Shares of Dicerna Pharmaceuticals Inc. (DRNA) - Get Dicerna Pharmaceuticals, Inc. Report slumped on Friday after the drug developer reported mixed results from a clinical trial of a treatment for primary hyperoxaluria, a genetic disorder that can harm the kidneys.
The trial included participants with primary hyperoxaluria subtypes 1 and 2.
And while the company saw positive results and plans to seek approval of nedosiran for the type 1 ailment, the type 2 data "showed inconsistent results."
"The results reinforce nedosiran’s potential to be a therapeutic option for patients with PH1, if approved," Executive Vice President Shreeram Aradhye said in a statement.
Dicerna shares at last check dropped 27% to $27.79. On July 20 they'd touched a 52-week high above $40.
The company expects to submit its new-drug application to the U.S. Food and Drug Administration for nedosiran in the fourth quarter.
Nedosiran met the primary goal of the trial, showing statistically and clinically significant sustained reduction in urinary oxalate excretion. (Hyperoxaluria occurs when a person has too much oxalate in his or her urine, the Mayo Clinic says.) A secondary endpoint was also met.
The reduction in Uox excretion seen in patients with PH1 confirms "the ability of Dicerna’s GalXC RNAi technology to silence disease-driving genes, derisking our growing pipeline of GalXC product candidates," Aradhye said.