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Quark Pharmaceuticals Presents Data on Synthetic siRNAs for Prevention of Ocular and Inner Ear Sensory Cell Damage

Posted on: Thursday, 14 May 2009, 07:00 CDT

FREMONT, Calif., May 14 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a development-stage pharmaceutical company discovering and developing novel RNA interference (RNAi)-based therapeutics, today announced that Elena Feinstein, M.D., Ph.D., Chief Scientific Officer, presented a study titled, "siRNA Therapeutics for Prevention of Ocular and Inner Ear Sensory Cells Death," at the DxRx Summit: Nucleic Acid Diagnostics & Therapeutics. The event was held May 12-13, 2009 in Boston, MA.

Dr. Feinstein presented data demonstrating that synthetic, chemically-modified 19-bp siRNAs, including PF 04523655 currently being studied by partners Pfizer and Quark in patients with diabetic macular edema (DME) and age-related macular degeneration (AMD), enter cells in the retina of the eye and exert their therapeutic effects while avoiding non-specific activation of the toll-like receptor 3 (TLR3) - the effect reported for other synthetic siRNAs. Similar data for QPI-1007, Quark's proprietary siRNA composition, were also reported. QPI-1007 was demonstrated to confer effective ocular neuroprotection and is currently in late-stage IND-enabling studies.

In addition to studies in the eye, Dr. Feinstein also reported results demonstrating effective delivery of synthetic siRNAs to the inner ear, which resulted in protection of hearing epithelium from ototoxic and noise-induced damage. These studies were performed in collaboration with Prof. Jayakrishna Ambati, University of Kentucky College of Medicine, Lexington, KY; Prof. Ann Logan, University of Birmingham, UK; Prof. Adriana Di Polo, University of Montreal; and Prof. Richard Salvi, SUNY University at Buffalo.

Daniel Zurr, Ph.D., President and Chief Executive Officer, said, "Results from the studies presented at DxRx demonstrate our successful track record in discovering and advancing industry-leading siRNA product candidates. Our strong position in the RNAi landscape has been validated by our ability to secure co-development agreements with major pharmaceutical partners, including Pfizer, to advance our siRNA product candidates further towards commercialization."

Dr. Feinstein added, "Our synthetic, chemically modified siRNA product candidates have been designed to elicit pharmacologic effects through target gene-knock down while avoiding potentially harmful off-target effects reported for other synthetic siRNAs. These results support our capabilities to discover and develop siRNA product candidates with greater specificity and reduced side effects."

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-04523655 (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-04523655 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.

Quark Pharmaceuticals, Inc. The Ruth Group (investors / media) Juliana Friedmann Sara Ephraim Pellegrino / Janine McCargo +972 89 30 5111 (646) 536-7002 / 7033 jfriedman@quarkpharma.com spellegrino@theruthgroup.com jmccargo@theruthgroup.com

SOURCE Quark Pharmaceuticals, Inc.

Source: PR Newswire

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