AnorMED has initiated a new Phase I clinical trial in healthy volunteers to measure the increase in stem cells when the Company's drug candidate AMD-3100 is used in combination with Neupogen, compared to either drug alone. AnorMED is evaluating AMD- 3100's ability to increase the stem cells available for transplant, improve the overall transplantation procedure and potentially enhance the patient outcome.
The new combination study, being conducted at the University of Washington in Seattle, is currently enrolling up to 18 healthy volunteers. As in AnorMED's first Phase I study in healthy volunteers, initiated in March 2001, AMD-3100 will be administered subcutaneously at doses previously determined to be safe. AnorMED expects to present additional data on AMD-3100 in this indication throughout 2002.
"Clinical results to date with AMD-3100 in the mobilization of stem cells are very encouraging and support further evaluation of the drug candidate as a new agent for stem cell transplant," said Dr. Michael Abrams, president and CEO of AnorMED Inc. "This is a program that we can potentially take through Phase II clinical trials independently, which are planned for the second half of this year, to establish proof of principle for its use in stem cell transplant. We view this a valuable commercial opportunity for AnorMED, particularly given the increasing use of peripheral blood stem cell transplants in cancer patients, as well as in autoimmune and inflammatory diseases."
Previous clinical studies in healthy volunteers indicate that a single injection of AMD-3100 increases the number of white blood cells, specifically stem cells, in the circulating (peripheral) blood. Peak stem cell increases were observed six hours after injection and subsequently returned to normal levels. Preclinical studies have shown that AMD-3100 acts synergistically with Neupogen to increase the number of white blood cells available for harvest for use in stem call transplants. These results were presented at the American Society of Hematology meeting in December 2001.
Neupogen, developed and marketed by Amgen, is the standard agent used in peripheral blood stem cell (PBSC) transplant for cancer patients and other diseases. AMD-3100, is a new drug candidate developed by AnorMED, that blocks a specific cellular receptor triggering the movement of stem cells out of the bone marrow and into circulating blood. AnorMED is also conducting additional preclinical studies to evaluate the potential use of stem cells mobilized by AMD-3100 in tissue regeneration and repair, among other indications.
PBSC transplant is a medical procedure used to restore the immune system in patients who have undergone chemotherapy and radiation therapy. It is used for patients who have cancers such as leukemias, multiple myelomas and lymphomas where radiation and chemotherapy destroy the immune system and certain types of blood cells. In addition, it is being evaluated as a new therapy in patients with autoimmune and inflammatory diseases, such as multiple sclerosis.
PBSC is less invasive than bone marrow transplants, which requires a surgical procedure to remove stem cells directly from the patients or donors bone. PBSC involves a series of injections to stimulate and collect stem cells, and is rapidly replacing bone marrow transplant as the standard procedure. In 1999 it is estimated that over 31,900 PBSC transplants were performed in North America and Europe. The use of PBSC is increasing for a number of other reasons including: an overall increase in both self and donor transplants for certain indications and increased donor availability as well as a shift toward the use in elderly patients due to improved procedure tolerability and transplant outcome.
Currently, in PBSC, stem cells (CD34+ cells) are collected from patients intravenously and stored prior to the patient undergoing radiation therapy and chemotherapy. These cells are then re-administered to the patient following radiation and chemotherapy regimens in order to re-establish the immune system. This process involves the administration of Neupogen before the stem cells are harvested in order to increase the number of stem cells in the peripheral blood available for transplantation. AMD-3100 may increase the stem cells available for PBSC transplant, improve the overall transplantation procedure and potentially enhance the patient outcome.
AnorMED's core strength involves the application of chemistry, biochemistry and biology to the discovery and development of small molecule therapeutics for the treatment of diseases including inflammation, cancer and HIV. AnorMED's late stage clinical products include: Foznol, exclusively licensed to Shire Pharmaceuticals Group plc, which is on track for a US filing and European market approval by mid-year 2002; AMD-473, a new platinum based anti-cancer agent, in ongoing Phase II trials; AMD-3100, a potential new agent for stem cell transplant in cancer patients currently in a Phase I trial; and Apomate, a prognostic imaging agent for cancer and other diseases, which employs a technology licensed to North American Scientific Inc. by AnorMED.