Quark Pharmaceuticals, Inc. Presented Positive Preclinical Results of Systemic RNAi Compound for Acute Renal Failure (ARF)
omniomix.com
Thu Aug 2 07:18:38 2007
Quark to initiate Phase 1 trial of AKIi-5 as the first human clinical study
involving the systemic delivery of siRNA
FREMONT, Calif., Aug. 2 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on discovering and developing novel RNA interference-based therapeutics, announced today the presentation of positive results from preclinical efficacy studies for its proprietary systemically-administered, siRNA compound, AKIi-5, in acute renal failure (ARF).
In the study, rats treated with a single bolus injection of rat AKIi-5, Quark's proprietary siRNA compound targeting the p53 gene, were significantly protected from ischemia/reperfusion-induced acute kidney injury. The compound was most efficacious when administered within a well-determined time window of 2-4 hours post injury. The study also proved that AKIi-5 has a favorable drug disposition (PK) profile, with short local exposure predominantly to the kidney-exhibiting rapid clearance of the drug post-administration. Quark's preclinical studies to date also indicate that AKIi-5 has a favorable safety profile, with a therapeutic index (toxic dose/effective dose) in rats greater than 250.
Elena Feinstein, M.D., Ph.D., the Company's Chief Scientific Officer, presented the results of the preclinical efficacy studies in a poster session at the Beyond Genome 2007 conference in San Francisco, CA. Quark's poster presentation was based on unpublished data. The studies were performed in collaboration with Bruce A. Molitoris, M.D., Director of the Division of Nephrology and Professor of Medicine at Indiana University School of Medicine, and lead academic investigator in the Quark program.
Daniel Zurr, CEO of Quark, commented, "Our pharmacokinetic, distribution, and toxicity studies indicate that siRNA-mediated, temporary inhibition of p53 by our systemically administered drug, AKIi-5, is a safe and efficient way to treat and prevent acute kidney injury caused by ischemia-reperfusion. Our drug could potentially offer a lifesaving treatment for this severe disease, which exhibits high rates of morbidity and mortality and represents a very serious unmet medical need. We have an open IND for AKIi-5 and expect to initiate a Phase I trial for the prevention of ARF through the systemic delivery of AKIi- 5 in patients undergoing major cardiac surgery. Based on publicly available information, we believe that this will be the first human clinical trial involving the systemic delivery of siRNA."
About AKIi-5
AKIi-5 is a synthetic, chemically modified siRNA molecule designed to temporarily inhibit the expression of p53, a gene which plays a significant role in ARF by inducing tubular cell death (apoptosis) in response to injury. AKIi-5 is based on Quark's proprietary, patented concept of temporary and reversible inhibition, for therapeutic purposes, of the expression of the transcription factor human p53, which is associated with DNA repair and apoptosis. Using RNA interference technology to temporarily inhibit p53 in acute settings such as acute kidney injury, apoptosis is delayed thereby allowing natural repair mechanisms to restore normal DNA and cellular integrity. The AKIi-5 molecule was designed and patented by Quark. The Company has licenses for AtuRNAi(TM) technology from Silence Therapeutics and additional RNAi intellectual property from Alnylam.
About Acute Renal Failure (ARF)
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