--Celgene halts late-stage study of Revlimid because of high
number of deaths
--Study was aimed at expanding Revlimid sales into a rare form
of blood cancer
--Celgene says study doesn't affect safety profile of Revlimid
in approved indications
(Updates throughout with details, company and analyst
comment.)
By Joseph Walker
Celgene Corp. (CELG) said it will discontinue a late-stage study
of Revlimid in elderly patients with a rare type of blood cancer
after finding that patients taking the drug had a death rate nearly
twice that of patients receiving chemotherapy.
The study was intended to help expand the approved uses for
Revlimid--the company's biggest-selling drug--to patients with
B-cell chronic lymphocytic leukemia, or CLL, a disease affecting
mostly older people. There are expected to be roughly 15,700 new
cases of CLL this year in the U.S., and Celgene is one of several
companies working on new treatments for the disease, which some
analysts project could grow into a $10 billion to $15 billion
market.
The study failure is a setback for Celgene, which is
aggressively seeking to expand its already strong position in the
treatment of blood-related cancers, a tumor area where patients can
stay on therapies for years and manufacturers have wide leeway in
setting prices. The market potential for such diseases has helped
make Onyx Pharmaceuticals Inc. (ONXX), which competes against
Celgene in multiple myeloma, into an acquisition target this
summer.
Celgene shares fell 2.8% to $132.80 in recent trading; the stock
is still up 69% this year.
Celgene said the failed study would not affect the safety
profile of Revlimid in its already-approved indications. Two other
company-sponsored trials of the drug in different CLL populations
will continue, the company said.
Revlimid had sales of $3.76 billion last year, and is approved
for certain patients with blood-related cancers like multiple
myeloma and mantle cell lymphoma, as well as anemia.
In March, an independent safety monitoring board informed
Celgene of an imbalance in patient deaths, data that was then
shared with the U.S. Food and Drug Administration, said Gregory
Geissman, a Celgene spokesman. Last week, the FDA, in consultation
with Celgene, halted the trial, which the company said would not
have met its primary endpoints.
"We believe the imbalance in deaths would make the study
unlikely to show a significant positive outcome on
[progression-free survival] or overall survival," Mr. Geissman
said.
Through March, there were 34 deaths in the Revlimid arm of the
study, and 18 deaths among patients receiving the chemotherapy
chlorambucil. However, as the study continued through July, the
imbalance narrowed, with 37 total Revlimid deaths compared to 28
deaths in the control arm, Mr. Geissman said.
The study, which enrolled 450 patients, examined the effect of
Revlimid in patients ages 65 years or older who previously had not
received treatment for CLL. The company said a majority of patients
suffered from other conditions, including diabetes and congestive
heart failure.
Some analysts said expectations for the trial hadn't been high
to begin with, primarily because of the assumption that Revlimid
would have been inferior to experimental drugs being developed by
competitors. The specter of safety issues for the drug, though,
could create some nervousness, said Christopher Raymond, a Robert
W. Baird analyst.
"Anything that plants a seed of doubt as to Revlimid's safety
has to catch investors' attention," Mr. Raymond said in a research
note to clients.
It is currently unknown what caused the mortality imbalance, but
Celgene said it would conduct a data analysis to determine whether
differences in patients' age, geography or other factors played a
role. Revlimid is known to sometimes cause a side effect known as
tumor flare reaction, or TFR, and the company will study whether
differences in the management of that condition also may have
contributed to the deaths.
The average age of patients found to have CLL is roughly 72
years, according to the American Cancer Society. Doctors say many
older patients are unable to tolerate the side effects of
traditional chemotherapy, creating a large need for new, less toxic
therapies. A drug being developed by Pharmacyclics Inc. (PCYC) and
Johnson & Johnson (JNJ) is considered by many analysts to be
the most promising of the experimental drugs, and last week the
companies submitted a new drug application to the FDA seeking
approval for the treatment.
-Ben Fox Rubin contributed to this article.
Write to Joseph Walker at Joseph.Walker@dowjones.com
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