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ASH Wrap-Up: Alphabet Soup and Drugs

By Brian Orelli, PhD - Updated Apr 5, 2017 at 8:01PM

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Blood disorders are big business.

Unlike solid tumors, which are usually named after the place the tumor originates, blood cancers tend to have crazy names that all seem to sound the same when shortened to acronyms. Let's take a look at the data that came out of the American Society of Hematology (ASH) meeting that wrapped up earlier this week and see if we can make sense of the alphabet soup.

Expanding the alphabet soup
Celgene's (NASDAQ:CELG) multiple myeloma treatment Revlimid has been doing pretty well lately, increasing sales 72% year over year last quarter to $343 million, but in order to keep sales increasing, the company needs to expand Revlimid into the treatment of other blood disorders.

Celgene presented two phase 2 trials at ASH, showing that the drug works well in patients with chronic lymphocytic leukemia (CLL). The company has already started a phase 3 trial and has another in the works.

The company also presented promising data from a phase 2 trial testing Revlimid in diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkins lymphoma. The company is starting a phase 3 trial to test the drug after standard chemotherapy treatment.

Genzyme (NASDAQ:GENZ) is another company trying to expand its drug's use. Clolar is already approved to treat acute lymphoblastic leukemia (ALL) in children, but the company is hoping to expand into acute myeloid leukemia (AML) in high-risk adults. Genzyme asked the FDA to approve the drug last month, so the ASH presentation acts like off-label marketing to doctors -- perfectly legal since it's done under the cover of science.

Same letters, hotter soup
The big news from Cephalon (NASDAQ:CEPH) wasn't about trying to change cancers that Treanda treats; instead it's trying to get the job done earlier. Treanda is currently approved as a second-line treatment for non-Hodgkins lymphoma, but Cephalon is testing the drug as a front-line treatment with Genentech (NYSE:DNA) and Biogen Idec's (NASDAQ:BIIB) Rituxan.

The drug seems to work just as well as the current front-line treatment, a combination of chemotherapy agents referred to as CHOP, but had a much better side effect profile. For instance, none of the patients taking Treanda lost their hair, compared to 91% on CHOP, and only 5% of patients needed a white-blood cell booster like Amgen's (NASDAQ:AMGN) Nuepogen, compared to 21% of patients on CHOP.

Still on the stove
Plenty of drugmakers present early data at ASH, but one treatment area is heating up to be a real slugfest. Incyte and Motley Fool Rule Breakers pick Exelixis (NASDAQ:EXEL) are both developing Janus kinase (JAK) inhibitors for treatment of myelofibrosis (MF), a disease of the bone marrow.

INCB18424 from Incyte is further along, with 150 patients already treated in a phase 2 trial. The results look promising enough that Incyte is planning to begin a phase 3 trial in the first half of next year.

Exelixis' XL019 is further behind, in a phase 1 trial. The company got delayed when the drug was originally tested at higher doses. It worked well, but had unwanted side effects. The new data from ASH suggest that Exelixis can dial the dose down and still get a decent response.

The results are early, but I expect nothing short of a bloodbath -- maybe even a head-to-head trial -- at future ASH meetings.

Give me an S for Science
Unless you're in the business, and sometimes even if you are, scientific meetings aren't exactly the most entertaining things to read about. But investors need to keep an eye on these presentations because they'll often give keys as to how good clinical trial data really is, along with information about what's in the pipeline, something investors should really keep an eye on.

More scientific Foolishness:

Exelixis is a Motley Fool Rule Breakers selection. Biogen Idec is a Stock Advisor pick. The Fool owns shares of Exelixis. Try any of our Foolish newsletters today, free for 30 days.

Fool contributor Brian Orelli, Ph.D., once tried to write the entire Fool disclosure policy in alphabet soup letters, but he got hungry after a few sentences and ate it. He doesn't own shares of any company mentioned in this article.

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Stocks Mentioned

Celgene Corporation Stock Quote
Celgene Corporation
Biogen Inc. Stock Quote
Biogen Inc.
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Amgen Inc. Stock Quote
Amgen Inc.
$250.58 (-0.25%) $0.63
Exelixis, Inc. Stock Quote
Exelixis, Inc.
$19.31 (-1.02%) $0.20

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