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- Dicerna Pharmaceuticals Inc (NASDAQ:DRNA) announced top-line results from PHYOX2 pivotal trial of nedosiran, RNAi therapeutic, for primary hyperoxaluria (PH).
- The trial hit the primary endpoint for a pivotal study, but shares plummeted after efficacy data in a sought-after patient subpopulation showed no benefit over placebo.
- PH is a family of ultra-rare genetic disorders that initially manifest with complications in the kidneys.
- The PHYOX2 clinical trial included participants with PH subtypes 1 and 2 (PH1 and PH2).
- The results showed benefit over placebo in patients with PH1, an indication with just one approved med, Alnylam Pharmaceuticals Inc's (NASDAQ:ALNY) Oxlumo, but not in PH2, one of two even rarer types of the disease without an approved therapy.
- In a release, Dicerna called the results in five nedosiran PH2 patients "inconsistent."
- Nedosiran works by inhibiting an enzyme that can cause an overproduction of oxalate in the blood and lead to chronic kidney stones.
- But now, Dicerna says it will focus its regulatory efforts with a marketing application filing expected in Q4 of 2021. Meanwhile, the drug has another study in PH3.
- Price Action: DRNA shares are down 28.8% at $27 during the premarket session on the last check Friday.
- Related content: Benzinga's Full FDA Calendar.
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