At the halfway point, a phase 3 trial of Mesoblast's off-the-shelf cell therapy for chronic heart failure is on track and should continue to completion, say advisers.
Shares in the Australian biotech hit a 12-month high on the update from the trial, which came after it enrolled 270 out of a planned total of 600 patients with moderate CHF and—crucially—revealed no safety issues with Mesoblast's MPC-150-IM candidate.
The trial is still blinded so there is now way to tell if the therapy is having an effect, but it's a case of so far, so good, as the biotech looks ahead to completing the study next year.
The trial is seeing whether delivery of mesenchymal precursor cells (MPCs) via a catheter into the left ventricular heart muscle, to see if it can reduce non-fatal heart failure-related major adverse cardiac events (HF-MACE), such as death, needing a pacemaker implanted or undergoing a heart transplant.
Last year, Israeli drugmaker Teva returned rights to MPC-150-IM to Mesoblast, which it had inherited through its takeover of Cephalon. Mesoblast however decided to go it alone, a decision helped by a recent $40 million placement intended to help bring the CHF trial to fruition.
Analyst John Savin at Edison said in a recent note that the biotech may not have to wait for its own trial to complete before filing for U.S. approval. That could happen before the end of the year if a National Institutes of Health-sponsored trial of the therapy in end-stage heart failure patients hits the target.
The 159-patient NIH study is expected to complete enrolment in the first half and—with luck—could report top-line data before year-end, according to Savin. He reckons that could "lead to an application for accelerated approval" under the U.S. 21st Century Cures Act, which provides a speedy route to market for regenerative medicines.
"Passing this interim futility analysis for MPC-150-IM is an important milestone for Mesoblast and our cardiovascular disease program," commented CEO Silviu Itescu. "This validates our strategy and our prioritization of this valuable program." Analysts at Credit Suisse have previously suggested that that drug could be worth $4.1 billion in peak annual sales.
Mesoblast is not the only biotech looking at a stem cell approach to cardiovascular diseases. BioCardia is developing CardiAMP for CHF, reporting positive data from small phase 2 trial last year, while CardioCell presented mixed results on its candidate at the 2016 ESC Congress in Rome, and Celyad's C-Cure product failed to meet its objectives in its phase 3 CHART trial. Meanwhile, Belgium's TiGenix said recently its acute myocardial infarction therapy AlloCSC-01 hit its targets in a phase 1/2 trial.
The Australian biotech claimed an FDA green light to start trials of an MPC therapy given alongside corrective heart surgery for children with hypoplastic left heart syndrome (HLHS).
It's also in the build-up to a decision by option partner Mallinckrodt on its phase 3 MPC for chronic low back pain—due in or before September—and a graft-versus-host disease (GVHD) candidate that should see pivotal results before year-end.