5
BioTech Stock Report, January 2004
Celgene Corp.
to mid-2004. The studies compare Re-
vimid plus dexamethasone versus
dexamethasone alone. The primary
endpoint is the time for the disease to
progress and the secondary endpoint
is overall survival. Data from the trial
could be available for ASH 2004 and
could enable a full FDA approval in
multiple myeloma in late 2005.
Enrollment in the Phase III trials in
metastatic melanoma is making head-
way. The primary endpoint is overall
survival since median survival for this
population is roughly 4-6 months. The
first trial is in North America and will
compare two different doses of Re-
vimid with no control arm, and the sec-
ond trial is in Europe will compare Re-
vimid plus standard of care, versus
standard of care plus placebo. These
trials could demonstrate Revimid's
potential activity in larger solid tumors,
but due to the nature of the disease,
melanoma, may provide a more limited
proof of concept in solid tumo rs. Data
from the trials could be available for
ASCO 2004 and these trials could also
enable full FDA approval of Revimid in
late 2005.
There were over 50 abstracts at
ASH that highlighted the use of Thalo-
mid as a monotherapy or in combina-
tion with other therapies, such as dexa-
methasone, mephalan, prednisone, and
cyclophosphamide, in newly diag-
nosed and relapsed/refractory multiple
myeloma patients and as a pre-
nance therapy.
The biggest focus for Celgene's
pipeline is on the FDA regulatory pro-
gress on Revimid for the treatment of
MDS, a disorder of blood cell produc-
tion. Enrollment in the first group of 36
patients is completed and in early
January/February 2004 the enrollment
of the second group should be fin-
ished. Management will have complete
clinical data from the first group of pa-
tients in the first quarter of 2004. Data
from the second group should become
available in mid-2004. At that point, the
company should release the data as
well as update the clinical status and
the FDA regulatory process for inves-
tors. An accelerated approval of Re-
vimid for MDS in late 2004 is possible
based upon data from ongoing trials in
the MDS 5q- patient population.
At ASH, Phase II data from trials
of Revimid in the MDS 5q- population
demonstrated 10 out of 11 or 91% pa-
tients had major erythroid responses
(transfusion dependence to transfu-
sion independence) and 100% major
cytogenic (cell development) re-
sponse. The compelling nature of this
data could enable the company to pur-
sue approval based on a primary end-
point of transfusion independence
versus more stringent endpoints seen
for other MDS treatments.
Enrollment in the Revimid Phase
III trials in relapsed refractory multiple
myeloma should be completed in early
Overview
Celgene Corp. (Celgene) develops
and commercializes small molecule
drugs for cancer and immunological
diseases. Its lead product, Thalomid, is
approved in the U.S. for leprosy and is
used off-label for multiple myeloma
and other cancers. In addition to ex-
panding the treatment usage of Thalo-
mid in other indications, Celgene's
pipeline includes second-generation
drugs, IMiDs (immuno-modulatory
drugs) and SelCIDs (selective cytokine
inhibitory drugs) in oncology and in-
flammatory diseases.
Analysis
With its variety of therapies, Cel-
gene was highly visible at the annual
ASH meeting.
Revimid and Actimid (both ImiDs)
are thalidomide analogues currently in
development for multiple myeloma,
myelodysplastic syndromes (MDS)
and solid tumors. In regards to Re-
vimid, the key abstract was on the use
of Revimid in treating anemia in MDS
and two other abstracts described the
use of Revimid in multiple myeloma.
For Actimid, a presentation on the use
of Actimid in patients with relapsed/
refractory multiple myeloma was a fo-
cal point. For Thalomid, clinical data
was presented on Thalomid in front-
line multiple myeloma, in various com-
bination regimens and as a mainte-
