This Week in Biotech


With the SPDR S&P Biotech Index up 20% over the trailing-12-month period, it's evident that investment dollars are willingly flowing into the biotech sector. Keeping that in mind, let's have a look at some of the rulings, studies, and companies that made waves in the sector last week.

Once again, earnings reports took center stage in the health-care sector, but four big stories still managed to wiggle their way into the fold during the week. However, unlike the previous week, this week's data was split down the middle between positive and negative news.

Leading with the positive, ZIOPHARM Oncology reported that its metastatic soft tissue sarcoma drug, palifosfamide, had reached the required number of progression-free survival events as outlined in its stage 3 trial to begin analyzing the data. Obviously, this gives us no indication of the safety or efficacy of the drug, but it's as good as taking one step up on the multi-step ladder toward an eventual approval of palifosfamide. The independent data monitoring committee will now review the data, and we should have top-line efficacy and safety results by the last week of March. ZIOPHARM shares rose 13% for the week.

Pharmacyclics and development partner Johnson & Johnson received phenomenal news midweek for their rare blood cancer drug, Ibrutinib. The Food and Drug Administration bestowed the "breakthrough therapy" designation on the drug -- the first such designation of its kind and a potentially positive sign that early-stage clinical data would be enough proof to approve the drug from the FDA's standpoint as long as it showed a marked improvement over existing therapies. J&J has already paid Pharmacyclics $300 million in royalties, and Pharmacyclics could nab an additional $675 million based on Ibrutinib's approval and sales totals. This is definitely a Cinderella story in the making.

On the flipside, clinical data from the recently spun-off AbbVie and Amicus Therapeutics may give investors heartburn this holiday-lengthened weekend.

On Friday, AbbVie announced the suspension of five clinical trials involving in its promising non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and small lymphocytic leukemia drug, ABT-199, after the death of two patients. According to AbbVie, the highly potent experimental drug can rapidly break down tumors and, in some cases, can trigger acute kidney failure -- a condition known as tumor lysis syndrome. The assumption of management is that, with a refined dosage, the suspension will be lifted and the FDA will allow the company to move on to late-stage trials. While I agree with management's assessment, color me a bit concerned nonetheless with the trial suspension.

Late in the week, Amicus did its best to mix the positives with the negatives, but investors failed to see it that way. On Thursday, Amicus announced positive clinical and pre-clinical data of AT2220, its co-administered pharmacological chaperone given with enzyme replacement therapy for the treatment of Pompe disease. However, Amicus spoiled that optimism just 24 hours later by reporting additional late-stage data from its Fabry monotherapy study. Management called the data "encouraging"; however, just as we saw when Amicus imploded in December, there wasn't enough statistical difference between migalastat HCl and the control arm to label it as significant. Investors pounced on the stock following this release and sent shares down nearly 26% on Friday.

Will AbbVie have an answer for Humira when its patents finally expire? Find out now!
In the pharma business, great success comes with a caveat. AbbVie is a perfect example, as investors in the new company are left wondering what the future holds once the company's golden goose, Humira, is cooked. The Fool's brand-new premium report on the company answers the high-profile questions that AbbVie investors are asking, and as a bonus, you'll receive a full year of free analyst updates as significant news hits. Simply click here now to claim your copy today.

The article This Week in Biotech originally appeared on

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.The Motley Fool recommends and owns shares of Johnson & Johnson. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

Copyright © 1995 - 2013 The Motley Fool, LLC. All rights reserved. The Motley Fool has a disclosure policy.