Quark Pharmaceuticals last week announced “favorable” interim results from a phase I trial of its siRNA-based ocular neuroprotectant QPI-1007, showing the drug may improve the vision of patients with a rare eye condition.
QPI-1007 is designed to inhibit the pro-apoptotic gene caspase 2 as a treatment for non-arteritic anterior ischemic optic neuropathy, or NAION, a disorder that can lead to permanent blindness. Quark began phase I testing of the agent in early 2010, with the intent of ultimately marketing the drug for glaucoma (GSN 3/4/2010).
According to Quark, the 38-patient study, which is ongoing, has two arms: one examining escalating doses of QPI-1007 in patients who are legally blind secondary to chronic optic nerve atrophy or retinal degeneration; and a second assessing the drug's safety and “potential biological activity ... in recent-onset NAION patients by monitoring changes in visual function following drug administration.”
In the second arm, two cohorts of 10 patients each received a single intravitreal injection of one of two doses of QPI-1007, the company said. After one month, 40 percent of patients in the first cohort and 60 percent of patients in the second cohort experienced improvements in visual acuity, with the effect extending out to three months for some patients.
“The data suggest that QPI-1007 may improve visual acuity in NAION patients,” Quark President and CEO Daniel Zurr said in a statement. “Based on the interim data from this clinical study, and from our positive results from preclinical studies in three different models of optic neuropathy, including a model of glaucoma, we believe QPI-1007 may protect neurons in the retina and optic nerve in NAION and in other types of optic neuropathy.”