Nonarteritic anterior ischemic optic neuropathy (NAION) remains without an established therapy, despite being the most common acute optic neuropathy in patients over 50. A large, multicenter Phase 2/3 trial evaluated intravitreal QPI-1007, a caspase-2–targeting siRNA, but failed to meet its primary endpoint of reducing ≥15-letter BCVA loss at 6 months. However, subgroup analyses revealed that eyes presenting with baseline BCVA ≤20/63 had significantly reduced risk of further BCVA decline and VF deterioration with repeated dosing. Importantly, the treatment demonstrated a favorable safety profile, limited to expected procedural adverse events.
These findings suggest potential neuroprotective benefit in a narrowly defined patient population, though general applicability remains uncertain. Unlock the full article in the journal. Click here In light of these findings, should future NAION…
