April 30, 2012
Preventing the Problem Before it Occurs – Athersys Meets with FDA to Discuss Novel Approach to Suppressing Graft Versus Host Disease in Leukemia and other Cancer Patients
Graft versus Host Disease (GvHD) is an area of great unmet medical that represents a significant risk to cancer patients throughout the world. The condition occurs when patients receive a bone marrow transplant following radiation and/or chemotherapy, and the transplanted immune cells begin to attack the patient, causing significant pain and disability, or even death.
Among patients being treated for cancers of the blood and immune system, roughly half will experience GvHD, and many of those will either experience chronic and debilitating disease, or eventually die. In fact, chronic GvHD kills up to 33 percent of all those who survive long-term after stem-cell transplants for leukemia. Several companies are pursuing new treatments for treating patients who are suffering GvHD. Acquisition news is in the headlines this week with Jazz Pharmaceuticals (NASDAQ: JAZZ) dolling-out nearly $700 million to acquire EUSA Pharma Inc, whose pipeline includes Leukotac® (inolimomab), an anti-CD25 monoclonal antibody in a phase III pivotal study in Europe for treatment of steroid-refractory acute graft versus host disease.
Many new therapies are designed to manage symptoms after the disease has already gotten out of control – and in the case of GvHD that’s analogous to trying to put out a forest fire after it’s started to rage out of control. However, one innovative company is taking a different approach – by developing a new therapy designed to prevent the GvHD from ever happening in patients that are at high risk of the condition. Based in Ohio near the renowned Cleveland Clinic and other notable medical and research institutions, including University Hospitals and the National Center for Regenerative Medicine, Athersys, Inc. (NASDAQ: ATHX) is moving to the forefront of the industry with some promising new therapies and an approach to preventing GvHD before it’s raging out of control – sort of like putting it out at the campfire stage.
Recently, Athersys, Inc. announced some encouraging initial data from a clinical study involving administration of MultiStem® to patients being treated for leukemia or other conditions that place them at risk of GvHD. That study showed some promising results, with a reduction in GvHD and potential for other clinical benefits. Last week, the company announced that it held a scheduled meeting with the FDA to discuss the results of its recently completed clinical trial. Additionally, the company discussed with the FDA its proposed plans for the next phase of clinical development, answered questions and obtained feedback on a number of specific issues related to the potential study parameters and proposed trial design. While it will undoubtedly take some time to map out the specifics of the next phase of clinical development, it would seem that since this could represent an opportunity for “fast track” development for a promising new therapy in a recognized orphan indication, and area of great clinical need.
“This was an important first step in defining the next phase of clinical development for this program. We sincerely appreciate the collaborative approach the FDA is taking regarding the development of innovative new therapies designed to address serious conditions and areas of significant unmet medical need,” commented Gil Van Bokkelen, Chairman & CEO at Athersys. “We look forward to getting additional input from the agency as we work with them to finalize the study design.”
About Graft versus Host Disease
Leukemia and certain related conditions are often treated with radiation and chemotherapy to eliminate cancerous or diseased cells, but this process also severely compromises the native blood forming and immune system in the patient, leaving them susceptible to infection and other complications. To address this, a patient will often receive an allogeneic HSCT, whereby following radiation and chemotherapy treatment a patient’s blood stem cells are replaced with a transplant of hematopoietic stem cells obtained from the bone marrow or peripheral blood of a healthy donor. Donors may be related or unrelated to the patient, but are matched according to tissue type in order to minimize the potential for GvHD, where donor immune cells transplanted with the donor HSCT attack tissue and organs of the patient. Following the transplant, the patient will often remain hospitalized in specialized units until successful engraftment provides a sufficiently functional immune system.
According to the Center for International Blood and Marrow Transplant Research, each year there are approximately 25,000 allogeneic HSCT performed globally, although this number is projected to increase due to the anticipated growth in incidence of hematologic malignancies associated with an aging population. While this treatment approach can be an effective medical therapy for these types of cancer, it is often associated with substantial tissue damage and side effects. GvHD is a frequent complication associated with allogeneic HSCT, affecting approximately half or more of transplant recipients, and advanced GvHD can be severely debilitating or even fatal. Several factors affect a patient’s likelihood of having GvHD and GvHD severity, including the treatment protocol used, the degree of tissue match between donor and recipient (with lower GvHD rates and severity associated with related donors and better tissue matches), and the condition of the patient among other factors.
MultiStem® cell therapy is a patented product that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of multiple therapeutic factors produced in response to signals of inflammation and tissue damage. MultiStem has demonstrated therapeutic potential for the treatment of inflammatory and immune disorders, neurological conditions, and cardiovascular disease, as well as other areas, and represents a unique “off-the-shelf” stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. The product is extensively characterized for safety, consistency and potency. Athersys has forged strategic partnerships with Pfizer Inc. to develop MultiStem for inflammatory bowel disease and with RTI Biologics, Inc. to develop cell therapy for use with a bone allograft product in the orthopedic market.
Athersys is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem® cell therapy product, a patented, adult-derived “off-the-shelf” stem cell product platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The Company currently has several clinical stage programs involving MultiStem, including for treating inflammatory bowel disease, ischemic stroke, damage caused by myocardial infarction, and for the prevention of graft versus host disease. Athersys has also developed a diverse portfolio that includes other technologies and product development opportunities, and has forged strategic partnerships and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at www.athersys.com <http://www.athersys.com> .
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