Celgene Needs a Phase III Trial

Yesterday, Celgene (NASDAQ: CELG) announced that its application to market Thalomid (thalidomide) for use in cancer patients would be delayed by at least two years. Celgene will conduct an additional clinical trial in multiple myeloma. Celgene said it will not submit mid-stage clinical trial data in cancer patients until the additional trial is completed.

Thalidomide, infamous because it can cause severe birth defects when given to pregnant women, has a wide variety of therapeutic uses. Since it is currently approved in the U.S. for the treatment of a specific complication of leprosy, it can be used off-label in cancer. In fact, it is prescribed far more often for cancer than for leprosy. However, use in cancer is not supported by active Celgene promotions to doctors, and more significant use in cancer awaits FDA approval for this indication. The FDA indicated that a phase III trial would strengthen Celgene's bid to treat multiple myeloma with Thalomid. The company wanted to rely on low statistical power phase II clinical trial data as well as evidence from cancer patients treated off-label to support a marketing application.

Multiple myeloma is a cancer in which defective plasma cells (antibody producing B cells) emerge from white blood cell precursors in the bone marrow. These renegade cells multiply and create both bone marrow tumors and a large quantity of abnormal antibodies in the blood or urine. Multiple myeloma has been a particularly challenging form of cancer to treat successfully. Now, thanks to advanced molecular biology efforts, promising agents are beginning to demonstrate enhanced therapeutic efficacy for multiple myeloma patients. In addition to Thalomid, promising developmental agents include Millennium Pharmaceuticals' (NASDAQ: MLNM) LDP-341 and Genta's (NASDAQ: GNTA) Genasense. Novartis's (NYSE: NVS) Zometa, approved last August, is quite promising for bone-related disease related to multiple myeloma.

Besides trials for multiple myeloma, Thalomid is in ongoing, phase III investigation for indications relating to non-specific immune disorders, apthous ulcers, and AIDS wasting syndrome. Thalomid is in phase II programs in inflammatory bowel disease, HIV-related diarrhea, AIDS-related Kaposi sarcoma, leukemia, and various solid tumors. Thalomid is currently used off-label in Crohn disease, and has generated encouraging mid-phase data in the management of Waldenström macroglobulinemia (WM), myelodysplastic syndrome, metastatic melanoma, and inflammatory bowel disease. However, the jury is still out for Thalomid's ability to treat conditions such as refractory pediatric malignancies and metastatic renal (kidney) cell carcinoma.

Drugs are rarely approved by the FDA based on phase II data. Novartis's (NYSE: NVS) Gleevec is a notable exception. Companies often try to gain approval with phase II data, especially if the drug can be used to treat deadly diseases refractory to other treatment options, as was the case with ImClone's (NASDAQ: IMCL) Erbitux, or if the drug has already demonstrated sufficient safety data to be approved for another indication, as was the case with Thalomid. However, recent events with ImClone and now Celgene clearly illustrate that seeking FDA approval based on phase II data is risky.

In spite of the delay in gaining approval for the treatment of multiple myeloma, we expect that Thalomid will become an important part of the management of a significant proportion of the disorders for which testing is ongoing. Thalomid's adverse effect profile continues to be reassuring, given that physicians know to avoid using thalidomide during pregnancy. 

BTECH NEWS
by Leon Henderson, M.D.
Bennett Weintraub, Ph.D.
Christopher Martin
www.btechnews.com
March 8, 2002

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BTECH NEWS, published by Btech Investor, Inc., highlights selected events in the life sciences sector that the Btech Investor team believes are particularly relevant to biotechnology investors. The Btech Investor team combines scientific, clinical, and business experience to perform comprehensive analysis of the biotechnology industry and to identify future industry leaders.

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