Expanding Sepofarsen Understanding in LCA10


After promising early findings for the investigative therapy in a rare form of blindness, investigators turn to the next phase.

After investigators presented new, one-year findings of a phase 1b/2 trial assessing RNA therapy sepofarsen in patients with a rare genetic variant of Leber congenital amurosis (LCA10) and found it able to significant improve visual acuity, they sought next steps of vindication.

In an interview with HCPLive® after what would have been the presentation of new data at the Association for the Research of Vision and Ophthalmology (ARVO) 2020 Annual Meeting, Steve R. Russell, MD, Dina J. Schrage Professor of Macular Degeneration Research at University of Iowa Health Care, discussed the already ongoing phase 2/3 assessments for sepofarsen.

The phase 3 trial, in particular, ups the ante for the investigative therapy to prove its benefit—more patients, lower doses, lower margins for improvement.

“What we’re looking for in the phase 3 is a larger cohort,” Russell explained. “We are looking at a slightly older population—a little older because we are edging because of the cataract. We’re also looking at patients with, in general, slightly better visual acuities.”

Nonetheless, the next rounds of trials for evidence in an indication without any currently marketed therapies is working with a foundation of promising findings.

“The improvements that we saw in the outperformers were so startling that it’s clear this was not just a placebo effect, or some sort of new discovery of a previous function,” Russell explained.

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