Quark Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company that is a leader in the discovery and development of novel RNAi-based therapeutics.

QPI-1002 is, based on public information, the first synthetic siRNA to be evaluated following systemic administration in human clinical trials, and is designed to temporarily inhibit expression of the stress-response gene, p53. QPI-1002 is being studied under two INDs for two indications - the prevention of acute kidney injury (AKI) in patients undergoing major cardiovascular surgery, and the prophylaxis of delayed graft function (DGF) in renal transplant patients. QPI-1002 was granted Orphan designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the prophylaxis of delayed graft function in kidney transplant patients. We completed Phase I studies in these patient populations and have recently initiated dosing in a Phase II trial for delayed graft function and expect to initiate dosing in a Phase II clinical trial for AKI within 2011. In August 2010 we granted to Novartis an option for a worldwide exclusive license to QPI-1002 for all indications.

QPI-1007 is being developed as a neuroprotective agent for the treatment of optic neuropathies that result in the death of retinal ganglion cells. We are currently evaluating QPI-1007 to treat sudden vision loss associated with non-arteritic anterior ischemic optic neuropathy (NAION), an unmet medical need. Demonstration of activity of QPI-1007 in NAION patients may provide the basis for examining activity in slower-progressing optic neuropathies such as glaucoma. QPI-1007 has a proprietary siRNA structure that preserves RNAi activity while reducing potential off-target and immunostimulatory effects. The current Phase I study is an open-label, dose escalation, safety, tolerability and pharmacokinetic study in which QPI-1007 will be administered to all participating patients. Enrollment of Stratum 1, the dose-escalation safety component of the study, was completed with no dose-limiting toxicities observed up to the highest dose level tested. Stratum 2 is designed to further evaluate safety and to determine potential biological activity in non-arteritic anterior ischemic optic neuropathy patients as assessed through changes in visual acuity and visual field following drug administration compared to historical control.

Quark’s partner, Pfizer, Inc, is is currently evaluating the first siRNA we developed, PF-655, in two Phase II clinical studies, one for the treatment of diabetic macular edema (DME) and the other in patients with wet, age-related macular degeneration (AMD). Pfizer has exclusive development rights to siRNAs that inhibit expression of a gene that was discovered by Quark, RTP801, for ophthalmic and non-ophthalmic indications, while Quark is eligible for development- and sales-based milestone payments and royalties from future product sales.

Preclinical Pipeline
In addition to the clinical drug candidates described above, Quark has a broad preclinical pipeline targeting serious unmet medical needs.

We are collaborating with Nitto Denko for the development of treatments to serious fibrotic diseases. The collaboration utilizes Quark’s technology and intellectual property in RNAi, potentially combined with Nitto Denko’s delivery technology to identify, select, optimize and develop a new siRNA drug based on a therapeutic concept and target gene identified by Nitto Denko and its collaborating scientists.

Intellectual Property
We have assembled a strong intellectual property position on the composition of matter of our drug candidates, siRNA structures, delivery methodologies and target genes that we have discovered. We own or control a portfolio of patents and patent applications to protect our technologies and products comprised of over 79 granted or allowed patents in the U.S., Europe and other countries, and approximately 140 patent applications in the U.S. and throughout the world.

Quark is a privately held company incorporated in California. Our headquarters is located at 6501 Dumbarton Circle, Fremont, CA 94555, USA, and we have R&D facilities at 2990 Wilderness Place, in Boulder Colorado 80301 and at QBI Enterprises, Ltd. in Nes Ziona, Israel. QBI Enterprises, Ltd. is a wholly owned subsidiary of Quark Pharmaceuticals, Inc.