Retina Society 2023: STARLIGHT Trial of MCO-010 Optogenetic Therapy for Stargardt Disease

Giulia Corradetti, MD
Doheny Eye Institute
Pasadena, CA

On the second day of the Retina Society Meeting in New York City, Dr. Allen Ho from Philadelphia, PA, presented the late-breaking 12-month outcomes of the STARLIGHT trial, a multi-center Phase 2 trial assessing a novel MCO-010 optogenetic therapy in subjects with Stargardt disease. The session was moderated by Drs. David Brown, and Aleksandra Rachitskaya. The MCO-010 is a multi-characteristic opsin ambient-light activatable optogenetic monotherapy designed to restore vision in patients with Stargardt disease and received FDA Fast Track designation and FDA orphan drug designation for Stargardt disease and retinitis pigmentosa.

The STARLIGHT trial aims to report the safety and tolerability of this novel gene therapy at week 24 and mean change in visual function metrics at week 48 in subjects with severe vision loss due to Stargardt disease. The STARLIGHT trial enrolled patients with a clinical diagnosis or a genetic diagnosis of Stargardt disease. 6 subjects were enrolled and received a single intravitreal injection of MCO-010.

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Dr. Allen Ho concluded his presentation with the following key messages:

  • MCO-010 was well tolerated with a favorable safety profile and no serious adverse events were observed in patients treated with MCO-010 at week 24 (primary endpoint);
  • Patients treated with MCO-010 with predominantly macular disease experienced clinically meaningful improvements in BCVA, and an approximate 2.6 dB improvement in mean sensitivity was measured by Visual Field Perimetry at week 48 (secondary endpoints);
  • Improvements in reading and color & contrast were recorded at week 48 (exploratory endpoints).