Nonarteritic anterior ischemic optic neuropathy (NAION), the most common acute optic neuropathy in individuals over 50 years, currently has no proven treatment. To address this gap, a phase 2/3 clinical trial evaluated the safety and efficacy of intravitreal tivanisiran (QP1-1007).,a small interfering RNA targeting caspase-2, in 725 participants with acute-onset NAION. The study found no significant difference at 6 months in the proportion of participants losing ≥15 letters of best-corrected visual acuity (BCVA) between the multidose and sham groups.
However, among participants with baseline BCVA ≤60 letters, significantly fewer in the treatment groups lost ≥10 letters of BCVA (P = 0.045 for 1.5 mg × 3; P = 0.0104 for 3.0 mg × 3), along with reduced visual field decline (7-dB loss; P = 0.023). Adverse events were similar across groups, with no unexpected safety concerns beyond known…
