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news.Quark Pharmaceuticals has reported promising results from preclinical efficacy studies for its siRNA compound in acute renal failure.
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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 two-four hours post injury, Quark said. The study also indicated that AKIi-5 has a favorable safety profile.
The drug is part of a new class of therapeutics known as RNA interference. AKIi-5 is designed to temporarily inhibit the p53 gene, which is responsible for controlling cell death (apoptosis). In acute settings such as acute kidney injury, apoptosis is delayed thereby allowing natural repair mechanisms to restore normal DNA and cellular integrity.
Daniel Zurr, CEO of Quark, said: “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.”