Mesoblast (MSB) Emerging Growth Conference 79 summary

Company overview and technology

• Develops scalable, off-the-shelf mesenchymal stromal cell therapies for severe inflammatory diseases.

• Robust supply chain from manufacturing to distribution enables broad patient access.

Recent FDA approval and product details

• Received first FDA approval for allogeneic mesenchymal stromal cell therapy in the US for pediatric graft-versus-host disease (GVHD).

• RYONCIL, administered intravenously, showed a 70% responder rate at 28 days and 60% six-year survival in children.

• Product launch in the US is imminent, pending final FDA release; pricing to be announced soon.

Market opportunity and adoption

• US market includes about 10,000 bone marrow transplants annually; 50% develop GVHD, with 375 severe pediatric cases expected per year.

• Product already widely used under expanded access, considered standard of care in major US hospitals.

• Anticipates 30% market penetration in pediatrics, with plans to expand to adults and other inflammatory diseases.