Image source: Flickr, Creative Commons.
Investing in biotechs is often seen as a feast-or-famine business. In fact, shorts and longs tend to converge on a stock as a key FDA date approaches, knowing it can send the stock soaring (or tanking) in the blink of an eye.
We're not advising that you follow their lead. In fact, here are a couple of surprising lessons you can profit from. First, research shows that negative FDA decisions hurt share prices more than the positive decisions help them. In addition, FDA decisions tend to influence share price long after they're made. In other words, even after a stock has popped from a regulatory thumbs up, there's still room for that stock to run.
With that in mind, our contributors are currently closely watching these three key FDA decisions. It can be incredibly helpful to know what's ahead, so you can be prepared. Knowing ahead of time what a company is facing can keep you from being surprised by the volatile trading that often surrounds an FDA decision.
So, let's get to it!
Todd Campbell: While not quite "this quarter," the FDA will issue a go-no-go verdict on Insys Therapeutics' (INSY) oral marinol on April 1, and in my book, that's close enough!
Marinol is a marijuana-derived drug that has been approved for use as a treatment for anorexia in AIDS patients and to relieve nausea and vomiting in chemotherapy patients. Insys Therapeutics' reformulated variation of marinol, which would be sold under the brand name Syndros, is dosed orally as a liquid, rather than swallowed as a pill, and because liquid dosing has a faster onset of action and dosing is more easily adjusted, management thinks Syndros can win significant market share.
If so, then Syndros has nine-figure annual sales potential, because the market for marinol products is worth $150 million per year and growing.
A successful launch of Syndros is also important because Insys Therapeutics revenue is made up almost entirely of Subsys, a fentanyl pain killer approved to treat breakthrough cancer pain. Last year, Subsys' sales totaled $330 million, up 49% from 2014.
However, Insys Therapeutics has been under investigation for improper off-label marketing of Subsys, and that has caused Insys Therapeutics to lose its CEO and more than half its value in the past year. Therefore, an approval of a second drug that could diversify the company away from Subsys would be welcome.
Brian Feroldi: One FDA decision I'll be watching closely this quarter is whether or not the FDA will give the green light to Rayaldee, a drug owned by Opko Health (OPK -1.99%). Rayaldee is an oral vitamin D prohormone designed to help patients with stage 3 or 4 chronic kidney disease, or CKD, better regulate their vitamin D levels. In phase 3 clinical trials, Rayaldee was able to correct vitamin D insufficiency levels in 96% of patients who used the drug, versus only 8% of patients who took a placebo.
CKD is a huge problem in the U.S.; the National Kidney Foundation estimates that 26 million Americans have the diseases, and 20 million of them are in stage 3 or later. It is estimated that more than 70% of patients with CKD have vitamin D insufficiency, and left untreated, it can lead to bone diseases or secondary hyperparathyroidism, and it's associated with increased mortality rates.
Right now, it's anyone's guess as to which way the agency will lean, but if Rayaldee wins approval, it could be a big deal for Opko Health's investors as the drug will compete in a market that rings up an estimated $12 billion in annual sales.
There's at least one big believer that Rayaldee will make its way to market: Opko Health's billionaire CEO Philip Frost has been aggressively adding to his huge position in the company recently as he has purchased more than 47 million additional shares since December alone. That's a big boost of confidence from an accomplished executive, so Opko Health could be a stock to watch in 2016.
Cheryl Swanson: Jazz Pharmaceuticals (JAZZ -1.88%) has a tiger by its tail with life-saving drug defibrotide, up for the FDA nod on March 31. Assuming defibrotide is approved, doctors should finally be able to reverse a complication of stem cell transplantation so devastating it causes mortality four out of five times.
Jazz believes it has now satisfied the FDA's requests. And with solid data from three clinical studies, and a priority review treatment designation, it seems likely that Jazz's gamble will soon pay off. That will be none too soon for Jazz investors, since right now, the lion's share of Jazz's revenues comes from sales of Xyrem, Jazz's narcolepsy medicine.
So, is there more investment upside in Jazz? Despite the compelling need for a treatment like defibrotide, approval isn't in the bag. Still, orphan drugs historically produce higher margins than those that treat common diseases, and defibrotide has potential for other rare diseases. The bottom line is that Jazz beat earnings and revenue expectations this quarter, and its growth and success thus far lend plenty of reason for optimism, assuming of course, defibrotide nails the expected approval.
