Clinical trials

ACTIVELY RECRUITING STUDY

Ongoing Observational Study in Advanced RP

NCT05820100 (actively recruiting), is a multi-center observational study in advanced RP patients 

NOT ACTIVELY RECRUITING and COMPLETE CLINICAL STUDIES

STARLIGHT: Phase 2  6-month Results for MCO-010 in Stargardt

STARLIGHT (6 patients dosed, not actively recruiting), is a Phase 2 trial of MCO-010 in Stargardt patients (NCT05417126).  

Efficacy

• Patients with predominantly macular atrophy experienced clinically meaningful improvements in visual acuity (BCVA)
• MCO-010 patients exhibited ~ 3 dB gain in mean sensitivity measured by Octopus Visual Field Perimetry
• High baseline vision-guided mobility (MLYMT) and object recognition (MLSDT) performance was maintained throughout study
• MCO-010 treated Stargardt patients reported significant improvements in key domain scores of Patient Reported Outcome measures: Reading, Color & Contrast

Safety

• Well-tolerated with no Serious Adverse Events
• Safety profile consistent with the favorable profile observed in Ph1/2a and Ph2b MCO-010 studies

Takeaways & next steps

Potential to address high unmet need: Substantial benefit to patients with vision loss due to Stargardt macular degeneration (no available treatment today)

Consistency of response: Visual function improvements consistent with MCO-010 Phase 1/2 study and Phase 2b RCT in RP

Next steps: Engage with FDA and other regulatory agencies on the future of MCO-010, with the goal of expeditiously getting this novel therapy to patients

RESTORE: Topline Phase 2b Results for MCO-010 in Advanced RP

RESTORE (18 patients dosed, not actively recruiting), is a Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). 

Efficacy

• 88.9% (16 / 18) of treated patients experienced clinically meaningful 2 or more luminance level improvement in vision-guided mobility (MLYMT) or object recognition (MLSDT)
• 94.4% (17/18) of treated patients experienced clinically meaningful improvement in MLYMT or visual acuity (BCVA)
• 100% (18/18) of treated patients experienced clinically meaningful improvement in MLYMT, MLSDT or BCVA
• Treated patients experienced a clinically meaningful improvement in BCVA by a mean of -0.34 LogMAR

Safety

• Well-tolerated with no serious or severe ocular or systemic adverse events reported
• One SAE occurred in a placebo treated patient
• Comparable incidence of TEAEs1across study arms

Takeaways & next steps

Potential to address high unmet need: Substantial benefit to patients with severe vision loss due to advanced RP (no available treatments today)

Consistency of response: Visual function improvements and safety profile consistent with previous MCO-010 Phase 1/2 study

Next steps: Engage with FDA and other regulatory agencies on the future of MCO-010, with the goal of expeditiously getting this novel therapy to patients; File RMAT application April 2023

Completed Phase 1/2a Trial  in Advanced RP

NCT04919473 (completed, 11 patients dosed), was a single-center Phase 1/2a clinical trial for retinitis pigmentosa

Efficacy

• 82% (9/11) of treated patients experienced clinically meaningful improvement in MLYMT or visual acuity (BCVA)
• 64% (7/11) of treated patients experienced clinically meaningful improvement in BCVA
• Treated patients experienced a clinically meaningful improvement in BCVA by a mean of -0.38 LogMAR

Safety

• Well-tolerated with no serious or severe ocular or systemic adverse events reported
• Mild to moderate, mainly self-resolving inflammation

Takeaways & next steps

First in Human Study: Substantial benefit to patients with severe vision loss due to advanced RP (no available treatments today)

Favorable Safety: Treatment well-tolerated across subjects, no patients on concomitant medication at week 52

Promising Efficacy: Visual function improvements across navigational and fine vision metrics

1. Treatment emergent adverse events