TiGenix Announces Top-Line Phase I/II Results of AlloCSC-01 in Acute Myocardial Infarction

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TiGenix, stem cell specialist focused on developing novel therapeutics from its two proprietary platforms of donor-derived expanded adipose derived stem cells (eASC) and donor-derived expanded cardiac stem cells (AlloCSCs), today announced top-line one-year results from the CAREMI clinical trial, an exploratory Phase I/II study of AlloCSCs in acute myocardial infarction (AMI).

CAREMI is the first-in-human clinical trial with the primary objective being safety and evaluating the feasibility of an intracoronary infusion of 35 million of AlloCSCs in patients with AMI and left ventricular dysfunction treated within the first week post-AMI. Importantly, the trial is the first cardiac stem cell study to integrate a highly discriminatory magnetic resonance imaging (MRI) strategy to select patients at increased risk of heart failure and late adverse outcomes. CAREMI was not powered to establish efficacy therefore no conclusion can be drawn on the secondary efficacy end-points.

The main findings of this study are:

  • All safety objectives of the study have been met. No mortality or major cardiac adverse events (MACE) have been found at 30 days meeting the primary end-point of the study. Moreover no mortality and MACE have been found at 6 months or 12 months follow-up
  • Of particular relevance to this allogeneic approach, no immune-related adverse events have been recorded at one-year follow-up
  • A larger reduction in infarct size was found in one pre-specified subgroup associated with poor long-term prognosis and representing more than half of the patient population of the randomization phase of the study. This finding has revealed valuable insight, and provides a specific direction for potential studies in a targeted subset of high-risk patients

“This is the first trial in which it has been demonstrated that allogeneic cardiac stem cells can be transplanted safely through the coronary tree, and in the worst possible setting represented by patients with an acute heart attack with left ventricular dysfunction,” commented Professor Fernández-Avilés, Head of the Department of Cardiology at the Hospital General Universitario Gregorio Marañón in Madrid (Spain), principal investigator on the trial in Spain. “It is especially encouraging that no cardiac or immunological side effects were observed.”

“This is the first study in which we have used a state of the art comprehensive MRI analysis to include patients with a large myocardial infarction in an innovative cell therapy protocol,” said Professor Janssens, Head of the Department of Cardiovascular Diseases, University Hospital, Leuven (Belgium), and principal investigator on the trial in Belgium. “Serial MRI analysis and extensive immunological profiling will allow us to further explore the encouraging signals we observed in cell treated patients with the worst MRI signature. These findings offer an exciting prospect for targeted follow-up studies in these high-risk patients.”

“Besides confirming the long term safety of the treatment these results suggest interesting opportunities in populations with high unmet medical need,” said Dr. Marie Paule Richard, Chief Medical Officer at TiGenix. “We look forward to working with our advisors to analyze the data in depth and determine the best way forward with AlloCSC-01 during the second half of this year.”

Full data results from the CAREMI study will be presented at an upcoming medical congress.

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