
· Data underlines the favourable safety profile of AGTC-501, with no clinically significant safety events related to the therapy
Beacon expects to present 12-month data from the SKYLINE trial in H1 CY2024, as well as commence the pivotal Phase II/III VISTA trial. 24-month data from the SKYLINE trial is expected to be presented in H2 CY2024.
Chris Hollowood, Chief Executive Officer of Syncona Investment Management Limited, said: “This data released by Beacon from the SKYLINE trial underlines the potential of the company’s lead AGTC-501 therapy in slowing the progression of XLRP, a devastating disease where patients become legally blind in their mid-40s. We are pleased to see the Beacon team continue to make significant progress as they progress towards key milestones, including the upcoming initiation of a pivotal trial. We also look forward to seeing further longer-term efficacy read-outs from SKYLINE in 2024, which will be key to demonstrating the durability profile of AGTC-501.”
