Ionis Pharmaceuticals Upbeat About Clinical Trial
15, May, 2017
Ionis Pharmaceuticals touted a clinical win for its investigational drug, inotersen, which just wrapped up Phase 3 trials. But safety concerns make it unclear if the drug will ever reach the market.
Inotersen is meant to treat a rare and crippling nerve condition called familial amyloid polneuropathy (FAP), which has no FDA-approved treatments in the U.S. Ionis tested its drug in 172 patients for 15 months and saw “statistically significant” improvement on two tests that measure their symptoms compared to placebo.
But the company also discovered some patient reactions to the drug that altered how the company monitors its patients. Three patients in the trial had dangerously low platelet levels while on the drug. Two patients recovered, but the other died from a brain hemorrhage. A fourth patient (whose platelet count dropped while on the drug) stopped treatment.
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