Sepofarsen Benefits Genetic Form of Blindness in Early Trials


A breakdown of a new 12-month, phase 1b/2 assessment of the RNA therapy in patients with LCA10.

New findings from a one-year phase 1b/2 trial assessing sepofarsen in patients with a genetic variant of Leber congenital amurosis (LCA10) show the therapy could provide significant improvement to patient visual acuity.

The data, planned for presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2020 Annual Meeting this year, showed that 4 of the 6 observed treated patients with the progressively worsening blindness-causing condition reported a 15-letter improvement in standard eye chart testing at 12 months.

In an interview with HCPLive®, study author Steve R. Russell, MD, Dina J. Schrage Professor of Macular Degeneration Research at University of Iowa Health Care, discussed the potential of the RNA therapy in effectively and safely treating the underlying cause of the genetic disease.

“Patients with LCA typically have very early-onset poor vision,” Russell said. “This is one of the poorer prognosis and more accelerated courses of patients who have LCA, so many times they are legally blind by the time you can measure them.”

He also discussed the therapy’s promise for a field barren of marketed options—there are currently no drugs approved by the US Food and Drug Administration (FDA) for treating patients with LCA10.

“This is a new gene,” he explained. “And so, there are no currently approved FDA therapies for this, so it would be a novel indication for an LCA.”

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