National Editor

The long and winding path of Campath has taken yet another turn, with ILEX Oncology Inc. - which last month granted complete worldwide rights to Schering AG for the drug as a leukemia treatment - gaining an option from BTG International Ltd. for all non-oncology therapeutic uses of the monoclonal antibody.

One major target outside cancer: multiple sclerosis. Barry Cohen, director of public relations for ILEX, said the company had rights to Campath for MS before London-based BTG granted the option.

"This gives us everything else," he told BioWorld Today, but "right now the biggest push is on MS. Campath, the hypothesis goes, would be [administered] for five days once a year" he added - a strong advantage over existing treatments for the disease.

Jeff Buchalter, president and CEO of San Antonio-based ILEX, speaking Monday at the Raymond James & Associates Institutional Investors Conference in Orlando, Fla., called Campath "a drug that's very early in its life cycle."

Marketed therapies in the $2 billion MS market have "very interesting limitations," he said. They must be self-administered daily or weekly for life and come with significant side effects. Also, he said, they "have never demonstrated, in most cases, an effect on the progressive phases of the disease."

ILEX in January began a Phase II trial to evaluate Campath against Rebif (interferon beta-1a) in patients with early active, relapsing-remitting MS. The 150-patient trial will examine the drugs against each other for efficacy in preventing sustained accumulative disability. Rebif was developed by Serono SA, of Geneva, and the drug received U.S. approval in March 2002.

Cohen said the focus now is on getting qualified patients into the trial. "They [must be] drug-na ve; they can't have taken any other drug," he said. "It's not every MS patient."

The trial will probably not yield conclusive data of the kind sought by ILEX - which wants to show Campath slows progression of the disease - for "several years," he added. A small study in the UK has come up with promising results and is ongoing.

Campath (alemtuzumab) is approved for B-cell chronic lymphocytic leukemia (CLL) patients who have been treated with alkylating agents and have failed fludarabine treatment, and Buchalter told attendees of the Raymond James conference "there's a very good story evolving for the use of Campath in CLL."

For example, in December at the 44th annual meeting of the American Society of Hematology in Philadelphia, independent investigators at the University of Cologne, Germany, presented data on 11 patients from an ongoing Phase II trial combining fludarabine phosphate and alemtuzumab therapy in patients with relapsed/refractory B-cell CLL showing efficacy, with a mild and manageable safety profile.

ILEX has given guidance of about $50 million in global sales for Campath this year, compared to $44 million last year. Approvals for earlier-stage CLL will be sought, Buchalter said, and Campath's value in treating such cancer disorders as non-Hodgkin's lymphoma also is being investigated.

Because the drug works by targeting and depleting T cells, it may have uses in other indications such as autoimmune and other diseases in which mediating T cells is important. Specifically, Campath binds to the CD52 antigen on cell surfaces, leading the body's immune system to destroy malignant cells and deplete B cells and T cells.

In 1999, LeukoSite Inc., of Cambridge, Mass., and ILEX granted worldwide marketing rights, except in Japan and East Asia, to Berlin-based Schering AG in a deal valued at up to $30 million. Richmond, Calif.-based Berlex Laboratories Inc., Schering's affiliate, handles marketing in the U.S. (See BioWorld Today, Aug. 25, 1999.)

First developed by ILEX and LeukoSite, Campath was part of the package when Millennium Pharmaceuticals Inc., also of Cambridge, bought LeukoSite later in 1999, thereby gaining LeukoSite's share of the drug.

Campath then was developed in a 50-50 joint venture by ILEX and Millennium, until Millennium sold its share back to ILEX in the fall of 2001 - Campath had won U.S. approval that spring - for about $140 million. (See BioWorld Today, Oct. 31, 2001.)

In February, ILEX granted Schering rights to the territories Schering needed for complete worldwide marketing of Campath, which was launched in the U.S. in June 2001 and launched in Europe, where it is called MabCampath, in August 2001.

Now, BTG, from which LeukoSite and ILEX licensed Campath originally, has granted ILEX the option for non-oncology uses. Those, Buchalter said, could be significant.

Campath is considered the "gold standard for organ transplant" therapy by some, he said, and has been "used for years in bone-marrow transplant," while showing promise in MS and in autoimmune diseases.

Cohen called Campath "almost exclusively a standard of care in renal transplants [off label] at two top-tier transplant centers in the U.S.," according to ILEX Pharmaceuticals Inc.. "It's sort of taken on a life of its own," he said, and a decision hasn't been made on whether to seek formal approval for the transplant indication.

Said Buchalter: "You don't find too many drugs that have that much potential. The true end of this is studies, data, and that's what we're focused on."

Meanwhile, the company has plenty more in the oncology field. Among its other efforts is a Phase III trial of eflornithine (difluoromethylornithine, or DFMO) in superficial bladder cancer, which is "still blinded, so we don't have data to report, but hopefully that [trial] will conclude this year," Buchalter said.

ILEX's stock (NASDAQ:ILXO) closed Tuesday at $6.45, down 7 cents.