Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-18

Not many boxes left to tick.

ACTIVITY REPORT Dec quarter

Graft versus Host Disease – Pediatric and Adult Phase 3 Programs

• Mesoblast has requested a meeting with FDA this quarter to provide additional potency and characterization data for its product RYONCIL which it believes demonstrate that the product used as second-line after corticosteroids in the pivotal Phase 3 trial GVHD001 in children with SR-aGVHD, which successfully met its primary endpoint of Day 28 Overall Response, was made to a standard supporting the trial as being adequate and well controlled.

• The new potency assay data show that the RYONCIL product made with the current manufacturing process that has undergone successful inspection by FDA, demonstrates greater potency than the earlier generation product, providing context to its greater impact on survival.

• Showing that the product used in the completed pediatric Phase 3 trial was standardized as to potency and characterization could provide support for approval of the pediatric indication given the absence of any approved therapies for children.