Ocugen shares fall, despite positive eye disorder trial
A midstage trial of Ocugen's gene therapy for sight-robbing disease geographic atrophy (GA) showed efficacy – but still sparked a selloff in its stock.
Shares in the Malvern, Philadelphia-based company were down almost 15% at one point after the announcement, but regained some of the lost ground after hours as investors digested the data.
The preliminary data from the phase 2 ArMaDa trial of Ocugen's OCU410 (AAV5-RORA) in patients with GA secondary to dry age-related macular degeneration (dAMD), a group that accounts for two to three million people across the US and Europe.
The efficacy result, coming from the first 50% or so patients enrolled in the study, revealed a 46% reduction in the growth of lesions in GA for the medium and high doses of OCU410 compared to a control group after a year.
What seems to have spooked investors was a better performance for the medium dose (54% reduction) compared to the high-dose group (36%), failing to show a linear dose response, although that variability could be explained by the fact that the patient population for statistical analysis is still small.
Ocugen also reported updated results from a phase 1 trial, which showed that ellipsoid zone (EZ) loss – which results in the impairment of central vision that is a characteristic of GA – was 60% reduced in OCU410-treated eyes compared to untreated eyes at 12 months.
Approximately three-quarters of people living with GA are thought to be undiagnosed and, without treatment, an estimated 66% of people may become blind or severely visually impaired.
Treatment options are limited, although there are two FDA-approved complement inhibitor therapies – Apellis' Syfovre (pegcetacoplan) and Astellas' Izervay (avacincaptad pegol) – which require regular injections into the eye.
In contrast, OCU410 would be delivered using a single subretinal injection, delivering a gene (RORA) that addresses multiple pathways thought to be involved in the GA, including inflammation, oxidative stress, and complement activation.
Outside the US, there are no approved therapies for GA at all, and Ocugen said it plans to move quickly ahead with a phase 3 programme that will support regulatory filings around the world.
"As a practising retinal specialist, OCU410's clinical profile is genuinely exciting for geographic atrophy patients," said Lejla Vajzovic of Duke University Eye Center, who chairs Ocugen's retinal disease advisory board.
"With these promising results, I believe OCU410 has the potential to set a new standard of care with a single treatment for life."
Ocugen said it plans to report the full results of the ArMaDa trial later this quarter and start a phase 3 programme in 2026, setting up potential regulatory filings in 2028. The company seems to be a little ahead of other groups developing gene therapies for GA secondary to AMD, including Sanofi, which has a candidate in phase 1/2 testing, and Johnson & Johnson (phase 2).
There was more good news for GA patients last year when a wireless retinal implant developed by Science Corp was shown to restore vision in patients with GA, in whom the loss of sight is usually irreversible.
