Beacon Therapeutics has initiated the final leg of the race for its lead asset, AGTC-501 (laruparetigene zovaparvovec), announcing in mid-June 2024 that the company has treated its first patient in the pivotal trial, VISTA. Currently, the only retinitis pigmentosa therapy on the market is Spark Therapeutics’ Luxturna (voretigene neparvovec), a retinoid isomerohydrolase activator, indicated for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. AGTC-501 is highly anticipated as it has the potential to become the first gene therapy specifically indicated for X-linked retinitis pigmentosa (XLRP).


AGTC-501 is an XLRP GTPase regulator (retinitis pigmentosa 15 or retinitis pigmentosa 3 GTPase regulator activator, which expresses the full-length RGPR protein. It thereby targets the full complement of photoreceptor damage that XLRP causes and is consequently anticipated to restore the function of rod and cone receptors, making it an exceptionally promising treatment option to enhance the quality of life for patients with XLRP.

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AGTC-501 is currently in Phase II/III of development for retinitis in the VISTA trial (NCT04850118), a randomized, masked, controlled, multi-centre pivotal trial set to investigate the impact that AGTC-501 has on vision and other symptoms in additional patients with XLRP, particularly studying the efficacy, safety and tolerability of AGTC-501 at two dose levels. The trial will enrol patients in the US and is then set to expand globally.

Results from AGTC-501’s ongoing Phase II SKYLINE trial (NCT03316560) revealed that at 12 months, 63% of eyes treated with high-dose AGTC-501 (6.8E + 11 vg/eye) experienced retinal sensitivity improvement of at least 7 decibels in at least five loci. Furthermore, patients in this study arm also demonstrated a strong improvement in visual function and mean retinal sensitivity. The low-dose AGTC-501 (7.5E + 10 vg/eye) group and the untreated fellow eyes group, on the other hand, demonstrated a 0% response rate. Beacon Therapeutics reported that AGTC-501 was well-tolerated; there were no clinically significant safety events related to treatment. Beacon Therapeutics announced that data from VISTA, SKYLINE and HORIZON, as well as long-term data, will collectively support the biologics licence application filing in the US, and the marketing authorisation application (MAA) in Europe.

Key opinion leaders (KOLs) interviewed by GlobalData noted that AGTC-501 has very good initial data, with Phase I–II trials showing that an improvement in microperimetry was observed in patients, and that optical coherence tomography demonstrated structural changes. The KOLs added that in the subsequent Phase II trial for AGTC-501, patients randomised to the higher dose had a pretty good improvement in microperimetry. Pivotal trial results are consequently highly anticipated.

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All in all, AGTC-501 has tremendous potential to be popular among XLRP patients and clinicians alike, given the promising results from its SKYLINE trial. Should VISTA further promote the efficacy and safety of this drug, AGTC-501 may be set on a trajectory to finally provide a long-awaited efficacious treatment for XLRP patients.

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