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Dicerna On Track with Drug Candidates, Initial Cancer Rx Data Expected in '15


NEW YORK (GenomeWeb) – Dicerna Pharmaceuticals this month confirmed previous guidance on its two drug candidates, the solid tumor therapy DCR-MYC and the primary hyperoxaluria 1 (PH1) treatment DCR-PH1, stating that both agents remain on track to enter new clinical trials later this year and in 2015, respectively.

The newly minted public company also released its financial results for the second quarter, reporting higher losses amid increased research and development spending.

DCR-MYC comprises the company’s Dicer-substrate RNAi molecules formulated in its EnCore lipid nanoparticles. It is designed to silence Myc, a regulator gene mutated in a variety of cancers, as a way to treat hepatocellular carcinoma.

In April, Dicerna announced that it had started a Phase I study examining escalating, once-weekly infusions of the drug to up to 66 patients with solid tumors, multiple myeloma, or lymphoma who are refractory or unresponsive to other treatments. Endpoints of the trial include determination of the drug's maximum tolerated dose, as well as its pharmacokinetics, pharmacodynamics, and potential antitumor activity.

Top-line data from this study is expected in 2015. Further, Dicerna continues to plan for a second Phase I trial in liver cancer patients in the second half of 2014.

Meanwhile, the firm's second clinical candidate, DCR-PH1, remains on track for a Phase I study next year.

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 candidate is designed to inhibit HAO1, a gene that produces glycolate oxidase, which is an enzyme upstream of AGT1.

For the three-month period ended June 30, Dicerna's net loss was $11.4 million, compared with a year-ago loss of $3.8 million.

Contributing to the increase was a rise in R&D costs to $6.8 million from $2.5 million, as well as higher general and administrative expenditures of $4.4 million versus $1.1 million the same period a year before.

At the end of the second quarter, Dicerna had cash and cash equivalents totaling $120.3 million.

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