Nanoscope Reports Positive End-of-Phase 2 Meeting for MCO-010 Gene Therapy

Credit: US Food and Drug Administration

Nanoscope Therapeutics has announced a positive End-of-Phase 2 meeting with the US Food and Drug Administration (FDA) for MCO-010 gene therapy to treat severe vision loss due to macular degeneration from Stargardt disease.¹

Reported by Nanoscope on September 12, 2024, the meeting supported the advancement of MCO-010 to a Phase 3 registration trial, with key input from the FDA. The study design will evaluate the safety and efficacy of MCO-010 in a single intravitreal dose group, compared with a control cohort receiving a sham injection.

“The current paradigm for managing Stargardt disease is one of diagnosis, followed by vision rehabilitation and a lifetime sentence of declining vision to severe blindness,” said Samuel Barone, MD, chief medical officer of Nanoscope. “The advancement of Nanoscope’s groundbreaking therapy for Stargardt to a Phase 3 registrational trial moves us much closer to having a viable restorative option for patients whose vision has been lost due to this terrible disease.”

Stargardt disease, known as juvenile macular dystrophy, is a genetic eye disorder with a variety of clinical presentations, which can vary in onset and rate of progression.² Patients with Stargardt Disease typically present with progressive visual impairment in the first or second decade of life.

For these patients, light-sensing photoreceptor cells in the macular degrade and lead to loss of central vision — several therapeutic agents are being evaluated for treatment, but none have received approval from the FDA.

MCO-010 is a genetic mutation agnostic optogenetic therapy being evaluated for multiple retinal degenerative disorders, including Stargardt and retinitis pigmentosa.¹ The therapy has received the FDA’s Fast Track designation and Orphan Drug Designation for both indications.

Positive data were reported in the Phase 2 STARLIGHT clinical trial, suggesting the benefit of MCO-010 for patients with Stargardt.³ After the End-of-Phase 2 Meeting, the FDA offered clear outcomes for the Stargardt trial program.¹

The regulatory agency confirmed a change in best-corrected visual acuity (BCVA) on the ETDRS was appropriate for a primary efficacy endpoint evaluating change from baseline to Week 52 in the planned Phase 3 trial. Regulators also provided insight on statistical methods for the proposed primary and secondary endpoints to inform a further conversation with the FDA to align on statistical analyses.

The FDA offered further discussion and assistance for evaluating the multi-luminance shape discrimination test as a key secondary trial endpoint, according to Nanoscope.

Nanoscope and the FDA achieved alignment on the enrollment of legally blind patients with vision worse than 20/200 and aged ≥12 years for the Phase 3 trial, extending the patient population evaluated in STARLIGHT.

The FDA signed off on the sufficiency of the current non-clinical package to support a future Biologics License Application (BLA) submission. Nanoscope announced the planned Phase 3 trial will represent the first randomized, controlled gene therapy trial for Stargardt disease.

“Following the positive results from the Phase 2 STARLIGHT clinical trial, the outcome of this meeting strengthens our conviction to advance clinical development of MCO-010 in this indication,” said Sulagna Bhattacharya, co-founder and chief executive officer of Nanoscope. “We appreciate the FDA’s guidance and look forward to finalizing preparation for the Phase 3 trial.”

References

1. _{Nanoscope Therapeutics. Nanoscope therapeutics announces end-of-phase 2 meeting with U.S. FDA and plan to initiate a phase 3 clinical trial of MCO-010 to treat stargardt macular degeneration. Nanoscope Therapeutics. September 12, 2024. Accessed September 13, 2024. https://nanostherapeutics.com/2024/09/12/nanoscope-therapeutics-announces-end-of-phase-2-meeting-with-u-s-fda-and-plan-to-initiate-a-phase-3-clinical-trial-of-mco-010-to-treat-stargardt-macular-degeneration/.}
2. _{Kohli P, Tripathy K, Kaur K. Stargardt Disease. [Updated 2024 Jan 8]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK587351/}
3. _{Iapoce C. Stephen H. Tsang, MD, Phd: MCO-010 for stargardt disease in starlight trial. HCP Live. November 5, 2023. Accessed September 13, 2024. https://www.hcplive.com/view/stephen-tsang-md-phd-mco-010-stargardt-disease-starlight-trial.}