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Safety Concerns Ding Ionis

The string of severe adverse events with Ionis' inotersen program puts competitor Alnylam in a more favorable position.

Safety concerns from

While Ionis’s pivotal NEURO-TTR study met both of its primary endpoints, several incidences of severe thrombocytopenia (platelet lowering) and renal complications dampened the otherwise encouraging top-line efficacy data. Three patients showed severe thrombocytopenia, one of whom died from brain hemorrhage, and four patients had renal complications with one of the four progressing to renal failure. Last year, a trial evaluating the same molecule in a slightly different patient population with familial amyloid cardiomyopathy was discontinued due to concerns of increased thrombocytopenia in the NEURO-TTR study.

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About the Author

Kelsey Tsai

Equity Analyst

Kelsey Tsai is an equity analyst for Morningstar Research Services LLC, a wholly owned subsidiary of Morningstar, Inc. She covers biotechnology companies. Before assuming her current role in 2016, Tsai was an associate equity analyst, supporting Morningstar’s coverage of the healthcare sector. She joined Morningstar in 2013 as a data analyst and specialized in exchange-traded funds and variable annuities.

Tsai holds a bachelor’s degree in economics from Georgetown University. She also holds the Chartered Financial Analyst® designation.

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