How Ionis Pharma is Beating the Pants Off of Competitors Like Alnylam, Arrowhead Pharma and Moderna

How Ionis Pharma is Beating the Pants Off of Competitors Like Alnylam, Arrowhead Pharma and Moderna September 20, 2016
By Mark Terry, Breaking News Staff

One of the hot trends in biotech research these days is RNA-based therapies. The biggest competitors in the field are currently Ionis Pharmaceuticals , Alnylam Pharmaceuticals , Arrowhead Pharmaceuticals and Moderna Therapeutics. Cory Renauer, writing for The Motley Fool, takes a look at why Ionis is at the head of the pack.

First up is sheer number of pipeline candidates. Ionis has 27 candidates in clinical-stage trials, as well as three drugs in Phase III studies for rare diseases. The drug furthest along is nusinersen to treat spinal muscular atrophy (SMA), a leading genetic cause of infant mortality.

That’s impressive, but not blindingly so. Moderna, a private company, with a real emphasis on “private,” has about 100 pipeline projects, but it’s recently shifted its focus toward vaccines, probably because vaccines are the quickest route to human trials. It’s raised $450 million venture capital, but so far only has two drugs in early-stage clinical trials.

Renauer writes, “Alnylam Pharmaceuticals and Ionis were in a tight race to develop treatments for nerve and heart damage associated with hereditary transthyretin amyloidosis. That was until Ionis’ partner, GlaxoSmithKline , decided to wait for safety data from an ongoing Phase III trial with IONIS-TTRrx for treatment of nerve damage before starting a trial with the same candidate for treatment of heart damage following some safety concerns.”

Alnylan also only has two drugs in Phase III trials, patisiran and revusiran, for transthyretin amyloidosis. Otherwise it has a few early-stage drugs in its pipeline.

And Arrowhead is well behind Ionis and Alnylam. It has only three candidates in mid-stage clinical trials. Furthest along is ARC-520 to treat hepatitis B virus.

Last month, Ionis provided early data from its Phase III trial for SMA, which showed promise. SMA has no current approved treatments. And Ionis has Kynamro to treat an ultra-rare inherited version of high cholesterol. Ionis had acquired Kynamro from Genzyme before Genzyme was acquired by Sanofi . After the drug was approved, Ionis reacquired the rights to the drug.

A lot of Ionis’ revenue is linked to partnerships with larger companies, which brings in a lot of milestone payments to date. One example is when Biogen exercised its option on nusinersen, which triggered a $75 million milestone payment to Ionis. If that drug should be approved, Ionis’s share of the pie could hit $1.7 billion annually.

Renauer does note that Ionis doesn’t lead in market capitalization. “At recent prices, Ionis’ market cap is around $3.8 billion, which is far below Alnylam’s $6.7 billion. One reason for this is Anylam’s willingness to shoulder more development costs for its candidates in return for commercialization rights in North America and Canada. This increases Alnylam’s risk, but could potentially lead to much more revenue if its candidates succeed.”

Of course, a success or failure in any of these companies’ pipeline could change the metrics Renauer is using. And Moderna, despite some turmoil in its corporate culture, is bringing in a lot of venture capital and funding from government agencies to develop a Zika virus. Only time will tell.

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