Beacon Therapeutics Treats First Patient in VISTA Trial of AGTC-501 for XLRP

Beacon Therapeutics announced the treatment of the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501 (laruparetigene zovaparvovec).

VISTA is a global randomized, controlled, masked, multicenter pivotal study evaluating the efficacy, safety, and tolerability of 2 dose levels of AGTC-501 for the treatment of X-Linked Retinitis Pigmentosa (XLRP) compared to an untreated control group. Beacon Therapeutics plans to use the data generated from the VISTA trial, in combination with data from the phase 1/2 HORIZON and phase 2 SKYLINE studies, including all available long-term data, to support a BLA in the US and a MAA in Europe for the treatment of patients with XLRP.

AGTC-501 has been awarded an Innovative Medicine Designation (ILAP) in the UK, Priority Medicine (PRIME) in the EU, and Fast Track in the US for the treatment of X-linked Retinitis Pigmentosa caused by mutations in the RPGR gene.

"This is an important milestone for the continued clinical development of AGTC-501, which offers the potential to transform the prognosis of patients with XLRP by enabling them to see better or maintain their current vision," Darin Curtiss, PharmD, Global Vice President of Clinical Development at Beacon Therapeutics, said in a company news release. "We look forward to progressing AGTC-501 in the clinic and moving toward our goal of bringing a treatment to market for this life-altering disease.”

XLRP is an orphan disease affecting approximately 17,000 patients in the US and EU5. XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age with no treatment options available. 

For more information about the VISTA study can be found at https://clinicaltrials.gov/study/NCT04850118.