NitroMed Inc.'s stock soared 73.1 percent Monday when the firm suggested it could launch BiDil for African-Americans with heart failure one year ahead of schedule, after stopping a confirmatory trial due to favorable preliminary results.
The company's stock (NASDAQ:NTMD) jumped $4.31 Monday to close at $10.21 once the market learned that early data from NitroMed's 1,000-patient study revealed a significant survival benefit in African-Americans.
Indeed, in a conference call, Anne Taylor, chairman of the trial steering committee and professor of medicine at the University of Minnesota, said it appears that BiDil improves survival, decreases hospitalization and improves quality of life in African-American patients.
The study's success represents a major milestone in the treatment of African-Americans, who generally suffer a disproportionate burden of heart disease and are underrepresented in clinical trials, Taylor said.
Referred to as A-HeFT (African-American Heart Failure Trial), the study was designed to evaluate the efficacy of BiDil when taken daily in addition to the best current therapy. BiDil is an orally administered nitric oxide-enhancing medicine, which combines isosorbide dinitrate and hydralazine.
NitroMed's decision to stop the trial followed unanimous recommendations from both the Data Safety Monitoring Board and the trial's steering committee, Michael Loberg, the firm's president and CEO, told BioWorld Today.
"I do think the company today is getting recognition that its clinical and research programs can bring value into this space," Loberg said. "Today is a day that we feel very, very good about ourselves."
Loberg would not discuss specifics about the data, saying it continues to be analyzed. He expects to begin releasing quantitative results in a peer-reviewed fashion during the fourth quarter.
The FDA requested the confirmatory trial in March 2001 after reviewing the firm's new drug application, which was based on two large-scale vasodilator heart failure trials (V-HeFT I and II). Published analyses from those trials showed that BiDil provided a selective benefit to black patients with heart failure as compared to non-black patients receiving the drug. (See BioWorld Today, March 9, 2001.)
The agency's request was contained in an approvable letter that said positive results from a confirmatory trial would support marketing approval, Liana Moussatos, an analyst with Pacific Growth Equities in San Francisco, said in a research note. Moussatos said the FDA will have six months to review the additional data once submitted.
However, Loberg was reluctant to offer a timeline as to when he anticipates filing the confirmatory study or when he expects FDA action. He said the company plans to have its sales and marketing programs in place by the first quarter of 2005 in the event of a quick FDA decision. "We have no evidence that will happen, but we are preparing for it," he said.
While the company has every intention of taking BiDil to market on its own, Loberg is cognizant of the fact that stopping the trial early limits the time the company has to prepare for launch. With that in mind, Loberg might consider an incremental field sales force.
The company had estimated that about 200 people could handle 80 percent of the physicians who treat African-Americans with heart failure.
Data from the U.S. Census Bureau and the Centers for Disease Control and Prevention estimate that each year there are 750,000 African-Americans in the U.S. with heart failure. The number is expected to grow to about 900,000 people by 2010, NitroMed said.
The A-HeFT clinical trial, conducted at 170 sites throughout the U.S., was designed to demonstrate that BiDil, when administered with standard heart failure therapies, can provide a combination of reduced mortality and hospitalization for heart failure and improved quality of life for African-Americans with heart failure.
The randomized, double-blind, placebo-controlled study was targeted to enroll 1,100 black men and women with moderate to severe heart failure, categorized as New York Heart Association heart failure functional classifications III and IV. Eligible patients also were required to have a reduced ability of the heart to pump blood efficiently, as characterized by a decreased ejection fraction as well as enlarged left ventricles.
The study treatment included either BiDil or a placebo in addition to the patient's standard course of treatment for their heart failure, which might have included angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, beta-blockers, diuretics, spironolactone and digitalis.
NitroMed in 1999 acquired BiDil's NDA and related intellectual property from Jay Cohn, a professor at the University of Minnesota.