Nanoscope Therapeutics is set to submit a Biologics License Application (BLA) to the FDA in the first quarter of 2025 for MCO-010 (sonpiretigene isteparvovec), a gene therapy intended for the treatment of retinitis pigmentosa (RP). This decision follows a productive meeting with the FDA, marking a significant step toward a potential new treatment option for patients with this inherited retinal disease.
MCO-010: A Novel Approach to Vision Restoration
MCO-010 is an ambient-light activatable, multi-characteristic opsin (MCO) optogenetic therapy designed to restore vision in patients with advanced RP, regardless of the specific gene mutation causing the disease. The therapy involves a single intravitreal injection of an adeno-associated virus (AAV2) vector that delivers the MCO transgene. This transgene increases the light sensitivity of bipolar cells in the retina, thereby improving vision in individuals who have lost rod and cone photoreceptors.
Clinical Trial Success and Data
Data from the Phase 2b/3 RESTORE clinical trial (NCT04945772) demonstrated that MCO-010 led to significant improvements in vision. At week 52, 40% of patients treated with MCO-010 showed an improvement of at least 0.3 LogMAR units in best-corrected visual acuity (BCVA), meeting the primary endpoint. By week 76, this number increased to 56%, with continued gains observed particularly in the high-dose group, fulfilling a key secondary endpoint. Furthermore, at 100 weeks, the BCVA area under the curve (AUC) was five times greater in the MCO-010 treatment arms compared to the sham control.
Allen C. Ho, MD, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, noted that the preservation of baseline visual acuity over several years represents a significant treatment effect that deviates from the natural progression of RP.
Disease-Modifying Effects
Recent findings published in Translational Vision Science & Technology (TVST) indicate that MCO-010 not only restores vision but also arrests further retinal degeneration. In an animal model of RP, MCO-010 treatment prevented further loss and disorganization of retinal cell layers, suggesting a disease-modifying effect. Following intravitreal MCO-010 treatment, approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed, unlike the control group.
Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope, stated that this disease-modifying aspect of MCO-010 has also been observed in animal models of Stargardt disease and nonhuman primate models for geographic atrophy. Preliminary evidence suggests stabilization of retinal structure in the RP clinical trial, with plans for long-term evaluation of this anatomical biomarker.
Regulatory Pathway and Future Plans
MCO-010 has received Fast Track designation and orphan drug designation from the FDA for both RP and Stargardt disease. Nanoscope has completed the Phase 2 STARLIGHT trial of MCO-010 in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in Q1 2025. The company is also developing MCO-020, a non-viral laser-delivered gene therapy for geographic atrophy.
With the planned BLA submission, Nanoscope aims to provide a viable, restorative option for patients facing vision loss due to RP and other retinal degenerative diseases.
