Mar 27, 2024

Nanoscope Plans to Apply for FDA Approval for its Optogenetic Therapy

Eye On the Cure Research News

The company reported positive two-year results for its Phase 2 clinical trial of MCO-010.

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Nanoscope Therapeutics has announced that it met the primary and secondary endpoints for its Phase 2b RESTORE clinical trial of MCO-010, an optogenetic therapy for restoring some vision to people with advanced vision loss from retinitis pigmentosa (RP). As a result, the company plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024. Approval of the BLA would enable the company to market MCO-010 to patients in the US.

MCO-010 is designed for people who have lost most or all of their photoreceptors, the cells that make vision possible. The treatment, a small drop of liquid delivered by an intravitreal injection, uses a human-engineered virus to deliver copies of a Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease. The company says that MCO-010 is enabling the recipient’s bipolar cells to sense ambient light, thereby working as a backup system for lost photoreceptors. The approach doesn’t use goggles or eyewear to enhance the visual information coming into the retinas. It’s also designed to work regardless of the mutated gene causing the RP.

The primary endpoint for the trial was change in LogMAR at 52 weeks after treatment. LogMAR is a test of best corrected visual acuity (BCVA) in which a lower number indicates better visual acuity. A LogMAR of 0.0 is equivalent to 20/20 vision, and a LogMAR of 2.0 is 20/2000 vision. If someone had a LogMAR of 2.0 and it improved to 1.5, that could be translated as improvement from 20/2000 to 20/640.

BCVA was obtained in the trial using a Freiburg Visual Acuity Test, which is designed for people with advanced vision loss who may not be able to read letters on a standard (Snellen) eye chart.

Baseline vision for the patients in the trial was hand motion or counting fingers — i.e., worse than a LogMAR of 1.9 or 20/1600.  A total of 27 patients were enrolled in the trial (9 high-dose, 9 low-dose, and 9 sham/control).

Patients’ BCVA at week 52 of the trial improved from baseline by -0.337 LogMAR in the high dose group and by -0.382 LogMAR in the low dose group. Patents’ BCVA at week 76 of the trial improved from baseline by -0.539 LogMAR in the high-dose group and by -0.372 in the low-dose group.

Some patients in the trial also had improvements in their ability to navigate Y-mobility and shape-recognition tests.