Biotech investing is a tricky space. Hope and miraculous claims often turn out to be hype and disappointment as drugs fail to make it through the approval process. To light the way, we assembled a team from the Fool community with knowledge of the biotech world and asked each to pick a company that they thought was poised for greatness.
Here is what they came up with:
Sean Williams (Fool Writer)
No bets in biotech are a sure thing, but Aeterna Zentaris (Nasdaq: AEZS), though small, looks as close to a sure thing as I see it.
The company, which I chose as one the 10 small caps to rule them all last month, has an impressive pipeline of 11 drugs of which perifosine is its lead candidate. Currently in phase 3 trials, perifosine is targeted at treating late-stage multiple myeloma and colorectal cancer. Perifosine received an orphan drug designation for the treatment of multiple myeloma from the FDA in September and fast-track status in December. It's true that the market for late-stage cancer treatments is small, but it's also practically free of competition -- potentially leaving the door wide open for Aeterna Zentaris' success.
Aeterna Zentaris has partnered with Keryx Biopharmaceuticals (Nasdaq: KERX) in the United States, Canada, and Mexico, Handok in Korea, and Yakult Honsha in Japan. The royalty payments from these partnerships are providing invaluable cash to fund the company's clinical trials instead of having to turn to a dilutive secondary offering to raise cash. Given the positive data presented thus far and the partnerships already in place, Aeterna Zentaris could provide your portfolio with a nice boost.
Brian Orelli (Fool Writer)
Investing in Seattle Genetics (Nasdaq: SGEN) not only gets you access to the company's lead drug, but potential royalties on 13 more and counting.
Brentuximab vedotin is currently under review at the Food and Drug Administration, which is expected to rule on or about Aug. 30. Considering the outstanding data we've seen so far, brentuximab vedotin seems likely to get a thumbs-up.
The market expecting an approval is sometimes a dangerous position to be in -- sell the news and all that jazz -- but even if shares take a post-approval dive, the long-term prospects for Seattle Genetics are still excellent. Pfizer (NYSE: PFE), Bayer, and others have licensed Seattle Genetics' antibody-drug conjugate technology, which connects a targeting antibody to a toxic payload in order to kill the tumor more effectively. Seattle Genetics stands to profit from milestone payments and royalties off the drugs its partners are developing using its technology.
Charly Travers (Fool Analyst)
One of my favorite biotechs with drugs in late-stage development is BioMarin Pharmaceutical (Nasdaq: BMRN). What I like is that the company focuses its R&D on niche markets where competition is limited. For example, the company's first two drugs to gain FDA approval, Aldurazyme and Naglazyme, are used to treat of rare genetic diseases that affect only a couple thousand patients worldwide. BioMarin essentially has a monopoly position serving these markets and will generate more than $400 million in revenue this year.
The future looks bright for BioMarin as it has a couple of additional drugs in late-stage clinical trials, again for the treatment of niche diseases. These products will be important growth drivers for the company over the next five years, and I see BioMarin doubling its revenue over this time frame. I'd be the first to admit that BioMarin's stock looks perpetually overvalued. On the other hand, small-cap drugmakers with attractive assets and substantial revenue are rare beasts in their own right. My expectation is that ultimately BioMarin will be acquired and at a price higher than where it's trading today.
David Williamson (Health-Care Editor)
The question surrounding Amarin (Nasdaq: AMRN) is whether the firm will be a long term multi-bagger after it gets its cholesterol drug AMR101 to market, or if it will get snatched up by a larger pharma player looking to add to its pipeline. Frankly, I don't particularly care; they would both be happy endings.
In case you aren't familiar with Amarin and AMR101, here is a quick synopsis. The company's triglyceride fighter passed its phase 3 trial with flying colors, and another recent trial, dubbed ANCHOR, potentially opened the drug up to a population 10 times larger than the phase 3 trial alone. AMR101 has been shown to lower bad cholesterol, while at the same time raising good cholesterol, something GlaxoSmithKline's Lovaza can't claim. Speaking of Lovaza, it made $800 million for Glaxo last year. If AMR101 is more than just a "me too" drug, as it appears to be, then the market is still undervaluing Amarin.
In the meantime Amarin is getting ready for its big debut after adding two additional suppliers for what it dubs an "aggressive" launch. Even though it has gone on a run, I think there is still time for investors to be aggressive with Amarin's shares before its FDA submission in the coming months and if approved, before it goes on sale next year.
Karl Thiel (Fool Analyst)
Unless you're sticking to the few blue-chip biotechs of the world, picking a winning investment in this space means weighing a relatively high level of speculation against the potential payoff. A company that sits in the sweet spot of risk and reward is Exelixis (Nasdaq: EXEL). The company doesn't have any approved products, but it does -- thanks to a 2010 decision by Bristol-Myers Squibb to bail out of a partnership -- have sole ownership rights to cabozantinib, a very promising drug for cancers, particularly prostate cancer. There are plenty of catalysts ahead: The company will file for approval of cabo later this year in medullary thyroid cancer, a rare disease that will serve to grease the skids for a more significant prostate cancer approval, hopefully around 2014. A pivotal trial in prostate cancer should also launch this year, with two more to follow in 2012. Exelixis is also one of the more anticipated presenters at the annual American Society of Clinical Oncology meeting, which is just about to kick off in Chicago.
The company is far from a one trick pony. Management has slashed expenses over the past couple years and narrowed its focus to just cabo, but it has an extensive pipeline, including programs that are advancing through partners and represent royalty opportunities -- but no expense -- to the company. Rumors that the company has signed on Goldman Sachs to seek a buyer, whether true or not, point to another potential catalyst, although it's one I'm not particularly eager for.
So there you have it Fools, four biotechs that could potentially find a home in your portfolios. Think we missed one of your favorites? Let us know in the comments below.
And if you have had your fill of biotechs and you are looking to round out your portfolio, why not take a look at our special free report on 13 High-Yielding Stocks to Buy Today.
