Nanoscope Therapeutics has agreed with the US Food and Drug Administration (FDA) on the design of its Phase III trial following a successful Phase II study of its gene therapy in Stargardt disease.
The Phase II STARLIGHT study (NCT05417126) found that a single intravitreal injection of gene-agnostic MCO-010 in six patients significantly improved vision. Nanoscope claims that this is the only trial to have demonstrated vision improvement in patients with Stargardt disease.
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In all patients, the one-time intravitreal gene therapy demonstrated a visual acuity improvement of 5.5 and 9 ETDRS letters, respectively, seen without and with a wearable low-vision aid.
In patients with atrophy confined to the macula, the mean improvement was higher, 12 ETDRS letters over 48 weeks. With the use of a wearable low-vision aid, there was an improvement of 32 ETDRS letters.
Nanoscope added that the gene therapy was well-tolerated with no reported serious adverse events over 48 weeks.
Dr Byron Lam, the STARLIGHT study’s principal investigator and professor of ophthalmology at the Bascom Palmer Eye Institute, said: “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.”
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By GlobalDataThe registration-enabling Phase III trial, STARGAZE, is set to be initiated by the end of this year. The randomised, double-blind, sham-controlled study aims to enrol 60 patients. The primary endpoint will be the change in BCVA using a standard vision chart versus a sham control while a key secondary endpoint will assess improvement in multi-luminance shape recognition. Top-line results are expected in 2027.
Stargardt disease is a genetic eye condition that causes vision loss. Also known as juvenile macular dystrophy, it affects more than 40,000 people in the US, leaving over 20,000 individuals legally blind. In Stargardt disease, the light-sensing photoreceptors in the macula deteriorate, leading to loss of central vision. Currently, there are no approved treatments.
MCO-010 acts by activating highly dense bipolar retinal cells to become light sensitive, utilising remaining visual circuitry following photoreceptor death. It has also been investigated in retinitis pigmentosa, with Nanoscope having submitted a rolling biologics licence application (BLA) to the FDA for the therapy in the indication.
While no therapies are approved, there are a lot of interesting candidates in development for this indication. Ascidian Therapeutics is investigating an RNA exon editor, ACDN-01, in a Phase I/II trial (NCT06467344). Kubota Vision is also running a trial of emixustat hydrochloride, a retinoid isomerohydrolase inhibitor. Ocugen is also running a Phase II/III pivotal confirmatory study of its modifier gene therapy candidate, OCU410ST.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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