The biotech sector is what you might call high-risk, high-reward. Because so many biotech companies are losing money, there's a lot of risk for failure if a company's pipeline doesn't pan out. However, if a drug, or a series of drugs, are approved, a company's growth rate can be incredible.
With that in mind, we posed a simple question to three of our healthcare contributors: "Can you name a biotech with with incredible growth prospects?" Here's what they had to say.
If you're ready to dream fearlessly, I've got a biotech bursting with potential. Portola Pharmaceuticals Inc. (NASDAQ:PTLA) presented stellar Phase III data on its anti-coagulent drug a few weeks ago. Now the emerging biotech is gearing up to file for accelerated FDA approval. Since this drug, called andexanet alfa, earned the agency's coveted breakthrough-therapy designation last year, it may well get a speedy pathway through review.
Andexanet alfa has already attracted the attention of Big Pharma players Bristol-Myers Squibb and Pfizer, who market the next-generation Factor Xa blood thinner Eliquis. Andexanet alfa is an antidote to this class of anticoagulant. By latching onto Factor Xa inhibitors like Eliquis, andexanet alfa eliminates the chance those drugs could create serious, even fatal bleeding episodes in patients who take them to avoid blood clots.
A $10 billion market could be on tap for next-generation anticoagulants. And since there is currently no approved reversal agent for these drugs, andexanet alfa's potential to boost sales of these drugs is enormous. Portola is also developing a Factor Xa inhibitor of its own, betrixaban, which is currently in a Phase III trial.
With multiple potential catalysts out there, including the potential for the company to entice an acquirer, this stock could go much higher. On the flip side, with no income being generated, Portola is only for investors with megasized risk tolerance.
How does the potential to go from $1.7 million in sales in 2014 to perhaps as much as $3 billion in sales in 2023 sound? If you're on board with these figures, then I'd suggest taking a closer look at clinical-stage biopharmaceutical company Intercept Pharmaceuticals (NASDAQ:ICPT).
Intercept isn't exactly what I would call "undiscovered." In early 2014 its stock shot from under $100 to more than $400 per share in the blink of an eye following positive midstage results from its FLINT trial involving lead drug obeticholic acid, or OCA. The FLINT study examined the effectiveness of OCA in treating nonalcoholic steatohepatitis, or NASH, a progressive liver disease that affects around six million people in the United States in its most severe form and can lead to death. The trial was actually stopped early due to efficacy (i.e., improved liver histology), which we later found out was highly statistically significant (p-value of just 0.0002).
This is the bread and butter indication for Intercept, although it did, just recently, file for marketing authorization in the U.S. and EU for OCA as a treatment for primary biliary cirrhosis (PBC). With the three studies behind its PBC indication all having met their primary endpoints with high levels of statistical significance, an approval in both the U.S. and EU appears more likely than not at this point.
Of course, it may not be a cakewalk for Intercept, which has also struggled with the long-term safety concerns of OCA. Elevated cholesterol levels in some OCA patients could threaten to narrow its market potential, so it'll be especially important for OCA in its phase 3 NASH studies to demonstrate a considerable benefits profile relative to its risk profile in order to get a green light from the Food and Drug Administration.
If OCA is approved in NASH and becomes a standard of care, I suspect it could see $3 billion in sales by 2023. It's a biotech stock with incredible growth prospects that you'll definitely want to be watching.
One of the most intriguing biotech stocks around in my view is also one of the hottest. Ligand Pharmaceuticals (NASDAQ:LGND) shareholders are no doubt pleased with the stock's year-to-date gains of over 50%, and there could very well be more big gains on the way.
Ligand expects 2015 to bring a year-over-year earnings increase of at least 40%. Wall Street thinks that estimate could even be too pessimistic. What's driving Ligand's incredible growth? A technology that many of the world's leading pharmaceutical companies rely on -- Captisol.
Captisol helps in developing drugs because it increases solubility and improves the stability of active pharmaceutical ingredients. It is used in seven drugs already on the market, most notably multiple myeloma drug Kyprolis. What's even more impressive is that over 100 pharmaceutical companies use the Captisol technology.
Look for increasing Kyprolis sales to help power Ligand's stock higher. Promacta, which increases the number of blood platelets, should also contribute to the biotech's good fortunes. And Ligand will continue to count on the nice revenue pulled in through its many Captisol licenses. I expect this biotech's momentum will keep going through the rest of the year.