The JPMorgan Healthcare Conference just finished up in San Francisco and was arguably the most important event of the entire year for the health care sector. This is one of the rarest opportunities for biotechnology, pharmaceutical, and medical device companies to open up about where they've been and where they're headed, so it pays to take notice.
With dozens of companies presenting each day last week, it was veritably impossible to review each and every one. That doesn't mean, however, that there weren't other companies squarely in my sights.
One of those companies is Exelixis (NASDAQ:EXEL), a biotechnology company that had its first drug, Cometriq, approved by the Food and Drug Administration in November for the treatment of metastatic medullary thyroid cancer. In trials, Cometriq's progression-free survival nearly tripled that of the placebo (11.2 months versus four months), and it should easily oust AstraZeneca's (NASDAQ:AZN) Caprelsa for the majority of the 2,250 cases of MTC reported in the U.S. annually. Needless to say, I was very intrigued to see what Exelixis had to say last week, and I wasn't disappointed.
Exelixis was very upfront about its plans for 2013, which include launching Cometriq commercially this month, focusing on expanding its indications through many ongoing clinical trials including the advancement of the Comet trials for metastatic castration-resistant prostate cancer, or mCRPC; and initiating trials in renal cell carcinoma and hepatocellular carcinoma.
Admittedly, the market for Cometriq looks pretty thin at the moment, with Exelixis targeting just 500 to 700 patients in the U.S. with a flat monthly pricing cost of $9,900. Where Exelixis sees its demand coming from is the additional indications for Cometriq.
According to Exelixis' presentation, the company currently has more than 30 ongoing or planned phase 1 or phase 2 studies. Exelixis plans on targeting such indications as non-small-cell lung cancer, ovarian cancer, multiple myeloma, breast cancer, and bladder cancer, to name a few.
Furthermore, it's testing Cometriq as both a combination therapy and by itself, bettering its chances of success and boosting the probability that a combination therapy would bring the shared expenses of trial costs and a drug launch. In the Comet 1 & 2 trials for mCRPC, Exelixis' most advanced and most important current trials, it's testing Cometriq on patients who've previously taken Medivation's (NASDAQ:MDVN) Xtandi or Johnson & Johnson's (NYSE:JNJ) Zytiga (depending on the study) to improve overall survival and induce a reduction in pain. Results of these studies are due out in the first half of 2014.
Even beyond its extensive research with Cometriq, Exelixis has an incredible number of collaborative partnerships. It's out-licensed Foretinib, which is currently in multiple phase 2 clinical trials for various types of breast cancer, to GlaxoSmithKline (NYSE:GSK); it has two PI3K pathway drugs in phase 2 trials with Sanofi (NASDAQ:SNY); and it has quite a few preclinical and early stage collaborations with Bristol-Myers Squibb (NYSE:BMY).
You might think running so many trials can be expensive – and to some point it is – but the company's well covered, with $675 million in cash at the end of the third quarter and multiple partnerships, which help spread the costs.
Following this presentation, I continue to feel that Exelixis is the No. 1 name to watch in biotech over the next year. I fully understand that Cometriq may not work as effectively on different types of cancers, but its PFS success rate in MTC just seems too good to be worthwhile for only one indication of cancer.