A clinical trial showed Mesoblast, a global leader in cell-based medicines, and its proprietary mesenchymal precursor cell product candidate, MPC-300-IV was safe and results in early and sustained clinical responses for patients with biologic refractory rheumatoid arthritis.
MPC-300-IV is being evaluated in a double-blind, randomized, placebo-controlled, two-dose escalating trial in the United States. The trial is designed to evaluate safety and explore efficacy of MPC treatment in 48 patients with active RA randomized 2:1 to either placebo or a single intravenous infusion of 1 million or 2 million MPCs/kg. All recruited patients are on a stable regimen of methotrexate and have previously failed or had an adverse or inadequate clinical response to at least one biologic agent.
The lower dose was evaluated in the first cohort of 24 patients, of whom 16 had failed 1-2 biologics. The second cohort of 24 patients, evaluating the higher dose of MPCs, is actively recruiting. Trial results for both cohorts are expected to be reported in the third quarter of 2016.
Here are five points:
1. Cell infusions were well tolerated with no cell-related adverse events.
2. The trial's 12 week, pre-specified ACR20 efficacy endpoint was achieved by 47 percent of all MPC-treated patients and by 60 percent of MPC-treated patients who failed 1-2 biologics, compared to 25 percent and 17 percent, respectively, of matched placebo-treated controls.
3. Seventy-one percent of MPC-treated patients who achieved ACR20 responses did so as early as week one.
4. At week 12, 27 percent of MPC-treated patients, but no placebo-treated controls, achieved ACR50 or ACR70 responses.
5. Remission at week 12, as defined by Disease Activity Score <2.6, was seen in 20 percent of MPC-treated patients but in no controls.