Beacon Therapeutics’ gene therapy AGTC-501 (laruparetigene zovaparvovec) is in the spotlight with encouraging interim results from SKYLINE trial in the retinitis pigmentosa (retinitis) market. Offering a new mechanism of action, it presents a formidable challenge to Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), the current frontrunner in gene therapy for retinitis. With promising safety profiles and ongoing trials, AGTC-501’s efficacy has the potential to redefine the treatment landscape for retinitis patients, says GlobalData, a leading data and analytics company.

As for AGTC-501’s safety profile, Beacon Therapeutics reported that it was well-tolerated; the majority of treatment-related adverse events were mild to moderate in severity, and there were no clinically significant safety events related to this treatment.

Sara Reci, MSc, Senior Pharma Analyst at GlobalData, comments: “AGTC-501, like Luxturna, is a gene therapy. However, it stands out by offering an alternative mechanism of action. AGTC-501 is an X-linked retinitis pigmentosa GTPase regulator (RPGR) activator. This has positive implications for patients, which includes potentially introducing a second gene therapy into the retinitis space, as well as providing a treatment with a new mechanism of action for retinitis patients.”

Nonetheless, as it currently stands, AGTC-501 is subject to head-to-head competition with MeiraGTx’s botaretigene sparoparvovec, also a gene therapy with the same mechanism of action, and currently in Phase III of development (NCT04671433).

Trial results for botaretigene sparoparvovec showed that at six months, patients had an improvement in mean retinal sensitivity of 1.96dB in the treated eyes compared to untreated eyes in the randomized concurrent control arm. Thus, based on efficacy profiles, AGTC-501 currently presents results that are more promising. The Phase II/III trial for AGTC-501, VISTA, is anticipated to begin in the first half of 2024.

Reci concludes: “AGTC-501 has tremendous potential to be popular among retinitis patients and clinicians alike, given its promising interim results from its SKYLINE trial. Should subsequent trials give rise to results that further promote the efficacy and safety of this drug, it may be set on a trajectory to compete with Luxturna in the retinitis space.”