Dicerna's RNAi drug shows 'inconsistent' results in rare kidney disease, tanking shares despite trial win
Sometimes even when you win, you lose.
Dicerna’s nedosiran, an RNAi therapeutic being tested in patients with a rare kidney disease known as primary hyperoxaluria, hit the primary endpoint for a pivotal study, the company said Thursday, but shares plummeted after efficacy data in a sought-after patient subpopulation showed no benefit over placebo.
The results paint the picture of a drug that showed benefit over placebo in patients with what is known as PH1 — an indication with just one approved med, Alnylam’s Oxlumo — but not in PH2, one of two even rarer types of the disease without an approved therapy.
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