Data highlights from the 8-week interim analysis include:
"These results confirm the consistent safety profile
"In the meantime, we continue to advance our other clinical and preclinical programs. We have a strong balance sheet, and are pursuing multiple exciting opportunities where we remain confident MultiStem and our other technologies will have an important impact," concluded Dr.
Phase 2 Clinical Study Design
The randomized, double-blind, placebo-controlled Phase 2 clinical trial is being conducted by Pfizer under a collaboration and license agreement with
Subjects enrolled in the efficacy stage of the study received either MultiStem treatment or placebo initially, followed by a second round of treatment with MultiStem therapy or placebo at eight weeks. The primary endpoints for the study include incidence and severity of adverse events over 16 weeks, change in endoscopic score (as measured by modified Baron score) at week eight, and changes in the Mayo rectal bleeding sub-score at weeks four and eight. Additionally, there are multiple secondary and exploratory endpoints evaluating disease indicators and markers over 16 weeks and through the entire study period.
Of the 88 patients enrolled in the larger cohort, 48 patients were enrolled in to the MultiStem treatment group and 40 patients were enrolled in the placebo group. The mean disease duration for patients enrolled in the efficacy stage of the study was 10 years and the other baseline characteristics confirm a patient population with advanced ulcerative colitis.
|Age, y, mean||41.2||41.0|
|Mayo score, mean||8.3||8.6|
|Prior anti-TNF therapy, %||60%||67%|
|Concommitant medication, %|
Enrollment was completed in
About the Disease Condition
Ulcerative colitis (UC) is an inflammatory bowel disease (IBD). IBD is a group of chronic, relapsing inflammatory and autoimmune conditions. The most common IBD conditions include ulcerative colitis and Crohn's disease, which are estimated to affect more than four million people in
In UC, the sub-mucosa of the colon becomes progressively dominated by infiltration of lymphocytes causing further damage. UC patients most commonly present with diarrhea, urgency, rectal bleeding, and abdominal pain. Patients may also experience fatigue, fevers, weight loss, and dehydration, and the symptoms can be incapacitating. The disease is characterized by periods of increased disease activity, or flares, separated by periods of disease remission, and by progression in disease severity over time. The complications of UC may require surgery to remove the affected region of the colon, and may also require temporary or permanent colostomy.
First-line therapies for mild to moderately active UC are usually 5-Aminosalicylate acids (5-ASA) agents. Corticosteroids are often used as the second-line therapy, but these medications are associated with side effects and many patients develop steroid dependency. Alternative therapies include immunosuppressants, such as azathioprine (AZA) or 6-mercaptopurine (6-MP), or in severe cases, cyclosporine. For moderately to severely active UC, biologics such as anti-TNF therapies may be used to induce and maintain remission, but these therapies are not effective in many patients and may be associated with serious side effects.
MultiStem cell therapy is a patented regenerative medicine 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. It 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.
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; the timing of and 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 email@example.com
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