Dicerna Provides Initial Observations From PHYOX™3 Trial of Nedosiran for Treatment of Primary Hyperoxaluria and an Update on Data Presentation Plan
Mar 31, 2020
— Preliminary Interim Data Show Normalization or Near-Normalization of Oxalate Levels in First Subset of Four Patients —
— Nedosiran Multidose Data to Be Presented at OxalEurope Meeting Now Rescheduled to
“We are encouraged by the initial data emerging from our multidose PHYOX3 open-label trial, which is designed to evaluate nedosiran’s effect on renal function and assess its safety and long-term effect in reducing urinary oxalate levels with chronic monthly dosing in patients with PH,” said
The PHYOX3 trial (ClinicalTrials.gov: NCT04042402) is designed to evaluate nedosiran’s long-term safety and efficacy in patients with PH1, PH2 or PH3. The PHYOX3 trial is an open-label extension study for patients six years of age or older with PH who have participated in any previous PHYOX clinical development program trial. Normal and near-normal urinary oxalate levels in the PHYOX3 trial are defined as below 0.46 mmol and from 0.46 to 0.6 mmol, respectively, during a 24-hour period.
The OxalEurope Meeting, previously scheduled for
About Primary Hyperoxaluria (PH)
Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. There are three known types of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of a substrate called oxalate. Abnormal production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis. Compromised renal function results eventually in the accumulation of oxalate in organs ranging from skin, bones, eyes and heart, especially in patients with PH1. In the most severe cases, symptoms start in the first year of life. A combined liver-kidney transplantation may be undertaken to resolve PH1 or PH2 but is an invasive solution with limited availability and high morbidity that requires lifelong immune suppression to prevent organ rejection. Currently, there is no approved therapy for the treatment of PH. Patients are limited to using hyperhydration and medication to attempt to increase solubility of oxalate in urine. Despite these interventions, oxalate may continue to accumulate in the kidneys, causing damage.
Nedosiran (formerly referred to as DCR-PHXC) is the only RNAi drug candidate in development for primary hyperoxaluria (PH) types 1, 2 and 3 and is Dicerna’s most advanced product candidate utilizing the proprietary GalXC™ RNAi technology platform. Nedosiran is designed to inhibit the lactate dehydrogenase (LDH) enzyme – an enzyme that catalyzes the final step in a common pathway resulting in oxalate overproduction in patients with PH1, PH2 and PH3. Dicerna is evaluating the safety and efficacy of nedosiran in patients with all known forms of PH as part of its PHYOX clinical development program.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding: (i) the therapeutic and commercial potential of nedosiran and the GalXC™ platform; (ii) research and development plans and timelines related to nedosiran, including additional patients receiving multiple doses of nedosiran, and the availability of more advanced data for presentation at the rescheduled OxalEurope Meeting in
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