Dicerna Presents PHYOX™2 and Primary Hyperoxaluria Healthcare Utilization Data at American Society of Nephrology (ASN) Kidney Week 2021
Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced results of the PHYOX™2 pivotal clinical trial of nedosiran, an investigational GalXC™ RNAi candidate for the treatment of primary hyperoxaluria (PH), in a late-breaker poster presentation at the American Society of Nephrology (ASN) Kidney Week 2021.
In the PHYOX2 pivotal trial, which included patients with PH type 1 (PH1) and PH type 2 (PH2), nedosiran demonstrated a statistically significant reduction from baseline in urinary oxalate (Uox) excretion compared to placebo. Additionally, a significantly higher proportion of patients treated with nedosiran achieved and sustained normal or near-normal Uox at two or more consecutive visits after Day 90 compared to placebo (p=0.0025). A post hoc subgroup analysis in participants with high baseline Uox (at least one value ≥ 1.6 mmol) also showed significantly greater Uox reduction in those treated with nedosiran (p=0.0186). These robust and sustained Uox reduction results observed in PHYOX2 were primarily driven by response to nedosiran in the PH1 subgroup, which met both primary and key secondary endpoints based on a post hoc analysis. There was no consistent pattern of Uox reduction observed in participants with PH2.
“Our pivotal PHYOX2 data emphasize the potential of nedosiran to be a safe and effective therapy option for people with PH1,” said Shreeram Aradhye, M.D., Executive Vice President and Chief Medical Officer at Dicerna. “Primary hyperoxaluria can lead to life-threatening kidney damage, and there continues to be an urgent need to provide the medical community with new therapeutic options and build an understanding of disease biology across the PH subtypes.”
“For people with primary hyperoxaluria, excessive oxalate production compromises kidney function and can have severe consequences,” said David Goldfarb, M.D., Clinical Chief of Nephrology at NYU Langone Health and co-director of its Kidney Stone Prevention Program. “More than 80% of people with PH1 taking nedosiran achieved normal or near-normal urinary oxalate excretions at Day 180 in the PHYOX2 trial. These data underscore the potential of nedosiran to be a meaningful treatment option for those affected by PH1, if approved.”
Separately, findings from a real-world study examining de-identified electronic medical record (EMR) data, including demographics, clinical characteristics and healthcare utilization among 47 patients with PH pre- and post-dialysis treatment were also presented at the conference. The study showed high rates of costly healthcare utilization, including emergency and inpatient visits, and confirmed that healthcare costs continued to be substantial, exceeding $200,000, in the first year following dialysis initiation. More than half of patients had no recorded PH diagnosis until after initiating dialysis.
“These healthcare utilization findings confirm what many of us know to be true – that people living with PH endure a high frequency of doctor visits. Over the course of the study, more than half of patients needed multiple emergency room visits and 70% had one or more hospital stays, which are even more expensive and disruptive to daily life,” Dr. Aradhye continued. “Of significant concern was the high rate of delayed diagnosis. More than half of patients did not receive a PH diagnosis until after their kidney function declined to the point of dialysis, underscoring the need for better education and understanding of the signs and symptoms of PH.”