Dicerna Pharmaceuticals has dosed the first patient in the Group B portion of the PHYOX trial, a Phase l clinical study of DCR-PHXC for the treatment of primary hyperoxaluria (PH).
The single-ascending dose trial comprises two groups, A and B.
Group A is a placebo-controlled, single-blind, single-centre trial, which has enrolled 25 healthy subjects.
Group B, which is an open-label, multi-centre trial plans to enrol up to 16 patients with PH type 1 (PH1) and PH type 2 (PH2).
PHYOX’s primary objective is to investigate the safety and tolerability of single doses of DCR-PHXC in both the groups.
Its secondary objectives are the evaluation of the pharmacodynamic effect of single doses of DCR-PHXC on biochemical markers, as well as changes in urine oxalate concentrations, and characterisation of the pharmacokinetics of single doses of DCR-PHXC in healthy subjects and PH patients.
Clinical proof-of-concept data from the PHYOX trial is expected to be available in the second half of this year.
Dicerna has already completed the Group A portion of the trial and has reported topline results from that portion.
Dicerna Pharmaceuticals chief medical officer Ralf Rosskamp said: “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.”
DCR-PHXC is an investigational drug in development for the treatment of all forms of PH, a type of severe, rare, genetic liver disorders caused by overproduction of oxalate, a natural chemical in the body that is normally removed as waste through the kidneys.
The drug has recently received orphan drug designation from the US Food and Drug Administration (FDA).