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Dicerna Doses First Human Subject in Clinical Trial of GalXC RNAi Product for Treatment of Primary Hyperoxaluria.

M2 PHARMA-December 11, 2017-Dicerna Doses First Human Subject in Clinical Trial of GalXC RNAi Product for Treatment of Primary Hyperoxaluria


- Cambridge, Massachusetts-based investigational RNA interference (RNAi) therapeutics developer Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA) has dosed the first human in DCR-PHXC-101, a Phase 1 clinical trial of DCR-PHXC, the company's most advanced GalXC product candidate, the company said.

GalXC is a proprietary platform invented by Dicerna scientists to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver.

Dicerna is investigating DCR-PHXC for the treatment of primary hyperoxaluria, a group of severe, rare, inherited disorders of the liver that often result in kidney failure.

The DCR-PHXC-101 clinical trial is a single ascending-dose study of DCR-PHXC in normal healthy volunteers (NHVs) and patients with PH. The study is divided into two groups: Group A is a placebo-controlled, single-blind, single-center Phase 1 study in NHVs; Group B is an open-label, multi-center study in patients with PH types 1 and 2.

The primary objective for the trial is to evaluate the safety and tolerability of single doses of DCR-PHXC in both groups. Secondary objectives are to characterize the pharmacokinetics of single doses of DCR-PHXC in NHVs and patients with PH, and to evaluate the pharmacodynamic effects of single doses of DCR-PHXC on biochemical markers including, but not limited to, changes in urine oxalate concentrations.

DCR-PHXC targets the lactate dehydrogenase A gene, a potentially optimal therapeutic target in patients with PH. DCR-PHXC yields potent, liver-specific LDHA inhibition in animal models of PH, an effect that reduces oxalate to near-normal levels, which may prevent the damage to the kidneys and other organs caused by oxalate accumulation.

Dicerna Pharmaceuticals is focused on the discovery and development of innovative RNAi-based therapeutics for diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases.

The company is leveraging its proprietary GalXC RNAi technology platform to build a broad pipeline in these core therapeutic areas, focusing on target genes where connections between target gene and diseases are well understood and documented.

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Publication:M2 Pharma
Date:Dec 11, 2017
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