Beacon Therapeutics, a Syncona portfolio company focused on ophthalmic gene therapies, has announced positive 36-month interim safety and efficacy data from its Phase I/II Horizon trial of AGTC-501 in patients with X-linked retinitis pigmentosa (XLRP). The data, presented at a conference in Barcelona, highlight the therapy's favorable safety profile and sustained efficacy.
Safety and Tolerability
The primary endpoint of the Horizon trial was safety, and the results demonstrated that AGTC-501 continues to be well-tolerated. No clinically significant safety events related to the gene therapy were observed during the 36-month follow-up period. This is a critical finding, as it supports the long-term safety of AGTC-501 in patients with XLRP.
Efficacy and Durability
In addition to the favorable safety profile, the data also revealed promising efficacy signals and a continued durability profile out to 36 months post-dosing. While specific efficacy data points were not disclosed in the announcement, the continued durability suggests a potential long-term benefit for patients receiving AGTC-501. Chris Hollowood, Chief Executive Officer of Syncona, noted that the durability profile was "particularly pleasing for an exploratory Phase I/II trial."
Ongoing Clinical Development
Beacon Therapeutics is actively advancing AGTC-501 through further clinical development. The Phase II SKYLINE trial, which incorporates an optimized dose and improved surgical repeatability, is expected to yield 24-month data. Furthermore, the Phase II DAWN and Phase II/III registrational VISTA trials are currently recruiting patients, utilizing an improved manufacturing process. These ongoing trials will provide further insights into the efficacy and safety of AGTC-501 in a larger patient population.
X-Linked Retinitis Pigmentosa (XLRP)
XLRP is a genetic eye disease that causes progressive vision loss. It primarily affects males and is characterized by mutations in the RPGR gene. Currently, there are limited treatment options available for XLRP, highlighting the unmet medical need for effective therapies like AGTC-501.
