Mestoblast’s Stem-cell Therapy for Heart Failure Reaches Phase III

The regenerative medicine company releases promising interim trial results for chronic heart failure therapy.

 The Australian company, Mesoblast, otherwise known as the “regenerative medicine company” is proud to report that it has not experienced any safety issues with their MPC-150-IM candidate and are looking to complete their study in full by next year.

There are currently 270 people who suffer from moderate chronic heart failure (CHF) enrolled, though the study is slated for recruitment of a total of 600 patients for further analysis and monitoring. Mesoblast has not released any information regarding the success of the trial as it is blinded, but the update that there are no safety issues with the candidate is serving as a green light and giving the company the feeling that the drug will successfully continue into the next year. The candidate has currently been in trial for approximately eighteen months now.

The trial involves the delivery of mesenchymal precursor cells (MPCs), which are sent directly into the left ventricular heart muscle. The trial then tests if this action helps to limit chronic heart failure issues or heart failure-related major adverse cardiac events (HF-MACE) as well as needing a pacemaker implantation, or even heart transplant and ultimately, including death.

Mesoblast CEO Silviu Itescu commented on the promising new candidate and his hopes for the future. “Passing this interim futility analysis for MPC-150-IM is an important milestone for Mesoblast and our cardiovascular disease program,” he said. “This validates our strategy and our prioritization of this valuable program,” Itescu added.

This has also helped propel the candidate to what the company hopes will lead to fast approval in the US. Although the company would need to wait for the end of the trail for approval and then add on another year in order for final result clearance, if a study sponsored by the National Institute of Health using the drug is both on track and meets its target in those afflicted by late stage heart failure, this approval could be even faster. The NIH is looking to complete enrollment in the 159-patient study by the first half of the year with the goal to have data to share by the end of the year. If this study is a success, the drug would likely move past the trial phase and onto approval, as it would fit within the 21st Century Cures niche for regenerative medicine.

Metroblast is currently exploring a similar drug option using MPCs for chronic lower back pain, alongside their option partner Mallinckrodt, which is also in Phase III and is likely to be released by September, if not prior to the third quarter.

The company is not the first to explore stem-cell therapy treatment options. BioCardia has developed the CardiAMP, also for chronic heart failure. Also, TiGenix has an acute myocardial infarction therapy AlloCSC-01 that has been reported as achieving their goals in both Phase I and II of trials.

 Metroblast is looking at promising profit margins if the drug is accepted, with analysts at Credit Suisse predicting that the MPC-150-IM candidate could be worth upwards of $4.1 billion annually. After separating from Teva, the company raised a total of $40 million in order to continue their clinical trial studies and push the potential stem-cell therapy forward.

There is a clear need for a drug of this sort, not to mention the optimism which comes with advancing cell therapies. As of last year, there were over 15 million patients suffering from chronic heart failure on a global scale, including those in the seven major markets of the US, Japan and EU 5 — all diagnosed with the aliment, according to PharmaPoint analysis. Fatality from chronic heart failure will affect approximately half of patients who will not survive past the fifth year being diagnosed with the disease. Chronic heart failure is characterized by weak heart muscles, which makes it difficult to pump blood throughout the body.

Significant Announcements and Forward Looking Dates

4/9/17     Successful Interim Analysis in Phase 3 Heart Failure Trial

4/5/17     Mesoblast Receives A$3.7m From Aus Govt For R&D

4/3/17     FDA Clears Heart Disease Trial At Boston Childrens Hospital

4/2/17     Change in substantial holding

3/31/17   Becoming a substantial holder from TEK


Nice Insight

Nice Insight, established in 2010, is the research division of That’s Nice, A Science Agency, providing data and analysis from proprietary annual surveys, custom primary qualitative and quantitative research as well as extensive secondary research. Current annual surveys include The Nice Insight Contract Development & Manufacturing (CDMO/CMO), Survey The Nice Insight Contract Research - Preclinical and Clinical (CRO) Survey, The Nice Insight Pharmaceutical Equipment Survey, and The Nice Insight Pharmaceutical Excipients Survey.