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Biotech / Medical : Ionis Pharmaceuticals (IONS)
IONS 74.30+0.2%Oct 31 9:30 AM EDT

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From: tuck4/7/2016 5:56:05 PM
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Ionis' TTR-rx program had a superficially strange hiccup today. It's running pivotal trials in two separate, but related indications: transthyretin (TTR) familial amyloid polyneuropathy and TTR amyloid cardiomyopathy. The latter trial has been put on clinical hold due to an unspecified issue in the former, even though the former is not on hold. Presumably, the FDA saw a signal in the NEURO-TTR trial that would be a more significant concern in the CARDIO-TTR trial, as the CARDIO-TTR population is more sickly. Note that the trials are regulated by different divisions within the FDA. The trials are being run by partner GSK, and neither company was especially forthcoming about this issue.

"FDA has placed a clinical hold on the CARDIO-TTR study while GSK provides answers to questions about the protocol stemming from Ionis' ongoing NEURO-TTR study."

That's as much information as investors are being given. Not a surprise, then, that the stock is down over 11%.

I bought a mini position at the close, looking for 30% or so retrace of the days loss. Short term dinner money, in theory.

Ionis next presents at Needham, 4/13. Don't know if there is Q&A there or what they might say on their own.

Cheers, Tuck
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