Quark Pharmaceutical's QPI-1007 Exhibits Neuroprotective Effects in Glaucoma Model

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Jun 28, 2010, 08:30 ET

FREMONT, Calif., June 28 /PRNewswire/ -- 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.

About Quark Pharmaceuticals, Inc.

Quark Pharmaceuticals, Inc., the world leader in novel RNAi discovery and development, has the largest clinical-stage siRNA pipeline in the industry. The Company's fully integrated drug development platform spans therapeutic target identification to drug development. Quark's approach to delivery allows targeting of tissues and organs including the eye, kidney, ear, lung, spinal cord and brain.

Quark's pipeline is led by PF-655, currently in two Phase II clinical trials for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME). In 2006 the siRNA therapeutic candidate was licensed to Pfizer, who is conducting both trials in collaboration with Quark. PF-655 targets Quark's proprietary gene, RTP801, discovered using its BiFAR™ target discovery platform that identifies clinically relevant critical genes and proteins that reverse the disease phenotype when inhibited. The Company owns a family of patents covering the RTP801 gene, its RNA and protein product sequences, specific antibodies, and gene inhibition across different pathologies.

Quark is also evaluating QPI-1002, the first systemically administered siRNA drug in human clinical trials. Enrollment was successfully completed in Phase I studies of QPI-1002 for the prevention of acute kidney injury (AKI) following major cardiovascular surgery and the prophylaxis of delayed graft function (DGF) after kidney transplantation and Phase II clinical studies are planned to commence shortly. For the structure of these products, Quark has obtained licenses from Silence Therapeutics and from Alnylam Pharmaceuticals.

Quark is currently conducting clinical trials of QPI-1007, its proprietary synthetic siRNA drug candidate for ocular neuroprotection. QPI-1007 utilizes a proprietary structure developed in collaboration with BioSpring GmbH that provides Quark with freedom to operate in the siRNA intellectual property arena and chemical modifications that are designed to preserve RNAi activity while ameliorating potential off-target and immunostimulatory effects of siRNAs.

Quark is also committed to leveraging a broad research pipeline of siRNA drug candidates and novel siRNA structures to develop additional RNAi drug candidates.

Quark is headquartered in Fremont, California and operates research and development facilities in Boulder, Colorado and Ness-Ziona, Israel. Additional information is available at www.quarkpharma.com

Quark Pharmaceuticals, Inc.

Juliana Friedman

+972 89 30 5111

[email protected]

The Ruth Group (investors / media)

Sara Pellegrino / Jason Rando

(646) 536-7002 / 7033

[email protected]

SOURCE Quark Pharmaceuticals, Inc.