Quark Pharmaceuticals Announces the Presentation of Data Indicating Potential Utility of QPI-1002 in Chronic Kidney Disease at the RNA Interference Summit
FREMONT, Calif., June 9 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a development-stage pharmaceutical company discovering and developing novel RNA interference (RNAi)-based therapeutics, today announced that Bruce A. Molitoris, M.D., Director, Division of Nephrology and Professor of Medicine, Indiana University, will present data demonstrating efficacy in models of acute and chronic kidney disease (AKI and CKD, respectively) of an animal analogue of QPI-1002 at the RNA Interference Summit being held June 8-10, 2009 in San Francisco, CA. QPI-1002, a siRNA drug candidate being developed by Quark Pharmaceuticals, Inc. is the first systemically-administered siRNA to enter human clinical trials. The drug is the subject of two multi-center Phase I studies for acute kidney injury (AKI) and one Phase I/II study for delayed graft function (DGF) in kidney transplantation. QPI-1002 targets p53, a gene that plays a pivotal role in the stress-response apoptotic pathway.
Using intravital 2-photon in life microscopy to follow the distribution of fluorescent labeled siRNA in the rat kidney, Prof. Molitoris demonstrated rapid and predominant distribution of the siRNA to kidney proximal tubular cells (PTC), which are the main type of suffering cells in the context of ischemic or toxic kidney injury. Dose response and time course studies exploring efficacy of the animal analogue of QPI-1002 in the AKI models in rats indicated excellent efficacy of the drug in reducing serum creatinine levels and ameliorating acute morphological damage to the kidney including PTC death. The data indicate that recurrent dosing of the drug concomitantly with monthly repetitive ischemic kidney insults minimizes the resulting kidney injury and can attenuate development of associated chronic kidney disease (CKD).
Daniel Zurr, Chief Executive Officer, commented, "We are excited to see this as a possible additional indication for QPI-1002; the compound is already being studied as the first systemically-dosed siRNA to be tested in humans for AKI following major cardiovascular surgery, and for delayed graft function (DGF) following kidney transplantation. CKD is a very serious condition leading to kidney failure and complications such as cardiovascular disease (CVD). In America alone, 26 million adults suffer from CKD and millions of others are at increased risk. We are eager to continue these studies to provide a remedy for this disease."
Dr. Molitoris said, "The current standard of care in the treatment of CKD is to help slow the rate of damage to the kidneys by treating the condition causing the disease and to modify the patient's diet and exercise. I'm excited by these data indicating that QPI-1002 could be an additional therapeutic tool to minimize the AKI associated progression of chronic kidney disease, which inevitably leads to loss of kidney function and development end stage renal disease that necessitates dialysis or a kidney transplant to maintain life. It would be gratifying to see a treatment that directly targets the condition of CKD, regardless of the underlying cause."
QPI-1002 is a synthetic, chemically modified siRNA molecule based on Quark's proprietary, patented invention of temporarily and reversibly inhibiting a master stress-response gene p53. QPI-1002 drug is also protected by Quark's patents. Temporary inhibition of p53 at the time of injury delays massive death of vulnerable affected cells, allowing natural repair mechanisms to take over, thus preserving tissue integrity and function. In acute kidney injury (AKI), the p53 gene induces tubular cell death resulting in kidney dysfunction. In the context of AKI, RNA interference (RNAi) technology used to temporarily inhibit p53 exploits the siRNA property to predominantly accumulate in target renal cells that activate p53 and are consequently prone to death. In collaboration with the University of Illinois at Chicago, Quark first published its therapeutic concept in a seminal paper in Science magazine (Science. 1999 Sep 10; 285).
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc. is a development-stage pharmaceutical company engaged in discovering and developing novel RNAi-based therapeutics. Quark has a fully integrated drug development platform that spans therapeutic target identification to drug development. Quark's RNAi technology includes novel siRNA structures and chemistry providing Quark with freedom to operate in the siRNA intellectual property arena, as well as the ability for non-invasive delivery of siRNA to other target tissues including the eye, ear, lung, and CNS.
In addition to QPI-1002, Quark's clinical pipeline includes PF-4523655 (RTP801i-14), licensed to Pfizer, which is currently in Phase II clinical trials in 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 discovered by Quark through the gene discovery platform BiFAR(TM). For the structure of these products, Quark has licenses from Silence Therapeutics and from Alnylam Pharmaceuticals. QPI-1007, a siRNA that utilizes a proprietary structure developed by Quark, is being evaluated in IND-enabling nonclinical studies as a neuroprotective agent for eye diseases. In addition, Quark has a broad pipeline of siRNA drug candidates based on internally developed novel structures.
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. The Ruth Group (investors / media)
Juliana Friedman Sara Ephraim Pellegrino / Janine McCargo
+972 89 30 5111 (646) 536-7002 / 7033
Source: Quark Pharmaceuticals, Inc.
Posted: June 2009