Interim results favorable for QPI-1007

Fremont, CA-Quark Pharmaceuticals Inc. has announced favorable interim results from the first two cohorts of its open-label, first-in-human phase I clinical study of QPI-1007, its proprietary synthetic siRNA drug candidate for ocular neuroprotection, in patients with recent onset of non-arteritic anterior ischemic optic neuropathy (NAION).

QPI-1007 exhibited no dose-limiting toxicities up to and including the highest dose of 6 mg enabled in this study, which is the highest dose of synthetic siRNA tested in the clinic following intravitreal administration.

After 3 months of follow up, patients treated in the study by a single dose of QPI-1007 exhibited no further loss of visual acuity, whereas vision loss was observed in previous studies in patients with non-treated NAION and similar initial disease severity. With the exception of one patient whose vision remained stable, all patients exhibited letter gains compared with baseline at screening, with some patients gaining three lines or more by 3 months after treatment.

The ongoing phase I open-label, dose-escalation study, which is being conducted at 22 sites in the United States and six in Israel, has enrolled a total of 38 patients in two strata. Stratum I was a dose-escalation safety study in patients who are legally blind secondary to chronic optic nerve atrophy or retinal degeneration and is fully enrolled. Stratum II was designed to evaluate safety further and to assess for potential biological activity of QPI-1007 in patients with recent-onset NAION by monitoring changes in visual function following drug administration.

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