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3 Things to Watch at Isis Pharmaceuticals

Brandy Betz
December 20, 2012

Isis Pharmaceuticals (NASDAQ: ISIS) hit rough waters with its lead drug, Kynamro. But the small-cap developer of antisense drugs has a populated pipeline and a wide range of big pharma partners. If the Food and Drug Administration denies Kynamro, will the company find its sea legs or sink underwater?

Here are three things to watch when considering an Isis buy.

1. Kynamro approval
Isis' only approved drug hit the market in 1998 and isn't commercially available anymore. That ups the importance of lead drug Kynamro, a partnered project with Sanofi's (NYSE: SNY) Genzyme subsidiary.

Kynamro treats a severely high cholesterol condition called homozygous familial hypercholesterolemia, or HoFH. Kynamro and Aegerion's (NASDAQ: AEGR) competing lomitapide are running nearly parallel down the pipelines. Both drugs are effective, making safety the deciding factor.

There's the (potential) rub.

Kynamro fared worse than lomitapide at regulatory safety concerns. An FDA advisory panel in October recommended approval for Kynamro, but questioned liver safety and neoplasm risk. The liver safety issue comes up with lomitapide, too, but Kynamro stands alone in questions concerning neoplasms, or abnormal tissue growth.

A European committee denial last week didn't help Kynamro's image. But while its approval would help further validate Isis' drug platform, Kynamro isn't the company's only hope.

2. Mid-stage pipeline
The Isis pipeline boasts around 25 projects, with