--Phase 3 Registrational Trial Expected to Commence in Near-term
DALLAS, Sept. 12, 2024 /PRNewswire/ -- Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases and age-related macular degeneration (AMD), today announced a productive End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its clinical program evaluating MCO-010 for the treatment of severe vision loss due to Stargardt Macular Degeneration (SMD), supporting advancement of MCO-010 to a Phase 3 registrational trial.
Key outcomes of the EOP2 meeting discussion included:
- The FDA provided clear input on the proposed Phase 3 registrational trial in patients with SMD intended to support safety and effectiveness of MCO-010. The proposed study design will consist of a single MCO-010 intravitreal dose group, randomized 1:1 to a control cohort receiving a sham injection.
- The FDA confirmed that a change in best corrected visual acuity (BCVA) that employs testing on the ETDRS Chart is appropriate for use in a primary efficacy endpoint assessing change from baseline to Week 52 in the proposed Phase 3 trial.
- The FDA provided valuable input on statistical methods for the proposed primary and secondary endpoints that will inform further engagement with FDA to align on statistical analyses.
- The FDA offered continued discussion and assistance to plan further evaluation of the multi-luminance shape discrimination test to support use as a key secondary endpoint.
- Alignment was achieved on the enrollment of legally blind patients with vision worse than 20/200, and as young as 12 years old, for the upcoming study, extending the population evaluated from that studied in Phase 2.
- The FDA agreed on the sufficiency of the current nonclinical package to support a future BLA submission.
"Patients suffering from Stargardt Macular Degeneration face a substantial disease burden with no restorative treatments presently available," said Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. "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. We appreciate the FDA's guidance and look forward to finalizing preparation for the Phase 3 trial."
"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. 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," said Samuel Barone, M.D., Chief Medical Officer.
The planned Phase 3 trial will be the first randomized, controlled gene therapy trial for Stargardt disease. Nanoscope's mutation-agnostic therapeutic approach has the potential to improve vision for many others suffering from severe vision loss, including macular degeneration patients.
About Stargardt Disease
Stargardt disease, often called as juvenile macular dystrophy, is an eye disease that presents in childhood and a major cause of vision loss in children and young adults. It is an inherited disease, meaning it is passed on to children via faulty genes from their parents. In patients with Stargardt disease, the light-sensing photoreceptors cells in the macula degrade leading to loss of central vision. Currently, there are no approved treatments.
About Nanoscope Therapeutics, Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company's lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for Retinitis Pigmentosa (RP) (NCT04945772). The company has also completed the STARLIGHT Phase 2 trial of MCO-010 in Stargardt disease patients (NCT05417126). MCO-010 has received FDA fast track designation and FDA orphan drug designation for both RP and Stargardt disease. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.
Investor Contact:
Argot Partners
(212) 600-1902
[email protected]
SOURCE Nanoscope Therapeutics
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