Recently, rumors have surfaced about an increase in deaths among MDS patients treated with Revlimid. During ASH, Celgene held a meeting for analysts where the company presented data from two recent studies.
A subset of patients with a 5q chromosomal deletion had the more benign form of MDS. Since MDS is a bone marrow disease and patients become anemic, Revlimid's activity was measured by a 50% decrease in transfusions or an increase in blood hemoglobin concentration (demonstrating that the body produces its own red blood cells).
The major erythroid response to transfusion dependence was 61% in the drug-treated arm of the study, according to the company. However, a number of deaths occurred in both studies, which, the company noted, were mostly not drug-related. The majority of deaths, the company's investigators concluded, were from secondary causes such as pneumonia.
Celgene reported that it would file an NDA in the first quarter of this year. The short-sellers of the stock, however, believe that the FDA will reject Revlimid's application for MDS because of its toxicity and that the pneumonia-associated deaths were a result of the drug. The bears also noted that the FDA will probably reject Celgene's application since the studies were not randomized and placebo controlled.
On the other hand, investors who are bullish on Celgene, anticipate that the real play in Revlimid is an anticipated approval for multiple myeloma, which will skyrocket Celgene's stock price.
Biotech analyst, Derek Taller, Ph.D., from Tradition-Asiel Securities (New York City), who is following Celgene, seemed less disturbed about Revlimid's drug-related deaths and more concerned about the efficacy of the drug and the durability of the response to the drug treatment in both patient populations.
Dr. Taller was concerned about Celgene's business strategy of trying to have Revlimid approved for MDS and also have the drug used off-label to treat MM (where there seems to be the real clinical benefit).
Other players in MDS arena are Pharmion (Boulder, CO), which is already marketing its drug Vidaza (azacitidine), and SuperGen (Dublin, CA), which reported on its Phase III study with Dacogen (decitabine) at ASH.
Both drugs are hypomethylation agents with a unique mechanism of action that recognizes that cells involved in this particular form of cancer have aberrant methylation patterns. By inactivating DNA methyltransferases with cytosine analogues, the aberrant cancer cells should inactivate.
Another interesting drug is Maxim Pharmaceuticals' (San Diego) Celpene for AML. Celpene is a histamine dihydrochloride that acts as a protectant to anti-cancer T cells that could be suppressed by blood phagocytes.
Maxim's investigators presented data from a Phase III trial demonstrating a leukemia-free survival benefit in AML patients in complete remission when treated with Celpene and Interleukin-2.
Diseases of the blood and cancer appear to be the power zone for biotech-based drug products. Wall Street's focus on clinical data emanating from the scientific conferences, such as ASH, reinforces the power of developing novel therapeutics to treat diseases that were once thought to be an exercise in futility for drug developers focused on these small disease markets.
Thalidomide reflects how understanding underlying disease mechanisms can lead to new clinical indications for drugs once considered niche market products.
It is conceivable that in the not too distant future we will have biotech products that not only delay disease onset or death, but also actually cure diseases.