Quark Pharmaceuticals said this week that it has dosed the first patient in a phase I study of its siRNA-based ocular neuroprotectant QPI-1007.
In the study, the drug will be directly delivered to the eyes of legally blind patients and patients with a recent onset of non-arteritic anterior ischemic optic neuropathy, a rare condition that can lead to permanent blindness. Primary endpoints of the trial are safety and tolerability, but Quark will also evaluate whether QPI-1007 has any biological activity in NAION patients.
NAION is the first indication Quark plans to pursue with the drug, but the company ultimately hopes to market QPI-1007 as a treatment for glaucoma.
Previously, the company released preclinical data showing the agent could prevent progressive retinal ganglion cell loss, which is believed to cause the vision loss characteristic of glaucoma, in an increased ocular-pressure rat model of the disease (see RNAi News, 7/30/2009).
Before that, the company released data demonstrating the drug's neuroprotective activity in two different animal models: one of retinal ganglion cell death by optic nerve crush and one of cell death induced by optic nerve axotomy (see RNAi News, 10/2/2008).