Doug Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

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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