Mesoblast announces clinically meaningful outcome in NIH trial of MPC-150-IM
Mesoblast announced that results from a 159-patient randomized, sham-controlled Phase 2 trial in end-stage heart failure patients implanted with a left ventricular assist device showed that Mesoblast's allogeneic cell therapy candidate MPC-150-IM achieved significant reduction in major gastrointestinal bleeding episodes and related hospitalizations, a complication affecting up to 40% of LVAD recipients. This clinically meaningful outcome confirms results seen in an earlier 30-patient pilot trial which provided the basis for the Regenerative Medicine Advanced Therapy designation granted to Mesoblast by the United States Food and Drug Administration for MPC-150-IM as adjunctive therapy to LVAD implantation. Under the RMAT designation, Mesoblast received specific guidance from the FDA that reduction in major GI bleeding episodes and related hospitalizations in the current trial is a clinically meaningful outcome with a high unmet need that could meet requirements for an approvable regulatory endpoint. In contrast, the FDA advised that the primary endpoint in the current trial of temporary weaning from full LVAD support is considered a biomarker and is not a clinically meaningful outcome in and of itself. MPC-150-IM is also being evaluated by catheter-based delivery in patients with NYHA Class IIb-III CHF. This ongoing events-driven Phase 3 trial has enrolled approximately 85% of approximately 600 total patients.