Analysis of Patient-Reported Outcomes of MCO-010 Mutation Agnostic Optogenetic Therapy for Retinitis Pigmentosa: 12-Month Results from a Phase 2b Randomized, Sham-Controlled, Patient- and Assessor-Masked Clinical Trial (RESTORE)
Author(s)
Anish Patel, PharmD1, Subrata Batabyal, PhD1, Ramesh Arjunji, PhD2, Samuel Barone, MD1, Samar Mohanty, PhD1;
1Nanoscope Therapeutics, Dallas, TX, USA, 2Independent Consultant, Collegeville, PA, USA
1Nanoscope Therapeutics, Dallas, TX, USA, 2Independent Consultant, Collegeville, PA, USA
OBJECTIVES: To evaluate patient-reported outcomes of MCO-010 optogenetic therapy at week 52 in patients with advanced retinitis pigmentosa (RP).
METHODS: Subjects with advanced RP clinical diagnosis and a baseline visual acuity worse than 1.9 LogMAR in the study eye and no better than 1.6 LogMAR in the fellow eye were included. Subjects were randomized to receive a single intravitreal injection of MCO-010 or sham injection in the study eye. Vision-related quality of life was assessed using National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) from baseline through week 52.
RESULTS: Compared to baseline, improvements in both NEI-VFQ-25 near and distance activities subscale scores were observed at week 52 in MCO-010 treated subjects (n=18). Unlike the sham-controlled treated subjects (n=9), the score at week 52 in the MCO-010 treated cohort was significantly higher (p<0.05) compared to baseline.
CONCLUSIONS: RESTORE data demonstrate improved patient-reported outcomes on vision-related quality of life at week 52 in MCO-010 treated patients.
METHODS: Subjects with advanced RP clinical diagnosis and a baseline visual acuity worse than 1.9 LogMAR in the study eye and no better than 1.6 LogMAR in the fellow eye were included. Subjects were randomized to receive a single intravitreal injection of MCO-010 or sham injection in the study eye. Vision-related quality of life was assessed using National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) from baseline through week 52.
RESULTS: Compared to baseline, improvements in both NEI-VFQ-25 near and distance activities subscale scores were observed at week 52 in MCO-010 treated subjects (n=18). Unlike the sham-controlled treated subjects (n=9), the score at week 52 in the MCO-010 treated cohort was significantly higher (p<0.05) compared to baseline.
CONCLUSIONS: RESTORE data demonstrate improved patient-reported outcomes on vision-related quality of life at week 52 in MCO-010 treated patients.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO50
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
SDC: Sensory System Disorders (Ear, Eye, Dental, Skin), STA: Genetic, Regenerative & Curative Therapies