Pharmaceutical

Dicerna Pharmaceuticals (Nasdaq:DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the Company has dosed the first primary hyperoxaluria (PH) patient with DCR-PHXC in the Group B portion of the PHYOX Phase 1 clinical trial. DCR-PHXC is an investigational GalXC™ product candidate in development for the treatment of all forms of …

Dicerna Pharmaceuticals (Nasdaq:DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the Company has dosed the first primary hyperoxaluria (PH) patient with DCR-PHXC in the Group B portion of the PHYOX Phase 1 clinical trial. DCR-PHXC is an investigational GalXC™ product candidate in development for the treatment of all forms of PH, a family of severe, rare, inherited disorders of the liver that often result in kidney failure.

As quoted in the press release:

“The Orphan Drug Designation for DCR-PHXC for the treatment of primary hyperoxaluria and the dosing of the first primary hyperoxaluria patient in the Group B portion of the PHYOX trial are important milestones for the Company as we seek to develop the first GalXC-based RNAi therapeutic to treat patients with all forms of this serious, life-threatening disease,” said Ralf Rosskamp, M.D., chief medical officer of Dicerna. “While the PHYOX study remains blinded to treatment assignment, the early safety and tolerability data from healthy volunteers in Group A are encouraging. The advancement of DCR-PHXC demonstrates our commitment to progressing novel science that has the potential to make a meaningful difference for this underserved patient population.”

The PHYOX trial is a Phase 1, 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 study that has enrolled 25 NHVs; Group B is an open-label, multi-center study enrolling up to 16 patients with PH type 1 (PH1) and PH type 2 (PH2).

Click here to read the full press release.

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