Pharmacology
N.O.V.E.L. 2 to Evaluate Sargramostim in Steroid-Dependent Patients of Crohn's Disease
By Berlex, Inc.
May 6, 2005, 18:22

Berlex, Inc., a U.S. affiliate of Schering AG, Germany , today announced patient enrollment has been completed for N.O.V.E.L. 2, a Phase 2 clinical trial evaluating LEUKINE in steroid-dependent patients as a potential new treatment option for Crohn's disease.

"We are excited to have reached this crucial milestone in our Crohn's disease trials," said Reinhard Franzen, President and CEO of Berlex Laboratories. "With the completion of enrollment into the N.O.V.E.L. 2 trial, and our continuing patient recruitment for the Phase 3 trials, Berlex is committed to ongoing research for safe and effective treatments for patients with Crohn's disease, a serious and debilitating condition for which there is no cure."

N.O.V.E.L. 2, a randomized, double-blind, placebo-controlled, multi-center trial in the United States and Canada, is evaluating the steroid-sparing properties of LEUKINE treatment in patients with active steroid-dependent Crohn's disease.

In the study, patients with moderately to severely active Crohn's disease who cannot taper steroids without disease relapse are randomized 2:1 to receive LEUKINE or placebo via daily subcutaneous (under the skin) self-injections for up to 22 weeks. Steroid therapy is withdrawn via a pre-determined schedule.

Results from N.O.V.E.L. 2 are expected to be available in 2006.

"Many of my Crohn's disease patients are unhappy with being on steroid treatment -- either because of the side effects they are experiencing with steroids, because the efficacy has decreased over time or because they don't like being on long-term immunosuppression," said N.O.V.E.L. 2 lead investigator John Valentine, M.D., Associate Professor, University of Florida, Gainesville. "This trial is an important step in looking at treatment alternatives for this disabling disease."

Berlex is actively recruiting patients in the United States for N.O.V.E.L. 3, a Phase 3 randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the potential for disease response and remission following retreatment with LEUKINE in Crohn's disease patients who have demonstrated response to an initial cycle of treatment.

During the initial eight week treatment course, patients will receive LEUKINE alone. Patients who respond to treatment will be followed regularly, then retreated if they experience a disease relapse. During the eight-week retreatment course, patients will receive either LEUKINE or placebo. Neither the patient nor physician will know if the patient is receiving LEUKINE or placebo. Patients will have a 50 percent chance of receiving LEUKINE. Both LEUKINE and placebo will be administered by daily subcutaneous (under the skin) self-injections.

Patients must be 18 years or older and must have active Crohn's disease to participate in this trial.

These trials are part of N.O.V.E.L.(TM) (New Opportunities to Verify Evolving Logic in Crohn's disease), a comprehensive, worldwide clinical trials program supported by Berlex, Inc., for the ongoing development of LEUKINE as a potential new treatment for Crohn's disease.

Evolving research suggests the primary defect in Crohn's disease may arise from a breakdown in the intestinal barrier composed of several layers of cells that protect the gastrointestinal (GI) tract. This breakdown may precede reactive inflammatory processes associated with Crohn's disease. LEUKINE may address the primary defect of Crohn's disease by helping to improve cell function within the intestinal barrier, unlike other therapies that broadly suppress immune response and inflammatory symptoms.

In N.O.V.E.L. 1, a multi-center, randomized, double-blind, placebo- controlled Phase 2 trial, patients receiving LEUKINE experienced significantly greater clinical response and remission rates than those receiving placebo. Follow-up to N.O.V.E.L. 1 showed that patients treated with LEUKINE maintained improvements in disease severity and quality-of-life even after therapy was completed.

About LEUKINE

LEUKINE, a man-made form of a naturally occurring growth factor that helps fight infection and disease in appropriate patients by enhancing cells of the immune system, was approved in the United States in 1991, and is marketed by Berlex, Inc.

LEUKINE is the only growth factor approved in the United States for use following induction chemotherapy in older adults with acute myelogenous leukemia (AML) to shorten the time to neutrophil recovery and reduce the incidence of severe and life-threatening infections and infections resulting in death.

LEUKINE has also been approved in the U.S. for use in four additional indications: myeloid reconstitution following allogeneic and autologous bone marrow transplantation (BMT), peripheral blood stem cell (PBSC) mobilization and subsequent myeloid reconstitution in patients undergoing PBSC transplantation, and bone marrow transplantation failure or engraftment delay. LEUKINE has been administered to more than 300,000 patients.

About N.O.V.E.L.(TM)

N.O.V.E.L. encompasses five major Phase 2 and Phase 3 trials designed to evaluate disease response and remission in both the induction and retreatment settings, as well as in steroid-dependent patients.

Enrollment is currently open for N.O.V.E.L. 3, 4 and 5. A phase I/II pediatric trial is also ongoing.

The N.O.V.E.L. Clinical Trials Program encompasses the following trials:

-- N.O.V.E.L. 1: A multi-center, randomized, double-blind, placebo-controlled Phase 2 trial;

-- N.O.V.E.L. 2: A Phase 2 trial evaluating steroid-dependent Crohn's disease patients in the United States and Canada;

-- N.O.V.E.L. 3: A Phase 3 randomized, double-blind, placebo-controlled, multi-center study in the United States designed to evaluate disease response and remission following retreatment with LEUKINE in Crohn's disease patients who have demonstrated response to an initial cycle of treatment;

-- N.O.V.E.L. 4: A Phase 3 randomized, double-blind, placebo-controlled, multi-center study in approximately 10 countries outside of the United States designed to evaluate the induction of disease response and remission in Crohn's disease patients treated with LEUKINE;

-- N.O.V.E.L. 5: An open-label extension study for all Crohn's disease patients who have previously participated in a N.O.V.E.L. clinical trial.

About Crohn's Disease

There are approximately 400,000-500,000 cases of diagnosed Crohn's disease in the United States and an estimated one million diagnosed cases worldwide. Crohn's disease affects men and women equally and seems to be more common within families. About 20 percent of people with Crohn's disease have a blood relative, most often a brother or sister, with some form of inflammatory bowel disease (IBD).

The most common symptoms of Crohn's disease are abdominal pain and diarrhea. Other symptoms include rectal bleeding, fever, loss of appetite and weight loss.

Crohn's disease can be difficult to diagnose because its symptoms are similar to other intestinal disorders such as irritable bowel syndrome (IBS) and ulcerative colitis. Crohn's disease causes inflammation in the GI tract and usually occurs as an inflammation of the small bowel or colon; however, it can affect any part of the digestive tract from the mouth to the anus. The inflammation from Crohn's disease can extend deeply into the lining of the affected organ, causing pain and frequent bowel movements.

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