Patients with heart failure can reduce the risk of further occurrences by as much as 80 percent using an experimental stem-cell treatment, according to a study whose results are among the most promising for such therapies.
In research by Mesoblast Ltd., 45 patients who had moderate to severe congestive heart failure were given a shot of the Revascor treatment and standard medicines, while 15 didn’t receive the experimental drug. The therapy was safe and well-tolerated at all doses, according to results of the Phase II study presented yesterday at an American Heart Association meeting in Orlando, Florida.
Mesoblast said it expects to enter Phase III trials in the first half of next year. Revascor may begin making a profit for the Melbourne-based company by 2015, when the treatment is expected to go on sale, David Stanton and Zara Lyons, analysts at Nomura Holdings Inc., wrote in a Nov. 4 note.
The data was “outstanding,” Stuart Roberts, an analyst at Bell Potter Securities Ltd. in Sydney, said by telephone today. “This is probably the biggest thing in heart failure in 10 years.”
Roberts rates the stock “speculative buy” and has a 12-month price estimate of A$16.
The trial involved patients between the ages of 20 and 80 who were tracked for at least 12 months. Revascor, derived from mesenchymal stem cells taken from another person, cut the overall risk of a major adverse cardiac event by 78 percent.
Over an average followup period of 22 months, one patient in the first group who received Revascor died, while there were three fatalities in the control group.
Heart Pumping Ability
Mesoblast said patients who got the highest dose of Revascor didn’t show improved ejection fraction, a measure of the amount of blood pumped out of the heart each time it beats. Those given the lowest dose showed a significant improvement over three months in ejection fraction, with a “sustained but less pronounced effect” over 12 months.
Mesoblast fell 5.5 percent to A$7.75 at the 4:10 p.m. close of trading in Sydney. The S&P/ASX 200 index declined 0.4 percent.
“In any clinical trial, the full data needs to be examined,” Craig Collie, an analyst at Macquarie Group Ltd. in Sydney, said by telephone. “In this instance, the trial missed every pre-defined efficacy endpoint, except for major adverse cardiac event.”
In a note to clients today, Collie raised the possibility that Teva Pharmaceutical Industries Ltd., Mesoblast’s partner for developing the drug, and the U.S. Food and Drug Administration may not provide funding and clearance for a phase III trial to proceed.
The analyst has an “underperform” rating on Mesoblast, with a 12-month price estimate of A$5.80.
The phase II trial result reinforces “Teva’s commitment to its strategic investment in Mesoblast’s adult stem cell technology and to our continued support for the clinical development of Revascor,” Kevin Buchi, Teva’s corporate vice president of global branded products, said in a statement.
Mesoblast is also developing a stem-cell treatment for leukemia patients needing a bone marrow transplant that may go on sale next year, its first product on the market. The company is conducting the final stage of tests generally required for marketing approval in the U.S., the world’s biggest drug market.
Cephalon Inc., owned by Teva, bought 20 percent of Mesoblast for $220 million and marketing rights for experimental stem-cell therapies from the Australian company in December 2010 for as much as $2 billion in initial and milestone payments.