Based on feedback from the FDA, Nanoscope Therapeutics Inc. plans to submit a Biologics License Application (BLA) for Multi-Characteristic Opsin (MCO)-010, intended to treat retinitis pigmentosa (RP), in early 2025.
Big plans! Let’s start with Nanoscope.
The Texas-based biotechnology company is developing MCOs—gene-agnostic, sight-restoring optogenetic therapeutics—for inherited retinal diseases (IRD)s, including:
- Cone rod dystrophy
- Dry age-related macular degeneration (AMD)
- Geographic atrophy (GA)
- RP (our topic of choice)
- Stargardt disease (SD)
- Read up on phase 3 plans.
- Usher syndrome
Now talk about MCO-010.
MCO-010 (sonpiretigene isteparvovec)—an ambient-light activatable MCO optogenetic agent—is a gene therapy vector designed to reprogram healthy retina cells into photosensitive cells via a single intravitreal (IV) injection.
How it does this: By using proprietary adeno-associated virus serotype 2 (AAV2) and promoter technology to deliver MCO genes into retinal bipolar cells to enable vision in different color environments.
- The potential result: Restoring vision with enhanced contrast and definition in real-world environments for patients with advanced RP.
And how is this unique from other gene therapies?
Current gene therapies: Generally target treating patients with specific genetic (inherited) mutations in the outer retinal cells.
This MCO: Targets “abundant inner retinal neurons” with the potential to restore RP-impacted vision loss by using degenerated outer retinal cells.
Interesting … and how has it performed in clinical trials?
Earlier this year, Nanoscope reported positive findings from the phase 2b RESTORE trial (NCT04945772) for RP, which enrolled 27 adult patients randomized to receive either MCO-010 (two doses) or a sham injection.
The primary endpoint: Efficacy of a single MCO-010 injection as evaluated via best-corrected visual acuity (BCVA).
And those results?
The study met its primary endpoint, demonstrating a statistically significant improvement in BCVA from Week 52 for both MCO-010 dose groups.
- Plus: Those improvements continued beyond Week 52, indicating MCO-010’s “durable effect of a single intravitreal injection.”
See our coverage for details of those findings—including how they compared to previous clinical data on the candidate.
How about its regulatory clinical status?
It’s important to note that the FDA has already granted MCO-010 both Orphan Drug and Fast Track designations for RP and SD.
Also: The candidate is currently the only broadband, fast, and most-light sensitive opsin in clinical trials.
Nice! Now what led to these BLA plans?
The company reported a “productive FDA meeting,” where the agency acknowledged Nanoscope’s proposed next steps for MCO-010’s RP-targeted BLA rolling submission.
These steps were outlined based on the candidate’s Fast Track Designation, according to Nanoscope.
Very exciting … so when is this submission expected?
While the company reported Q1 2025, no specific date was provided.
Gotcha. So what else is Nanoscope up to?
Speaking of Q1 2025, the company also recently announced its intent to kick off a phase 3 registration trial evaluating MCO-010 for SD within that quarter.
Needless to say, Q1 2025 looks like it’ll be a busy time for Nanoscope. So stay tuned!