AlloCSC-01 consists of adult allogeneic cardiac stem cells isolated from donors, and expanded in vitro. The product has just completed a phase I/II study in acute myocardial infarction (the CAREMI study), demonstrating that allogeneic cardiac stem cells can be transplanted safely through the coronary tree and lead to a reduction in infarct size in patients with a large myocardial infarction.
MEDICAL NEED AND POTENTIAL MARKET
Cardiovascular disease is the most common cause of death, leading to 17.5 million deaths worldwide in 2012, of whom 7.4 million people died of ischemic heart disease, or decreased blood flow to the heart, according to the World Health Organization (WHO). Up to 1.9 million people annually are diagnosed with acute myocardial infarction in the United States, Europe and Japan, according to the Acute Coronary Syndrome Cardium Study by Decision Resources (January 2015), most of whom are treated by percutaneous coronary angioplasty and the implantation of one or more stents. Congestive heart failure following myocardial infarction affects 26 million patients.
In 2016, the American Heart Association (AHA) estimated that the direct and indirect cost of coronary heart disease, the main cause of myocardial infarction, was $182 billion and is expected to reach $322 billion in 2030. Similarly the cost of heart failure in the United States was estimated at $24 billion for 2015, reaching $47 billion in 2030.
Successful treatment of acute myocardial infarctions (AMI) has increased short-term survival but contributed to a Chronic Heart Failure (CHF) epidemic (26M patients worldwide, according to Ambrosy PA et al., (J Am Coll Cardiol. 2014;63:1123-1133). CHF post-AMI is a terminal disease with an annual mortality rate of ~5% after the first episode, for which there is currently no curative treatment with the exception of heart transplantation.
We believe that AlloCSC-01 can be used within a few days after the stent is inserted to limit the extent of tissue damage, through three potential modes of action:
- By secreting protective factors in the recently damaged cardiac tissue, AlloCSC-01 could reduce cell death produced both when blood flow is interrupted and when it is restored, thus salvaging valuable tissue.
- By controlling inflammation, AlloCSC-01 could limit the scarring of cardiac tissue in the infarcted region, which would lead to an improved prognosis.
- AlloCSC-01 could support the regeneration of new viable tissue from resident cardiac cells, improving the functional capacity of the cardiac muscle.
Clinical evidence was gathered in the CAREMI trial, which was designed as a Phase I/II trial in acute myocardial infarction (AMI). The trial enrolled 51 patients with AMI and left ventricular dysfunction, of which 35 received a single intracoronary administration of AlloCSC-01 and 16 patients received placebo. Patients were treated within the first week post-AMI.
On 13th March 2017, TiGenix released top-line one year results of the CAREMI Phase I/II study. The study met all safety objectives, demonstrating that allogeneic cardiac stem cells can be transplanted safely through the coronary tree. Study results also indicated a larger reduction in infarct size in one pre-specified subgroup associated with poor long-term prognosis (patients with a large myocardial infarction).
Further information on the CAREMI trial can be found on: Clinical Trials
The CAREMI trial is part of the European CARE-MI project (Cardio Repair European Multidisciplinary Initiative), whose main objective is to advance the development of cell therapies for myocardial infarction. This project is funded by the Seventh Framework Programme of the European Union under the coordination of the Centro Nacional the Investigaciones Cardiovasculares (CNIC) and the Centro Nacional de Biotecnología (CNB) and the participation of research institutions and companies from nine EU countries.
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