SAN DIEGO -- Institutional biotech investors crowded into a hotel room here Sunday night to hear
(CELG - Get Report)
detail -- some would say defend -- the efficacy and safety of the company's experimental drug Revlimid.
In light of recent reports of excessive patient deaths, Dr. Alan List of the H. Lee Moffitt Cancer Center in Tampa, Fla., defended Revlimid's safety Sunday evening at the American Society of Hematology conference. List cited statistics from medical literature that these patients would be expected to have a 10% annual death rate; therefore, the 9% death rate in the Revlimid study is reasonable, especially since he claimed only two patient deaths were tied to the drug.
"There is nothing out of the ordinary," List said, referring to the death rate in the study and the observed toxicities of the drug. List is the lead investigator in Celgene's phase II trial of Revlimid as a treatment for so-called 5 q minus, or low-risk, myelodysplastic syndrome study (MDS), a cancer-like bone marrow disease.
But such assurances of Revlimid's safety did not necessarily gibe with some of the other data presented Sunday, nor did they assuage critics who believe the company is glossing over Revlimid's safety problem and hiding the fact that more of the deaths in the study are being caused by the toxic effects of the drug.
"Celgene is not telling us the full story about Revlimid," said one hedge fund manager who is short the stock. "Nothing I heard tonight convinces me that the [<b>Food and Drug Administration</b>] will approve this drug. In fact, the drug is causing more deaths than I previously thought."
Celgene plans to use this 5 q minus MDS study to seek marketing approval for Revlimid from the FDA during the first quarter of 2005. Despite the drug's compelling efficacy, this plan could be delayed or derailed entirely if Revlimid is deemed too toxic for patients with the 5 q minus form of MDS.
On Nov. 17,
exclusively that there were 11 patient deaths in the phase II Revlimid study, developments which raised questions about the drug's safety. At that time, the cause of the deaths was not known. The deaths were particularly concerning because the patients in the study are all diagnosed with a low-grade form of MDS, a disease which progresses slowly and generally does not present a near-term risk of death.
For the first time, Celgene acknowledged Sunday night that 14 patients died in the study, but only two of these deaths were caused by Revlimid, the company claimed. The other 12 patients died as a result of their disease. With 148 patients enrolled in the study, the overall death rate was just over 9%, with a drug-related mortality rate a bit more than 1%, according to the firm.
The two patients in the study whose deaths were tied to Revlimid died because the drug caused a drastic reduction in their white blood cells (neutropenia), red blood cells (anemia) and platelets (thrombocytopenia). This so-called pancytopenic condition left the patients vulnerable to infections, including pneumonia. For these patients, the pneumonia was so severe that they died.