Key points:

• The biologic refractory rheumatoid arthritis (RA) population accounts for approximately one-third of all RA patients who have received anti-TNF or other biologic agents, is the fastest growing branded market segment, the hardest to treat, and requires new therapies that are both effective and safe
• Intravenous infusions of allogeneic Mesenchymal Precursor Cells (MPCs) were well tolerated in biologic refractory RA patients and were without serious adverse events over 12 weeks
• A single intravenous MPC infusion in biologic refractory RA patients resulted in dose-related improvements in clinical symptoms, function, and disease activity, with the 2 million MPCs/kg dose providing the greatest benefit
• Importantly, ACR70, the most meaningful measure of clinical improvement, was achieved by significantly more of the high dose MPC-treated than placebo-treated patients at 12 weeks
• In patients who had previously received 1-2 biologics, a single infusion of 2 million MPC/kg resulted in 55% and 36% ACR50 and ACR70 responses, respectively, compared with 11% and 0% of placebo treated patients, and in 91% of patients achieving the minimum clinically important improvement in physical function, defined as a reduction of at least -0.22 in the HAQ-DI, compared with 33% placebo treated patients
• The safety and efficacy results of this trial provide support for the potential of Mesoblast’s allogeneic MPCs to be positioned as first-line treatment option in RA patients who have previously received a prior anti-TNF or other biologic agent
• Given the large market opportunity, Mesoblast’s Tier 1 product candidate, MPC-300-IV,  is well-positioned to advance through a strategic partnership into Phase 3 development for biologic refractory rheumatoid arthritis

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