3 Hematology Successes Biotech Investors Need to Know

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The American Society of Hematology has just ended its 54th annual meeting, and there's plenty of news for investors to get excited about. Numerous drugs and therapies in various stages of clinical trials were on display, with many from your favorite companies -- from small biotechs like Pharmacyclics (UNKNOWN: PCYC.DL  ) to big pharma giants like Bristol-Myers Squibb (NYSE: BMY  ) . Three biotechs took home some good news from the meeting -- enough to make investors very happy with the results.

First up: Pharmacyclics
Pharmacyclics' BTK inhibitor ibrutinib, which the company is developing alongside Johnson & Johnson (NYSE: JNJ  ) to shut down infected leukemia cells while preserving healthy ones, was on full display at the ASH meeting. The company proceeded to blow any lingering doubts away, presenting a full load of good news from two studies on the drug.

In the first study -- a phase 1b/2 trial -- ibrutinib was used as monotherapy against both chronic and small lymphocytic lymphoma. The study recorded a resounding overall survival rate of 96% in previously untreated patients, with a survival rate of 85% in a relapsed or refractory patient group. Neither group showed any long-term safety issues, either.

The second phase 2 study combined ibrutinib with another drug, rituximab, and showed "profound activity" in combating high-risk chronic lymphocytic lymphoma. In both studies, the verdict was clear, however: Ibrutinib met all expectations and looks good in both effectiveness and safety standpoints. Considering that analysts say that the drug could end up pulling in $5 billion a year eventually, Johnson & Johnson's hefty licensing deal to back the drug seems to have paid off.

It's paid off even more for Pharmacyclics investors: The stock jumped nearly 5% on Monday and performed even better on Tuesday, leaping more than 7%. Trust the medical professionals on this one: The director of the hematology division at the Ohio State University Comprehensive Cancer Center, Dr. John Byrd, said at the meeting, "Ibrutinib offers great potential to significantly change the treatment landscape in CLL."

It could significantly change the landscape of your portfolio, as well.

Ariad joins the party
Pharmacyclics wasn't the only company on tap. Ariad Pharmaceuticals (NASDAQ: ARIA  ) showed off results for its chronic myeloid leukemia drug ponatinib at the ASH meeting, and it's looking better and better for the drug heading toward its date with the Food and Drug Administration early next year.

While there are several therapies for CML on the market already, some patients have grown resistance to those established drugs over time. That's where ponatinib steps in: The phase 2 trial reported on at the meeting utilized a patient base already heavily treated with existing drugs. The results look good, as more than half of all previously treated patients achieved a "major response" from the drug.

With only limited side effects reported, ponatinib's safety record looks acceptable by the FDA's standards. Ariad claims that around 2,500 CML patients each year in the U.S. become resistant to existing treatments, forming the core of ponatinib's target market. University of Texas professor of medicine Dr. Jorges Cortes, who presented the data at the meeting, summed up the potential of ponatinib for previously treated patients in saying, "We have simply never had any treatment produce such high rates of durable response in a heavily pretreated group of patients."

Ponatinib's phase 3 trial is already under way, but all signs point to Ariad gaining regulatory approval by the FDA's PDUFA date of March 27. With analysts predicting ponatinib could hit blockbuster status, this company -- and its stock -- is on the cusp of real success.

Finally, some optimism for Geron
The ASH conference's last bit of good news comes from a company that has punished shareholders as of late: Geron  (NASDAQ: GERN  ) . While Geron's drug imetelstat failed in a phase 2 breast cancer study earlier in the year and sent shares down an apocalyptic 56% in one day, the company managed to bounce back a bit with some promising news for the drug.

Geron reported data on a phase 2 trial of using imetelstat to treat patients with the blood disorder essential thrombocythemia, or ET. The results showed a 100% response rate among all the study's patients, with an 86% response rate among those with a specific mutation. It's not quite treating breast cancer, but it's a small victory for Geron considering the study's patients couldn't tolerate or respond to other ET treatments.

The news did wonders for weary investors in sending the stock up 15% on Monday and an additional 14% Tuesday, but unlike Ariad and Pharmacyclics, Geron still has a long road to go before it can declare imetelstat a success. Given Geron's troubles this year, cautious investors would be best advised to wait for more news before taking a gamble on this beleaguered stock.

Two real victories -- and one to hold off on
While many companies ultimately presented data at the ASH meeting, Pharmacyclics and Ariad really scored big. Geron's news sent shares soaring over the last two days, but that company's got a lot more ground to make up after the beating it's taken recently. Ariad seems the best-positioned of the three, with ponatinib seemingly destined for FDA approval next year -- but Pharmacyclics' ibrutinib could be a smashing hit in the future. In the wake of the ASH meeting's results, you'd be justified in placing your faith in either stock.

Johnson & Johnson saw enough in Pharmacyclics' ibrunitib to give it its backing for a hefty price. Pharmacyclics is looking good for your portfolio, but how about Johnson & Johnson, the colossus of the health care industry? Offering everything from baby powder to biologics, critics think the company has spread itself too thin, becoming nothing more than a bloated corporate whale. Is this true, or is J&J a well-diversified giant that's perfect for your portfolio? Make sure you understand the full story behind the stock, along with its key opportunities and risks, by checking out our brand new premium report on Johnson & Johnson. To claim your copy simply click here now for instant access.

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