Quark reports results from QPI-1007 siRNA drug study in rat ocular hypertension model of glaucoma

Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, today announced results from a second study of QPI-1007, a neuroprotective siRNA drug, in rat ocular hypertension model of glaucoma, conducted by Prof. Adriana Di Polo of the Department of Pathology and Cell Biology, Universite de Montreal.  Prof. Di Polo's recent study in a rat model of glaucoma shows that QPI-1007, when given in two intravitreal injections after induction of ocular hypertension (at two weeks and 10 days later), can deliver a sustained neuroprotective effect, as measured by a significant attenuation in RGC death five weeks after disease induction. Ocular neuroprotection, encompassing prevention of retinal ganglion cell (RGC) death, represents a novel approach for treating glaucoma, non-arteritic anterior ischemic optic neuropathy (NAION), and other ocular diseases.

These results support the further evaluation of QPI-1007 as a potential novel treatment for glaucoma.

Dr. Daniel Zurr, Quark's Chief Executive Officer, stated, "These findings suggest that QPI-1007 delivers a sustained therapeutic effect against RGC death. These results support our novel approach to neuroprotection and, together with our IP position, have the potential to strongly differentiate QPI-1007 from other glaucoma treatments in the market and clinic. In addition, these results support the study of QPI-1007 in other retinal diseases, such as the ongoing Phase I clinical trial in NAION."

Prof. Di Polo commented: "We are encouraged by additional QPI-1007 data that further confirm protection of retinal ganglion cells in experimental glaucoma. Therapeutic effect was observed at five-weeks post-induction of ocular hypertension when the drug was administered at two weeks after the initial insult, and long after RGC loss has commenced. The rat ocular hypertension model is a highly relevant model of prevalent forms of human glaucoma because in both cases RGC loss is likely to result from axonal injury caused by elevated intraocular pressure (IOP). Given these similarities, we believe that further study of QPI-1007 is warranted in glaucoma."

In addition to sustained neuroprotective effects in the rat glaucoma model, QPI-1007 has demonstrated robust efficacy when administered immediately after optic nerve injury in two models of acute RGC death induced by optic nerve transection (using crush or axotomy). QPI-1007 utilizes a proprietary structure and modifications that is designed to preserve RNAi activity while ameliorating potential off-target and immunostimulatory effects of siRNAs. A Phase I clinical trial of QPI-1007 is currently underway in Non-arteritic Anterior Ischemic Optic Neuropathy (NAION). QPI-1007 is Quark's first synthetic siRNA drug candidate with proprietary structure and chemical modifications, providing the Company freedom to operate in the siRNA IP space. QPI-1007 is designed as nuclease stable molecule possessing high specific RNAi activity while lacking potential off-target and immunostimulatory effects.