Following regulatory discussions with the FDA, Nanoscope Therapeutics Inc. announced next steps for the clinical path of MCO-010, an optogenetic therapy for the treatment of retinitis pigmentosa (RP), toward U.S. market approval.
Give me a sentence about this company.
The Dallas, Texas-headquartered biotechnology company has focused on developing gene-agnostic, sight-restoring optogenetic therapeutics for inherited retinal diseases (IRDs).
Now MCO-010.
MCO-010 (sonpiretigene isteparvovec) is an ambient-light activatable multi-characteristic opsin (MCO) developed to be administered as a single intravitreal injection to potentially restore vision with enhanced contrast and definition in real-world environments.
More specifically: The gene therapy could reprogram 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 possibly enable vision in different color environments.
And its current clinical status?
The therapy has 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.
How has it performed so far?
These trials include:In August 2023, the phase 2 STARLIGHT trial (NCT05417126) for Stargardt disease reported favorable 6-month data.
And in May 2023, the RESTORE phase 2b trial (NCT04945772) demonstrated promising key efficacy data.
Gotcha. So what’s the FDA update?
So… the first thing to know is that the agency met with Nanoscope as part of a Type B End-of-Phase 2 discussion following the May 2023 data readout from the RESTORE trial.
According to the company, the FDA conveyed its input on the specific primary endpoint that would prove to be an “appropriate primary efficacy assessment in an adequate and well-controlled study” to provide the necessary evidence and data needed to support a future Biologics License Application (BLA) approval.
And this primary endpoint is… ?
Per Nanoscope: Change from baseline in a measure of visual acuity (VA) in low vision (RP) patients.
To note, the original primary efficacy endpoint from that RESTORE trial was:
- Efficacy of a single intravitreal injection of MCO-010, as assessed via best-corrected visual acuity (BCVA).
- Determined by the change from baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye (at Week 52)
Gotcha. Now what’s next?
Nanoscope has since updated the ongoing RESTORE trial’s primary endpoint to reflect the FDA’s recommendation.
“End-of-study, 100-week data are expected to be reported in the first half of 2024,” the company stated.
And the bigger picture of this?
With no current treatment approved in the U.S., MCO-010 could prove to be groundbreaking in the quest to restore some level of vision for these RP patients.