Quark Granted European Orphan Drug Status for QPI-1002 for the Prophylaxis of Delayed Graft Function in Kidney Transplant Patients

FREMONT, Calif., June 15 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, today announced that the European Commission has granted Orphan Medicinal Product Designation for QPI-1002 (also referred to as "I5NP") for the prophylaxis of delayed graft function (DGF) in kidney transplant patients. QPI-1002 is a synthetic siRNA targeting p53 mRNA and is the first synthetic siRNA to be administered systemically to humans. The designation is granted under the name of the European sponsor, Verius, Ltd. of the United Kingdom.

Dr. Daniel Zurr, Quark's Chief Executive Officer, stated, "The addition of orphan designation for the European Union, following the US FDA orphan designation for Quark's QPI-1002 announced in February, provides the opportunity for regulatory support for this indication in that region as well. We believe the EU renal transplant community will welcome development of QPI-1002 with the same enthusiasm already demonstrated in the US. A therapy that succeeds in reducing the incidence and severity of DGF may improve longer-term graft survival and help to reduce the widening gap between donor organ availability and the number of patients awaiting transplantation."

Orphan designation in the EU grants special status to a product to treat a rare disease or condition affecting less than five in 10,000 persons in the EU per year. The provisions for the EU orphan designation allow for incentives to pharmaceutical companies developing such products, thus providing better access to treatments for patients having conditions that otherwise might not be investigated. For marketing approved products this includes potential market exclusivity of up to 10 years in the EU as well as various fee reductions for certain regulatory activities.

About Delayed Graft Function (DGF) in Kidney Transplantation

DGF is one of the most common complications during the immediate postoperative period in renal transplantation and affects 25-40% of the deceased donor renal transplants in the United States and Europe. DGF most often results from ischemia/reperfusion injury when blood flow is re-established to the kidney following transplantation and initiates a chain of events that can lead to severe renal damage.  DGF is associated with longer hospital stays and higher rates of graft rejection, which decreases transplanted kidney survival (graft survival). There is no currently marketed drug therapy for the preventive or treatment of DGF.

Ongoing Study for DGF

Quark has completed enrollment for the dose escalation safety portion (Part A) of a multi-center, two-part Phase 1/2 clinical trial for prophylaxis of delayed graft function (DGF) in patients undergoing deceased donor kidney transplantation. An independent Data Safety Monitoring Board (DSMB) has recommended continuation as planned to evaluate the safety and potential clinical activity of selected dose(s) of QPI-1002 in the Phase II portion (Part B) of the study. Part B of the study is expected to commence enrollment of adult kidney transplant recipients by mid 2010.  Additional information on this study may be found at www.clinicaltrials.gov.  

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-04523655, currently in two Phase II clinical trials for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME). The siRNA therapeutic candidate was licensed to Pfizer in 2006 and both trials are being conducted by Pfizer in collaboration with Quark. PF-04523655 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 several families of patents covering the RTP801 gene, its RNA and protein product sequences, specific antibodies, and gene inhibition across different pathologies.

Quark's 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 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 also conducting clinical trials of QPI-1007, its first proprietary synthetic siRNA drug candidate. QPI-1007 utilizes a proprietary structure developed in collaboration with BioSpring GmbH. The proprietary structure provides Quark freedom to operate in the siRNA intellectual property arena and the chemical modifications 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

SOURCE Quark Pharmaceuticals, Inc.