MESOBLAST says the three-month interim results of the company’s Phase 2 clinical trial of its stem cell treatment for congestive heart failure show the product leads to significantly improved heart muscle function in patients.

HomeNews5/17/2009

Nick Evanswww.angioblast.com

The results, from the first 20 patients enrolled in the trial of Revascor, show moderate to severe congestive heart failure patients who received a single injection into damaged heart muscle showed significantly improved ejection fraction (the fraction of blood pumped out of a ventricle with each heart beat) results compared to patients treated with the current standard of care.

No treatment-related adverse events were noted in patients, according to a Mesoblast spokesperson.

The randomised, placebo-controlled Phase 2 trial is being run at multiple centres in the United States by Mesoblast’s United States-based associated company, Angioblast Systems.

The trial aims to compare one of three increasing doses of Revascor against standard of care in up to 60 patients suffering from moderate-severe congestive heart failure (defined as a baseline ejection fraction 40% or lower by echocardiogram).

Mesoblast said, in the first 20-patient cohort, 15 were randomised to receive the lowest dose of Revascor, with five randomised to the control arm.

While control patients with baseline EF 40 or below demonstrated an 11% mean decrease in EF over the first three months (mean EF values decreased from 31 to 27), patients with baseline EF 40 or below who received a single injection into damaged heart muscle of the lowest dose of Revascor showed a 37% average increase in EF over the period (mean EF increased from 28 to 37, p=0.017).

The 10-point mean difference in absolute EF at three months between cell-treated and control patients was also significant (p<0.05), according to Mesoblast.

Patients with the most severe and advanced heart failure, defined as a baseline EF<30 (9 patients treated with Revascor), had an even greater improvement in heart function, with mean increase in EF over three months of 50% (p=0.02).

The company said that, on the basis of the positive interim efficacy results and the excellent safety profile seen to date in the first cohort, the second group of patients set to receive the next higher dose is being actively recruited.

The company expects to be able to announced results from this group within six months, and may be able to move into a full Phase 3 trial of Revascor by the end of the year, if trial results continue to return these kind of results.

Mesoblast executive director Professor Silviu Itescu said he was encouraged by the results.

"We eagerly await the six-month results to see whether these effects are sustained or even further augmented," he said.

"If the clinical trial continues to parallel our pre-clinical results, we anticipate even better outcomes in the next group of patients receiving a higher dose of Revascor."

Director of Monash University Centre of Cardiovascular Research & Education in Therapeutics, Professor Henry Krum, who Mesoblast engaged to conduct an independent review of the trial data, described the initial results as "exciting".

"Equally as important is the lack of any safety concerns to date, meaning that for the first time we could potentially have a safe and effective off-the-shelf cell therapy product which could change the treatment paradigm for patients with chronic heart failure."

Revascor, Damaged heart muscle, Improved ejection fraction, Mesoblast, Mesoblast United States-based associated company, Angioblast Systems, Moderate-severe congestive heart failure, Echocardiogram, Lowest dose of Revascor, Professor Silviu Itescu, Monash University Centre of Cardiovascular Research and Education in Therapeutics, Professor Henry Krum, Chronic heart failure