American Society of Clinical Oncology
(ASCO) annual meeting is full of orphans looking for a home.
By orphans, I mean beaten down drug and biotech companies long left for dead by much of Wall Street. These are the companies that have blown-up drugs -- and the bottomed-out stock prices to go along with them -- but are trying to make a comeback, either by resurrecting old drugs or starting from scratch with something else in the pipeline.
One medical conference does not a comeback make, but the ASCO meeting is a good place to kick the tires on some of these bio-orphans and get started on some further fundamental research.
Let's take a look at three such companies that caught my eye this weekend at the ASCO meeting:
Keryx blew up in March 2008 when its lead drug for diabetic nephropathy failed a pivotal phase III study. The setback sent the company's stock plunging below $1 where it has stayed ever since. Money became tight and the company was forced to restructure operations and cut back on the development of two remaining drugs. A new CEO took over Keryx's reins in May.
This weekend, Keryx presented mid-stage data from one of those remaining pipeline drugs. Perifosine, or KRX-0401, is an oral anti-cancer drug that targets the AKT pathway in cells, which when activated, promotes cell survival and resistance to chemotherapy. High levels of AKT are seen in many types of cancer and are often correlated with poor prognosis.
The data presented on perifosine at ASCO came from a small, randomized phase II study in patients with second- or third-line metastatic colon cancer. Patients treated with a combination of perifosine and the chemotherapy Xeloda reported that it took 28.9 weeks before their tumors started growing again. That's more than double the 11 weeks for patients treated with Xeloda alone.
Twenty percent of patients in the perifosine arm of the study reported tumor shrinkage compared to 7% for patients in the Xeloda-alone arm. Perifosine patients were also living longer, although survival data is not yet mature.
The data are encouraging, especially for such advanced colon cancer patients, but the study was small, with only 38 patients enrolled. And less than half the patients in the study received prior treatment with Erbitux, the colon cancer drug from
that is commonly used in second- and third-line colon cancer patients.
Late last year, Keryx also presented encouraging data with a combination of perifosine and the drug Velcade in patients with multiple myeloma.
One other interesting note about perifosine is that last week,
signed a global partnership deal with
to gain access to two of Exelixis' early stage drugs that target a similar anti-cancer pathway.
ASCO holds its plenary sessions in huge meeting halls that can seat 15,000 people comfortably. Usually, these highly attended sessions feature cutting-edge research from the top names in the biotech and pharmaceutical sectors.
Well this year, those big companies shared the stage with a tiny drug firm sporting a penny stock trading on the pink sheets.
Accentia and its majority-owned subsidiary
(yes, another bulletin board stock) presented data on the first cancer vaccine purported to work in patients with non-Hodgkin's lymphoma.
BiovaxID is made from cells taken from a patient's own tumors. The vaccine works by training the body's immune system to recognize and attack a protein found only on cancerous B cells.
In a phase III study, patients with non-Hodgkin's lymphoma who were treated with BiovaxID experienced a median disease-free survival of 44.2 months compared to 30.6 months for patients treated with a control vaccone.
After almost five years of follow-up, the BiovaxID patients experienced a 38% lower risk of disease recurrence compared to patients receiving the control vaccine.
Two other now-defunct companies --
-- failed with similar vaccines for non-Hodgkin's lymphoma.
Despite the imprimatur that an ASCO plenary session brings, there are reasons to be skeptical about these seemingly positive results.
Accentia and Biovest first announced the positive results from this study of BiovaxID in June of last year, but since then the stock prices of both companies have fallen even further below $1 a share. (Only Biovest's stock price has recently recovered.)
The results might also be a bit misleading as well because the study takes a funny accounting of patient responses, essentially not starting the clock until six months after patients are treated initially with a standard regime of drugs used to put the cancer into remission.
The disease-free survival benefit reported in this study was barely under the limit of statistical significance. If patient responses were counted in a more traditional way, i.e., as soon as the patients were admitted into the study, the results might not look so robust.
That's an issue for the U.S. Food and Drug Administration if or when it starts a review of BiovaxID. Still, a plenary session at the ASCO meeting is a high honor, so with investors buzzing about cancer vaccines in the wake of
success, BiovaxID is worthy of further research.
It's a bit unfair to group Medarex into this bio-orphan niche, if only because the company is much larger than the other two, and actually has a successful humanized antibody development business.
But Medarex has not yet successfully developed a drug on its own, and the biggest disappointment to date has been ipilimumab, a drug for skin cancer, which posted disappointing results from three pivotal trials in late 2007.
The failure of those three studies, coupled with the failure of a similar skin cancer drug being developed by
, put ipilimumab in Wall Street's penalty box. Both Medarex and its partner
insisted that ipilimumab wasn't done, but investors have been skeptical.
Perhaps perseverance is paying off. Medarex and Bristol-Myers continued to follow the patients treated with ipilimumab in those studies, and the results after two years show that between 30% and 42% of these skin cancer patients are still alive, according to data presented Sunday at the ASCO meeting.
Now, there were no control patients in any of these studies to make appropriate survival comparisons, but Medarex says that a search of the old melanoma studies covering about 2,000 patients shows that similar patients typically have about a 25% survival rate at one year.
For Medarex, this is encouraging news on ipilumumab and a big reason to look forward to later this year, when results are expected from another phase III study of ipilummab -- this one prospectively designed to assess the drug's ability to prolong survival in patients with newly treated melanoma.
Adam Feuerstein writes regularly for TheStreet.com. In keeping with TSC's editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet.com. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback;
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