Washington Editor

WASHINGTON - Early results from a National Cancer Institute-sponsored Phase III trial showed that multiple myeloma patients who received Celgene Corp.'s Revlimid (lenalidomide) after an autologous blood stem cell transplant had a statistically significant reduction in disease progression, the agency reported Friday.

"This study answers the important question for multiple myeloma patients regarding maintenance lenalidomide therapy," said lead investigator Philip McCarthy, associate professor of medicine at Roswell Park Cancer Institute.

Because of the superior efficacy seen in the trial, the study was stopped early, officials said.

Shares of Summit, N.J.-based Celgene (NASDAQ:CELG) shot up $5.33 Friday, or 10.5 percent, to close at $55.95.

Revlimid currently is approved in the U.S. in combination with dexamethasone as a treatment for multiple myeloma in patients who received at least one prior therapy.

Oppenheimer analyst Brian Abrahams predicted that maintenance use of Revlimid could add $400 million in sales to Celgene's bottom line by 2013.

But Baird & Co. analyst Christopher Raymond was even more optimistic, surmising that with 5,000 U.S. patients receiving autologous stem cell transplants each year, with two years of treatment potential, additional revenues in the out years could reach up to $900 million.

The NCI-funded trial, known as CALGB 100104/ECOG 100104, was conducted by a network of researchers led by the Cancer and Leukemia Group B, in collaboration with the Eastern Cooperative Oncology Group and the Blood and Marrow Transplant Clinical Trials Network, a network co-sponsored by the NCI and the National Heart, Lung and Blood Institute.

The study enrolled 568 multiple myeloma patients ages 18 to 70 who had received no more than 12 months of prior therapy and no prior transplant. All patients received autologous transplantation following a high dose of melphalan, a drug commonly used to treat multiple myeloma.

Of those, 460 patients with adequate organ function and no evidence of progressive disease were randomized about three months after transplant to receive Revlimid or placebo. Results showed that there was a 58 percent reduction of disease progression in patients receiving Revlimid as a maintenance therapy after transplant, which the NCI said was "highly statistically significant," compared with half of those on placebo whose disease worsened within 778 days, or nearly 26 months. The NCI said that the study was the first Phase III trial to demonstrate a clinical benefit of Revlimid following transplant for multiple myeloma. Officials noted, however, that the trial has yet to show evidence of an overall survival benefit with Revlimid.

An FDA advisory panel last week gave the thumbs down to OSI Pharmaceuticals Inc.'s Tarceva (erlotinib) as a maintenance therapy in lung cancer, insisting that perhaps treating at progression might be better for that drug. (See BioWorld Today, Dec. 15, 2009, and Dec. 17, 2009.)

"To answer that question, survival becomes a critical endpoint," said Credit Suisse analyst Michael Aberman. Nonetheless, he insisted, the NCI study coupled with an earlier Revlimid trial "would lead to meaningful adoption in the maintenance setting."

Import Blockers Got Big Drug Bucks

Senators who voted last week to block prescription drug importation legislation, which failed 51-48, received an average of $85,812 in contributions over the past six years from the drug industry - a 69 percent increase over the $50,767 received by those who voted in favor of the measure, the bipartisan watchdog MAPLight.org reported.

The provision, offered by Sen. Byron Dorgan (D-N.D.) as an amendment to the Senate's health reform package, would have relaxed restrictions on imports from Canada and other highly developed countries. But 30 Democrats sided with 17 Republicans and one Independent to take down the measure. MAPLight.org found that the Senate Democrats who voted against the legislation received an average of $73,729 each from drugmakers over the past six years - 76 percent more than the $41,894 received by Democrats who voted in favor of imports.

MAPLight.org's research previously has shown alignment of pharma campaign money and votes in Congress.

For instance, Democrats on the Senate Finance Committee who voted down a measure in September that would have required drugmakers to pay $106 billion over 10 years in rebates aimed at closing the Medicare drug gap received 4.2 times more in campaign money from the industry than those who voted in favor of the measure. (See BioWorld Today, Sept. 28, 2009.)

H1N1 Flu Vaccine Now Abundant in U.S.

Health officials last week said 100 million doses of the H1N1 flu vaccine have been shipped, with restrictions now lifted in most states. "Many people have been patiently waiting their turn to get vaccinated, so our message is take advantage of the increased supply and get vaccinated as soon as you can," said Health and Human Services Secretary Kathleen Sebelius, who said she got her shot last Wednesday.

"We have a chance to lessen the impact or even prevent a big third wave when flu season really hits, and we need to seize that opportunity right now," she told reporters.

NCI SBIR Grants $11.5M in Bridge Awards

The National Cancer Institute Small Business Innovation Research Program (SBIR) last week announced $11.5 million in Phase II Bridge awards to four small businesses, including Altor BioScience Corp. Each of the awardees has raised substantial matching funds from investors. Bridge funding is aimed at helping small businesses overcome the funding gap known as the "valley of death" between the end of the SBIR Phase II award and the subsequent round of financing needed to advance a product or service toward commercialization, which officials said was "especially critical during these economic times."

Altor BioScience's award is intended to help advance the Miramar, Fla.-based biotech's bifunctional T-cell receptor-based immunotherapeutic directed against multiple types of cancer. The other three awardees in the round were device makers.