Ionis Pharmaceuticals (IONS 1.65%) eked out a 5% gain in 2017, which isn't much compared to the average healthcare stock. A muted gain like that might lead you to conclude the company had a ho-hum year where nothing important happened.
In reality, Ionis shared a number of positive updates with investors. Here's a look back at the three biggest events of the year and how they position the company well for future success.
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Spinraza takes flight
In late 2016, Ionis and its collaboration partner Biogen won FDA approval for Spinraza, a treatment for spinal muscular atrophy, a life-threatening genetic disease. While this wasn't the first time that Ionis managed to get the thumbs-up from the Food and Drug Administration, Spinraza was the first potential blockbuster drug to get the green light. Needless to say, expectations were running high for this important drug.
Thankfully, Spinraza got off to a very fast start. Sales eclipsed $520 million through the first nine months of 2017, which means that this is one of the most successful rare-disease-drug launches in history.
Of course, Ionis doesn't get to pocket all of that money itself. It receives tiered royalty payments based on total sales, plus milestone payments. Still, those payments are adding up fast and are helping turn Ionis into a profitable business.
The Akcea spinoff
In March, Ionis took investors by surprise by announcing that it would be spinning off a few of its drugs into a new subsidiary. The new spinoff, Akcea Therapeutics (AKCA +0.00%), was handed the rights to four drugs that treat lipid disorders. The most advanced compound, volanesorsen, is designed to treat extremely high levels of triglycerides in the blood.
Akcea began trading on the public markets in July and raised about $144 million from the offering. Just a few months later, the company submitted volanesorsen for regulatory review in the U.S., Europe, and Canada as a treatment for familial chylomicronemia syndrome, a genetic disorder in which the body can't break down fats correctly.
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What's more, Akcea and Ionis also quickly found a partner to help with the commercialization process should volanesorsen win approval. Pharma giant Novartis (NVS 0.65%) agreed to make a $75 million up-front payment and another $150 million equity investment in Ionis to become a strategic partner. The deal provides Novartis with an exclusive worldwide option to develop and commercialize two of Akcea's drugs. If all goes well, Akcea and Ionis would also be entitled to receive milestone payments and royalties on any sales.
A potential blockbuster comes back home
Investors learned in August that pharma giant GlaxoSmithKline (GSK 0.26%) had declined to exercise its option to move forward with inotersen, a possible treatment for hereditary TTR amyloidosis, a disorder that can cause serious heart or nerve damage. This move was a bit of a head-scratcher because the clinical data on inotersen looked quite good and the drug could be a cash cow should it win approval.
Once the drug was back in Ionis' hands, the company wasted no time with its development. The drug was submitted to regulators in the U.S. and Europe in November, which puts it on pace for a go/no-go decision in mid-2018.
The only potential wrinkle here is that Ionis isn't expected to have this area of the market all to itself for long. Another RNA-focused company, Alnylam Pharmaceuticals, has developed a drug, patisiran, that looks like it will give inotersen a serious run for its money.
Ionis' management team argued that there's room for both drugs in the marketplace, even though they feel that inotersen will have the upper hand.
I'm still a bull
Between Spinraza, volanesorsen, inotersen, and the rest of the company's pipeline, I think there are plenty of reasons for investors to be excited about 2018 and beyond. Ionis' stock performance in 2017 may have been muted, but I'm a firm believer that you can't keep a great company down for long. That's why I plan on remaining a shareholder for years to come.
