Nanoscope Therapeutics Inc. announced key efficacy findings from its RESTORE phase 2b trial assessing the use of MCO-010 as an optogenetic therapy for the treatment of retinitis pigmentosa (RP).
Give me more on MCO-010.
MCO-010 (sonpiretigene isteparvovec) is an ambient-light activatable multi-characteristic opsin (MCO) optogenetic received as a single intravitreal injection to potentially restore vision with enhanced contrast and definition in real-world environments.
In other words: the gene therapy reprograms healthy retina cells to make them photosensitive by using proprietary adeno-associated virus serotype 2 (AAV2) vector and promoter technology to deliver the MCO genes into retina bipolar cells to enable vision in different color environments.
What else?
The therapy has also already received both Orphan Drug and Fast Track designations from the FDA in 2022 for RP and Stargardt disease; it’s also currently the only broadband, fast, and most-light sensitive opsin in clinical trials.
Talk about this trial.
The randomized, double-masked, sham-controlled, multicenter trial (NCT04945772) enrolled 27 patients (ages 18+) with severe vision impairment due to RP.
A total of 18 participants received one injection of MCO-010 (1.2E11gc/eye or 0.9E11gc/eye) in the experimental arms, while 9 participants received one sham injection.
Primary outcome measures included mean change in multi-luminance y-mobility test (MLYMT, vision-guided mobility) score vs sham; other key efficacy assessments were multi-luminance shape discrimination test (MLSDT, near-object recognition) and best-corrected visual acuity (BCVA).
Note: A 0.3 LogMAR change for BCVA would be considered clinically meaningful (negative change means improved visual acuity [VA]).
Didn’t we just see results from this?
Yes … the company released positive topline data from the RESTORE trial in April 2023.
See here for more details.
Now talk about the latest data.
Key efficacy measures at 12 months found that 100% of MCO-010-treated patients showed vision improvement in the MLYMT, MLSDT, or BCVA (compared to 55.6% of placebo patients) 94.4% of MCO-010 patients showed vision improvement in just MLYMT or BCVA (compared to 44.4% of placebo patients).
A total of 88.9% MCO-010 patients demonstrated a 2 or more luminance level improvement in MLYMT or MLSDT (compared to 44.4% receiving placebo), while 77.8% of MCO-010 patients showed vision improvement in MLYMT or BCVA (compared to 33.3% of placebo patients).
Any adverse events?
Investigators reported no serious / severe ocular or systemic adverse events (AEs). However, a comparable incidence of treatment emergent AEs (TAEs) were noted across study arms.
The most commonly observed ocular TAEs were anterior chamber cells, ocular hypertension, and conjunctival hemorrhage.
Significance?
The clinically significant vision improvements observed among MCO-010-treated patients supports the potential for the therapy to treat advanced RP, which currently has no approved treatment.
What’s next?
According to Nanoscope co-founder/CEO Sulagana Bhattacharya, the company is planning to discuss the latest data as well as next steps with the FDA regarding an expeditious path to market for MCO-010.