First Synthetic siRNA Administered Systemically to Humans for the Prophylaxis of Acute Kidney Injury and Delayed Graft Function
FREMONT, Calif., Jan. 5 Quark Pharmaceuticals, Inc., the world leader in clinical development of RNAi-based therapeutics, announced today that the independent Data Safety Monitoring Board (“DSMB”) recommended that QPI-1002 (I5NP) continue on to the next phase of clinical testing. This followed review of available safety data from two clinical trials for Quark’s siRNA QPI-1002, at doses up to 10 mg/kg, the highest evaluated to date. QPI-1002 is designed to temporarily inhibit expression of the stress-response gene, p53, and is the first systemically administered siRNA drug to enter human clinical trials.
The DSMB is an independent group of experts enlisted by Quark to review and evaluate the safety data generated from the dose-escalation portions of Quark’s clinical program for QPI-1002. These include a Phase 1/2 study in renal transplant patients for the prophylaxis of delayed graft function (DGF) and the Phase 1 trial for prevention of acute kidney injury (AKI) in patients undergoing major cardiovascular surgery. The primary responsibility of the DSMB is to review data in order to make recommendations regarding the continuation, modification or termination of the trial due to safety concerns.
Daniel Zurr, Ph.D., President and Chief Executive Officer of Quark Pharmaceuticals, said, “The recommendation of the DSMB represents a landmark in the development of synthetic siRNAs, as QPI-1002 is the first siRNA to be administered systemically to humans. This recommendation by the DSMB enables Quark to move forward with our human clinical trials for QPI-1002 in both AKI and DGF. Quark has an established track record for taking siRNAs into human clinical trials at a rate unmatched in the industry. Part of the Company’s ongoing mission is to bring novel and clinically relevant siRNA compounds to health care providers. By the end of 2010, Quark will have three compounds in clinical studies in five different disease indications, making Quark the industry leader in developing therapeutics in the Nobel Prize-winning field of RNAi.”
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc. is a leader in the discovery and development of novel RNAi therapeutics. Quark has a fully integrated drug development platform that spans 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 delivery allows targeting of tissues and organs including the eye, kidney, ear, lung, spinal cord and brain.
Quark’s partner, Pfizer Inc, currently is evaluating PF-4523655 (RTP801i-14) 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(TM). Quark’s development pipeline also features 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 after kidney transplantation. Enrollment was successfully completed recently in Phase I studies in these indications. For the composition of these products, Quark has obtained licenses from Silence Therapeutics and from Alnylam Pharmaceuticals.
In the first quarter of 2010, Quark will begin a Phase 1/2 study of a new synthetic siRNA, QPI-1007, as a neuroprotective agent for eye diseases. QPI-1007 utilizes a proprietary composition with freedom to operate in the siRNA intellectual property arena that was developed in collaboration with BioSpring GmbH. The structure and modifications utilized in QPI-1007 preserve RNAi activity while ameliorating potential off-target and immunostimulatory effects of siRNAs. In addition, Quark has a broad pipeline of siRNA drug candidates that have arisen from Quark’s research activities. The Company is committed to developing novel siRNA structures and expects to utilize these improvements 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.