June 12th, 2013
During our first-quarter earnings call last month, I was pleased to report that we began treating patients in the ixCELL-DCM Phase 2b trial, which is evaluating ixmyelocel-T for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy (ischemic DCM). This clinical trial offers a potentially historic advancement in the treatment of patients with advanced heart failure due to ischemic DCM, an indication for which we have an orphan drug designation in the United States.
DCM is a condition in which the heart becomes weakened and enlarged and cannot sufficiently pump blood throughout the body. DCM is the third most common cause of heart failure and the most frequent cause of heart transplantation. A majority of the advanced heart failure patients who no longer respond to medical therapy — more than a quarter of a million patients in the U.S. — have DCM, and approximately 60% of these cases are of ischemic origin due to prior heart attacks or atherosclerosis. These patients typically have maximized their use of prescription and device therapies, and are no longer candidates for procedures to restore blood flow such as angioplasty and bypass surgery. At this stage of the disease, they have very limited treatment options, which generally include placement of left ventricular assist devices and heart transplantation.
What makes advanced heart failure due to ischemic DCM so challenging for patients is its impact on daily life. The condition significantly reduces exercise capacity and quality of life, because for many patients, walking even a short distance causes severe shortness of breath and fatigue. There is a strong rationale for developing ixmyelocel-T for the treatment of ischemic DCM. In preclinical studies, ixmyelocel-T was found to significantly reduce cardiac tissue damage and demonstrated additional cardioprotective effects in relevant disease models. In Phase 2a clinical trials, ixmyelocel-T was found to be well-tolerated and demonstrated positive efficacy trends, including improved symptoms and improved walking distance in treated patients compared to placebo.
The ixCELL-DCM Phase 2b trial is designed to enroll 108 patients at about 30 sites in the U.S. and Canada. This is a double-blind, placebo-controlled trial that will primarily measure mortality, hospitalizations, and heart failure emergency room visits in trial participants. The trial also will examine a range of clinical, functional, and symptomatic measures. The study is on track to complete enrollment by the end of Q1 2014 and show top-line results in Q2 2015.
Aastrom is one of the few companies working to develop a treatment for this major unmet medical need, and our clinical research program for ixmyelocel-T is one of the most advanced global research efforts in regenerative medicine. As we continue to make progress, we look forward to the opportunity to share our results and insights with patients, physicians, and researchers around the world.
April 4th, 2013
Our recent decision to implement a strategic change in our R&D programs to focus on the development of ixmyelocel-T for the treatment of dilated cardiomyopathy (DCM) and stop enrollment in the Phase 3 REVIVE trial in critical limb ischemia (CLI) reflects the significant opportunity that we see to treat advanced heart failure caused by DCM and the challenges that we faced in enrolling the REVIVE study in a reasonable timeframe. While we believe that ixmyelocel-T has strong therapeutic potential to treat CLI, based on previous clinical results showing that ixmyelocel-T was efficacious and well-tolerated in this patient population, the decision was based on our need to allocate resources to advance ixmyelocel-T toward commercialization as quickly as possible. We believe that the DCM program represents our best near-term opportunity to accomplish this goal.
Our previous results in DCM —in both preclinical and clinical studies — suggest that our patient-specific multicellular therapy can produce a range of clinical benefits for patients with severe heart failure whose limited treatment options include heart transplantation. Read More…
February 1st, 2013
Dear Friends of Aastrom,
February is American Heart Month, and it is during this time that we recognize many of the important advances in medical research that may help us prevent and treat cardiovascular disease more effectively. In recent years, breakthroughs in surgery, drug therapy and devices have expanded options for patients, making it possible to lower the risk of a cardiac event and treat the effects of peripheral artery disease (PAD). Despite these advances, cardiovascular disease remains the leading cause of death in the United States. Each year it disrupts the quality of life of millions of people and is the cause of approximately 160,000 amputations and 1.5 million heart attacks in the U.S.
