The investigational therapy QPI-1007 (Quark Pharmaceuticals) did not improve vision outcomes for most patients with acute nonarteritic anterior ischemic optic neuropathy (NAION), according to study findings published recently in Ophthalmology.

The phase 2/3 study enrolled 725 participants across 85 sites in eight countries. Patients presenting within 14 days of symptom onset were randomized to receive intravitreal injections of QPI-1007, a small interfering RNA targeting caspase-2, or sham injections. The primary endpoint was the proportion of patients who lost at least 15 ETDRS letters in best-corrected visual acuity (BCVA) at 6 months. The trial was terminated in July 2019, when an interim analysis did not warrant continued enrollment. 

In the recently published data, no statistically significant differences were observed between the multidose treatment groups and sham (15.4% for 1.5 mg, 18.2% for 3 mg, and 12.1% for sham). Secondary analyses also showed no significant differences overall. However, in patients with poorer baseline vision (BCVA ≤60 letters), the multidose 3 mg group had lower rates of vision loss compared with sham on both BCVA (5.9% vs 25.4%; P=.01) and visual field measures (P=.023). QPI-1007 was well tolerated, with adverse events largely related to the injection procedure.

Investigators suggested that future trials may benefit from enrolling patients with moderately reduced vision and using composite outcome measures. RP