Nanoscope's Gene Therapy Shows Promise in Phase II with Endpoint Adjustment

Nanoscope Therapeutics has reported positive results from its Phase IIb clinical trial for a gene therapy aimed at treating vision loss caused by retinitis pigmentosa. The biotech firm is now preparing to seek FDA approval for MCO-010, a gene therapy that is designed to be effective for all patients with the eye disease, irrespective of the specific genetic mutation causing their condition.

Retinitis pigmentosa is a progressive genetic disorder that leads to the degeneration of the retina. While Spark Therapeutics' Luxturna received FDA approval in 2017 for treating a subset of patients with the RPE65 gene mutation, MCO-010 is intended to offer a broader treatment option. The therapy employs an adeno-associated virus vector to introduce multi-characteristic opsins, which enable retinal bipolar cells to detect light, thus improving vision.

Initially, the trial's primary endpoint was to measure the effectiveness of MCO-010 using the multi-luminance mobility test after 52 weeks. However, after the trial did not meet this endpoint, Nanoscope altered its primary endpoint in January 2024. The FDA suggested that a change from baseline in visual acuity for low vision patients could serve as an appropriate primary efficacy endpoint.

The revised trial focused on the best-corrected visual acuity (BCVA) at week 52, and the results showed statistically significant improvements with both doses of MCO-010, meeting the new primary endpoint. Despite the low dose not outperforming the control group at week 76, the high dose demonstrated a significant advantage over the sham treatment. The biotech recorded reductions in logMAR scores from baseline of 0.539 for the high dose and 0.374 for the low dose after 76 weeks, which aligns with the Icelandic Medicines Agency's endorsement of a 0.3 logMAR change as clinically meaningful for patients with severe vision loss.

Following these findings, Nanoscope is set to file for FDA approval in the latter half of 2024, potentially offering a new treatment option for individuals suffering from retinitis pigmentosa. The success of MCO-010 could represent a significant advancement in the field of gene therapy for vision loss, providing hope for a wider range of patients affected by this genetic eye disease.