Fortunately, there are reasons to be optimistic. Recent advances in regenerative medicine and cell therapy are encouraging and have shown the potential to improve the lives of patients with the most severe forms of cardiovascular disease. In preclinical and clinical studies, cell therapies have demonstrated the potential to repair damage to the heart muscle and other vascular tissue that can occur with a cardiac event. They also have the potential to help restore blood flow or repair genetic defects. If we continue to make clinical progress in this area, cell therapies may become an effective, less costly treatment option for people with advanced cardiovascular disease. Read More…
December 1st, 2012
Dear Friends of Aastrom,
Aastrom’s commitment to transparency was reinforced by the recent announcement from GlaxoSmithKline (GSK). GSK made headlines when it announced a new policy to disclose all clinical data from the company’s drug development programs. Previously, the company had followed prevailing industry standards by releasing results from only some of its clinical studies (generally, only the positive ones). A 2008 study[i] by the University of California, San Francisco found that many drug companies do not routinely report negative clinical data, while some do not make any data available to the public. We welcome GSK’s initiative – it is good clinical practice and consistent with our commitment to transparency at Aastrom. This move toward greater transparency is an important trend with profound implications for the future of research.
We believe that the benefits of disclosing all clinical results, not just the positive study results, far outweigh the real or perceived risks. Full disclosure allows any interested party to review all of the potentially relevant data about experimental therapies. This allows for a much broader and more accurate assessment of both safety and efficacy – the mechanisms of action – and also enables scientists and physicians to identify potential areas for future research. When publicly available information is incomplete, it can lead the medical community to flawed conclusions that may limit the chances of future clinical success or, in extreme cases, actually cause patients harm.
November 1st, 2012
Dear Friends of Aastrom,
There have been many studies over the years highlighting the fact that drug discovery and development is typically a very long, risky, complex and costly process. It is not uncommon for drug development programs to last a decade or more, beginning with early-stage discovery and research, and progressing through late-stage clinical development, regulatory review and approval. Within that timeline, companies must continually monitor progress and determine when and whether to expand their focus to include essential commercialization strategies to prepare to bring promising late-stage product candidates to the patients who need them.
Over the past year, Aastrom has initiated a number of important activities to support the future commercialization of ixmyelocel-T, which is currently in Phase 3 clinical trials for the treatment of patients with severe peripheral arterial disease (PAD) and existing tissue loss, and in Phase 2 trials for the treatment of patients suffering with dilated cardiomyopathy (DCM). Read More…
August 1st, 2012
Dear Friends of Aastrom,
As investors and the media continue to follow the progress of stem cell therapy companies, the need for clarity and transparency about our work is growing. We welcome this trend and the responsibility it places on us to describe clearly, openly and on a regular basis what we do.
Over the past year, we have found that investors, clinicians, patients and advocates are most interested in our answers to three key questions:
1. Why do you believe Aastrom’s stem cell therapy will work more effectively than other methods of treating disease?
2. What makes critical limb ischemia (CLI) an appropriate target for Aastrom’s stem cell therapy?
3. When will a large pharmaceutical company embrace stem cell therapy and partner with Aastrom?
The answer to the first question is that bone marrow cells have been used safely and effectively for over 30 years to fight certain diseases. Our stem cell therapy is produced from each patient’s bone marrow. Certain cell types found in and expanded from bone marrow – especially the CD90+ mesenchymal stromal cells and the CD14+auto+ M2 macrophages that comprise the majority of our stem cell therapy – have been shown to support tissue remodeling, address chronic inflammation and promote angiogenesis. No other stem cell therapy has this unique combination of cells. Based on compelling preclinical and clinical results, we believe that delivering our stem cell therapy directly to damaged tissue has therapeutic effects for CLI patients that are not possible with other treatment methods such as open surgical procedures, endovascular devices or traditional pharmaceuticals. Our clinical results thus far provide strong support for this conclusion. Read More…
June 4th, 2012
Dear Friends of Aastrom,
Whenever I am asked what makes Aastrom a leader in cell therapy, I usually begin my answer by citing the evidence of our leadership.
During the past year, we have demonstrated the therapeutic potential of our unique product to treat cardiovascular diseases for which there are no other approved therapies. We have validated and expanded our efficient, proprietary, high-margin, state-of-the-art cell-production technology. We have launched the largest and most advanced clinical research program in critical limb ischemia and attracted significant new capital from institutional investors. These achievements reflect the enormous progress we have made to advance our unique cell therapy into late-stage clinical development.
The qualities that make Aastrom a leader take longer to describe but they are no less important to understanding why we have been successful. They include how we work, the values we uphold and the company we keep.
How we work at Aastrom is a reflection of our unique corporate culture and the talented team we have assembled. We are not unique among biotechnology companies simply because we work hard and believe in what we do. We are unique because we have skills and experience unlike any other company and bring unwavering dedication to our mission. As a result, we have identified important unmet medical needs and markets where our technology can best be applied – and we have moved rapidly to address these new opportunities. Read More…