Highlights First Systemic siRNA Administered in Humans and Quark’s Proprietary siRNA Structures
Fremont, CA – March 9, 2009 – Quark Pharmaceuticals, Inc., a development-stage pharmaceutical company discovering and developing novel RNA interference (RNAi)-based therapeutics, today announced that James D. Thompson, Ph.D., Vice President, Pharmaceutical Development presented an overview of Quark’s novel pipeline of synthetic siRNA compounds at the Asia TIDES Oligonucleotide and Peptide® Technology and Product Development Conference, held February 24th, 2009 in Tokyo, Japan.
Dr. Thompson’s presentation, entitled “Design and Development Strategies for Clinical Applications of Synthetic siRNAs,” highlighted Quark’s ongoing clinical studies. His case study on QPI-1002 detailed the first systemic administration of a synthetic siRNA in man, results from IND-enabling efficacy and toxicology studies and the largest cGMP manufacture of synthetic siRNA of its time. QPI-1002, also known as I5NP and AKIi-5, is currently being evaluated in two Phase I/IIa clinical studies for the prevention of acute kidney injury (AKI) and a Phase I/II clinical study for the prophylaxis of delayed graft function (DGF) following renal transplantation.
In addition to the case study of QPI-1002, Dr. Thompson provided an update on QPI-1007, Quark’s first proprietary siRNA drug candidate based on its intellectual property covering a host of novel structures. The chemically modified siRNA exhibits optimal activity and stability while attenuating potential off-target effects. The novel structures developed in collaboration with BioSpring GmbH give Quark freedom to operate in the siRNA IP space. QPI-1007 is in advanced IND-enabling preclinical studies for the treatment of non-arteritic anterior ischemic optic neuropathy and has demonstrated proof-of-concept efficacy as a neuroprotective agent in animal models of eye diseases.
Dr. Thompson, commented, “Quark has kept pace or out-performed on every major benchmark by which RNAi therapeutics companies are measured. With QPI-1002, Quark became the first to administer a siRNA drug systemically in humans. Our proprietary structures have supplied us with freedom to operate in the siRNA IP space and Quark is currently developing a pipeline of novel synthetic siRNA compositions starting with QPI-1007.”
Dr. Thompson has been at the forefront of siRNA development for several years. Prior to joining Quark, he was Director of Research and Development at Genta, and previously was Director of Biology Research at Ribozyme Pharmaceuticals, which was acquired by Merck after changing its name to Sirna Therapeutics.
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, spinal cord and brain.
PF-4523655 (RTP801i-14), currently in Phase II clinical trials, is a synthetic, chemically modified siRNA designed to inhibit the expression of the gene RTP801 discovered by Quark through the gene discovery platform BiFAR. PF-4523655 is licensed to Pfizer. In addition, Quark’s current clinical pipeline includes QPI-1002, the first systemically administered siRNA drug in human clinical trials, developed by Quark for the prevention of acute kidney injury (AKI) following major cardiac surgery and of delayed graft function in kidney transplantation. 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 advanced IND-enabling preclinical studies as a neuroprotective agent for eye diseases. In addition, Quark has a broad pipeline of siRNA drug candidates based on novel structures developed internally. The Company expects to utilize the 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