"We are pleased to have been granted the benefits of orphan drug designation in
Patients with leukemia or other related malignancies are typically treated by radiation and chemotherapy, which are administered to destroy cancerous cells, but also substantially impair the blood forming and immune system of the patient. These procedures are followed by a HSC transplant to reconstitute the immune system to fight infection and any remaining malignancy. Patients undergoing donor derived, or allogeneic, HSC transplants are at significant risk for serious complications, including GvHD, which results when transplanted immune cells attack various tissues and organs in the patient. GvHD can be severe and life-threatening, with substantial impact on overall treatment requirements and costs, as well as on the patient's quality of life. Annually, there are estimated to be more than 25,000 allogeneic HSC transplants in the developed countries.
Currently, the Company is preparing for a Phase II/III clinical study in the area. It has met with the
Orphan drug designation, which is intended to facilitate drug development, provides substantial potential incentives to the sponsor, such as fee reductions for agency meetings, study-design assistance, opportunities for expedited product development, orphan grant access, tax incentives and market exclusivity for the product upon regulatory approval (7 years for the U.S. and up to 10 years for the EU).
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. These forward-looking statements relate to, among other things, the expected timetable for development of our product candidates, our growth strategy, and our future financial performance, including our operations, economic performance, financial condition, prospects, and other future events. We have attempted to identify forward-looking statements by using such words as "anticipates," "believes," "can," "continue," "could," "estimates," "expects," "intends," "may," "plans," "potential," "should," "suggest," "will," or other similar expressions. These forward-looking statements are only predictions and are largely based on our current expectations. A number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face that could cause actual results to differ materially from those implied by forward-looking statements are the risks and uncertainties inherent in the process of discovering, developing, and commercializing products that are safe and effective for use as human therapeutics, such as the uncertainty regarding market acceptance of our product candidates and our ability to generate revenues, including MultiStem for the treatment of inflammatory bowel disease, acute myocardial infarction, stroke and other disease indications, including lysosomal storage disorders, and the prevention of graft-versus-host disease. These risks may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements. Other important factors to consider in evaluating our forward-looking statements include: our ability to raise additional capital; final results from our MultiStem clinical trials; the possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete clinical trials; changes in external market factors; changes in our industry's overall performance; changes in our business strategy; our ability to protect our intellectual property portfolio; our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; our ability to meet milestones under our collaboration agreements; our collaborators' ability to continue to fulfill their obligations under the terms of our collaboration agreements; the success of our efforts to enter into new strategic partnerships and advance our programs; our possible inability to execute our strategy due to changes in our industry or the economy generally; changes in productivity and reliability of suppliers; and the success of our competitors and the emergence of new competitors. You should not place undue reliance on forward-looking statements contained in this press release, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.
CONTACT: William (
B.J.) Lehmann, J.D. President and Chief Operating Officer Tel: (216) 431-9900 firstname.lastname@example.org Investor Relations: Lisa M. Wilson In-Site CommunicationsTel: (917) 543-9932 email@example.com
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