Dicerna Presents Positive New Interim Data From PHYOX?3 Long-Term, Open-Label Extension Study of Nedosiran for Treatment of Primary Hyperoxaluria (PH)

- A total of 16 trial participants from the completed PHYOX1 trial entered the PHYOX3 trial. Of these, 13 who had reached 180 Days and had received six monthly doses in the PHYOX3 trial were included in this analysis. Three participants were not included in the efficacy analysis as they had not yet reached Day 180 at the time of the analysis.
- All participants in this analysis, regardless of PH subtype, achieved normal or near-normal Uox excretion by Day 180.
- 92% of all participants (12 of 13) achieved normal Uox excretion at one or more visits through Day 180:
- 100% of participants (10 of 10) with PH1
- 67% of participants (2 of 3) with PH2
- The single participant with PH2 who did not achieve normal Uox excretion during the analysis period reached near-normal Uox excretion rates.
- The mean 24-hour Uox excretion for all 13 participants at Day 180 was in the normal range at 0.44 mmol/1.73 m2?BSA/24 hr.
- 62% of all participants (8 of 13) demonstrated normalized Uox excretions on at least three consecutive visits, meeting protocol-defined eligibility for gradual reduction in fluid intake requirements:
- 70% of participants (7 of 10) with PH1
- 33% of participants (1 of 3) with PH2
- The mean maximum reduction in Uox excretion for all participants was 70.9% (range, 54.9% to 87.5%) by Day 180.
- Nedosiran was generally well tolerated, and no serious safety concerns were identified in this ongoing study. There were no treatment discontinuations or study withdrawals during the observation period. Two participants had serious treatment-emergent adverse events (TEAEs) (pyelonephritis and nephrolithiasis) that were determined by the investigator to be unrelated to nedosiran treatment. The most common TEAEs were mild to moderate administration-site reactions. Protocol-defined injection-site reactions (ISRs) occurred in 18.8% of participants.
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Media: Amy Trevvett +1 617-612-6253 atrevvett@dicerna.com Investors: Lauren Stival +1 617-514-0461 lstival@dicerna.com Source:?Dicerna Pharmaceuticals, Inc.