Chronic Heart Failure

MPC-150-IM is a Phase 3 product candidate being developed as a treatment for both advanced and end-stage chronic heart failure (CHF).

Disease Indication and Patient Population

CHF is characterized by an enlarged heart and insufficient blood flow to the organs and extremities of the body. The condition, which affects 2% of the adult population of the United States, is progressive and can be caused by many factors that put an excess demand on the heart muscle such as high blood pressure, faulty valves, infections or congenital heart problems. CHF prevalence is expected to grow 46% by 2030, affecting more than 8 million Americans1.

CHF is classified in relation to the severity of the symptoms experienced by the patient. The most commonly used classification system was established by the New York Heart Association (NYHA) and ranges from Class I (mild) to Class IV or end stage (severe).

Patients with advanced or Class II/III heart failure disease continue to represent the greatest unmet medical need despite recent advances in new therapies.


MPC-150-IM is a tier 1 product candidate which consists of 150 million mesenchymal precursor cells (MPCs) administered by direct injection into the heart muscle in patients suffering from CHF and progressive loss of heart function.

Mechanism of Action

MPCs release a range of factors when triggered by specific receptor-ligand interactions within damaged tissue. Based on preclinical data, it is believed that these factors induce functional cardiac recovery by simultaneous activation of multiple pathways, including induction of endogenous vascular network formation, reduction in harmful inflammation, reduction in cardiac scarring and fibrosis, and regeneration of heart muscle through activation of tissue precursors.

Clinical Trials

Ongoing Phase 3 Trial

Approximately 600 patients are being enrolled in a placebo-controlled Phase 3 trial to evaluate a single dose of MPC-150-IM in Class II/III CHF patients across multiple sites in North America. Patients with advanced heart failure are expected to constitute the majority of the patients enrolled in this clinical trial program.

The objectives of this Phase 3 events-driven trial are to evaluate the ability of MPC-150-IM to reduce the primary endpoint of recurrent non-fatal heart failure-related major adverse cardiac events (HF-MACE) in patients with left ventricular dysfunction, as well as delay or prevent disease progression to end-stage HF and terminal cardiac events, defined as death, left ventricular assist device (LVAD) implantation, or cardiac transplant. 

In April 2017, the trial achieved a successful pre-specified interim futility analysis of the efficacy endpoint in the first 270 patients and an independent data monitoring committee formally recommended the continuation of the trial.

Additional information about this trial is available at: and at

Phase 2 Trial

A Phase 2 dose-ranging study in patients with Class II/III (New York Heart Association) heart failure has been completed. Click here for clinical trial results.

End-Stage Heart Failure

MPC-150-IM is also under clinical investigation for the treatment of end-stage heart failure.

The definitive method of treating end-stage chronic heart failure is a heart transplant or implanting a mechanical assist device. Although there are many patients awaiting a transplant, due to limited supply, there were only 2,378 transplants performed in the United States in 20121.

Patients with end stage or Class IV heart failure disease represent a significant unmet medical need.

Clinical Trials

Ongoing Phase 2b Trial

Enrollment of a 159-patient Phase 2b trial in patients with end-stage heart failure being implanted with a left ventricular assist device (LVAD) has been completed. 

It is being conducted by a multi-center team of researchers within the United States National Institutes of Health (NIH)-funded Cardiothoracic Surgical Trials Network (CTSN), led by Icahn School of Medicine at Mount Sinai, New York. The National Institute of Neurological Disorders and Stroke, and the Canadian Institutes for Health Research are also supporting this trial.

The double-blind, placebo-controlled 2:1 randomized trial, is being conducted across multiple North American sites. The primary objectives of this trial are to evaluate the safety and efficacy of injecting up to 150 million MPCs into the native myocardium of LVAD recipients. The primary efficacy endpoint of the study is the number of temporary weans from LVAD tolerated over 12 months. Additionally, the study will evaluate patient survival and re-hospitalization over 12 months.

In December 2017, the United States Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for Mesoblast’s MPC therapy in the treatment of heart failure patients with left ventricular systolic dysfunction and LVADs. The RMAT designation under the 21st Century Cures Act aims to expedite the development of regenerative medicine therapies intended for the treatment of serious diseases and life-threatening conditions.

Phase 2a trial results

The results of the Phase 2a pilot trial were presented at the American Heart Association Scientific Sessions 2013 and published in the journal Circulation in June 2014. Click here more information on the trial results.


1Go AS, et al. Heart disease and stroke statistics—2014 update: a report from the American Heart Association. Circulation. 2014; 129:e28–e292.

2U.S. Department of Health and Human Services Organ Procurement & Transplantation Network data (accessed 10 Oct 2014)