Kenji Fujita, MD, on Clinically Meaningful FST and MLMT Improvements in LCA1 With ATSN-101

“In addition to FST, we also measured an endpoint for functional vision called MLMT, or multi luminescence mobility test. This is a full room physical maze that patients run under 7 different light conditions, and it's the same endpoint that was used for the approval of Luxturna. And we measured this in some of our high dose patients and we found a very nice correlation with our improvements in FST. And in the majority of the patients, we saw a 2 or greater step improvement in MLMT, which is the threshold considered to be clinically meaningful.”

Positive 6-month safety and efficacy data from the ongoing phase 1/2 clinical trial (NCT03920007) of ATSN-101 (Atsena Therapeutics), an investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1), were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, held April 23-27, 2023, in New Orleans, Louisiana, by Christine Nichols Kay, MD, Affiliate Assistant Professor, University of South Florida, and Clinical Ophthalmology Advisor, Atsena.

CGTLive spoke with Kenji Fujita, MD, chief medical officer, Atsena, to learn more about the data and how ATSN-101 could help address current unmet needs in the patient population of LCA1. He discussed unique advantages of the gene therapy, including the use of Atsena’s vector and photoreceptor-specific promoter, that may improve efficiency of gene expression. He overviewed the updated data presented at ARVO, including positive results on both full-field stimulus test and MLMT scores that were clinically meaningful. He also touched on the recent investigational new drug approval of ATSN-201 to be assessed in X-linked retinoschisis.

REFERENCE

Kay CN. Six-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). Presented at: ARVO 2023 Annual Meeting; April 23-27, 2023; New Orleans, LA. Presentation 1914