Stephen H. Tsang, MD, PhD: MCO-010 for Stargardt Disease in STARLIGHT Trial

New data from the phase 2 STARLIGHT trial suggest the promise of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) optogenetic therapy in patients with Stargadt Disease, according to a presentation at the 127th Annual American Academy of Ophthalmology (AAO) Meeting in San Francisco, California.

Results from the phase 2 STARLIGHT trial showed patients with Stargadt Disease had a mean best-corrected visual acuity improvement of +5.5 ETDRS letters.

“In some suggestions, there are improvements in vision,” presenting investigator Stephen H. Tsang, MD, PhD, a professor of ophthalmology, pathology, and cell biology at Columbia University, told HCPLive. “It would provide support to go into a phase 3 trial in a larger number of patients to see whether or not there is efficacy.”

Stargardt disease is a rare inherited eye disorder caused by fatty deposits in the macula and leads to center-field loss of vision in affected patients. There are approximately 8,000-10,000 patients in the United States, and symptoms often begin in childhood or adolescence. Currently, there are no treatments approved by the US Food and Drug Administration (FDA).

The phase 2 STARLIGHT Trial evaluated the effects of MCO-010 gene therapy on patients with Stargardt disease. MCO-010 uses a well-established intraocular injection for the delivery of a gene that encodes the ambient light-sensitive MCO protein into retinal cells. As a result, the therapy enables retinal cells to detect light, allowing patients with rare retinal diseases, including retinitis pigmentosa or Stargardt disease, to see again.

MCO-010 has been granted Orphan Drug and Fast Track designation by the FDA for both Stargardt Disease and retinitis pigmentosa.

“Since Stargardt, although relatively rare, has a lot of features as in age-related macular degeneration,” Tsang said. “It works on this younger group of patients who have Stargardt juvenile macular degeneration, we hope that it would have a broader impact on age-related macular degeneration as well.”

References

_{Tsang SH. MCO-010 Optogenetic Therapy for Vision Loss in Stargardt Disease: Topline Data From the Phase 2 STARLIGHT Trial. Presented at the 2023 American Academy of Ophthalmology Annual Meeting, November 3 – 6, 2023.}