MCO-010 intravitreal optogenetic therapy in Stargardt disease. 6-month outcomes from the Phase 2 STARLIGHT trial

Abstract

Purpose : MCO-010 is an intravitreal, Adeno-Associated Virus (AAV2)-delivered optogenetic therapy designed to treat severe vision loss from outer retinal degenerative disease. Stargardt disease is a rare, inherited condition with significant unmet need, characterized by progressive photoreceptor degradation and loss of central vision eventually leading to profound vision loss. STARLIGHT (NCT05417126) is a Phase 2 open label multi-center clinical study to evaluate the safety and effects of a single dose level of MCO-010 in subjects with Stargardt disease.

Methods : Participants in the STARLIGHT trial were required to have a documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1. All subjects received a single intravitreal injection of 1.2E11 gc/eye MCO-010 in the study eye at baseline. Ocular and systemic safety, as well as multiple vision function tests, including Best-Corrected Visual Acuity (BCVA) using ETDRS charts at 50 cm, Multi-Luminance Y-Mobility Test (MLYMT) and Low-Vision Multi-Parameter Test (LVMPT) were key outcome measures.

Results : 6 participants, 4 males and 2 females, with severe vision loss due to Stargardt disease were enrolled between June and September 2022. Mean age was 49 years (range 32-71). At baseline mean study eye BCVA letter score was 23 (range 13-34) or approximately 1.8 LogMAR. Safety and efficacy data through 6 months follow up will be presented for the first time.

Conclusions : STARLIGHT is the first clinical study of optogenetic therapy for severe vision loss due to Stargardt disease. The 6-month outcomes from STARLIGHT will help inform the future clinical development of MCO-010 optogenetic therapy for vision restoration in Stargardt disease and other retinal degenerative conditions.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

 

MCO-010 Vector Design

 

Starlight Phase 2 Study Design Schema