Nanoscope Therapeutics Inc. has announced the publication of positive results from its STARLIGHT clinical trial in eClinical Medicine, part of The Lancet Discovery Science.
STARLIGHT is the first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease (SD).
STARLIGHT is a phase 2, open-label study designed to assess the safety and efficacy of a single intravitreal injection of gene-agnostic MCO-010 therapy in six participants with severe vision loss due to SD.
Key findings include:
Mean improvement in best corrected visual acuity (BCVA) from baseline to week 48 was:
For all patients, +5.5 and +9.0 ETDRS letters without and with a wearable low-vision aid, respectively.
In individuals with atrophy confined to the macula, +12.0 and +32.0 ETDRS letters, without and with a wearable low-vision aid, respectively.
Some patients observed vision improvement as early as 4 weeks post-treatment that was maintained throughout the duration of the study.
MCO-010 was well tolerated with no reported serious adverse events over 48 weeks.
Stargardt disease, also known as juvenile macular dystrophy, is a major cause of vision loss in children, affecting more than 40,000 people in the US and leaving over 20,000 legally blind. In patients with SD, the light-sensing photoreceptors in the macula degrade, leading to loss of central vision. Currently, there are no approved treatments for SD.
“While this was an open-label study, this is one of the first studies to demonstrate vision improvement in Stargardt disease patients. This is a remarkable finding as these patients typically experience irreversible central vision loss over their lifetimes,” said Byron Lam, professor of ophthalmology at Bascom Palmer Eye Institute, lead author and principal investigator of the STARLIGHT study.
“I am thrilled to be part of the upcoming Phase 3 trial of MCO-010 in Stargardt disease, helping advance this promising therapy towards potential approval.”
“We are grateful to the patients and investigators who participated in the trial,” said Samarendra Mohanty, president/chief scientific officer.
“The results we have seen in STARLIGHT for SD patients reinforce the therapeutic potential of our gene-agnostic MCO-010 platform, building on positive outcomes in both Stargardt and retinitis pigmentosa populations.”
The published manuscript, “Safety and Efficacy of MCO-010 Optogenetic Therapy in Patients with Stargardt Disease in USA (STARLIGHT): an Open-label Multi-Center Ph2 Trial,” is available online and will appear in an upcoming print issue of The Lancet.
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