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Dicerna Presents Preclinical Data on Primary Hyperoxaluria Type 1 Drug


NEW YORK (GenomeWeb) – Dicerna Pharmaceuticals this week announced preclinical data showing that its investigational primary hyperoxaluria type 1 (PH1) therapy DCR-PH1 could silence expression of its target by as much as 97 percent in a mouse model of the disease.

PH1 is a rare, inherited autosomal-recessive condition characterized by the liver's inability to metabolize a precursor of oxalate due to disruption of an enzyme called alanine-glyoxylate aminotransferase 1 (AGT1). As a result, calcium oxalate builds up in renal tubules causing kidney stones and fibrosis.

Dicerna's drug is designed to inhibit HAO1, a gene that produces glycolate oxidase, which is an enzyme upstream of AGT1.

In the preclinical studies, which were detailed at the 11th International Primary Hyperoxaluria Workshop, Dicerna and academic collaborators specifically demonstrated that a single dose of DCR-PH1 led to a 97 percent reduction of the HAO1 transcript in the liver and significant reductions in urinary oxalate to near baseline levels.

Dicerna has said it expects to begin Phase I testing of DCR-PH1 in 2015.