West Coast Editor
Genzyme Corp.'s Phase III win with Mozobil (plerixafor) for non-Hodgkin's lymphoma vindicates those who cheered the high-priced buyout in a bidding war for AnorMED Inc., but the market for the CXCR4 chemokine antagonist might not be huge, and investors still are waiting for results of the trial in multiple myeloma, due in the next few weeks.
"We'll await the results with MM," said Dan Quinn, spokesman for Cambridge, Mass.-based Genzyme. "If they are as positive as this, we would certainly file on both indications." Based on the latest Phase III data, Genzyme plans to seek U.S. and European approval for NHL in the first half of next year.
The company's shares (NASDAQ:GENZ) closed Thursday at $60.88, down 42 cents.
Designed under a special protocol assessment with the FDA, the trial included 298 patients undergoing hematopoietic stem cell transplants (HSCTs) for NHL at medical centers in the U.S. and Canada. The drug is intended to boost the number of HSCTs collected for a transplant. The study compared the hematopoietic stem cell yield from patients treated with Mozobil in combination with standard-of-care granulocyte-colony stimulating factor (G-CSF) to patients treated with G-CSF in combination with placebo.
In the primary efficacy endpoint, 59 percent of patients treated with Mozobil and G-CSF achieved the target threshold for collection of at least 5 million CD34+cells/kg from the peripheral blood with four or fewer days of apheresis sessions, compared with 20 percent of patients in the G-CSF/placebo group - a threefold increase that was highly statistically significant in favor of the Mozobil-treated patients (p<0.0001). The 40 percent absolute difference between the two treatment groups was almost twice the target that Genzyme prospectively defined in the study protocol.
Phil Nadeau, analyst with Cowen and Co., predicted the success of the NHL, but noted that physicians had mixed opinions regarding how deeply Mozobil might penetrate the market. "While some say they would use it in all of their stem cell transplant patients, others say they will reserve it for the 10 percent to 20 percent of patients who fail to mobilize sufficiently using the current standard of care," Nadeau wrote in a research report, and predicted the product would sell only $100 million to $200 million.
But Quinn said the market potential will depend on the results from both trials. "We'll know more about that in the near future," he added.
Genzyme gained Mozobil when the firm outbid Cambridge, Mass.-based Millennium Pharmaceuticals Inc. for Vancouver, British Columbia-based AnorMED last fall, paying $13.50 per outstanding share, or about $580 million in cash, after Millennium declined to increase its $12 per share offer. (See BioWorld Today, Oct. 18, 2006.)
Christopher Raymond with Robert Baird & Co. at the time called the move "a pretty interesting, smart little deal by [officials at] Millennium. They dodged a bullet," and collected $19.5 million, the agreed-upon breakup fee.
Whether Mozobil penetrates well or not, the market is small and getting smaller, Raymond said, arguing that physicians have opted instead for therapies such as Revlimid (lenalidomide) from Summit, N.J.-based Celgene Corp., Millennium's Velcade (bortezomib), and Thalomid (thalidomide) also from Celgene.
Genzyme, though, estimated that 55,000 transplants are performed each year for NHL, MM, Hodgkin's lymphoma and other conditions in markets where the firm has a commercial infrastructure, including the U.S., Europe, Latin America and the Asian Pacific countries.
Mark Goldberg, senior vice president for clinical research at Genzyme, said transplants "haven't been dwindling at all" and could increase significantly with the advent of drugs such as Mozobil.
High-dose chemotherapy, followed by stem cell transplant, is the standard of care for malignancies such as NHL, MM, Hodgkin's disease and leukemia. "I think it's very clear, there's strong evidence for the utility of autologous and allogeneic stem cell transplants in various stages of those diseases," Goldberg said.
"Certainly, in [chronic myelogenous leukemia], over the last several years, with the introduction of Gleevec, the amount of allogeneic transplants has markedly declined," Goldberg noted, but that's old news. Basel, Switzerland-based Novartis AG's Gleevec (imatinib) was approved for CML in May 2001.
"Ultimately, we all want more targeted therapies, more Gleevecs," but such products are years away, he said. Mozobil and others might make stem-cell transplants more feasible in the meantime.