Washington Editor

Millennium Pharmaceuticals Inc. on Monday released data from a Phase II trial of the cancer drug Velcade, showing a 35 percent response rate in patients with relapsed and refractory multiple myeloma who were progressing on their last therapy.

According to the company, a majority of the 202 trial participants showed a reduction or stabilization in their disease severity, with some patients experiencing complete remission. Furthermore, the trial demonstrated a 16.4-month median survival time in a disease that often kills patients within six months of diagnosis.

The data were presented at the 44th annual meeting of the American Society of Hematology (ASH) in Philadelphia, just days after Millennium released a statement saying it would file a new drug application in early 2003 based on the Phase II trial. (See BioWorld Today, Dec. 6, 2002.)

While company officials could not be reached for comment, Paul Richardson, the lead study investigator from the Dana-Farber Cancer Institute in Boston, released a statement saying, "These outcomes indicate an important potential advance in the treatment of multiple myeloma and may offer new hope for increased survival in this patient population. Consistent with the preliminary Phase II results, these final Phase II Velcade data continue to suggest encouraging activity with durable - including complete - responses with multiple myeloma patients who have exhausted most other treatment options."

Millennium's stock (NASDAQ: MLNM) closed Monday at $10.28, down 91 cents.

Velcade (bortezomib), an injectable drug, is a small molecule designed to inhibit proteasome. It was granted fast-track status by the FDA.

Even though the announcement to file a year early took Wall Street by surprise, Bill Tanner, managing director with Leerink Swann & Company in Boston, told BioWorld Today that many physicians were looking forward to the 2003 filing. "Some of our physician consultants expressed some time ago the notion that Velcade might be available toward the end of 2003, so maybe the Street was less perceiving the fact that Velcade could be on the market late next year."

He added that the physicians believe Velcade works. "These patients are definitely sick and have few other options, so there's a high level of buy-in by the physicians," he said.

The data released in Philadelphia were from the multicenter Phase II trial, known as SUMMIT, designed to look at patients who had a survival time of six to nine months and averaged six prior therapeutic regimens. The company said 4 percent of the patients experienced complete remissions and another 6 percent who had a 100 percent reduction in M-protein but a positive immunofixation test (a sensitive antibody test for trace amounts of M-protein) also experienced complete remissions. A total of 59 percent of the patients demonstrated a response or achieved stable disease.

Once on the market, Tanner expects peak U.S. sales of Velcade in refractory or relapsed patients to reach $100 million. Last week, Yaron Werber, a vice president and biotechnology analyst with SG Cowen Securities in New York, told BioWorld Today that worldwide peak sales could total about $300 million.

Tanner believes Velcade has the potential to generate higher sales in other indications. "As it stands now, the drug would likely be used for a second-line or salvage regimen for multiple myeloma. But if you use it in earlier stages, obviously the real upside for Velcade would be if it proves to be effective in treating solid tumors, but there's no clinical data available to speak to that," he said.

Velcade is the subject of Phase I studies in cancers including prostate, breast, pancreatic and lung.

Meanwhile, the 600-patient Phase III trial in refractory or relapsed multiple myeloma is ongoing. The trial is designed to compare Velcade with high-dose dexamethasone. The primary endpoint is time to disease progression and secondary endpoints are survival, measurements of clinical benefit, quality of life and response rates. (See BioWorld Today, June 13, 2002.)

Millennium acquired Velcade, formerly known as MLN341, LDP-341 and PS-341, in 1999 when it merged with LeukoSite Inc., also of Cambridge, Mass., in a $635 million stock deal. (See BioWorld Today, Oct. 18, 1999.)

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