FREMONT, Calif., February 16, 2010 – Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, announced today that the US FDA has granted orphan drug designation for QPI-1002 (also referred to as “I5NP”), a synthetic siRNA targeting p53 mRNA for the prophylaxis of delayed graft function (DGF) in kidney transplant patients. Quark has completed enrollment and dosing in the dose-escalation safety portion (Part A) of a Phase 1/2 study and has received a positive recommendation from an independent Data Safety Monitoring Board (DSMB) to progress the drug to the second portion (Part B) of the study to evaluate the safety and potential clinical activity of QPI-1002
“DGF is currently an unmet medical need, and we anticipate a great deal of interest in QPI-1002 from the renal transplant community. We are confident that obtaining orphan status will facilitate the rapid development of this innovative compound and hope that ultimately, we can reduce the wait time for kidney transplants with fewer failed grafts,” Daniel Zurr, Ph.D., President and Chief Executive Officer of Quark Pharmaceuticals commented.
US orphan drug designation is granted to products intended to treat a rare disease or condition affecting less than 200,000 Americans. The Orphan Drug Act allows for possible R&D incentives and qualifies the sponsor for tax credits and a period of market exclusivity following approval.
QPI-1002, the first systemic siRNA drug to enter human clinical trials, is an investigational drug designed to temporarily inhibit the expression of the pro-apoptotic gene, p53, to protect normal cells from acute injury. Preclinical studies have shown that p53-targeted siRNAs can protect kidneys from ischemia/reperfusion injury that can occur following transplantation.
About Delayed Graft Function (DGF) in Kidney Transplantation
DGF is one of the most common complications during the immediate postoperative period in renal transplantation, affecting 25-40% of deceased donor renal transplants. 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 or the prevention or treatment of DGF.
Ongoing Study for DGF
QPI-1002 has completed enrollment for the dose escalation safety portion (Part A) of a multi-center, two-part Phase 1/2 clinical trial for prevention 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 a selected dose of QPI-1002 in the second portion (Part B) of the study. Part B of the study is expected commence enrollment of adult kidney transplant recipients in the second quarter of 2010. Additional information on this study may be found at www.clinicaltrials.gov.
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc., is a leader in the discovery and development of novel RNAi-based drugs. Quark has a fully integrated drug development platform spanning therapeutic target identification to drug development. The Company’s technology platform includes novel disease targets and siRNA structures and chemistry, providing Quark with freedom to operate in the siRNA intellectual property arena. Quark’s approach to therapeutic delivery allows targeting of tissues and organs including the eye, kidney, ear, lung, spinal cord and brain.
Quark’s partner, Pfizer Inc, is advancing PF-4523655 (RTP801i-14), currently in two Phase 2 clinical studies in patients with diabetic macular edema (DME) and age-related macular degeneration (AMD). PF-4523655 is a synthetic, chemically modified siRNA designed to inhibit the expression of the gene RTP801 that was discovered by Quark through the gene discovery platform BiFAR™.
Quark’s clinical pipeline includes QPI-1002, the first systemically administered siRNA drug in human clinical trials. QPI-1002 is being evaluated for the prevention of acute kidney injury (AKI) following major cardiovascular surgery and the prophylaxis of delayed graft function (DGF) after kidney transplantation. Enrollment was successfully completed in a Phase I study and in the Part A portion of a Phase 1/2 studies in AKI and DGF, respectively. Quark has licenses for the structure for these product candidates from Silence Therapeutics and Alnylam Pharmaceuticals.
In the first quarter of 2010, Quark will begin a Phase 1/2 study of its latest siRNA drug candidate, QPI-1007, as a neuroprotective agent for eye diseases. QPI-1007 utilizes a proprietary structure with freedom to operate in the siRNA intellectual property arena developed in collaboration with BioSpring GmbH. In addition, Quark has a broad pipeline of siRNA drug candidates that have arisen from Quark’s research activities. The Company is committed to development of novel siRNA structures and expects to utilize these structures to develop additional RNAi drug candidates based on the Company’s productive R&D engine.
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.