FREMONT, Calif., March 1st, 2010 – Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, announced today the dosing of the first patient in a Phase I study of the investigational neuroprotective agent, QPI-1007. This drug candidate, having a proprietary siRNA structure, is being developed for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION). The current trial represents the seventh clinical study of Quark synthetic siRNAs. QPI-1007 has been evaluated in several preclinical models of ocular neuroprotection, and has been shown in these models to protect retinal ganglion cells from injury-induced apoptosis.
The study is an open-label, dose escalation, safety, tolerability and pharmacokinetic study, where active study drug will be given to all patients who participate. This study will determine whether QPI-1007 is safe when it is injected into the eye and will also reveal if there are any side effects of the drug and how long it takes for the body to clear the drug. In addition, the study will examine the biological activity of the drug in NAION patients. The study will enroll patients in two strata, and is being conducted in the US and Israel. Stratum I will enroll legally blind patients, while stratum II will enroll patients with recent onset of NAION.
Shai Erlich, Ph.D., Chief Medical Officer of Quark, commented, “Based on the pre-clinical results we’ve seen for QPI-1007, we are pleased to initiate dosing in human clinical trials. QPI-1007 is a siRNA molecule with Quark’s proprietary structure and its entry into clinical trials reflects the Company’s success in developing novel siRNA compounds. These accomplishments further validate Quark’s ability to advance innovative product candidates from discovery into the clinic.”
Dr. Daniel Zurr, Quark’s Chief Executive Officer, said, “The initiation of dosing in this trial brings the total number of siRNA programs in the clinic to five, an unprecedented number in the RNAi industry. Beyond the size of our clinical stage siRNA portfolio, we are proud that this particular product, QPI-1007, was developed using entirely independent structural IP, further demonstrating our leadership in the field.”
QPI-1007 is Quark’s first siRNA drug candidate to utilize intellectual property developed internally in collaboration with BioSpring GmbH, and gives Quark freedom to operate in the siRNA IP space. Preclinical studies have demonstrated a robust neuroprotective effect of QPI-1007 when administered immediately after injury induced by optic nerve crush or axotomy in two models of retinal ganglion cell (RGC) death as well as in a rat intraocular pressure (IOP) elevation model in which the drug was injected 2 weeks after IOP elevating operation.
NAION is an acute ischemic disorder of the anterior optic nerve. It is believed to be caused by acute ischemia of the optic nerve head resulting from insufficient arterial blood flow via the posterior ciliary artery. Vision loss resulting from NAION is typically sudden, painless, and accompanied by pallid swelling of the optic disc. In most cases patients have permanent severe visual deficits . There is currently no approved treatment for NAION
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 for 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 addition, Quark has a broad pipeline of siRNA drug candidates that have arisen from its 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 its 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 atwww.quarkpharma.com.