The US Food and Drug Administration (FDA) has approved Ocugen’s investigational new drug (IND) amendment to initiate a Phase II/III pivotal confirmatory study of the modifier gene therapy candidate, OCU410ST, under development for all Stargardt disease.
This follows the agency’s orphan drug and rare paediatric disease designations for the therapy to treat ABCA4-associated retinopathies.
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The Phase II/III trial of OCU410ST will involve 51 subjects with Stargardt disease, out of which 34 will be given a subretinal injection of the therapy, whereas 17 subjects will form an untreated control group.
The trial’s main aim is to assess the decrease in the size of the atrophic lesion.
Key secondary goals include improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA), against controls.
The trial’s one-year follow-up data will support Ocugen’s biologics licence application.
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By GlobalDataOcugen co-founder, CEO and chairman Dr Shankar Musunuri said: “We have had a highly productive and collaborative engagement with the FDA’s Center for Biologics Evaluation and Research (CBER) in establishing the pivotal confirmatory trial for OCU410ST.
“It’s evident that there is a real sense of urgency by the agency in providing treatment options for patients who currently have nothing available to them. As we initiate the Phase II/III registration trial, we are expediting the clinical development of OCU410ST by two to three years and potentially providing an innovative gene therapy to patients desperate for a treatment option.”
OCU410ST showed a favourable tolerability and safety profile in the Phase I GARDian trial, which showed improvements in visual function.
It leverages an adeno-associated virus (AAV) delivery platform to introduce the RAR-related orphan receptor A (RORA) gene into the retina.
Stargardt disease, a genetic disorder leading to retinal degeneration, affects vision significantly.
In January 2025, Ocugen dosed the first subject in a multi-centre Phase I trial of OCU200 for diabetic macular oedema treatment.
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