Nanoscope Therapeutics Inc. released key 6-month data from the phase 2 STARLIGHT clinical trial assessing MCO-010, an optogenetic therapy, for the treatment of vision loss due to Stargardt disease.
Give me more on MCO-010.
MCO-010 (sonpiretigene isteparvovec) is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic administered as a single intravitreal injection to potentially restore vision with enhanced contrast and definition in real-world environments.
How, exactly?
The gene therapy reprograms healthy retina cells to make them photosensitive by using a proprietary adeno-associated virus serotype 2 (AAV2) vector and promoter technology to deliver the MCO genes into retina bipolar cells, potentially enabling vision in different color environments.
MCO-010 could be administered in a clinical office setting.
Where is it in the clinical process?
The therapy has also already received both Orphan Drug and Fast Track designations from the FDA in 2022 for retinitis pigmentosa (RP) and Stargardt disease.
It’s also currently the only broadband, fast, and most-light sensitive opsin in clinical trials, according to Samarendra Mohanty, Nanoscope co-founder, president, and chief scientific officer (CSO).
To note, in May 2023, Nanascope released key efficacy data from a phase 2b trial on MCO-010 for the treatment of RP.
Read more here.
Now talk about this trial.
The multicenter, open-label study (NCT05417126) is evaluating a single dose of virally carried Multi-Characteristic Opsin (vMCO-010; dosed 1.2E11gc/eye) administered to six patients (≥16 years of age) diagnosed with Stargardt disease, followed by 48 weeks of assessment.
What’s being measured?
Primary outcomes include the type, severity, and incidence of ocular and systemic adverse events (AEs) following vMCO-010 injection (48 weeks).
Secondary outcomes, measured at Weeks 12, 24, and 48, include the effect of vMCO-010:
- Assessed via visual acuity (VA), measured by change in baseline of best-corrected visual acuity (BCVA).
- On light-guide mobility, measured via the Multi-Luminance Y-MobilityTest (MLYMT)
- On shape, measured by change from baseline via the Low-Vision Multi-Parameter Test (LVMPT).
- On optical flow, measured from baseline via LVMPT.
Now this data.
Per Nanoscope, vMCO-010-treated patients demonstrated a clinically meaningful improvement in BCVA (a secondary outcome) and achieved an approximate 3 dB gain in mean sensitivity (measured via Octopus visual field (VF) perimetry).
Treated patients also reported significant improvements in reading, color, and contrast outcome measures.
Further, both high baseline MLYMT and Multi-Luminance Shape Discrimination Test (MLSDT) performances were maintained.
Any adverse events?
The company reported no serious AEs.
Overall?
vMCO-010 demonstrated a favorable safety profile consistent with that observed in Nanscope’s phase 2a/2b trials for RP.
Significance?
The promising data on vMCO-010-treated patients supports its potential as the first approved therapy for Stargardt disease patients.
What’s next?
According to Clinical Trials, the STARLIGHT study is slated to conclude by October 2023.
Nanscope is also planning to engage with regulatory agencies (including the FDA) on the possibility of “expeditiously getting this novel therapy to patients.”