The 1 Biotech Stock I Want to Own in 2017

For most biotechs about to turn the profitability corner, launching the first ever treatment for the most common genetic cause of infant mortality might be their biggest headline of the year. But, the launch of Spinraza is just the beginning of a year packed with catalysts for Ionis Pharmaceuticals (IONS -0.24%) -- and that's what it's the one biotech stock I want to own in 2017.

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In addition to launching Spinraza in partnership with Biogen (BIIB -0.85%) in the first weeks of the new year, Ionis signed a $1.6 billion deal with Novartis (NVS -0.55%), reported a clinical trial success with a first-in-class diabetes candidate, and revised its full-year financial guidance in the right direction. Here's a look at how these developments could make Ionis a top-performing biotech stock in 2017 and beyond.

How much?

The FDA's last approval of 2016 is becoming one of the most closely watched of 2017. Spinraza is the first drug to significantly benefit patients with severe and milder forms of spinal muscular atrophy (SMA), but an eye-popping price tag of about $125,000 per dose could limit its sale to the most severely affected. Up to six doses may be necessary in the first year of treatment, followed by three doses in subsequent years.

In the U.S., it's hard to imagine insurers refusing to pay for the potentially life-saving treatment in the relatively small group of patients with infantile-onset SMA. Investors will want to keep an eye open for uptake among the larger population with less severe, later-onset forms of the genetic disorder. Peak annual sales of Spinraza are expected to brush the $1 billion mark in the infantile onset indication, but they could reach $2.5 billion with strong uptake among the wider SMA population.

Fat Novartis deal

Ionis Pharmaceuticals has a problem that many larger companies envy: more new drug candidates with potential than it can afford to develop as quickly as possible. Earlier this month, it inked a deal with Novartis over AKCEA-APO(a)-LRx, a new drug candidate that significantly decreases circulating levels of a certain fat transport protein often linked to cholesterol, and AKCEA-APOCIII-LRx, a drug that lowers triglycerides.

Both candidates have shown they can effectively reduce their respective targets, but proving whether or not doing so actually lowers the risk of heart attack and stroke isn't going to be cheap. In addition to $75 million upfront and a $100 million equity investment in Ionis Pharmaceuticals, the Swiss Pharma will keep its eyes on a couple of mid-stage dose-determination studies. If the data is positive, Novartis will be responsible for gargantuan expenses associated with running long-term cardiovascular outcome studies, and it will pay Ionis a $150 million licensing option fee for each candidate advanced to late-stage studies.

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If lengthy and expensive outcome studies lead to approvals and highly successful launches, Ionis could be eligible for a further $1.13 billion in milestone payments from Novartis for these two candidates. For now, investors will want to keep an eye open for mid-stage data that suggests this pile of biobucks might become actual capital it can use to advance its wholly owned candidates, such as volanesorsen and IONIS-GCGRRx.

Beyond rare diseases?

An estimated 29 million Americans have some form of diabetes, and at least 90% of adults are type 2 diabetics. The later onset type 2 variety of the disease is marked by a poor response to insulin, and trial results announced earlier this month suggest Ionis' candidate IONIS-GCGRRx could become a valuable new weapon in the battle against this epidemic.

In a 79-patient mid-stage study, those receiving 50mg and 75mg of IONIS-GCGRRx showed 0.7 percentage point and 1.4 percentage point blood sugar reductions from baseline measurements, respectively. While the smaller dose was just strong enough to be considered statistically significant, the larger dose was highly encouraging. Seeing the magnitude of response increase with dosage also points in the right direction.

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It's early still, but this candidate is particularly exciting because it could be the first diabetes treatment that reduces production of glucagon receptors. Blockbuster type 2 diabetes treatments such as Novo Nordisk's Victoza reduce blood sugar by inhibiting secretion of glucagon. If skipping ahead to limit the number of receptors glucagon can act on becomes equally popular, it could lead to huge long-term gains for Ionis.

Recently, Ionis also announced a well-received improvement on its full-year outlook. Management expects to report a $20 million operating profit for 2016 instead of a $60 million loss. Receiving $280 million from partners should allow Ionis to report a slim operating profit for last year. However, Spinraza's launch plus plenty of cash to develop a slew of partnered and wholly owned candidates make this one of the best biotech stocks to own this year.