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

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Nanoscope Therapeutics announced the meeting with the FDA supported the advancement of MCO-010 into Phase 3 for Stargardt disease.

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.

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