Precious Metals

[gfp927z]

>>> Ionis Pharmaceuticals: candidates galore



http://www.fool.com/investing/2016/06/29/these-3-biotechs-are-down-more-than-36-this-year-a.aspx?source=yahoo-2&utm_campaign=article&utm_medium=feed&utm_source=yahoo-2



Ionis is another biotech recently beaten up for bad news concerning just one clinical-stage program, despite plenty of other opportunities. In April, the the FDA put one of its GlaxoSmithKline-partnered trials on hold because of a relatively minor issue with IONIS-TTRRx that emerged during a nerve-damage trial. It became a major issue for Ionis' stock when Glaxo announced it wasn't going to begin a planned phase 3 trial with the same drug in patients suffering heart damage.

I think Wall Street is missing the bigger picture here:

Ionis Clinical Stage Pipeline

The drug in question is one of the company's older, more advanced clinical-stage candidates, but Ionis has a very deep bench. For example, Nusinersin, in partnership with with Biogen, is in phase 3 for treatment of spinal muscular atrophy, the leading genetic cause of infant mortality. If successful, Ionis could receive up to $346 million from the bigger biotech, and tiered royalties in the mid-teens on any sales of this drug.

Another phase 3 hopeful, volanesorsen, for treatment of patients with high triglyceride levels, is partnered with Akcea, a wholly owned subsidiary of Ionis. The company is taking on more risk, but it's entitled to all potential sales. The ongoing phase 3 studies could support applications for treatment of extremely rare inherited diseases. If it's successful, more general use among 20,000 or more U.S. patients without a genetic cause for extremely high triglyceride levels could help drive growth in the years ahead.

Behind its enormous clinical program is a drug-discovery machine that churns out potential clinical candidates faster than any biotech I can think of. With a recent market cap of about $2.7 billion, this stock is a real bargain.

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