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Nexell Therapeutics Files IND for Blood Stem Cell Therapy
Registration Trial in CGD is First for Novel, Curative Approach to Non-Malignant Blood Disorders

IRVINE, Calif.--(BUSINESS WIRE)--July 2, 2001--Nexell Therapeutics Inc. (Nasdaq:NEXL - news) announced today that it has filed an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration (FDA) to allow the company to initiate a pivotal study of hematopoietic (blood) stem cell transplantation as a treatment for chronic granulomatous disease (CGD), a rare inherited blood disorder that leaves patients vulnerable to potentially fatal infections. The trial will be conducted at the National Institutes of Health (NIH) under a Cooperative Research and Development Agreement (CRADA).

``This is a significant milestone for the company,`` said William A. Albright, Jr., President and Chief Executive Officer of Nexell Therapeutics. ``This filing represents the transition of our product development strategy from cell processing instruments and disposables to the manufacture of therapeutic cellular products. We expect the data from this trial will support an indication for a stem cell product with demonstrated clinical efficacy, the first in a series of potential indications to treat a variety of non-malignant, inherited blood disorders that afflict tens of thousands of people every year.``

Nexell plans to enroll approximately 45 patients in a controlled trial to compare the efficacy of current standard therapy (antibiotics/interferon gamma) with a non-myeloablative (not destructive of bone marrow) conditioning regimen followed by a T-cell depleted allogeneic (donor origin) hematopoietic stem cell transplant. The principal clinical endpoint of the trial is a reduction in the incidence of catalase positive infections (typical in CGD) in the transplanted patients with acceptable side effects. Nexell hopes to complete patient enrollment in 2002 or early 2003 and plans to seek FDA approval based on a Biological License Application (BLA).

``Because it is of manageable size, can be conducted in a single center and has near-term endpoints, this trial design is an efficient way to provide the first proof of efficacy for a stem cell product,`` said Mr. Albright. ``We have worked hard with the FDA to refine the protocol for this trial and confirm the viability of the BLA approval pathway for this and future therapeutic products based on adult blood stem cells.``

CGD is caused by a group of gene mutations that prevent white blood cells called neutrophils from producing oxygen compounds, their main weapon against bacteria or fungi. As a result, patients suffer from recurrent, often life-threatening infections and the formation of inflammatory nodules called granulomas in their lungs, livers and other organs. To help prevent potentially fatal complications, most CGD patients must receive antibiotics and other drugs on a regular basis. Despite the availability of this treatment, the annual mortality rate for CGD is two to five percent and many patients succumb to fatal infections by the time they reach their thirties. Nexell anticipates that stem cell therapy, if successful, will not only increase life expectancy but also improve the quality of life for patients with this disease.

Independent research performed at the NIH and published last March in The New England Journal of Medicine(a), demonstrated that blood stem cells from matched related donors could be transplanted successfully into CGD patients to generate functioning neutrophils, producing a persistent reduction in the signs and symptoms of the disease. Although the researchers considered their approach high risk, they were able to improve its safety profile by removing T-cells from the donor grafts with Nexell cell processing technology to reduce graft-versus-host disease (GVHD), a potentially fatal complication of allogeneic stem cell transplantation.

More information about the CGD clinical trial is available from http://clinicaltrials.gov or by calling the NIH Clinical Center patient recruitment office at 1-800-411-1222.